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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.”

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

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.

More people are being diagnosed with Crohn’s disease and ulcerative colitis than ever before, but researchers aren’t exactly sure why. A variety of factors including genetics, weakened immune systems and the environment may be at play.

In this podcast produced for this year’s Crohn’s and Colitis Awareness Week, Cathy Ferrone, director of patient advocacy at Celgene, and Laura Wingate, senior vice president of Education, Support and Advocacy at the Crohn’s and Colitis Foundation, discuss the rise in worldwide incidence rates of inflammatory bowel disease and why research in this area remains so important.

 


CATHY FERRONE FROM CELGENE AND LAURA WINGATE FROM CROHN’S AND COLITIS FOUNDATION DISCUSS THE RISE IN INFLAMMATORY BOWEL DISEASE.

 

To learn more about the lifelong struggle of having an inflammatory bowel disease, read “What It’s Really Like to Live with Ulcerative Colitis.”

To explore the Crohn’s & Colitis Foundation’s resources available to patients, caregivers and health care professionals, visit their website at http://www.crohnscolitisfoundation.org/ or call 1-888-My-Gut-Pain.
 

Lung cancer remains the leading cause of cancer-related death in both men and women in the United States. And for America’s veterans, that risk is even higher as they are 25 percent more likely to be diagnosed with lung cancer than those who did not serve in the military.

As we recognize this year’s Veterans Day, Laurie Fenton Ambrose, President and CEO of Lung Cancer Alliance, explains how early detection and the treatment of lung cancer is evolving in and benefiting the veteran community, and what still needs to be done to improve the care for the men and women who have served our country.

Why are veterans disproportionately affected by lung cancer?

“Veterans are disproportionately affected for two key reasons: their smoking history and their occupational exposures. First, veterans have a higher prevalence of smoking than the civilian population. Many use smoking as a way to cope with the stress of their occupation and cigarettes were readily available to them while in service. At the same time, veterans were also exposed to a variety of chemicals that are linked with an increased risk of lung cancer, such as asbestos, Agent Orange, burn pits and chemical weapons.”

Why is treating veterans with lung cancer challenging?

LAURIE FENTON AMBROSE

LAURIE FENTON AMBROSE, PRESIDENT AND CEO OF LUNG CANCER ALLIANCE, IS HELPING TO LEAD THE CHARGE IN IMPROVING PREVENTION, SCREENING AND TREATMENT OF LUNG CANCER IN THE VETERAN COMMUNITY.

“Research has shown that many Veterans’ Affairs facilities are not prepared to implement comprehensive lung cancer screening programs. More work needs to be done to support the investment in infrastructure and resources to offer more coordinated care to American veterans with lung cancer as quickly as possible.

We also need to address the stigma associated with having lung cancer—which affects how the disease is both resourced and advocated for — and the comorbidities that many veterans face in addition to lung cancer.”

How is Lung Cancer Alliance working to improve care for veterans with lung cancer?

“We are working with military facilities and programs to reduce tobacco use and exposure. We are also working on awareness campaigns to alert veterans to their elevated risk for lung cancer as well as directing them to responsible screening and care so that we catch and manage the disease early.”

Why is early detection so important?

“Because it can save your life. Just like other diseases with approved screenings, you see higher survival rates in those whose disease has been caught early by screening. Now lung cancer can join this fold. Simply put, if you find lung cancer in its earliest form, you improve your treatment options and your quality of life. But right now, approximately 75 percent of lung cancers in the general population are diagnosed at late stage when there are fewer effective treatment options.

We need to highlight the benefits of screening and make sure that it is made available to our veterans. Studies have shown that low-dose CT screenings can decrease lung cancer death rates if we provide adequate resources and infrastructure. It’s key that we make sure everyone at risk—including our military community—is aware that these screenings are available to them.”

We’re at a pivotal moment right now with lung cancer screening and treatment advances, and we are seeing more lung cancer survivors than ever.

How are the efforts to improve screenings going so far?

“Screening is a fairly new preventive service. The federal government gave a green light just four years ago for coverage. So, we are now working hard to “ramp-up” this service in communities across the country. This involves bringing national awareness to the issue as well as educating providers and those at risk of the benefits and risks. We are also committed to making sure screening and care is being provided in the most responsible way in medical centers and considering how we collect information to help further improve early detection and treatment options. It is not easy. It takes time. But we are focused on moving this forward as rapidly as possible.

The challenge I see is that there isn’t the same sense of urgency and focus around lung cancer screening as we have seen with other cancers such as breast and colon. That’s likely related to the stigma around this disease. We really need more national attention on this issue.”

How are new treatment options helping patients with lung cancer?

“New treatment options for patients with lung cancer are improving care and bringing hope. For example, we have more targeted therapies and immunotherapies being paired with chemotherapy to treat lung cancer today than we have had in decades.

When you combine these new therapies with the ability to detect lung cancer earlier, outcomes can be more favorable for early stage patients. This one-two punch is making a dent in the high mortality rates. We’re seeing five-year survival rates rise to 19 percent; a leap forward from where we were in the 1980s, when five-year survival was only 13 percent. We’re at a pivotal moment right now with lung cancer screening and treatment advances.”

What else should veterans know about lung cancer?

“No veteran should ever feel alone. We are ready to support them. That’s what Lung Cancer Alliance is here to do. Veterans should know that there’s a place where they can go to feel part of the community and find information. We are honoring their service by making sure our service is there for them.”

To learn more about how researchers are developing new treatments strategies for lung cancer, read “Hitting Moving Targets in Lung Cancer Subtypes.”

In August 2005, Amelia List celebrated her first birthday, and her mother Julie breathed a sigh of relief. She had feared Amelia would develop severe food allergies, as her five-year-old sister Autumn had, by the time she turned one. But it was so far, so good.

Unfortunately, everything went downhill from there, Julie recalls. A month later, the entire family got the stomach flu. Everyone recovered well except for Amelia, whose vomiting and diarrhea continued. Six weeks later, she had lost 20 percent of her body weight. Julie and her husband took their daughter to a gastroenterologist, who diagnosed Amelia with eosinophilic esophagitis.

Having already joined several food allergy forums online, Julie knew what that meant. Eosinophilic esophagitis was not your typical allergic reaction to food. She turned to her husband. “We’re going to be one of those people whose kid can only eat one or two foods,” she told him.

More than 150,000 children and adults in the United States live with eosinophilic esophagitis, a relatively new disease that was only first recognized in the 1990s. There are currently no FDA-approved pharmaceuticals to treat EoE. Symptoms may be managed with elimination diets and other methods. Julie is sharing their story to raise awareness of the disease, with the hope that more can be done to improve their daily struggle.

Identifying Triggers

Julie knew eosinophilic esophagitis was not a typical food allergy, but she was stunned when their local gastroenterologist told them that he could only diagnose but not treat the condition. In fact, at that time, there was no doctor near their home in South Carolina who treated this rare disease. For the next four years, the family traveled eight hours to Cincinnati to see a specialist whenever necessary.

The doctor explained that proteins in the foods Amelia was eating were triggering a type of white blood cell called eosinophils to inflame her esophagus. This inflammation led to her vomiting, difficulty swallowing and recurring stomach pain.

Amelia underwent food trials to identify her food triggers, eating one or more foods at a time for two months to see whether they made her sick. If they didn’t, she’d get an endoscopy to check her upper digestive tract for inflammation. Her doctor would put her under anesthesia and insert a flexible tube with a camera into Amelia’s upper digestive tract.

Her doctor also took six to ten biopsies throughout the esophagus to determine if eosinophils were present. “The biopsies can reveal if eosinophils are present and causing damage that is not visible to the eye,” Julie said. “We never knew if food was safe until the biopsy results were returned.”

She went through this process with a dozen foods. To get her required nutrition, Amelia was given an amino-acid based formula through a gastric feeding tube. “The formula had no proteins that would trigger allergies, but it tasted terrible,” Julie said.

We never tell them they can’t do anything.
We’ve tube-fed Amelia while hiking.

Overcoming Setbacks

In 2007, the List family received more devastating news when their middle child, Abby, was diagnosed with eosinophilic esophagitis at the age of six. Studies have shown that siblings of someone with eosinophilic esophagitis are at increased risk for the disease, suggesting a role for genetic factors. Environmental factors are also thought to have a role.

Then in 2016, Amelia had a severe, life-threatening allergic reaction to white rice, her first allergic reaction unrelated to her eosinophilic esophagitis. Her immune system reacted, and Amelia soon found she could no longer eat any of the foods she previously tolerated.

Amelia resumed consuming formula through the gastric tube but then started reacting to that, too. To help tolerate the formula, she takes medication twice a day. She once again began food-testing with endoscopies to validate new safe foods. Since her initial diagnosis, Amelia has had 32 endoscopies and counting.

Today, she continues to consume formula, but it is not her sole source of nutrition. She can eat seven foods: apples, sweet potatoes, kidney beans, soy, millet flour, turkey and black olives. “For sure, you get sick of them, but I just have to keep eating them,” Amelia said. “I really don’t have any other choice.”

Growing Up Quickly

Amelia has been administering her own tube-feedings since she was a kid. Now 14, she has it down to a science. She eats this way three times a day, and she takes her equipment with her everywhere, plus the formula and water to mix.

Amelia reminds herself not to let eosinophilic esophagitis hold her back from living the life she’s dreamed of living. “It’s part of you, but it doesn’t control you. It’s not who you are,” Amelia said.

Going out—whether to school, on a field trip, to a friend’s house or on vacation—requires planning. If she goes to a party or sleepover, she brings a can of olives or a sweet potato in case she gets hungry. Sometimes, of course, she chooses to forgo events if she decides that they’re not worth the effort.

“We never tell them they can’t do anything,” Julie said. “We’ve tube-fed Amelia while hiking. But they definitely analyze situations ahead of time, which most kids don’t have to think about. These kids are very responsible and have to grow up quickly.”

To learn more about how Celgene is committed to supporting research for rare diseases, read Supporting Research to Find Cures for Rare Diseases.

While understanding the impact of myelodysplastic syndromes (MDS) on the lives of people diagnosed with these blood cancers can help inform their care, assessments of quality of life in MDS have been, for the most part, lacking.

But a recent survey commissioned by the MDS Foundation, Inc. is helping to fill that gap, to shed some much-needed light on the experience of patients and their caregivers. According to the results, many people with MDS surveyed said that fatigue had a significant impact on their daily lives.

“The number one complaint that we hear, by far, from patients with MDS is that they don’t have the energy to do the things that make them feel like they’re living,” said Tracey Iraca, executive director of the MDS Foundation, which is raising awareness of quality-of-life issues during this year’s MDS World Awareness Day. “There are so many little things we take for granted that these people struggle with.”

A Closer Look at the MDS Symptom of Fatigue

Tracey Iraca

TRACEY IRACA, EXECUTIVE DIRECTOR OF THE MDS FOUNDATION, INC., BELIEVES A BETTER UNDERSTANDING OF HOW MYELODYSPLASTIC SYNDROMES AFFECT PATIENT LIVES IS THE FIRST STEP TOWARDS IMPROVING THE MANAGEMENT OF THE DISEASE.

The fatigue and tiredness that people with MDS experience interferes with their daily activities, according to the survey. Patients surveyed reported that they often struggled with tasks such as cooking, cleaning, shopping, climbing stairs and taking care of their pets. Several respondents said that they relied on other people to complete many of those chores.

“My house is not nearly as clean since [I was diagnosed with] MDS,” one person responded. “I am exhausted a lot of the time. I can only work short times, and I have to sit down—then I usually fall asleep.”

Some people reported feeling tired all day, every day, while others only experienced exhaustion in the afternoon. Some also said that naps in the afternoon had become a necessity in their everyday life.

Fatigue drains people with MDS not only physically but emotionally, according to the survey. People with MDS reported losing patience with themselves and worrying about their loss of independence. Several also said they experienced feelings of isolation and loneliness from not being able to visit their family and friends.

Why Fatigue Is a Symptom of Myelodysplastic Syndrome

It didn’t surprise Iraca that fatigue was of significant concern for people with MDS. She has heard it dozens of times over the past decade at the Foundation. And she understands why.

In MDS, the bone marrow doesn’t produce enough healthy red blood cells, which transport oxygen to different cells and tissues. Young red blood cells are then inhibited from properly maturing, caused by what is known as erythroid maturation defects.

“The normal development of all blood cells is a complex process that relies on both stem cells and the environment within the bone marrow,” explained Sandra Kurtin, board member of the MDS Foundation, assistant professor of clinical medicine and assistant professor of nursing, The University of Arizona Cancer Center. “This process goes awry in MDS due to a variety of issues.”

As a result of this ineffective development of red blood cells, up to 90 percent of people with MDS have low red blood cell counts, a condition known as anemia. Without enough healthy red blood cells to transport oxygen, it leaves people feeling continuously tired and weak throughout the day.

“Patients are becoming much more aware of what’s happening—they are learning to talk with their doctor about their fatigue and other symptoms of anemia.”

Managing MDS Linked Anemia

Understanding how MDS affects the daily lives of people is the first step toward improving care for the thousands living with this disease. When people with MDS, doctors and caregivers discuss the complete patient experience, they can address what matters most.

“Patients are becoming much more aware of what’s happening—they are learning to talk with their doctor about their fatigue and other symptoms of anemia,” Iraca noted. “We want to educate people to identify these symptoms earlier on so that they can get treatment sooner for anemia.”

People with MDS may receive red transfusions to raise their low blood counts and antibiotics to prevent or fight infections. Some people may also receive a bone marrow transplant, chemotherapy, or other treatment options. Iraca is hopeful that research will help us learn more about the disease and how to treat it.

“Researchers are working to identify genetic defects in MDS so that they can develop therapies to target them,” Iraca said. “The research that’s happening now makes us hopeful.”

To learn about the high unmet need for people with MDS, read “Why I Advocate for People with Myelodysplastic Syndromes.”

What’s on the outside can often distract us from what’s happening on the inside. For people with plaque psoriasis, that can mean treating their outer symptoms, but sometimes their treatments aren’t addressing the deeper problem within.

On this year’s World Psoriasis Day (October 29), it’s time to gain a better understanding of psoriasis: an autoimmune condition that causes an overactive immune response. It’s this immune response that causes the rapid growth of skin cells, leading to red, raised, itchy and scaly patches. By understanding this, people with psoriasis can better understand all of their possible treatment options.

 
To learn more about plaque psoriasis and available treatment categories, visit DeeperLevelPSO.com

Never judge a book by its cover: for TV host and fashion journalist Louise Roe and the millions of people living with psoriasis in the United States, it is more important than ever to understand this chronic condition.

Roe has teamed up with Celgene to launch INSIDE PERSPECTIVE, a campaign that aims to raise awareness about plaque psoriasis – an autoimmune condition that appears on the surface of the skin but starts inside the body.

While working at a fashion magazine in London at the age of 25, Roe was diagnosed with plaque psoriasis. Initially, she struggled with the diagnosis and managing the condition – planning her wardrobe and work events around hiding plaques on her skin.

“It was quite tough for me at first given the fashion industry is very focused on outward appearances,” said Roe. “At times, when the symptoms were too severe to hide, I canceled plans and social events – it definitely affected my confidence and my life in general.”

After working with her dermatologist, she was able to better understand plaque psoriasis and found ways to help manage the symptoms, all while building a successful fashion career as a journalist, author and blogger. Because of her personal experiences with her condition, Louise is speaking out to further educate the psoriasis community to treat the condition from within.

Through INSIDE PERSPECTIVE, Roe also had the opportunity to swap worlds for a day with Alycia, one of her fans who lives with moderate to severe plaque psoriasis. Their experiences are chronicled in an inspiring video where they share heartfelt insights about living with the condition, as well as the importance of looking beneath the surface and embracing who they are both inside and out. The full story and video can be viewed at www.PsOPerspective.com.

“What I’ve learned through this experience is that there’s always more to the story,” said Roe. “By opening up about my experience, I hope the campaign will help inspire and empower others.”

Louise is one of the estimated 7.5 million people in the United States living with plaque psoriasis—an autoimmune disease that appears on the skin as red, itchy patches that starts inside the body. Though often seen as a skin condition, plaque psoriasis can have both a physical and an emotional impact on those living with it.

To learn more about the INSIDE PERSPECTIVE campaign and Louise’s personal story, visit www.PsOPerspective.com.

Although survival rates for blood cancer have improved over the past decade, it remains the third deadliest type of cancer in the U.S. That’s why supporting blood cancer research is so crucial, according to Louis J. DeGennaro, Ph.D., president and CEO of The Leukemia & Lymphoma Society.

“About 25 years ago, a handful of Leukemia & Lymphoma Society volunteers in Minneapolis wanted to organize a family-friendly walk at dusk to celebrate cancer survivors and their supporters. That walk evolved into Light The Night, which now takes place in over 200 communities across the United States and Canada.

We also now recognize people we’ve lost to blood cancer, as well as survivors and supporters. At each walk, you’ll see red, white and gold lanterns. The red lanterns recognize supporters, the white lanterns are for survivors and the gold lanterns are to remember those we’ve lost.”

How is the money raised from an event like Light The Night spent?

LOUIS J. DEGENNARO

LOUIS J. DEGENNARO FROM THE LEUKEMIA & LYMPHOMA SOCIETY BELIEVES THE LIGHT THE NIGHT EVENTS REMAIN AS IMPORTANT AND AS MOVING AS EVER.

“These walks are a major fundraiser for The Leukemia & Lymphoma Society. Last year, we raised $60 million to support our research, patient services and advocacy work.

Research has always been the bow of the ship for The Leukemia & Lymphoma Society. We were founded 70 years ago by a father and mother who lost their son to leukemia, and their mission was to raise funds to support research and find a cure. The investment is paying off: the U.S. Food and Drug Administration (FDA) approved 18 therapies for blood cancer last year. To put that into perspective, a good year would involve the FDA approving two new blood cancer therapies.”

With all these advances, why is fundraising still so important?

“For starters, some blood cancers are stubbornly resistant to treatment. For example, acute myeloid leukemia (AML) is a commonly diagnosed cancer, but we hadn’t seen advancement in survival rates for 30 years, until four new approaches were approved by the FDA between 2017 and 2018. Without continued investment in research, those new treatments would not have been developed.

There’s also an even more important reason for continuing this support: when you make an advance in blood cancer research, it may benefit other cancers as well. About 40 percent of all cancer therapies approved by the FDA since 2000 were first approved to treat blood cancers.”

CAR T cell therapy has the potential to help people with certain types of blood cancer and is in early testing for other types of cancer.

What are some of the promising treatment approaches that The Leukemia & Lymphoma Society is investing in?

“We’re very keen on immunotherapy. In fact, we’ve contributed $40 million to research since 1998 for an immunotherapy called chimeric antigen receptor (CAR) T cell therapy, which reprograms immune cells so a person’s immune system can fight the blood cancer. CAR T cell therapy has the potential to help people with certain types of blood cancer and is in early testing for other types of cancer, including colon, ovarian and lung cancers. We’re also excited about targeted therapies, which are designed to go after specific, bad-acting genes.”

Light The Night Helps Lead the Way for Blood Cancer Research

Why are Light The Night walks so meaningful to you?

“A few years ago, I attended a walk in Minneapolis at the major league baseball stadium. I was walking around that stadium at dusk, carrying my red lantern and walking beside folks carrying gold lanterns for loved ones that they have lost to blood cancer. Suddenly, the outfield gate opened and hundreds of survivors carrying white lanterns came walking onto the field.

Then, a child survivor broke out from the pack and started running around the stadium. I was in tears. That moment felt emblematic of the progress we’ve made – and how quickly we’ve made it. That child, and all the people at these walks who carry those white lanterns, are a symbol of the success of our work. There are a growing number of white lanterns every year. It’s truly moving.”

To find a Light The Night event near you, visit The Leukemia & Lymphoma Society’s Light The Night website.

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At 6:21 p.m. on September 18, 2016, the sun was beginning to set in New York City, and Eric Gelber started his final lap around Central Park. A crowd of supporters were running with him and cheering him on, and cowbells were ringing along the race course. Every step was painful. When he finally crossed the finish line, he broke down and kissed and hugged his wife. He had just run 200 miles and raised over $320,000—bringing his lifetime fundraising total to more than $1.1 million and moving us one step closer to a cure for multiple myeloma.

But for Gelber, the journey is not over yet. Now, he’s inviting other runners from the New York City area and beyond to join him on The Journey Towards a Cure initiative by taking part in a 12-hour endurance run/relay on October 6, 2018, to support the Multiple Myeloma Research Foundation (MMRF).

In anticipation of this new chapter in a personal journey he started more than a decade ago, Gelber explains what keeps him coming back to this cause, how this year’s event will be different than his previous efforts and why it remains vital to support multiple myeloma research.

What did it feel like to finally achieve that 200-mile run?

“It was unbelievable. I was somewhat out of it when I finished, but it felt like a dream. After two failed attempts, it was an amazing feeling to fulfill that goal. I was thinking of what all the people on my support team and I had just accomplished. Together, we pushed our cumulative fundraising total to over $1.1 million.

I’m not sure what would have happened if I did not complete all 200 miles. I decided it would be my final attempt before I did it. The following year, a documentary was released at the Tribeca Film Festival to tell my story and the stories of my friends who have been affected by multiple myeloma. I hope our story inspires more people to support this effort.”

ERIC GELBER AND HIS WIFE DISCUSS HIS PROGRESS AFTER COMPLETING 176 MILES DURING HIS FINAL ATTEMPT TO RUN 200 MILES IN SUPPORT OF THE MULTIPLE MYELOMA RESEARCH FOUNDATION IN 2016.

What kept you coming back year after year to push yourself to your limits and raise money for multiple myeloma research?

“Over time, this became a massive part of our lives, and I keep coming back because I want to do as much as I can to support the MMRF.

I’m not a scientist. I’m not a doctor. But I can run, and I can fundraise. That’s my way of getting us one step closer to a cure for multiple myeloma.”

This year, you are encouraging others to run a 12-hour relay race. Why did you feel it was time to change the event?

“This year, there is a different format. I wanted others to feel like they were actively helping the cause as well. An event that gets people to come out and push themselves—either solo or in a team—expands the idea of building a community. I didn’t expect it would take this format, but it was time to evolve.

There will be the race followed by a celebration with food, kids’ activities and a music festival with four live bands. It’s important to give people a reason to come together, share their stories and celebrate life in a fun atmosphere. I’m looking forward to this new chapter.”

What advice do you have for someone who wants to get involved with this year’s event?

“If you’ve never run a race before, be cautious and set achievable goals. Pace yourself so you avoid getting injured. There is an athlete in all of us, but you don’t have to run for 12 hours. You can a join a five- or 10-person relay team and run for as long as you can. As long as you’re challenging yourself, there’s a lot to be proud of.

If you are unable to run, there’s the option to walk a loop on the course. We also need volunteers to help direct traffic, register participants and manage aid stations. All of that is critical to making this event possible.”

One of my friends has been living with multiple myeloma for 20 years. Part of that is because there are more treatments available now. It’s very encouraging.

How do you feel about the progress made in the treatment of multiple myeloma over the last decade?

“I see progress in the faces of my friends who have multiple myeloma. They’re living longer. One of my friends has been living with multiple myeloma for 20 years. Part of that is because there are more treatments available now. It’s very encouraging.

There’s still a considerable amount of work to be done. We still don’t have a cure. That’s why we’re opening the event to a broader audience. I’m still learning about organizing and putting together an event as big as this. But sometimes you have to go for it and try—just like attempting to run 200 miles. You take it one step at a time.”

To learn more about the upcoming The Journey Towards a Cure event, visit The Journey Endurance Run/Relay website.