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

CDKL5 in Color, a patient advocacy organization for CDKL5 Deficiency Disorder, expresses enthusiastic support for the Haystack Project citizen petition. They argue that the current FDA drug approval framework creates insurmountable barriers for ultra-rare diseases and advocate for the adoption of Bayesian adaptive designs, biomarker-stratified enrichment, and formal early-stage consultation with patient experts.

Dr. Jesse Sengillo, a clinician at the Bascom Palmer Eye Institute, supports the Haystack Project's petition for a rulemaking framework to evaluate clinical investigations in rare diseases. He argues that traditional development paradigms often fail to account for disease-specific factors like genotype heterogeneity and variable progression rates, and advocates for a "fit-for-purpose" approach to study design.

Dr. Stephen H. Tsang, a professor at Columbia University, supports the Haystack Project's petition for a new FDA rulemaking framework. He argues that clinical investigations for rare diseases, such as inherited retinal diseases, should be evaluated based on disease-specific contexts and "fit-for-purpose" study designs rather than traditional development paradigms.

Dr. Paul Yang, a clinician and professor at the Paul H. Casey Ophthalmic Genetics Division, supports the Haystack Project’s Petition for Rulemaking. He argues that the FDA should adopt a framework that considers disease-specific contexts—such as heterogeneity and progression rates in rare diseases—when evaluating whether clinical investigations are adequate and well-controlled.

CURE SYNGAP1, an advocacy organization for SYNGAP1-related disorders, supports the Haystack Project's petition to modernize rare disease drug development frameworks. They argue that the FDA should integrate disease-specific endpoints, real-world evidence, and alternative study designs to better address the unique challenges of rare diseases like SYNGAP1.

Dr. Robert K. Koenekoop, a clinician scientist at McGill University and the Montreal Children’s Hospital, supports the Haystack Project’s Petition for Rulemaking. He argues that the proposed framework is necessary to ensure that clinical investigations for rare diseases, specifically inherited retinal diseases, are evaluated based on disease-specific contexts rather than just traditional development paradigms.

Dr. Tomas S. Aleman, a professor at the University of Pennsylvania, supports the Haystack Project’s Petition for Rulemaking. He argues that the FDA should adopt a framework that considers disease context when evaluating clinical investigations for rare diseases, specifically to ensure that study designs are fit-for-purpose and reflect clinical realities.

Professor Mariya Moosajee, a clinician scientist at the UCL Institute of Ophthalmology, supports the Haystack Project’s Petition for Rulemaking. She argues that the proposed framework is necessary to ensure that clinical investigations for rare diseases, specifically inherited retinal diseases, are evaluated based on disease-specific contexts rather than just traditional development paradigms.

The Luka Shai Foundation, representing families affected by ultra-rare proton-pump diseases, supports the Haystack Project citizen petition. They argue that the FDA should establish a formal rulemaking to create a durable framework for rare disease research that accounts for small patient populations, multisystem benefits, and early engagement with disease-specific experts.

Prof. Elfride De Baere, a physician-scientist and professor at Ghent University, supports the Haystack Project’s Petition for Rulemaking. She argues that clinical investigations for rare diseases like inherited retinal diseases require a framework that accounts for disease-specific factors, such as heterogeneity and variable progression, rather than relying solely on traditional development paradigms.