PRO-ving Ground: FDA Review Teams Commit to Patient-Reported Outcomes
This article was originally published in RPM Report
Executive Summary
FDA recently announced a significant step in furthering the development of patient-reported outcomes: the planned creation of a web-based compendium of clinical outcomes assessments, including PROs. But an even more significant sign that FDA is committed to developing new patient-reported endpoints came directly from reviewers – including some divisions that have not historically been out front on the issue.
You may also be interested in...
Patient Advocacy With FDA Review Staff Will Be Tougher Post-Sarepta
Efforts by CDER management to encourage more interaction between patients and FDA reviewers may be part of the collateral damage from the difficult FDA review of Sarepta’s Exondys 51. The hopes of parents of boys with DMD pushed the regulatory flexibility by CDER management on this application – but may end up limiting the willingness of FDA reviewers to engage with patients in the future.
Patient-Reported Outcomes in Oncology: The Jakafi and Zytiga Exceptions
Oncology is the biggest area of biopharmaceutical R&D where patient-reported outcomes are not routinely collected during clinical trials. There are some good reasons why. Jakafi and Zytiga remain outliers oncology treatment with a PRO in the approved label.
Confluence of Symptoms and Treatments at FDA Patient Meetings
The first round of four PDUFA patient-focused meetings concluded with the Sept. 24 meeting on narcolepsy. FDA is beginning to look ahead to the fiscal 2014 meetings – to start with fibromyalgia on December 10. One pattern that is emerging is that three of the first five conditions discussed with patients have overlapping symptoms and treatments.