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

Rare Diseases: CBER Looks To ‘Lean Into’ Accelerated Approval, Align More With CDER

Executive Summary

US FDA biologics center officials spoke about their efforts to increase collaboration and harmonization with the drugs center, and to internally involve more review disciplines in evaluating biomarker evidence, during a Reagan-Udall Foundation meeting that weighed potential use of accelerated approval for neuronopathic mucopolysaccharidoses disorders.

You may also be interested in...



Sanfilippo Syndrome Patients, Drug Developers At Odds With US FDA Guidance

Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.

Cell/Gene Therapies: US FDA Wants ‘Quantitative Correlation' Between Surrogate And Clinical Outcome, Former Reviewer Says

When weighing accelerated approval, the FDA wants to see that ‘X change in the surrogate translates into Y change in the clinically meaningful outcome,’ Parexel’s Steve Winitsky says. CBER Director Peter Marks opines on the types of therapeutic settings where accelerated approval is best suited, and what happens when a confirmatory trial disappoints.

‘No Simple Formula’: US FDA Decisions On Surrogate Endpoints Are Complex, Case-Specific

Office of New Drugs’ Peter Stein runs down the types of evidence and other considerations weighed in determining that a surrogate is reasonably likely to predict clinical benefit; however, some panelists at a National Academies meeting say the agency needs to increase transparency around its thinking on surrogates that can support accelerated approval.

Related Content

Topics

Latest Headlines
See All
UsernamePublicRestriction

Register

PS149838

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