Pink Sheet is part of the Business Intelligence Division of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


FDA Accelerated Approval For Rare Diseases Again Under Congressional Pressure

This article was originally published in The Pink Sheet Daily

Executive Summary

Letter being circulated by Rep. Bilirakis states the agency’s draft expedited approval guidance does not provide clarity for rare disease sponsors to use accelerated approval.


Related Content

FDA Expedited Pathways Guidance Expands Range Of Eligible Products
Accelerated Approval May Support Smaller Trials In Acute Diseases – FDA
FDA Directives From House Range From Antibiotics To Zohydro
Priority Review Vouchers For Pediatric Rare Disease: Will Popularity Force A Quick Halt?
Hamburg Tries To “Cope” With FDA Budget, Emphasizes Predictability
Biomarkers In Orphan Studies Still Need Past Experience, FDA Says
Regulatory Freelancing: Rare Disease Group Hopes To Shape FDA Guidance
FDA To Provide Expedited Approval Desktop Reference
TREAT Act Wants Accelerated Approval Change, But Uses Existing Language
Regulatory Flexibility Staring Contest: Will Industry Or FDA Blink First?





Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts