FDA Offers Flexibility
For their part, FDA representatives stated, “It all starts with well-defined and reliable PRO instruments.” They emphasized the importance of content validity—that is, proof that the instrument measures what it purports to measure.
The FDA acknowledged that the standards in their 2009 guidance on PRO measurement tools are strict, but pointed out that they were intended to address all therapeutic areas.
The agency may be willing to exercise flexibility when evaluating PRO claims for oncology products.
The FDA encouraged industry to work more closely with the Center for Drug Evaluation and Research to increase the number of publicly available outcome assessments for use in multiple clinical trials.
Patients Remain Frustrated
Cindy Geoghegan, a patient representative on the panel, objected strongly to the failure thus far to include patient experiences in drug labeling.
“I find it hard to understand how the FDA can continue to evaluate the potential benefits and harms of cancer drugs that are toxic, with survival benefits measured only in days or weeks, without a comprehensive understanding of patient experiences elicited through PROs,” she said.
Although patients will always strive for survival, she said, “When faced with a decision to choose between treatments that have shown very modest benefit without extending life, they need to know what they can expect to feel and how they can expect to function.”
PRO Consortium Agrees to Try
The PRO Consortium, a collaboration among the FDA, industry, and the Critical Path Institute, will focus on the development of a PRO measurement instrument for use in trials of drugs for non-small cell lung cancer (NSCLC) as a test case.
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Given the hurdles that manufacturers currently face, development of measurement tools for other cancers will be put on hold until the feasibility of developing a tool for NSCLC is determined.
In its conclusions, the panel recommended that the FDA and industry collaborate more closely on developing PRO measurement tools, analyze existing tools for validity, and support training in the use of PRO assessments.
- Basch E, Geoghegan C, Coons SJ, et al. Patient-reported outcomes in cancer drug development and US regulatory review. Perspectives from industry, the Food and Drug Administration, and the patient. JAMA Oncology. 2015; Published online April 16, 2015. doi:10.1001/jamaoncol.2015.0530.