Archive for the ‘comparative effectiveness research’ Category

PCORI Methodology Committee Named; No Private Sector PM Experts Included

January 21, 2011

The  Government Accountability Office (GAO) today announced the members of the Methodology Committee for the Patient Centered Outcomes Research Institute (PCORI). We don’t  dispute the credentials of the 17 appointed members,  and we appreciate NIH Director Francis Collins’ participation on the committee. Yet it is nonetheless disappointing to see that none of these appointed members from the private sector bring expertise in personalized medicine, particularly since including experts in genomics was one of the specific fields identified in statute.  This will make it all the more important for leaders in the personalized medicine field to continue to advocate for alignment of PM and CER in development of research methods and study designs.   For the past year, the Personalized Medicine Coalition (PMC) has been a strong advocate in support of CER that is aligned with personalized medicine.   In October, PMC submitted a letter to GAO nominating ten experts in personalized medicine from across academia and industry.   While many on the methodology committee  bring strong expertise in their research fields, the lack of in-depth expertise in genomics and personalized medicine will make it more challenging to advance research methods that are aligned with personalized medicine and recognize “advances in medical technology” as specified by statute. 

Perhaps  the PCORI Board could create an  ad hoc advisory panel focused on personalized medicine and innovation that would help to ensure that sufficient focus is brought to these important issues as PCORI  moves forward with developing research methods and priorities .   Having such a committee could help PCORI fulfill other mandates such as public engagement, stakeholder representation, and keeping the science up-to-date.

The Role of Comparative Effectiveness Research in Total Cancer Care™

December 20, 2010

In my previous entry, I discussed how the launch of the Total Cancer Care™ initiative at Moffitt Cancer Center nearly eight years ago led to the development of one of the largest prospective observational studies in the world.  Through the enrollment of more than 60,000 patients and collection and genetic profiling of tens of thousands of tumors, Total Cancer Care™ collaborators have generated a vast information system to be leveraged as a clinical decision tool, and as a means of quality performance and comparative effectiveness research (CER).

One of the stated aims of Total Cancer Care™ is to raise the standard of care for all patients by integrating new technologies in an evidence-based approach to maximize benefits and reduce costs.  Although we developed this aim over seven years ago, I believe it is completely consistent with the current definition of comparative effectiveness being used by AHRQ and other policymakers. 

As I mentioned in my previous entry, strategic partnerships are an essential component to achieving the goals of Total Cancer Care™, and this is clearly demonstrated in our efforts in CER. Dr. David Fenstermacher and colleagues from Moffitt as well as the Institute of Human and  Machine  Cognition (IHMC), in Pensacola, Fla., are collaborating on a major NIH/NCI grant to enhance the Total Cancer Care™ infrastructure to support CER by expanding data management resources, integrating  automated data extraction methodologies (including natural language processing technology a particular area of expertise for IHMC), and creating user interfaces to data for researchers, clinicians and even patients. 

A major focus of our current efforts in CER is to determine the information and technology gaps in the CER infrastructure for data capture and data sharing.  Ultimately, it will be important to involve the community at large who are enrolling patients in the Total Cancer Care™ Protocol so that they can use the Total Cancer Care™ data warehouse as a decision tool based on evidence generated by the study itself.  The importance of the community network cannot be over emphasized both for populating the Total Cancer Care™ biorepository and database, and the ultimate utilization of the information and evidence generated for delivering the right treatment for the right patient.

To enhance Moffit’ts ability to establish this large research initiative the cancer center formed a wholly owned for-profit company, M2Gen, in 2006.  Merck and Co., Inc., through a Merck affiliate, signed on as our ”Founding Collaborator”. This experience has taught us how to service a global healthcare client and produce measurable scientific insights to accelerate drug candidates through translational medicine advances.

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

September 23, 2010

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

September 20, 2010

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.

* * *

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
Personalized Medicine Coalition
Washington, Sept. 13, 2010

* * *

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

September 10, 2010

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-1st RI) partnered with Rep. Anna Eshoo (D-14th 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.

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