The Food and Drug Administration on Thursday finalized its streamlined application process designed to help put experimental drugs into the hands of terminally ill patients.
But both supporters and opponents of the FDA's “compassionate use” program agree that it's not the federal agency, but drugmakers that need to change their processes to improve access.
FDA Commissioner Dr. Robert Califf Thursday acknowledged that the former application process could be “challenging and time-consuming” and saw the changes as “reducing the procedural burdens on physicians and patients.”
Califf said the new form should take about 45 minutes to complete. Critics had estimated the old form could take up to 100 hours. It required physicians to provide 26 types of information and included seven attachments. The new process drops the amount of information to eight types and requires only one attachment.
The FDA established compassionate use in 2009 as a way to allow patients with serious or life-threatening conditions to gain access to therapies that have not yet completed clinical trials if no viable alternative treatments exist.
While the agency has said it approves more than 99% of compassionate-use applications within a matter of days or sometimes hours, critics argue the process can take months.
Some contend the FDA should not be involved at all in granting patients access to potentially lifesaving medicines, and that those decisions should be left up solely to the discretion of drugmakers.
After all, the FDA posts information about which drugs are available for compassionate use only if drugmakers inform the agency of their availability.
"You don't wind up on the FDA page until a company has said 'We're okay with giving you whatever it is,' ” said Arthur Caplan, director of the division of medical ethics at New York University's Langone Medical Center. Caplan also heads a panel that advises Johnson & Johnson on compassionate use.
Drug companies are disincentived from participating in the program however, because they could face limited supplies of a drug or challenges in gaining FDA approval if a patient has an adverse reaction.
Christina Sandefur, executive vice president of the Goldwater Institute, wants to see access to those drugs become a mandate.
“While I applaud any effort the FDA takes to make the process easier for dying patients, we think dying patients deserve no less than the right to try, and that compassionate use should be the law of the land and not the exception,” she said.
The institute has led a national campaign to pass so-called “right-to-try” state laws. The laws allow terminally ill patients who have exhausted all other approved treatment options to request a prescription for an experimental drug or medical device that has passed the first phase of the FDA's clinical trial process. Companies are not legally required to provide the drug or device, and are not liable if they cause harm or death.
Such bills have been signed into law in 28 states with two states—New Hampshire and South Carolina—currently awaiting governors' signatures.
But critics of right-to-try laws say they offer patients no substantive advantage in gaining greater access to experimental drugs.
“There are no incentives in right-to-try laws to obligate companies to do anything,” Caplan said. "Right-to-try supporters give the impression that the FDA is a big roadblock; I don't think they are.”
Caplan says patients and physicians have difficulty finding out about clinical trials and the processes through which drugmakers make an experimental therapy available.
“Whether you know what trials are going on depends on whether your doctor is savvy and has been keeping up with literature, whether you're affiliated with a patient advocacy group that might tell you, or whether the company's website is easy to use,” Caplan said.
The FDA Thursday also reiterated that drugmakers could not charge Medicaid, Medicare nor private insurers, only patients, for the cost of experimental treatments used under compassionate use.
