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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.”

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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.

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.

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:

Last year, chimeric antigen receptor (CAR) T cell therapy was recognized as the Advance of the Year in the American Society of Clinical Oncology (ASCO) Annual Report for its demonstrated benefits in certain blood cancers and its potential in many other tumor types. At this year’s ASCO annual meeting, the interest in CAR T cell therapy remains strong with the availability of more data for approved and investigational therapies.

“So far, the oncology community has greeted CAR T cell therapy with extraordinary enthusiasm,” said Dr. Jeremy Abramson, clinical director for the Center for Lymphoma at Massachusetts General Hospital. “We’ve had few effective treatment options for difficult-to-treat blood cancers like diffuse large B-cell lymphoma [DLBCL]. The newest data from ASCO continues to suggest that CAR T cell therapy may represent an important advance for some patients.”

DR. JEREMY ABRAMSON FROM MASSACHUSETTS GENERAL HOSPITAL BELIEVES THAT MANY QUESTIONS ABOUT CAR T CELL THERAPIES WILL BE ADDRESSED AT THIS YEAR’S ASCO ANNUAL MEETING.

DR. JEREMY ABRAMSON FROM MASSACHUSETTS GENERAL HOSPITAL BELIEVES THAT MANY QUESTIONS ABOUT CAR T CELL THERAPIES WILL BE ADDRESSED AT THIS YEAR’S ASCO ANNUAL MEETING.

A Long Time in the Making

The first investigational CAR T cells were developed over 30 years ago, using genetic engineering advances with the goal to reprogram a patient’s immune system to recognize and attack cancer cells. Early attempts were lackluster; the engineered T cells were slow to reproduce, died quickly and produced weak immune responses that weren’t effective at killing tumor cells.

Advances in genetic engineering tools and techniques, as well as a far better understanding of the human genome, have advanced this type of technology. Over the past five years, the number of clinical trials involving CAR T cell therapies has skyrocketed from just a handful to more than 180.

In 2017, the U.S. Food and Drug Administration approved the first two CAR T cell therapies — one for children with relapsed or refractory acute lymphoblastic leukemia and another for non-Hodgkin lymphoma in adults who have failed at least two other kinds of treatment.

These are just two examples of diseases for which CAR T cell therapy represents a radically different therapeutic approach.

“CAR T cells are specifically engineered to recognize, go after and attack the cancer cells,” Abramson said.

How CAR T Cell Therapy Works

CAR T CELL THERAPY BEGINS BY REMOVING A PATIENT’S T CELLS, WHICH FIGHT INFECTIONS IN THE BODY, THROUGH A BLOOD DRAW. THOSE CELLS ARE THEN SENT TO A MANUFACTURING SITE WHERE THEY ARE GENETICALLY ENGINEERED TO RECOGNIZE AND ATTACH TO ANTIGENS EXPRESSED ON CANCER CELLS AND SOME NORMAL CELLS. PATIENTS THEN RECEIVE CHEMOTHERAPY BEFORE THESE PROGRAMMED CELLS ARE RETURNED TO THEIR BODIES TO SEEK AND ATTACK CANCER CELLS. PATIENTS ARE MONITORED FOR SIDE EFFECTS AFTER CAR T CELL THERAPY.

Exploring Questions in Blood Cancers

While the first two CAR T therapies have been approved, Abramson notes that many questions remain, including CAR T cell therapy production, safety and longevity.

Scientists are still fine-tuning the process of creating CAR T cell therapies. For instance, studies continue around different ratios of two subtypes of T cells—CD4+ and CD8+ T cells— that may behave differently. That’s because CD8+ T cells have a cancer-killing effect, while CD4+ T cells produce chemical messages that boost T cell production. Finding the right ratio of CAR T cells created from these subtypes may impact the efficacy and safety of these treatments, according to Abramson. But the clinical significance of CD4:CD8 ratio remains unknown.

“Even the most effective therapies can only be administered if the toxicities can be identified and successfully treated and reversed,” Abramson explained. “We’re continuing to learn about potential toxicities with the different approved and investigational CAR T cell therapies, and how to optimally manage and prevent them.”

We are studying ways to make these therapies work better, designing more effective CAR T cells that may target different or multiple cancer proteins and combining them with other medications.

Adverse events that have been noted in trials of CAR T cell therapy include cytokine release syndrome (CRS) and neurotoxicity. CRS symptoms include fever, nausea, or headaches, and neurotoxicity symptoms include delirium, headaches and problems speaking. ASCO attendees will get a better understanding of the severity and timing of these and other potential safety issues as well as insight into paths for their prevention and treatment.

As for the durability of CAR T cell therapies, Abramson believes there’s work to be done.

“Less than half of patients with DLBCL who receive CAR T cell therapy are still in remission a year later,” he said. “We are studying ways to design more effective CAR T cells that may target different or multiple cancer proteins and learn how to combine them with other medications like checkpoint inhibitors or immunomodulators to see if we can enhance CAR T cell activity.”

Beyond the Blood

So far, CAR T cell therapies have only been approved for the treatment of blood cancers. The major challenge in solid tumors, such as lung and breast cancers, has been identifying a target that’s restricted to the tumor, so the CAR T cells don’t also attack the patient’s healthy cells.

CAR T cells kill healthy immune cells called B lymphocytes, for instance, as well as lymphoma cells, but patients can often do without those particular cells. But a treatment that attacked an entire organ — or organs — would have catastrophic effects.

According to Abramson, one potential way to get around the problem may be to create CAR T cells that attack only when they encounter a specific combination of targets. While a single protein might be shared by cancer and healthy cells, researchers are searching for patterns of multiple targets only found on cancer cells. Whether or not this tactic succeeds, Abramson is optimistic that scientists can find a way.

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

Dr. Abramson is a lead principal investigator for Juno and has consultant/advisory roles with Celgene.

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.

Last year, Americans took more prescription medicines than ever before due in part to efforts to improve adherence. In addition, the number of new medications approved more than doubled from 2016. But if you think the country is paying substantially more at the pharmacy as a result, you may be surprised.

Prescription medication spending increased just 0.6 percent last year, less than the rate of inflation and the lowest growth rate since 2012, according to a report published by the IQVIA Institute for Human Data Science. Murray Aitken, executive director of the IQVIA Institute, explains why prescription spending growth has remained in check, why growth in prescription spending isn’t necessarily a bad thing and what we can expect in the near future.

MURRAY AITKEN, EXECUTIVE DIRECTOR OF THE IQVIA INSTITUTE, BELIEVES HIGH GROWTH IN PRESCRIPTION SPENDING CAN REPRESENT GOOD VALUE IN TERMS OF LONGER, HEALTHIER LIVES.

MURRAY AITKEN, EXECUTIVE DIRECTOR OF THE IQVIA INSTITUTE, BELIEVES HIGH GROWTH IN PRESCRIPTION SPENDING CAN REPRESENT GOOD VALUE IN TERMS OF LONGER, HEALTHIER LIVES.

Why was the growth rate for prescription medication spending just 0.6 percent in 2017?

“That relatively low rate is due to patent expirations and lower cost generics, which are offsetting the growth from new therapies and price increases. In fact, a record-setting 1,027 generic medications were approved in 2017. If we look at retail and mail-order prescriptions, spending actually declined 2.1 percent last year, as we had more new injectable and infusible treatments than oral treatments last year. The prescription spending growth rate in 2017 is significantly lower than the 9 to 10 percent that we saw in 2014 and 2015, which were historically high levels of growth.”

What happened in 2014 and 2015 that caused those high rates of growth in prescription spending?

“Between 2014 and 2015, we saw the rise and fall of hepatitis C spending, as therapies that cured a significant number of patients were introduced. Since that wave of innovation, spending has grown more slowly.

“When innovative therapies are introduced that address an unmet need for many patients, spending will go up as we saw in hepatitis C. That is not a bad thing. That growth is supported by the value that innovative therapies provide in the form of longer and better lives. When we get an effective therapy for Alzheimer’s, for instance, we will see spending increase as we treat the millions of Americans to improve their lives and reduce other healthcare costs of caring for those with the disease.”

Prescription Drug Spending Increased Just 0.6% in 2017

How are the prescription spending growth rates in your report different from those that we see elsewhere?

“First, we are not basing our analysis on list price. We are looking at the revenue manufacturers receive after all the rebates, discounts, coupons and vouchers have been accounted for. Secondly, we are reporting on the total use of all medicines through all distribution channels, not just individual medications. Individual medication costs tend to make headlines but aren’t necessarily reflective of the overall market.”

Are patients paying more for prescription medications because of price increases?

“Over the past five years, patient out-of-pocket costs have actually declined by 15 percent to $8.69 per prescription on average in 2017. These lower out-of-pocket costs are due to higher usage of generics and manufacturers’ coupons.

“But patients’ out-of-pocket costs remain high for a small number of prescriptions. Patients paid more than $500 for about 0.2 percent of all prescriptions in 2017. These patients are usually those in the coverage gap of Medicare plans or the deductible phase of their insurance plans. They may get the same medicine for less at a different time of year.”

Patient Out-of-Pocket Costs Down 15% Since 2013

What is the outlook for prescription spending and patient costs over the next few years?

“While there are many uncertainties, including government policy, we don’t foresee any major disruptions over the next five years. So we’re forecasting spending growth to average between 2 and 5 percent per year. Growth will continue to be driven by innovation in various disease areas, but especially in oncology. That growth will continue to be offset by expirations of patents, which are designed to keep spending in check over the long term.”

To learn how medical innovation saves lives and has reduced health expenditures, read “When It Comes to Healthcare Costs, New Medicines Are the Solution, Not the Problem.”

More than 32,000 oncology professionals will soon gather in Chicago for the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting. This year’s meeting, with a theme of “Delivering Discoveries: Expanding the Reach of Precision Medicine,” promises to be the most exciting yet because researchers are further unraveling the different mechanisms by which cancer starts, grows and metastasizes, as well as how the immune system responds to it.

This progress is ushering in a new era of precision medicine, according to Wim Souverijns, Corporate Vice President of Global Marketing for Hematology & Oncology at Celgene. Much of the research Celgene will present at this year’s meeting includes updates on its chimeric antigen receptor (CAR) T cell therapy platforms, as well new data related its investigational pipeline across hematologic malignancies and solid tumors.

CAR T Cell Data Continue to Roll In

One area of personalized medicine with incredible potential in cancer is CAR T cell technology. This process involves a patient’s immune cells being collected, modified in a laboratory to recognize cancer cells and reinfused to attack the cancer.

At this year’s ASCO meeting, Souverijns is looking forward to seeing longer-term safety and outcome data for several clinical trials involving investigational CAR T cell therapies in relapsed or refractory patients with difficult to treat blood cancers like multiple myeloma and diffuse large B-cell lymphoma.

While these therapies come with great promise, their safety profile needs to be properly characterized to ensure appropriate use of these therapies, Souverijns explains. At ASCO he expects further insights about particular side effects such as cytokine release syndrome (CRS) and neurotoxicity.

“These longer-term data will help us to better understand these effects and how to manage them,” he said. “This will be crucial to realize the promise of these investigational treatments for patients.”

Three years ago, most patients were getting doublets, and people were questioning the need for triplets. Today, people are talking about quadruplets.

CAR T beyond Blood Cancers

The big hope is that CAR T cell therapies will be able to go beyond hematological malignancies and effectively target solid tumors as well. Preclinical data of CAR T cell therapies in solid tumors are emerging. The challenge, though, is that solid tumors often aren’t responsive to treatment simply because therapies physically can’t reach the tumor.

“CAR T also holds promise in solid tumors, but it’s going to be a much harder nut to crack from a technological and scientific perspective than with blood cancers,” Souverijns said. “The integration of Juno’s recently acquired CAR T cell science powerhouse with Celgene’s deep disease and cellular therapy expertise provides a great opportunity with these new investigational technologies for cancer patients.”

AT THIS YEAR’S AMERICAN SOCIETY OF CLINICAL ONCOLOGY MEETING, ATTENDEES WILL DISCUSS ADVANCES IN CANCER RESEARCH, INCLUDING ADVANCES IN CAR T CELL THERAPY AND COMBINATION THERAPIES BUILT ON THE FOUNDATION OF IMMUNOMODULATORS.

AT THIS YEAR’S AMERICAN SOCIETY OF CLINICAL ONCOLOGY MEETING, ATTENDEES WILL DISCUSS ADVANCES IN CANCER RESEARCH, INCLUDING ADVANCES IN CAR T CELL THERAPY AND COMBINATION THERAPIES BUILT ON THE FOUNDATION OF IMMUNOMODULATORS.

Triplets Becoming More Common in Multiple Myeloma

At last year’s ASCO conference, clinicians saw data combining immunomodulators with other therapies to treat diseases like multiple myeloma. Souverijns expects to see even more data from trials this year testing combination therapies. While these combinations used to focus on later lines of treatment when patients have exhausted other options, the new data are showing options for earlier patient segments as well.

More seems like it may be better as more triplet therapies are being approved and utilized, while even quadruplets are being tested now. This is something he expects will be discussed at length at the conference as doctors are realizing that adding new therapies to the foundation of an immunomodulator may drive better outcomes.

“Three years ago, most patients were getting doublets, and people were questioning the need for triplets,” Souverijns said. “Today, people are talking about quadruplet therapy. It’s amazing how quickly cancer care is progressing, making each ASCO more extraordinary than the last.”

For all the potential advancements, Celgene’s Vice President, U.S. Medical Affairs Teng Jin Ong, M.D., pointed to precision medicine at this year’s conference.

“Our increasing insight into the biology of cancer drives the discovery of new therapies that are targeting very specific cancer mutations and allow for greater improvements in outcomes for patients with such mutations,” said Dr. Ong. “We’ll remember 2018 as the tipping point for precision medicine and showing how it could work in practice.

To learn more about how CAR T cells may help immune cells identify cancer cells, read “Revealing Cancer Cells to the Immune System.”

 

When Ralph Hills was diagnosed with an aggressive blood cancer called acute myeloid leukemia (AML) more than three years ago, his doctor told him to get his affairs in order. He interpreted that statement as a polite way of saying that he didn’t have long to live. But a last-minute phone call from his doctor led him to the right treatment for his disease.

AML is driven by as many as 76 gene mutations. Approved and investigational therapies that target those mutations are opening up the possibility to tailor treatments for individual patients. As a preview to a Facebook Live event hosted by Celgene on Wednesday, April 18 at 9:30 a.m. EDT in recognition of the third annual AML World Awareness Day on April 21, Hills discusses how precision medicine is helping to save lives by sharing his journey with AML.

How did you learn that you had AML?

“In December 2014, I was 70 years old and experiencing back pain. I thought my doctor was going to schedule surgery, but instead, he told me to make an appointment with our local cancer center in Connecticut. A couple days later, my wife and I were sitting in a room with an oncologist who told me I had AML. He said that he was going to prescribe chemotherapy and that I should get my affairs in order. He also suggested that we get a second opinion.”

How did you react to learning you had AML?

“That meeting left me in a haze. I felt like I lost control of everything. We cried. I knew that the prognosis for AML was not good for someone my age, and I lost hope. But a few days later, we took my doctor’s advice about getting a second opinion and went to the leukemia department of Weill Cornell Medicine in New York City.

“Targeted therapy inhibits the activity of specific genes, proteins or antibodies that have mutated and are helping cancer cells to grow. This inhibition can slow or stop the growth of cancer cells.”

Targeted therapy inhibits the activity of specific genes, proteins or antibodies that have mutated and are helping cancer cells to grow. This inhibition can slow or stop the growth of cancer cells.

Targeted therapy inhibits the activity of specific genes, proteins or antibodies that have mutated and are helping cancer cells to grow. This inhibition can slow or stop the growth of cancer cells.

Did your treatment plan change after receiving the second opinion?

“It didn’t, at first. My new oncologist recommended the same decades-old standard of care for AML, which is an aggressive chemotherapy regimen. I knew the treatment would have harsh side effects and would not cure my AML.”

“The week before my treatment was scheduled to start, I visited a friend who was being treated with chemotherapy for his blood cancer. He told me that he was tired of fighting and ready to give up. I imagined that I was going to be in his position in a matter of months.”

What happened next?

“The night before my chemotherapy was going to start, my doctor called during her ski vacation. She asked me if I would consider changing my treatment plan. Apparently, I had a molecular mutation in a gene that might respond to a targeted therapy that was in clinical trials. So I had a choice of being treated with chemotherapy or an unknown treatment. I trusted my doctor and knew that she wouldn’t have offered it to me unless she thought it was the right treatment for me at that time.”

AML Awareness: A Patient's Perspective

Did you know that your doctor had ordered molecular profiling of your disease?

“No, we didn’t know at the time. She took my blood and bone marrow samples when we first met with her, but I thought that was just pretty standard routine tests. I didn’t know that she went the extra mile and ordered molecular profiling to see if I had any mutations. But that test was what qualified me for this trial.”

What was your treatment experience like?

“For six months, I could hardly move. I was in my bed unable to eat regular foods, getting all my sustenance from nutritional supplements. I lost 45 pounds.”

“After that, the bad leukemia cells began to disappear, leaving room for the healthy blood cells and platelets. Every two weeks, I went in for testing and watched the number of bad blood cells drop. Then I was told that I have indiscernible leukemia. The doctor told me to go home because she had sick people to see. That moment was the start of my second life.”

The idea to target the bad guys and avoid affecting other healthy cells makes sense to me.

What kept you going during your treatment?

“My dedicated wife, Dorcas, handled much of the physical and emotional load. She made phone calls and wrote newsletters to keep my friends and family up to date. They sent me get-well cards and flowers. I played card games with my grandchildren for five minutes at a time, which was all I could manage. But little things like that kept me going.”

How do you feel now?

“My life has changed in many, many ways. Like all cancer survivors, I visit my doctor for regular medical check-ups. I’m eliminating all the stuff that wasn’t important and enjoying every moment more. I’m semi-retired now. This summer, I spent a couple of months in Canada. In September, I will celebrate my third birthday after my diagnosis. That’s the way I’m treating all this — as a complete reset on my life.”

What do you hope the future has in store for AML patients?

“The idea to target the bad guys and avoid affecting other healthy cells makes sense to me. I’m delighted that my doctor did the molecular profiling to qualify me for the right clinical trial among the 7,000 that are ongoing in the United States so I could get the right treatment. I hope every person, young and old, rich and poor, gets the right treatment at the right time like I did. I feel like I am one of the very, very lucky ones.”

To learn more about the progress that is being made in the treatment of AML, join us for a special Facebook Live event with AML patient Ralph Hill on Wednesday, April 18, 2018 at 9:30 a.m. EDT.