A Holiday Reading List from The Age of Personalized Medicine Blog

As we head into the final weeks of 2009, I wanted to share some recent articles and reports that you might want to read between glasses of eggnog and gatherings with family and friends. As the year draws to a close, these articles point to the tremendous opportunities that are ahead for personalized medicine, as well as the challenges in policy, business, and health care practice .

1) ObamaCare Threatens Personalized Medicine, Forbes – Dec 21, 2009 

Gregory Conko of the Competitive Enterprise Institute and Henry I. Miller, a physician and a fellow at Stanford University’s Hoover Institution, wrote a compelling op-ed this week on a topic we have discussed prominently at this blog – the need to align priorities for health care reform, particularly comparative effectiveness research, and personalized medicine. Conko and Miller point to the importance of taking into account the effects of a medicine on sub-populations and how the value of medicine (in terms of diversifying treatment indications) can increase over time. An approach to CER that lacks emphasis on such important advances may stunt the growth of personalized medicine. 

As Tony Coelho pointed out at this blog last week, the language in the Senate version of the health care bill does provide for an approach to CER that embraces patient differences and focuses on providing information that will enable patients and providers to make more-informed decisions. We’ll be standing by on Christmas Eve to see if this is what we get! 
 

2) Welcome to the Era of Personalized Medicine, Huffington Post – Dec 22, 2009 

In this article, WIRED Editor Thomas Goetz suggests that the era of personalized medicine is not only about advances in pharmacogenomics, but also about how advances in bioinformatics and consumer-oriented tools are generating huge amounts of data that can inform a more personalized approach to care. Goetz contends, “Personalized medicine isn’t something that happens to us; it’s something that we have to choose to engage in.” With such an engaged patient population emerging, health care providers also need to consider how interactions will change with patients bringing self-generated records and research into discussions about their health care.  

I look forward to seeing Goetz’s book The Decision Tree: Taking Control of Your Health in the New Era of Personalized Medicine in February. You can also check out the Decision Tree blog to hear more of his thoughts on predictive medicine and the future of health care. 
 

3) Personalized Medicine Market To Grow 11% Annually, Pharmaceutical Executive – Dec 15, 2009 

Earlier this month, PricewaterhouseCoopers released The new science of personalized medicine: Translating the promise into practice, highlighting how the “disruptive innovation” of personalized medicine is creating opportunities and challenges for traditional health care practice. In case you won’t have a chance to read the 50-page report, this article from Pharm Exec gives a great summary of its salient conclusions. Echoing Goetz’s suggestion that personalized medicine encompasses consumer-oriented products and services, the report contends that a growing emphasis on prevention and wellness is paving the way for advances in personalized medicine.

Personalized Medicine and Health Care Reform: Looking Back, Looking Ahead

Will health care reform support personalized medicine?  In my mind, that depends on two important factors: 1) the extent to which health care reform is truly patient-centered (does it make room for patient differences, room for patient voices, and time for patient care?) and, 2) the extent to which it is innovation-friendly.

I’m focusing on the first topic in this post.  Earlier posts have rightly focused on comparative effectiveness research as one key provision.  If CER is structured correctly, it can help inform patients about optimal medical and health care options based on our differing needs.  These differences come from a number of factors, including different clinical conditions we may have, differences in our preferences and the way we view risk/benefit trade-offs, cultural differences, and certainly molecular differences.  CER structured to recognize, and respect, these differences can only accelerate the move to personalized care.  Yet it remains unclear if this is the kind of CER we’ll get.  I think only the Senate bill’s CER language gets us close to this goal, by fully including patients and providers in the process, fully embracing patient differences, and focusing the program on results communications and not national policy recommendations. 

CER is one of several aspects of health care reform that will have an impact on patient-centeredness and personalized medicine.  Just as important are provisions that will apply the scientific evidence to policy decisions.  This includes proposals to establish an independent Medicare advisory board, define physician “best practices” and performance standards, and establish standards for use of health information technology.   

These types of policies, when deployed as cost-control levers, could be one of the single biggest factors influencing the future of personalized medicine.  Payment policy measures designed to control costs and expand access—but that fail to encourage continued development and adoption of personalized medicine—could substantially delay or diminish opportunities for meaningful, measurable improvements in health care value and quality.

That’s because cost containment proposals that impose access restrictions based on average, population-wide study results risk ignoring the different needs of individual patients and discouraging adoption of personalized tests and therapies based on these differences.  For example, “pay for performance” programs focused on short-term provider efficiency could discourage physicians from using gene-based tests and targeted therapies to optimize care for the individual.  As a patient with epilepsy myself, I take a very personal interest in how these policies get developed and applied.  

This nexus of CER and policy levers was highlighted earlier this year in an NPR commentary from Anne Brewster, an internist and instructor at Harvard Medical School.  “Physicians may agree with the end goal, but many of us worry about the methods and unintended consequences.  Comparative effectiveness research sounds sensible.  Of course we need more studies to define best practices.  But I find myself afraid that the results will be used by policy makers, hospital administrators, and lawyers to further limit my autonomy by setting hard and fast rules about what is “right”.  Clinical situations are always nuanced, never black and white.  Perhaps it is semantic, but I want to feel that CER will empower rather than constrain me.” 

Enacting, and implementing, health care policies that support patient-centered care and the science of personalized medicine won’t be easy; but it is absolutely essential.  Let’s work together to make it happen.

By Tony Coelho, Chairperson, Partnership for Improved Patient Care

Companion Diagnostics Regulation Clarification: On the Horizon at the Food and Drug Administration

Federal regulation of diagnostic tests used in companion therapeutic-diagnostic products is seen by some to be a barrier for personalized medicine.  Others argue that definition of a clear, reasonable regulatory pathway for these tests will remove a barrier to personalized medicine. Today, drugs are regulated by one part of the Food and Drug Administration (FDA), lab kits (lab tests sold packaged in ‘boxes’) by another part of FDA, and laboratory test service companies are regulated by the Center for Medicare and Medicaid Services.  Companion diagnostics (lab tests and kits designed inform drug selection or dosage) pose a challenge to this system, and the opportunity to create a better one.

At the suggestion of the FDA, PMC convened a workgroup to suggest alterations to the agency’s 2005 white paper on the subject.  Stakeholders around the table sometimes had competing views, but all agreed that promoting high quality patient care is the central goal. This enabled the groups (including lab service companies, diagnostic kit manufactures, and pharmaceutical companies) to achieve consensus on several over-arching recommendations:

• Regulation must recognize the various types of companion products and work for all of them.
• Any oversight of in vitro diagnostic devices and laboratory-developed tests should be developed through an open and transparent process.
• Regulatory policy should promote rapid access to new diagnostic information to improve clinical care and sustain innovation.

Since FDA has suggested that it might pursue a series of white papers or hold a series of workshops on topics related to companion diagnostic regulation in advance of developing draft guidance on it, PMC also suggested a series of topics that might prove valuable to the community.  They are:

• Analysis of regulatory precedent for companion diagnostics.
• Review and analysis of evidence requirements for companion diagnostics and co-developed drug-diagnostic products.
• Intra-agency coordination among the office for drug regulation, the office for device regulation, and the Office of Combination Products.

The full text of the PMC’s submission to FDA can be found here: https://ageofpersonalizedmedicine.files.wordpress.com/2009/12/pmc-rx-dx-co-development-letter-to-fda-9dec09.pdf

Regulations to Implement Title I of GINA Move One Step Closer to Finalization

In October 2009, the Genetic Information Nondiscrimination Act (GINA) reached a major milestone when the agencies released the regulations outlining their interpretation of the protections afforded by Title I of this civil rights law.  The interagency team tasked with implementing GINA released these regulations as interim final, giving members of the regulated community six months to fully comply with GINA.  Despite this interim status, these regulations officially became effective on December 7, 2009. 

After the passage of GINA, the agencies solicited comments and information from the public to aid their drafting of the regulations.  In response to concerns about GINA limiting the use of genetic information in making payment determinations, the interim final regulations provide a safe harbor that allows insurers to condition payment on the outcome of a genetic test as long as this policy is applied uniformly to all plan members and uses the minimal amount of information necessary to make a determination.  For example, an insurer can request a beneficiary’s BRCA mutation status before covering the cost of a yearly mammogram starting at age 30. 

In October 2009, the Office of Civil Rights (OCR) at the Department of Health and Human Services (HHS) also released their regulations amending the Privacy Rule issued under the Health Insurance Portability and Accountability Act of 1996.  The regulations were released in draft form with a comment period expiring on December 7, 2009.  There are two sections of the draft regulations that prompted the most comments from the community.  First, the draft regulations extend the Title I protections beyond health insurers to long-term care policies, excepted benefit plans (vision, dental, etc.), the military and veteran’s health care plans, and others.  GINA advocates applauded this interpretation while others question whether or not this extension is consistent with the intent of Congress. 

The second area of contention has to do with the definition of underwriting as it applies to employer based wellness programs.  The OCR draft regulations allow the collection of genetic information (including family history) in wellness programs as long as the programs are voluntary.  They interpret GINA to prohibit the use of financial incentives beyond premium sharing, such as cash, gym memberships and other gifts, in exchange for the completion of a health risk assessment that collects genetic information.  The draft regulations view these incentives as penalties to those who choose to keep their genetic information private, and hence, the wellness program is not considered to be voluntary. 

Over 250 organizations advocating for GINA sent a letter to the agencies supporting their interpretation of underwriting as it applies to wellness programs.  At the same time, supporters of using financial incentives to encourage enrollment in employer based wellness programs that collect genetic information have also sent comments to the agencies requesting a modification to the draft regulations.  Additionally, they have tried to seek assistance from Congress, requesting an amendment to the pending health care reform legislation in the Senate to allow financial incentives when collecting genetic information in wellness programs.  With the comment period now closed, advocates on both sides of the issue patiently await the outcome of their lobbying efforts and the final OCR regulations.