Personalized medicine is turning a corner. FDA approved four new indications for personalized medicine in July of 2015, a record for the agency and the field. As you may remember, 20 percent of FDA’s 2014 drug approvals were personalized medicines. At this rate, that percentage will likely be matched or exceeded this year.

But what does that mean for the field?

It means policymakers throughout the health care ecosystem have to play catch up, because ethically and scientifically there is no going back. Science has led us here because personalized medicines save and extend lives. They keep people at work, enjoying their families and contributing to society. They reduce morbidity and the adverse health events associated with older treatments. And for some patients they extend lives long enough to allow for participation in promising clinical trials, which can extend lives even further.

For evidence of personalized medicine’s potential, look no further than this month’s approvals (see table below). While the list covers a variety of disease conditions, it is clear that we are making significant headway in turning deadly diseases into chronic conditions, and in some cases, finding functional cures. For example, through significant investments by industry and the patients they serve, we now have treatments for cystic fibrosis that allow young people with the disease to live longer, fuller and more fulfilling lives.

Table 1. July’s Approved Indications for Personalized Medicines

It is incumbent upon decision-makers to ensure that health policy and delivery systems support the use of these important medicines so that they can continue to deliver on their promise.

Reimbursement policies are part of that discussion. As noted in a recent article from 118 oncologists that was published in Mayo Clinic’s academic journal, Mayo Clinic Proceedings, current trends in coverage policies can result in individual patients being required to make co-payments that are as high as 30 percent of the total cost of a prescribed therapy. That is not sustainable, but the factors that lead to these unfortunate circumstances are not linear. They involve multiple actors. And so should the solution.

In this context I encourage all the field’s stakeholders to work together toward ensuring that personalized medicine makes us healthier, because as FDA’s recent decisions make clear, personalized medicine is no longer just a hypothetical paradigm.

It is our current reality.

—
Amy M. Miller, Ph.D.
Executive Vice President
Personalized Medicine Coalition

Companion diagnostic tests (CDTs) can offer a multitude of potential benefits such as improved effectiveness, improved side effect profiles and streamlined clinical trials, but assessing the clinical and economic value of these tests can be challenging. The technology is evolving rapidly, but a clear idea of what evidence is necessary to enable a payer to fully evaluate the diagnostic may be lagging.

The lack of consistent value-based coverage and reimbursement of diagnostic tests presents a significant barrier to patient access to personalized medicine products. As a co-author of a peer-reviewed study funded by the National Pharmaceutical Council (NPC) and published in the Journal of Managed Care & Specialty Pharmacy, I worked with Robert Dubois at NPC and our colleagues at the University of Washington to outline a framework to assist managed care organizations in determining the value of CDTs.

How do payers assess value? Based on the information gathered from a literature review and interviews with payers, we developed and tested a value assessment tool aimed at providing clarity and consistency in the evaluation of diagnostics. The tool reflects perceived value drivers and consists of four main sections:

• Eligibility for review: is the test a companion test?
• Prioritization of review: is a standalone evidence review of the test warranted?
• Clinical review: are the test results valid and clinically useful?
• Economic review: does the test provide good value?

Existing frameworks for evaluating the value of companion tests are often too cumbersome or inconsistently applied for the average managed care decision-maker to complete in a timely manner, thereby slowing down access to personalized medicines. Our value assessment tool is designed to capture all the different types of companion diagnostics, and be simple to use, thus bringing greater efficiency to the CDT evaluation process.

“As more CDTs enter the market,” we write, “it will become increasingly important for those evaluating their use to quickly gain a sense of their value… [U]sing a formal tool to guide the evaluation process by prioritizing assessments and focusing on key drivers may improve the efficiency and effectiveness of CDT value assessment.”

—
Daryl Pritchard, Ph.D.
Vice President, Science Policy
Personalized Medicine Coalition

While issues in science, regulation and reimbursement are still the focus of most stakeholders in personalized medicine, an increasing number of providers are integrating personalized medicine into the health care system despite obstacles. The 2^nd Annual PMC/BIO Solutions Summit will convene representatives from industry, patient groups and payers as well as the increasing number of academic health centers and community health care systems that are already delivering personalized care to discuss those obstacles and, more importantly, identify the best practices for speeding the integration of personalized medicine into clinical care.

The event, co-hosted by PMC and the Biotechnology Industry Organization (BIO), will take place October 14, 2015 at the Renaissance Downtown Hotel in Washington, D.C. It features a distinguished line up of speakers.

Following introductory remarks, a panel including Centers for Medicare and Medicaid (CMS) Hospital and Ambulatory Group Acting Director Marc Hartstein will explore the issues involved in reviewing evidence for coverage of personalized medicine products and services. The session is designed to shed light on the kinds of data the personalized medicine community might focus on generating in order to convince payers that personalized medicine is worth paying for.

The event’s second session focuses on education. During this discussion, experts including David Flockhart of the Indiana Institute for Personalized Medicine will present strategies for ensuring that payers, providers, patients and other stakeholders stay up to date on the latest trends in the quickly evolving field.

The second session anticipates a luncheon keynote presentation that will feature a discussion between Illumina Senior Vice President & Chief Medical Officer Richard Klausner and AstraZeneca Vice President of Translational Sciences J. Carl Barrett. Klausner and Barrett will elevate the education discussion to the systems level, exploring the development of a framework for the efficient utilization of personalized medicine information throughout the health care ecosystem. Turna Ray, an editor at GenomeWeb, will moderate the discussion.

A third panel offers a look at how personalized medicine’s value can be demonstrated from a variety of perspectives. Bonnie J. Addario of the Bonnie J. Addario Lung Cancer Foundation will provide a patient voice, while representatives from an academic health center, a diagnostics company and Aetna will round out the discussion. The conversation will reveal arguments with which stakeholders can prove that personalized medicine is a worthwhile effort.

The event’s final panel will examine the infrastructure supporting personalized medicine. During this discussion, experts including Oracle Global Vice President of Health Sciences Jonathan Sheldon will explain the challenges involved in generating and managing the data required to deliver personalized care, as well as the options for overcoming these challenges.

At the conclusion of the final panel, representatives from PMC and BIO will summarize the solutions that were uncovered and provide an overview of next steps.

We look forward to seeing you at the event.

—
Chris Wells
Communications Director
Personalized Medicine Coalition

NIH’s Kathy Hudson Outlines Potential PMI Implications of 2016 Appropriations Decisions

PMC’s briefing on Capitol Hill yesterday emphasized the importance of personalized medicine as the Senate Health, Education, Labor and Pensions (HELP) Committee assesses its own priorities in medical innovation following the passage of the “21^st Century Cures” bill in the House, with Sen. Amy Klobuchar (D-MN) leading the way in advocating for funding of personalized medicine research initiatives.

“Precision medicine got a lift from the president talking about it in the State of the Union, but we need to continue on that,” Klobuchar told the standing-room only crowd of more than 125, which included more than 50 Hill staffers. “We need to increase the money we invest in research.”

Many of the panelists echoed Sen. Klobuchar’s emphasis on research funding.

Foundation Medicine CEO Michael Pellini, M.D., noted that the personalized medicine era is inevitable. But how quickly we get there, he said, depends on favorable public policies, especially in reimbursement, which he said is particularly challenging because decisions are often decentralized at the Centers for Medicare and Medicaid Services (CMS).

For her part, National Institutes of Health (NIH) Deputy Director for Science, Outreach and Policy Kathy Hudson said the agency plans to begin building a volunteer cohort that will enable research to explore the molecular underpinnings of disease in the beginning of the fiscal year, as part of President Obama’s Precision Medicine Initiative (PMI). She did add, however, that the initiative will have to move slower if lawmakers decide to pass a continuing resolution extending fiscal year 2015 funding levels. In that case, she said, the initiative would likely begin with funding opportunity announcements, instead of the development of the cohort.

The briefing was co-hosted by PMC and Sens. Klobuchar and Orrin Hatch (R-UT). Designed to provide an overview of what personalized medicine is and why it is important, it also featured comments from Stephanie Haney, a stage IV lung cancer patient, Keith Stewart, M.B., Ch.B., director of Mayo Clinic‘s Center for Individualized Medicine, and Greg Keenan, M.D., vice president of medical affairs and U.S. head medical officer at AstraZeneca. Amy M. Miller, Ph.D., PMC executive vice president, moderated the discussion.

The fact sheet below was distributed to the event’s attendees.

Personalized Medicine 101: Improving Patient Care in the 21st Century (PDF)

—
Chris Wells
Communications Director
Personalized Medicine Coalition

Something called precision medicine, in some cases people call it personalized medicine, gives us one of the greatest opportunities for new medical breakthroughs that we have ever seen.
– President Barack Obama (Jan. 30, 2015)

With these words, President Obama turned the eyes of the public toward the emerging field of personalized medicine. Last week, the House of Representatives passed the 21^st Century Cures bill, which contains several provisions designed to accelerate personalized medicine’s progress, and the Senate Health, Education, Labor & Pensions Committee is now examining the issue through the lens of its Innovation for Healthier Americans initiative.

With the issue clearly on the radar for U.S. policymakers, the office of Senator Amy Klobuchar (D-MN) invited the Personalized Medicine Coalition to organize the upcoming Hill briefing scheduled for July 16, 2015 from 12:00 – 1:30 p.m. ET. The event is co-hosted by Orrin Hatch (R-UT), a long-time champion of personalized medicine, and will feature a panel of experts representing patients, health care providers, government and the biopharmaceutical and diagnostics industries.

Against the backdrop of the Russell Senate Office Building’s awe-inspiring Kennedy Caucus Room, Stephanie Haney will kick off the conversation by explaining how personalized medicine has assisted in her eight-year battle with stage IV lung cancer. Keith Stewart, the director of Mayo Clinic’s Center for Individualized Medicine, will follow with a description of what personalized is and why it is so important. Industry representatives Greg Keenan of AstraZeneca and Michael Pellini of Foundation Medicine will then provide an explanation of the development challenges applicable to the field, and Kathy Hudson of the National Institutes of Health (NIH) will discuss how the President’s Precision Medicine Initiative will contribute.

As I look forward to the event, I cannot help but get excited. Policymakers have demonstrated a renewed interest in regulatory issues at FDA following the release of the agency’s framework for regulating laboratory diagnostic tests, and alternative payment models are clearly becoming a priority for the Department of Health and Human Services. The briefing is a powerful opportunity to demonstrate how we can incentivize the development of a more efficient health system by incorporating the personalized medicine perspective into all regulatory and reimbursement decisions. Doing so will ultimately improve the lives of patients across the country. That is an opportunity we cannot afford to miss.

—
Amy M. Miller, Ph.D.
Executive Vice President
Personalized Medicine Coalition