2006N-0062

Expanded Access to Investigational Drugs for Treatment Use

FDA Comment Number :

EC17

Submitter :

Dr. Mark Thornton

Date & Time:

03/13/2007 06:03:16

Organization :

The Alliance Against Exceptionally Rare Diseases

Category :

Consumer Group

Issue Areas/Comments

GENERAL

The proposed regulations for expanded access do not go nearly far enough. On March 1, a federal appeals court will hear oral arguments in the case of a lawsuit to change systems at the FDA to allow terminally ill patients access to promising drugs that have completed Phase I testing. Because of my former role in the oncology division at the FDA, and in my experience as a cancer patient advocate, I can shed some light on the regulatory policy, medical drug development and patient rights issues surrounding this case, and why it is reasonable and timely. Victory in this lawsuit, or passage of the ACCESS Act (modified to apply to allow access to patients that cannot participate in a clinical trial) is what is needed to provide proper access to patients in dire need.

If the lawsuit is successful, drug companies could seek early approval of a drug for patients who are terminally ill and who are ineligible for participation in clinical trials. The only requirement would be that the drug must have surpassed Phase I clinical trials. Critics of that position claim that current regulations fully meet the needs of such patientsdoctors merely need to ask the government, on behalf of their patients, for access to any unapproved drug they wish. Change of the status quo is therefore unnecessary. If these critics could be a fly on the wall at FDA after such a special request is made by a doctor, they might change their tune. I can attest to the burden the sponsors of these requests have to go through with FDA reviewers as they run the gauntlet. Manufacturing, pharmacology, toxicology, pharmacokinetic, clinical and even statistical issues raised by FDA staff, aimed at the applying physician, can sometimes rival receipt of an audit from the IRS. Requests are on occasion withdrawn by exasperated doctors or refused by FDA, leaving the patient to fend for themselves.

Critics also claim that at the threshold for early approval for the terminally ill, i.e., post-Phase I testing, the safety of the drug is still very much a concern. The fact is that all drugs, no matter what stage of development, have the potential to evolve new safety concerns. Since a drug is safe enough for the hundreds of patients in Phase 2 of human testing, how is it not safe enough for a patient whose only option is the terminal progression of his disease?

Critics of early access also present doomsday scenarios of the impact of success of the lawsuit on Phase 3 clinical trials. Some with a vested interest in maintaining the status quo, such as medical society groups, might feel threatened if the perception is that a patient will be able to easily opt out of clinical trials and just get the drug directly. In my opinion this threat is non-existent. One criterion of the patients under consideration by the court is that they cannot even be eligible for a clinical trial. In addition, the FDA will still have enormous authority in who is truly terminal, who is eligible for other clinical trials, and whether other treatment options exist.

What about the rights of the dying patient in all this? In the case of those with cancer, the patient turns to their oncologist for options. These physicians are amongst the most well-intentioned individuals in our society. The oncologist already has much access to off-label use of approved medications for their patients in a terminal setting. The system is well suited to adapting should the court add additional options to the patients and physicians faced with advancing and otherwise untreatable cancer.

Patients have valid arguments in demanding greater access to promising agents under development, and government officials should have very compelling reasons for denying such access. New drug development will not suffer if a small minority of patients fighting for their lives, with no other options and in concert with their physician, gain access to a potentially beneficial agent with an established basic safety profile.