Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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

UsernamePublicRestriction

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.

You may also be interested in...



FDA Expedited Pathways Guidance Expands Range Of Eligible Products

Drugs intended to prevent a serious condition or reduce likelihood of disease progression may qualify, as may products intended to diminish serious adverse events associated with current treatment.

Accelerated Approval May Support Smaller Trials In Acute Diseases – FDA

Final guidance on expedited regulatory pathways suggests FDA is open to accelerated approval for some acute conditions; agency also provides examples outside of HIV/cancer arena where surrogate and intermediate clinical endpoints have supported approval.

FDA Directives From House Range From Antibiotics To Zohydro

Draft appropriations committee report instructs FDA to undertake tasks both profound (offer a better accounting of how user fees are spent) and routine (meet with patient communities for specific diseases).

Related Content

Topics

Latest Headlines
See All
UsernamePublicRestriction

Register

PS076048

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

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

Cancel