Protocol

Develop a protocol that ensures the institutional review board (IRB) or research ethics committee (REC) is reviewing the trial not just for safety and ethics principles, but also for its justice principle. The latter takes into consideration fairness in distribution of benefits realized from the research study as well as its burdens.

Subgroups

Include information on how the drug, device, or other therapy being studied could work in different subgroups and any prior data on heterogeneity of treatment effect. This will further justify your rationale for the selected inclusion and exclusion criteria, and will facilitate the review of your protocol.

Communication

Initiate early and regular conversations with regulators to establish changes to the protocol, including eligibility criteria, that will broaden enrollment. See the FDA Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program for more information.