Policy Update Archive - 2010

December 7, 2010

Issue Brief Highlights Need for Update of Reimbursement System
A discord exists between the rapidly increasing number and therapeutic power of personalized medicine diagnostics (PMDs) and a payment system that does not recognize their clinical utility or economic value, according to an issue brief released in December by the Personalized Medicine Coalition and Boston Healthcare Associates.

The brief, The Adverse Impact of the US Reimbursement System on the Development and Adoption of Personalized Medicine Diagnostics, outlines systemic problems facing reimbursement for PMDs. “Because appropriate reimbursement results from a complex interplay of codes, coverage decisions, policies, and payment amounts, addressing any of these aspects individually is inadequate. Instead, obtaining the full benefits of PMDs will require a fundamentally different reimbursement paradigm,” writes report author David Parker, Ph.D., Vice President of Boston Healthcare Associates.  

The issue brief was the product of a working group that met during the summer of 2010 to focus on Medicare reimbursement issues. The working group, co-chaired by Scott Allocco of BioMarker Strategies and Steve Phillips of Johnson & Johnson, included representatives from more than 30 diverse PMC member organizations. The issue brief is available for download at http://www.personalizedmedicinecoalition.org/.

November 16, 2010

Study Confirms Pharmaceutical Industry’s Commitment to Personalized Medicine
An increased number of personalized medicine products are in the drug pipelines of large pharmaceutical and biotechnology companies, according to a study released by the Tufts Center for the Study of Drug Development. Between 12 and 50 percent of current pipelines comprise personalized medicine products, said the 25 companies in the study, which was released last November. The study projected that investment in personalized medicine product development would increase by an average of 53 percent over the next five years.

October 25, 2010

Draft Guidance for Industry on Qualification Process for Drug Development Tools; Availability
The Food and Drug Administration (FDA) announced the availability of a draft guidance for industry entitled “Qualification Process for Drug Development Tools.” This draft guidance describes the qualification process for drug development tools (DDTs) intended for potential use, over time, in multiple drug development programs. The draft guidance provides a framework for interactions between the Center for Drug Evaluation and Research (CDER) and DDT sponsors to support work towards qualification of an identified DDT and creates a mechanism for formal review of data by CDER to qualify the DDT and ensure that the evaluation is comprehensive and reliable.

Although you can comment on any guidance at any time (see21 CFR 10.115(g)(5)), to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit either electronic or written comments on the draft guidance by January 24, 2011. To view the document, please click the following link: Qualification Process for Drug Development Tools (PDF -190KB). To view the full notice from the FDA, visit: http://edocket.access.gpo.gov/2010/2010-26927.htm.

October 8, 2010

FDA Releases Regulatory Science Framework
At a National Press Club luncheon on October 6, 2010, Food and Drug Administration (FDA) Commissioner Margaret Hamburg released a strategic framework for FDA’s initiative to improve regulatory science and to create a new office dedicated to it.

The framework, outlined in a paper titled “Advancing Regulatory Science for Public Health,” seeks to improve FDA’s ability to effectively evaluate and regulate emerging products in the genomics and personalized medicine field, among other fast-changing areas of health care innovation. The goal of this effort, Hamburg said, “is to reduce the gap between the advances in science and the practice of patient care.”

Commissioner Hamburg pointed to the need to utilize the breakthroughs in genomics and tailored therapies to improve patient care. In particular, the new initiative will seek to expand collaborations with industry, academia, and other government entities to improve product development process and speed. It is hoped that this will reduce the time currently needed to bring new innovations into clinical practice.

Recognizing that innovation is increasingly focused on personalized medicine, these changes are designed to open the door to smaller, shorter clinical trials for targeted therapies, to speed the approval of new drugs, especially cancer drugs, for individuals with specific biomarkers.

In the words of the Commissioner, the FDA wishes to pragmatically evolve regulatory science to enable “21st century medicine” and “move out of a one-size-fits-all” paradigm of regulation.

September 28, 2010

EHC Program Update: Clinical Heterogeneity Methods Report Is Now Available
The Agency for Healthcare Research and Quality (AHRQ) Effective Health Care (EHC) Program is pleased to announce that a report titled Comparative Effectiveness Review Methods: Clinical Heterogeneity is now available.

To access the report and associated products, please visit: http://www.effectivehealthcare.ahrq.gov/index.cfm/search-for-guides-reviews-and-reports/?pageaction=displayproduct&productid=533.

We are taking a survey of our guides! If you use our consumer or clinician guides, take a moment to complete our online survey. Just look for and click on the “Take our Survey” button for the guide(s) you use. To see the clinician guides in the survey, visit: http://effectivehealthcare.ahrq.gov/index.cfm/guides-cmece-and-other-resources-for-clinicians/. To see the consumer guides, visit: http://effectivehealthcare.ahrq.gov/index.cfm/guides-for-patients-and-consumers/.

Learn how you can personalize your interactions with and share what you learn on the EHC Program Web site. Visit: http://www.effectivehealthcare.ahrq.gov/index.cfm/personalization-and-social-media-tools/.

We welcome your comments on the EHC Program Web site! Please use our “Contact Us” form available at: http://www.effectivehealthcare.ahrq.gov/index.cfm/contact-the-effective-health-care-program/.

Thank you,
Effective Health Care Program
http://www.effectivehealthcare.ahrq.gov
 

September 23, 2010

Personalized Medicine Coalition Nominee Selected For PCORI Board of Governors

The Personalized Medicine Coalition (PMC) is pleased that the Government Accountability Office (GAO) included a PMC nominee with expertise in and a commitment to personalized medicine as a member of the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors. PMC nominated a slate of individuals representing the diversity of our coalition that included representatives from patient advocacy groups, hospitals and health systems, the diagnostics industry, and public health agencies. Picked by GAO from among our nominees, Freda Lewis-Hall, MD, Chief Medical Officer for Pfizer was selected for her commitment to and knowledge of both comparative effectiveness research and personalized medicine. The 21-member PCORI Board of Governors, established by the Patient Protection and Affordable Care Act, is tasked with assisting patients, clinicians, purchasers, and policy-makers in making informed, evidence-based, health decisions.

August 30, 2010

Notice of Meeting: Secretary’s Advisory Committee on Genetics, Health, and Society

Pursuant to Public Law 92-463, notice is hereby given of the twenty-third meeting of the Secretary’s Advisory Committee on Genetics, Health, and Society (SACGHS), U.S. Public Health Service. The meeting will be held from 8:30 a.m. to approximately 5:30 p.m. on Tuesday, October 5, 2010, and from 8:30 a.m. to approximately 3:45 p.m. on Wednesday, October 6, 2010, at the National Institute of Health, Building 31, Conference Room 6C6, 9000 Rockville Pike, Bethesda, MD 20892. The meeting will be open to the public with attendance limited to space available. The meeting will also be Web cast.

The main agenda item will be a review of the revised draft report on genetics education and training and discussion of the final draft recommendations. The meeting will also include sessions on genomic data sharing and the implications of affordable whole-genome sequencing, an update on the implementation of the Genetic Information Nondiscrimination Act, and a briefing from the Food and Drug Administration on activities related to genetic testing.

Individuals who would like to provide public comment should notify the SACGHS Executive Secretary, Ms. Sarah Carr, by telephone at 301-496-9838 or e-mail at carrs@od.nih.gov.

The draft meeting agenda and other information about SACGHS, including information about access to the Web cast, will be available at the following Web site: http://oba.od.nih.gov/SACGHS/sacghs_meetings.html.

July 27, 2010

House Subcommittee on Oversight and Investigations Holds Hearing on DTC Tests

On July 22, the House Energy and Commerce Subcommittee on Oversight and Investigations held a hearing on “Direct-To-Consumer Genetic Testing and the Consequences to the Public Health,” where members raised concerns about DTC testing including: the validity of the tests, the unintended consequences of the tests stemming from misinterpretation and incorrect results, the protection of patients’ privacy, and the lack of current data for ethnic minorities. The Subcommittee heard testimony from Gregory Kutz of the Government Accountability Office, which had conducted an investigation of the tests offered by 23andMe, Navigenics, Pathway Genomics, and deCODE Genetics. Jeffrey Shuren, M.D., Director, Center for Devices and Radiological Health, Food and Drug Administration, testified that the FDA is taking the initiative to deftly oversee these tests, and he believes that “smart regulation can encourage innovation and more investment into the field” as all companies would then be held to a higher standard. Ranking Member, Representative Michael Burgess (R-26^th TX) noted that “the science will get there, the FDA needs to be ready to regulate it.” Navigenics’ President and Chief Executive Officer, Vance Vanier, M.D., responded to the Subcommittee stating that these tests are “probabilistic not predictive” and addressed the other concerns of the committee. He was joined by representatives from 23andMe and Pathway Genomics, who all welcomed improving genomic testing, which the Subcommittee recognized could “revolutionize the daily practice of medicine.” 

FDA Holds Two Day Meeting on LDTs

The FDA/Center for Devices and Radiological Health hosted a public meeting July 19-20 to discuss and solicit feedback on “Oversight of Laboratory Developed Tests.” Wayne Rosenkrans, PMC Chairman, participated as a panelist in the Direct-to-Consumer (DTC) Marketing of Testing session on July 20. This meeting comes as the FDA has taken a closer look at genetic testing, including sending letters to fourteen manufacturers regarding the possibility that their products fall into the medical device category. PMC will continue to monitor and provide updates regarding the actions of the FDA on laboratory developed tests and DTC genetic testing. To view GenomeWeb’s full story on the meeting, click here.

June 3, 2010

FDA Official Speaks with Policy and Science Committees, Task Forces Move Forward
Vicki Seyfert-Margolis, Ph.D., Senior Advisor, Science Innovation and Policy, Office of the Chief Scientist, Office of the Commissioner, Food and Drug Administration (FDA), spoke to a joint meeting of the PMC Policy and Science Committees on June 3. Dr. Seyfert-Margolis reiterated the FDA's promise to release new companion diagnostics guidance before the end of the year, as well as comparative effectiveness research guidance. She also discussed the FDA's advances in regulatory science, “collaborating in key areas of overlap between the various subsets of the FDA," and their goal to "interrogate data during clinical trials to analyze results by subpopulations." 

The co-chairs of PMC’s Centers for Medicare and Medicaid Services (CMS) Coverage, Coding and Reimbursement Task Force, Scott Allocco of BioMarker Strategies and Steve Johnson of Johnson & Johnson, discussed task force efforts to create an issues brief that clearly outlines reimbursement challenges for personalized medicine products. David Parker of Boston Healthcare Associates, who facilitates the task force calls and will author the issues brief, discussed the importance of this work given the personalized medicine provisions in health care reform and the reintroduction of Genomics and Personalized Medicine Act (GPMA).

The Personalized Medicine Coalition has formed a health information technology (HIT) task force, chaired by Tony Hess of SAIC and staffed by Marcia Kean of Feinstein Kean Healthcare. The task force is working with Darrell West, Vice President, Governance Studies of the Brookings Institution, to issue a white paper this fall. PMC believes this is a critical opportunity to inform HIT policy makers, as policy in this area has largely ignored the principles of personalized medicine in the past.

Genomics and Personalized Medicine Act Reintroduced
The Genomics and Personalized Medicine Act (GPMA) was reintroduced on May 27 by Reps. Patrick Kennedy and Anna Eshoo. PMC applauds the reintroduction of this legislation, as it calls for organizing personalized medicine activities at the Department of Health and Human Services, funds biobanking activities and allocates funds for personalized medicine research and educational activities. It is a needed step to advance legislation to include tangible incentives for personalized medicine, such as tax incentives, and shift from price-controlled testing to value-based testing. PMC is encouraged by the co-sponsorship of Rep. Eshoo given her focus in life sciences.

April 20, 2010

CMS Lacks Clear Guidance on Reimbursement, Policy Committee Says
Members of the Personalized Medicine Coalition’s Public Policy Committee told officials from the Centers for Medicare and Medicaid Services (CMS) at a meeting on February 24 that guidance is lacking on the scientific evidence CMS requires to approve reimbursement of a genetic test. Jim Rowlands, M.D. and Jeffrey Roche, M.D., medical officers in CMS’s Office of Clinical Standards and Quality, told the committee they want to “ensure that Medicare beneficiaries have access to any demonstrated improved health outcomes of pharmacogenomic testing.” The Public Policy Committee has formed a workgroup comprising a broad variety of PMC members to express its concerns to CMS in an effort to help the agency clarify its regulations.

PMC Committee to Propose Changes to GPMA
Members of PMC’s Public Policy Committee in March discussed proposed changes to the Genomics and Personalized Medicine Act. Mark Hoeft, M.D., a legislative fellow for Representative Patrick Kennedy, D-R.I., sought the committee’s help at its December 15 meeting. PMC members praised the draft’s codification of the Personalized Healthcare Initiative and its funding for workforce training and genomics research. Without business incentives, however, they said it is difficult to overcome the barriers to personalized-medicine adoption that result from existing business models. PMC will continue to advocate for provisions such as a research and development tax credit and patent extension in the Senate bill.

HHS Office to Meet in April on Policy Framework for HIT
The Office of the National Coordinator for Health Information Technology at the Department of Health and Human Services will hold seven workgroup meetings in April to discuss a policy framework for a nationwide infrastructure that permits the electronic exchange of health information. The announcement of the workshops comes as PMC has asked the Brookings Institution to write a new white paper on the intersection between health information technology and personalized medicine. For further information, contact Judy Sparrow at 202-205-4528 or judy.sparrow@hhs.gov.