CBI's Patient Reported Outcomes

December 15, 2009

Applied Clinical Trials

Last month saw the 5th Forum on Patient Reported Outcomes from CBI. Over 90 attendees came to the two-day event in Center City Philadelphia to learn more about Patient Reported Outcomes (PRO) planning, updates on FDA acceptance, ePRO, and more.

Central to PRO knowledge was the FDA Draft Guidance from 2006, which provided the first roadmap for industry in working with the regulatory agency in regard to PRO. That document has just been recently superseded by the final guidance released on December 9, “Patient-Reported Outcomes Measures: Use in Medical Product Development to Support Labeling Claims.”

The conference did offer insight into how FDA and other regulatory authorities can be brought into the process to make a more acceptable PRO. In fact, it’s not just FDA that should be brought into these discussions. Robert DiGregorio, D.O., F.A.C.O.O.G., Director, Worldwide Regulatory Affairs, for Wyeth Research now Pfizer, a presenter at the CBI conference, told Applied Clinical Trials that it would also be beneficial for the commercial and business units in the sponsor company to get in on early PRO planning.

“Typically not represented in these conversations are the commercial and business units,” said DiGregorio. “But PRO is based label claims and primary efficacy claims that need to be integrated very early in the process.” And, as DiGregorio stressed, the label development will include language on how the patients will use or respond to the drug. The FDA will ask, “did you develop the [PRO] tool based on what’s important to the patient.”

DiGregorio’s presentation “Integrate PRO Development into Your FDA Interaction Strategy” led to a good discussion on integration strategies. DiGregorio also offered a timeline of PRO development and agency interaction where he served as the lead on the PRO task force at Wyeth.

CBI attendee and former keynote speaker, Ari Gnanasakthy, PRO Expert at Novartis, noted that presentations offered by Nancy Kline Leider, PhD, Sr. Vice President, Scientific Affairs United BioSource Corporation, and Jean Paty, Co-Founder and SVP, Scientific, Quality and Regulatory Affairs for Invivodata, were very informative.

“Leidy’s [Explore Key Issues in Content Validity] had good content and a good grounding for newcomers,” remarked Gnanasakthy. Both DiGregorio and Gnanasakthy felt that Paty’s presentation on Marketing Insight versus Instrument Development Patient Interviews — Are They Really Different? was interesting. “The premise is that how patients’ view of disease and treatment are collected at different time points during drug development and we could consider doing it all at the beginning” said Gnanasakthy. “Although noble, this is not practical. Pharma companies cannot possibly invest in ‘once and for all’ market research activities so early for every compound in the pipeline.”

Editor’s Note:Applied Clinical Trials and CBI are owned by Advanstar Communications.

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