But some women, relying on the medical literature and like-minded surgeons, rejected this decades-old procedure in favor of smaller operations, including an early version of today’s lumpectomy. Opposed by most surgeons, these women took their case to the public through newspapers, magazines and television shows like Today. Eventually, surgeons had to concede that these less aggressive operations were viable options for localized breast cancers.
A decade later, when young gay men were dying of a frightening new disease, now known as AIDS, scientists developed the first medications that potentially prolonged life. Yet when doctors told these men they would need to enter a clinical trial of one of these medicines, zidovudine, in which only one-half of them would actually receive the pill, they rebelled, pleading their case in the press and in public protests. This activism led the Food and Drug Administration to speed the process by which it approves new medications.
There are many other examples of successful patient activism. In 1983, thanks to the efforts of the National Organization for Rare Diseases, Congress passed the Orphan Drug Act, which gives incentives to pharmaceutical companies to develop medicines for rare diseases that might otherwise be ignored. Perhaps the apogee of patient activism occurred when Augusto and Michaela Odone, parents of a boy with a progressive degenerative neurological disorder, leapfrogged over doctors to develop an oil that prevented the onset of the disease, a story commemorated in the 1992 film Lorenzo’s Oil.
Not all of these efforts have led to successful therapies and the saving of lives. But what activist patients have demanded is the opportunity to benefit from potentially helpful medications or procedures, such as organ transplants.
Which is why the Murnaghans and the parents of another child with end-stage cystic fibrosis, a boy named Javier Acosta, used the legal system to challenge existing rules that prohibited the use of adult lungs in children. And it is why two Illinois women petitioned their state’s Medicaid system, which funds medical care of the poor, to pay for a risky, unproven multi-organ transplant that they believed would improve their dire medical conditions.
Illinois declined to pay, terming the procedure “experimental.” But rejecting such requests is difficult, especially when patients are in pain or dying and are out of other options. Children, understandably, generate the most sympathy. There is an old expression—“You cannot ration at the bedside”—which I have always found to be true. It may be hard for insurers to say no to patients in need, but it is even harder for health professionals to do so.
We are thus left with a difficult dilemma. The Affordable Care Act promotes the use of cost-efficient interventions while seeking to improve health care coverage for the neediest Americans. But the ACA is being implemented in a country with a legacy of vibrant patient activism, which has not only saved lives, but has also promoted innovation and, at times, enabled poor patients to obtain unproven or expensive innovations that they otherwise could not have afforded.