Citizen Petition from Haystack Project, Inc., et al.

Professor Matthew Simunovic, an academic and vitreoretinal surgeon, supports the Haystack Project's petition for rulemaking regarding the evaluation of "well-controlled" clinical investigations in rare diseases. He argues that regulatory frameworks should allow for flexibility in outcome measures, specifically advocating for the use of validated psychophysical assessments over the repeated development of complex, bespoke functional assessments.

Dr. Jason J. Begalke supports the Haystack Project's petition to modernize FDA regulatory frameworks for rare diseases. He argues that the current reliance on large-scale randomized controlled trials is unsuitable for small patient populations and advocates for context-driven evidence like natural history controls and disease-specific endpoints.