In a video posted on YouTube two weeks ago, Diane Gant, 46, says that she is dying of Stage 4 breast cancer. Much like a scene from a film called 'My Life Without Me,' in which a dying mom tape records messages for her young daughters to offer advice and hope for each of their successive birthdays, Gant holds up letters to her young son in order to touch base with him when he graduates high school and college, and marries. She does not expect to be there in person.
And Gant also laments that a potentially life-saving Genentech drug is not yet available. "The most unfortunate part is that there is a very promising drug for me, pertuzumab, that the FDA is just sitting on since December and does not plan on releasing until June 2012. I am not expected to make it to June, my liver is engulfed with tumors and I am wasting away," she writes in a note accompanying the video.
The FDA, however, was not the issue. "Patients and healthcare providers seeking expanded access to unapproved drugs need to understand that drug companies must agree to provide these drugs to patients," Richard Pazdur, who heads the FDA Office of Oncology Drug Products, wrote in an FDA blog last week. The stumbling block was actually Genentech. And so, a few days and several thousand views later, the Roche unit blinked and agreed to make pertuzumab available to Gant based on compassionate use.
"Genentech is committed to a fair and impartial evaluation of each request for access to our investigational medicines, and takes these requests very seriously," a spokeswoman tells ABC News. "Appropriate decisions regarding potential access to investigational or unapproved medicines can only be made after in-depth discussions between Genentech clinical teams and the person's qualified treating doctor."
The episode underscores the debate over experimental treatments and the willingness of some patients to assume risks. Drugmakers "don't want a death attributed to their new drug," University of Pennsylvania bioethics professor Art Caplan tells ABC. "They may not have any idea how much drug to use and they often have a very limited supply on hand, which they hope to use in testing the drug not for one person's last desperate gasp at a bit more life."
"Every case is a huge maelstrom of hope, begging, corporate self-interest, slow bureaucratic due process, media spotlighting and public frustration," he continues. "It is simply very hard to solve access issues with a single policy given the many interests besides the patients that are in play in these cases. Each winds up being unique."
The Gant video, however, also illustrates how social media can change the equation. In years past, patients had to rely on a letter-writing campaign, a petition or, perhaps, a coveted connection with someone who had influence at a drugmaker in order to achieve the same result. By posting her video, though, Gant succeeded in breaking through barriers. Of course, there is also the possibility that such videos will become so commonplace that drugmakers will be unwilling to respond to each entreaty.