Answering to Patients Who Yell the Loudest

Considering the sometimes crucial but sometimes costly role of patient activism


Few people would argue that scarce medical resources should simply go to patients and families who yell the loudest, but the recent case of a ten year-old Pennsylvania girl with cystic fibrosis shows how such a strategy can work. The parents of Sarah Murnaghan went to court to demand that their daughter be placed on the transplant list for new lungs.  The court agreed, and she has now received a second set of lungs after her immune system rejected the first set.

There is a long—and storied—history of activist patients bucking the system to obtain treatments initially denied to them. These successes have created strategies, often involving the media, for other desperately ill people to follow.  But in an era of rising health care costs and passage of the Affordable Care Act (ACA), which seeks to reign and regulate spending, the privileges of activist patients need to be reexamined.

There have always been patients who pushed their physicians to obtain unusual therapies, but modern patient activism can be traced back to the efforts of women with breast cancer in the 1970s.  At that time, the treatment of choice for the disease was the disfiguring radical mastectomy, which entailed removal not only of the cancerous breast but the underarm lymph nodes and chest wall muscles on the side of the cancer.

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.

So what can be done? At the very least, it makes sense to openly acknowledge how individual instances of activism and system-wide cost containment may often be directly at odds. That is why the media attention to the recent cases in Pennsylvania and Illinois is so helpful.  Understanding how money allotted for certain procedures may prevent funding of other, more scientifically proven interventions—particularly those that prevent disease—can promote healthy debate among insurers, health care professionals and the public. And, whenever possible, patients should be enrolled into clinical trials of innovative new medications or procedures, even if they, themselves, may ultimately not benefit.