Patients Speak Louder Than Politics

Right to Try became federal law through bipartisan grassroots advocacy

By Christina Sandefur

You wouldn’t know it from examining today’s drug approval system, but there was once a time when federal regulations empowered patients to make informed treatment choices. Years of growing bureaucracy, however, have dramatically transformed this system into a tangled web of red tape that can be impossible for patients to navigate. Today, rather than ensuring that physicians and patients are equipped to make their own decisions, the government has become the decision-maker. It restricts what treatments doctors can prescribe and patients can take—all because it thinks it knows what’s best for us. Fortunately, this system is starting to change, thanks to a new reform called Right to Try, which allows dying Americans to access safe investigational treatments without having to first get permission from the FDA.

Patients vs. Paternalism 

At their inception over a century ago, federal drug regulations focused on ensuring that products marketed to the public were safe and correctly labeled, so that patients had the facts to make informed decisions about the medicines they were going to take. Originally, federal law didn’t require manufacturers to obtain federal permission before marketing their products. Then in 1938, Congress passed the Federal Food, Drug and Cosmetic Act, requiring manufacturers to prove that a drug was safe before marketing. At that time, the statute did not mandate federal evaluation of a drug’s efficacy, only of safety.

But federal law gradually shifted from a focus on empowering patients to make safe choices to a slow, costly, paternalistic approach that delays availability rather than eases suffering. This trend reached fruition in 1962, when Congress passed the Kefauver-Harris Drug Amendments, compelling manufacturers to “provide substantial evidence of effectiveness”—not just safety—for their products.

These amendments were passed in reaction to the infamous incident involving thalidomide, a sleep aid sometimes prescribed to pregnant women as a treatment for morning sickness, which was found to cause birth defects. But thalidomide posed a safety problem, not an efficacy problem (it actually was effective in alleviating morning sickness). And it was not approved in the U.S. In fact, only 17 of the more than 10,000 worldwide thalidomide-related birth defects occurred in the U.S., and American consumers had been protected by already-existing safety rules. Nevertheless, American politicians used a foreign safety concern as an excuse to erect additional roadblocks.

The result is today’s complex, multistep drug approval process, which can go on for decades before the FDA approves a medicine for sale—even a potentially lifesaving drug. Until the multistage testing process is completed, pharmaceutical manufacturers may not sell it, and doctors may not prescribe it. And because these stages of approval can take so long, patients often find themselves blocked from using medicines that have not only passed basic safety evaluations but are currently being administered to other patients in clinical trials. As a result, countless patients suffer and die, unable to access medicines that could help them—and that the FDA considers safe enough to administer to those patients fortunate enough to be allowed into clinical trials.

Americans have lived with (or died under) this system for more than half a century. Despite patients’ desperate demands, only once did the bureaucrats yield, when intense political pressure from AIDS sufferers prompted the federal government to take minor steps to improve its process. In 1987, the FDA instituted the “compassionate use” program, also known as expanded access, allowing people to obtain investigational medications outside the traditional (and extremely limited) clinical trial mechanism. But while political pressure changed the law, it did not change the mindset at the FDA. The expanded access process is extremely cumbersome and time-consuming, and decades later, it is still applied inequitably, on a case-by-case basis.

In the midst of this deadly delay, it became clear that decades of efforts to change the system in Washington were yielding little success. Patients were no longer interested in finding different ways to beg the government for permission to obtain treatment. Instead, they wanted to tear up the permission slip. And that change would come from the ground up, starting in state capitols and with the people themselves.

Overcoming Bureaucratic Inertia 

In April 2014, Colorado became the first state to adopt a Right to Try law. Over the next four years, 40 other states adopted Right to Try, each time with overwhelming bipartisan support in state legislatures. Dozens of newspapers endorsed the bills, including USA Today and the Wall Street Journal, and celebrities such as actress Mira Sorvino advocated for the reform. The movement became so successful that it got Washington’s attention, and even as the FDA refused to change course, Congress could no longer turn a blind eye to patient suffering.

California’s Right to Try sponsor, Democratic Assembly member Ian Calderon, explained the strategy in 2015: “[T]he only way you can get change from the FDA is pressure from the states.” In 2014, the Right to Try movement inspired a U.S. Senate investigation of the FDA’s compassionate use process and a House bill to prevent the FDA from blocking implementation of any state Right to Try law. In 2016, the Senate held hearings on the issue of access to investigational drugs, and a Senate bill complementing the House proposal was introduced that same year.

The FDA had long maintained that its system worked, but the Right to Try movement exposed the difficulty (and often impossibility) of patients’ securing treatments or even obtaining a compassionate use exception. Ultimately, the FDA was forced to acknowledge that its program was too cumbersome and slow. In 2016, the FDA said it would establish an “expanded access navigator” to guide patients through the FDA’s labyrinthine application process (though it took the agency nearly two years to roll out the website). Two months later, the agency announced that it had streamlined its compassionate use process, purportedly reducing the paperwork burden of filing a compassionate use petition.

After decades of FDA obstinacy, this was significant progress, at least in the eyes of The New York Times and the Wall Street Journal, both of which took note of the announcement’s importance. The Times called it a “breathtaking reduction of red tape.” But the Journal was more insightful. “The decision to expedite access,” it noted, either “means the agency knows its processes are broken or is merely an act of bureaucratic realpolitik.”

These improvements were welcome—and a clear indication that Right to Try had transformed the national conversation about patient rights. But the FDA’s shorter forms and hand-holding websites didn’t address the system’s fundamental flaw—the rule barring access to potentially lifesaving medicines without a government permission slip. It became clear that Congress would have to force that change.

In February 2017, Vice President Mike Pence, who as governor signed Indiana’s Right to Try law, invited terminal patients and their families to the White House to talk about protecting medical autonomy rights. Shortly after, Pence announced on Twitter that he and President Donald Trump supported “restoring hope and giving terminally ill patients a fighting chance” with Right to Try. Six months later, the U.S. Senate unanimously passed a Right to Try law sponsored by Wisconsin Senator Ron Johnson, a Republican, and Indiana Senator Joe Donnelly, a Democrat, along with 45 additional bipartisan co-sponsors.

Then Trump made Right to Try a focal point of his 2018 State of the Union Address. “People who are terminally ill should not have to go from country to country to seek a cure,” he said. “I want to give them a chance right here at home. It is time for Congress to give these wonderful Americans the ‘right to try.’” That’s when Right to Try hit its first (and, really, its only) speed bump.

In March 2018, the U.S. House took up Right to Try—and the bill failed. The legislation had been fast-tracked by a special motion and required approval by at least two-thirds of the representatives, which it narrowly failed to garner. For the first time since the Right to Try concept had been introduced in the states, most of the opposition was partisan and came from Democrats, who parroted the usual objections a handful of university bioethicists had been voicing since the reform’s early days. But for years, scores of Democratic legislators had supported Right to Try in their states, Democratic governors had signed the bill into law and Democrats from across the country had even traveled to Washington to advocate for the bill. The reform was the same, but its endorsement by a polarizing political figure had suddenly put years of success in jeopardy.

The people ultimately spoke louder than the politicians. Pressure from patients, doctors and advocates continued to mount, calling on Congress to put aside partisan politics for the benefit of dying patients. And only one week later, the House took up the bill again and passed Right to Try by a majority vote, with bipartisan support. 

The Present and the Future

Though Right to Try was signed into federal law in May 2018, it took more than five months for the FDA to grudgingly acknowledge the law’s existence and Congress’ intent to “promote access and protect patients.” But thanks to Right to Try, desperate patients now have new hope and an alternative pathway to access treatments—one that doesn’t require bureaucratic buy-in.

Now that it’s the law of the land, Right to Try is not just saving lives across the country—it’s changing the way we think about the drug approval process. It’s forcing the federal government to respect the doctor-patient relationship, stop stifling innovation and get patients better access to the best treatments. And it’s paving the way for a system that enables patients to decide for themselves, rather than the government dictating to them the terms on which they can live—and save—their lives.