Posts Tagged ‘Comparative effectiveness research’

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

Harnessing the Power of Health IT in a New Era of Translational Research

March 8, 2010

In the final years of the 20th century and the first decade of the 21st century, tremendous progress has been made toward bridging a recognized chasm between science and the real world, and specifically in medicine, between biomedical research and its application in healthcare.  Three identified “blocks” to translation have impeded the use of research findings to better the lot of our patients: T1, the translation of laboratory findings to clinical care, T2, the application of best evidence identified during T2 to everyday clinical care; and T3, wider generalization of research findings to improve the health of the community and, more broadly, the public.  The recent deluge of funding for comparative effectiveness research (CER) represents, in large part, an attempt to conquer T2 and T3.  T1, however, persists and presents a fundamental impediment to personalized medicine.

To overcome T1, and transfer T1 knowledge to T2, will require true integration of the clinical and research spheres – an integration that necessitates bidirectional information flow from the patient and physician in the clinic to the research scientist and back again, in an iterative cycle of hypothesis, question, answer, and testing of that answer in the real world setting.  To support this sort of information exchange, we will need: new coordinated health information technology (HIT) systems that span former “silos” in the biomedical community, and that can collect and manage large volumes of disparate and heterogeneous data; culture change that engages clinicians and researchers in a common mission of inquiry to improve care; communication channels that fuel hypothesis generation, and that support the translation of research findings into change in clinical practice, and; decision support mechanisms that help clinicians leverage the power of large-scale aggregated data to improve care for the individual patient.  In short, we need a new model of care, one that harnesses the potential of HIT and integrated clinical/research data to dismantle the T1 block. The purpose, fundamentally, of such a model will be to enable personalized medicine.

In advancing “rapid learning healthcare,” the Institute of Medicine has spearheaded the development of a new healthcare paradigm in which personalized medicine could become a reality.  Efforts are underway to develop this paradigm and its prerequisites.  As one such example, the Cancer Biomedical Informatics Grid (caBIG®) championed by the National Cancer Institute has tackled the development of an infrastructure promoting large-scale data interoperability spanning the data type boundaries from the basic sciences to clinical care and the patient report.  As we seek to match novel therapeutics and trials to patients, and to personalize care using individually relevant information, critical steps will be: (1) providing access to data, (2) generating data, and (3) making sense of the data.  Making sense of data needs to be facilitated at the levels of basic science (to guide translation of in silico research results into clinical practice change and further discovery), the population (to allow CER to guide health services decisions and policy), and the individual patient (to enable personalized medicine).  New data generated in any of these steps should be reinvested in the system to iteratively update the knowledge base.

The caBIG® experience has taught us that just having access to better HIT does not, in itself, advance personalized medicine.  Though a powerful tool, HIT alone is not enough to bulldoze the translation blocks.  Why?  Because healthcare is not a purely technical matter; rather, it is a human system, fundamentally dependent upon human understanding, acceptance, and behavior.  All of these must change in order to transform information flow through HIT, implement a new data-driven model of healthcare, and thus realize the vision of personalized medicine.  The individual stakeholders in medicine need to be aligned behind the new vision – through incentives to participation that speak to each one.  First, the new model needs to be structured, and to function, so that the HIT makes sense to real human beings using the system (clinicians, staff, patients, administrators, clinical researchers, basic scientists); HIT must represent “value added” to the existing system from the perspective of each stakeholder.  Second, interoperable data must be generated, so that the system has “grist for the mill” of inquiry; we have to start somewhere and someone needs to be encouraged to put their first big toe in the water — there is nothing like a “big story” to bring along the naysayers.  Third, to build confidence in the approach, we must make sure that privacy, confidentiality and the sanctity of personal health information are preserved. And fourth, novel ways to make sense of ever-growing databanks need to be developed; these methods may include new approaches for visualization, decision support systems, Bayesian and other branched analytic approaches, CER, and in silico research.  Current efforts focus on generating interoperable data (the middle step), but neglect to create systems that make sense of the data and promote its use, or that provide a structure and an engine to produce the data.

Finally, and, in my mind, most importantly, a critical next step is to define a reorganization of medicine at the point of care.  The new model must fully utilize available data and linked datasets, and must help clinicians understand the data and apply it in tailoring care to their individual patients.  If it makes sense to them, clinicians and patients will drive this.  In the background, interdigitated with the growing body of clinical experiences captured in linked clinical/research databases, will be the robust evidence base comprising published results of basic science, clinical research, and translational studies.  The resulting combination creates a system in which each patient’s care is guided by personal history and characteristics, the experiences of similar patients included in local and massive national longitudinal datasets, and the historical evidence base constituting an up-to-date state of the science.  Our challenge, today, is to develop this system beginning at the point of care, with the patient.

A Holiday Reading List from The Age of Personalized Medicine Blog

December 23, 2009

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

December 18, 2009

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

Aligning CER and PM – Progress on the Hill

November 5, 2009

The debate on comparative effectiveness research (CER) policy in the House of Representatives took two interesting turns over the last few days.  First, the House released its health reform bill (H.R. 3962) that combined three separate versions.  In the section related to CER, research on genetic and molecular sub-types was added to the scope of work for the Center for Comparative Effectiveness Research, some independence was added to the organization, and some patient protections were outlined. 

PMC sent a letter to House leadership on the bill.  In it, we laud their inclusion of language on genetic and molecular subpopulations as an important step forward for personalized medicine, and ask them to take the additional step of combining this language with a CER bill (H.R. 2502) introduced by Rep. Kurt Schrader (D-OR-5th) as the best approach to aligning PM and CER. 

In addition, Rep. Schrader sent a letter to colleagues stating his intention to continue pursuing his CER bill as an alternative to the House bill’s CER section.  His letter also highlighted the importance of aligning CER and PM. H.R. 2502 has a similar approach to CER as outlined in the Senate Finance version that recognizes personalized medicine throughout its structure. 

While the CER section is only a small part of a very big bill, the extent to which it aligns with personalized medicine could have a huge impact on continued progress in our field.

By Amy Miller, Public Policy Director, Personalized Medicine Coalition

CER and PM: Contradictory or Complementary?

October 28, 2009

Comparative effectiveness research (CER) is poised to play a major role in the promotion of a personalized approach to health care. Done right, CER could do more to advance personalized medicine than any other policy under consideration right now. Done wrong, it could lock us into 20th century medicine and discourage innovation. Congress is considering two, very different policies for conducting CER. One allows for personalized medicine and medical innovation to emerge, the other would take us down the path of traditional, “one-size-fits-all” medicine.

The Senate Finance Committee’s approach – which incorporates the science of personalized medicine throughout the process and builds an independent Institute to make the most of scientific expertise in the public and private sectors – is an important step towards aligning CER and PM.  Researchers, policymakers, patients, and caregivers must continue working together to define pathways in support of personalized medicine. In contrast, other CER proposals in the Senate HELP bill and the House bill (H.R. 3200) provide more funds to conduct existing CER programs, which usually leads to broad, population-based conclusions.

Today, in concert with the release of the Comparative Effectiveness Research and Personalized Medicine: From Contradiction to Synergy report, the Personalized Medicine Coalition and National Pharmaceutical Councilare hosting a conference that we hope will point the way forward to evidence-based, personalized medicine. Titled Comparative Effectiveness Research and Personalized Medicine: Policy, Science, and Business, this conference has attracted leaders in all three sectors to discuss the topic. We will consider the two policies currently being debated, and discuss the scientific possibilities and business implications of both policies.

This conference addresses just one of several topics ripe for discussion at the intersection of personalized medicine and health policy. This blog will continue to explore policy, science, and business topics of interest to proponents of personalized medicine. We will explore the barriers to personalized medicine, and ways around them. We hope you will join the conversation.

By Amy Miller, Public Policy Director, Personalized Medicine Coalition

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