We propose the preparation of a Qualification Plan to support qualification of the PROMIS® Short Form v2.1—Physical Function-Multiple Sclerosis 15a (PROMIS PFMS—15a) as a patient-reported outcome measure of physical function in individuals diagnosed with all forms of multiple sclerosis (MS). Over 1 million people in the United States are living with MS, a disease that has significant impacts on physical functioning and its associated quality of life. For example, many of the symptoms of MS (e.g., fatigue, weakness, spasticity, numbness) limit physical function, such as the ability to carry out activities of daily living (e.g., household chores and personal care) and other physical activities (e.g., rising from sitting, standing for a short time, and climbing a flight of stairs). However, a psychometrically sound, publicly available measure of physical function optimized for persons with MS has not been recognized by the FDA as fit-for-purpose for use in drug development. A measure of physical function was accepted into the Center for Drug Evaluation and Research’s (CDER’s) Clinical Outcome Assessment (COA) Qualification Program under DDT #000123 on June 21, 2017. The PRO Consortium’s MS Working Group has selected the PROMIS PFMS—15a as the physical function measure for qualification as it has been optimized for use in persons with MS. The next step in the qualification process will be to submit a Qualification Plan to the FDA to address the MS Working Group’s research plan for obtaining the quantitative evidence necessary to support qualification of the measure for use in MS drug development. Our approach includes two aims. For Aim 1, we will develop the final protocol and cross-sectional and longitudinal statistical analysis plans for an existing dataset that includes the PROMIS PFMS—15a. Planned analyses will examine the measure’s validity, reliability, and responsiveness as an outcome measure in MS populations. The protocol and analysis plans will provide the foundation for Aim 2, in which we will finalize and submit a Qualification Plan for the PROMIS PFMS—15a using the CDER COA qualification plan specifications. The long-term result of this proposal will be the development of a publicly available and accessible COA for measuring physical function in MS clinical trials. As such, qualifying the PROMIS PFMS—15a will fill a critical gap in the measurement of physical function in MS treatment trials.
|Effective start/end date||8/1/20 → 7/31/22|
- Critical Path Institute (DDT COA #000123//1 U01 FD006999-01)
- Food and Drug Administration (DDT COA #000123//1 U01 FD006999-01)
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