FDA Quality Of Life Guidance Receives Renewed Interest After Industry Input
Executive Summary
FDA has renewed development of a guidance on the use of quality of life claims in drug labeling and promotional materials after gathering input from industry
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FDA has renewed development of a guidance on the use of quality of life claims in drug labeling and promotional materials after gathering input from industry. The Division of Drug Marketing, Advertising & Communications has renewed work on a "Patient Reported Outcomes" guidance; FDA is using the term in place of "quality of life outcomes" to discuss general health-related issues. An FDA "Patient Reported Outcomes" working group, which includes members from Center for Drug Evaluation & Research, the Center for Biologics Evaluation & Research and the Center for Devices & Radiological Health, has been meeting since 2000. However, in March 2001, the agency delayed development of the guidance until it collected additional information from several professional groups (1 (Also see "FDA Quality Of Life, Pharmacoeconomic Guidance Development On Hold" - Pink Sheet, 19 Mar, 2001.), p. 24). Four associations formed the "Patient-Reported Outcomes Harmonization Group" to work with FDA: the International Society for Pharmacoeconomics and Outcomes Research; International Society for Quality of Life Research; Pharmaceutical Research & Manufacturers of America; and European Regulatory Issues on Health-Related Quality of Life Assessment. Following ongoing discussions with FDA, the coalition is planning to publish a series of documents on health-related patient-reported and quality of life claims. By the end of the year, ISPOR plans to publish a scientific document that FDA could use in development of the guidance. Issues raised by ISPOR during the past three years also could be considered in the guidance. ISPOR has suggested that FDA could be more specific on requirements for quality of life clinical trial submissions. The agency should also address whether "responsiveness [should] be proven prior to a clinical trial being undertaken - or, if discovered in the trial, is this good enough?" Other issues that ISPOR believes FDA should consider include how clinical significance is determined and whether a trial should be "powered to detect clinical significance up-front?...If a difference is found to be statistically significant but it is not 'clinically significant' - can a claim be filed?" "Can a study that is powered solely to detect quality of life be enough for labeling/promotion when there is not enough power to detect a (secondary) clinical endpoint?" ISPOR asked. The patient-reported outcomes guidance is one of several under development at CDER, including a guidance that would clarify FDA's policy on advertising and labeling of Treatment IND protocols. FDA Oncologic Drug Products Division Director Richard Pazdur, MD, has suggested that large expanded access programs can transform from premarketing awareness to premarketing promotion of investigational drugs (2 (Also see "Cancer Expanded Access Plans Need Focus On Data Collection – FDA’s Pazdur" - Pink Sheet, 27 May, 2002.), p. 36). AstraZenaca has an ongoing 12,000-patient expanded access program for Iressa (gefitinib). FDA is also working on a guidance entitled "Dispute Resolution Involving Pediatric Labeling" that will address issues highlighted by Merck's attempts to gain pediatric exclusivity for the cholesterol-lowering agent Mevacor . FDA denied Merck's request for Mevacor pediatric exclusivity in June 2001 because the agency believed the company did not adequately meet the terms set out in its request for pediatric studies. Merck challenged FDA in D.C. federal court (3 (Also see "Merck Mevacor Pediatric Exclusivity Gets July 3 Hearing; 5 Generics Stayed" - Pink Sheet, 25 Jun, 2001.), p. 28). The D.C. court granted Merck a temporary stay against lovastatin generics, and allowed FDA to reconsider its petition, citing an incomplete administrative record. FDA later reversed its decision and granted pediatric exclusivity for Mevacor through Dec. 15, 2001, following consideration of clarified data submitted by Merck (4 (Also see "Merck Mevacor Exclusivity Will Run Until Dec. 15 Following FDA Reversal" - Pink Sheet, 23 Jul, 2001.), p. 9). Other guidances under development by FDA include "Promotion of Combination Oral Contraceptive Products"; "Bioequivalence Studies With Clinical Endpoints for Vaginal Antifungal Drug Products"; and "Chemistry, Manufacturing, and Controls Documentation Unique to Radiopharmaceuticals Submitted in ANDAs." |