Congressmen Paul Tonko (D-NY) and David McKinley (R-WV) introduced the HEART Act (HR 1184) on Feb. 16, 2021. Please contact Emily (Emily.Silverberg@mail.house.gov) or Kirsten (Kirsten.Wing@mail.house.gov) to cosponsor the HEART Act.

The HEART Act contains 5 provisions critical to rare patients:

• Advisory Committees When an advisory committee was largely “saved” and resulted in a positive vote recommending approval by the “accidental” attendance of one physician with experience in the science of small studies, we went looking for a requirement that this should occur systematically. There is no such requirement today.
  • Solution. Require a rare/ultra-rare expert in the science of small population studies at Advisory Committee meetings when the application under review is for a low prevalence condition.
• Review Division Transparency The “orphan” category is actually fairly broad with respect to impacted population, and there may be issues in patient populations below 15-20,000 that are not present when prevalence approaches 200,000. When a review division asks questions that clearly indicate a lack of experience in ultra-rare applications, we went to see how many applications they had reviewed before. This data is unavailable. Is it also unavailable to Congress and to the FDA itself?
  • Solution. Require annual report to Congress that sets out, by division, how many rare applications were reviewed, Agency actions, and the prevalence #s for that rare condition (this could be pulled from sponsor submission on orphan designation request.)

Read more about how this bill will help patients like Casey who fall into the ultra rare category, with patient populations <200,000 that are typical sizes needed for additional research orphan drug research.