Barbara Kopple’s documentary film, “Fight to Live,” concerns the ability to get drugs approved by the Food and Drug Administration. The FDA approves drugs and approves the clinical trials to get the drugs in the pipeline. It allows some drugs to be used for “compassionate use” (drugs that are not approved but could be used when someone is near death).
The problem is the Food and Drug Administration has not released many drugs for compassionate use and in the case of one drug, MTP (Mifamurtide, the trade name Mepact for osteosarcoma, a bone cancer that mainly affects children and teens).
The drug was approved in Europe in March 2009, but not in the United States. In Kopple’s documentary, there is heart-wrenching testimony by a physician whose life was saved as a teenager by MTP, but there were others who lost their lives because MTP was not available to them. He survived; they didn’t.
Another drug that “Fight to Live” discusses is Iplex, which has several uses, including to treat Lou Gehrig’s disease, medically known Amyotrophic lateral sclerosis, or ALS (also known by its generic name, Mecasermin Rinfabate). The data has been less than conclusive, but why not give the drug to people who have the fatal disease? The drug is available elsewhere in the world, but not in the U.S.
Kopple’s movie shows a protest at the FDA in the fall of 1988 by ACT UP. People were demanding drugs as they were dying. The first of these protests took place in Boston, and I was present at a famous “die-in” in the summer of 1988 held by young men who had very little time to live. It took place at a conference I was coordinating for the Physicians for Human Rights.
FDA Commissioner Dr. Frank Young was speaking, and the dying young men with AIDS wanted new drugs fast tracked so they could be available to people who otherwise had no hope. Men held up watches and “died” on the stage in front of the commissioner. Some drugs were moved through the pipeline more quickly then. However, since then, many potentially lifesaving drugs have not been approved.
There have been, however, some brave souls who have taken up the cause and have devoted time and energy toward trying to get drugs to people who otherwise would have no access. One of the groups featured in Kopple’s “Fight to Live” is the Abigail Alliance, whose mission is “helping create wider access to developmental cancer drugs and other drugs for serious, life-threatening illnesses.”
They have worked to get drugs more quickly into the approval process and to get drugs approved and available for compassionate use. Faster Cures, an organization from the Milken Institute, is another group working to reform the FDA process. Their manifesto includes having patients as active participants in the process, that the process of clinical trials is “streamlined and laser focused” and that intellectual property is not “hoarded in a mattress.”
FightToLive.org, which shares the same name as the movie, declares on its mission page:
“The development and approval process for drugs customarily takes 15 or more years and costs an average of $1.2 billion.
“In today’s environment, venture capital firms, whose funding is the fuel for the innovative biotech and drug development industry, see little prospect of a return on investment and impossibly long timelines.
“Efforts to bring drugs to market are seen as thwarted by an FDA shackled by rigid guidelines outdated and irrelevant to today’s medical breakthroughs.”
Times have changed since the ACT UP demonstrations of 1988. The Internet and Twitter now exist, but those innovations may have reduced the number of protests that brought about change to combat and end the dying from AIDS. It is time we all got active. We will all die someday, and some of us sooner than our natural lifetimes.
Groups like the Abigail Alliance, Faster Cures and Fight to Live are trying to swim against the current of trial lawyers and the entrenched bureaucracy of the FDA.
We can only hope they can win what seems to many a difficult, if not impossible, fight.