In response to patient demands for access to unapproved medicines, the US Food and Drug Administration and the Reagan-Udall Foundation, an independent, nonprofit created by Congress to help the agency accomplish its work, will hold a public meeting next week to create a navigator. What is a navigator? An online tool that would help patients and doctors find drugs that quality for an FDA program called expanded access, or compassionate use. These are drugs not currently approved, but are being tested and may be suitable to help someone who has a fatal illness. The notion seems helpful, but Alison Bateman-House, a bioethicist and public health researcher at the New York University Langone Medical Center, believes the navigator would be a Band-Aid. We asked her to explain…
Pharmalot: What is a navigator supposed to do?
Bateman-House: That’s still being decided. There are a number of options on the table. It could be a portal to get information on expanded access, to follow up a request for a medicine, or to follow up to see if a medicine was clinically helpful or not. It may also tabulate requests so we can have data.
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Any navigation system should include a history of successes and failures (and what is success or failure). Desperate patients are unduly encouraged to try experimental drugs without recognizing they typically do not work and cause in many instances side effects described as “torture.” False Hope is not to be encouraged!
The answer is to make compassionate use available when there is at least preliminary evidence of efficacy. The optimal time would be for the sponsor to incorporate a compassionate use proposal into the End-of-Phase 2 Meeting with FDA. Unless there is some evidence the drug works you basically take the compassion out of compassionate use.
My, my, talk about damning with faint praise ….. “We do not want a webpage that promises assistance only to result in frustration. We already have one of these, and it is called ClinicalTrials.gov.”
Actually, that’s just damning – period, at least as I read it.
Hello Observer,
Thanks for writing in. My sense is that this is a way of delivering a message and trying to shape the discussion, of course, before the meeting next week.
Regards
ed