As drugs enter clinical trials for Huntington's, it's time to learn about and get involved in the process that will promote early access (compassionate use) for patients who desire access to drugs before the FDA approval process is complete. In this article, Dr. LaVonne Veatch Goodman reports on a new FDA plan to allow early access to drugs and reviews the history of early access in other neurodegenerative diseases.
FDA Response: On Dec. 11, 2006, the FDA submitted an 83 page proposal to amend regulations that presently control how patients can access drugs that are still undergoing the clinical trials needed for FDA approval. I suspect that the timing of this proposal was prompted by political pressures brought by the Abigail Alliance in its lawsuit against the FDA. In this lawsuit, the court ruled that patients who have serious illness and no other available treatment have a constitutional right to access drugs that have passed first safety and efficacy testing.

Though this court decision is being appealed, the FDA is responding to the compelling arguments and political pressures brought by patients with serious disease who desire early access to drugs still in development.

The Proposal: The amended regulations simplify the process for applying for early access which now is so convoluted and time consuming that it acts as a roadblock to all but a few patients and doctors. A key change is that individual doctors can request drugs for patients using a procedure (Treatment IND) set in place by the sponsoring pharmaceutical company. Previously each doctor was required to complete a separate Treatment IND procedure which effectively blocked access except by doctors and patients at academic centers.

The FDA also proposed working more closely with the pharmaceutical industry by encouraging companies to provide a drug access program when there is patient interest and request: "When FDA receives a significant number of similar requests for individual patient expanded access, the agency may ask the sponsor to submit an IND or protocol for the use." They further defined this number as 10 or more requests for the same drug within a 6 month period. In effect, this is a mechanism for patient groups (like HDDW) to petition the FDA and drug companies for specific drugs.

The FDA also proposed to better inform appropriate persons and health groups about the availability of industry sponsored access programs, but they have not defined how this will be accomplished.
History of FDA Access Programs in Neurodegenerative Disease

Though the majority of past programs have been for AIDS and cancer patients, the FDA has been willing to grant early access for various neurodegenerative diseases. L-dopa was granted conditional FDA approval for Parkinson's in the late 1960's. This kind of approval allowed not only for early access, it also allowed marketing of L-dopa prior to completion of phase 3 trials. Tacrine, the first anti-cholinesterase inhibitor for Alzheimer's, was approved for early access prior to FDA approval in 1993; more than 7,000 patients received tacrine under this program. There have also been smaller programs for amyotrophic lateral sclerosis and multiple sclerosis.

Next Steps: The FDA's proposal is definitely a step in the right direction, but it will only be useful if pharmaceutical companies are brought on board to allow access to their drugs. The FDA must provide incentives to industry sponsors for support of early access programs, because industry support and sponsorship is the initial step. If the FDA is really serious about these programs in more than a token way, they must step up to the bar, and offer real incentives to companies who run early access programs.