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AML is a rare aggressive cancer of the blood and bone marrow. It is the most common form of acute leukemia in adults with approximately 21,000 people diagnosed each year in the United States.

To recognize World AML Awareness Day, Celgene’s Kim Hodges sat down with Anna Ferguson, RN, BSN, an oncology research nurse at Johns Hopkins Cancer Center for a Facebook Live discussion about acute myeloid leukemia and how it impacts patients as well as doctors, nurses and caregivers. In our discussion, Anna highlighted the basics of the disease and what groups like the first global AML coalition called KNOW AML are doing to raise awareness.

Having started her career 25 years ago, Anna spoke about her personal journey to becoming an oncology nurse and working with patients in clinical trials. She spoke at length about studying the subject of hope over the past five years and the role that it plays in the live and treatments of patients with cancer. “The first person I took care of in the oncology unit had AML and I remember being so struck and moved by the intensity of the illness and the lack of treatments available,” Anna said, “Yet, I was also struck by the amount of hope and joy and perseverance in the patients. It was the first time that I saw that illness and hope could – and should – live side by side.”

Throughout her career, Anna has learned the true meaning of hope through patients’ inspiring perspectives and their stories about why hope matters and the hopes they keep that give them the strength to wake up and battle their disease each and every day. One of Anna’s patients described how she views hope, “I’m only human. Of course, I want to be cured. But the hope for a cure is not what drives me every day. It’s not what gets me out of bed in the morning. It’s not what lights my fire. It’s different, smaller hopes that keep me going every day. The hope of having enough energy to cook a meal for my family, hoping to make it to my daughter’s soccer game, hoping to avoid transfusions for just a week so I can go visit my sister. These are the things I hope for day to day that make the quality of my life better. These are the things I need my treatment team to know, to see if they can help me make them happen.’” For Anna, it is perspectives like this one that helped shape her nursing practice.

Yet, I was also struck by the amount of hope and joy and perseverance in the patients. It was the first time that I saw that illness and hope could – and should – live side by side.

To help support the educational needs of patients and their caregivers, Anna and her team at Johns Hopkins developed a set of important questions for patients to ask themselves in order to help them have an open dialogue with their oncologists. “There are so many things patients discuss at these visits, but in between those things, it is very important to discuss how AML and its treatments are affecting your day to day life and how you’re managing emotionally when it comes to your illness,” remarked Anna.

Join the full discussion on Facebook and share Anna’s message of why hope matters as we recognize AML patients around the world on World AML Awareness Day.

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Today, Celgene and Bristol-Myers Squibb stockholders voted to approve the combination of our two companies announced on January 3, 2019. The companies continue to expect to close the transaction in the third quarter of 2019, subject to customary closing conditions and regulatory approvals. You can read our press release here.

“Today’s stockholder vote represent a strong expression of confidence in the strategic merit of this combination and are a significant milestone towards close,” said Mark Alles, Celgene’s Chief Executive Officer. “Together, our two companies will have a greater impact on the patients who rely on our therapies, as well as create opportunities for our people and value for our stockholders over the long-term. We will have leading franchises and a deep and broad pipeline that will position the combined company to drive sustainable growth and deliver new options for patients with cancer, inflammatory and immunologic disease and cardiovascular disease.”

As we highlighted when the combination was first announced, the combined company will have a broad portfolio of leading in-line products, and an expanded pipeline of early stage programs and near-term product launches to build a sustainable platform for future growth.

We will have nine marketed products with more than $1 billion in annual sales, enabling us to create:

  • Leading oncology franchises in both solid tumors and hematologic malignancies led by OPDIVO and YERVOY as well as REVLIMID and POMALYST;
  • A top five immunology/inflammation franchise led by ORENCIA and OTEZLA; and
  • The #1 cardiovascular franchise led by ELIQUIS.

In addition to six expected near-term product launches (representing greater than $15 billion in revenue potential), the combined company will have a deep and diverse early and mid-stage pipeline across solid tumors and hematologic malignancies, immunology and inflammation, cardiovascular disease and fibrotic disease leveraging combined strengths in innovation.

The early-stage pipeline includes 50 high-potential programs, many with important data readouts in the near-term.

Together, we’ll also further our cutting-edge technologies and discovery platforms to sustain innovation leadership over time, including through the expansion of our capabilities in small molecule design, biologics/synthetic biologics, protein degradation, antibody engineering and cell therapy.

Consistent with Celgene’s long history of cultivating partnerships to benefit patients worldwide, the combined company will also have strong external partnerships with access to additional scientific platforms.

“Celgene has always been committed to changing the course of human health through bold pursuits in science with a promise to always put patients first,” Alles concluded. “We have accomplished so much already, and I look forward to the opportunities ahead as we join Bristol-Myers Squibb and further advance our mission for patients.”

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While most people living with follicular lymphoma—the most common subtype of slow-growing indolent non-Hodgkin’s lymphoma—respond to treatment, studies conclude that approximately 20 percent of patients relapse within two years of receiving first-line therapy. One area of research to understand this is the tumor microenvironment, which researchers have recognized as a significant driver of this blood cancer.

At the University of Texas MD Anderson Cancer Center in Houston, Dr. Loretta J. Nastoupil leads the Lymphoma Phase I Drug Development team, which is exploring the role of the tumor microenvironment in lymphoma. In this Q&A, she discusses how targeting the microenvironment may change how we treat follicular lymphoma and why treatment combinations may be helping to eliminate these tumor cells.

What is the tumor microenvironment?

DR. LORETTA J. NASTOUPIL

DR. LORETTA J. NASTOUPIL FROM THE UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER IS EXPLORING THE ROLE OF THE TUMOR MICROENVIRONMENT IN FOLLICULAR LYMPHOMA.

“Recently, researchers have been paying increased attention to the tumor microenvironment—the cells, molecules and pathways that surround and support the growth of tumor cells.

When B-cells, a type of white blood cell, become cancerous in follicular lymphoma, they have migrated from the bone marrow, where they are produced, to the lymph node. The microenvironment within the lymph node is essential in maintaining this disease. Complex interactions between cells in the nodes keep the cancerous B-cells growing.”

Why is targeting the microenvironment in follicular lymphoma worth studying?

“We tend to consider follicular lymphoma as more of a chronic disease than a fatal one. Patients generally do well with treatment, but we can’t cure it. Current therapies can put most people in remission, when there are no signs of cancer, but it usually comes back. If we can understand the tumor biology better, we hope to develop treatments that lead to a more durable remission.

We know that the microenvironment plays a critical role in maintaining this disease, helping it to thrive and to resist treatment. Right now, characterizing the microenvironment is our best predictor of how well someone with follicular lymphoma is going to do. If we can figure out better ways to target whatever structures support the lymphoma, instead of helping to stop DNA replication like some current therapies do, we might progress how we treat patients with follicular lymphoma.”

Why can’t we just target the genetic drivers of follicular lymphoma?

“With solid tumors, immune cells infiltrate in small numbers. If you can target or block that mutation, you can stop or slow that tumor from growing. But in follicular lymphoma, it’s not the same.

About 90 percent of patients with follicular lymphoma have an abnormal rearrangement between parts of two chromosomes, called a translocation. But you can’t target that abnormality for treatment. Acquiring the translocation is just the first in a multi-step process that leads to follicular lymphoma. The other steps lead to more genetic alterations, which provide some survival advantage to that cancer cell, including masking them from the immune system’s search-and-destroy process.”


Do any current therapies target the microenvironment?

“People with follicular lymphoma are often treated with anti-CD20 antibodies that interact with effector cells of the immune microenvironment. Essentially, you’re putting a flag on the follicular cancer cells, which express the CD20 proteins on their surface, and hope the other key immune system players see those flags and eliminate the cancer cells. Significant developments in treatment over the last two decades have led to an improvement in the relative five-year survival rate for patients with follicular lymphoma, which increased from 70 percent in 1990 to 89 percent in 2010.

Therapies that target the microenvironment offer a promising approach that has the potential to improve outcomes in follicular lymphoma.”

How important is combination therapy in helping the immune system to eliminate follicular lymphoma cells?

“With follicular lymphoma, when you block one pathway, usually cancer cells find another way to escape the immune system.

Now, it’s critical to be more rational about the combinations we’re studying. We could do a better job selecting combinations if we understood the key players in the microenvironment and their roles in maintaining the lymphoma.”

We want 100 percent of patients with follicular lymphoma to be looking at an average life expectancy, and we want to achieve that with as little impact on their quality of life as possible.

How important is targeting the microenvironment to the future of treating patients with follicular lymphoma?

“With follicular lymphoma, many patients do well with the current standard of care treatment options. So why do we focus so much on the 20 percent that relapse early, within the first couple years? Why do we talk about it so much at our meetings and in our research? It’s because we want 100 percent of patients with follicular lymphoma to be looking at an average life expectancy, and we want to achieve that with as little impact on their quality of life as possible. Targeting the tumor microenvironment is the next step forward towards accomplishing this goal.”

To learn more about novel combination approaches for some patients with lymphoma, read “The Goal to Treat Lymphoma without Chemotherapy.”

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Best Places to Work for LGBTQ EqualityCelgene is thrilled to announce that it has been recognized as a Best Place to Work for LGBTQ Equality with a stellar score of 100 percent for 2019.

The Corporate Equality Index (CEI) is a national benchmarking tool on corporate policies and practices relating to lesbian, gay, bisexual, transgender and queer employees (LGBTQ).  Administered by the Human Rights Campaign Foundation, an annual CEI survey is sent to hundreds of global companies to evaluate an array of LGBTQ-related policies and practices.

Best Place to Work 2019The CEI rating criteria have three key pillars:

  Non-discrimination policies across business entities;
  Equitable benefits for LGBTQ workers and their families;
  Supporting an inclusive culture and corporate social responsibility

Celgene’s focus on these important areas of workplace equality and inclusion resulted in a 100 percent ranking and the proud designation as a Best Place to Work for LGBTQ Equality.

Joe Hand, EVP, Global HR & Corporate Services says, “We are pleased that our commitment and efforts to ensure all Celgene colleagues feel welcomed were recognized with the designation as a Best Place to Work for LGBTQ Equality! Our inclusive benefits, workforce protections and the ongoing support and leadership of employee resource groups, like our Celgene Pride Alliance, all contributed to this perfect score on the Index.”

What Makes Celgene The Best Place To Work

A heavy emphasis on mentorship, volunteerism and employee inclusion groups has also been an area of focus across the organization.  Employee resource groups, including the Celgene Pride Alliance, offer opportunities for colleagues to connect and network. Supported by Executive Sponsors who are members of the Celgene Executive Committee, these resource groups host events and campaigns that bring together Celgene employees in an inclusive and safe environment to advance the dialog of social awareness and acceptance and drive necessary change.

Inclusivity and diversity in the workplace not only creates a more welcoming environment – it fosters new ideas, approaches and innovation across our entire business.

“The main goal for our growing list of employee resource groups is to promote employee development and learning, drive engagement and model inclusive behavior where all colleagues can be their authentic selves at work,” said Juli Blanche, VP, Talent Acquisition & Employee Experience. “Inclusivity and diversity in the workplace not only create a more welcoming environment – it fosters new ideas, approaches and innovation across our entire business.  I’m proud of everyone at Celgene for their commitment to recruit and retain the top industry talent inclusive of all sexual orientations and gender identities,” she added.

Celgene Pride AllianceIn recent years, employees also benefited from exciting enhancements to many U.S. benefits including important Work-Life benefits like increased paid parental leave for either parent, a “bridge back to work” policy that allows new parents to transition back to work on a part-time basis but with full pay, comprehensive and inclusive family building and fertility benefits, increased adoption support and assistance and a policy for flexible work arrangements, to name a few.

“Earning a spot on the Human Rights Campaign’s “Best Places to Work for LGBTQ Equality” list, with a 100% rating, is a great accomplishment! It is evidence of the pro-active collaborative efforts between the Celgene Executive Committee and the Celgene Pride Alliance. More importantly, it exemplifies the company’s indelible commitment to diversity and its actions to cultivate an inclusive work environment,” remarked Olivier J. Gouedard, Chair-Celgene Pride Alliance.

Looking Forward into the Future of Celgene

Celgene continues to strive to make the workplace a welcoming and inclusive environment for all colleagues.

“While we are excited with the ranking and designation as a Best Place to Work for LGBTQ Equality, we also recognize there is always more to be done to ensure parity and inclusion.  We look forward to continuing to move the needle while remaining focused on our collective goal to improve patient outcomes,” said Joe Hand.

To learn more about Celgene’s people and culture, click here.

Long Island isn’t the best place to train for climbing Iceland’s mountains and volcanoes. But it was here, in his hometown of Oceanside, NY, where Rich Appelbaum prepared for the Moving Mountains for Multiple Myeloma (MM4MM) team Fire & Ice trek across Iceland.* While five straight days of hiking for seven to eight hours a day would be tough for almost anyone, it posed an extra challenge to Appelbaum, who was diagnosed with multiple myeloma in 2016 at the age of 62. Two years later, he found himself on a life-changing excursion.

MM4MM organizes treks that are more than just a physical challenge for its teams, with members who are all directly affected by multiple myeloma as patients, family members, friends, clinicians or advocates. This collaboration between CURE Media Group and the Multiple Myeloma Research Foundation (MMRF) brings attention to the need for additional research and treatments for the more than 118,000 people in the U.S. living with this cancer and an estimated 32,000 others were diagnosed in 2018. Team members each raised at least $7,500 for the MMRF, and together the Iceland team raised more than $135,000.

Pushing past the diagnosis

Appelbaum’s 2016 diagnosis was unexpected, arising out of a routine blood test for a life insurance application. “I was honestly expecting to receive a preferred status,” he said. Instead, he was not eligible, based on extremely high levels of protein in his blood. After a biopsy and bone marrow test, Rich was perplexed by his diagnosis, because he had never heard of multiple myeloma.

Rich Appelbaum's Team

MULTIPLE MYELOMA DIDN’T STOP RICH APPELBAUM FROM KEEPING UP WITH HIS TEAMMATES. AS THE TOP FUNDRAISER OF THE GROUP, HE RAISED MORE THAN $20,000 FOR THE MULTIPLE MYELOMA RESEARCH FOUNDATION.

He started treatments in January 2017 but continued to encourage himself. “When you’re diagnosed with cancer, certain doubts creep into your mind as to what you’re going to be able to do,” he said. The idea of hiking volcanoes, glaciers, lava beds, gorges and up to waterfalls was a personal challenge that motivated him.

Surpassing expectations

Appelbaum was a novice hiker. “I’d never even carried a backpack before, and I was told I’d need to get used to carrying an extra 30 or 40 pounds on my back,” he said. he filled a backpack with weights, put on hiking boots and walked around Long Island with his wife. Eventually, he joined friends on day trips to nearby trails on the weekend. “My motto has always been, if it’s worth doing – it’s worth overdoing.”

Appelbaum encountered his first hurdle before the trek even began. Initially, the MMRF didn’t respond to his application. As the closing date approached, he sent them videos of himself at the gym with accompanying music from the film “Rocky.” Before long, the organizers offered him a spot on the trip. Good thing for the MMRF, too – Appelbaum not only completed and in some instances excelled on the hike but was the top team fundraiser, bringing in more than $20,000 for multiple myeloma research.

In the process of raising funds, he also raised awareness of multiple myeloma and the MMRF among the thousands of his followers who read his social media updates about the trek. “I made it clear that all the funds would go to the MMRF. That made it understandable for people to support me.”

My motto has always been, if it’s worth doing – it’s worth overdoing.

RICH APPELBAUM BONDS WITH HIS TEAMMATES DURING THEIR TREK ACROSS ICELAND.


RICH APPELBAUM, CENTER, IS MOST GRATEFUL FOR THE BONDS HE MADE WITH HIS TEAMMATES DURING THEIR TREK ACROSS ICELAND.”

 

Forging friendships on the trek

While completing the trek was a major personal accomplishment, Appelbaum’s most valuable takeaways were the friendships he forged with his teammates and the stories they shared while hiking through the craggy landscape. Even eight months later, the teammates are still in touch daily. “I’m inspired by all of them,” he said. “One of my teammates was told she had a 20 percent chance of living five years when she was diagnosed; she has already lived 10.”

Appelbaum is also grateful for the bonds he made with the health care professionals on his team. “They’ve been very supportive and I consider them good friends as well.”

Stepping out of his comfort zone

Appelbaum sees parallels between the excursion and his battle with multiple myeloma. “The trek definitely forced me out of my comfort zone, and I think dealing with cancer does the same,” he said. “You may no longer necessarily be able to do things on your own terms or your own schedule.”

The experience also made him appreciate recent treatment advances, including those supported by the MMRF, which has supported more than 350 research grants worldwide and has helped bring ten drugs to market since its inception. “I’m truly amazed by how often I read about advances in medication for multiple myeloma and ways to identify which medication is best for each patient,” Appelbaum said.

To learn more about the MMRF and how you can support multiple myeloma research, visit the MMRF website.

*The MM4MM Fire & Ice trek across Iceland was partially sponsored by Celgene Corporation.

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Over the past decade, researchers have begun to uncover the complex biology behind myelofibrosis. Nearly 5,000 people in the United States are estimated to be diagnosed with this blood cancer each year.

RUBEN MESA, M.D.

RUBEN MESA, M.D., FROM THE UT HEALTH SAN ANTONIO MD ANDERSON CANCER CENTER, BELIEVES ONGOING RESEARCH IN MYELOFIBROSIS OFFERS HOPE THAT THERE WILL BE MORE TREATMENT OPTIONS IN THE FUTURE.

“We do not yet fully understand the biology behind why myelofibrosis advances in patients,” explained Ruben Mesa, M.D., director of UT Health San Antonio MD Anderson Cancer Center. “Improving our understanding can help us advance treatment beyond the current approved and investigational therapies, which are still being examined to determine how they might slow progression of disease.”

What Is Myelofibrosis?

Myelofibrosis is a rare blood cancer that starts in the stem cells of the bone marrow, leading to the production of faulty blood cells that don’t mature or function properly. Eventually, the disorder results in scarring of the bone marrow, further stifling its ability to produce healthy blood cells.

While some patients may live years without symptoms, others see their disease progress rather quickly. Without enough healthy red blood cells, these patients can develop anemia, leading to fatigue, weakness and shortness of breath. Myelofibrosis can also cause an enlarged spleen and the disorder has a higher incidence in people over age 50.

In addition to monitoring patients closely for any signs of disease progression or other conditions, physicians face the challenge of tailoring treatment. “Myelofibrosis is a variable disease,” Mesa said. “A better understanding of the role of genetic mutations that cause the cancer and refining the prognostic scores could help doctors have a much more detailed assessment of prognosis and determine which treatment options are best for each individual patient.”

Understanding Options

While a stem cell transplant is potentially curative in some cases of myelofibrosis, doctors reserve it for patients fit enough to endure the procedure due to the risk of life-threatening complications. Eligibility depends on several factors, including prognosis, age, overall health and the availability of an appropriate donor.

“The average age of diagnosis for myelofibrosis is about 60 years old, which is toward the latter end of when transplantation is considered safe or effective,” Mesa said. “So it’s not an appropriate therapy for all patients.”

For many patients, the goal of treatment is focused on relieving symptoms, reducing an enlarged spleen and improving blood cell counts. Therapy options for those who are ineligible for a transplant include JAK inhibitors, chemotherapy, immunomodulators and corticosteroids. Not all of these agents target the signs and symptoms associated with myelofibrosis, and researchers are working to find additional options.

There are many reasons to be hopeful in myelofibrosis research – a better understanding of the disease, better understanding of its genetic drivers and better therapies.

Hope for the Future

As with many rare diseases, several unmet needs in myelofibrosis are attracting attention, according to Mesa. “Currently, there are very few targeted therapies approved for myelofibrosis, and we do not have effective therapies for patients with the most advanced forms of the disease.”

There is hope. At December’s American Society of Hematology (ASH) Annual Meeting, researchers presented data from more than 20 clinical trials in myelofibrosis. Treatment options being investigated include JAK inhibitors and combinations that incorporate other agents, like immunomodulatory therapies which adjust the body’s immune responses to disease. Researchers are also exploring treatment strategies that target multiple disease-signaling pathways, which are showing promise in early studies, according to Mesa.

These are positive developments for patients with a disease like myelofibrosis. More targeted therapies may one day allow doctors to tailor a patient’s treatment to their specific symptoms and unique genetic makeup of the disease.

“There are many reasons to be hopeful in myelofibrosis – a better understanding of the disease, better understanding of its genetic drivers and better therapies,” Mesa said.

To learn more about Celgene’s commitment to myelofibrosis and other rare diseases, read “Supporting Research to Find Cures for Rare Diseases.”

Since 2006, Medicare Part D has helped millions of American seniors gain access to their prescribed medications. In particular, the federal prescription medication coverage program has been remarkably effective for helping individuals with the blood cancer multiple myeloma in accessing their medicines. According to a new study published in the Journal of Clinical Oncology, not only has the program increased the use of newer or oral anticancer therapies among people with multiple myeloma, but may also be associated with helping those patients live longer.

For this year’s Multiple Myeloma Action Month, study author and Yale University professor Amy J. Davidoff, Ph.D., explains how Medicare Part D may have an impact on patient outcomes and what this means for the future of access to oral therapies for cancer.

AMY J. DAVIDOFF, PH.D.

IN A RECENT STUDY, AMY J. DAVIDOFF, PH.D., FROM YALE UNIVERSITY AND HER CO-AUTHORS FOUND THAT PATIENTS WITH MULTIPLE MYELOMA WHO HAD PRESCRIPTION MEDICATION COVERAGE HAD BETTER SURVIVAL OUTCOMES.

How does Part D impact what treatment a patient receives for multiple myeloma?

“It’s a complicated situation because there are the oral and IV (intravenous) options for multiple myeloma treatment. The out-of-pocket costs depend not only on the medication’s method of delivery but also on whether a patient has supplemental medical coverage (e.g. Medigap), prescription medication coverage or both.

These supplemental coverage programs are vital to protecting patients from large out-of-pocket burdens. In some cases, the types of coverage they have may lead them to choose one therapy over another or enable them to afford a combination of medications, which is common for multiple myeloma.”

Does Part D and other insurance plans impact survival for multiple myeloma?

“Our study revealed that multiple myeloma patients were more likely to receive any myeloma therapy, but less likely to received older IV therapies if they had prescription medication coverage compared to those without such coverage. We could not directly observe, but presume that they were receiving oral therapy. Patients covered by Part D or other creditable prescription medication coverage programs had median overall survivals that were 16 percent higher than the non-covered group.

Prescription drug coverage becomes an important part of the equation when patients have the option to take oral therapies, which is the case with multiple myeloma.

Why is prescription medication coverage particularly important in multiple myeloma?

“Prescription drug coverage becomes an important part of the equation when patients have the option to take oral therapies, which is the case with multiple myeloma. Patients with other cancers, such as diffuse large B cell lymphoma, are most often prescribed IV chemotherapy, which is administered in an outpatient setting and is covered under a patient’s medical benefits. As a result, our study found Medicare beneficiaries with this lymphoma saw no significant difference in survival with Part D coverage.

Oral therapies for multiple myeloma, however, were introduced in 2006, so our study was able to cover an adequate period of their use, from 2006 to 2013. During the last decade, oral therapies have been developed for other malignancies such as breast cancer and melanoma. As time progresses, we could see similar research on prescription coverage and patients with various cancers.”

Why don’t more seniors opt for prescription medication coverage?

“The few seniors who decline Part D coverage may perceive themselves as relatively healthy. They may assess the cost of Medicare Part D coverage compared to the cost of their current medications and decide that they don’t need to enroll. Health care professionals understand that your health status today does not necessarily determine your status tomorrow, but not all people understand the risk of future health problems and may not understand the potential risks of not enrolling.

Many of those seniors regret this decision later on. For example, 41 percent of non-covered patients enrolled in a prescription plan the year after being diagnosed with multiple myeloma. There is definitely an ongoing need to better inform the Medicare population of their coverage options during open enrollment.”

What are your thoughts on the implementation of Part D and its future?

“We need to change the way seniors enroll in Medicare programs. The process as it is now is confusing and far from ideal. There are a lot of opportunities to improve the enrollment site and provide beneficiaries with better information.

In addition, there’s a need to establish better caps on out-of-pocket costs. This would be very useful. The goal is to make prescription medication coverage as affordable as possible for Medicare beneficiaries.”

To learn why policymakers must act to protect prescription coverage for America’s seniors, read “Medicare Part D: Challenges Loom Ahead.”

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What do space, the ocean and the desert have in common with health care? That is the question posed by Healthcare’s Great Expedition, a unique interactive experience created by Celgene and focused on innovation in medicine.

The display made its debut at the 2018 World of Technology and Science (WoTS) congress in Utrecht, Netherlands, and Celgene plans to take this Expedition experience on the road, including to the 11th World Conference of Science Journalists, July 1-5, 2019, in Lausanne, Switzerland.

At WoTS, thousands of visitors travelled through the booth navigating three sections named “Space,” “Ocean” and “Desert” to reflect environments that hold potential for human discovery and offer an interesting analogy to some of the issues faced in health care. In these exhibit areas, participants learned about the various stages of medical discovery and development, while hearing Celgene’s perspectives on the complexities of the health care environment. Visitors shared their feedback and interacted with features, including a short science-fiction film and several games.

“Celgene is proud to be part of this great health care expedition,” said Kevin Loth, Celgene Vice President, Corporate Affairs and Policy, Worldwide Markets. “We appreciate the opportunity we have to engage with members of the public about what we are doing as a company and industry. Through interactions like this, we can help people understand the critical roles pharmaceutical companies play in bringing new medicines to patients.”

Communication and Collaboration

Visitors began their tour with Space, the area of the booth focused on communication and collaboration. There, they viewed This is Axiom, a story of an astronaut lost in space and fighting for a chance to return home. Her story and the stories of those who help her along the way serve as a metaphor for the complex health care environment and the urgent need for collaboration among health care stakeholders when making decisions about patient care.

The booth also allowed stakeholders to contribute ideas regarding sustainability in health care. According to at least one attendee, rising medical costs are not the fault of any one individual stakeholder.

“It is too easy to point at other stakeholders; that will not lead us to a solution,” Arnt Wolter, strategic health care contractor from the Diakonessenhuis Hospital in Utrecht, said. “Every stakeholder can take responsibility for a part of the solution.”

Through interactions like this, we can help people understand the critical roles pharmaceutical companies play in bringing new medicines to patients.

Discovery and Exploration

The second area, Ocean, focused on the high-risk nature of medical discovery and the vast areas of exploration still open. An interactive game gave visitors the opportunity to develop an investigational medicine from its discovery; through clinical trials, regulatory approval and manufacturing; and finally launch. This exercise highlighted the many difficulties in real-world medical discovery and development. This part of the exhibit provided greater detail about the complex discovery process and the high rates of failure in pharmaceutical development.

At the end of Ocean, visitors were asked what they thought was the greatest challenge to discovering new treatment options. Answers included difficult science, challenging government policies, technological challenges, high costs and high risks. Most participants chose difficult science, followed by high costs and high risks.

Access to Medicines

Finally, visitors arrived at Desert, where access to medicines was the theme. An interactive pricing simulation game allowed participants to step into the role of a pharmaceutical company leader. In this simulation, they evaluated decisions on pricing and future investments. When asked at the end of this area to identify the primary driver of the cost of medicines, most visitors chose research and development.

Next Steps for Healthcare’s Great Expedition

The display enabled valuable conversations from diverse perspectives regarding the many facets of health care, including investments, research and development, value and innovation and the future. Celgene looks forward to gathering further perspectives at additional events with the Expedition in the coming year.

In 2011, Mary Ellen Kelly was diagnosed with myelodysplastic syndromes (MDS), a disorder in which the bone marrow does not produce enough healthy blood cells. As a result, her red blood cells often do not mature and function properly, causing anemia. Other blood cells, such as white blood cells and platelets, may also be affected, but the most common sign in MDS is a shortage of red blood cells. In order to maintain her health, Kelly’s priorities have shifted to focus on managing her condition. A significant part of this management involves receiving frequent blood transfusions, which treat her MDS-associated anemia and help with her excessive fatigue – one of the most prevalent symptoms among patients with MDS.

Neil Horikoshi, CEO of the Aplastic Anemia & MDS International Foundation, advocates for patients like Kelly and is vocal about the struggles they face. “Everyone with MDS will experience some fatigue, even if they have improved blood counts after transfusions,” said Horikoshi. “It’s the nature of bone marrow failure disorders.”

Patients are feeling the exhaustion. According to a survey, one-third of patients describe transfusions as a burden to their family, and two-thirds would prefer a therapy that lessens their need for transfusions. For this year’s National Aplastic Anemia and MDS Week, Kelly provides a diary detailing five days during one of her transfusion weeks to raise awareness of how her chronic and rare disease affects her daily life.

Monday: Blood Work

For Kelly, each week begins with a 20-minute drive to her local hospital for a blood test to check her red blood cell count. “If it’s too low, the doctors and nurses schedule a transfusion for me within the next day or two,” Kelly said. “The lower my count, the more units of blood I’ll receive. Typically, it’s one. But if it’s extremely low, I get two.”

Each visit takes about three hours. By the time she gets home in the late afternoon, Kelly is ready for a nap. In fact, she typically naps for about an hour every afternoon due to the persistent fatigue associated with MDS. In one survey, 89 percent of patients with MDS reported experiencing excessive fatigue—a persistent sense of physical, emotional and cognitive exhaustion.


MARY ELLEN KELLY HAS BEEN LIVING WITH MDS-ASSOCIATED CHRONIC ANEMIA SINCE 2011 AND RELIES ON HELP FROM HER SISTER FOR DAILY TASKS WHEN SHE IS FATIGUED.

 

Tuesday: Yoga, Laundry and Shopping

In addition to naps, Kelly manages fatigue by attending yoga classes twice a week. This also helps her to manage her stress and to sleep better at night. “I find that it gives me more energy to get through the day,” Kelly said.

Energy is something that she tries to conserve as much as possible. She sets priorities, paces herself and asks her sister to help with chores, such as grocery shopping and carrying the laundry up from the basement.

“My pace of walking is slower than most people’s, and I have a hard time walking upstairs,” Kelly said. “I avoid really big stores because I can’t deal with all the walking. I can’t run around the store and get something quickly.”

Fatigue often affects other patients with MDS in a similar way. About 25 percent of patients report that it takes an effort to engage in normal activities, and 16 percent said they could not perform active work at all.

I devote a lot of time to my blood transfusions. It’s not what I want to be doing, but I’ve gotten used to it…

NEIL HORIKOSHI

NEIL HORIKOSHI CEO OF THE APLASTIC ANEMIA & MDS INTERNATIONAL FOUNDATION BELIEVES THAT BONE MARROW FAILURE DISORDERS HAVE A SIGNIFICANT IMPACT ON THE LIVES OF PATIENTS AS WELL AS THEIR CAREGIVERS.

Wednesday: Blood Transfusions

By Wednesday, Kelly is ready to go back to the hospital for a transfusion to boost her blood count levels. While transfusions do not treat the underlying disease, they do help to relieve the symptoms of her chronic anemia.

The transfusion process can also be lengthy and draining. “If I’m getting one unit of blood, it takes about a half day,” Kelly said. “If it’s two units, it could be seven to eight hours depending on the wait time.”

When she was still working as a paralegal, Kelly couldn’t afford to take time off for her transfusions. So she would bring her laptop and work from the transfusion center.

Now, three years into retirement, Kelly spends her time in the transfusion center napping, reading books or catching up on the news. On most of these days, she is alone.

“I devote a lot of time to my blood transfusions,” Kelly said. “It’s not what I want to be doing, but I’ve gotten used to it over the years.”

Thursday: A Free Day to Plan for Some Fun

What Kelly would prefer to be doing is traveling more. In June, she’s going to see her favorite singer, Barry Manilow, perform in Las Vegas.

“Sometimes, I take trips during the year,” Kelly said. “But I cannot be gone longer than a week at most, because I need my treatments. So, I could never go to someplace like Australia.”

The few times she did travel for longer stretches, Kelly felt like she pushed her limits. Earlier this year, she had to get a blood transfusion a couple of days after returning from a weeklong cruise because her blood levels were so low.

For the most part, though, Kelly spends her free time gardening in her backyard, meeting friends for lunch or dinner, or going to the theatre or movies. She’s recently seen—and highly recommends—A Star Is Born.

Friday: More Medical Appointments

Aside from the hours spent in the hospital for blood tests and transfusions, Kelly also has regular follow-up visits with her doctors, during which she gets a check-up and has the opportunity to discuss her symptoms and treatments.

Her doctors watch for warning signs that her MDS is progressing to acute myeloid leukemia (AML). Kelly counts herself as lucky to have low-risk MDS, with only a 20 percent chance of developing AML. Others with high-risk MDS have more than double the chance of progressing to AML.

With such a high risk of progression, attention to treating MDS and other bone marrow failure disorders is increasing, according to Horikoshi. For instance, over the past decade, Congress has invested more than $35 million in research for the prevention and treatment of bone marrow failure diseases through the Congressional Directed Medical Research Program.

For patients like Kelly, treatment advances cannot come soon enough. “I hope researchers find better treatments for MDS. But for now, and for as far as I can see, MDS is a significant part of my life,” she said.

To learn more about the progress in understanding MDS, read “Searching for New Ways to Help Red Blood Cells Mature in Myelodysplastic Syndromes.”

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In 2017, Wendell Ott was diagnosed at age 78 with myelodysplastic syndromes (MDS), which quickly progressed to a rare cancer called acute myeloid leukemia (AML). Less than 20,000 people are diagnosed with AML each year in the United States. Stem cell transplants are available as a potentially curative option, but unfortunately, Ott did not qualify for the risky procedure due to his age and overall health.

Over 500 Rare Disease Therapies Have Been Approved Since the Orphan Drug Act Was PassedWhen the U.S. Food and Drug Administration approved three new therapies for AML in 2018, Ott’s doctor recommended giving one a shot. Three months later, he was doing so well that he qualified for a stem cell transplant. After the procedure, his AML went into remission. “I have my life back, and I attribute much of the success I’ve had to the newly approved treatment,” Ott said.

Ott is just one of the more than 25 million people in the U.S. who are hoping to benefit from research being driven by the Orphan Drug Act, which is celebrating its 35th anniversary during this year’s Rare Disease Day.

Today, many people with rare diseases are devoting their time to advocating for the Act, as lawmakers are considering changing the legislation. The proposed changes could limit the development of rare disease therapies.

35 Years of Rare Success

PAUL MELMEYER

PAUL MELMEYER FROM THE NATIONAL ORGANIZATION FOR RARE DISEASES BELIEVES THAT THERE COULD BE DIRE CONSEQUENCES IF THE ORPHAN DRUG ACT IS NOT PROTECTED.

There are potentially 7,000 rare diseases that could affect more than 25 million people in the United States. Before the Orphan Drug Act was passed in 1983, only an estimated 34 treatments were approved to treat them.

Disappointed patients with rare diseases, in need of more treatment options, advocated for legislative changes to facilitate the development of new medicines. The Orphan Drug Act offered incentives to companies developing treatments for rare diseases, including grants for clinical trials, seven years of marketing exclusivity and a 50 percent tax credit on research and development costs.

With more than 500 therapies approved, rare diseases have become a popular area of research due to the Orphan Drug Act. In 2017 alone, there were 80 new orphan drug approvals for an array of diverse diseases, including lysosomal storage disorders, neuromuscular diseases and hemophilia A – rare diseases that previously had little or no treatment options. Following these accomplishments, another 57 therapies were approved within the first eight months of 2018.

“The Orphan Drug Act isn’t just working to incentivize the development in certain therapeutic pockets, but instead across the entire disease spectrum,” said Paul Melmeyer, director of Federal Policy at the National Organization for Rare Disorders. The Orphan Drug Act encouraged pharmaceutical companies to develop therapies with very small patient populations. In fact, about a quarter of approved orphan drug indications target populations wither fewer than 5,000 people.

That progress is only expected to continue, given that the FDA granted orphan drug status to more than 400 investigational therapies in 2017.

I’m hopeful that we have an opportunity to change lawmakers’ minds before it’s too late.

Changes and Misconceptions

Despite its success, the Orphan Drug Act was targeted by Congress in 2017, when the tax credit was cut in half. More changes in the Act’s incentives appear to be on the horizon.

WENDELL OTT

WENDELL OTT FEELS LUCKY THAT NEW TREATMENTS WERE AVAILABLE WHEN HE WAS DIAGNOSED WITH A RARE CANCER.

For patients like Ott, that prospect is baffling. “I’m hopeful that patients have an opportunity to change lawmakers’ minds before it’s too late,” he said. “Any efforts to limit or restrict the Orphan Drug Act do not seem well-considered.”

Ott’s suspicions may be well-founded, according to Melmeyer, who notes that the proposed changes are being driven by several misconceptions about rare disease therapies and the approval process.

One popular misconception is that treatments for rare diseases are driving up the cost of healthcare in the United States. Melmeyer countered that orphan therapies account for less than 10 percent of prescription medication spending.

Another misperception is that the incentives delay the development of generics and biosimilars. But in fact, a recent study found that the far more important factor for a lack of generic or biosimilar competition in the space is a potential low return on investment for generics manufacturers. Of note, generic or biosimilar competitors exist for just over half of the 217 orphan therapies that no longer have exclusivity.

Orphan Drug Exclusivity Does Not Delay the Development of Genetics“The orphan designation – and seven-year marketing exclusivity after approval – is focused on a single specific orphan disease and, in my opinion, doesn’t block generics and biosimilars from coming to the market to treat other diseases,” Melmeyer said. “In actuality, we don’t believe that the Orphan Drug Act is being abused; it’s working as intended.”

Dire Consequences

Cutting the tax credit is already forcing some companies to question the practicality of developing rare disease therapies, which will mean fewer treatments, according to Melmeyer.

“The vast majority of patients with a rare disease — 95 percent — are still waiting for that very first treatment indicated for them specifically,” Melmeyer said. “The consequences would be quite concerning, even dire, for many of those patients currently waiting.”

Melmeyer believes that we should be reaffirming support for the legislation and looking for new ways to drive the development of medicines to treat rare diseases. For patients with a rare disease like Ott, these incentives could mean the difference between life and death.

“When I hear about the prospect of limiting the Orphan Drug Act, it’s hard for me not to take it personally,” Ott said. “I don’t know where I’d be right now had the new drug not been approved, which made me eligible for the stem cell transplant. I’ve returned very close to normal, and I’m grateful for that.”

To learn more about the impact that rare diseases have on the lives of patients, read “Rare Disease Forces Abrupt Career Change” and “Rare Disease Day: What It’s Like Living with Behçet’s Disease.”

“Where are you?” Gina Leonardi recognized the voice on the other end of the phone; it was her boss. But, she had told him the day before where she would be.

As a toddler, Leonardi was diagnosed with beta-thalassemia, a rare blood disorder that reduces the level of oxygen-carrying hemoglobin in her blood, resulting in anemia — a lack of functioning red blood cells that causes fatigue and weakness. To help restore her red blood cells, for the last 48 years, she has received regular blood transfusions to help treat her anemia. That’s where she sat when she got the latest interruption from her boss. Within months, she would be out of her job.

Dealing with a rare disease like beta-thalassemia can make maintaining a job tough. In a recent survey, 38 percent of patients with rare diseases said they missed more than 30 days of work in the last 12 months because of their illness. In addition, in a survey of European employees, 41 percent of patients and caregivers said they needed special leave at work, but could not obtain it. For this year’s Rare Disease Day, which carries the theme of “bridging health and social care,” Leonardi explains how support from her family throughout her treatment has helped her deal with those who may be insensitive to her condition.

GINA LEONARDI LIVES WITH A RARE DISEASE CALLED BETA-THALASSEMIA AND FINDS SUPPORT FROM HER FAMILY.

How often do you receive transfusions to treat your anemia from beta-thalassemia?

“I get transfusions every three weeks. While I recognize these are vital to treat my anemia, it disrupts my routine because I’m in the hospital for a couple of hours, depending on how many units of blood I get. It takes up my entire day. I can’t do much while I’m there and don’t feel like doing anything after either. I’m just drained.

I used to drive from my house in New Jersey to a hospital in New York City. I would leave at 10 in the morning and not get home until 10 at night. Then four years ago, I had my daughter and I couldn’t spend that much time away from her, so I started going to a hospital about a half hour drive from where I live.”

What routines have your treatments disrupted?

“Beta-thalassemia has disrupted my career. I’ve been in the mortgage industry for years, and I was a loan officer assistant for a while. It’s a cut-throat business. I had informed my boss that I had a medical condition, and I told him ahead of time that I was going to be out every three weeks because I needed to get treatment.

Despite that, the demands of the job added even more stress to an already stressful situation.”

My job was very important to me, but my health has to come first.

How did that eventually turn out?

“My medical condition forced me to leave my job. I did not feel like my employer was supportive or that I could meet the time commitments required to perform my role.

My job was very important to me, but my health has to come first. So I left.”

PHILLIP CUTALO HAS ACCOMPANIED HIS DAUGHTER GINA LEONARDI TO HER BETA-THALASSEMIA TREATMENTS FOR DECADES.

Did you end up finding another job?

“Quitting was the best thing I could have done. Now, I’m a real estate closer, collecting and reviewing the documents when someone buys a home. It’s much more flexible than being a loan officer assistant for a mortgage brokerage, so if I need to miss a day of work, I can do so without repercussions.”

How important has support from family and friends been in this process?

“My parents have always been there for me. My mom took me into the city for my treatments when I was younger, and then my dad took over when she started working. We’d go to lunch together, and he’d turn on the TV in the hospital. It’d always be too loud, and I would have to yell at him to turn it down.

He still comes with me to this day. We have a very close relationship. He doesn’t leave my side.”

Do you have any tips for other people living with a rare disease?

“The first thing is to stay positive and keep up with your treatments and tests. Do what you have to do to live your life.

I would also like people to know that they are not alone. Some people are lost and feel like they don’t have anyone. I’m blessed to have my family and friends. I want people to know that it’s okay to talk with others. There’s so much support out there for people with rare diseases, and I hope that they are taking advantage of it.”

To learn more about this rare disease and others, read “Rare Disease Day: What It’s Like Living with Behçet’s Disease.” 

Like many people living with a rare disease, people with Behçet’s Disease often experience a delay in their diagnosis and have few treatment options for their recurrent physical symptoms such as oral ulcers, inflammation and skin lesions. This chronic, multi-system inflammatory disease affects just approximately 16,000 to 20,000 people in the United States, and no diagnostic test or cure has been developed yet.

But receiving an accurate and timely diagnosis is just the tip of the iceberg. These patients may also face emotional and social challenges associated with their rare chronic disease. Their time can be consumed by doctor’s appointments, treatment regimens and dealing with fatigue, according to patients.

As we recognize the theme of this year’s Rare Disease Day, “bridging health and social care,” two women—Ashley Pelletier and Rochell Magliocco—discuss how they cope with the emotional and social struggles of living with Behçet’s Disease.
 

Rochell Magliocco

BEHCET’S DISEASE HAS IMPACTED THE EMOTIONAL AND SOCIAL LIVES OF PATIENTS SUCH AS ROCHELL MAGLIOCCO.

When were you diagnosed with Behçet’s Disease, and what were some of your symptoms?

Ashley Pelletier: I started experiencing genital ulcers in 2016 when I was 19 and then developed oral ulcers and skin rashes. My OB-GYN thought it was herpes, but the test was negative. They didn’t know any other disease that caused such ulcers, so they treated me for herpes. I also had extreme fatigue, arthritis, migraines and some gastrointestinal issues, including nausea and vomiting. Eventually, a rheumatologist gave me a tentative diagnosis of Behçet’s, but he himself had never seen it before. In the next few years, doctors would sometimes diagnose me with something else, like fibromyalgia, or tell me that it was just in my head.

Rochell Magliocco: I was diagnosed in 2014 at age 34, but my first symptoms—ulcers in my mouth—went back to when I was a little girl. They progressed to genital ulcers, then nodules on my lower limbs, which is related to vasculitis (inflammation of the blood vessels). I have a history of blood clots in my lower legs. In 2015, I was diagnosed with meningitis, with brain and spinal cord swelling and lesions. I received treatment for this newly diagnosed infection while I was trying to control my Behçet’s symptoms. I was paralyzed from the waist down for three months. My left leg function returned, but not fully. I can’t bend my right hip or knee and I now walk with a cane.

I’ve lost friends because I’ve had to cancel plans last minute or leave early because I was sick.

How does Behçet’s affect your daily activities, including your work and social life?

Ashley Pelletier: Behçet’s is unpredictable, so it’s hard to plan ahead. I don’t know what symptoms I may face each day. I don’t have control. Even doing laundry, showering, getting dressed or making a meal can be challenging. It’s inconvenient for my family and friends. I let people down. I’ve lost friends because I’ve had to cancel plans last minute or leave early because I was sick. I used to be athletic, running races and kickboxing. I had to give up those activities. I loved soaking in the sun at the beach, but now I get severe skin lesions no matter how much sunblock I use.

Rochell Magliocco: I work part-time as an optician. I’m on my feet all day, and by the end of the day, I’m exhausted. We have a beach house. It’s hard to get out to the beach, but I still do it. I’m slower at doing things like walking on soccer fields for my son’s games. I try not to let it affect my life, but I am slower than I used to be.
 

Ashley Pelletier

ASHLEY PELLETIER SAW 30 TO 40 DOCTORS BEFORE BEING DIAGNOSED WITH BEHCET’S DISEASE.

How has your disease affected your family and friends?

Ashley Pelletier: I feel lonely, anxious, depressed and scared sometimes. But I have friends and family who don’t judge me and help me with medical appointments and at home. It’s comforting. My disease has shaped the way my family looks at chronic illness. My mom and I had to explain to my brothers why I was sick so often and had so many doctor’s appointments. It’s taught us that some people may be sick even if we can’t see it. Just because someone looks fine on the outside doesn’t mean they’re fine on the inside.
 

How important is communication between your doctors in your care?

Ashley Pelletier: I saw 30 to 40 doctors before a Behçet’s specialist diagnosed me two years after my first symptoms. I saw a vasculitis specialist, dermatologist, neurologist, gastroenterologist and others. I got bounced around. No one understood my case because Behçet’s is so rare. Doctors took me off treatments prescribed by other doctors without giving it time to see if it was helping. If they had worked together, we might have found an effective treatment earlier. Now I have a Behçet’s specialist who is finding doctors close to me, and they coordinate my care together.

Rochell Magliocco: My doctors are all in the same health system, so they can access all my medical records and lab results and view my other appointments. They’re great about coordinating care. I hadn’t seen a specialist until a year ago when a newly trained neurologist who treated Behçet’s joined a nearby medical system. He agreed with the treatment I had been receiving.
 

Do you have tips for people who have been newly diagnosed with a rare disease?

Ashley Pelletier: You should try to be really organized about coordinating your care and tracking all your medical documents. It can be really time-consuming. My mom and I spend more than half our time going to appointments, traveling for care or coordinating care. I invested in a big planner that keeps all my medical records together. I like to use my phone to organize reminders and sign up for patient portals.

Rochell Magliocco: Be your own advocate. Join groups on social media. I’ve met so many different people and learned from what they are doing to help manage their rare diseases.
 
To learn more about this rare, chronic inflammatory disease, read “Understanding the Common Symptoms of Behçet’s Disease.”

Over the past 25 years, significant innovation has advanced the treatment of multiple sclerosis  (MS), addressing the frequency of relapses for this chronic neurological disease. But on a daily basis, the 2.5 million people living with MS worldwide still face many challenges associated with their disease.

Hoping to help address these persistent challenges, Celgene has joined with the chronic disease-focused, patient-empowerment platform Lyfebulb to launch the Lyfebulb-Celgene 2019 “Addressing Unmet Needs in MS: An Innovation Challenge,” which will provide $25,000 to one patient entrepreneur who is developing solutions to address an unmet need in MS. The goal of the initiative is to seek new solutions, beyond therapy, to help address either challenges faced by people with MS in their daily lives or an unmet need that could potentially improve outcomes and experiences for both people with the disease and their support partners.

To introduce this innovation challenge, Lyfebulb and Celgene hosted an event on January 24 in New York. The event featured a diverse panel which included a neurologist, MS patient and patient advocacy leaders who gave their perspectives on the current challenges in MS care and the evolving needs of patients, caregivers and clinicians.

Supporting Patient Entrepreneurs

While most people may not be familiar with the concept of “patient entrepreneurs,” Lyfebulb is hoping that will soon change. When Lyfebulb was founded in 2014, its founders wanted to empower patients and those who support them to become active participants in the search for solutions to issues associated with chronic diseases like MS. Lyfebulb provides these patient entrepreneurs with opportunities to take part in summits and challenges – like the Lyfebulb-Celgene 2019 Addressing Unmet Needs in MS: An Innovation Challenge – to help themselves and others impacted by the disease.

These entrepreneurs not only have the drive to improve their lives and the lives of those who have experienced MS firsthand, but also have inside knowledge of the problems that patients with this chronic disease face. Or, as Elizabeth Jones, a patient and an advocate, put it, “No one understands what it’s like to have MS other than another person who has experience with it.”


KARIN HEHENBERGER, M.D., PH.D., MODERATED A PANEL FEATURING MS EXPERTS FROM ACROSS THE COUNTRY, INCLUDING A PERSON WITH MS, ADVOCATES AND A PHYSICIAN TO KICKOFF THE LYFEBULB-CELGENE 2019 “ADDRESSING UNMET NEEDS IN MS: AN INNOVATION CHALLENGE.”

 

Needs Left Unaddressed

While it is essential to continue studying treatment for MS, patients and their caregivers have other daily challenges that need to be addressed, as well.

“Addressing physical activity and diet and having conversations around holistic care with clinicians—that wasn’t the case 10 years ago,” explained Tim Coetzee, Ph.D., chief advocacy, services and research officer at National Multiple Sclerosis Society and a panelist at the recent discussion. “We want to approach innovation holistically by looking for tools that are useful not just to those who want small bites, but also to those who want it all.”

For instance, 28 percent of people with MS surveyed in one study said that psychological support was their most significant unmet need. “I lived in denial for years—I didn’t tell my friends or coworkers that I had a chronic illness,” Elizabeth Jones said during the panel. “I really needed to connect with people.”

Like people with MS, caregivers also experience high levels of stress and are often referred to as the “hidden patients” of the disease. But now, we’re seeing increased awareness of the burden of the disease on caregivers, according to Amanda Montague, Ed.M., vice president of Education and Healthcare Relations at the Multiple Sclerosis Association of America.

“We’re seeing a focus on the holistic experience of MS and bringing care partners into conversations,” she said during the panel discussion. “I’m excited to see innovations that incorporate care partners in both clinical and home settings.”

No one understands what it’s like to have MS
other than another person who has it.

Overcoming Information Overload

During the discussion, panelist Darin T. Okuda, M.D., M.S., director of the Neuroinnovation program and director of the Multiple Sclerosis & Neuroimmunology Imaging program at the University of Texas Southwestern Medical Center in Dallas, explained that because every individual with MS has a different experience of the disease, there are many opportunities for innovation to improve the lives of patients.

As an example, Okuda explained that he realized patients were coming to their appointments with him often feeling overwhelmed by the amount of information available on the Internet about their disease and confused by which resources to trust.

So Okuda and his team developed an app, called Pre-Meet: Multiple Sclerosis, to help patients prepare and focus on what is essential before appointments with their MS specialist to make the most of those visits. The app lets them know what to expect during their exam and understand the next steps following the appointment.


DARIN T. OKUDA, M.D., M.S. FROM THE UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER IN DALLAS AND HIS TEAM DEVELOPED AN APP, CALLED PRE-MEET: MULTIPLE SCLEROSIS, TO HELP PATIENTS PREPARE FOR THEIR APPOINTMENTS WITH THEIR MS SPECIALIST.

 

“Responsible and reliable content is key,” Okuda said. “When a patient has way too much information, we often find that they define themselves by their disease, which we don’t want them to do.”

While all the panelists at the event brought their own unique perspectives and priorities to the discussion, they all agreed that the MS space is ripe for innovation and that those directly impacted by the disease – patients, caregivers and experts in the field – can lead the charge.

To learn more about the Lyfebulb-Celgene 2019 “Addressing Unmet Needs in MS: An Innovation Challenge” or to apply for the Challenge, visit the Lyfebulb web page.

When Dr. John Marshall first started treating patients with pancreatic cancer nearly 30 years ago, the goal of treatment was to extend a patient’s life; the quality of the patient’s life was a secondary consideration. Despite some gains in survival for pancreatic cancer, the survival rate is still in the single digits; currently 8.5 percent of patients are alive five years after diagnosis. While research to improve treatment options continues, quality of life has become increasingly important.

“Patients want to be tough and compliant with their treatment because they want their cancer to go away,” said Marshall, Director, The Ruesch Center for the Cure of Gastrointestinal Cancers, at Lombardi Comprehensive Cancer Center, Georgetown University Medical Center. “But if their treatments are preventing them from enjoying life, they may need to reconsider their options.”

DR. JOHN MARSHALL

DR. JOHN MARSHALL, DIRECTOR OF THE RUESCH CENTER FOR THE CURE OF GI CANCERS, HAS SEEN QUALITY OF LIFE BECOME INCREASINGLY MORE IMPORTANT IN MAKING PANCREATIC CANCER TREATMENT DECISIONS.

The management of cancer is a continuum, and priorities can change between a focus mostly on treatment effectiveness to a greater emphasis on quality of life, according to Marshall. “Doctors aren’t good at documenting quality of life,” Marshall said. “We need to be better at focusing on the specific aspects that are most relevant to patients with pancreatic cancer.”

Doctors struggle for a couple of reasons. First, while survival can be objectively measured in months and years, quality of life is determined by subjective perceptions of physical, emotional, social and cognitive aspects of a patient’s life. Many doctors do not collect this sort of data because it is difficult to measure, especially in a busy medical practice.

Additionally, according to Dr. Marshall, each patient has a unique set of priorities. Some patients want to live longer, no matter what it takes; others may prioritize the quality of the remaining time they have.

Multiple factors affect quality of life, including symptoms and comorbidities associated with the disease. Sometimes it can be difficult to tell the difference, according to Dr. Michael Pishvaian, an assistant professor in the hematology/oncology division at MedStar Georgetown University Hospital and Lombardi Comprehensive Cancer Center.

“Pancreatic cancer is a miserable disease that causes a tremendous number of symptoms,”  Pishvaian said. Symptoms include loss of appetite, weight loss, back or belly pain, nausea, vomiting, diabetes and more.

Many patients expect pain or discomfort when being treated for cancer and sometimes suffer in silence. But when doctors know about their patients’ issues, they can often provide solutions. Doctors should be diligent about asking the right questions and encouraging their patients to respond honestly, reporting how they feel on both good and bad days throughout their treatment, according to Pishvaian.

Experts suggest there are a few ways patients and physicians can ensure that quality of life will be a consideration during treatment. First, communication is key to maintaining quality of life.

“As patients go through their diagnosis and treatment for pancreatic cancer, things continually change, making the need for information that much greater,” said Julie Fleshman, JD, MBA, president and chief executive officer of the Pancreatic Cancer Action Network. “Patients need to not only communicate their needs with the people who are supporting them, but also advocate for themselves by asking for the latest information on treatment options, clinical trials and support resources.”

Secondly, patients who feel that their doctors respect them and are treating them as “whole people” report a higher quality of life. Finally, doctors do not need to care for their patients alone; they can help their patients assemble a team that includes a nutritionist, a psychiatrist and others to provide comprehensive support for their patients.

Given the extent to which pancreatic cancer affects quality of life for patients, doctors need to be proactive in managing their disease, according to Marshall. “That might mean seeing them more often than you would see patients with other cancers. It might mean seeing them every other week or more, depending on how that patient is responding to treatment.”

While about 10,590 children in the United States are diagnosed with cancer each year, more than 80 percent of them survive 5 years or more. But in sub-Saharan Africa, the statistic is vastly different – 90 percent of the 100,000 children diagnosed with cancer annually die from their disease.

At Celgene, we believe that cancer treatment should be available to patients no matter who they are or where they live. That is why Celgene recently supported expanded care capacity in resource-constrained countries through  $1 million in grants as part of the Celgene Cancer Care Links™ program.

In recognition of World Cancer Day 2019, Dr. David Poplack, M.D., director of the Global HOPE program at Texas Children’s Hospital’s Cancer and Hematology Centers and Baylor College of Medicine, explains how two grants received by Baylor College of Medicine and Texas Children’s Hospital from the program are being used to improve cancer care in sub-Saharan Africa.

This year marks the launch of the “I Am and I Will” World Cancer Day campaign, which encourages people to make a personal commitment to reduce the impact of cancer. So let’s start there. Can you describe what you’re doing to help fight cancer?

“Our goal at Global HOPE is to improve the survival rate for children with cancer in sub-Saharan Africa by increasing their capacity in this underserved region. To do so, we are establishing centers of excellence in six countries where we train pediatric doctors and nurses to become experts in diagnosing and treating children with cancer and blood diseases.”

What challenges are clinicians facing in sub-Saharan Africa?

“In my frequent travels to the region, there’s a noticeable lack of understanding of cancer. Many children lose their lives without ever being diagnosed. In some regions, they don’t even have a name for cancer.

A major reason so many children go undiagnosed is because of the inadequate health care infrastructure in most of these countries. For example, in most African countries, there are very few pediatric oncologists, the experts in the diagnosis and treatment of childhood cancer. In addition, many of the needed effective cancer therapies are simply not readily available.

We need to better educate the public to raise the awareness of cancer and train pediatricians to become specialists in how to properly diagnose and treat children with cancer. There’s also a tremendous need for adequate resources, like well-equipped medical centers that have the expertise, infrastructure and therapies to treat childhood cancer.”

PATRICK STONE

DAVID POPLACK, MEDICAL DIRECTOR OF TEXAS CHILDREN’S HOSPITAL’S CANCER AND HEMATOLOGY CENTERS HUGS A CANCER SURVIVOR AT THE GROUNDBREAKING FOR A CHILDREN’S CANCER AND HEMATOLOGY CENTER OF EXCELLENCE IN BOTSWANA IN 2017. SOURCE: BAYLOR COLLEGE OF MEDICINE/SMILEY POOL

Why is cancer becoming an important health care priority in sub-Saharan Africa today?

“In recent years, we have developed a better understanding of the burden of non-communicable diseases in low-resource countries. Of the 56.9 million global deaths in 2016, 71 percent were due to non-communicable diseases.

As a result, people have become more aware that cancer is a significant killer in sub-Saharan Africa – a region with some of the most resource-scarce countries. It’s fair to say that we are behind in improving cancer care in these countries. So, support for the Global HOPE program is critical to get closer to where we need to be.”

One of the grants you received focuses on pediatric Kaposi sarcoma in Malawi. What are the challenges that this program seeks to address?

“Kaposi sarcoma is a malignancy that affects both adults and children and is epidemic in sub-Saharan Africa, with especially high incidence in people with HIV. Our project seeks to better understand this disease and to develop an effective treatment.

Specifically, we are developing an approach known as risk-based treatment. For low-risk patients, the goal is to treat them with effective medication that has a lower toxicity. For those with high-risk disease, we must give them a more intensive treatment.”

We cannot achieve a world free from cancer unless we fund innovative approaches to address the problem of the disease in under-resourced countries.

The other grant is focused on pediatric Burkitt lymphoma in sub-Saharan Africa. How does the program propose to address this problem?

“Burkitt lymphoma is associated with Epstein–Barr virus and the endemic type originates in East Africa and the sub-Saharan countries that fall into the so-called ‘malaria belt.’ It presents frequently in children, and is universally fatal if not diagnosed and treated quickly.

A variety of therapies have been used to treat Burkitt lymphoma over the years, and our program is aimed at developing a treatment approach based on disease stage and risk factors. This approach requires making sure we can accurately diagnose the disease, using sophisticated forms of clinical diagnosis, including approaches such as flow cytometry, immunohistochemistry and biopsy.”

How did the Texas Children’s Hospital’s Cancer and Hematology Centers get involved in these global health programs?

“The Texas Children’s Hospital’s Cancer and Hematology Centers has the largest, and one of the most effective, training programs for pediatric oncologists in the United States. So, we have looked at where else in the world we could bring our expertise to impact childhood cancer.

In 1999, Baylor International Pediatric AIDS Initiative and Texas Children’s Hospital, supported by the Bristol-Myers Squibb Foundation’s (BMSF) Secure the Future Program, developed a program and built centers of excellence in African countries to help deal with the problem of pediatric HIV/AIDS. That program, directed by Dr. Mark Kline, is highly successful and treats more than 300,000 children in over a dozen countries. The Global HOPE program for managing childhood cancer developed from additional BMSF funding and leveraged the infrastructure we built for the original.”

What makes the funding that Global HOPE has received from Celgene so unique?

“There are many grant opportunities out there to help improve research for cancers that affect children in the United States, but few speak to the tremendous burden of pediatric cancer in under-resourced countries such as those in sub-Saharan Africa. We cannot achieve a world free from cancer unless we fund innovative approaches to address the problem of the disease in under-resourced countries. With this additional, extraordinary support from Celgene, we will specifically be able to study the biology and improve the care of two cancers that often strike children in sub-Saharan Africa.”

To learn more about Celgene Cancer Care Links™ program, read “Improving Cancer Care in Resource-Constrained Countries.”

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More people are being diagnosed with—and dying from—pancreatic cancer, due to an aging population and rising rates of risk factors such as obesity. As a result, costs for treating this disease are on the rise.

Incremental improvements in treatment have been made, but progress remains slow, so pancreatic cancer still carries a poor prognosis. Dr. Hani Babiker, a pancreatic cancer specialist at The University of Arizona Cancer Center, offers his thoughts on the meaning of value in pancreatic cancer care – for which survival rates are low – and how the value of care could be improved.

How do you define value in cancer care?

“I define value as pushing ourselves to deliver more comprehensive approaches to providing care for our patients. That means addressing all patient issues and concerns related to their cancer.

With my patients, I discuss not only treatment options and goals but also what foods, vitamins and supplements can help them manage their disease. I refer my patients to social workers who can help them deal with the stress that can accompany a cancer diagnosis. A palliative care physician in our clinic helps patients to control symptoms such as pain, nausea and vomiting. We focus on treating the whole patient. That’s how I seek to provide the best value.”

DR. HANI BABIKER

DR. HANI BABIKER, A PANCREATIC CANCER SPECIALIST AT THE UNIVERSITY OF ARIZONA CANCER CENTER, BELIEVES DOCTORS AND PATIENTS SHOULD FOCUS ON THE OVERALL VALUE THAT TREATMENTS PROVIDE.

Does the definition of value change for cancers with low survival rates and few treatment options, such as pancreatic cancer?

“Absolutely. It is difficult to compare the value of treatments across all cancer patients; the options and goals are not the same.

We should not deprive pancreatic cancer patients of the most effective treatment options for their particular disease because their prognosis is not as good as patients with other cancers. But when the quantity of life we can offer patients is short, quality of life becomes an increasingly important factor to the value we provide.

Most people with pancreatic cancer experience pain, and I’ve seen firsthand how chemotherapy has helped patients manage that pain.”

Why has progress in pancreatic cancer lagged behind other cancers?

“It is an inherently unique disease. The microenvironment surrounding pancreatic cancer tumors creates a barrier that is difficult for therapies to penetrate. It also suppresses the body’s immune cells that would typically hunt down and eliminate cancer cells. We still have a ways to go in understanding and treating this disease better.”

If we can identify pancreatic cancer earlier, by screening people who are at high risk because of their family or medical history or genetic predisposition, we can potentially treat more patients using the Whipple procedure. We may provide better outcomes and, therefore, may deliver better value, even though hospitalizations and surgeries are expensive.

So how can we continue to improve the value of pancreatic cancer care?

“Pancreatic cancer is tough to diagnose. We usually see patients who are in advanced stages, when the disease has spread beyond the pancreas. As a result, only 20 percent of patients diagnosed are eligible for a surgery known as the Whipple procedure, in which doctors remove the cancerous part of the pancreas. Surgery gives appropriate patients the best chance for a cure.

If we can identify pancreatic cancer earlier, by screening people who are at high risk because of their family or medical history or genetic predisposition, we can potentially treat more patients using the Whipple procedure. We may provide better outcomes and, therefore, may deliver better value, even though hospitalizations and surgeries are expensive.”

What role do innovative therapies play in reducing hospitalizations and improving outcomes?

“Surgery gives patients the best chance for a cure, but less than 20 percent of patients live at least five years after their operation. This statistic highlights the fact that most pancreatic cancers have spread and cannot be cured through surgery alone.

Doctors are now using chemotherapy before and after surgery to improve outcomes in pancreatic cancer. And we are seeing more therapies being developed that will continue to improve survival and, one day, reduce the need for costly hospitalizations and surgeries. In the future, the best way to add value for pancreatic cancer patients will be to invest in better screening and to continue funding research into more treatment options.”

Learn more about how screening in individuals at risk for pancreatic cancer can help to save lives: read “Family History Helped This Survivor Catch Pancreatic Cancer Early.”

Millions of Americans who rely on Medicare Part D may be facing sticker shock in 2020 if solutions that address out-of-pocket costs and access are not enacted quickly, based on a report from the Lupus Foundation of America.

Medicare Part D provides prescription drug coverage to more than 44 million Medicare recipients. While prescription drug coverage under part D hasn’t been around for as long as people may think, its popularity stems from its far reaching importance to the overall health of its millions of beneficiaries.

“Medicare did not offer a prescription drug benefit until Part D was enacted in 2003,” said Patrick Wildman, vice president of Advocacy & Government Relations for the Lupus Foundation of America. “Since then, it has greatly improved the health of so many people.” Beneficiaries include lupus patients, who incur an average of $12,643 per year in direct health care costs.

While Part D has been a big step, Wildman believes policymakers need to address looming challenges, including the impending “out-of-pocket” cliff, specialty tiers and more. The right policies will protect prescription medication coverage for America’s seniors, but Wildman notes we need the political will to make them happen.

The “Out-of-Pocket” Cliff

In 2005, the Lupus Foundation of America established the Medicare Access for Patients Rx (MAPRx) coalition — a group of more than 55 patient advocacy organizations representing seniors who depend on Medicare Part D for their prescription medications. The coalition recently published a report highlighting some of the challenges faced by Part D beneficiaries.

The most pressing issue, according to the report, is a steep increase in the “out-of-pocket” threshold, the amount patients pay before entering catastrophic coverage where their payment responsibility for their medicines drops to five percent for the remainder of the plan year. The Affordable Care Act capped how much that limit could increase each year. But the limit on growth of the threshold is set to expire in 2020, adding $1,250 to threshold in just one year, unless Congress acts.

“It is a looming crisis,” Wildman said. “As a coalition, we are worried that with that big of a spike, people may have challenges accessing medication they need.”

Infographic of Medicare Part D, Out of Pocket Spending

Specialty Tiers on the Rise

Part D plans also have been increasingly placing more medications on specialty tiers, which force seniors to pay up to a third of the costs for certain particularly expensive treatments. A study found that Part D beneficiaries pay an average of $3,949 annually out of pocket for specialty treatments for rheumatoid arthritis, $5,238 for multiple sclerosis and $6,322 for chronic myeloid leukemia. These chronic illnesses require several treatments that are frequently listed on specialty tiers.

Part D beneficiaries have no recourse to request exceptions to specialty tier pricing, either.9 The MAPRx coalition is fighting to change that.

“I think we need to fundamentally address the role of specialty tiers in Part D,” Wildman said. “Placing a medication on a specialty tier may stop some patients from filling their prescriptions and getting the treatment that they need.”

There is a lot of work still to be done to improve Medicare Part D for our seniors.

Improving Part D with an Out-of-Pocket Cap

While Medicare Part B beneficiaries can enroll in additional coverage that limits their annual out-of-pocket spending, most Part D beneficiaries must pay 5 percent of their medication costs after they hit the catastrophic phase without limit. Depending on what medications they have been prescribed, this can amount to thousands of dollars each month. But by putting an out-of-pocket cap in place, however, policymakers could help Part D beneficiaries better manage the financial burden of their conditions and illnesses, according to Wildman.

Strengthening and protecting access to medications is critical for millions of seniors with chronic diseases. The first step in this process, according to Wildman, is to convince policymakers how important the benefit is for seniors and people with disabilities, as well as others who depend on it. The coalition also wants to ensure that existing patient protections within the program stay in place and that recently introduced pricing policies do not restrict access.

“There is a lot of work still to be done to improve Medicare Part D for our seniors,” Wildman said. “We are looking forward to strengthening the program in the years to come.”

To learn more about Medicare Part D, read “Medicare Part D: 10 Years of Successfully Meeting Seniors’ Needs

Approximately 3.5 million women in the U.S. are living with breast cancer, including more than 154,000 with disease that has spread beyond the breast to other parts of the body, known as metastatic breast cancer (stage IV). The outlook for non-metastatic breast cancer patients has overall improved, with an average five-year survival rate reaching close to 100 percent for people with stage 0 or I breast cancer, and 93 percent for people with stage II breast cancer.

The prognosis for those women diagnosed with metastatic breast cancer is not as promising. But, as research continues, progress is emerging. The percentage of women surviving five years with metastatic breast cancer (aged 15-49) doubled from 18 to 36 percent between 1994 and 2012.

“Sometimes metastatic breast cancer can be considered much more of a chronic disease,” said Denise A. Yardley, M.D., a senior investigator at the Sarah Cannon Research Institute in Nashville, TN. “I’ve seen a positive impact on patients who continue relatively normal lives despite their disease and treatment.”

DENISE A. YARDLEY, M.D.

DENISE A. YARDLEY, M.D., FROM THE SARAH CANNON RESEARCH INSTITUTE BELIEVES WE ARE SEEING PROGRESS IN THE TREATMENT AND PROGNOSIS OF METASTATIC BREAST CANCER.

Treatment Advances Are Constantly Occuring

There are many forms of metastatic breast cancer. Patients’ tumors can be either positive or negative for growth receptors, signaling the presence or absence of the known drivers of the disease. And they may or may not have disease driven by HER2 (human epidermal growth factor receptor 2) receptors. That complexity and the cross signaling from the HER2 receptor to other growth factor receptors, as well as the multitude of treatments available to treat metastatic breast cancer are part of the reason it has been a particularly difficult disease to treat. But translational researchers are making significant strides to further understand tumor biology and the genomics behind breast cancer subtypes.

The end result is a more tailored treatment approach based on a patient’s specific tumor biology and other clinical factors. With an expansion in targeted therapies, there is greater value in having patients’ tumors thoroughly examined so treatments are better selected. “We’re continuing to try to improve our precision medicine and really tailor treatments to what’s going on in that specific patient’s tumor,” Yardley said.

Doctors also have a better understanding of how to use the growing array of treatments. While combination therapy is used in early stages of breast cancer, recent studies have provided additional evidence on how to  sequence treatment options for their patients. We now also focus  on balancing symptom control with quality of life and partnering with our patients to make appropriate treatment selections at any given time.”

There’s every reason to be optimistic for patients facing the diagnosis and challenges of metastatic breast cancer today.

Preventing Metastasis from the Start

About 30 percent of women with early stage breast cancer eventually develop metastatic disease. So in addition to improving the treatment of metastatic breast cancer, researchers are trying to continue to improve the cure rate and thus prevent breast cancer from becoming metastatic in the first place.

Metastatic Breast Cancer TreatmentMost women with early stage breast cancer will have surgery during the course of their treatment. Now, many are also candidates for  a variety of systemic therapies, either before or after surgery, to reduce the number of potentially microscopic cancer cells left behind and prevent the disease from coming back. Chemotherapy  is usually reserved for patients at higher risk for a recurrence or metastasis.

“We want to make sure we’re appropriately recommending specific therapies but sparing patients who have a lower risk of disease recurring and becoming metastatic,” Yardley said.

By administering these medications earlier, when the disease is still operable, researchers aim to increase the cure rate and prevent—or at least delay—recurrence and metastasis in breast cancer patients.

More Work to Be Done

Admittedly, much work remains to be done, according to Yardley. Over the last 60 years, breast cancer survival rates have tripled, but metastatic survival rates have a long way to go before they reach that level.

Clinical trials play an essential role in exploring new treatments and approaches in metastatic breast cancer, and these trials continue to become more targeted as researchers learn more about the disease subtypes. For instance, while immunotherapies have not proven effective in studies for metastatic breast cancer in general, trials investigating immunotherapy in specific breast cancer subtypes such as triple negative breast cancer have shown promising activity. Thus, targeted treatments in combination with chemotherapy continue to demonstrate great promise.

“The science has become astounding, allowing us to manipulate the biology of metastatic breast cancer through very tailored approaches,” Yardley added. “There’s every reason to be optimistic for patients facing the challenges of metastatic breast cancer today.”

To learn more about a patient’s experience with metastatic breast cancer, read “How This Metastatic Breast Cancer Survivor Told Her Family About Her Diagnosis.”

When treating a chronic disease such as psoriasis or psoriatic arthritis, time is of the essence: every day that a patient goes without an effective treatment is another day of suffering. Unfortunately, three-quarters of large employers offer their employees insurance plans that use step therapy policies, which can often delay patients from getting access to the medications prescribed by their doctors.

“You could be looking at a nine-month process before you get access to the doctor’s recommended treatment,” said Patrick Stone, vice president of government relations and advocacy at the National Psoriasis Foundation (NPF). “Insurance plans need to get patients access to medications their doctors determined were right for them sooner than that. They deserve better.”

Many states have stepped up to protect patients from step therapy procedures by enacting legislation that limits the use of step therapy, with Minnesota and New Mexico being two of the latest examples.

The Problems of Step Therapy

PATRICK STONE

PATRICK STONE FROM THE NATIONAL PSORIASIS FOUNDATION SAYS THAT THE ORGANIZATION IS NOT STOPPING UNTIL LEGISLATION PROTECTING PATIENTS FROM STEP THERAPY IS PASSED IN ALL 50 STATES AND ON THE FEDERAL LEVEL.

If a prescribed treatment isn’t on the insurer’s preferred medications list, the insurer may deny it until a patient tries and “fails” on one or more of the preferred options. This process, called step therapy, is commonly practiced among major private insurance plans.

Step therapy is based on a one-size-fits-all approach, assuming that patients respond similarly to treatments. But in reality, patients with chronic diseases such as psoriasis and psoriatic arthritis can have very different responses to the same medication.

Step therapy is not unusual in rheumatology and dermatology despite the fact that many of these chronic diseases are associated with serious comorbidities. Psoriasis and psoriatic arthritis patients can suffer from other ailments, making it even more important to address the disease effectively and promptly with appropriate therapies.

4 The Average Number of Treatments Psoriasis Patients Try

“Step therapy reform is a high priority for the psoriasis and psoriatic arthritis community,” Stone said. “If you’re not treating psoriatic arthritis in a timely and appropriate manner as determined by your doctor, it can certainly become a disabling disease.”

Reigning in Step Therapy

Recently passed step therapy reform legislation does not stop insurance carriers from enacting cost control measures. Instead, the laws are intended to protect patients by providing a timely exemption process to override step therapy procedure.

Over the past four years, the NPF has led a number of campaigns at the state and federal levels with other patient and provider groups across the country.

In 2018, New Mexico passed step therapy reform legislation. “As a result of step therapy legislation, people living with a psoriatic disease in New Mexico have better access to prescribed treatments,” Stone said. The total number of states that have enacted step therapy legislation is now up to 19.

The most effective spokespeople for step therapy legislation have been the patients, according to Stone. When legislation was being considered in Texas last year, a 16-year-old with psoriatic arthritis named Michael from San Antonio met with state legislators. In a room filled mostly with lobbyists, the Speaker of the House only wanted to hear from one person: Michael, who shared how step therapy delayed his treatment and the trouble that caused him. “Michael did a better job than any lobbyist could in articulating the issue,” Stone said.

As a result of step therapy legislation in Minnesota and New Mexico, the more than 190,000 Americans living with a psoriatic disease in those two states have better access to prescribed treatments.

Next Steps

With most state legislatures having already adjourned for the year, Stone and the NPF are already planning for 2019. “In the upcoming year, we plan to renew efforts in Florida, Georgia, Washington and Maine, while also exploring options in other states with no prior legislative attempts,” said Stone. “Meanwhile, states like Pennsylvania, Virginia and Oregon are considering folding step therapy regulations into larger bills aimed at protecting patients from insurance practices.”

“The momentum is behind us going into the 2019 legislative sessions,” Stone said. “We have a game plan in place already. We know what states we’ll be in, and we’re excited about the potential for large amounts of legislative victories during the next couple years regarding step therapy reform.”

Meanwhile, at the federal level, the administration recently announced that it would allow step therapy in Medicare Advantage plans for Part B medications, which are typically administered in a hospital or clinic setting. Step therapy is already allowed in Medicare Part D plans, which covers at-home prescription medications.

“Medicare Advantage holders are senior citizens and those who are permanently disabled,” Stone said. “That makes it even more difficult for them to understand how the mediation process works and how to appeal it. So we’ve still got plenty of work to do to protect them.”

To learn more about why the one-size-fits-all approach doesn’t work in psoriasis, read “Psoriasis Patients Deserve Their Prescribed Therapy Without Delay.”

At Celgene, bold science that benefits patients is at the core of our values and our business. Each year, Celgene’s cutting-edge medical research helps more patients around the world. While patients are the focus of our mission, we also take very seriously our responsibilities to our employees, our communities and our environment—responsibilities that we continue to live up to. Celgene’s 2018 Corporate Responsibility Report, released this week, highlights how we are working to make a difference in the lives of patients, communities and economies around the world.

“We are at a very important time in our company’s journey to help patients and create increased value for our stockholders,” Mark J. Alles, Chairman and Chief Executive Officer of Celgene Corporation, said. “Over the next two years, we expect to advance five late-stage products toward regulatory approval. These therapies represent the promise of our industry-leading investments in research and the beginning of our next wave of innovation.”

Patients First

Celgene’s commitment to changing the course of human health includes helping patients living in developing parts of the world. In 2017, Celgene joined 24 biopharmaceutical companies in the Access Accelerated partnership to improve access to treatment and care for non-communicable diseases — such as cancer — in low- and middle-income countries.

2018 Corporate Responsibility Report

During its first year, the partnership moved toward its goal by advancing 62 programs, including the Celgene Cancer Care Links™ grant program1 and the Academic Model Providing Access to Healthcare (AMPATH) Oncology Partnership, which Celgene joined in 2011 to increase access to cancer diagnosis and treatment in Kenya.

Celgene introduced the Cancer Care Links™ in December 2017 to improve cancer care in resource-constrained countries by giving organizations with existing infrastructure an opportunity to apply for grants to support oncology training, cancer prevention and detection, nursing programs, general medical support, pharmacy programs, and awareness and education initiatives. The first grant recipients were announced in December 2018.

This past March, Access Accelerated hosted a forum in Kenya, where Zeba Khan, Ph.D., Vice President of Corporate Responsibility for Celgene, took part in a panel to discuss the AMPATH Oncology Partnership. “Our partnership with AMPATH works within the current healthcare system to address a local need and to improve multiple myeloma care in Kenya,” Khan said.

ENGAGING ACROSS SECTORS AND DISCIPLINES TO ADDRESS NCDs

THE “ENGAGING ACROSS SECTORS AND DISCIPLINES TO ADDRESS NCDs” PANEL INCLUDED PATIENTS, HEALTHCARE PROFESSIONALS, BIOPHARMACEUTICAL COMPANIES, NON-PROFIT ORGANIZATIONS AND KENYA’S MINISTRY OF HEALTH.

Supporting Communities and the Planet

At Celgene, we’re committed to making a positive impact on the communities where we work and live through giving and volunteerism, and to the health of the planet through environmental stewardship and resource conservation.

Last year, more than 2,000 Celgene employees ran, cycled or walked in races and events to support finding a cure for serious diseases, including blood cancers, pancreatic cancer, psoriasis and psoriatic arthritis. In 2017, more than 1,000 Celgene employees helped raise $700,000 to support the Leukemia & Lymphoma Society’s Light The Night® events, making Celgene its top biopharmaceutical fundraising partner. Celgene was also the first National Presenting Sponsor of PurpleStride®, the Walk to End Pancreatic Cancer, in 2017. More than 280 employees participated in 52 PurpleStride walks and runs, raising $57,000.

Meanwhile, Celgene is making strong progress to meeting our 2020 environmental goals, which focus on reducing our carbon footprint, investing more in renewable electricity, reducing water withdrawal and reducing waste generation. This year, Celgene expanded its portfolio of LEED®-certified buildings and continued our trend of purchasing renewable energy. Renewable sources now make up 50 percent of all power used by Celgene worldwide, and wind-powered electricity fuels 73 percent of U.S. facilities’ energy. This achievement has resulted in an invitation to join the U.S. Environmental Protection Agency’s Green Power Partnership.

Built on Integrity

Celgene’s business culture is built on integrity, ethics, sound decision-making and behaviors that reflect our values and focus on patients. This year, Celgene joined the Pharmaceutical Supply Chain Initiative (PSCI) to enhance sustainability within our supply chain and was recognized for excellence in responsible clinical trial data sharing in the Bioethics International Good Pharma Scorecard, ranking in the top five for Clinical Trial Transparency.

Whether it’s sharing clinical trial data or providing grants to organizations in resource-constrained countries to improve the cancer care infrastructure, the common thread that binds our employees and all our activities is our focus on putting patients first. “We remain steadfastly committed to our mission, as we work to discover, develop and deliver to patients new and even more effective ways to improve and extend the lives of patients around the world,” Alles said.

To learn more about Celgene’s Corporate Responsibility activities, read the 2018 Celgene Corporate Responsibility Report.