By Dr. Robert Roth
As recently reported, FDA has held a public workshop entitled, “Clinical Outcomes Assessment Development and Implementation: Opportunities and Challenges.” For this initiative, FDA prepared a pilot document entitled “Clinical Outcome Assessment Compendium”, and has established a docket for suggestions and comments to the Compendium (FDA-2015-N-5106). The Compendium, which is intended to aid sponsors in the design of their clinical trials, is not FDA guidance but does present some insight into FDA thinking. Based on preliminary response to the docket, it can be expected that some very specific, cutting-edge instruments will be suggested to the Compendium.
Established to aid in the development of new drugs, the compendium includes an evaluation of how specific clinical outcome assessments have been used in clinical trials to measure disease-related symptoms and other patient-related outcomes. Some of the outcome assessment instruments have been validated for use in drug development programs under FDA’s Drug Development Tool Qualification Program. Intended as tools to directly quantify patient-related outcomes, the endpoints are clinical rather with or without the addition of specific biomarkers which can help define the endpoint.
The pilot Compendium version released for public input is organized by clinical indication, with separate contents prepared by one of the CDER offices (e.g., hematology-related endpoints presented by the Office of Hematology and Oncology Products) or one of the Office of Drug Evaluation groups. Some of the entries identify specific patient-reported or physician-reported outcome instruments including diaries and questionnaires, whereas others describe the use of performance instruments such as distance walking tests or visual acuity eye exams.
Of particular use, sources for additional information about a specific outcome assessment are provided when available. Such sources can include divisions within NIH, working group consortiums, and commercial entities. Although mention of a specific endpoint in the Compendium does not necessarily mean that FDA considers the endpoint fully validated or to be the choice for primary efficacy endpoint in a clinical trial, or officially endorsed by FDA, it does provide some direction for clinical research sponsors to consider. That said, many such endpoints in fact did provide key clinical evidence for recent new drug approvals.