The U.S. Food and Drug Administration (FDA) and the American Society of Hematology (ASH) co-hosted the Sickle Cell Disease (SCD) Clinical Endpoints Workshop, a one-time event that aimed to identify opportunities to bring uniformity and standards to existing SCD endpoints, identify gaps, and propose development of new endpoints as a focus for future research.

For regulatory approval of therapeutics for the treatment of SCD, the FDA requires clinical endpoints that show direct evidence of clinical benefit or improvement in an established surrogate for clinical benefit. Since the original hydroxyurea approval, science and technology have evolved, and medical care delivery for patients with sickle cell disease has changed. The robust SCD drug development pipeline is poised to deliver new therapies to patients; however, there is general agreement that a timely discussion about endpoints is needed. To facilitate this discussion, the FDA and ASH convened this public workshop focused on clinical endpoints for SCD. The discussion and recommendations from the workshop will be published and shared with the SCD community.

The workshop included seven panels on the following topics:

• Patient reported outcomes (PRO)
• Pain (non-PRO)
• Brain
• End organ considerations, such as cardio-pulmonary and renal
• Biomarkers
• Endpoints related to curative therapies, including bone marrow transplantation, gene therapy, gene editing, and gene switching
• Considerations of endpoints for low-resource settings

Public Feedback Portal

The workshop included opportunities for the public to provide feedback throughout the meeting.

Who Should Attend?

The SCD Clinical Endpoints Workshop was intended for a diverse group of clinicians and scientists responsible for the development of SCD therapies, interested parties involved in ongoing efforts on SCD research, and program officers from various federal agencies with an interest in SCD.

The primary audience for this event included:

• Experts, scientists, and clinicians from academia, industry, and other health care sectors
• Patient advocates and representatives from patient advocacy groups
• Government agencies in the U.S. and abroad
• Representatives from non-profit organizations and industry with an interest in SCD
• Pharmacokineticists (clinical, preclinical, and toxicokinetics) with an interest in SCD

Workshop Co-Chairs

• Julie Panepinto, MD, MSPH, Medical College of Wisconsin
• Ann Farrell, MD, Office of Hematology and Oncology Products, U.S. Food and Drug Administration

Support

This workshop made possible through generous support from the Doris Duke Charitable Foundation, as well as support from numerous donors who contributed to the ASH Foundation’s Sickle Cell Disease Initiative Fund.

The mission of the Doris Duke Charitable Foundation is to improve the quality of people’s lives through grants supporting the performing arts, environmental conservation, medical research, and child well-being, and through preservation of the cultural and environmental legacy of Doris Duke’s properties. The foundation’s Medical Research Program supports clinical research that advances the translation of biomedical discoveries into new preventions, diagnoses, and treatments for human diseases.

Questions?

Please submit all general inquiries about this workshop to ASH Sickle Cell Disease Policy and Programs Coordinator Karina Ngaiza at kngaiza@hematology.org.

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