September 2020
Rare Across America looked a little different this year but our voices were still heard! Thank you to Cure CMD Advocacy Team members who participated in this event and met with their Members of Congress by phone or video meeting.

Proposed Legislation Highlight

What it would do: This bill will permanently authorize the Pediatric Priority Review Voucher (PRV) program. It will allow further opportunity to spur innovation in rare and neglected diseases that disproportionately impact children.

Why it is important: Since 2012, this program provides incentives for pharmaceutical companies to develop treatments for rare pediatric diseases by providing them with a priority review voucher that entitles the company to receive a 6-month review that would normally be reviewed under FDA’s standard 10-month review period. The Priority Review Voucher provides critical incentives to rare disorder product developers. 

Why it is important to the Cure CMD community: The PRV may help future treatments for CMD when they are ready for FDA review and approval. This bill would make the PRV permanent so it is guaranteed to be available if and when we need it in the future.

  • Attend the RDLA Monthly Webinar on September 10th at Noon EST to learn more about the current state of Newborn Screening. Register here.

  • Attend a Listening Session on Rare Diseases and the Pandemic co-hosted by NORD and the FDA on September 16 at 2pm EST. Register here.

  • Attend the Virtual Rare Disease Caucus Briefing on September 22nd at Noon EST. The topic of this briefing is Saving Lives: Newborn Screening and Diagnostic Testing. Register here.

  • Attend the EveryLife Foundation's 2020 Newborn Screening Virtual Bootcamp starting September 30th. Learn more here.

Know someone who would like to join the Cure CMD Advocacy Team? Email us and we'll add them to the list!