The FDA is proposing that doctors have easier access to experimental drugs for patients suffering dire illnesses who have no other options for treatment.
An editorial published in the New York Times commended the feds for reducing the time involved in applying for the right to use an experimental drug for an ill person from 100 hours to less than one.
The FDA’s proposal reflects a policy implemented when the AIDS epidemic began. Its intent was to allow “compassionate use” of experimental drugs even though their safety and efficacy hadn’t been proved. The policy has expanded, but, like many regulatory functions, has become too complex.
Currently, doctors must provide 26 separate pieces of information accompanied by seven attachments; the new form seeks only eight kinds of information, including the patient’s clinical history and the doctor’s reason for choosing the experimental drug, and a single attachment.
In a breath of fresh bureaucratic air, the FDA won’t deny doctors who want the drugs now, although the proposed new process, which was announced in early February, won’t be final until the 60-day comment period has elapsed.
But be advised that just because someone wants an experimental drug that doesn’t mean he or she will get it. As The Times wrote, “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 feds, of course, can’t force a manufacturer to provide a drug to someone; its role is providing guidance for doing it. But the FDA authorizes most requests, the paper reported, within days or even hours.
Terminally ill people are entitled by law to try experimental drugs that have passed at least the first of three phases of testing required by the FDA to assess safety. Drug trials take a long time, so these patients are allowed access to them when they’re still years away from being marketed. But manufacturers aren’t required to provide the treatment, and insurers are not required to pay for it.
The laws are known as “right to try,” and are intended to get medicine to seriously ill patients by bypassing the FDA’s role in moving drugs to market. “Once a doctor and patient decide that an experimental drug is the right choice,” The Times explained, “the laws let them apply to the drug company directly.”
The “right to try” is legal in five states – Colorado, Louisiana, Michigan, Missouri and Arizona, and 26 others are considering such a law. Delaware is the most recent, having introduced legislation earlier this month.
So far, the editorial said, “right to try” has not helped anyone obtain a drug, but supporters hope some patients will have access and benefit from it soon.
Of course such quick action is not without risk – there’s a reason drug trials take so long, and many manufacturers welcome the FDA’s role in the process. The agency “has more information about potential risks and benefits of drugs under development than a doctor or patient is apt to know,” as the newspaper noted.
But the government’s reliance on drug manufacturers to self-report side effects from their products has been called into question, so even if you’re out of treatment options and desperate for help, know that the effects of even drugs not considered experimental might be unknown.