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

As Orphan Drugs Gain More Attention, FDA Releases Development Guidance

This article was originally published in The Pink Sheet Daily

Executive Summary

Straightforward draft document seems aimed at entities that have limited experience working with the agency.

You may also be interested in...



Rare Diseases: US FDA Gives More Guidance On Premarket Safety Evaluation, Historical Controls

Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.

PDUFA VI: Price Tag For Patient Experience Data Validation May Go Up

FDA wants to advance PRO projects through the user fee reauthorization, but ironically is having difficulty quantifying how much support it might need.

In Manufacturing, Absence Of Evidence Is Not Evidence Of Absence, FDA Tells BioMarin

BioMarin showed that the Vimizim BLA did not have enough data to prove that clinical and commercial sites are different. That’s not the same as concluding there is no difference between the sites, FDA said.

Related Content

Topics

Related Companies

Latest Headlines
See All
UsernamePublicRestriction

Register

PS078807

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