The Food and Drug Administration has proposed a greatly simplified process for doctors to obtain experimental drugs for patients who are suffering from serious or life-threatening illnesses and have no other alternative. In a breathtaking reduction of red tape, the simplification should reduce the time it takes a doctor to apply for experimental drugs from 100 hours to less than one hour. Their desperately ill patients can only benefit.
Instead of making doctors provide 26 separate types of information and seven attachments, the new form asks for only eight elements of information, one of which requires the patient’s clinical history and the doctor’s rationale for wanting to use the experimental drug, and a single attachment.
Although the proposed simplified form won’t become final until after a 60-day comment period, the F.D.A. says it won’t turn away doctors who want to use it before then.
The proposal was announced last week by Dr. Peter Lurie, associate F.D.A. commissioner for public health strategy and analysis. Dr. Lurie described the new form as a continuation of a policy that started in the early years of the AIDS epidemic to allow “compassionate use” of experimental drugs even though their safety and efficacy had yet to be demonstrated. That policy has since been broadened, but, in the process, it has become too complex.
Not every patient who wants an experimental drug can get it. Patients are eligible only when there is no other product that can diagnose, monitor or treat the patient’s disease or condition and the patient cannot be enrolled in a clinical study testing it. The doctor must determine that the probable risk from the experimental drug is not greater than the probable risk from the disease. And the doctor must ensure that the manufacturer is willing to provide it. The F.D.A. can’t compel the manufacturer to provide the drug to an individual; it simply offers guidance on how to do it. Once an application has been filed, the F.D.A. authorizes a vast majority of requests within days or even hours.
The new policy comes on the heels of a campaign by the libertarian Goldwater Institute to persuade states to pass “right to try” laws to make it easier for terminally ill patients to obtain unapproved drugs. So far, “right to try” is now law in five states — Colorado, Louisiana, Michigan, Missouri and, most recently, Arizona. Some 26 other states have had legislation introduced.
The laws give terminally ill patients the right to try experimental drugs that have passed at least the first of three phases of F.D.A. testing (to determine safety) but are still years away from reaching pharmacy shelves. The laws don’t require manufacturers to provide the treatment or insurers to pay for it.
Instead of relying on the F.D.A. to move quickly, the “right to try” laws seek to speed up access by eliminating the F.D.A. from the process entirely. Once a doctor and patient decide that an experimental drug is the right choice, the laws let them apply to the drug company directly.
There are downsides to that approach. Many manufacturers prefer to keep the F.D.A. in the loop. And there could be safety issues in some cases. The F.D.A. has more information about potential risks and benefits of drugs under development than a doctor or patient is apt to know. Thus far, the laws have not helped anyone obtain a drug, but backers are hopeful that some patients will gain access in coming months.
The F.D.A. has no position on the state “right to try” laws but encourages patients to use its new simplified process for obtaining experimental drugs.
An editorial on Feb. 12 about patients’ use of experimental drugs incorrectly stated that a Food and Drug Administration form could take a doctor 100 hours to complete. That estimate applies to the time it would take manufacturers to apply for clinical drug trials using that form.