Exploring the PDUFA V agreement: Patient-reported outcomes and study assessment tools

As you are probably aware, several media outlets have reported that the FDA and industry groups have reached agreement on the proposed PDUFA V technical letter. The letter deals with a lot of technical issues, and we have been working to explore what these issues really mean for patients. Today, we are tackling an issue that that has recently been discussed in the news:

Patient-reported outcomes and study endpoint assessment tools

As defined in FDA's guidance, a Patient Reported Outcome (PRO) is any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician. The outcome can be measured by the severity of a symptom, state of a disease or as a change from the previous condition. In clinical trials, a PRO instrument can be used to measure both the safety and efficacy of new medicines. Effectively measuring PROs in clinical trials is an important step in ensuring new medicines make it to patients in need.

According to the September 27, 2010 minutes posted on FDA's website, FDA identified a need for industry user fee funding to be allocated to ensuring quality of patient-reported outcomes and other tools that assess clinical trial endpoints. Within drug development, clinical trial endpoint assessments are increasingly becoming an important part of the development process. Developing a consistent and valid process around evaluation and statistical design are critical. FDA had proposed to institute and resource an ongoing unit that would manage patient reported outcomes by determining qualifications and issuing much-needed guidance to industry.

According to FDA, a significant number endpoints that are not established early in the drug development process end up failing -- threatening the chances of new medicines making it to market. The biopharmaceutical industry and FDA agreed to language on a technical proposal that will increase the clinical and statistical capacity of the FDA to ensure quality with these endpoints -- helping get new medicines to patients in need in a timely manner.