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

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. Taizo A. Nakano, a professor and clinician at Children's Hospital Colorado, supports the Haystack Project's petition for rulemaking. He argues that the FDA should adopt a more flexible, disease-specific framework for evaluating clinical investigations in rare and ultra-rare diseases, where conventional trial designs may not be feasible or optimal.

Lipodystrophy United, a patient-led nonprofit organization, supports the Haystack Project petition to modernize FDA drug development frameworks for rare and ultra-rare diseases. They argue that traditional frequentist statistical models are unsuitable for small, heterogeneous patient populations and advocate for the adoption of Bayesian adaptive designs, synthetic control arms, and subtype-specific endpoints.

Kelly Paul, a patient living with mycosis fungoides, supports the petition to establish a context-based evidence framework for rare diseases. She argues that standard trial designs are often unsuitable for rare diseases with small populations and that a disease-specific framework would provide more meaningful evidence and investment certainty for patients.

The International Cancer Advocacy Network (ICAN) supports the Haystack Project's petition to modernize the FDA's drug approval framework for rare and ultra-rare diseases. They argue that the current framework creates insurmountable barriers for small patient populations and advocate for flexible study designs, disease-specific endpoints, and expert consultation to improve access to life-extending therapies.

AIM at Melanoma, a nonprofit organization, supports the Haystack Project's petition for rulemaking to clarify how the "adequate and well-controlled" standard should be applied to rare diseases and orphan-designated products. They argue that the proposed amendments to 21 CFR § 314.126(b) and 21 CFR § 312.47 will improve regulatory predictability and ensure that study designs are fit-for-purpose for small, heterogeneous patient populations.

The FCS Foundation, a patient-led nonprofit for Familial Chylomicronemia Syndrome (FCS), supports the Haystack Project's proposed framework for improving drug approval for rare diseases. They argue that the FDA should adopt modern statistical methods (like Bayesian adaptive designs) and patient-specific endpoints to address the unique challenges of small patient populations.

Mark E. Villeneuve, a patient living with Mycosis Fungoides, supports the Haystack Project Petition to modernize the regulatory framework for rare disease treatments. He argues that the current system should be updated to recognize clinically meaningful endpoints and unique study designs that reflect the realities of rare diseases without lowering safety and effectiveness standards.

The National Organization for Rare Disorders (NORD) supports the Haystack Project's petition to improve the FDA's review process for rare diseases. They argue for the use of clinically meaningful endpoints from natural history studies, the consideration of alternative trial designs to randomized controlled trials, and the inclusion of patient and caregiver expertise in the regulatory process.

Theresa Owhady, a parent of a child with Neurofibromatosis Type 1, supports the Haystack Project’s petition to create a modernized regulatory framework for rare diseases. She argues that traditional drug development pathways are often unworkable for small, heterogeneous patient populations and calls for a framework that recognizes clinically meaningful endpoints and diverse study designs.