PRO Q&A with FDA

In preparation for a recent conference on patient reported outcomes, I reached out to FDA’s Dr. Laurie Burke in the Center for Drug Evaluation and Research. Burke, Associate Director, Office of New Drugs, was actually brought up during the PRO meeting, when a presenter credited her early involvement/familiarity with ePRO as meaningful. Nowadays she’s on the road a lot, so what follows is general feedback from FDA based on questions sent via email in Burke’s direction. Thus, the answers cannot be wholly attributed to her.

Q: I’ve heard the agency is increasing pressure on sponsors to focus on the patient. Why is this the case?

A: For some treatment effects, the patient is the only source of data. For example, pain intensity and pain relief are the fundamental measures used in the development of analgesic products. There are no observable or physical measures for these concepts. It would be difficult if not impossible to evaluate the efficacy of some medical products without the patient’s perspective and input. With that in mind, we would not agree that we have increased pressure on drug developers to focus on PROs; rather, we have put effort into encouraging it, and helping facilitate it.

Q: On FDA’s part, what’s behind the shift toward more patient-reported data?

A: PROs offer the opportunity for developers to be successful at demonstrating certain treatment effects pertaining to how patients feel and function that are often of value to patients and that cannot be identified using traditional, non-patient reported measures.

Q: How would you sum up the Final Guidance published in December?

A: The final guidance describes how FDA evaluates PRO instruments for their usefulness in measuring and characterizing treatment benefit as perceived by the patient. It emphasizes that FDA recognizes the importance of the patient perspective in medical product development where appropriate. The guidance also explains how FDA reviews evidence that a PRO instrument measures the concept represented by a treatment benefit-labeling claim.

Q: Why do you think more sponsors are relying on patient-reported data for primary and secondary endpoints?

A: It depends on the disease being treated, the measurement concepts of interest, the patient-population, and the medical therapy being evaluated as to whether a PRO is useful or necessary to evaluate medical product efficacy. Sponsors are more aware now that for certain conditions/therapies, efficacy cannot be adequately evaluated without patient input. Furthermore, in some cases, PRO instruments may generate more sensitive and interpretable clinical trial data.

Q: How much does the fact that patient reported outcomes are now electronic influence FDA’s acceptance of them?

A: I don’t think the ability to record PROs electronically alters FDA’s decision to accept or reject a PRO tool; either it has been rigorously developed and determined to be fit for purpose and FDA accepts it, or it has not been, and FDA has substantial reservations about it. The format for data collection is part of FDA’s review of the instrument. Advances in electronic PRO data collection has made electronic entry of the data by patients much more practical and thereby sponsors can better acquire complete and good quality data. Quality of data will affect FDA’s willingness to accept or not accept the data.

For more on the PRO conference mentioned, stay tuned.