FDA Rare Disease Day 2024 Virtual Public Meeting

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We are pleased that Carla (DeMuro) Romano, M.S., Vice President of Patient-Centered Outcomes Assessment at RTI Health Solutions, will be presenting at the US FDA’s Virtual Public Meeting in recognition of Rare Disease Day 2024. In the afternoon session, Romano will be part of the discussion surrounding patient committee participants on the FDA-sponsored study to adapt an in-clinic outcome assessment tool for ALS to remote-use at home.   

Panel 3: Patient Engagement at FDA
March 1, 2024
1:30 – 3:00 pm EST

As shared in the FDA event agenda, having outcome measures that can be assessed remotely may decrease travel burden and expense for patients and their families and help remove obstacles for subject recruitment and retention that often lead to underrepresentation of certain groups of otherwise eligible subjects in clinical trials. This panel also includes discussion of patient engagement in public private partnerships and ways patients and care partners can participate and advance clinical research for rare diseases including regenerative medicine, such as gene and cell therapies.

Carla (DeMuro) Romano has extensive experience in the rigorous development and psychometric evaluation of clinical outcome assessment measures for use in clinical trials, as well as large scale epidemiologic and population-based studies. She has specialized in both the qualitative and quantitative aspects of instrument design and has worked towards creating a clear path from measure development to interpretation. She is working closely with the FDA to develop and evaluate remote access versions of a widely used clinical outcome assessment in ALS as part of the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS) initiative. Romano shares, “I am truly honored to facilitate the Patient Committee as part of this research and to participate in FDA Rare Disease Day.”