Last week, President Trump signed the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, capping off a long and controversial legislative journey.
Supporters viewed the effort as a defense of desperate patients against the slow, paternalistic bureaucracy that has historically been the Food and Drug Administration. President Trump at the signing ceremony claimed that the bill would save hundreds of thousands of lives and give patients what they were previously lacking: hope.
Unfortunately, this bill will likely fail to save many lives, if any. The law does not improve on the current compassionate use programs run through the FDA. It also fails to address the biggest problem: the companies that manufacture new drugs. The legislation may indeed give patients hope, but in many cases it will be a false hope, ending in crushing disappointment.
In 2016, right-to-try found a political champion in Wisconsin Senator Ron Johnson. He made the issue central to his difficult battle for re-election. Against the odds, he successfully defended his seat and immediately introduced Senate Bill 204, which would eventually be signed by President Trump.
No major action was taken on the bill since the Senate’s focus was on attempting to repeal and replace the Affordable Care Act for the first eight months of 2017. But that August, Johnson threatened to disrupt an important FDA funding bill in order to secure a vote on his right-to-try bill.
Changes to the bill were hashed out by Senators Lamar Alexander and Patty Murray, both on the Senate health committee, in order to make the legislation palatable to the sixty members needed to pass it. The full body then passed the amended version by unanimous consent in early August, sending the measure to the House.
Once again, the bill sat for months. In October 2017, the FDA Commissioner Scott Gottlieb testified at a hearing on the bill held by the House Energy and Commerce Committee. Gottlieb expressed some reservations about the scope of the bill and suggested editing the language.
The chairman of that committee, Representative Greg Walden, announced his own, updated version of Johnson’s bill in March 2018. Walden’s bill was much narrower in scope and came about as a result of extensive collaboration with FDA officials, among other stakeholders. The bill passed the House with strong Republican support and some Democratic support.
Throughout this period, both President Trump and Vice President Mike Pence heavily pressured Congress to pass some sort of right-to-try bill. Pence has long supported the right-to-try movement and signed a right-to-try bill during his tenure as Indiana’s governor. He and his staff communicated with House leadership and encouraged them to get a bill through by early 2018. Trump also applied public pressure, telling Congress it was time to pass right-to-try in his 2018 State of the Union address.
Due to this intense pressure, House leaders decided to abandon Walden’s narrowly tailored bill when it became clear that it would not pass the Senate quickly. Instead, the House voted on and passed the Johnson version that had already cleared the Senate chamber.
The Johnson bill allows a patient who is diagnosed with a life-threatening disease or condition, has exhausted all approved treatment options, is not eligible to participate in a clinical trial, and has given their treating physician a written statement of informed consent to ask a drug manufacturer or distributer for access to certain unapproved treatments. To be eligible, an unapproved treatment must have gone through a Phase 1 trial (the purpose of which is to establish safety), is actively being developed or produced by the manufacturer, and has not been placed under a clinical hold by the FDA. The bill allows the manufacturer or distributer to provide eligible drugs to eligible patients without having to obtain the FDA approvals normally required by the Food, Drug, and Cosmetic Act before drugs can be marketed to patients.
The bill further forbids the FDA from considering patient outcomes (positive or negative) associated with the use of eligible drugs under the law in its decision to approve the drug, unless the agency determines that it is critical to determining the drug’s safety.
Manufacturers, dispensers, and doctors are also shielded from liability for deciding to give the patient the experimental drug or for deciding not to give the patient the drug. But these actors can still be sued for reckless or willful misconduct, gross negligence, or an intentional tort under applicable state law.
Finally, the legislation requires each manufacturer of an eligible drug to send the FDA an annual report detailing any use of the drug under the right-to-try law. The agency then must compile its own annual report on the use of the pathway spelled out by the bill and post that report on its website.
Good Foundation, Poor Execution
The foundational ideas of the right-to-try movement affirm the patient’s ability to determine their own health. Patients, working with their physicians, have the right and the capacity to make decisions about how best to achieve their own well-being. A government agency should not have the power to forbid a patient from making a decision about which they have come to by weighing costs and potential benefits. In this respect, right-to-try is a good thing, because it promotes patient well-being.
This certainly does not mean that the FDA ought to be disbanded. The agency has a tendency to be over-cautious, resulting in serious, if unseen, losses in both lives and medical progress. But the agency has also done much good for public health by curbing predatory practitioners, serving as a source for reliable medical information, and identifying safe and effective treatments.
The right-to-try movement becomes problematic when right-to-try legislation is nothing more than the opening salvo in a total assault on the FDA. It is too easy to lose sight of what is actually good for patients in an ideologically motivated battle against medical regulators.
Soon after President Trump signed the bill, Senator Johnson sent a letter to Gottlieb requesting a meeting to discuss the implementation of the bill, and clearly stating that his legislation’s intent was to weaken the FDA. While there is certainly extensive room for reform at the agency that will result in benefits for patients, Johnson’s comments bolstered the argument that his primary purpose was a symbolic attack on a government bureaucracy and not helping real patients. This argument is further strengthened when the likely effects of the law are examined.
One of the most powerful objections that critics of the bill raised was that it did not substantially improve on existing compassionate use pathways. The FDA already has a process by which patients who have exhausted available, approved treatments and who do not qualify for any clinical trials can apply to the agency for a special exemption that would allow them to use an experimental drug outside the confines of a trial. The FDA grants around 99% of those requests, usually within a matter of hours. Additionally, the agency can offer physicians information on how to safely administer a drug for which there is little data.
Someone might counter that even the possibility that a government agency could deny a patient access to a drug is a violation of that person’s rights. That is a conversation that is both ongoing and worth having. But the main focus of medical legislation ought to be more pragmatic: Does it improve real patients’ well-being? Considering the facts of the FDA’s expanded access program, it seems this law will not make a noticeable difference.
The law also fails to address the biggest hurdle in a patient’s quest to use experimental drugs: the company manufacturing that drug.
Vibhav Rangarajan and his wife Sonal have a daughter, Radha, who has a rare genetic disease that affects the nervous system. The pharmaceutical company Shire has developed an enzyme replacement therapy for this disease and has completed a Phase 1/2 trial with what appear to be encouraging results. After obtaining the FDA’s approval, Radha’s treating physicians applied to Shire for a dose of their experimental drug through their compassionate use program. The company quickly replied, denying their request.
Radha’s family was devastated. Vibhay, a cardiologist, used every connection that he had to try and speak to someone from Shire and personally beg them to allow his daughter to be treated with the drug. The company continually rejected these requests and refused to correspond with the Rangarajans directly.
Vibhay wrote a heartbreaking account of his family’s experience in Stat, concluding that the right-to-try law is nothing more than a “cruel joke” since it fails to correct the root cause of Radha’s problem.
This is not to say that Congress should force companies to give their experimental drugs to each person that asks. These companies often have legitimate reasons for rejecting individual claims. Small pharmaceutical companies sometimes do not have the resources to both run an expensive clinical trial, and give away already scarce doses of the drug under development.
Companies also must manage scarce patients, especially when they are developing drugs for rare conditions. In order to offer a drug to most patients through the general market, a company must run several clinical trials that require large numbers of patients. Granting too many compassionate use requests inhibits a company’s ability to find enough patients to successfully conduct a clinical trial, especially when there are only a few hundred patients or less that even have a rare disease.
Pharmaceutical companies must also grapple with the possibility that someone using a drug through compassionate use will experience an adverse event and induce the FDA to reject the eventual approval application. While the right-to-try bill seeks to curb this by ostensibly forbidding the agency from considering patient outcomes, the FDA can simply claim the outcome is critical to determining the drug’s safety and circumvent this protection. And that is a good thing. The FDA ought to be able to consider an event like a death in their evaluation of the drug. It could save more lives in the future.
These are all compelling reasons to protect a drug company’s property rights and allow them to refuse compassionate use requests. Yet that means that right-to-try will never be completely effective in saving the hundreds of thousands of lives that Trump claimed it would.
At its core, right-to-try is motivated by a desire to empower patients and ultimately improve their well-being. Unfortunately, this bill seems unlikely to offer new, more effective tools to desperate patients. It does, however, run the serious risk of offering false hope to patients, many of whom will experience the same gut-wrenching despair that Vibhay and Sonal felt.
Patients and advocates should continue to work to empower patients and fix the hurdles that stand in their way, be they caused by bloated government bureaucracy, anti-competitive special interest groups, or unscrupulous providers. But there are more effective actions that can be taken that do not pose the same risks to patients as the formulation of right-to-try contained in this new law. FDA Commissioner Scott Gottlieb has spent the last year working to encourage medical innovation in emerging fields like artificial intelligence and advanced cancer treatment. Institutionalizing this pro-innovation mindset will greatly benefit suffering patients, especially over the long term.
Patient well-being ought to always be the primary goal of anyone involved in the health care field. Unfortunately, the new right-to-try law seems to fall short of achieving that goal.