PMC Nominee Dr. Freda Lewis-Hall Selected for PCORI Board of Governors

In June, PMC nominated a slate of individuals representing the diversity of our coalition including representatives from patient advocacy groups, hospitals and health systems, the pharmaceutical and diagnostics industries, and public health agencies for appointment to the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors.  We were pleased that the Government Accountability Office (GAO) included Pfizer’s Chief Medical Officer Dr. Freda Lewis-Hall, a PMC nominee with expertise in and a commitment to personalized medicine, as a member of the PCORI Board of Governors.

But while we thank GAO for selecting a group of individuals for the PCORI Board of Governors who are committed to improving the health of the American people through research that will, we hope, improve outcomes, we also recognize that our work is not finished. We must continue to advocate for personalized medicine both with the PCORI Board of Governors and eventually with its Methodology Committee, to ensure that comparative effectiveness research considers population subtypes, individual variation, and personalized medicine in general.

 The 21-member PCORI Board of Governors, established by the Patient Protection and Affordable Care Act, is tasked with assisting patients, clinicians, purchasers, and policymakers in making informed, evidence-based, health decisions.  It will identify research priorities, carry out a research agenda, and receive support from a Methodology Committee and expert advisory panels.

We were disappointed that the newly selected members of the PCORI Board of Governors did not include more individuals with specific expertise in personalized medicine, in particular, individuals representing small, innovative diagnostics companies.  Small diagnostics companies, which develop new tests for detecting disease, are central to the field of personalized medicine and will play a necessary and important role in the research PCORI will conduct. We hope that the yet-to-be-named Methodology Committee will include more representation from this important sector.  

The members picked today by the GAO include the 19 members below, as well as the Director of the National Institutes of Health and the Director of the Agency for Healthcare Research and Quality, or their designees:

• Freda Lewis-Hall, Pfizer
• Eugene Washington, MD, UCLA
• Steven Lipstein, MHA, BJC Health Care
• Christine Goertz, Palmer College of Chiropractic
• Sharon Levine, MD, Permanente
• Ellen Sigal, PhD, Friends of Cancer Research
• Harlan Weisman, MD, Johnson and Johnson
• Robert Zwolak, MD, Dartmouth-Hitchcock
• Lawrence Becker, Xerox
• Arnold Epstein, MD, Harvard/Brigham and Women’s
• Andrew Imperato, AAPD
• Robert Jesse, MD, Department of Veterans Affairs
• Grayson Norquist, MD, Dept of Psychiatry and Human Behavior,  University of Mississippi Medical Center
• Debra Barksdale, UNC Chapel Hill School of Nursing
• Kerry Barnett, The Regence Group
• Allen Douma, AARP
• Leah Hole-Currty, JD, Washington State HTA program
• Harlan Krumholz, MD, Yale University
• Richard Kuntz, Medtronic

Letter to the Editor: The Proper Integration of Comparative Effectiveness Research

A recent editorial in The New York Times (Is Newer Better? Not Always, Sept. 11, 2010) suggests that the comparative effectiveness research (CER) provisions of health care reform are a solution to curtailing ever-increasing medical costs by providing patients, doctors, and the government better information about the efficacy of different drugs and treatment strategies. It also suggests that the real opportunity is in giving policymakers more power to enforce CER-informed practice guidelines. 

In response, I sent a Letter to the Editor, agreeing that CER can be part of the overall solution. However, I also cautioned that thinking simplistically about medical innovation as the “problem” and CER-based guidelines as the “solution” overlooks the value that medical advances like those in personalized medicine hold for patients, and for helping address rising health care costs.

“If we lock ourselves into a one-size-fits-all paradigm, we will strangle the power of personalized medicine to prescribe the right drug for the right patient at the right time.” This approach could turn CER “into a blunt instrument that smothers the opportunity to improve care and reduce costs.”

Also worth noting is a separate letter from Tony Coelho, head of the Partnership to Improve Patient Care, who argued that the CER provisions of the health reform law “can be an important part of the solution to rising health care costs over the long term, but only because the health reform law gives patients and doctors the assurance that the program is centered on our health care needs.”

Read both of these published responses below and continue the discussion by contributing your own comment at The Age of Personalized Medicine Blog.

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To the Editor:

Your editorial correctly states that new health care technologies often lead to medical progress, but also regrettably to increased and unaffordable costs, unlike in other fields where new technology usually causes costs to decline.

The danger is that the proposed solution, comparative effectiveness research (CER), could turn into a blunt instrument that smothers the opportunity to improve care and reduce costs.

When President Obama sought to explain how it would work, he said that with the new funds allocated for CER, researchers would be able to compare the red pill with the blue pill to determine which worked better and thereby, presumably, save money.

The key question to ask, as neither your editorial nor President Obama does, is which medicines work for whom? While the blue pill may work for me, there is a good chance it won’t work for you, especially if the diagnosis is cancer.

If we lock ourselves into a one-size-fits-all paradigm, we will strangle the power of personalized medicine to prescribe the right drug for the right patient at the right time. Personalized medicine enables us, over time, to save enormous sums because patients who should not receive particular medicines will not get them.

Edward Abrahams
President
Personalized Medicine Coalition
Washington, Sept. 13, 2010

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To the Editor:

Creating a body with the power to enforce national guidelines for treatment based on clinical and cost effectiveness would be a serious distortion of the comparative effectiveness research (CER) provisions of the health reform law. It would confirm the fear of many that the real intent was to give government decision makers new power to cut costs by denying patients access to medically beneficial tests and treatments.

Fortunately, Congress took a different tack in the health reform law and got it right. It acted not in reaction to fear-mongering but because, as stated last year by one Democrat, the CER research center must be a partner to patients.

Yes, CER can be an important part of the solution to rising health care costs over the long term, but only because the health reform law gives patients and doctors the assurance that the program is centered on our health care needs. Newer isn’t always better, but in the case of the new patient-centered approach to CER in the health reform law, it is.

Tony Coelho
Washington, Sept. 14, 2010
The writer, a former member of Congress, is chairman of the Partnership to Improve Patient Care.

A version of these letters appeared in print on September 20, 2010, on page A30 of the New York edition.

Fall Forecast: Personalized Medicine Policy and Events

Labor Day has come and gone, Congress is preparing to go back in session, and shortening days remind us that Daylight savings will be here before we know it. Even as the long days of summer draw to a close, the fall promises to be a busy and productive time in advancing policies to support personalized medicine, as well as convening industry stakeholders to connect, share ideas, and collaborate toward advancing the science, business, and policy of personalized medicine.

This fall we expect to see a maturing of activities started over the summer.  FDA hosted a 2-day meeting in July on the regulation of laboratory-developed tests (LDTs).  The meeting docket (FDA-2010-N-0274) provides a good overview to any who missed the event.  After that meeting, the House of Representatives held a hearing on consumer genomics.  This hearing made it clear that Congress is not satisfied with how gene scans are regulated, and sometimes that dissatisfaction was aimed at LDT regulation in general. The Senate is also concerned; GenomeWeb reported that Senator Hatch (R-UT) is considering legislation on the subject. 

In other legislative news, Rep. Patrick Kennedy (D-1^st RI) partnered with Rep. Anna Eshoo (D-14^th CA) to introduce an updated version of the Genomics and Personalized Medicine Act (HR 5440). PMC will continue our work to strengthen this bill through inclusions of research  incentives for personalized medicine and other improvements in the Senate version as we look for it to be re-introduced  in the next Congress. 

Next week, I will be joining Carolyn Clancy, Francis Collins, and  many other leading experts at the 2nd National Comparative Effectiveness Summit which will feature presentations and discussion on the legislative history and future of comparative effectiveness research (CER), how federal stimulus dollars for CER are being put to use, as well as the intersection between CER and personalized medicine. The focus of this meeting is particularly timely as the expected announcement of the federally-appointed Board of Governors for the Patient-Centered Outcomes Research Institute (PCORI) is expected later this month. The discussion on balancing CER and personalized medicine objectives will continue in October at the Comparative Effectiveness and Personalized Medicine:  An Essential Interface meeting hosted by the ECRI Institute, Agency for Healthcare Research and Quality, and the National Institutes of Health.

There are a number of other meetings taking place throughout the fall focused on a variety of topics and issues relevant to personalized medicine, beginning with the 6th Annual Burrill Personalized Medicine Meeting on September 27 & 28. Hosted by Burrill & Co and the Duke Institute for Genome Sciences & Policy, the meeting will focus on the impact of healthcare reform on personalized medicine. On October 14 & 15, the 3rd Annual Personalized Health Care National Conference is taking place at Ohio State University Medical Center’s Center for Personalized Health Care. This conference will include discussion of the challenges of making predictive, preventive, personalized, and participatory medicine a reality.

The conference season will culminate on November 17 & 18 with the 6th Annual Personalized Medicine Conference. Each year, the Partners HealthCare Center for Personalized Genetic Medicine, Harvard Medical School, and Harvard Business School bring together representatives from academia, diagnostic and pharmaceutical companies, healthcare providers, policymakers, and other industry experts making contributions to advancing personalized medicine. This year we’ll explore the ways personalized medicine impacts clinical care, and how lessons from these experiences can inform policies and plans to implement personalized medicine to improve patient care.

For more information on these and other conferences of importance to personalized medicine, please visit the Events page at The Age of Personalized Medicine website. And throughout the fall, stay tuned to The Age of Personalized Medicine Blog as an opportunity to engage in discussions stemming from these events and new policy developments.

Leveraging Partnerships and Personalized Medicine to Find a Cure

The sixth scientific workshop of the Progeria Research Foundation (PRF) earlier this year highlighted many ways that an increased understanding of the genetic underpinnings of Progeria is informing promising new approaches for treating the disease.  A record 140 attendees congregated from 10 different countries to hear expert oral presentations and view poster presentations. Doctors and scientists – who often work in separate worlds, either in the clinic or in the lab – inspired one another as they came together to share new discoveries and directions for future research.  Speakers included leading scientists in the fields of heart disease, aging, genetics and lamins.

Researchers at the workshop had a unique opportunity to meet some of the children who are benefiting from their work.  Children and their parents talked about what it’s like to live with Progeria, and answered questions from the attendees who wanted to better understand how they can continue to conduct beneficial research.  Leslie Gordon, PRF’s medical director, shared Progeria’s journey from obscurity, through gene discovery, on to treatment trials, and presented a vision of where the field is headed in the push towards new treatments and a cure. 

We heard from clinical trial investigators, who shared updates on the progress of their current studies.  We heard from world renowned scientists such as George Martin, Elizabeth Nabel, and Judy Campisi, who addressed the links between normal aging, cardiovascular disease, inflammation and Progeria.  And we got a glimpse into the future from several scientists who are working on cutting edge strategies for future drug development and gene therapy for Progeria.  

Dr. Tom Misteli (NCI) presented a drug development assay which uses genetic strategies to measure chemicals that decrease or eliminate the production of the disease-causing protein, progerin.  His promising studies point us in the direction of new treatments or even a cure for Progeria.

 Dr. Fyodor Urnov looked to the possibility of a genetic cure for Progeria using “genome editing” with zinc finger nucleases.  This method of repairing genetic mutations within cells of the human body is now in the first stages of clinical trials for other diseases, and may be applied to Progeria in the near future.

PRF is proud that Progeria research has made such tremendous progress, moving from bench to bedside in only 10 years.  This would not be possible without the collaborations and innovative partnerships that have been established over the last decade.  From our collaboration and partnerships with NIH (NHGRI, NCI and NHLBI), to pharmaceutical companies like Merck Schering-Plough to first-rate hospitals like Children’s Hospital Boston,  we are moving at lightning speed in the direction of finding a cure.

Audrey Gordon, Esq. is the President and Executive Director of the Progeria Research Foundation