The parents of Charlie Gard announced on Monday that they’d given up on treating their 11-month-old child, who suffers from a rare and deadly gene mutation affecting his mitochondrial DNA. The roller-coaster case began in February when physicians at the London hospital treating the infant said it was time to remove Charlie’s life support. They refused to let the British couple fly him to New York City for a last-ditch, experimental treatment that, according to its inventor, had a “small but significant chance” of reversing his brain damage. Over the past five months of legal battles, the hospital never wavered from its claims that every reasonable means of saving Charlie had been tried already and that he should be spared any further suffering that might come with a form of therapy that has never been tested on a patient with exactly his condition, and which isn’t part of any clinical trial.
Charlie’s parents now say that it’s too late for any intervention and that it’s time to “let him go.” But for several months now, the #CharlieGard saga has served as the focus for a broader push for patients’ rights in Washington. Conservative politicians were quick to champion Charlie’s parents’ cause—President Trump tweeted his support and the House tried to grant the couple permanent residency—in keeping with the GOP’s strong endorsement of so-called right-to-try laws. These measures—lately passed in 33 states—are meant to guarantee very sick people access to “experimental or nonconventional” medical treatments that haven’t yet passed muster with the Food and Drug Administration. In practice, that means the parents of a dying patient such as Charlie wouldn’t need to ask permission from the FDA to move ahead with therapy; they could just request it directly from the manufacturer.
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It’s hard to argue with vocal patient-advocates who say their lives were saved by gaining access to experimental treatments. The right to try sounds like common sense: It should be up to patients to decide whether the potential upside of a treatment (surviving a terminal illness) seems worth any risk of painful side effects. Why not let them give it hell and go down swinging? But stepping back from anecdotes, the spread of experimental access laws (like the calls for Charlie’s puddle-jumping medevac) suggests that critical decisions about the final months of people’s lives are often based on biased judgments of reality. Patients seem to overvalue innovation, as a rule, and assume that newer drugs have a better chance of working than any other treatment, just because they’re new. Not only does this sanguine view of scientific progress fail to fit the facts; it also leads patients to the converse, false impression that “nonconventional” treatments aren’t likely to be harmful in themselves. A more sober view suggests that the hope that often moves people to seek out these types of treatments—and the ever-present pressure to fight until the end—is not as useful as we think.
Unfounded optimism tends to be the rule in medicine. A 2015 review of several dozen studies of people’s expectations from treatment, comprising data from more than 27,000 subjects, found systematic evidence of a Pollyanna Patient problem: We overestimate the value of the care that we receive and underestimate its harm. That work is cited in an excellent article by Liz Szabo of Kaiser Health News, on the surprising ineffectiveness of cancer drugs that have been FDA approved. It’s not just that these treatments do little to prolong survival, Szabo says; according to one study, many patients never grasp this fact. In a sample of several thousand adults, 39 percent said they believe the “FDA only approves prescription drugs that are extremely effective”; 1 in 6 asserted that “drugs that have serious side effects cannot be advertised to consumers.” Neither statement is even close to being true. According to Vinay Prasad, an oncologist and expert in evidence-based medicine at Oregon Health and Sciences University, we don’t have any hard evidence of benefit—in terms of patients’ living longer lives—for the majority of cancer drugs approved in recent years.
If FDA-approved drugs often fail to offer substantive benefits, then experimental ones—those that haven’t even passed the suspect bar for agency signoff—are even less likely to be helpful. In fact, about 90 percent of experimental treatments flunk out during clinical trials, either because they aren’t shown to be any more effective than the standard treatment or because their side effects are too severe. In some cases, experimental treatments once thought to be miraculous—like the use of bone-marrow transplants as a cure for breast cancer, which started in the 1980s—have turned out to be worse than ineffective in clinical trials. In the bone marrow case, the procedure could be deadly on its own. This abysmal failure rate persists in spite of the enormous cost of running trials and researchers’ clear incentive (read: bias) to produce positive results.
Such dire stats have done little to discourage eager patients, though. When it comes to clinical trials, we seem to harbor a version of the favorite–long shot bias—the tendency of horse-track gamblers to overvalue the underdog at the expense of the odds-on favorite. In medicine, this translates to fixation on the value of experimental treatments—and the remote possibility that they might turn out to be wonder cures. Indeed, for those who are faced with imminent death, the desire to bet one’s health on long-shot drugs (and the right to do so, when all other options have been tried) is so insistent that patients even deride clinical trials as another structure blocking access to potentially life-saving treatments. The trials’ randomized treatment groups and stringent inclusion criteria mean the majority of patients never get the chance to serve as guinea pigs at all.
In certain cases—think of early AIDS drugs or Ebola vaccines—this rigidity can indeed have tragic consequences. But how much rigor should be sacrificed, and how many rules should be suspended, on behalf of patients whose expectations may be substantially inflated? In late June, that question served as the backdrop for a two-day symposium of doctors, bioethicists, patient-advocates, and public-health officials on the future of randomized controlled trials. The problems with RCTs are legion, speakers said: They’re not well-suited to emerging threats; they’re too expensive; they’re too slow.
But it seemed just as clear from the proceedings that patients should think twice before they clamor for inclusion in these trials and for greater flexibility in their administration. “The randomized trial is the single greatest medical innovation of the 20th century,” said Prasad, who was in New York City for the meeting. But he warned against the use of massive studies of experimental treatments that may have only very tiny benefits in the end. It’s unethical, he said, to put so many desperate patients on a drug unless you have good reason to believe in its effectiveness.
Even in this era of informed consent, patients may not understand exactly what they stand to gain (or lose) by entering a trial. Research going back to 1982 has found that many suffer from a “therapeutic misconception”: They assume they’ll benefit personally from being in a clinical trial, though in fact they may not get the tested treatment—and even if they did, chances are it wouldn’t help. (In fairness, some researchers now say this problem has been overstated.) “My advice is, you’re better off in the control group,” warned former FDA chief Robert Califf in his keynote lecture at the symposium, speaking to prospective patients in the audience who had been arguing for greater access to experimental drugs. “Most things don’t work or they’re dangerous.”
The fact that an experimental drug is usually a bad bet isn’t likely to dull our instinct to gamble on untested treatments, though. The idolatry of experimentation has even spawned a sinister, for-profit industry, lurking in the shadows of the FDA approval process. In a disturbing paper published last week, bioethicist Leigh Turner describes how the government website ClinicalTrials.gov—a registry established in 1997 to improve the reliability of formal research on potential treatments—is being used to market sketchy medical practices. Patients who are looking for a way to break into a clinical trial may scan the registry for opportunities to volunteer; now, instead of finding only legitimate, government-sanctioned research trials, they could land on so-called patient-funded or patient-sponsored ones. In these, they have to pay for access to a therapy that isn’t necessarily based on any peer-reviewed, preclinical data, and which may lack any evidence of safety or effectiveness. (Already there have been reports of patients suffering severe complications from their participation in these ersatz trials.)
What makes us so gung-ho for things that aren’t fully tested? It may in part be human nature, but aspects of the bias seem to be conditioned, too. Even honest science coverage tends to focus on putative medical breakthroughs that have either just occurred or may be coming soon; less scrupulous media figures hawk salves or potions with little basis whatsoever. Taken altogether this creates an intoxicating atmosphere of progress—a sense that new and better treatments are always on the verge of coming out.
Yet the excitement in the air rarely matches up to reality: Actual medical advancement tends to be incremental and excruciatingly slow. The discord this creates—between the feeling of innovation inspired by the media and the real options that we’re offered in the clinic—may distort our view of experimental treatments. It could make us think there must be some reason why our cancers haven’t yet been cured; there must be some external factors preventing us from getting access to the new and better drugs we’ve heard so much about. If only regulators weren’t so overcautious and uptight, we end up thinking, it would be possible to tap this cache of innovation.
Right-to-try laws indulge the fear that unbending bureaucrats in Washington have kept patients from medical cures with an excess of red tape. In fact, these laws have little real effect. That’s because the FDA already offers access to experimental treatments with very modest oversight—and in recent years the agency has done away with a few unnecessary rules that slowed the process down. The problem isn’t that patients (or their parents) have insufficient freedom to decide how they’d like to balance out potential risks and benefits from experimental treatments. It’s that our bias often makes them victims of false hope.