Mark McClellan, director of the Duke-Margolis Center for Health Policy, is hopeful that attendees of the group’s March 19 meeting on novel statistical methods and trial designs will walk out with the tools they need to design and launch platform trials in rare diseases.
McClellan, who was previously FDA commissioner and administrator of CMS, joined Duke-Margolis in 2015 as its inaugural director to lead the center’s work on translational health policy research, analysis and implementation.
Monday’s meeting will be co-hosted by FDA and include patients, academics, biostatisticians and companies.
The small numbers of rare disease patients can make it difficult to enroll enough subjects to achieve a statistically significant treatment effect in all comers, let alone in patient subgroups.
Multidrug platform trials could make better use of the available patients by increasing the proportion