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

PDUFA VII: US FDA Will Offer Additional Meetings To Boost Rare Disease Endpoint Development

Executive Summary

If they reach a public disclosure agreement, a handful of sponsors will be admitted to a new pilot program that allows four meetings with the FDA on rare disease endpoint issues.

You may also be interested in...



Don’t Call It A Center Of Excellence: Accelerating Rare Disease Cures Program Launched By CDER

‘Umbrella’ program is intended to ensure all rare disease and related programs at CDER are working together, which seems similar to the rare disease center of excellence concept that stakeholders want to coordinate activities across the FDA.

Firms Hope To Adapt COVID Vaccine Trial Design, FDA Communication Approaches To Other Diseases

Moderna has adopted more inclusionary enrollment criteria and real-time epidemiology monitoring for its RSV vaccine program, while Pfizer is expanding its internal ‘Lightspeed’ approach. US FDA is considering how to extend the fast, frequent and informal interactions.

Rare Disease Challenges At US FDA: CDER Director Says More Staff, Not Reorganization, Will Fix Issues

Rather than changing the agency's structure to create more focus on rare diseases, Patrizia Cavazzoni wants funding for people to do the necessary work.

Related Content

Topics

UsernamePublicRestriction

Register

PS144865

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