Today, senior FDA officials and representatives from leading patient and health policy organizations are meeting in Washington, DC to discuss the urgent and contentious issues surrounding expanded access to investigational drugs. Yesterday I keynoted the “Expanded Access Programs: New Models for Stakeholder Collaboration, Program Design, Supply Equity and Access for Investigation Drugs” conference. I was pleased to share the day with colleagues such as Richard Klein (Patient Liaison Program Director, FDA’s Office of Health and Constituent Affairs) and Diane Dorman (Vice President for Public Policy at NORD).
The full program can be found at www.cbinet.com/eap.
Some points from the conference worth mentioning:
There is “therapeutic misinterpretation” of early scientific data resulting in some patients asking for access to investigational products based on mouse data! Patients see reporting of promising early data in places like USA Today and immediately call the FDA.
Better “expanded access IQ” isn’t just reserved for patients – but is also needed for physicians. Many doctors are unsure of just what there roles and responsibilities are relative to expanded access protocols. Is this even taught in medical school? And then there's the issue of physicans being concerned (and rightfully so given our litigious society)) about liability issues.
The urban myth that adverse events reported through expanded access programs derail the success of FDA approval is not supported by facts. Richard Klein repeatedly told the conference that (to his knowledge) there were no examples of this – and he’s contacted his FDA colleagues in search of such circumstances. Perhaps it’s time for industry to gather examples – if they, in fact, exist – and share them with the FDA as a tool to correct the problem.
Is “expanded access” even the right term to use? It means one thing in the US, and completely unrelated things in other countries. In the UK, for example, “expanded access” is a reimbursement strategy for approved products.
How can IRBs be restructured to expedite review rather than being a bottleneck?
In the words of the great health policy guru, Buffalo Springfield, There's something happening here / What it is ain't exactly clear.
Perhaps, as part of the FDA’s current initiatives to enhance both the timeliness and weight of the patient voice, expanded access plan development and execution should involve patient organizations. Maybe it’s time to harness that power to make the process both more-inclusive and better.
Another issue that remains at-large is who pays for access to these unapproved drugs? What a company can charge is regulated (via draft guidance), but sometimes the drug company will bare all costs, other times some costs, and just as often it’s the patient who writes the check. And IRB and other related costs are often borne by the patient. Perhaps there’s a role for the Federal government.
How about a fund that pays for access for any approved FDA expanded access IND or protocol? I propose that this issue should be a key part of the pending 21st Century Cures legislation being drafted by Representative Fred Upton, the Chair of the House Energy & Commerce Committee and by Senator Lamar Alexander in the Senate.
All sides want the same thing -- expedited expanded access programs. But name-calling and bridge burning doesn't bring anyone closer together or experimental drugs to dying patients any faster.
Let’s expedite access by enlarging the Expanded Access Ecosystem.
My complete keynote remarks (including commentary on Abigail Alliance v. von Eschenbach and the Right-to-Try debate can be found here.
