The U.S. Food and Drug Administration (FDA) will soon be deciding whether to approve tetrabenazine. Drs. LaVonne Veatch Goodman and Nathan Goodman put the tetrabenazine story into the broader context of providing rapid access to drugs for people with serious illnesses like Huntington's.
The U.S. Food and Drug Administration (FDA) is the gatekeeper to drug services. We depend on this agency to evaluate drug data from basic research through clinical trials, and to deliberate over the benefits and risks of new drugs. For Huntington’s patients, the many years it takes for a drug to wind through the FDA approval process means loss of quality of life and greater disability. In the case of tetrabenazine, although access isn’t easy, it is possible. Those who can afford it and have a physician willing to supervise its use are able to obtain the drug from other countries. But what about everyone else and what about new drugs? Will we be forced to wait the years it takes to complete phase 3 clinical trials and FDA final approval?

The Abigail Alliance for Better Access to Developmental Drugs is a patient advocacy organization working for earlier access to experimental drugs for those with terminal diseases. They are advancing this cause through the court system and the political process. In a case brought by Abigail against the FDA, the U.S. Court of Appeals for the D.C. Circuit ruled that “patients with life-threatening and otherwise untreatable diseases have a constitutional right to seek experimental treatments”. This issue has been taken up by the U.S. Congress as a legislative proposal, The Access Act, which would permit use of experimental drugs for seriously ill patients who have exhausted all other treatment options. In such a plan, individuals who receive the drug waive the right to sue, and permit the collection of clinical data.

As consequence of patient and political pressures advanced by Abigail Alliance, the FDA is working to streamline and expand existing programs for “compassionate” use of experimental drugs. They have said that they are working on mechanisms that will make it easier for physicians to request and receive experimental drugs for use in their patients when the drug enters either late phase 2 or phase 3 clinical trials. They will try to persuade sponsoring drug companies to perform large open-access trials in parallel to placebo-controlled trials, so that more patients can receive an experimental drug.

We at HDDW salute the work of the Abigail Alliance. We also report that this group has joined HDDW in advocating FDA approval of tetrabenazine.

The Access ActS.1956, The Access, Compassion, Care and Ethics for Seriously Ill Patients Act, short name The Access Act, filed by Sens. Brownback (R-KS) and Inhofe (R-OK) with cosponsors Allen (R-VA), Isakson (R-GA), and Sessions (R-AL). Full text from U.S. Government Printing Office