Experimental Treatments? Unapproved But Not Always Unavailable
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.
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.