Parkinson Pipeline Project-treatment development database, research, FDA drug approval, PD clinical trials and sponsors, patient involvement

Expanded Access to Experimental Therapies

“A major goal of drug regulation must be to speed the journey from laboratory to bedside of important new drugs for devastating illnesses.” (1) This shift in emphasis by the FDA, pharmaceutical industry, and health professionals allows patients who are seriously ill or have life-threatening conditions to access investigational drugs not yet on the market.

Treatment use of experimental drugs is generally grouped into two categories according to the number of people treated: expanded access and single patient use.

One type of expanded access program is called the Treatment IND, which allows use by large numbers of patients of investigational drugs that have shown promising results in clinical trials, but have not yet reached the marketing stage. There cannot be other approved, satisfactory treatments currently available for these patients. They receive the treatments outside of the ongoing clinical trials.

Single patient use (sometimes called “compassionate use”) is the treatment use of experimental drugs for an individual patient rather than a group of patients. The FDA allows an individual patient to become in effect, his or her own study. The FDA can grant a special exception for single patient use when a patient might benefit from an unapproved treatment still in development, but is ineligible for the current clinical trial. The commercial IND sponsor would provide the drug and would be responsible for reporting to the FDA.

However, if the commercial sponsor is unwilling to assume responsibility for a special exception, an investigator or the patient’s doctor may take responsibility for treating the patient. They would need to obtain the drug from a manufacturer, apply directly to the FDA for an IND and report information about the effects of the drug to the FDA. Additionally, an Institutional Review Board must evaluate the situation and grant approval.

The FDA may grant a compassionate use or Treatment IND (investigational new drug) if:

The patient gives informed consent, understanding that the drug is not approved and may cause side effects from mild to fatal.
No comparable or satisfactory alternative drug is available.
The drug does not expose patients to an unreasonable and significant additional risk of illness or injury.
The drug is under investigation in a controlled clinical trial (Phase II or III) or all clinical trials have been completed.
The sponsor is pursuing marketing approval with “due diligence.”

"The FDA has worked diligently to balance two compelling, and sometimes competing, factors," says FDA Commissioner Jane E. Henney, M.D. "On one hand, there is the need for the disciplined, systematic, scientifically controlled studies necessary to identify treatments that may improve patient health and that lead to the approval of new drugs. At the same time, there is the desire of seriously ill persons, with no effective options available, to have the earliest access to unapproved products that could be the best therapy for them." (2)

Are the risks greater than the benefits when trying an experimental medication? That depends on your viewpoint. Even if an unproven treatment does not increase the long-term survival prognosis, the patient and the family may feel better knowing they are taking action and are not simply victims of some serious disease.

FDA has no authority to require that the company make its drug available outside of the clinical trial.

Staff from the Office of Special Health Issues and the Center for Drug Evaluation and Research's drug information branch often provide detailed information and explain the process for getting access to an experimental medication, the agency does not steer patients in one direction or the other. Information is provided so patients, in consultation with their physicians, can make their own informed decisions.

"Continuing to shorten review times and work with industry to shorten development times for drugs, biologics and medical devices is the best way to provide all Americans with access to useful medical treatments", Dr. Henney said.

Treatment INDs benefit everyone in that they generate useful information about how specific drugs affect larger segments of the patient population than might otherwise receive it in a clinical study. The process:

Involves wider treatment use of unapproved agents.
Accelerates the FDA’s new drug application review process without compromising approval requirements for safety and effectiveness.
Shortens the time devoted to pre-approval drug testing, eliminating unnecessary, duplicative studies, and expediting the review of innovative agents for the most serious or life-threatening conditions.
Encourages FDA reviewers to help drug developers plan studies that generate information necessary to make decisions about approvability.

The FDA has more than 13,000 active drug and biologic studies on file. These range from a few dozen to as many as 50,000 patients participating in a single investigational new drug trial. More than 100,000 patients are enrolled each year in nation-wide National Institutes of Health-sponsored studies. (2)

Since the final treatment IND rule was published more than a decade ago, the FDA has made more than 40 drug or biologic investigational products available to patients early and has approved 36.

Biomedical research advances rapidly and breakthroughs come from unexpected places, all feeding the hope that the next experimental drug will be the one that cures our ills. (2)

Sources:

FDA Consumer magazine, January-February 2000, “Experimental Treatments? Unapproved but Not Always Unavailable” , by Larry Thompson

Code of Federal Regulations 21CFR312.34, Treatment use of an investigational new drug.

Food and Drug Administration. Protecting human study subjects