PHARMACOECONOMIC ANALYSIS PRINCIPLES REPORT FROM GOVERNMENT/INDUSTRY TASK FORCE WILL BE PUBLISHED IN AIM: FIRST ATTEMPT AT CONTROLLING BIAS IN PE STUDIES

A Leonard Davis Institute of Health Economics task force report on pharmacoeconomic analyses will be published soon in the Annals of Internal Medicine, Merck Human Health Division Outcomes Research and Management Director Marc Berger, MD, said March 17 at a Drug Information Association meeting on outcomes research methodologies in Vancouver.

The "Report on the Task Force on Principles of Economic Analysis" from LDI is the first cooperative public/private sector attempt to create guidelines for controlling bias in pharmacoeconomic studies. The report will contain a disclaimer to the effect that, although reviewed by the task force participants, they do not officially endorse the document. Nonetheless, it is understood that the task force sees the guidelines as a "white paper" and hopes the recommendations will be followed.

The institute, located at the University of Pennsylvania, formed the task force to develop guidelines on economic analyses two-and-a-half years ago. The group included 20 private and public sector researchers.

On the task force were representatives from HHS' Agency for Health Care Policy & Research, the Centers for Disease Control & Prevention, 13 pharmaceutical/health care companies that rotated into the guideline creation process four at a time, FDA, the Federal Trade Commission, the Institute of Medicine, the Health Care Financing Administration and the American College of Physicians. Additionally, the task force heard from FDA Division of Drug Marketing, Advertising and Communications Marketing Practices and Communications Chief Lou Morris; Stephen Schondelmeyer, PhD, of the PRIME Institute; an ethicist and an attorney.

Creation of the task force was sparked, in part, by a "sounding board" article in the May 9, 1991 New England Journal of Medicine by Alan Hillman, MD, et al. The article, "Avoiding Bias in the Conduct and Reporting of Cost-Effectiveness Research Sponsored by Pharmaceutical Companies," suggested that guidelines were needed to standardize cost-effectiveness studies and that researchers conducting those analyses should be allowed to publish their findings regardless of whether the findings were negative or positive.

The task force report focuses on defining bias and reportedly includes 11 guidelines for researcher independence, including study design, reasons for termination of a study and how to avoid conflicts of interest. The task force report does not formulate a new methodology because that field is so vast, but does provide a dozen or so pages on basic approaches.

Recently, NEJM announced its editorial policy that if a researcher paid directly by the industry is a co-author of a pharmacoeconomic analysis, NEJM will consider the article a review article only. Berger called the policy "a little bit naive," saying the policy also "demonstrates the depth of suspicion" about company involvement in PE studies.

Berger said he believes the NEJM policy indicates "that they were less interested in the kind of research that was industry-sponsored unless there was a complete arms-length distance: `Here, give the money to an academic researcher, let them do the study hands off, and whatever the results are, the results are.'"

Berger compared the skepticism about PE studies with the "suspicion" that arose 30 years ago when randomized, prospective clinical trials were first required to prove efficacy as well as safety. "We, over the course of time, evolved a very rigorous and consensus set of guidelines about how these studies can be done, how you quality control them to believe the results." Alluding to the nascent state of cost-effectiveness economic analyses, Berger said: "I think now we're at the beginning of a discourse that I think will take a 20-year time or some long period of time" after which "we will again as a society come to a set of consensus rules" about PE studies.