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
UsernamePublicRestriction

FDA Orphan Designations At Record Levels; Resources Need To Keep Up

This article was originally published in RPM Report

Executive Summary

The development of drugs and biologics for rare diseases has reached a regulatory milestone that underscores industry’s interest in the orphan drug market. FDA’s Office of Orphan Product Development is now granting one new orphan drug designation, on average, every day of the year. That activity underscores the success of the Orphan Drug Act, but creates a new problem: Finding the funding to support it.

You may also be interested in...



Separating Gene Therapy ‘Wheat’ From ‘Chaff’: CBER Facilitators Could Help Inexperienced Sponsors

Surge of interest prompts US FDA to consider how to adjust feedback, including potentially creating a separate team to answer more basic questions.

FDA/Sponsor Meetings During COVID: How To Overcome The Loss Of ‘Hallway Chitchat’

When the closest you can get to US FDA staff is Webex, the meeting minutes and interactions with the review team project manager take on increased importance, consultants suggest during an Informa-sponsored webinar.

US FDA’s Oncology Center In The Lockdown: ‘Business As Usual’

Amid the COVID-19 pandemic, Richard Pazdur doesn’t want the cancer patient forgotten. His solution: keep churning out approvals.

Topics

Related Companies

UsernamePublicRestriction

Register

LL1133951

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

Cancel