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Celgene is a company built on a foundation of bold innovation to address areas of significant need for patients with cancer and other debilitating diseases. This unyielding drive has led the company to developments that have transformed the care of diseases like multiple myeloma and pancreatic cancer and has provided important new options for patients with psoriatic diseases.

As the company has grown, so has its commitment to innovation. In fact, the company has increased its investment in research and development by more than 36 percent per year on average since 2006, when its lead therapy for multiple hematologic diseases was approved. During this time, the company also built what would be a defining part of its research efforts, the distributed research model.

By coupling Celgene’s internal research and traditional business development efforts with a program focused on identifying and nurturing disruptive science outside the company, Celgene was able to option promising candidates for rare and debilitating diseases. The program currently features more than 50 collaborations with 21 unique compounds in clinical development.

Robert Hershberg

ROBERT HERSHBERG, M.D., PH.D., CELGENE’S EXECUTIVE VICE PRESIDENT AND HEAD OF BUSINESS DEVELOPMENT AND GLOBAL ALLIANCES.

“We are focused on great science, first and foremost,” said Robert Hershberg, M.D., Ph.D., Celgene’s Executive Vice President and Head of Business Development and Global Alliances. “We seek in our partners what we seek in our own scientific and development teams. Notably, we seek passion, a commitment to excellence, and a strong desire to bring novel treatments to improve patients’ lives.”

“The distributed research model—in place at Celgene for almost a decade—recognizes and embraces the fact that contributions to progress in human health can be readily seen in academic institutions, private foundations, small and large biotechnology companies, and in the pharmaceutical sector,” he continued. “Importantly, no single institution, company or entity can do this alone and there is an increasing interdependence on a range of efforts to bring this promise to patients. We seek partners in our core areas of scientific interest (Protein Homeostasis, Immuno-Oncology, Epigenetics, Immunology/Inflammation, and Neuroscience) and clinical interest (Hematology, Oncology, Inflammatory diseases) and hope to identify programs in adjacent, novel areas as well.”

One of the keys to the growth of the programs was to identify and incentivize this innovation early on.

“As products increase in both their complexity and their precision, intense support early in the development process is critical. The establishment of relevant pre-clinical models and deep interrogation of novel pathways provide the appropriate roadmap for moving early science forward towards the clinic,” continued Hershberg. “In early studies of these novel therapies in patients, an intense focus on ‘translational’ medicine—developing tools to gather as much data as possible in early clinical trials. These early efforts both improve the likelihood of clinical success and can dramatically reduce the timelines required to bring novel therapies to patients that desperately need them.”

Along with groundbreaking research, these partnerships also provide the opportunity to learn critical lessons in discovery research through new platforms for Celgene, and a chance to advance programs alongside an industry veteran for the partner companies.

An important example of this was the partnership with Agios Pharmaceuticals, Inc. The long-term research partnership provided the opportunity to evaluate multiple programs, to adjust the terms to fit each company, and most importantly, to deliver the first FDA-approved product to come out of Celgene’s distributed research model.

“Close collaboration from the very start has been key to the success of our relationship with Celgene” said David Schenkein, M.D., Chief Executive Officer of Agios. “By focusing on each other’s strengths, we were able to pursue the science and develop a new medicine, less than four years from the first in human study to approval in a blood cancer that had not seen a new medicine in nearly 40 years.”

Hershberg has a unique perspective on partnering with Celgene as he has now served on both sides of the model, in his current role as head of the company’s business development efforts, and as a partner during his time as CEO of VentiRx.

“Celgene has been on the leading edge of business development to engage external partners to extend our Research and Development footprint. Importantly, many of these partnerships allow and facilitate a partner’s ability to do what they do best—and to advance programs into early and mid-stage clinical development. The business structures are flexible and ideally designed to meet both Celgene’s and the partner’s unique needs,” said Hershberg.

Another long-term partner was recently in the news as well. Acceleron Pharma, alongside Celgene, announced top-line results from multiple pivotal studies of a collaboration that had been ongoing for more than ten years.

“Our collaboration is focused on developing and delivering transformational therapies to patients in areas of disease with few options,” said Habib Dable, President and Chief Executive Officer of Acceleron. “Because of our mutual commitment to this focus, we have advanced to the point where we are preparing for potential approval. Our shared experience has kept the combined team energized and on mission throughout.”

The close collaborations that make up Celgene’s distributed research model have bolstered a leading biopharmaceutical pipeline for Celgene, provided vital, early support for its partners’ promising programs and even delivered a new therapy to patients in need.

“The opportunity to partner early and leverage our research platform to identify potentially disruptive therapies has led to the opportunity to expand our collaboration twice already to encompass new targets and new areas of disease,” said Werner Lanthaler, Chief Executive Officer of Evotec AG. “We believe our work together across these multiple platforms have the potential to make meaningful impacts on patients’ lives and we continue to partner closely to make this happen.”

Celgene will continue to look for transformational science both within and outside of its walls as it seeks to deliver on its mission to improve the lives of patients worldwide. Successful partnerships, like those it has fostered already, will be essential in that effort.

Celgene has long maintained a dual focus on not only the patients that its therapies treat but the employees at Celgene that work to develop those therapies and get them to market. In October, Celgene was ranked #9 on the Forbes’ World’s Best Employers 2018 list. This year Forbes also gave Celgene placements on the 2018 Global Growth Champions list and America’s Best Midsize Employers 2018.

Celgene Values WallTogether with Statista, Forbes World’s Best Employers list was compiled from an analysis of more than 430,000 global employee recommendations. Along with 30 detailed questions, respondents were asked to rate their own employer and the likelihood they would recommend their company to a friend or family member. They also listed other companies they admired.

Among the qualifying factors, Celgene has continued expansion of Diversity and Inclusion initiatives which likely contributed to its high ranking. Parallel with these efforts, Celgene recently announced its participation as a founding member of the Healthcare Businesswomen’s Association’s Gender Parity Collaborative where several Celgene leaders now sit on the collaborative’s Gender Parity Council.

“Our ranking as one of the best employers in the world highlights Celgene special culture and the direct connection every colleague has to our patients,” said Joe Hand, EVP, Global HR and Corporate Services. “As our global organization evolves, we continue to look for innovative and genuine approaches to enhance our employee experience and attract and retain talented people.”

Celgene Change Makers - Boudry 2017Celgene leadership also places a heavy focus on mentorship, volunteerism and employee inclusion groups. Women Advancing Leadership at Celgene and Celgene Pride Alliance are employee resource groups that offer employees opportunities to connect and network. Supported by Executive Sponsors who are members of the Celgene Executive Committee, these resource groups host events and campaigns that bring together Celgene employees in an inclusive and safe environment to advance the dialog of social awareness and acceptance and drive necessary change.

In 2018 Celgene employees also saw enhancements to many U.S. employee benefits including Work-Life benefits like increased paid parental leave, a “bridge back to work” policy that allows new parents to transition back to work on a part-time basis but with full pay, and a policy for flexible work arrangements, to name a few.

“We felt it was important that our policies matched our commitment to creating an inclusive environment for our employee base,” said Joe Hand. “We’re proud to provide more balance between employees’ personal and professional lives to enable our workforce to bring their best self to work at Celgene and to their families and loved ones at home.”

Celgene Named Among World’s Best Employers 2018 - Run

To learn more about Celgene continued progress toward Diversity; read our official Diversity Mission Statement.

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.

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.”

Over the past decades we have developed an intuitive understanding that better patient involvement in clinical trials delivers better patient outcomes.

Patients' Partners logoFrom involving representative patient populations in trials through to empowering patients to manage their own care, the impact of not listening to patients’ lived experience has resulted in significant change.With the benefit of experience, historical shortcomings have been rectified and opportunities have been identified to involve patients across the entire patient pathway in a more collaborative, less transactional way.

Patient organisations and their representatives offer unique insights through their lived experiences, but are often under-represented in medicine discovery, development and discussion. Celgene, with its Patient Partners is now seeking to redress this balance with the ChangeMakers programme.

But what is a ChangeMaker? A ChangeMaker is an individual, organisation or movement that supports and advocates for improvements in the world around them, using their lived experiences and expertise as the basis for this change.

Putting patients at the heart of everything we do is not only one of our company values, it is in our very DNA as a business

The Celgene ChangeMakers mission is to recognise and amplify the voice of patient organisations. We believe that by working with patient organisations to qualify and articulate the key opportunities for patient involvement we can provide a catalyst for change.

To provide an evidence base for the need for change, a Steering Group comprised of Celgene and patient organisation representatives reviewed the evidence and identified the key areas where patient organisations demonstrate the most value in the delivery of good healthcare.

The ChangeMakers Goals reflect the key areas where increased patient engagement can lead to the most positive changes for healthcare delivery. Celgene and patient organisation representatives reviewed evidence from over 100 clinical studies and other existing programmes to better understand and demonstrate the value of the patient voice. This evidence formed the foundation for the Celgene ChangeMakers Goals.

The Goals provide an evidence-based framework for achieving the four principle ChangeMakers Visions:

  • All clinical trials conducted in Europe will incorporate the patient perspective as early as possible. They will be designed and developed in partnership with patients and patient representatives, ensuring the real-world concerns voiced by patients are at the centre of scientific research
  • More patients to be aware of and participating in biopharmaceutical clinical trials across Europe
  • Patients have access to the right treatment and care at the right time
  • Every patient is empowered to make decisions related to their own health and care through active participation and increased understanding

“Putting patients at the heart of everything we do is not only one of our company values, it is in our very DNA as a business,” said Mark Alles, Chief Executive of Celgene. “ChangeMakers is a programme that strives for constant improvement and change. A Celgene ChangeMaker is never satisfied with the status quo, or resigned to accepting things because effecting change may be too complicated or take too much time.

“A ChangeMaker is an individual, organisation or movement that supports and advocates for improvements in the world around them, using their lived experiences and expertise as the basis for this change. We wanted to recognise this behaviour and its impact with our patient representative attendees, providing a forum during this year’s Summit to celebrate achievements to date and a framework to increase the visibility and voice of patients in the future.”

The ChangeMakers Goals were launched to over one hundred patient organisations from across Europe for the inaugural ChangeMakers Summit, part of the Celgene Patient Advocacy Framework, Patients’ Partners. This year’s Summit then saw not only patient representative delegates, but all of Celgene’s Senior Management team, led by Mark Alles, sign up to the Goals.

Once the Goals were officially launched to the delegation, the focus of the Summit then considered what actions are needed to achieve the Goals. Commitments also came from Celgene Senior Management on the steps the company will take to ensure patients feel more empowered and engaged in our activities and in their own health. To support this change and progress, the Summit also included capability sessions delivered by Celgene to share knowledge and insights with delegates from topics ranging from effective communications to building high impact teams.

The Goals are just the first step on the journey – an evidence-based framework for change and progress. Areas of joint responsibility for working together to secure broader commitments by listening to and actively involving the patient voice will be identified. Celgene ChangeMakers has created a powerful community of patients and patient organisations who are dedicated to improving the patient voice across the patient pathway. We will continue to work with our Patient Partners and ChangeMakers to uphold the Goals and our commitment to achieving them.

“The Celgene ChangeMakers programme is just the beginning,” said Anita Atema, Executive Director for Patient Advocacy for Europe and International Markets. “We now have our vision for change and concrete steps to get there. Now we need to make a concerted effort, with our patient partners, to keep up the momentum and ensure patients’ voices are heard as they should be.”

For 25 years, Dr. Linda Burns, MD, cared for patients with acute myeloid leukemia (AML) at the University of Minnesota in Minneapolis. She strived to give patients the best care possible. But too often she made decisions based on what treatments her patients’ insurance would cover.

Now, Burns is furthering the conversation about the value of AML treatments. Her goal is to find opportunities to reduce costs without restricting access to appropriate treatments.

“AML is an aggressive disease, and patients will die in weeks or months without proper treatment,” said Burns, who is now vice president and medical director of Health Services Research at the National Marrow Donor Program/Be The Match in Minneapolis. “If a treatment can help these patients, it is creating value for those patients. Cost alone should not be guiding treatment decisions.”

Dr. Linda Burns, MD, vice president and medical director of Health Services Research at the National Marrow Donor Program/Be The Match, believes that costs alone should not guide treatment decisions for AML patients.

Dr. Linda Burns, MD, vice president and medical director of Health Services Research at the National Marrow Donor Program/Be The Match, believes that costs alone should not guide treatment decisions for AML patients.

In reality, even those costs are not well understood. Physicians and patients lack access to the prices of treatments and procedures. To shed light on the costs for AML treatments, Burns and her colleagues analyzed the Truven MarketScan database of private insurance claims from 2007 to 2011 for patients aged 50 to 64 who were treated with either chemotherapy or chemotherapy and stem cell transplantation. Their recently published findings demonstrate the expense involved in treating this rare disease.

There are two primary treatment options for AML: chemotherapy alone or in combination with a stem cell transplant. While stem cell transplants cure more patients than chemotherapy alone, the procedure in combination with chemotherapy costs over $540,000 on average.

Patients who undergo this aggressive treatment option need lengthy hospitalizations that drive up the total costs. On average, patients had five hospitalizations, 92.5 inpatient days and 74.5 outpatient visits.

Cost of Care for Acute Myeloid Leukemia

“The treatment weakens the patient’s immune system and their ability to fight infections, so they need to be hospitalized,” Burns explained. “And we know that hospitalization is very, very expensive.”

The study provided a price benchmark for AML treatment costs. Now, further information about how the treatment improved or extended the patient’s life is needed to understand the value that those treatments create for patients. To this end, Burns and her colleagues are linking cost data to patient health and outcomes information.

It’s important that we understand the value of AML therapies within the current treatment landscape and ensure patients have access to them as well.

The question of value will only grow in importance as new options for AML become available. Progress in AML treatment has been slow over the past 40 years, but clinical trials are exploring therapies that target specific molecular genetic changes in the disease. As we continue to better understand AML and can more precisely target the disease, new treatments have the potential to improve outcomes and, therefore, may help to relieve burdens on the healthcare system.

“Bring them on,” Burns said. “We need new treatments, and targeted therapies would add to the care we can provide to AML patients. It’s important that we understand the value of all AML therapies within the current treatment landscape and ensure patients have access to them as well.”

By Mark J. Alles

A decade ago, receiving a medicine designed for your specific genetic makeup or modifying your own immune cells to fight cancer may have seemed like something out of a science fiction novel. But today, “precision” medicines — tailored therapeutics based on a patient’s distinct genetic characteristics — are turning fiction into fact for many patients.

Since every person is unique, not only do precision medicines have the potential to bring highly effective therapies and high-value care to patients, they can also lower the overall cost of treating many of the most serious diseases. The investments needed to discover and develop these medicines can substantially improve health outcomes, and reduce the cost of failing to appropriately target treatment, estimated to be tens of billions of dollars every year.

Tailoring medical treatment to the profile of each patient can enable physicians to identify the best course of treatment and often avoid or reduce adverse drug reactions and the toxic effects of medicines that may not be necessary.

Mark Alles

Mark J. Alles serves as Celgene’s Chief Executive Officer.


For instance, according to a recent study published by JAMA Oncology, genetic profiling can predict which women with early-stage breast cancer have a lower risk for their cancer coming back after surgery, allowing up to 15 percent of patients to avoid unnecessary chemotherapy.

Most importantly, precision medicines can help patients live longer, healthier lives. Already, the first wave of precision medicines have entered mainstream clinical practice, including targeted therapies that now make it possible for patients with a once incurable form of leukemia, chronic myelogenous leukemia, to live close-to-normal life spans. Similarly, precision medicines are dramatically changing the treatment landscape for deadly cancers like non-small cell lung cancer and metastatic melanoma, not only increasing survival rates but also reducing the need for the costly procedures and hospitalizations that are now part of the standard of care for these diseases.

As a case study, consider acute myeloid leukemia (AML), one of the most serious and challenging blood cancers. Progress understanding and developing effective and safe therapy for patients with AML has been modest, and overall survival for patients with this terrible disease is measured in months. According to a study published by the journal, Biology of Blood and Marrow Transplantation, the average cost for the chemotherapy and stem cell transplantation involved in treating many patients with AML has been estimated to be between $280,000 and $500,000. Discovering why this disease occurs and developing targeted medicines to treat it are really the only alternatives to help these patients and to reduce the cost of treatment failures.

Precision medicines are dramatically changing the treatment landscape for deadly cancers like non-small cell lung cancer and metastatic melanoma, not only increasing survival rates but also reducing the need for the costly procedures and hospitalizations.

Yet, to realize the promise of precision medicines, we must act collectively across the health-care ecosystem to ensure that patients who desperately need these transformational therapies have access to them.

A problem that too many Americans face when prescribed specialty medicines to treat complex or rare conditions is high out-of-pocket costs. Many patients with the most serious illnesses face high deductibles and coinsurance requirements, which often put the latest, safest and most-effective treatments out of their reach. These patient cost-sharing barriers are one of the reasons half of the medicines used to treat chronic diseases are not taken as prescribed, contributing to the estimated $100 billion to $290 billion of unnecessary costs to the U.S. health-care system from medication non-adherence, as cited by the Annals of Internal Medicine and the New England Journal of Medicine.

We must do better. We need to work together to ensure access to these medicines and reduce the financial burden on patients. Towards this end, Celgene is proactively working with major commercial U.S. health-care payers on arrangements designed to give eligible patients access to our most recently approved medicine — a precision therapy with an accompanying diagnostic test — without deductibles, co-pays and co-insurance. By partnering with payers to offset and even eliminate patient cost-sharing as an obstacle to treatment, our hope is to prevent some of the financial burden that leads to many of the problems currently impacting patient care.

Our action is just one step in what will be needed to ensure access to the medicines Americans grappling with devastating diseases need. As health-care stakeholders, it is up to all of us to work together to develop market-based solutions to ensure that medical innovation continues to be valued, and that patients have affordable health care. We’re not there yet, but we are getting closer. Celgene is working with U.S. commercial health-care payers to step up to that challenge. We are also committed to engaging with policy-makers on finding ways to develop innovative contracting strategies that can benefit patients with government insurance as well. We encourage others in the health-care ecosystem to join us in finding solutions to these challenges.

This article was originally published on CNBC.com on August 3, 2017. 

Cancer survives and thrives by working around the body’s natural defenses and turning off the immune system’s roadblocks before it can attack the disease. One way tumor cells flourish is by using the programmed death-1 (PD-1) receptor and programmed death-ligand 1 (PD-L1) pathway to dampen the immune system. Innovative new therapies are now tackling this pathway in an attempt to slow the progression of certain tumors

PD-1 is a “checkpoint,” which immune cells use to determine whether they should attack an enemy, such as a tumor cell or a cell infected with a virus, or shut themselves down. Cancers, though, have found ways to manipulate PD-1. For example, they make high levels of its ligand, PD-L1. So when immune cells approach tumors, they become anesthetized by the PD-L1 and lose their ability to attack.

New immunotherapy research is examining whether antibodies that block the PD-1/PD-L1 pathway can awaken and reactivate immune cells so they can once again kill tumor cells.

PD-1 and PD-L1 antibodies release the brakes on the immune system and can restore its natural antitumor response

There are other therapies designed to work with the immune system to combat cancer, but PD-1 and PD-L1 inhibitors may hold unique potential for some hard-to-treat cancers.

“PD-1 and PD-L1 antibodies release the brakes on the immune system and can restore its natural antitumor response,” said Robert Hershberg, Executive Vice President, Head of Business Development and Global Alliances, at Celgene Corporation,former Chief Scientific Officer and leader of the Immuno-Oncology Center of Excellence. “I think there’s very little doubt now that the future of oncology is inextricably linked to the immune system.”

While targeted therapies effectively shut down just one target within cancer cells, immunotherapy has more widespread effects — working with the body’s immune system as a whole to make it more difficult for the cancer to survive. Early clinical research suggests that a range of solid tumor cancers, including melanoma, lung cancer, bladder cancer, head and neck cancer (among others), respond to immunotherapy. Using sophisticated immune monitoring techniques to determine which patients respond to these immune-targeting agents remains a crucial endeavor at Celgene.

Disrupting the PD-1 checkpoint may also result in an unchecked immune response that may lead to adverse effects for some patients. Researchers are learning how to engineer these therapies to not only be more effective but also minimize molecular interactions that may have undesirable consequences.

Down the road, combination therapy with PD-1 and PD-L1 antibodies could be even more advantageous. “It’s a breakthrough and revolutionary, but really the tip of the iceberg,” Hershberg said.

 

RAFAEL FONSECA, MD, MYELOMA EXPERT AND CHAIR OF THE DEPARTMENT OF MEDICINE AT THE MAYO CLINIC IN ARIZONA, AND COLLEAGUES DISCOVERED THAT COST DRIVERS IN THE CARE OF MULTIPLE MYELOMA INCLUDE HOSPITALIZATIONS AND OUTPATIENT SERVICES, NOT INNOVATIVE THERAPIES ALONE.

RAFAEL FONSECA, MD, MYELOMA EXPERT AND CHAIR OF THE DEPARTMENT OF MEDICINE AT THE MAYO CLINIC IN ARIZONA, AND COLLEAGUES DISCOVERED THAT COST DRIVERS IN THE CARE OF MULTIPLE MYELOMA INCLUDE HOSPITALIZATIONS AND OUTPATIENT SERVICES, NOT INNOVATIVE THERAPIES ALONE.

Over the past decade, treatment advances in multiple myeloma are helping patients live longer, better lives. But many doctors are concerned that those advances have made treatment unaffordable for some patients. Leading up to this year’s American Society of Clinical Oncology (ASCO) meeting, oncologists are learning that might not be the case.

Since 2000, outpatient services and inpatient admissions have driven more of the total cost increases for multiple myeloma treatment than new therapies, according to a study published earlier this year in Leukemia.

“That was a bit of a surprise to us,” said Rafael Fonseca, MD, a myeloma expert and chair of the Department of Medicine at the Mayo Clinic in Arizona and one of the study authors. “We just didn’t know exactly how much we were spending on the various components of multiple myeloma care.”

One of the primary drivers of the cost increases for multiple myeloma care is outpatient services, which increased by $5,200 per patient per month from 2000 to 2014. This rise was fueled by an increased use of imaging technologies, diagnostics and other tools needed for the care of myeloma patients.

Meanwhile, hospitalizations and prescription medication costs increased by $4,100 and $3,833, respectively, due to increased utilization of stem cell transplants and other new therapies.

Fonseca and his co-authors dug deep into “real world” data from the Truven Health MarketScan Research Databases and Medicare Supplemental Databases. Those databases provide information about what is paid for health services and medications, including rebates and discounts that insurers and pharmacy benefit managers receive.

In contrast, conversations about treatment-related costs often focus on list prices rather than actual prices negotiated by insurers, according to Fonseca.

Multiple Myeloma Treatment Cost Increases Per Patient Per Month

Of course, the study’s data had some limitations. For instance, it only included people with commercial health coverage or private Medicare supplemental coverage, so the results may not apply to people with other or no health insurance. Also, the identification of multiple myeloma patients relied on diagnosis codes, which may not always be accurate or complete.

“Despite those limitations, it’s fairly representative of reality,” Fonseca said. “Without an objective analysis, the community risks perpetuating the narrative that patients can’t afford treatment. I would argue that’s not true in most cases.”

At the Mayo Clinic, Fonseca treats patients from all walks of life and some on fixed incomes. Most of his patients ultimately get the care they need because their health insurance or Medicare help with the costs and foundations like the Leukemia and Lymphoma Society provide additional copay support. Even though some patients needed help with their efforts to secure financial assistance, Fonseca does not remember a single instance in his practice where a multiple myeloma patient couldn’t access their treatment because of cost.

That is not to say it never happens, but at least not for his practice. “Commercially insured patients get a fixed maximum copay, Medicare patients receive support from foundations, and we can obtain free medications for uninsured patients,” Fonseca said.

Without an objective analysis, the community risks perpetuating the narrative that patients can’t afford treatment. I would argue that’s not true in most cases.

The false narrative that cancer therapies are unaffordable to patients can have very negative consequences. Fonseca knows the stories; one oncologist in Arizona feared treatment might be a financial burden for one newly diagnosed myeloma patient and his family, so he sent that patient to hospice instead.

“That’s just mind-boggling,” said Fonseca. “At the Mayo Clinic, we have always found a way to get affordable treatment for our patients. Narratives, true or not, can indeed have real-world consequences.”

That narrative has also given credence to the increased use of strategies like prior authorization and specialty tiers that insurers and pharmacy benefit managers use to reduce costs by restricting or discouraging the use of medications.

But many of these strategies, which are not guided by clinical evidence, affect patient care, according to a recent ASCO policy statement. ASCO believes that these strategies should follow evidence-based clinical pathways.

“It’s unethical to ask sick patients to pay more when they are faced with expensive medical costs,” Fonseca said. “The consumerism trend in health care is exerting pressure to prevent utilization. I am pleased that ASCO decided to speak out against these practices. They recognize these innovative therapies for multiple myeloma are worth the cost.”

To learn more about how new treatments are giving multiple myeloma patients hope, read “A Decade of Progress in Myeloma, And More to Come.”

ONCOLOGIST DEBRA PATT BELIEVES THE AMERICAN SOCIETY OF CLINICAL ONCOLOGISTS’ RECOMMENDATIONS WILL IMPROVE CANCER CARE.

ONCOLOGIST DEBRA PATT BELIEVES THE AMERICAN SOCIETY OF CLINICAL ONCOLOGISTS’ RECOMMENDATIONS WILL IMPROVE CANCER CARE.

By Debra Patt, MD, vice president of Texas Oncology

The American Society of Clinical Oncology (ASCO) is speaking out against the strategies that health insurers use to control the use of innovative therapies for cancer and other diseases and provides guidance for improving these strategies to improve patient care.

In a policy statement published earlier this year, the organization said these strategies often block access to quality cancer care. Instead, the group recommends guidelines that are based on medical evidence and the best interests of patients.

ASCO should be praised for stepping up and providing this guidance for improving cancer care. Following these guidelines will allow more cancer patients to get the right treatment without inappropriate barriers.

Each cancer therapy has a unique combination of efficacy, safety and tolerability that may best suit an individual patient, according to a doctor’s determination. ASCO believes insurers’ strategies can often hinder doctors from giving patients appropriate treatments.

These strategies operate under the false assumption that all cancer treatments are interchangeable. Sometimes there are therapeutically equivalent options, but that’s the exception, not the rule.

Over the past year, I have seen a dramatic rise in the use of approaches meant to save insurers money, such as prior authorization, step therapy, specialty tiers, restrictive formularies and a lack of parity for oral treatments. These strategies are blunt instruments targeted at decreasing utilization. They can be aggressive and arbitrary. What cancer patients need are finer instruments that consider the complexity of cancer and allow appropriate access to therapeutic intervention.

This trend isn’t stopping anytime soon. New cancer therapies continue to enter the market, and insurers continue to face pressure to keep cancer care costs down.

These strategies are blunt instruments targeted at decreasing utilization. They can be aggressive and arbitrary.

Prior authorization, in particular, has hindered patient care at Texas Oncology. A year ago, oncologists started chemotherapy the day after determining a patient’s treatment plan. Today, the wait time is up to a week before the practice can obtain prior authorization from the insurance companies. At times, approval has taken up to a month. It should take a couple of hours if all the information required to make the determination is available when the order is written. This process needs to be streamlined.

Waiting for treatment to begin can be especially tough on patients, who are often afraid and want to get started as soon as possible. It’s in the best interest of patients to start treatment in a timely manner, not a month later. But we face delays and uncertainties in care with these policies today.

Other insurance strategies also compromise cancer care. Step therapy forces patients to try and fail one therapy before getting access to another. Specialty tiers require a higher cost-sharing burden for cancer therapies than for other diseases. Formularies, the list of prescription medications covered by a plan, are not being developed with transparency or the appropriate clinical oversight. Finally, patients often have higher copays, coinsurance and deductibles for oral cancer therapies than for treatments given in a hospital, such as intravenous or other modes of injectable medicines.

Insurers' and Pharmacy Benefit Managers' Policies Undermine Cancer Care

Instead, these insurer strategies should be based on evidence-based clinical pathways. This approach will ensure the appropriate use of cancer therapies and the best cancer care.

ASCO’s statement galvanizes support for state and federal legislative proposals to protect patients from aggressive cost-reduction strategies. For instance, legislation introduced in Texas this year would ensure step therapy is only used when medically appropriate and offers protections for cancer patients’ treatments on Medicaid formulary.

The policy statement advocates for approaches that provide the right care for patients at the right time. ASCO is trying to provide guidance to payers regarding how to manage a real, complex, and growing problem of managing utilization and cancer treatment costs while allowing the right care to the right patient. ASCO’s recommendations will help improve cancer care for patients as strategies like these remain a continuous challenge for doctors and the patients we serve.