Though the number of drug access petitions on Change.org has gone up over the last five years, the number of compassionate use applications submitted to the FDA for approval has fluctuated and appears to be trending downward. (To submit an application to the FDA, a doctor must have the drug company on board to provide the drug.)
In the fiscal year ending in September 2011, the FDA received 1,200 applications for compassionate use programs for investigational new drugs, or INDs, and approved 1,199 of them, according to the agency. But in the fiscal year ending in September 2013, the most recent one available, it received applications for only 977 programs and approved 974 of them.
Compassionate use can occasionally hurt a company’s ability to bring a drug to market quickly, slowing the process by diverting the drug itself, resources and time, experts said. If a patient dies while taking the experimental drug under compassionate use –- even of unrelated causes -- it may slow down the approval process for a drug because the company has to prove that the drug didn’t contribute to the patient’s death.
“Compassionate use often fails,” NYU's Caplan noted. “It is not due to the drug –- just that the drug frequently does not cure.”
The alternative for these patients is often death, so they fight for access to the experimental drugs because they want to know they did everything they could.
"I have been given a zero chance of survival past the next few months without this drug," Change.org petitioner Nick Auden told ABCNews.com in an email last October.
Auden’s campaign was one of the most successful compassionate use campaigns on Change.org, gathering more than half a million signatures to convince Merck or Bristol-Myers Squibb to give him access to their breakthrough cancer drug. His wife came up with the idea to post a video of Auden playing with their young children to drum up support, and they named it the “Save Locky’s Dad” campaign for their oldest son, Locky, 7.
"I want my dad to get the PD-1 drug because then I can do the things I like to do with him all the time," Locky says in the video, flashing a smile that's missing two front teeth.
Merck eventually expanded access to its drug to other melanoma patients this month, but it was too late for Auden.
He died at home in November.