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ASCO 2018 will be remembered for the potential paradigm changing innovations across hematologic and solid tumor diseases, but there was a critical discussion also taking place around the tremendous value these advances are offering patients.

On Friday, June 1, Celgene presented a workshop entitled “Understanding the Value of Innovation in Oncology” that looked at what value means to different stakeholders, including patients, physicians and health economists on a series of panels throughout the day.

The first session featured Dr. Rafael Fonseca of the Mayo Clinic and Dr. Anupam Jena of Harvard Medical School who spoke about why measuring value matters in oncology during this current era of increasing costs. The doctors discussed the concept of healthcare as an investment and the inherent value that research and innovative therapies are providing for patients with cancer.

Dr. Fonseca

Dr. Jena then brought Dr. Peter Neumann of Tufts University School of Medicine, Dr. John Romley of the University of Southern California, and Dr. Jason Shafrin of the Innovation and Value Initiative to talk about how we currently measure value and why context matters in these assessments. The group also discussed new approaches to measuring value including a new open-source, consensus-based approach to measure the benefits, risks, and costs involved in assigning value.

Importantly, the patient voice was well-represented during the event as Jennifer Hinkel, a patient and Partner at McGivney Global Advisors, hosted a second panel that explored what patients believe are important aspects of innovative therapies. Dr. Michael Millenson of Health Quality Advisors, Sarah Krüg, CEO of Cancer101 and patient Stuart Cornew explored the many considerations that contribute to a therapy’s profile such as clinical benefit, survival, toxicity, novelty, and quality of life, and how those factors contribute to value.

Finally, after Dr. Fonseca grounded the group in physicians’ considerations around value, he hosted a panel including Jennifer Hinkel, Dr. Jena and Dr. Neuman that looked toward the future at how different audiences can be engaged in discussions around value and how the community can overcome challenges and limitations of current approaches.

“This event was a true opportunity to hear from some of the most important voices in any discussion about value,” said Dr. Safiya Abouzaid, host of the workshop from Celgene Corporation. “By understanding what value means for each stakeholder- how it is currently assessed and how we can reflect different perspectives in that assessment- we can ensure a continuation of the kind of innovation that has improved the lives of many patients over recent years.”

Dr. Jena
 

By Mark J. Alles, Celgene Chairman and Chief Executive Officer

A few decades ago, drug discovery was, for the most part, a matter of trial and error. The world’s most successful pharmaceutical companies would often collect dirt from far-flung locales and screen that soil or sand for medically active components. Biology was often a secondary concern. It was the speed of trial and error that was important.

Today, drug discovery is more targeted and intentional, building on a wealth of data that has grown exponentially since the early days of trial and error. Our knowledge of cell biology and genomics is now sufficiently advanced that it is possible to create medicines based on our individual immune cells or genetic profile. These advances promise patient-tailored medicines, such as the new CAR T therapies that “train” the patient’s own immune system to fight cancer, that have the potential to transform how cancer is treated.

This kind of innovation, however, requires investment to be sustained. Both the basic science and the delivery of treatments require investment to foster the next generation of therapies. The challenge is ensuring access to and reimbursement for those new medicines, thereby enabling innovators to reinvest in research and development. This is the virtuous cycle of innovation.

Mark Alles

Mark J. ALLES, CELGENE CHAIRMAN AND CHIEF EXECUTIVE OFFICER


If the costs of a new intervention far exceed the benefits, or if patients cannot get access, we’ve failed – as a system – to provide value. Conversely, when we see benefits that far outstrip costs, we can be confident that we are moving the health system in a direction to higher value, with better health and lower costs.

Understanding that balance has never been more critical. The United States spends $3.3 trillion on health care every year, or 18 percent of the nation’s gross domestic product (GDP). That’s about equal to the entire GDP of Germany. Among the primary drivers of this spending are hospital care (32 percent); physician and clinical services (20 percent); retail prescription drugs (10 percent); and other health, residential, and personal care services (5 percent). We have a responsibility to make sure that those dollars are spent wisely and to define what we – as a company and as a part of the health care system – believe is a good investment in the health of the nation.

Celgene’s Value and Innovation Framework Report is an effort to help meet that responsibility. We have developed a framework that outlines our approach to value and defines our role as a driver of value.

 “We have a responsibility to define what we believe is a good investment in the health of the nation.”

Though spending on biopharmaceutical products remains a relatively small piece of the overall health care environment, the biopharmaceutical sector has had an outsized impact on outcomes, and we are proud of our role in a therapeutic revolution that has cut the cancer death rate by 25 percent since 1991. While overall spending on biopharmaceutical products has indeed increased over the years, it has contributed to significant improvements in health outcomes. In fact, one study found that over 70 percent of recent life expectancy growth is due to the increased use of medicines.

Even with the increasing innovation coming from the biopharmaceutical sector, there are signs that drug spending is stabilizing. In 2017, per-person spending on prescription drugs rose just 1.5 percent across plans covering employees and their families, less than half of the increase reported in 2016 and the lowest increase in 24 years of tracking drug-trend data. Another study found that after accounting for rebates and discounts, spending growth on prescription drugs in the United States slowed to 0.6 percent in 2017.

But we cannot simply proclaim successes, declare that we have provided value, and avoid further discussion. Instead, we must start with a clear definition of our goals and a fair-minded examination of our impact at every level of the health care system. This report is evidence of our commitment to evaluating our performance as “value drivers” so that we can continually refine our role in the virtuous cycle of innovation.

To learn more about how Celgene defines value and measures it through medical innovation, read the 2018 Value and Innovation Framework Report.

Pancreatic cancer is once again an area of focus at this year’s American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. New research is increasingly important as estimates show that the mortality rate of pancreatic cancer could surpass that of breast and colorectal cancers by 2030 in some countries. Celgene is at the forefront of the fight against pancreatic cancer, and at this year’s ASCO conference, new data will continue to expand our understanding of emerging investigational treatments.

Celgene continues the fight against pancreatic cancer through our commitment in advancing research, our collaboration with advocacy groups and our support to patients.

Some look. We envision.

Celgene is committed to advancing research to help change the lives of patients with pancreatic cancer. In 2017, more than 20 percent of our revenue was reinvested into R&D, one of the highest rates in the industry.

Part of our research aims to inform today’s treatment choices in pancreatic cancer. Given the availability of more treatment options for patients with metastatic pancreatic adenocarcinoma, it is time to develop a treatment plan for patients. Celgene is contributing to this important discussion by helping physicians and patients make informed treatment decisions. With an eye toward the future, we are also conducting research and collaborating with researchers around the world on 91 ongoing investigational trials that combine novel agents with the foundation of approved Celgene therapies. These trials involve more than 40 unique novel compounds across more than 30 mechanisms of action and target enrollment of more than 4,500 pancreas cancer patients.

Celgene: Your partner in the fight against pancreatic cancer.

Some hear. We listen.

Celgene collaborates with patient advocacy groups to bring hope to the fight against pancreatic cancer. In partnership with 29 international patient groups, Celgene created the first-ever World Pancreatic Cancer Day in 2014 and continues to grow the event. Celgene also supports events such as Purple Stride Walks in the United States, the European Multi-Stakeholder platform on Pancreatic Cancer in November 2014, 2015 Pancreatic Cancer Forum in Milan and grants to help transport patients to their treatment appointments through the CancerCare GetYouThere program.

Some touch. We feel.

Celgene is dedicated to offering compassion and support to patients with pancreatic cancer and those who care for them. To help them, Celgene developed NavigatePanc.com, a personal pancreatic cancer information center. The site provides information about the disease, clinical trials, support groups, finances and communication and allows patients and caregivers to create their own online libraries of relevant information. Celgene also recognizes that pancreatic cancer patients often have special nutritional needs due to compromised pancreatic function. Our response: a source for recipes and meal planning. Lastly, we are committed to increasing the awareness of pancreatic cancer in the healthcare community.

Pancreatic Cancer: Cooking. Comfort. Care.

We support all who fight pancreatic cancer.

Celgene is committed to providing information and support to people with pancreatic cancer, and their loved ones, to help them in making informed decisions throughout their journey.

To learn more:

The cost and value of medical innovation in oncology is one of the hot topics on the agenda for this year’s American Society for Clinical Oncology Annual Meeting (ASCO). One cancer that has seen an increase in relative survival rates over the past decade is multiple myeloma. So it makes sense that multiple myeloma is at the center of a debate at ASCO on the cost and value of new therapies.

As one of the participants in that debate, Dr. Rafael Fonseca, a hematologist, oncologist and chair of the Department of Internal Medicine at Mayo Clinic in Arizona, will argue that society can’t afford not to provide patients with multiple myeloma access to the best care possible. In this Q&A, Fonseca shares his views about the affordability of cancer therapies, why many doctors hold onto the notion that medications are too expensive and the implications for future multiple myeloma treatment.

DR. RAFAEL FONSECA FROM THE MAYO CLINIC IN ARIZONA BELIEVES THAT SOCIETY CAN’T AFFORD NOT TO PROVIDE PATIENTS WITH MULTIPLE MYELOMA ACCESS TO THE BEST CARE POSSIBLE.

DR. RAFAEL FONSECA FROM THE MAYO CLINIC IN ARIZONA BELIEVES THAT SOCIETY CAN’T AFFORD NOT TO PROVIDE PATIENTS WITH MULTIPLE MYELOMA ACCESS TO THE BEST CARE POSSIBLE.

In the debate, you will argue that we can’t afford not to provide patients with access to the right treatment for them. How did you come to that conclusion?

“Over the past decade, I’ve witnessed so many patients with multiple myeloma increasingly beating the odds for survival. So I wanted to know what exactly was responsible for this. After some research, I concluded—as many of my colleagues have also—that it was the new medications. These innovative therapies provide tremendous value to our patients and society.”

When you discuss this topic with your fellow oncologists, what is the most compelling evidence supporting your position?

“I usually take a stepped approach to presenting my point of view. I ask them to consider the progress that we’ve made in survival in cancers like multiple myeloma and what they have seen in their own patients. I help them understand the value by walking them through all the new therapies that those improvements are attributed to. You can’t just look at the price tag of a specific medication, which they often focus on most.”

The opposing position is that patients and society cannot afford multiple myeloma therapies. Why don’t you believe that argument?

“The data don’t support the argument. One study found that 98 percent of patients paid $50 or less to fill their prescriptions in 2017. While that could be a hardship for some people, it is far different than the list prices of thousands of dollars that make headlines. So we need to talk about what patients are paying in the real world and what’s best for our patients.”

“Beyond that, there are the ethical considerations. Doctors should prescribe the medications they believe will benefit their patients the most.”

Why do doctors continue to say that new cancer therapies are unaffordable if the data suggest they aren’t for most patients?

“I feel like most cancer doctors are concerned about the cost of prescription medications out of their sense of compassion and responsibility for their patients. They see their role as treating patients responsibly. Prescribing a therapy that may cost them thousands seems inconsistent with that mission.”

“But they are so busy caring for patients that they don’t have the time to research the real world data about what patients actually pay for their prescriptions. As we see in other areas of discourse, facts matter. I believe that misunderstood empathy and baseless rhetoric can have real-world consequences for patients.”

Everyone agrees that today’s cancer treatments are simply not good enough and that innovation is key to improving cancer care.

Have you ever had a patient —or many—who could not access treatments that you prescribed?

“I cannot think of a single patient who could not access a medication due to financial reasons. I’ve had patients who have chosen other treatment options but for other reasons—never financial. In a few instances, we have had to go above and beyond to get them financial help from the manufacturer or non-profit groups. But those cases are the exceptions, not the rule.”

What do those on the other side suggest should be done about the affordability of multiple myeloma care and what are the potential consequences?

“When we say that new therapies are too expensive, what we’re doing is calling for price regulations. But without a doubt in my mind, those regulations will kill innovation. Medical innovation is a high-risk, high-reward endeavor. We should not fool ourselves into thinking that there won’t be consequences; we’ll have fewer new treatments for our patients as a result.”

Is there common ground in this debate over the value of multiple myeloma care that can be used to move forward?

“There is plenty of common ground. I think today’s cancer treatments are simply not good enough and that innovation is key to improving cancer care. So we should make sure that we do not hinder that innovation.”

“Most cancer doctors also agree that clinical trials should be as fast as possible without sacrificing safety, so they cost less and lead to faster approvals. And I think that we all think patients should have access to the best treatment options but have different ideas on how to provide that access.”

To learn more about the how medical innovation has improved the treatment of multiple myeloma, read “A Decade of Progress in Multiple Myeloma, and More to Come.”

Dr. Fonseca has received speaker fees, advisory board fees, travel support in connection with consulting services, and research support from Celgene.

An estimated 1.5 million Americans are affected by lupus, and each of these patients has a different story to tell. This inflammatory disease — in which a patient’s immune system begins attacking healthy organs and cells — has many different symptoms and affects each person differently. Some people with lupus have a few mild symptoms, but others can experience many symptoms and severe complications.

In this video produced for this year’s Lupus Awareness Month (May), we asked several patients to share how this complicated, incurable disease affects their daily lives, what they want others to know about their condition, and how they have learned to manage their symptoms.

Celgene wants to thank the Lupus Research Alliance for their partnership in the making of the video.

To learn more about how advances in lupus research are leading to potential new treatment options, read “Genetic Research in Lupus Offers New Lines of Attack.”

In 2017, California’s state health insurance exchange became the first to implement caps on what insurers could charge patients for a prescription medication, topping them at $250 per month, in an effort to protect the chronically ill from ever-increasing insurance cost sharing. While some critics argued that caps would substantially increase pharmacy spending and premiums for everyone, several studies suggested otherwise.

GABRIELA DIEGUEZ, A PRINCIPAL AND CONSULTING ACTUARY WITH MILLIMAN, DID NOT SEE CALIFORNIA INSURERS EXPECTING MUCH IMPACT ON SPENDING OR PREMIUMS FROM COST-SHARING CAPS.

GABRIELA DIEGUEZ, A PRINCIPAL AND CONSULTING ACTUARY WITH MILLIMAN, DID NOT SEE CALIFORNIA INSURERS EXPECTING MUCH IMPACT ON SPENDING OR PREMIUMS FROM COST-SHARING CAPS.

Now, California’s insurers have released their cost projections for 2017, the year that the cost-sharing caps went into effect. The data reveal that insurers did not project much, if any, change in spending or premiums due to the patient out-of-pocket caps, according to an analysis by the actuarial firm Milliman.

“If insurers thought that the sky was falling, we would have seen it in their projections,” said Gabriela Dieguez, a principal and consulting actuary with Milliman and the lead author of the report. “But they didn’t seem to be anticipating spending to grow disproportionately.”

Dieguez and colleagues compared the change in pharmacy spending projections from insurers in California to those in other states without caps. Not only did California insurers not project higher prescription spending increases between 2015 and 2017 than did other states, but their projections actually came in about 1 percent lower for people who buy their own insurance and 3 percent lower for those who get their insurance through a small employer.

Cost Sharing Caps

Some have worried that insurers might raise premiums substantially to guard against any losses from the caps. But this doesn’t seem to be the case, according to the Milliman analysis, which estimated that premiums increased by only about 1 percent for average members.

That relatively small increase leads to significant savings when an innovative therapy for a chronic disease is needed. Dieguez and colleagues projected annual savings of $586 per family member for families that include someone with blood cancer and $683 per family member for those including someone with rheumatoid arthritis.

Savings Due To Cost-Sharing Caps

The findings will help California and other states who have passed similar legislation understand the impact of their caps. Delaware, Louisiana, Maine, Maryland, Montana, New York and Vermont have all limited how much insurers can ask patients to pay for their prescription medications.

“While several states have regulations that limit cost sharing for prescription medications, we chose California because its large insured population provided a credible basis for our analysis,” Dieguez said.

If insurers thought that the sky was falling, we would have seen it in their projections. But they don’t seem to be anticipating spending to grow disproportionately.

The analysis will also inform states who are looking to introduce such legislation, including Connecticut, Illinois, Kansas, Kentucky, Massachusetts, Michigan, Mississippi, Nevada, New Jersey, Ohio, Oregon, Pennsylvania and Washington.

While these numbers are based on projections from insurers, the true impact of the caps on prescription spending and premiums will be revealed when California’s insurers release the final data for 2017 later this year.

“We’re very much looking forward to those data so we can see if the caps made as small of an impact on spending and premiums as the insurers projected,” Dieguez said. “Many people are following the California case closely.”

To learn more about how states are taking action to improve patient access, read “Protecting Patients from Higher Cost Sharing.”

Most people have never heard of Behçet’s disease a painful, chronic condition that causes sores in the mouth and elsewhere on the body. Since only 16,000 to 20,000 people in the United States are affected by this condition, that’s hardly surprising. But for those suffering in silence, it’s important to know that, while there is no cure, diagnosis and treatment can help manage symptoms.

The diagnosis is based on the combination of clinical findings, and most doctor may not be very familiar with the condition and its symptoms as it is so uncommon in the United States many patients face significant delays in getting properly diagnosed and treated. One study suggested an average of nearly three years to diagnosis for patients with Behçet’s disease.

Knowing the signs and symptoms is essential to managing the disease. As part of this year’s Behçet’s Disease Awareness Day (May 20), we’re highlighting the five most common signs and symptoms that may develop as the disease progresses, with the hope that more patients can take control of their condition by talking to their doctor and getting treated as soon as possible.

Mouth Sores

Mouth sores are often the first sign of Behçet’s and occur in almost everyone with the disease. These painful ulcers, which can appear anywhere in the mouth, including the lips, tongue and cheek lining, look like common canker sores but can be far more abundant. Patients have reported dealing with multiple mouth ulcers at a time. Not surprisingly, they can make eating, drinking and even speaking difficult. These sores usually heal within a week or two without scarring but often return.

Genital Sores

Appearing in about 75 percent of people with Behçet’s disease, genital sores can occur almost anywhere in the groin area. These painful ulcers tend to be larger and deeper than mouth sores and, as a result, can lead to scarring. These sores will typically heal within two to four weeks but recur. While genital sores are not contagious, they can make sexual intercourse painful. Doctors can misdiagnose genital sores associated with Behçet’s disease as herpes.

Skin Sores

Between 60 and 90 percent of people with Behçet’s disease experience skin lesions beyond the mouth and genital areas. These lesions can manifest anywhere on the body as painful acne-like bumps or on the legs, arms, face and neck as red, tender nodules called erythema nodosum. These lesions typically heal within two to three weeks but, like mouth and genital ulcers, frequently return.

Eye Inflammation

More than half of people with Behçet’s disease experience a condition called uveitis, an inflammation of the eye that can result in pain, redness, blurry vision and increased sensitivity to light. Eye inflammation due to Behçet’s happens more commonly in younger people and men. While eye inflammation can be the first sign of Behçet’s in some patients, it may not appear until years later for others. If left untreated, repeated recurrences can lead to vision loss or blindness.

Joint Pain and Swelling

Joint diseases such as arthritis and spondylitis affect 50 percent of patients with Behçet’s disease, resulting in pain, swelling and stiffness in the knees, ankles, wrists and elbows. Spondylitis can cause pain in the low back or buttock. Joint pain and swelling can make it difficult to move and perform everyday tasks. Although bouts of joint pain and swelling typically last about days to weeks, as with most of the common symptoms of Behçet’s, it often recurs. Permanent joint damage, however, is rare.

Beyond these five most common symptoms, Behçet’s disease can affect the brain and spinal cord, gastrointestinal tract, large blood vessels, heart, lungs and kidneys. If you have Behçet’s and are experiencing a flare up of either a recurring or new symptom, consult with your doctor.

To learn more about the need for new treatment options for Behçet’s disease, read “Behçet’s Disease: The Key to a Breakthrough Is Medical Innovation.”

 

While some brides-to-be spend the week before their big day worrying about the weather or fitting into their dress, Danielle Kroft was dealing with a flare-up of her ulcerative colitis (UC). And a wedding dress wasn’t exactly going to make the situation easier.

“I couldn’t imagine being stuck in that dress for nine hours,” Kroft recalled. “So I was in my doctor’s office begging him to give me anything and everything to get it under control.”

This is what life is like for more than 900,000 people in the United States living with UC, an unpredictable inflammatory bowel disease (IBD) with symptoms that come and go over time. Some patients go years without symptoms; others have frequent flare-ups. In a survey, almost two-thirds of patients feel like UC controls their lives.

While more people are being diagnosed with IBD, Kroft believes that there is hope to accepting and managing the chronic disease. She has been reluctant to talk about her UC but is sharing her story for this year’s World IBD Day (May 19) to raise awareness so others can learn to better manage their disease.

A WEEK BEFORE HER WEDDING DAY, DANIELLE KROFT HAD A FLARE-UP OF HER ULCERATIVE COLITIS THAT THREATENED HER PLANS. PHOTO CREDIT: TARA WILEY PHOTOGRAPHY

A WEEK BEFORE HER WEDDING DAY, DANIELLE KROFT HAD A FLARE-UP OF HER ULCERATIVE COLITIS THAT THREATENED HER PLANS. PHOTO CREDIT: TARA WILEY PHOTOGRAPHY

Dinner Table Conversation Non-starters

When Kroft began noticing blood in her stool and experiencing abdominal pain at the age of 14, she didn’t tell anyone at first, thinking—or maybe hoping—that it would go away on its own. After all, the symptoms are hardly appropriate for the dinner table. But as the symptoms continued, Kroft eventually confided in her mother, who scheduled a doctor’s appointment. Five months, multiple doctor visits and a colonoscopy later, she was diagnosed.

“When you are 14, you don’t know what symptoms you should be watching for,” Kroft said. “Part of the reason that it took so many months to be diagnosed with UC was that I wasn’t sharing all the information about my symptoms with my doctors.”

This experience is common; in a survey, 45 percent of patients with UC regret not sharing more information with their doctors during visits. UC is most frequently diagnosed between the ages of 15 and 30, when patients may feel self-conscious talking about symptoms such as bloody stools, diarrhea and urgent needs to use the toilet.

When Kroft’s sister was diagnosed a couple of years later—genetic factors are thought to play a role in the disease—Kroft felt like she had someone close to her who could relate. But she also learned first-hand that no two people with UC have the same experience.

“Our food triggers, for example, are very different,” Kroft said. “I can eat bananas, but they upset her abdomen. I don’t do well with pancakes, but for her, pancakes aren’t a problem. And I really like pancakes.”

When you have a chronic illness, you need to take your medicine and build in time for blood work and doctor visits.

Things Only Got Harder

Being diagnosed with UC was just the start of Kroft’s lifelong struggle to control the disease, which is the best that she can hope for with no current cure. Instead of worrying about what shoes she was going to wear to school, Kroft had to focus on taking her medication every day or risk getting sick.

Sticking with her treatment plan was not easy. At one point, she was taking 26 pills a day—a mix of over-the-counter and prescription medications and supplements. UC treatment remains a trial-and-error process, and her first few medications didn’t work out. Her treatment plans have included enemas and foams that had to be administered rectally, which she recalls was as uncomfortable as it was embarrassing.

Things only got harder in college. Waking up at 4 a.m. each day for crew practice and the logistics of taking her medications stressed her out. “Where are you going to lay down to take an enema when you’re living in a dorm? Ask your roommate to leave because you have to do that? No way.”

At one point during her sophomore year, she had a month without any symptoms and convinced herself that she didn’t need her medication anymore. She stopped her treatment, which led to a really bad flare-up.

“I broke down emotionally at that point,” Kroft said. “I was young and didn’t take it as seriously as I should have. I didn’t make it a priority.”

AFTER TEN YEARS OF TRYING DIFFERENT THERAPIES, KROFT AND HER DOCTOR FINALLY FOUND A TREATMENT PLAN THAT HELPS HER MANAGE HER UC SYMPTOMS.

AFTER TEN YEARS OF TRYING DIFFERENT THERAPIES, KROFT AND HER DOCTOR FINALLY FOUND A TREATMENT PLAN THAT HELPS HER MANAGE HER UC SYMPTOMS.

Talking Poop

About ten years, multiple medications later, Kroft has finally found a treatment plan that works for her. She also landed a job she loves in public relations and married her high school sweetheart.

She still feels the need to be close to a bathroom during conference calls or while commuting. And she understands that’s always going to be a part of her life. For the most part, she’s feeling well.

“The biggest thing I have realized is that you need to take care of yourself,” Kroft said. “When you have a chronic illness, you need to take your medicine and build in time for blood work and doctor visits.”

Taking good care of yourself means learning more about your chronic disease and getting support, according to Kroft. She wishes she had asked her doctor more questions about the condition and had spent time reading the Crohn’s & Colitis Foundation’s website when she was first diagnosed. Recently, Kroft has begun attending seminars from the Foundation, where she has learned from other patients and experts. She’s presently most concerned with learning the effects, if any, of UC and its treatments on pregnancy.

Over the past 14 years, Kroft has also learned to be more open with people about her disease. She can’t believe she waited six years before thoroughly explaining what living with UC is like to her now-husband. “People are a lot more willing to talk about poop issues and cut you some slack than you might think,” Kroft said.

To learn how combining endoscopic and microscopic analyses is providing a more complete picture of UC, read “A Closer Look at Mucosal Healing in Ulcerative Colitis.”

 

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When Ahmet Varoğlu was diagnosed with beta-thalassemia at the age of four, his family knew nothing about the disease. But they would soon learn that it is a rare, inherited blood disorder for which there is no cure. Now 49, Varoğlu serves as President of the North Cyprus Thalassaemia Society and Vice President of the Evrensel Hasta Haklari Derneği (Universal Patient Rights Association) in Northern Cyprus. He is dedicated to sharing his story to help improve understanding and increase patient access to disease education and other resources. This International Thalassemia Day (May 8), Varoğlu reflects on his experience and the challenges still facing patients today.

Beta-thalassemia is caused by a genetic defect in the production of hemoglobin, a protein that carries oxygen to red blood cells throughout the body. The defect leads to fewer and abnormal red blood cells, which can cause chronic anemia. The treatment of anemia often requires frequent blood transfusions, which are associated with serious health risks such as iron overload and diabetes.

The years following his initial diagnosis were difficult. “No one knew how to treat the disease,” he said. “There were no medications or specialized doctors. We didn’t even have a hospital to go to for treatment.”

As a result, Varoğlu and his family had to search for answers on their own after his diagnosis. Not until the 1970s were pilot programs launched in Cyprus to drive public awareness, education and prevention efforts. Patients in Northern Cyprus also banded together with family members caring for those impacted by the disease to form a local thalassemia patient society. According to Varoğlu, since its development in 1978, the society has been instrumental in helping patients make informed treatment decisions, providing access to healthcare professionals and educating physicians about beta-thalassemia.

Today, national education and prevention programs for beta-thalassemia can be found in several countries, including Cyprus, as well as Greece, Iran, Thailand and the UK. These programs provide information about risk factors, offer detection services to identify carriers and implement measures to reduce the spread of disease. Local organizations together with larger advocacy groups, such as the Thalassemia International Federation, are also helping to improve disease awareness and patient care. However, more needs to be done.

Current treatment approaches help to mitigate symptoms but do little to target the cause of the disease. With numerous doctors’ appointments and a need for ongoing blood transfusions, treatment requires a significant time commitment and can be costly. In addition, life-altering complications such as heart disease, bone and liver abnormalities and growth deficiencies often accompany beta-thalassemia. Patients and their loved ones have to accept that the disease will require life-long management.

Varoğlu has come to terms with the fact that beta-thalassemia will forever be a part of his life, but he’s hopeful that research efforts will improve care in the future.

“I can’t say that we are in a bad position. There have been many improvements within the past few years. But there is still no cure,” Varoğlu said. “Being informed, sharing resources and asking questions are critical for improving the future of patient care. Thankfully, research is ongoing.”

Watch this video to learn more Ahmet Varoğu’s story and hear his hope for future beta-thalassemia patients.

By Zeba Khan, Vice President of Corporate Responsibility, Celgene

John Chahilu is a proud Kenyan who is living with multiple myeloma. John could have easily been another statistic—another preventable death resulting from cancer and other noncommunicable diseases (NCDs). Delayed cancer diagnoses and poor-quality treatment are all too common in low- and middle-income countries like Kenya. However, increasingly, multi-sector partnerships are taking action so that patients, like John, can have better health outcomes.

John is being treated by physicians at the Academic Model Providing Access to Healthcare (AMPATH) program and Moi University Referral and Treatment Hospital, which are working in partnership with the Kenyan Ministry of Health to create equitable access to health care.

The Ministry is currently advancing universal coverage through a National Health Insurance Scheme and a corresponding population health model that operates through a network of rural clinics focused on the provision of quality primary care and a strong referral network for more advanced or specialty cases.

AMPATH’s Oncology Institute has worked to improve diagnostic and treatment training for multiple myeloma. In 2010, physicians at AMPATH were surprised to have diagnosed five patients with myeloma. With more awareness and better diagnostics, that number has grown steadily to 34 new patients in 2012 to 60 new patients in 2017. The expected number of new multiple myeloma diagnoses in the country is about 800 per year.

ZEBA KHAN, VICE PRESIDENT OF CORPORATE RESPONSIBILITY AT CELGENE, PARTICIPATED IN A PANEL DISCUSSION ON “ENGAGING ACROSS SECTORS AND DISCIPLINES TO ADDRESS NCDS” DURING AN ACCESS ACCELERATED-HOSTED EVENT IN NAIROBI, KENYA.

ZEBA KHAN, VICE PRESIDENT OF CORPORATE RESPONSIBILITY AT CELGENE, PARTICIPATed IN A PANEL DISCUSSION ON “ENGAGING ACROSS SECTORS AND DISCIPLINES TO ADDRESS NCDs” DURING AN ACCESS ACCELERATED-HOSTED EVENT IN NAIROBI, KENYA.

In late March, in Nairobi, Kenya, John participated in a panel discussion on engaging across sectors and disciplines to address NCDs. John kicked off the panel with his own patient story, an example of the power of cross-sector collaborations to create new possibilities for patients.

The discussion was part of a larger meeting hosted by Access Accelerated, an initiative that has brought together over 20 biopharmaceutical companies to tackle the challenges of NCDs. The meeting brought together representatives from patient groups, civil society, government and the private sector to discuss the specific challenges of addressing NCDs in Kenya, as well as current and potential sustainable solutions.

The Access Accelerated meeting extended an important discussion that began earlier in the week at an event convened by the World Bank and the Kenyan Ministry of Health in which NCD stakeholders discussed strategies for realigning NCDs with Kenya’s Universal Health Coverage agenda.

Collaboration is key to making measurable progress on NCDs. We are working across health systems and sectors to advance sustainable solutions for patients.

John’s story and the discussion that followed emphasized how organizations across all parts of the health care sector—private and public, large and small—can help in the fight against NCDs, in Kenya and elsewhere, by working together.

This is the aim of Celgene’s partnership with AMPATH Oncology. Together, we have created the first and only multiple myeloma program in Kenya that educates patients and healthcare professionals. Through our continuing education programs for oncologists, we have helped to improve diagnostics so that patients are more likely to get accurate diagnoses and consequently, earlier treatment options.

John is one of the approximately 100 Kenyans with multiple myeloma who are seeing specialists through the AMPATH program. Hearing his story was a gratifying reminder of how global health partnerships are making an impact on patients’ lives.

Our partnership with AMPATH works within the current healthcare system to address a locally identified need and to improve multiple myeloma care in Kenya. Through the AMPATH program, Celgene works with Moi University Teaching and Referral Hospital in Eldoret, Kenya, providing additional training for oncologists who are already treating patients so that they can provide better care. This complementary approach was emphasized by several participants during the panel session.

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.

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.

At the meeting, it was encouraging to see so many stakeholders from different sectors come together and learn from one another. For example, the panel discussion featured patients, healthcare professionals, biopharmaceutical companies, non-profit organizations and Kenya’s Ministry of Health.

To meet the challenges of noncommunicable diseases in Kenya, and the rest of the world, we need to continue listening to diverse perspectives from all sectors and to find ways to complement and enhance what others are already doing.

As Celgene continues to support global health initiatives, patients will remain at the heart of everything we do. Our vision is for many more patients to have stories like John’s, in which they receive a timely diagnosis and appropriate treatments, regardless of where they live.

To learn more about how partnerships like Access Accelerated are strengthening the global healthcare system’s capacity for cancer care, read “Burden of Cancer Growing in Developing Countries.”