By Deborah Borfitz
March 3, 2008 | Since at least 2001, the volume of Phase IV studies has been growing more than 20 percent per year. The trend isn’t apt to change anytime soon, according to Richard Gliklich, MD, president and CEO of Cambridge, MA-based Outcome Sciences.
The latest push for post-approval studies came from congressional reauthorization of the Prescription Drug User Fee Act in September 2007, which expanded FDA resources for post-marketing surveillance to $29.3 million. Presumably, this will improve industry follow-through on post-approval commitments, Gliklich says.
At about the same time, the FDA was given new authority to use a range of regulatory tools to help assure drug safety. These include the ability to require post-marketing studies and special medication guides for patients as well as restrict which physicians can prescribe a drug. The authority gets exercised through a required “risk evaluation and mitigation strategy.”
Product globalization is also driving demand for post-approval studies, says Gliklich. Offshore countries desire “a safety profile within their own market.”
The latest report from the Institutes of Medicine, Knowing What Works in Healthcare: A Roadmap for the Nation, has stirred up discussion about the need for understanding drug and device effectiveness relative to other treatments. The report recommends creation of a new agency charged with the study of comparative effectiveness based on a systematic review of all scientific evidence.
The Congressional Budget Office has also concluded that the nation’s healthcare spending rate could be slowed by studying comparative effectiveness. “There is a lot of pressure now on manufacturers to produce evidence that their product not only works in the real world but that it is better than the alternatives.” says Gliklich.
During the post-approval phase, companies may need to start collecting new kinds of information to ensure their products are covered and appropriately reimbursed by Medicare and private health plans, says Gliklich. Since July 2006, Medicare has had two new national coverage categories: one stipulates further study of the effectiveness of a drug, device, or procedure, and another mandates outcomes data collection by procedure-performing centers. Consumers migrating into high-deductible plans may also start demanding information about the comparative effectiveness of products.
At Outcome Sciences, the number of patient registries is up more than 50 percent year over year, says Gliklich. Some sponsors also individually report that they are doing 20 percent more registry trials this year than last. Challenges include creating non-monetary value for sites to participate, such as quality-of-care measures they can use for other purposes. It is also critical that information technology be easy to use, since physician and nurses, rather than experienced study coordinators, often oversee data collection.
“What we’re seeing is more studies with mixed purposes,” says Gliklich. “[All] of the programs we launched last year have been for more than one purpose. Sponsors respond to safety requirements but take the opportunity to look at effectiveness, quality of life, and cost in the same study context.”
How to collect “good-quality evidence” is the topic of a how-to handbook, an effort led by Outcome Sciences and released by the Agency for Healthcare Research and Quality last May. “A number of pharmaceutical and device companies have incorporated parts or the entire book into their SOPs [standard operating procedures],” says Gliklich.
How information from observational research should be reported in the medical literature will be dictated, at some point, by the international collaborative initiative known as STROBE (STrengthening the Reporting of OBservational studies in Epidemiology), says Gliklich. “As soon as the International Council of Medical Journal Editors adopts [the STROBE Statement], it will essentially become mandatory.”
Editor’s Note: Outcomes Sciences will be supporting the Post-Approval Summit at Harvard Medical School on May 14-15. Topics include drug safety surveillance and comparative effectiveness. Details at www.postapproval.org.
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