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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.

By Mark Alles, Celgene CEO

Mark Alles

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

When the President’s Cancer Panel convened recently to consider priorities for cancer policy, its focus was exactly where it should be: encouraging innovations that produce life-saving medicines, and increasing patients’ access to those medicines.

There is legitimate concern about healthcare costs, so it is important to understand what matters and what works. Simply put, medical innovation saves lives and over the past decade has reduced health expenditures by more than $1.5 trillion. But the real measure of this progress is the millions of Americans who are living active lives because of disease-altering treatments, despite their medical conditions. Take Don Wright, who at age 62 was diagnosed with multiple myeloma, an incurable blood cancer, and told he had no more than three years to live. Thanks to medical innovation, Don is alive 13 years later. He recently completed his 100th marathon since his diagnosis.

Don Wright is but one example of the many Americans now living longer, healthier and better lives due to new treatments. Today, more than 30 million diabetes patients are benefitting from new medicines that offer better control of glucose levels. Innovative therapies have helped cut deaths from heart disease in half and reduce by 20 percent cardiovascular disease’s share of national health expenditures. Also of note, new cancer therapies are linked to a 25 percent decrease in disease-related deaths since 1991 and a societal economic benefit of $2 trillion.

Treatments that provide the greatest health impact for seriously ill people should have the lowest cost sharing requirements.

All of this tremendous progress has been achieved while retail expenditures on life-enhancing therapies have remained steady at just 10 percent of total U.S. healthcare spending. In fact, as a percentage of the healthcare dollar, retail prescription medicine costs are the same today as in 1960, and are projected to remain no higher than this level for at least the next 10 years. That’s remarkable stability – maintaining the same rate of spending when more than 550 new medicines have been approved over the past 15 years.

It is certainly true that many of the newest innovative medicines have a high cost. But the value innovative medicines deliver to patients and society is immense. Innovative “specialty” medicines such as treatments for cancer, HIV/AIDS and multiple sclerosis comprise less than four percent of total healthcare expenditures and are taken by less than one percent of all patients. Yet notwithstanding the immense value of these medicines, many insurance plans impose disproportionate cost burdens on patients who need them, requiring patients to pay on average five times more out of pocket for medicines than for hospital care.

To change this situation, we propose a straightforward principle: treatments that provide the greatest health impact for seriously ill people should have the lowest cost sharing requirements. Additionally, the most vulnerable patients must be protected from insurance company practices such as adverse tiering (in which all new drugs for a serious disease are assigned to the highest cost sharing level) and step therapy (where patients are required to try one or more drugs before the medicine their doctor wants to prescribe is covered).

All healthcare stakeholders should work together to promote reforms that enhance broad, affordable access for patients and eliminate cost-sharing hurdles that may discriminate against patients who need care the most.

Despite these insurance shortcomings, the good news is that the virtuous cycle of medical innovation continues to deliver. Today 89 percent of prescriptions are filled as generics, at prices 75 to 90 percent lower than the original patented medicines. This incredible value is built into our healthcare system by design. The cost of innovative medicines plummets after a period of patent protection, providing the best medical solutions in the world at the lowest costs to society for generations while incentivizing medical researchers to continue developing new and even better therapies.

We are in an unparalleled time of medical advances that can continue to improve patients’ lives, increase life expectancy and reduce the economic burden on healthcare systems. The United States is leading the way, developing 57 percent of the world’s breakthrough medicines. But we can maintain this momentum only if patients have access to the medicines they need. What’s the point of developing innovative new medicines if patients cannot benefit from them?

All healthcare stakeholders should work together to promote reforms that enhance broad, affordable access for patients and eliminate cost-sharing hurdles that may discriminate against patients who need care the most.

This article was originally published on Forbes.com on June 2, 2017. 

AMY BURD, VICE PRESIDENT OF RESEARCH STRATEGY AT THE LEUKEMIA & LYMPHOMA SOCIETY (LLS), IS LEADING A CLINICAL TRIAL THAT IS BRINGING A MORE PERSONALIZED APPROACH TO AML TREATMENT.

AMY BURD, VICE PRESIDENT OF RESEARCH STRATEGY AT THE LEUKEMIA & LYMPHOMA SOCIETY (LLS), IS LEADING A CLINICAL TRIAL THAT IS BRINGING A MORE PERSONALIZED APPROACH TO AML TREATMENT.

There has been limited advancement in the treatment of acute myeloid leukemia over the past 40 years, with most patients undergoing intensive, toxic chemotherapy. Today, AML remains a deadly blood cancer, taking the lives of more than 10,000 patients each year, and less than 30 percent of patients live five years or longer after being diagnosed.

As the blood cancer community recognizes April 21 as the second annual AML Awareness Day, an innovative approach to clinical trials holds promise to overcome the research stagnation and explore tailored alternatives tailored for individual patients.

“A high unmet medical need exists for AML treatment,” Amy Burd, Ph.D., vice president of Research Strategy at The Leukemia & Lymphoma Society (LLS), said. The limitations of chemotherapy are especially notable for the elderly, she explained. “Older patients — those who are the most likely to be diagnosed with AML — cannot tolerate the standard chemotherapy regime. It’s just too toxic for them.”

One reason treatment remains challenging is that AML is not a single disease but a complex group of more than 10 different subtypes, some of which are more aggressive than others. And not all types respond to treatment equally.

In 2008, researchers sequenced the genome of AML tumor cells, improving the understanding of how the disease develops and, potentially, treatments it might respond to. With this knowledge in hand, scientists have developed targeted therapies that open up the possibility to tailor treatments for individual patients.

AML Treatment Milestones Timeline

Last October, the LLS launched a unique clinical trial initiative called the Beat AML Master Trial, which matches patients with an investigational drug or drug combination potentially best suited to attack the specific mutations causing their cancer.

Their goal is to create nothing short of a paradigm shift in how AML is treated, moving away from the current one-size-fits-all approach.

But that shift requires a change in the clinical development culture — shaking up business as usual for clinical trials. A typical trial focuses on one or two therapies; but the Beat AML Master Trial is examining multiple treatments — including small molecule therapies, immunotherapies and checkpoint inhibitors — at multiple medical research centers across the country. The LLS has been coordinating the activities with the U.S. Food and Drug Administration, multiple biopharmaceutical companies, and at least 10 trial centers. LLS plans to enroll approximately 500 patients in this trial

We’re trying to put all these pieces together in a way that is focused on what is best for each patient. This is a new approach for clinical trials.

“Putting all the pieces together is the biggest challenge with this initiative,” Burd said. “We have the DNA sequencing technologies, genomic analysis and targeted therapies. But how do we get to the point where they are working together in a way that is focused on what is best for each individual patient? This is a new approach for clinical trials and we think this Master Trial has the potential to stand as a model for future cancer clinical trials.”

Since the launch, they’ve already exceeded the targeted preliminary enrollment of 25 patients and provided those patients with personalized treatment plans. Enthusiasm among investigators and patients has been high, according to Burd.

“It speaks to the high unmet need in AML and desire for patients and doctors to have better treatment options,” Burd said. “Our mission has always been to put patients first. So we’ve incorporated patient-reported outcomes into the study to ensure they are getting the results that matter to them — whether that’s being able to walk around the block or run a marathon.”

ELEANOR DEHONEY, VICE PRESIDENT OF POLICY AND ADVOCACY AT RESEARCH!AMERICA, BELIEVES POLICY MUST BALANCE INCENTIVES FOR INNOVATION AND AFFORDABLE ACCESS FOR PATIENTS.

ELEANOR DEHONEY, VICE PRESIDENT OF POLICY AND ADVOCACY AT RESEARCH!AMERICA, BELIEVES POLICY MUST BALANCE INCENTIVES FOR INNOVATION AND AFFORDABLE ACCESS FOR PATIENTS.

When the European Commission compiled its annual EU Industrial Research & Development Investment Scoreboard last year, Celgene led the world’s largest companies in percentage of revenues reinvested in research and development (R&D), at nearly 40 percent in 2015.

For Celgene, this reinvestment into the creation of more and better innovative medications is essential to fulfilling its promise to patients, ensuring that scientific advances continue to improve human health. The development of new therapies relies on substantial investments in R&D.

“The clock is ticking for many patients, and robust pipelines in the biopharmaceutical industry give them hope for the future,” said Eleanor Dehoney, vice president of policy and advocacy at the public health advocacy organization Research!America. “The purpose of mission-driven companies like Celgene is to invest and fuel medical progress continuously.”

Investments by Celgene and America’s other biopharmaceutical companies are already changing the course of serious and life-threatening diseases such as cancer. According to a recent report by the American Cancer Society, the death rate from cancer in the United States fell 25 percent between 1991 and 2014, with more than 2.1 million deaths averted. Researchers have credited new treatments—including innovative medications—with 79 to 86 percent of survival gains between 1980 and 2000.

The future of cancer care is also giving patients a reason to be hopeful. More than 800 new cancer therapies are under development, and 80 percent of cancer treatments in the pipeline represent potentially first-in-class medicines that use new approaches—such as CAR-T cells and immunotherapies.

Virtuous Cycle of Medical Innovation

A robust pipeline of cancer therapies would not have been possible without a commitment to ensuring approval of and reimbursement for the cancer medications that have been developed previously. This virtuous cycle of innovation is essential to ensuring continued progress in the fight against cancer and other diseases.

“We must continue to balance incentivizing innovation and providing affordable access for patients everywhere,” Dehoney said. “If policy isn’t developed with a holistic view of the entire health care ecosystem, the innovation that has the potential to drive down costs will suffer.”

Research supports the idea that increased spending on prescription medications can help reduce total cancer care costs. In a 2014 study published in the Journal of Oncology Practice, pharmaceutical spending rose by 179 percent, but overall cancer care costs decreased by 34 percent due to reductions in hospital care and radiation therapy. Meanwhile, patients continued to receive high-quality care.

Reinvesting revenue into R&D is very patient-centric, and we as a society need to support those investments to improve human health.

That study tested a new payment model that rewarded doctors based on how well their patients did. This scheme is a departure from the current fee-for-service system where doctors are paid for how often they see their patients not the quality of that care. Such a holistic view will ultimately benefit innovation and provide patients with the most appropriate treatment, according to Dehoney.

“We tend to undervalue medical innovation, but it’s going to take a village to tackle the health care challenges we face today,” Dehoney said. “Reinvesting revenue into R&D is very patient-centric, and we as a society need to support those investments to improve human health.”

To learn more about the why patient access is essential to supporting innovation, read “The Virtuous Cycle: Investing in Better Health Care.”

A study from Padmaja Ayyagari, an assistant professor at the University of Iowa, and colleagues found Medicare Part D has reduced emergency department use among enrollees.

A study from Padmaja Ayyagari, an assistant professor at the University of Iowa, and colleagues found Medicare Part D has reduced emergency department use among enrollees.

Medicare Part D is helping keep seniors out of the emergency room, according to a recent study.

Emergency department visits for nonemergency reasons have dropped 18 percent for Medicare Part D beneficiaries since the program launched in 2006, according to research published in Health Economics. The authors attribute the decline to reduced prescription costs for seniors covered by the program, which has helped them stick to their treatments.

“Our study shows that prescription drug coverage can not only improve health but also potentially save costs by reducing expensive emergency department care,” said Padmaja Ayyagari, an assistant professor of health management and policy at the University of Iowa and lead author of the study. “We specifically looked at emergency department visits because of the high costs associated with this type of care and its increased use.”

Emergency room visits rose 47 percent between 1992 and 2012. That increase translates into major costs, since experts estimate that care in an emergency department setting costs two to five times more than it does the in primary care setting.

Many emergency room visits and hospitalizations have the potential to be avoided if care is managed well before the patient’s health condition becomes severe.

Better use of primary and preventative care could save the U.S. health care system $18 billion annually, according to one estimate. Medicare Part D is contributing by reducing emergency department spending for a large population of Americans, according to Ayyagari.

“Many emergency room visits and hospitalizations have the potential to be avoided if care is managed well before the patient’s health condition becomes severe,” Ayyagari said.

Medicare Part D: Improving Access, Keeping Patients Healthy

Other studies support the idea that Part D is improving seniors’ health. A report from North Carolina State University published earlier this year, for example, found that the program has cut in half the number of seniors who stop taking their medications due to cost.

By keeping seniors healthy, Medicare Part D is reducing spending in other health care categories and making the health care system more efficient. Most consider Part D a success by almost all measures, and so we need to maintain the structure of this program to continue to improve the health of our seniors.

Discover why 88 percent of seniors are satisfied with their prescription drug coverage under Medicare Part D and how we can make the program better for seniors by reading “Medicare Part D: 10 Years of Successfully Meeting Seniors’ Needs.”