AUGUST 29, 2012

Welcome to the Comparative Effectiveness Update eNewsletter
Editor: Philip L. Ronning
This issue is sponsored by the Comparative Effectiveness Summit

REMINDER: Public comments are due by September 14, 2012 on the Patient-Centered Outcomes Research Institute's (PCORI) Draft Methodolgy Report which can be found here: http://pcori.org/assets/MethodologyReport-Comment.pdf. The survey tool can be found here: http://www.pcori.org/survey/methodology-report/. Also refer to a short article by PCORI staff here: http://www.pcori.org/blog/why-methods-matter/.

Health IT's Next Big Challenge: Comparative Effectiveness Research
Comparative clinical research has traditionally relied on randomized controlled trials (RCTs) requiring a large group of control subjects who receive either the therapy under study or a placebo. RCTs are very expensive. Comparative effectiveness research compares two or more treatments to determine which is more effective. Medical informatics allows huge groups of patients to be studied by searching electronic records, often for a fraction of the cost of RCTs. According to Paul Cerrate, this article's author and Editor of InformationWeek Healthcare, "Given the fact that comparative effectiveness research will likely cost far less than a randomized clinical [sic] trial, it's time healthcare stakeholders take a closer look at this approach. The challenge for IT departments is going to be getting searchable patient data repositories up and running. Few hospitals have the resources to create their own version of Clinical Query. But at the very least, they need to start ramping up their data warehousing and data mining initiatives." (InformationWeek Healthcare, August 21, 2012)

FDA Balances Costs, Patient Safety in the Biologics and Personalized Medicine Revolution
"Biologics" are a new class of treatments made from living organisms intended to treat not the consequence of a disease, but rather to block the diseases in their early development. Biologics represent serious patient safety and expense concerns. The Hatch-Waxman Act of 1984 established the structure to bring generics to market after a patent exclusivity period. Consumer groups are pushing for biologics to be subject to similar regulations, resulting in biologics known as "biosimilars." The Affordable Care Act (ACA) clears the way for the FDA to create a "biosimilar pathway" with a 12 year patent exclusivity period. The problem is patient safety. "Because biologics are created using DNA technology, rather than a uniform chemical reaction with a predictable outcome as are traditional drugs, their risks are significant and often unpredictable. How can the government ensure that biosimilars are as safe and as effective as the original innovator drug?" The question at hand is "scientific equivalency" or ensuring the quality of biosimilars. (Forbes, July 23, 2012)

Sorting Out Options on Breast Cancer Screening
An estimated one in eight women will develop breast cancer in their lifetime, with the risk increasing with age. Mammography has been recommended for screening all women over 40, but with no agreement on the frequency of the tests or the specific commencement date of such screening. The Comparative Effectiveness Research on Cancer in Texas (CERCIT) is studying cancer screening. See this article for links to their research. For now, women and their doctors should discuss the risks and benefits of screening. "The decision should be based in large part on patient preferences, risk factors, age and overall health -- not blanket screening recommendations." (Chron.com, August 14, 2012)

Can Effective Cancer Care Become Affordable Again? Novel Approach to Health Economics Research
Seattle's Fred Hutchinson Cancer Research Center recently announced the launch of a first-of-its-kind research institute called the Institute for Cancer Outcomes Research and Evaluation. The Institute will be dedicated to cancer outcomes and health economics research. In this interview with the Institute's Director, Scott Ramsey, MD, PhD, he says, "We, at our Institute, have collected research showing patients with cancer are going bankrupt at unprecedented levels and many are experiencing severe financial strain. And for this reason we feel that the promise of cancer care is now being threatened by the cost of cancer care. It is our goal at this new Institute to try to reduce that trend." The Institute's intent is to collect information on cost and outcomes in order to identify the most cost-effective care and translate that into a national model. (cancernetwork.com, August 22, 2012)

Study: CT, MRI Use Dropped After 2005
After a decade of growth, MRI and CT use slowed between 2006 and 2009 to between 1% and 3% according to a July 25, 2012 Health Affairs article. The authors, from GE Healthcare and the Massachusetts Institute of technology, said this: "We hypothesize that higher cost sharing, prior authorization, reduced reimbursements, and fear of radiation are, for different parts of the population, countering some of the nonmedical incentives to order an imaging study." They concluded, "What has occurred in the imaging field suggests incentive-based cost control measures can be a useful complement to comparative effectiveness research when a procedure's ultimate clinical benefit is uncertain." (Modern Medicine, August 8, 2012)

Experts Say Ethical Dilemmas Contribute to 'Critical Weaknesses' in FDA Postmarket Oversight
The halting in September 2010 of the Avandia postmarket trial was according to the authors of an article in the New England Journal of Medicine's August 22, 2012 article entitled "Ethical Considerations in Studying Drug Safety -- The Institute of Medicine Report," "a lesson in how our current approach to the oversight of drug-safety and postmarketing research can fail both the public and the research participants." The Institute of Medicine's (IOM) May 22, 2012 report serves as the starting point for these authors as they highlight and expand on key findings and recommendations. Ethical issues vary widely, which makes context clarity crucial. The authors say, "Depending on who is initiating the research, for what reasons, and when, the same study design may have very different ramifications for the benefit--risk balance of the study and what patients need to know in order to provide meaningful informed consent." (Medical Express, August 22, 2012)

We'll Explain the Side Effects Later
A related story in Scientific American's Salon.com focuses on translating the risks of a randomized controlled trial (RCT) to patients and presents a much different picture on the article's findings. This story presents a hypothetical situation in which a patient is offered a new blockbuster drug under an RCT. "What you might not know--even after you sign up for the trial and have inked the informed-consent form--is that scattered reports are starting to suggest that the new medication might occasionally cause severe side effects. And the real reason the trial is being conducted with these previously released drugs is to test whether the new medication really is a lot riskier to everyone or just to a subset of patients. If you found that out, would you still sign up for the trial?" Ruth Faden, PhD, MPH, Director of the Johns Hopkins Berman Institute of Bioethics and co-author of the NEJM article cited in the above item believes you may not agree to participate in such a trial and therefore the rules surrounding such testing must change. "Now you've got this heightened ethical obligation toward these people," Faden says. "They need to understand why this particular trial is being conducted," and this step is often lacking in postmarket trials. (Salon.com, August 22, 2012)

Topol: Get Rid of the Randomized Trial; Here's a Better Way

Eric Topol, MD, Director of the Scripps Translational Science Institute and Editor-in-Chief of Medscape Genomic Medicine and theheart.org, offers his opinions on the future of research in this video and text from his series "The Creative Destruction of Medicine."

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Methods and Data Infrastructure Needed for Comparative Effectiveness Research

Sean Tunis, MD, MSc
President and CEO, Center for Medical Technology Policy