Improve FDA Myelodysplastic Syndromes Guidance

Researchers from Johns Hopkins and the University of Miami say myelodysplastic syndromes (MDS) therapeutic development is challenged by the syndromes’ biological diversity and regulatory risk. Writing in Blood Advances, the researchers say MDS is “ideal for FDA to implement flexible, biology-informed regulatory pathways. Establishing such frameworks in MDS could set a precedent that may extend across other rare and heterogeneous malignancies.”

The paper makes recommendations for improving an FDA 7/2025 draft guidance clarifying the agency’s approach to evaluating investigational products for MDS, with a particular focus on improvements in disease classification and trial eligibility, endpoint selection and validation, inclusion of older adults, and safety reporting frameworks.