TABLE 1.

Working Group 1: scientific priorities and innovative implementation strategies for FVIII inhibitor clinical trials

Co-chairs	Margaret Ragni, MD, MPH and Lindsey George, MD
Charge	The design of investigator-initiated clinical trials to determine optimal integration of non-IV, non-factor therapeutics, including gene therapy, into the standard of care for FVIII inhibitor patients
Goals	Overcome the challenges associated with conducting small clinical trials in rare diseases; resources and partnerships required to facilitate them • Leverage the HTC infrastructure • Potential for the CDC surveillance databases and central laboratory to serve as a platform for launching prospective clinical trials • Optimization of private-public partnerships to fund clinical trials • Engage the patient community in clinical trials • Embed training opportunities within the implementation strategy for clinical trials