CDER Launches Clinical Outcomes Assessment Compendium, Seeks Input on Future Expansions

By James E. Valentine –

On January 13, 2015, CDER’s Clinical Outcome Assessments Staff (formerly Study Endpoints and Labeling Development (SEALD)) announced the launch of Stage 1, or the pilot stage, of its Clinical Outcomes Assessment (COA) Compendium. A COA measures patients’ symptoms, overall mental state, or the effects of a disease or condition on how the patients function. There are four types of COA measures:

• Patient-reported outcome (PRO) measures
• Clinician-reported outcome (ClinRO) measures
• Observer-reported outcome (ObsRO) measures
• Performance outcome (PerfO) measures

Based on prior statements from FDA officials, the COA Compendium, which will be available at www.fda.gov/COACompendium, is supposed to provide clinical trial sponsors a database of: (1) qualified tools; (2) ongoing qualification programs; and (3) previously labeled COAs (from new molecular entity labeling approved 2003 and later). This pilot follows through with CDER’s pledge from spring of 2015 (see our previous coverage here) to provide a collated and summarized list of “potentially acceptable endpoints” that could support labeling claims, as well as encourage development of COAs more generally. Consistent with CDER’s intent for this compendium to serve more as a starting point when considering how measures might be utilized in clinical trials, the compendium is not expected to include detail on the endpoints. Therefore, the COAs currently available can serve to inform discussions between research sponsors and FDA, particularly early in drug development.

The COA Compendium also compliments FDA’s efforts to foster patient-focused drug development (see our coverage of these efforts here). CDER intends for the compendium to facilitate the use of measures that capture outcomes that are important to patients (e.g., PROs) – a “high priority for FDA.” In addition, CDER previously indicated its interest in a second stage of the compendium that would directly incorporate input from Patient-Focused Drug Development meetings to identify gaps in available measurements. Specifically, a list of could be generated of concepts highlighted as important by patients, but for which no tool exists.

CDER is seeking input on the utility of the pilot compendium and approaches for future iterations, including any suggested expansions of its scope. Comments can be submitted to FDA’s docket here.