COURT TO HEAR CASE ON EXPERIMENTAL DRUGS
The debate over access to experimental drugs for chronically ill people continues as the US Court of Appeals for the District of Columbia in November decided to rehear a case next summer between the Washington Legal Foundation and the Abigail Alliance for Better Access to Developmental Drugs versus the FDA.
Last May, the FDA had appealed the court's decision that would have given chronically ill patients early access to drugs deemed unlikely to be approved before they die. The agency asked for the full 10-judge court to rehear the case. In December the FDA proposed regulatory changes to expand access to experimental drugs to patients under a treatment plan when there is no satisfactory alternative therapy. The proposed rules which are open for comment through March, are available at www.fda.gov/cder/regulatory/applications/IND_PR.htm.
The Washington Legal Foundation, a public interest law firm, and the Abigail Alliance for Better Access to Developmental Drugs – an advocacy group founded and named in 2001 for a young woman who died after being denied experimental cancer drugs – sued the FDA in 2003 for greater access for terminally ill cancer patients to drugs that had been tested in preliminary safety trials, but had not yet been FDA-approved.
The FDA argued that increased access to experimental medications would lead to unacceptable risk, and said that programs to provide experimental medications do currently exist. In January 2006, the FDA decided to allow researchers to test small amounts of experimental drugs in patients before formal clinical trials begin and all animal studies are completed. (See Neurology Today's “FDA Approves Earlier Clinical Trials For Experimental Drugs,” Mar. 7, 2006, page 36.)
Investigators and patient advocacy groups for patients with neurological conditions offered differing perspectives on the issue of experimental drugs. The ALS Association, according to spokesperson Gary Wosk, encourages individuals to make informed decisions. If they choose experimental drugs, they should know that such “treatments or therapies are not developed under appropriate scientific review and ethical guidelines,” he said.
The Muscular Dystrophy Association, according to spokesperson Bob Mackle, also contends that experimental therapies, by definition, have unproven benefit. “Early access to experimental therapies by individuals with chronic, progressive, and disabling conditions may provide false hope and expose them to undue risk.” He added, “Patient should have access to experimental therapies in a controlled manner through clinical trials.”
Rita Vareha, a coordinator for the movement disorder clinical outreach program at the University of Pittsburgh Institute for Neurodegenerative Diseases, who also coordinates research for the Brain Bank and the Huntington disease studies there, said that she is unaware of any neurology advocacy groups supporting the use of medications “outside of current channels.”
She said most of her patients “want to try every compound they hear about, but it is still difficult to get them to agree to participate in a study.” Vareha noted that patients, who initially hold off on conservative treatment, sometimes at the last minute express interest in the latest promising treatment.
Vareha agreed that all stages of clinical trials are necessary prior to patient access. “We cannot police ourselves, and the current system of informed consent, FDA oversight, and going logically from one step to another – however painfully slow it may appear to be to desperate patients and their families – is necessary. We also need the controlled environment of a study to give these drugs to patients so that we are certain that the outcomes are indeed related to the drug (in that dose and without confounding variables).”