SEPTEMBER 26, 2012

Welcome to the Comparative Effectiveness Update eNewsletter
Editor: Philip L. Ronning
This issue is sponsored by the National Pharma Council's "Myth of Average" Conference

Patient-Centered Outcomes Research Institute Seeks Applications to Fund up to $96 Million in Comparative Clinical Effectiveness Research
The Patient-Centered Outcomes Research Institute (PCORI), the entity established by the Affordable Care Act to fund effectiveness research, has launched the second cycle of soliciting grant requests. Applicants in this round will be competing for up to $96 million in comparative effectiveness research grants. Joe Selby, MD, MPH, PCORI's Executive Director, said, "PCORI continues to build a portfolio of research across a variety of conditions and treatments that will provide patients and those who care for them with information that they can help them make complex prevention, diagnostic and treatment choices. (O)ur latest funding cycle seeks applications for rigorously designed studies that involve patients and stakeholders on the research team and that answer questions of greatest concern to patients." This Funding Announcement (PFA) corresponds with four of the five National Priorities for Research established by PCORI including 1) Assessment of Prevention, Diagnosis, and Treatment Options, 2) Improving Healthcare Systems, 3) Communication and Dissemination, and 4) Addressing Disparities. (redOrbit, September 17, 2012)

FDA Pushes Device Surveillance Plan that Could Involve PCORI; Off-Label, Data Access Debated
The Food and Drug Administration (FDA) is continuing to push for a "postmarket surveillance monitoring system" using Unique Device Identifiers that will be included in medical records, registries and other adverse event reporting data bases. Joe Selby, MD, MPH, has expressed interest in PCORI becoming involved in the discussion to advocate for including patients in the discussion and to assist by providing analytic methods. Amid wide-spread initial support from consumers and academics, concerns have arisen over off-label uses and access to the data. Paul Brown, the Government Relations Manager for the National Research Center for Women and Families, argued that the system could provide an incentive for companies to not conduct clinical trials thereby exacerbating problems with off-label promotion. (Inside Health Policy, September 11, 2012)

DCI and UNM Health Sciences to Partner in Creating Kidney Research Institute
Dialysis Clinics, Inc. (DCI) and the University of New Mexico's Department of Internal Medicine are forming the UNM Kidney Research Institute in Albuquerque. The Institute is being funded through a $6 million gift from DCI. The Institute's focus will be on comparative effectiveness research related to Nephrology and Kidney Disease. (Renal Business Today, September 18, 2012)

A Look into the Integrated Healthcare Policy Consortium and U.S. Healthcare
Bill Reddy, LAc, Dipl. Ac., the author, is a long-time board member of the Integrated Healthcare Policy Consortium (IHPC), an association representing 420,000 licensed practitioners, including acupuncturists, naturopaths, chiropractors, midwives and massage therapists and MD/DO Homeopaths. Reddy reports that he has been "...very pleased with our progress in influencing the language of the Patient Protection and Affordable Care Act (ACA) and nominating key people with integrative backgrounds to committees involved in executing the spirit and letter of the law." The bulk of the article is an interview Reddy conducted with the IPHC's executive director, Alyssa Wostrel, MBA, DIHom, on her goals and views of US healthcare. A part of the IHPC strategy is to use Comparative Effectiveness Research (CER), which she says has been championed by IHPC, to advance the interests of the AOM (Acupuncture and Oriental Medicine) professions and their patients. The CER method will be helpful "... because instead of performing basic science to study the efficacy of acupuncture therapy - it's comparing AOM to 'standard care' which would include drugs and surgical procedures." (Acupuncture Today, October, 2012)

More Effectiveness Research Needed for Diabetes Treatments
A meta analysis of comparative effectiveness studies for two treatments (CSII and MDI) and two methods of monitoring blood glucose (SMBG and rt-CGM) was reported in the September 4, 2012 issue of the Annals of Internal Medicine. The researchers, who were led by Hsin-Chieh Yeh, PhD of Johns Hopkins University, say, "Although these technologies are widely used, their effectiveness has not been consistently demonstrated and the populations most likely to benefit are unclear." They go on to state, "Future research should include larger studies in populations in which diabetes is increasing -- elderly persons, persons with insulin-requiring type 2 diabetes and minority populations. Studies should report on important clinical outcomes, consider effects of adherence on outcomes and assess cost-effectiveness," the researchers concluded. (Cardiovascular Business, September 6, 2012)

How Do Oncologists Sort Through All the New Anticancer Drugs?
In an article related to last month's Comparative Effectiveness Update eNewsletter story Can Effective Cancer Care Become Affordable Again? Novel Approach to Health Economics Research (Volume 3, Issue 29, August 29, 2012) the former president of Pfizer R&D and now a senior partner at PureTech Ventures, wonders how oncologists can keep up with the rapid evolution of research "into the various molecular mechanistic pathways that cause the constellation of diseases known as 'cancer.'" Not only must physicians evaluate efficacy by evaluating many alternative and evolving medicines, but they should also be evaluating the economy of the medicines - they are expensive and many patients have limited resources. The author points to the Institute for Cancer Outcomes Research and Evaluation (ICORE) located at the Fred Hutchinson Cancer Research Center in Seattle, which is focusing efforts on understanding the cost effectiveness and economic implications of cancer care. For another story on the topic see "A Lonely Voice Asking Tough Questions on Cancer Cost-Effectiveness" at http://www.xconomy.com/national/2012/07/30/a-lonely-voice-asking-tough-questions-on-cancer-cost-effectiveness/. (Forbes, August 22, 2012)

Large Data Sets in Primary Care Research
Electronic Health Records, (EHRs) are giving researchers in more settings than simply those of large managed care plans growing access to large data sets. The University of Wisconsin -- Department of Family Medicine began a research project asking the question, "What are the patterns and predictors of medication use in our family medicine clinics?" Previous studies had been limited to small sample sizes and principally elderly populations. The study in question analyzed nearly 2 million unique pieces of data from more than 110,000 patients in a network of 28 ambulatory clinics. The authors concluded, "Academic family medicine programs are ideally situated to perform influential studies on population health, treatment effectiveness, disease prognosis, and social determinants of health. This research will not only enhance our understanding of disease, but shape how we practice medicine in the future." (Annals of Family Medicine, September/October 2012)

Leading Researchers Outline Principles for Conduct of Comparative Effectiveness Research
Comparative Effectiveness Research (CER) may find increased consistency if principles developed by a group of leading researchers are successfully applied. So says a study published in The Journal of Comparative Effectiveness Research. "These are voluntary principles that can help to improve the quality of CER, which in turn can help patients and providers make better treatment decisions," said Bryan R. Luce, PhD, MBA, of United BioSource Corporation and the University of Washington, the article's principal author. The thirteen principles are:

  • Study objectives: The objective of a CER study should be meaningful, explicitly stated and relevant for informing important clinical or health care decisions
  • Stakeholders: All relevant stakeholders should, to the extent feasible, be actively engaged, or at least consulted and informed, during key stages of a CER study
  • Perspective: CER studies should address the perspectives of affected decision makers
  • Relevance: From planning to conduct, study relevance should be evaluated in light of decision maker needs
  • Bias and transparency: Attempts should be made to minimize potential bias in CER studies and to conduct them in a transparent manner
  • Broad consideration of alternatives: CER studies should make all reasonable efforts to include the full range of all relevant intervention, prevention, delivery, and organizational strategies
  • Outcomes: CER studies should evaluate those clinical, other health-related, and system outcomes most relevant to decision makers
  • Data: CER studies should take advantage of all relevant, available data, including information that becomes available during the course of the study
  • Methods: CER studies should incorporate appropriate methods for assessing outcomes of alternative interventions and intervention strategies
  • Heterogeneity: CER studies should identify and endeavor to evaluate intervention effects across patients, subpopulations, and systems
  • Uncertainty: CER studies should explicitly characterize the uncertainty in key study parameters and outcomes
  • Generalizability: CER studies should consider the generalizability and transferability of study findings across patients, settings, geography and systems of care
  • Follow-through: CER studies should include a plan for dissemination, implementation and evaluation (gnom.es, September 11, 2012)

Harnessing the Promise of Comparative Effectiveness Research

This Health Affairs Blog identifies four important challenges to harnessing the power of Comparative Effectiveness Research: 1) CER must strive to remain patient-centric, 2) the successful implementation of CER will require better decision support for physicians and some degree of utilization management, 3) CER must evolve beyond medications and procedures and evaluate the comparative effectiveness of health care systems, and 4) priority-setting for federally-funded CER should consider not only clinical priorities but the likelihood that evidence will be generated by private rather than federal funding (Health Affairs Blog, September 12, 2012)

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Comparative Effectiveness Research and Personalized Medicine

Amy Miller, PhD
Vice President, Public Policy, Personalized Medicine Coalition, Washington, DC