American Academy Of Optometry’s Post

Our latest Whitepaper, 'Navigating the Rare Pediatric Disease Priority Review Voucher Program, Ahead of September 2024', is now available to read and download, https://lnkd.in/g8SdRH9E - The paper, written by Iheoma Anosike, provides useful information relating to The rare pediatric disease designation and RPD PRV program, with important considerations for Sponsors. For support ahead of the upcoming 30 September 2024 sunset date, get in touch to speak with a member of the team www.scendea.com/contact - #whitepaper #drugdevelopment #regulatoryaffairs #pediatricdisease

U.S. President Biden signed a bill postponing the sunset date of the rare pediatric disease Priority Review Voucher (PRV) program from September 30, 2024, to December 20, 2024. The bill, the “Continuing Appropriations and Extensions Act, 2025” is a temporary measure to continue the rare pediatric disease PRV program. There are currently other ongoing legislative efforts to renew the program through 2028 and later. We have summarized these proposals here: https://lnkd.in/exn-tcha #PediatricHealth #PriorityReviewVoucher #ContinuingAppropriationsandExtensionsAct2025 Authors: Komal Karnik Nigam, Deborah Cho, and Bryan Walsh.

Read our new #NORD, the National Organization for Rare Disorders, report on the rare pediatric disease priority review voucher program, and its ability to spur #raredisease #drugdevelopment.

An insightful read on the Rare Pediatric Disease Priority Review Voucher Program!

Our latest whitepaper, 'Navigating the Rare Pediatric Disease Priority Review Voucher Program Ahead of September 2024', written by Senior Consultant Iheoma Anosike is now available to read and download: https://lnkd.in/g8SdRH9E - This whitepaper discusses the rare pediatric disease priority review voucher (RPD PRV) program which was introduced by the US FDA in 2012 and also provides important considerations for Sponsors ahead of the upcoming 30 September 2024 sunset date. - #whitepaper #drugdevelopment #rarepediatricdisease

In this short video, Dr. Leslie Gordon, Co-Founder and Medical Director of The Progeria Research Foundation, explains the vital importance of Rare Pediatric Disease PRVs for #progeria and other #RareDiseases. If you have not taken action on this issue, do so here: https://lnkd.in/ettvJA2p

🔑✨ TOP PSORIASIS TAKEAWAYS FROM AUGUST: #PsoriasisAwarenessMonth was packed with updates on psoriatic disease: Alumis Inc.’s ESK-001 phase 3 trial, the impact of green spaces on risk, and apremilast becoming available for pediatric patients. Explore our comprehensive recap to stay informed on these important developments and more. ⬇️ https://lnkd.in/gtmadrk9

Get your drug to market 4 months faster with a "Rare Pediatric Disease Priority Review Voucher" but hurry, this program ends September 30, 2024! Refer to FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers (here: https://lnkd.in/eStfxZSi) for details on what to include in your request. Source (see section 5): https://lnkd.in/ems7aMY3 #RarePediatricDisease #PriorityReviewVoucher #PRV #PediatricDisease #RareDiseases #FDA #Regulations #RegulatoryAffairs

As a voice for the rare disease community, a recent op-ed in STAT really hit home for me. The article focuses on how the FDA’s rare pediatric disease priority review voucher program will disappear unless Congress reauthorizes it before September 30. As NORD's Pamela Gavin shares in the byline, more than 30 million Americans live with a rare disease, and nearly half are children – the program disappearing would be a devastating end for patients and their families eagerly waiting for treatment.    At Aldevron, we are committed to advocating for individuals with #rarediseases by helping develop the tools to create breakthrough treatments, and we understand how important increased funding and programming is in this often-forgotten space. We must continue to push the government to incentivize #drugdevelopment for rare diseases, ensuring that patients continue to have hope for their future. #PRVs #RareDisease #FDA 

Boost your clinical trial success with our on-demand webinar on building trust with rare disease patients and accelerating enrollment. This webinar is designed to unveil the essential strategies and practices for fostering trust among rare disease patients, which is pivotal in accelerating clinical trial enrollment. Watch now: https://lnkd.in/gqfzUh3D #ClinicalTrials #RareDiseases #PatientTrust