How CMS and the FDA May Respond to the Evidence Gap

Jennifer Erickstad

February 2011, Vol 2, No 1 -


San Diego, CA—Citing a “significant level of concern about how clinical trials are conducted in this country,” Steve Phurrough, MD, MPA, chief operating officer and senior clinical director at the Center for Medical Technology Policy, discussed the Centers for Medicare & Medicaid Services (CMS) and the US Food and Drug Admin istration (FDA), speculating on how these agencies may change their policies to help “rectify some of the evidence gaps” in trials of new technologies.

Approximately 18,000 randomized controlled trials are performed each year, with thousands of other types of studies adding to the literature, yet “new technologies are rapidly spreading without sufficient data,” said Dr Phurrough, former director of coverage and analysis at CMS.

To show conference attendees how the field of radiation oncology can better develop this sufficient evidence, Dr Phurrough discussed at the annual meeting of the American Society for Radiation Oncology (ASTRO) what the FDA and CMS will be looking for in their technology assessments.

Sufficient Evidence Is Necessary
Dr Phurrough explained that the FDA is currently undergoing 510(k) reform, which will bring about a higher level of evidence required for device ap provals. Specifically, greater evidence will likely be needed to prove a device is the substantial equivalent of an already marketed product, with special attention paid to demonstrating parallel outcomes.

Similarly, Dr Phurrough estimated that greater weight will also be placed on outcomes in the premarket approval process, noting that the FDA is “quite interested in seeing if technologies will change outcomes, not just [asking] ‘does it do what it is supposed to do?’”

CMS and CER
CMS will likely focus on using the coverage process to improve quality, Dr Phurrough suggested. The agency’s current standard is to cover anything that is “reasonable and necessary,” yet these terms have never been formally defined, leaving individual national coverage determinations (NCDs) to provide clarification.

As a result of this ambiguity, Dr Phurrough wagered that if Medicare were to conduct NCDs around radiation therapy, “a significant number of therapies would not have sufficient evidence to meet their reasonable and necessary standard.”

To use the coverage process to influence care quality, Dr Phurrough believes CMS will likely continue to use “coverage with evidence development”— which provides treatment coverage only to those in a trial or other kind of research program—and may, despite significant industry concern, also use “conditional coverage,” in which a device is quickly brought to market and reimbursed but can be removed if subsequent data collection does not provide confirmatory benefit.

Dr Phurrough also examined the question of whether CMS will require comparative effectiveness research (CER) for its coverage decisions. He thinks that CMS is unlikely to use CER in a type of “winners and losers” fashion, where the better-performing treatment would be covered and the lesser-performing intervention would not. “Most CMS coverage decisions are based on evidence that something does or does not work, not that it works better or worse than something else,” he explained. Dr Phurrough believes CER “should certainly be used as a payment tool, and if A is better than B, then A gets some incentives or B gets some disincentives.”