PURPOSE

: This workshop will briefly describe FDA efforts to advance the development of publicly-available standard core sets of clinical outcome assessments (COAs) and their related endpoints for specific disease indications, including progress on an anticipated new FDA grant program. The workshop will focus on identifying opportunities for key stakeholders including patients, academics, Health Technology Assessors, payers, health care providers, and drug developers, to help inform this work.

DESCRIPTION

: The development of a COA that is fit-for-purpose for its intended use in drug development is a lengthy, rigorous, and resource-intensive process. There are many efforts underway to develop, modify or select COAs, and there is little to no external coordination across or even within disease areas. The current landscape requires repeated significant start-up costs. The siloed efforts also lead to variability in the quality of the COAs, COA development that may be incomplete, and a continued “learning curve” for developers, implementers, end users and regulators. For long-term sustainability, there is a need for a more collaborative pre-competitive multi-stakeholder approach to the development, modification and/or selection of COAs within specific disease area(s) that incorporates input from patients, caregivers, drug developers, researchers, regulators, payers, and other decision makers.

FDA anticipates establishing a new grant program to support the development of publicly-available standard core sets of COAs and their related endpoints for specific disease indications. A standard core set can include different types of COAs (i.e. patient-reported outcome, clinician-reported outcome, observer-reported outcome, performance outcome instruments) and their related endpoints that assess a minimum list of impacts that matter most to patients, are likely to demonstrate meaningful change and should be reported in a clinical trial. A standard core set might be relevant across several disease populations or subgroups or be focused on attributes of a specific disease.