Pink Sheet is part 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

UsernamePublicRestriction

Regulatory Freelancing: Rare Disease Group Hopes To Shape FDA Guidance

Executive Summary

EveryLife Foundation is working on a white paper that it hopes will inform FDA’s guidance clarifying accelerated approval and fast track pathways, which is mandated in FDASIA.

You may also be interested in...



FDA Accelerated Approval For Rare Diseases Again Under Congressional Pressure

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.

Biomarker Approvals At Pre-IND Stage Requested For Orphan Drugs

Updated draft white paper on expanding use of accelerated approval suggests FDA approve the use of biomarkers as surrogate endpoints in the pre-IND stage, which could enhance sponsors’ abilities to raise funds for trials and manufacturing.

“Complete Response” Letters Might Include Benefit-Risk Framework Discussion

FDA official says idea could be helpful; meanwhile, sponsors and others remain concerned about the framework’s undefined decision-making methods.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

PS055292

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