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The current benchmark for efficacy in clinical trials of new plaque psoriasis treatments is a 75 percent reduction in the Psoriasis Area and Severity Index (PASI), which is a measure of the area of skin affected along with the skin’s appearance. But the most bothersome symptom, according to a survey of patients with plaque psoriasis, was not skin appearance, but itching.

As thousands of dermatologists gather at the 27th European Academy of Dermatology and Venereology Congress in Paris to discuss the latest data from ongoing clinical trials in psoriasis, Dr. Colby Evans, M.D., a dermatologist in Austin, TX, and also the immediate past chair of the Board of Directors of the National Psoriasis Foundation, discusses how researchers measure the effectiveness of treatments in clinical trials and how those measurements could be more comprehensive.

How do researchers measure the effectiveness of new plaque psoriasis treatments in clinical trials?

“Efficacy of plaque psoriasis treatments has immensely improved over the last 20 years, making more ambitious endpoints realistic. While a 75 percent reduction in PASI score has been the Food and Drug Administration’s benchmark, we see a growing interest in PASI 90 or 100 in more current trials. We want to get patients as close as we can to PASI 100. That being said, achieving a PASI 50 along with an improvement to quality of life is still clinically beneficial.”

DR. COLBY EVANS, M.D.

DR. COLBY EVANS, M.D., FROM THE NATIONAL PSORIASIS FOUNDATION BELIEVES THAT THERE IS ROOM FOR IMPROVEMENT IN THE MEASUREMENT AND GOALS OF EFFICACY IN PSORIASIS CLINICAL TRIALS.

Does PASI comprehensively capture all the clinical benefits of psoriasis treatments?

“PASI measures the size, as well as the level of scale, redness and thickness of psoriasis plaques. It does not capture other debilitating symptoms associated with psoriasis, such as psoriatic arthritis, itching or social stigma.”

How impactful are those symptoms on the lives of patients with plaque psoriasis?

“Itching can be debilitating and miserable, and can interfere with daily functioning. Survey data showed that people with psoriasis reported having a higher level of sleep-related problems compared to the general population.

Psoriatic arthritis is also of high concern among patients with psoriasis. Approximately 30 percent of plaque psoriasis patients develop psoriatic arthritis. It’s painful, can distort the joints and can be permanently disabling if untreated.”

What are the potential consequences of not capturing improvements in these symptoms?

“It’s important to take full measure of the patient and their life before you decide on treatment. PASI is a reasonable place to start, but it is more complicated than just the extent and thickness of their plaques. If you’re ignoring specific symptoms, patients can get left behind, and treatment decisions may be made without factoring in critical information.”

The more safe and effective treatment options we have, the better for patients with plaque psoriasis.

Do you measure PASI to make treatment decisions for patients who are not in clinical trials?

“In the real world, outside of trials, we don’t use a strict algorithmic treatment of psoriasis. In the regular clinical setting, I am more interested in knowing if the patient can live their life socially, occupationally and recreationally, and without feeling limited by their psoriasis. If they are, I know we’re on the path to success.”

How can clinical trials evolve to more comprehensively measure meaningful improvements for patients with plaque psoriasis?

“It’s becoming fairly common to have secondary endpoints that include itching and quality of life, which is progress. We’re seeing better management of psoriasis sub-types, such as patients with severe hand and foot psoriasis or patients who have severe arthritis without a lot of skin disease. Since the condition is quite diverse, there’s no one treatment for every patient. So the more safe and effective treatment options we have, the better for patients with plaque psoriasis.

With that in mind, many people who have had plaque psoriasis for decades aren’t aware of some of the new treatments. So a tremendous amount of patient education needs to be done.”

To learn more about treatment challenges that patients with psoriasis continue to face, read “Moderate Psoriasis Patient Needs Should Not Be Overlooked.”

Most patients diagnosed with lymphoma discuss the possibility of chemotherapy with a healthcare professional at some point. Chemotherapy is a standard of care for many forms of lymphoma, but most patients will experience multiple relapses.

Chemotherapy is a broad spectrum treatment that stops cell growth and division throughout the body, which can lead to side effects. Chemotherapies cannot differentiate between cancer cells and normal cells, so they also attack fast-growing but healthy cells, such as hair follicles and the cells lining the gut. That damage can lead to both short and long-term side effects, such as fatigue, nausea, a compromised immune system, fertility loss and an increased risk of infection or a second primary cancer.

While the benefits of chemotherapy often outweigh the risks, patients are eager for alternative solutions. Thankfully, research continues to look at different treatment pathways.

Meghan Gutierrez

MEGHAN GUTIERREZ, CHIEF EXECUTIVE OFFICER OF THE LYMPHOMA RESEARCH FOUNDATION (LRF), EXPLAINS WHY RESEARCHERS ARE EXPLORING CHEMOTHERAPY-FREE TREATMENTS

“We are learning a great deal about lymphoma subtypes and making progress in the discovery and development of new approaches that may improve quality of life,” Meghan Gutierrez, chief executive officer of the Lymphoma Research Foundation (LRF), said. “There is meaningful interest in exploring potential new treatments and combinations, many of which are chemotherapy-free.”

Lymphomas are caused by changes in immune cells called lymphocytes. In patients with lymphoma, the body makes many of these defective lymphoma cells that may not be detected by normal immune cells, which can properly fight infection and disease, including cancer. Restoring the immune system’s ability to fight cancer is a growing trend that has led to the development of immunomodulatory therapies, which can boost the tumor-killing cells of the immune system.

Investigators continue to explore new approaches focused on stimulating the immune system for patients with lymphoma.

The inherent ability of some types of immune cells to attack tumors relies on “tags” called antibodies on the surface of cancer cells. This killing process is known as antibody-dependent cell-mediated cytotoxicity (ADCC). In fact, several approved lymphoma therapies are antibodies that attach to cancer cells, leading the immune system to better identify and attack them. Researchers are now studying whether combining these antibody therapies with immunomodulatory therapies might further enhance cancer-killing ADCC, without the need for chemotherapy.

With further understanding of how both of these types of treatment work, separately and in combination, there is a potential to improve outcomes.

“Investigators continue to explore new approaches focused on stimulating the immune system for patients with lymphoma,” Gutierrez said. “It’s an incredibly exciting time as research is constantly evolving.”

Over the past few years, there has been important progress in treating certain blood cancers with chimeric antigen receptor (CAR) T cell therapy, leading to the approval of two of these novel treatments last year. But ensuring patients can access these innovative therapies remains a top concern for doctors, patient advocacy organizations and other stakeholders in the life sciences community.

Those challenges were the focus of a special panel sponsored by Celgene during the 2018 Biotechnology Innovation Organization (BIO) International Conference, which was held from June 4-7 in Boston. Richard Bagger, executive vice president of Celgene Corporate Affairs and Market Access, moderated the panel, which included experts with backgrounds as varied as medical research, economics and health policy.

“We are at the threshold of a very exciting time,” said panelist Nupoor Raje, Massachusetts General Hospital. “The progress that we are seeing is promising.”

This progress also comes with challenges and potential barriers for patients. The panelists agreed that those challenges should not prevent patients who could potentially benefit from CAR T cell therapies from having affordable access to them. Part of the discussion highlighted how a number of stakeholders will need to work together to shape the regulatory and reimbursement environment for optimal patient access.

Richard Bagger

RICHARD BAGGER, EXECUTIVE VICE PRESIDENT OF CORPORATE AFFAIRS AND MARKET ACCESS AT CELGENE, MODERATED A PANEL ON ENSURING ACCESS TO TRANSFORMATIVE CANCER THERAPIES AT THE 2018 BIO INTERNATIONAL CONFERENCE.

Ensuring Access

Operationally, the production of approved CAR T cell therapies is as complex as each treatment is highly personalized and unique to every patient.  A patient’s T cells are extracted at a treatment center, sent to a manufacturing facility where they are engineered to recognize a specific protein or antigen and attack cells, including cancer cells that have this antigen, and then sent back to be infused into the patient. The process requires an unprecedented level of collaboration between doctors, health centers and manufacturers.

One of the approved CAR T cell therapies is for pediatric patients with relapsed or refractory acute lymphoblastic leukemia. Both approved CAR T cell therapies are for adults with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of therapy.  Celgene CAR T cell therapies are still investigational and are not yet approved by the Food and Drug Administration.

However, a significant blockade to patient access exists—these personalized therapies don’t fit neatly into private payer or government payment models, including Medicare, meaning delays for patients and complications for providers.

The last thing we want to do is deny a patient a therapy that could potentially benefit them.

“Waiting two to four weeks for insurance approval could be too long for many of these patients,” panelist Dr. Gwen Nichols, the Chief Medical Officer of The Leukemia & Lymphoma Society, said.

And despite the financial losses that hospitals face when Medicare and insurance reimbursement does not adequately cover the costs of CAR T cell therapy, many centers continue to try to deliver timely treatments to meet patient demand, according to panelist Beth Roberts, a health policy lawyer at the firm Hogan Lovells.

As a possible solution to expedite patient access and simplify issues for providers, panelist Henry Grabowski, Ph.D., Professor Emeritus of Economics at Duke University, suggested that private and public insurers should explore innovative payment arrangements such as payment plans, flexible pricing and value-based contracts.

Patients Are Waiting

Beyond the issue of reimbursement, the health care system is still increasing capacity to provide CAR T cell therapies to more people. For instance, because of the highly specialized expertise required, only a few community cancer centers can administer CAR T cell therapies, so patients often must travel far for treatment.

Preparing today so patients can access innovative cancer treatments such as the CAR T cell therapies must be a top priority for our health care system. “We need to manage the hype and continue to let the science lead the way,” Raje said. “The last thing we want to do is deny a patient a therapy that could potentially benefit them.”

To learn why CAR T cell therapy represents an important advance for some patients, read “CAR T Cell Research Continues to Advance.”

While Blood Cancer Awareness Month provides an opportunity to raise awareness and understanding of the various forms of blood cancer, it also serves as a reminder of the progress that has been made in treating these diseases. This progress would not have been possible without the numerous individuals who are bound together to improve the lives of people diagnosed with blood cancers.

A blood cancer diagnosis significantly alters the lives of not only patients but also their caregivers, friends and family. In 2012, more than 900,000 people worldwide were diagnosed with blood cancer. In the United States, someone is diagnosed with blood cancer approximately every three minutes.

Explore a model “Day in the Life” of various individuals through the following infographics to better understand the effect blood cancer has on their daily lives.

Blood Cancer Awareness Month
Leukemia
Multiple Myeloma
MDS
lymphoma
myelofibrosis

 

As director of Patient and Non-Personal Promotions at Celgene in Seattle, Becky Holman works to ensure information is communicated to patients as clearly as possible. She knows that the work she does could potentially make a difference in the lives of patients with blood cancers, but hearing it from the patients themselves is something altogether different.

Recently, Holman had just such an opportunity when she and her Celgene colleagues were asked to read letters written by blood cancer patients for a video to mark Blood Cancer Awareness Month. It was a powerful reminder of the special bond that everyone who works at Celgene shares with patients.

“At Celgene, we are bound together with patients in this fight to find a potential cure for blood cancers,” Holman said. “We share a common goal. They are fighting for their lives, and we are fighting alongside them. Celgene and the blood cancer community are truly bound by blood.”

 

Ripple Effect

The letter that Holman read was from a man with non-Hodgkin lymphoma (NHL) named Nick, whom she’d met earlier this year in Celgene’s Seattle office. He had recently had his 100-day treatment follow-up after treatment on a clinical trial with an investigational therapy.

Nick is a firefighter who puts his own life on the line to save others, playing an essential role in his community. He had faced heat and flames—and now cancer—with bravery.

He is also memorable because of his age; while lymphoma is most commonly diagnosed among people who are aged 65 to 74, Nick is in his early 30s. He and his wife discovered they were expecting their first child after he found out that his cancer had come back following treatment with chemotherapy.

“Knowing Nick, I could already see his face while I was reading his letter,” Holman said. “He helped me understand the ripple effect of blood cancer therapies. If we can help him, he has a chance to continue to save lives in his community through his job as a firefighter. That’s an immeasurable impact.”

Every time that we meet patients or read a letter from a patient, we deepen our commitment to the people who are fighting blood cancer alongside us.

Constant Reminders

The bonds between Celgene and the blood cancer community run deep. Patients with blood cancer, like Nick, rely on the research community and companies like Celgene to continue researching new treatment options. Meanwhile, Celgene relies on patients to take part in clinical trials of those therapies to ascertain the safety and effectiveness so we can hopefully offer them to the blood cancer community.

Celgene’s promise to the blood cancer community is to always put patients first, and the company takes steps to ensure that everyone who works there never loses sight of that promise. While it’s easy to get caught up in the strategy and operations of a large company, Celgene employees embrace the opportunity to meet and engage with people like Nick whose lives have been affected by blood cancer.

“Every time that we meet patients or read a letter from a patient, we deepen our commitment to the people who are fighting blood cancer alongside us,” Holman said. “We’re reminded why we are here, why we’re doing what we’re doing. It keeps us anchored, focused, grounded. It gives us purpose and hope.”

To learn more about the special bond shared by Celgene employees and blood cancer patients, watch the “Bound by Blood” video.

Patients with plaque psoriasis who see their doctor frequently can increase their odds of finding an effective and appropriate treatment for them, especially as there are more ways to treat psoriasis than ever before. According to an analysis of the MarketScan Medicaid database, the average time between a psoriasis patient’s initial visit to the doctor’s office and a follow-up appointment is 153 days.

To raise awareness about why it’s important for patients to work with their doctors to help manage psoriasis during this year’s Psoriasis Awareness Month (August), Mark Jackson, M.D., Clinical Professor of Medicine (Dermatology) at the University of Louisville and Forefront Dermatology, explains how often patients with psoriasis should see their doctors, how psoriasis treatments have evolved and why it’s important for patients to feel empowered to help take control of their disease.

DR. MARK JACKSON

MARK JACKSON, M.D., FROM THE UNIVERSITY OF LOUISVILLE BELIEVES PATIENTS WITH PSORIASIS SHOULD SEE THEIR DOCTORS FREQUENTLY TO HELP ENSURE THE BEST POSSIBLE OUTCOMES.

How often do you recommend your patients with psoriasis see you?

“Plaque psoriasis requires active management. It’s essential that doctors make sure that their patient’s condition is improving within four to six weeks of starting a new treatment for them. Even if their current treatment is effectively controlling their symptoms, they should still see their doctors every three to six months. Seeing their physician frequently is imperative for patients with psoriasis.

That being said, if a patient is experiencing new symptoms or treatment side effects, they should call their doctor immediately.”

Why is it important that people with psoriasis see their doctors frequently?

“Psoriasis is not just a rash; it’s an inflammatory disease. Patients with psoriasis are more likely to have other health conditions, including metabolic syndrome, diabetes, heart disease and obesity. Up to 30 percent of psoriasis patients eventually develop psoriatic arthritis as well.

During office visits, doctors check the signs and symptoms of psoriasis and its comorbidities. Dermatologists are not just skin experts; we’re physicians. We can optimize our patients’ overall health and if we can assist in controlling the inflammation from psoriasis, we might possibly be able to help reduce the risk of these other conditions as well.”

What are some symptoms that you tell your patients to look out for?

“For most patients, psoriasis symptoms can fluctuate on a seasonal basis or with life changes. Cold, dry weather and stress can both trigger a psoriasis flare, and certain medications can make some symptoms worse.

Because of the increased risk of developing psoriatic arthritis, I make sure my patients are on the lookout for any joint pain and swelling. Patients should also know and watch out for the signs and symptoms of other comorbid conditions.”

The more that people with psoriasis take charge of their overall health and treatment, the better their outcomes can be.

How can doctors encourage their patients to see them more frequently?

“Doctors should foster a partnership with their patients through regular communication. The best outcomes occur if patients and doctors are both engaged and involved in their care. The more informed a patient is, the better decisions they make and the more compliant they can be with treatment.

Some patients may get scared when they learn about the risks that come with some systemic psoriasis treatments. It’s our job as physicians to thoroughly explain to our patients the risks and benefits of all their options and address their concerns, so we can make the most appropriate choice together.”

How have psoriasis treatments evolved over the past decade?

“We’ve seen a dramatic increase in the number of available psoriasis therapies as well as the efficacy and tolerability of those treatments. Newer treatments have become much more targeted compared with many of the older systemic medications, which cast a wider net on the immune system.”

Why is it important that patients feel empowered to manage their psoriasis?

“We can’t make patients take their medicines or schedule appointments, but we need to work to make them engaged participants. We can only provide the best information and try to build their confidence with the hope of helping improve their treatment compliance.

I encourage my patients to get involved with the National Psoriasis Foundation and learn more about their disease. My patients don’t always love hearing about diet and exercise, but I try to explain to them that their therapy may be more effective if they maintain a healthy body weight. The more that people with psoriasis take charge of their overall health and treatment, the better their outcomes can be.”

To learn more about how comorbidities can affect the health and care of people with psoriasis, read “Painting a Clearer Picture of Psoriasis Comorbidities.”

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While innovative therapies have helped improve the relative survival rate for patients with multiple myeloma, the disease remains incurable with most patients experiencing repeated relapse and ultimately becoming refractory to treatments. Today, research continues to delve into whether treating relapsed multiple myeloma with combination therapies may be an appropriate option for certain patients. More agents could also result in increased toxicities and patients must be monitored closely.

­At the 54th Annual Meeting of the American Society of Clinical Oncology (ASCO), new data regarding the use of triplet therapy for relapsed multiple myeloma were presented. Now four or more drug combinations are being examined as well. In this interview, Dr. Paul Richardson, the RJ Corman Professor of Medicine at Harvard Medical School and clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute, discusses the new data presented at this year’s ASCO meeting and how the future of relapsed multiple myeloma treatment could look.

DR. PAUL RICHARDSON

DR. PAUL RICHARDSON FROM DANA-FARBER CANCER INSTITUTE BELIEVES THAT WE WILL CONTINUE TO SEE PROMISING RESULTS FROM COMBINATION THERAPIES FOR RELAPSED/REFRACTORY MULTIPLE MYELOMA.

Why have triplet and quadruplet regimens become more frequently used to treat relapsed multiple myeloma?

“Multiple myeloma is a heterogeneous disease, which means that patients typically have multiple subpopulations of malignant plasma cells with different molecular profiles and characteristics. When patients with multiple myeloma are given a single therapy, some of these subpopulations recede while others may grow. This phenomenon is believed to contribute to treatment resistance and eventual relapse.”

“So the theory that combination therapies provide a multi-pronged attack to target multiple myeloma subpopulations may be a reason why we are seeing more relapsed multiple myeloma patients being treated with combination therapies. Quadruplet regimens are also being investigated for high-risk patients with particularly resistant multiple myeloma.”

How have data presented at this year’s ASCO meeting moved the field forward?

“At ASCO, we saw new data from clinical trials comparing triplet with doublet regimens for relapsed multiple myeloma. Those findings support the idea that if you throw a wider net around the illness early with a triplet, you may achieve greater clinical benefit. At the same time, as we continue to explore new combinations, we are finding more regimens that may be better tolerated by patients.”

Why do many of these combinations include immunomodulators?

“Immunomodulators are an integral part of multiple myeloma treatment in both the newly diagnosed and relapsed/refractory setting. These therapies have numerous effects on tumor cells, the immune system, and the tumor microenvironment which we believe leads to their anti-multiple myeloma activity. In fact, they constitute a foundation for therapy regimens in multiple myeloma.”

“At this year’s ASCO, we saw important new data from trials exploring the interactions between these therapeutic classes in the relapsed setting.”

Although the treatment of relapsed multiple myeloma remains an unmet medical need, we’re making progress. The data presented at ASCO on combination therapies and potential breakthrough approaches like CAR T cell therapy are particularly exciting.

How are researchers taking a more precision-focused approach to treating relapsed multiple myeloma?

“Choosing the most appropriate therapy based on the genetic causes of a disease makes sense but is challenging in multiple myeloma, because the cancer is so genetically unstable. For example, we sequenced the genome of one patient at diagnosis and identified over 5,000 disease-associated mutations. By the time he relapsed, we had found more than 12,000. When you have that number of genetic changes, targeting a single mutation will be unlikely to make much of a difference on its own.”

“To help address this problem, we have to take a modified precision medicine approach. Researchers are studying whether combining backbone agents such as immunomodulators and proteasome inhibitors that have broad success in the disease with more targeted agents and evaluating these combination regimens, and in particular with monoclonal antibodies.”

What are your thoughts on the future of relapsed multiple myeloma treatment following this year’s ASCO meeting?

“Although the treatment of relapsed multiple myeloma remains an unmet medical need, we’re making substantial progress. The data presented at ASCO on combination therapies and potential breakthrough approaches like CAR T cell therapy are particularly exciting. While the FDA approved CAR T cell therapies have already shown promise in other cancers, multiple myeloma is a much tougher nut to crack. So we’ll have to wait and see what happens with these trials in the longer term, but early data are encouraging.”

To learn more about the advances discussed at ASCO 2018, read “ASCO 2018 Preview: Precision Medicine, CAR T Cells and Immunomodulators.”

Dr. Richardson regularly provides input as a paid consultant for Celgene.

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, Chairman and Chief Executive Officer, Celgene Corporation

Americans want and deserve better health care.1  Yet many patients are also concerned about the affordability of health care, including out-of-pocket costs which are rising faster than health care spending.2  When it comes to health care, we should not have to choose between access, quality and affordability. This is especially important for those patients suffering with life-threatening diseases who may only be able to be effectively treated with the newest, most innovative medicines. For the benefit of all patients, we must find ways to ensure that American health care improves access and quality, while becoming more affordable over time.

Mark Alles

Mark J. Alles, CELGENE CHAIRMAN AND CHIEF EXECUTIVE OFFICER

Celgene has long held to a set of principles used to guide our decisions about the pricing of the medicines we discover, develop and distribute worldwide. These principles reflect our commitment to patient access, obligation to provide value for patients and the health system, drive for continuing innovation for the future, and the need for flexibility. Today, we are enhancing these principles by setting forth an approach to price increases reflecting medical inflation and increased value. This approach is intended to continue to provide transparency around the value of our therapies and contribute to policy solutions that support access and affordability for patients.

First, if Celgene increases the price of any individual therapy across our portfolio, the price increase will be limited to no more than once a year and at a level no greater than the Centers for Medicare and Medicaid Services projected increase in National Health Expenditures for the year. For 2018, this rate is 5.3%. Because value is a guiding principle of our pricing decisions, there may be exceptional circumstances in which additional clinical or health economic evidence demonstrates a clear and significant increase in the value of one of our medicines where this standard would not apply. We believe this action will provide greater certainty for all stakeholders and contribute to limiting the growth of health care spending.

Second, we agree that transparency about value and pricing is important. Patients, healthcare professionals and policymakers are asking for more information, and we are committed to providing it. That’s why earlier this year we introduced Celgene’s first annual Value and Innovation Framework Report, offering comprehensive evidence on the value we provide to patients, the health system, the economy and society, and future innovation. We also plan to provide information related to any increases in price, including enhancements in value, at: www.celgene.com/value.

Third, we are committed to advocating for public policy solutions that will improve patient access and affordability, encourage value-based payment models, and expand competition.

Increasingly, the health care system is pushing more and more of the cost of care to patients, especially those with serious medical conditions, in the form of high deductibles and percentage-based coinsurance.3  We believe that limits on patient out-of-pocket spending will improve access and affordability and lead to greater treatment adherence, better health outcomes and lower overall health system costs. It is positive to see proposals to include a patient out-of-pocket cap in the Medicare Part D drug benefit included in the President’s Blueprint to Lower Drug Prices.4

Celgene supports value-based payment models, which, when properly designed, can provide access for patients, predictability for payers, and incentives for continued innovation by biopharmaceutical companies. The development of new and bolder value-based payment approaches can be facilitated through necessary modernization of government regulations to accommodate value-based arrangements. We applaud the Department of Health and Human Services’ interest in identifying regulatory solutions that would enable the evolution of these new payment models and ensure that access and reimbursement innovation keeps pace with medical innovation.

Finally, Celgene supports the robust balance between incentives for innovation and generic competition. The Hatch-Waxman Act has created a sustainable system where nearly 90 percent of prescriptions every year are generics, representing massive price reductions compared to the original innovator brand.5  Last year, the Food and Drug Administration approved a record-high 1,027 new generic medications.5  Between 2018 and 2022, based on anticipated loss of exclusivity, an estimated $105 billion in price decreases will be realized by the healthcare system.5  Policies that enhance competition after innovators’ patents expire drive the virtuous cycle of incentives for new innovation and the long continuing value from generic competition.

At Celgene, our long-standing purpose is to change the course of human health through bold pursuits in science and a promise to always put patients first. After spending many years and significant resources to discover and develop innovative new therapies, our greatest priority is ensuring that patients have access to them. Affordable access for patients is essential to our ability to reinvest in research and development that will lead to the next generation of treatments — and ultimately cures — for diseases that affect millions of people worldwide.



1 Blendon RJ, Benson JM, SteelFisher GK, and Weldon KJ. Report on Americans’ Views on the Quality of Health Care. Harvard School of Public Health. March 22, 2011. https://www.rwjf.org/en/library/research/2011/03/report-on-americans–views-on-the-quality-of-health-care.html?cid=XEM_807207 Accessed June 2018.
2 Consumers for Quality Care Survey Finds Americans Acutely Worried about Health Care Costs. Consumer for Quality Care. April 25, 2018. https://consumers4qualitycare.org/research/ Accessed June 2018.
3 Commercially-Insured Patients Pay Undiscounted List Prices for One In Five Brand Prescriptions, Accounting for Half of Out-of-Pocket Spending on Brand Medicines. Phrma. March 2017. http://phrma-docs.phrma.org/download.cfm?objectid=C6A51770-0FCD-11E7-ACCC0050569A4B6C Accessed June 2018
4 U.S. Department of Health & Human Services. American Patients First: The Trump Administration Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs. May 2018. https://www.hhs.gov/sites/default/files/AmericanPatientsFirst.pdf. Accessed June 2018.
5 Uhl K. 2017 Was Another Record-Setting Year for Generic Drugs. FDA Voice. 2018.
https://blogs.fda.gov/fdavoice/index.php/2018/02/2017-was-another-record-setting-year-for-generic-drugs/ Accessed June 2018.

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.