Updates are coming to expanded access procedures and data use. Read the latest from FDA Law Blog featuring key messages from our public meeting on leveraging expanded acccess data.

Pink Sheet featured an article authored by Kate Rawson which highlights important takeaways from our public meeting, Leveraging Real-World Treatment Experience from Expanded Access Protocols on November 19, 2018 at the FDA White Oak Campus. Find the summary and access to the article below.

Executive Summary

Formal reg not needed, according to agency; officials agree that regulatory flexibility is best way to offer sponsors advice, saying data collected during compassionate use can be helpful in guiding regulatory decisions.

Mark McClellan and his colleagues at Duke-Margolis Health Policy Center outline how real-world evidence can increase value in health care in this Growth Commentary referencing Reagan-Udall Foundation for the FDA’s IMEDS program.

Dr. Scott Gottlieb indicated that the upcoming changes to the FDA Office of New Drugs would likely be in place this summer, when taking questions at the Foundation's Annual Public Meeting in this article from RAPS.

FDA hopes to bolster its scientific staff in 2018 and this piece in FDA News explains how the Reagan-Udall Foundation's upcoming Fellowship program could funnel talented post-docs into the agency it was created to support. 

In one powerful blog post, Dr. Gottlieb waives requirements to wait for a full convening of IRBs to approve single patient expanded access treatments, announces the future expansion of the Foundation's Navigator to rare diseases and offers clarity on which adverse events during EA treatment need to be submitted to FDA.

Dr. Joette Giovinco shared the launch of the Expanded Access Navigator in a Fox Medical Team TV and online report. She credited the FDA and advocacy groups who provided input for the online tool and the clip shows how companies are listing single patient expanded access therapies in the Navigator’s Directory.

More

Saving time is critical for physicians treating seriously ill patients who have exhausted alternatives to expanded access. So Forbes contributor Alison Bateman-House explains how the Navigator spells out timelines in this piece called "How To Try An Experimental Drug When You Don't Qualify For A Clinical Trial."

In the article, Wasser explains why the FDA asked its foundation to create a public-private partnership launching the Navigator. Legislation like the 21st Century Cures Act and changes in regulation require drug companies to make any expanded access policies and contact information public. The Navigator streamlines the way to find that information.

This short MedPage article focuses on the Navigator's step-by-step guided experience for patients and physicians: from identifying treatment and exploring clinical trials to seeking IRB approval and treatment/reporting requirements.