Bringing the platform trial model to scleroderma

A new platform study sponsored by the Scleroderma Research Foundation could spur much needed therapeutic innovation for the rare autoimmune indication, while also serving as proof of concept for the role master protocols can play in rare diseases. 

There’s no shortage of innovation in autoimmune diseases, but most is directed at the larger indications such as psoriasis, rheumatoid arthritis and Crohn’s disease. Underlying disease mechanisms are often shared across autoimmune disease indications, but there’s a commercial incentive problem that can prevent sponsors from pursuing less prevalent indications. The Inflation Reduction Act (IRA), which disincentivizes development in multiple rare indications, isn’t helping...