www.fdanews.com/articles/187775-fda-issues-advice-on-replacement-or-corrective-therapies-for-certain-rare-diseases
FDA Issues Advice on Replacement or Corrective Therapies for Certain Rare Diseases
July 27, 2018
The FDA published a draft guidance recommending ways to demonstrate if new drugs or drug uses are effective for certain low-prevalence rare diseases caused by single enzyme defects.
The guidance applies to diseases that have well-characterized pathophysiology and whose changes in substrate deposition can be effectively measured in the affected tissues; it does not apply to rare diseases that progress rapidly.
“There are many reasons that make demonstration of effectiveness extremely challenging for drugs intended to treat slowly progressive, low-prevalence rare diseases that result from defects in a single enzyme,” the agency said.