Experimental Treatments: Getting Access, Beyond Clinical Trials

by Food and Drug Administration (FDA)

(Page 2 of 3)

Getting Access

For patients in search of a cutting edge treatment, the possibilities have improved dramatically. First of all, there are more clinical studies under way than ever before. FDA has on file more than 13,000 active drug and biologic studies. These range from a few dozen patients to as many as 50,000 participating in a single investigational new drug trial. More than 100,000 patients are enrolled each year in National Institutes of Health-sponsored studies conducted all over the United States.

Studies with investigational new drugs can be conducted by the federal government, primarily through the National Institutes of Health; by research universities, usually with federal funding, though also through private foundations or drug companies; and by private, for-profit companies on behalf of pharmaceutical manufacturers.

Clinical trials are essential to the development and approval of new drugs. In these studies, a group of human volunteers receiving the investigational therapy are compared with another group that receives either the standard treatment or a placebo. Placebos, sometimes called sugar pills, are any fake treatment that has no therapeutic benefit. This allows the researchers to compare the effect of the treatment to no treatment in otherwise similar patients. When the control group is given the standard treatment, researchers are able to determine whether the experimental treatment provides a better outcome than what is already available.

The clinical trial setting helps ensure that risks are minimized because the research protocol, the set of rules by which the clinical trial is conducted, have been scrutinized by FDA and a local ethics committee called an institutional review board.

"We want to encourage people to participate in the clinical trial process because that is where information is best developed about the drug product," says David Lepay, M.D., director of the division of scientific investigation in FDA's Center for Drug Evaluation and Research.

The downside of being in a clinical trial, indeed the downside of using any unproved medication, is that the new drug may not work. It may even be dangerous and, sometimes, deadly.

Not everyone who wants to participate in a clinical trial can do so. Limits on the number of participants and specific eligibility criteria keep some people out. In addition, it is often inconvenient for the patient to travel to the research center.

When individuals are unable to participate in a clinical study, FDA provides alternative mechanisms for patients and their doctors to get their hands on a promising new drug.

Beyond Clinical Trials

In 1987, FDA created a regulatory mechanism (first proposed in 1982) to permit expanded access to investigational drugs outside of controlled clinical trials. The "treatment IND" allows people with serious and life-threatening illnesses to take investigational drugs while the products are being tested in a clinical trial. Typically, however, drugs allowed under treatment INDs already have shown promise and proven safety. In addition to the benefit to individual patients, treatment INDs generate useful information about how the drug affects larger segments of the patient population than might otherwise receive it in a clinical study.

For example, the AIDS drug Videx (ddI) was made available to people with AIDS outside the clinical trial at a time when the choices for AIDS therapy were few and many people had already exhausted the then available options. Although patients seeking treatment with ddI were told that it was still under study and that there were risks, more than 20,000 decided to take ddI anyway. This not only gave them a better chance to survive but also gave researchers more information about the drug's safety than would have been possible from the some 4,000 patients involved in the clinical studies.

Since the final treatment IND rule was published more than a decade ago, FDA has made more than 40 drug or biologic investigational products available to patients early and has approved 36. Of these, nearly a dozen were for cancer and another dozen for AIDS or AIDS-related conditions.

Single-Patient INDs

As with a clinical trial, there may not be an appropriate treatment IND for an individual patient's condition, but there may be a new drug still working its way through development. If enough is known about the drug's safety, and there is some clinical evidence of effectiveness, FDA may allow a patient to become his or her own study. This so-called single-patient IND, or compassionate use IND, virtually ensures that any patient can get access to any investigational new drug.

Although FDA's requirements for a single-patient IND are relatively simple, setting up this kind of access for an individual patient is not. First of all, the company must be willing to provide the new drug to the patient. This can be expensive and time consuming for the company since, in addition to providing the drug, the company needs to track shipments of the drug, create special instructions for its use, and create a way of collecting safety data and a mechanism for tracking outcomes for each patient. Second, the patient must give informed consent, understanding that the drug is not approved and may cause side effects from mild to fatal. Third, the patient's physician must be willing to take responsibility for treating the patient and agree to collect information about the effects of the drug.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.