Faced with "right-to-try" laws and diseases like Ebola with no known treatment, the US Food and Drug Administration (FDA) today announced that it will make it easier for physicians to request "compassionate use" of unapproved investigational drugs and devices outside of a clinical trial.
An application form that now takes 100 hours to complete will become a 45-minute chore, according to an agency proposal released for public comment before it is made final.
Compassionate use is another name for the FDA's expanded-access program, which began at the dawn of the HIV/AIDS epidemic when the agency allowed physicians to prescribe investigational drugs to patients considered otherwise doomed. More recently, the FDA has allowed the compassionate use of the experimental Ebola drug brincidofovir (Chimerix) and the antibiotic miltefosine for primary amebic meningoencephalitis. In each case, the agency has not yet determined that the drug is safe and effective.
The FDA has come under pressure to expedite compassionate-use approvals with the spread of right-to-try laws in various states. Such laws allow terminally ill patients to receive unapproved, experimental drugs and devices. The last state to pass such legislation was Arizona, in November 2014.
The FDA said that it's heeding requests for a speedier application process.
"We know why patients want access to these drugs and we know how busy their treating physicians can be," wrote FDA Acting Associate Commissioner Peter Lurie, MD, MPH, in an agency blog post today. "So we streamlined the new draft form to be shorter and simpler for physicians to fill out."
The current application form asks physicians to fill in 26 blanks and attach seven documents. The proposed application cuts down the paperwork to eight data elements and one attachment.
An organization campaigning for state "right-to-try" laws said today that the FDA is "finally getting the message," but still poses an obstacle to dying patients.
"Regrettably, a 'simpler form' is window dressing on an archaic and inhumane system that prevents the vast majority of Americans with terminal illnesses from accessing promising investigational treatments," said Darcy Olsen, president of the conservative, small-government Goldwater Institute, in a statement today. "Patients must still beg for permission to try to save their own lives — it's just a shorter form."
More information on today's FDA announcement is available on the agency's website.
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Cite this: FDA to Simplify 'Compassionate Use' Drug Applications - Medscape - Feb 04, 2015.