The Right to Try, to Hope, to Live

When Abigail Burroughs was nineteen, she was diagnosed with cancer. She tried a variety of treatments, but none worked. After exhausting all of her government-approved options, Abigail had one last hope: Her doctors identified a promising treatment called Erbitux. The drug had not yet been approved for sale by the Food and Drug Administration (FDA); it was still being evaluated in government-monitored clinical trials. But Abigail could not be admitted into the clinical trials because the trials were only testing patients with colon cancer. Abigail’s cancer was in her head and neck. “She had the right cancer cells in the wrong part of her body,” explained her father, Frank Burroughs.

Even though a viable treatment existed—one that the FDA deemed safe enough to administer to patients within the trials—federal law prohibited Abigail from using it. On June 9, 2011, she lost her battle with cancer. She was twenty-one.¹

In the United States—the supposed Land of the Free—thousands of patients like Abigail suffer and die every year while treatments that could help them are awaiting the outcome of a slow, bureaucratic federal process—a process that takes, on average, fourteen years.²

The FDA’s approval process generally consists of three phases—each requiring clinical trials with between one hundred and several thousand test subjects. During phase one, experimenters evaluate how safe drugs are, testing about one hundred people.³ During phase two, which can take up to two years, experimenters continue to assess not just safety, but also efficacy in trials of between one hundred and three hundred people. The third phase tests the drug against placebos as well as the currently available treatments. Phase three typically requires between three hundred and three thousand test subjects. For some treatments, there is yet another phase of clinical trials, and on the whole, only about a quarter of drugs make it through the process.⁴

Because this process can take so long, countless patients such as Abigail find themselves blocked from using medicines that have passed basic safety testing and are being administered to other patients in clinical trials.

After losing his daughter, Frank Burroughs founded the Abigail Alliance for Better Access to Developmental Drugs to fight on behalf of other terminal patients for a freer, fairer system.⁵ In 2003, the Abigail Alliance and Washington Legal Foundation filed a petition asking the FDA to adopt a policy that would let terminally ill patients access treatments after they passed phase one clinical trials.⁶ The FDA denied the petition.

Abigail Alliance sued, arguing that terminally ill patients have a constitutional right to access experimental drugs that have been proven safe.⁷ A three-judge panel of the D.C. Circuit Court of Appeals initially ruled in the Alliance’s favor. However, when the other Circuit Court judges reconsidered the case, they reversed that ruling. The right of terminal patients to decide for themselves whether to use investigational medicines “is not fundamental,” they said, and was outweighed by the act of Congress that established the cumbersome FDA approval process.⁸

After this defeat, Mr. Burroughs and the Abigail Alliance were successful in getting legislation passed that speeds up the FDA approval process for certain drugs. However, Congress shelved a second proposal that would permit all terminally ill patients to access treatments that are still undergoing testing.⁹

An Unjust and Immoral System

When the FDA was created more than a century ago, it was charged with focusing on ensuring that products marketed to the public were safe and correctly labeled,¹⁰ so that patients had truthful information with which to make informed decisions. Over time, federal law shifted from a focus on empowering patients to one empowering regulators to make decisions affecting the lives of hundreds of thousands of people.¹¹

Among the many problems with the FDA’s drug-approval system is that the agency has stronger incentive to prevent treatments from reaching the market than to approve them. If a drug that might work is held back from the market, few patients will even learn of the fact, and Congress will do little or nothing to penalize the FDA for its slowness. But if an unsafe drug does reach the market, the FDA will be subjected to humiliating public hearings and condemnation. This makes the agency risk-averse in ways that inevitably result in greater suffering and earlier death for many patients.

That risk-adversity is reflected in strict rules that the FDA uses in administering clinical trials. Clinical trials are an important aspect of scientific methodology, helping scientists determine whether and how a drug works. (I won’t address here the question of whether the government should be involved in evaluating drugs.) But the rules governing clinical trials are strict, and because trials aim to investigate drugs, not to cure patients, those rules often exclude people because they are considered “too sick” or “too healthy” to satisfy the scientific protocols.

As a result, the system can have unintended—and horrific—consequences. For example, Jenn McNary’s sons Austin and Max were diagnosed with Duchenne Muscular Dystrophy—an incurable, fatal muscle disorder. Jenn tried to enroll both boys in a clinical trial for a promising treatment; Max was admitted to the trial, but Austin’s disease had progressed too far, so he did not qualify. Jenn was forced to watch while Max improved under treatment, whereas Austin’s condition worsened until he could no longer dress himself or use the bathroom without help. By the time the FDA allowed Austin into a different trial three years later, his disease had progressed to the point that he was unable to regain the ability to walk.¹²

When government denies a patient’s right to decide for himself (or his child) whether to pursue medical treatment, it often leaves patients to suffer.

What we have long needed is a return to a system that recognizes and respects the rights of individuals to make their own decisions about their health care, a system that protects people’s right to try medicines, especially people whose lives hang in the balance—regardless of what government agents think of those medicines.

The Principles of Right to Try

In order to sustain his life, a person must make all kinds of decisions: what to eat, where to live, what type of work to pursue, whether to go to school, how to spend his time and money. Each person must weigh his options, interests, opportunities, and so forth, in order to decide, given his preferences and risk tolerances, how best to live his life and pursue his happiness.

Individual rights, as enshrined in the United States Declaration of Independence and Constitution, recognize this fact and—in principle—protect each person’s mastery over his own actions. The most basic of all rights is the right to one’s own mind and body.¹³ That right includes not just such things as the right to choose what one eats and drinks, or whether to move from one place to another, but also the right to decide whether to seek medical treatment—and what kind of treatment to seek.

Few situations more clearly demonstrate why humans require liberty than those involving terminally ill patients. Zoning laws, for example, require us to ask permission from the city or county before we build a shed or a fence, restricting the way we use our own property. Professional licensure laws require us to ask permission from a state agency before we can design floral arrangements or blow-dry hair, restricting our ability to earn a living. But federal drug regulations require people to ask the FDA for permission before taking potentially lifesaving drugs—in some cases, determining whether we will live at all. Although each of these laws keeps people from living their lives on their own terms, federal drug regulations are an injustice of another magnitude. They keep people from acting on their judgment in life-or-death situations.

Why should government have the power to make life-or-death decisions for individuals? It shouldn’t. For government to do so is to violate the individual’s right to life. Even if the FDA had all of the information necessary to make the “right” decision about treatment for an individual—which it doesn’t and can’t—the agency still would not have a moral right to make that decision.

It is unethical for government to violate a person’s right to seek medical treatment, regardless of whether the treatment has been approved by the FDA. Yet, regulators have long been empowered to do exactly that.

A system that prioritizes the preferences of bureaucrats and politicians over each person’s right to shape his own destiny is fundamentally inconsistent with the principle that each person owns his life.

A New Solution: The Right to Try

Decades of effort (beginning in the 1980s) to change the slow, bureaucratic system in Washington met with little success. Although the FDA occasionally would promise reform, changes rarely materialized. For example, pharmaceutical companies explained that they would be more willing to provide treatments to terminal patients if they could be assured that the FDA would not hold it against them in the event that patients—who willingly and gladly took the drugs—did not improve or suffered adverse effects. The FDA claimed that it rarely penalized companies in such cases, but it refused to put that policy in writing, making companies hesitant to offer treatments outside of clinical trials.

Even more cynical was the FDA’s “expanded access” policy—a rule that allowed terminal patients to access drugs prior to approval if they submitted information and obtained the agency’s permission. The agency claimed that the form was easy to fill out and that some 90 percent of applicants were approved. In reality, the form required detailed information that patients often could not obtain or doctors did not have time to compile, particularly for the sickest patients who most desperately needed access. The result, as the CEO of one pharmaceutical company put it, was that applying for “expanded access” was like forcing dying patients to climb a mountain first: “If you . . . deliver the application at the top level of Mount Everest, they will approve it.” But most patients couldn’t even take the first steps.

“Expanded access” missed the point, anyway. Patients did not want a different way to beg the FDA for permission. They wanted the government to recognize their right to act without permission.

Along with patients, doctors, and policy makers nationwide, the Goldwater Institute crafted Right to Try legislation. It offered a new approach that began at the state level. Although states aren’t involved in the drug approval process, they—not the FDA—regulate the practice of medicine. Right to Try aimed at leveraging states’ constitutional powers in order to put medical decisions back in the hands of patients and their physicians. State Right to Try laws would guarantee that states would not punish patients, doctors, or drug makers when terminal patients were treated willingly with drugs that were still undergoing FDA testing.

Opponents of Right to Try argued that it might open the door to unsafe and unproven treatments. But that argument ignored the fundamental point that choices about medical treatment should be up to patients, not bureaucrats—even if those decisions involve risks. Patients already are free to take serious risks. Chemotherapy, after all, is not safe, in the sense that it is, technically, poison.¹⁴ Acetaminophen kills more than four hundred people per year.¹⁵ The federal drug approval system allows healthy volunteers to test medicines that could be fatal. And in five states, terminal patients have the option of ending their lives with a physician’s help.¹⁶

Right to Try’s opponents also claimed that the law would give patients “false hope” by leading them to believe that a treatment could help them when it would not. But it was the opponents of Right to Try, who were essentially advocating for the violation of patients’ rights, who were quashing hope for many. “I have no hope now,” said California firefighter Mike DeBartoli, suffering from debilitating Lou Gehrig’s disease. “I will take false hope. . . . I don’t know who the FDA thinks they are protecting. Who are they to tell me what I should be hopeful or not hopeful for?”¹⁷

In fact, regulations both create “false hope” for some and lead others to give up all hope—in the first case by mandating that some patients get placebos instead of treatments in clinical trials—and in the second case by barring qualified candidates from receiving treatment altogether.

By contrast, Right to Try reclaims for patients the right to consider all options and to act on their own best judgment. It thereby rekindles hope and gives them a chance to live.

State Right to Try laws proved a spectacular success. Over the course of four years, Right to Try was approved in forty states by large bipartisan majorities. It helped numerous patients across the country. For instance, Houston-based oncologist Dr. Ebrahim Delpassand successfully treated more than 150 terminally ill cancer patients using LU-177 (or Lutetium Dototate), a drug that had successfully completed three phases of FDA-approved clinical trials and that was widely available in European countries for many years but would not be approved by the FDA until January 2018. When the FDA would not allow additional patients into Delpassand’s FDA-governed treatment program, he invoked his rights and those of patients recognized under Texas’s Right to Try law. Without treatment, “many of these patients were only given three to six months to live,” he said. “Now, a year later, many of them are still alive.”¹⁸

Finally, the wide adoption and incredible success of Right to Try created a groundswell of popular support that Washington officials could not ignore. On May 30, 2018, President Donald Trump signed S.204, the federal Right to Try Act, which protects the right of patients nationwide to seek treatments not yet approved for sale by the FDA.¹⁹ No longer can bureaucrats stand between the likes of an Abigail Burroughs and her only chance at life.

Though much more work still needs to be done, Right to Try is a critical step in the effort to restore and safeguard patient autonomy. It reclaims for all patients the freedom to choose whether to pursue a course of treatment. Right to Try thereby restores hope and gives many a chance to live.

About Christina Sandefur

Christina Sandefur is executive vice president of the Goldwater Institute and coauthor of the Right to Try model legislation upon which the state and federal legislation is based.

View all articles by Christina Sandefur →