Discover and read the best of Twitter Threads about #compassionateUse
Most recents (1)
Easing 'compassionate use' access to investigational drugs has been a major priority for the @FDAOncology @SGottliebFDA @tmprowell @BlumenthalG - now in @JAMAOnc our analysis of outcomes from this mechanism over 5 years @sloan_kettering ja.ma/2Ej78li [1/5] 
Key findings: 208 applications IRB-approved (185 administered), 43 cancer types treated, 66 unique products. ORR 20.1% (95% CI, 14.2%-27.2%). PFS at 1-year 24.5% (95% CI, 18.2%-31.4%). Meaningful benefit - albeit in minority of patients. [2/5]
At the time of use, products were in Phase 1 (19%), Phase 2 (36%), and Phase 3 (39%) development. Children 34% of use (drug access remains major peds issue!) - no unique safety signals. Applications citing genomic rationale not associated with improved outcome @JackWestMD [3/5]
