A review of the recent literature confirms that “comparative effectiveness” research—studies designed to help physicians use existing treatments more effectively—is severely lacking.
Fewer than a third of the studies published in the six top journals qualified as comparative effectiveness (CE) research. This finding “supports concerns that only limited clinical research is currently devoted to helping physicians” improve the use of existing therapies and determine which interventions and strategies are the most effective and safe, and the least costly, said Dr. Michael Hochman and Dr. Danny McCormick of Cambridge (Mass.) Health Alliance and Harvard Medical School, Boston.
Congress recently passed legislation to provide more than $1 billion to support CE studies, and President Obama's budget for 2011 recommends further funding of CE research. Noting that few data are available on the current status of CE research, the investigators reviewed all clinical medication-assessment studies published between June 2008 and October 2009 in the New England Journal of Medicine, Lancet, JAMA, Annals of Internal Medicine, British Medical Journal, and Archives of Internal Medicine. These publications “are the most widely read, quoted, and covered by the media, and thus are disproportionately likely to influence clinicians,” the investigators said (JAMA 2010;303:951–8).
Of the 328 randomized trials, observational studies, or meta-analyses, only 104 (32%) were CE studies. Only 11% of the CE studies compared medications with nonpharmacologic treatments, confirming a relative lack of such research. Such studies are important because they help clinicians “make fundamental therapeutic decisions,” the investigators said.
Nearly 90% of the CE studies relied on noncommercial funding, primarily from government sources, a finding that highlights how essential such funding is. “Commercial entities presumably devote much of their research to the development of novel therapies and to funding inactive-comparator studies aimed at expanding indications for their products,” they noted.
More than half of the randomized trials in this analysis used an “inactive comparator” such as placebo, rather than comparing a medication against existing treatments. Such trials were disproportionately funded by commercial sources and were disproportionately likely to show that a medication produced positive results. In addition, 24% of the randomized trials that did use an active comparator sought to demonstrate only the noninferiority of a medication to that comparator; there was no effort to clarify the optimal therapy, only to test equivalency. Such trials were exclusively funded by commercial sources.
Only 19% of the CE studies focused on patient safety, which implies that safety concerns are not adequately emphasized in medication studies.
Only 2% of the CE studies and 1% of all studies in the analysis included formal cost-effectiveness analyses, which are critical to promoting efficient health care.
Overall, the findings “underscore the importance of the recent legislation passed in the United States to expand public funding for CE studies. In particular, our findings suggest government and noncommercial support should be increased for studies involving nonpharmacologic therapies, for studies comparing different therapeutic strategies, and for studies focusing on the comparative safety and cost of different therapies,” Dr. Hochman and Dr. McCormick said.
Disclosures: The investigators reported no financial conflicts of interest.