June 2, 2020
By Naomi Lopez
This past week marked two years since President Trump signed Right to Try into federal law—a milestone moment for terminally ill patients seeking hope. The law allows patients facing life-threatening or terminal illness to seek treatments that are considered safe enough to test on humans but have not received U.S. Food and Drug Administration (FDA) approval, under their doctor’s care and recommendation. And two years on, we’re seeing the positive impacts of this Goldwater Institute-developed law.
When the President signed the federal Right to Try law in 2018, he was surrounded by terminal patients and their loved ones, all advocates for this important right. Matt Bellina was one of those patients. A former U.S. Navy pilot, his service to the country was cut short in 2014 when he was diagnosed with amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). Before long, he was out of treatment options. But there was another way—and that way was Right to Try. As one of the patients most strongly advocating for Right to Try, it’s fitting that he was one of the first beneficiaries of the federal law. In January 2019, Bellina began receiving an investigational treatment under the law, and within weeks, he was able to breathe more easily and even pull himself to standing from his wheelchair.
Right to Try becoming federal law was the culmination of years of hard work on the part of many individuals—individuals like Laura McLinn and her son, Jordan. Just a few months before his fourth birthday, young Jordan was diagnosed with Duchenne muscular dystrophy, a fatal muscle-wasting disease that affects about one in 5,000 boys. Luckily, Jordan was one of the fortunate few to be admitted into a clinical trial for one of the promising treatments makes its way through the federal approval process. But only a handful of boys were able to access those treatments, so the McLinns took up the cause of protecting the right of anyone with a terminal disease to fight for their lives by trying promising medicines that aren’t FDA-approved. Jordan and Laura started advocating for the Right to Try in their home state of Indiana, and Jordan was at then-Governor Mike Pence’s side when he signed the bill into law.
And individuals like Dr. Ebrahim Delpassand. In Texas alone, Dr. Delpassand helped nearly 200 patients access a treatment for advanced stage neuroendocrine cancer that had completed clinical trials but was not yet fully approved. Many of these patients were told they had only months to live but are still alive years later, thanks to Right to Try. After a two-and-a-half-year wait, that drug received full approval by the FDA.
Right to Try continues to move us closer to the important goal of bringing the right treatment, to the right patient, and at the right time. Today, during the coronavirus crisis, we should celebrate the fact that, by removing red tape and bureaucratic hurdles, Right to Try has provided a policy beacon for making treatments accessible for those patients whose lives hang in the balance.
There are currently no FDA-approved vaccines or treatments for COVID-19, and that is why the FDA should be lauded for moving quickly to begin clinical evaluation of potential vaccines and treatments for this virus. But this speed and attention on COVID-19 is a tacit reminder that, when lives hang in the balance, the drug approval process is often too slow and burdensome. That is why the Goldwater Institute championed Right to Try laws.
Clearly, there are still important steps that need to be taken to ensure that doctors and patients have access to the full range of their treatment options. There are three important steps that lawmakers and policymakers can take today to continue so that patients can realize the full potential of the Right to Try law.
1. The government’s clinicaltrials.gov website lists important information about ongoing clinical trials in the U.S. Unfortunately, the federal agencies that have jurisdiction over the management of this site are choosing to not include information about medicines available under the recently adopted Right to Try law even when the manufacturer has requested being listed.
This means drugmakers whose medicines are currently being used to save lives—and have already received safety approval from the FDA—cannot share crucial information with doctors and patients via the government’s online registry. Yet that same registry does include studies for non-approved, potentially dangerous treatments that are not under FDA review and are being administered in places such as Azerbaijan and Togo.
While not all manufacturers may wish to be listed, there is no reason why the federal government should exclude information that might make it simpler and faster for doctors and patients to learn about promising treatments that they have agreed to make available under Right to Try.
2. The federal Right to Try Act requires that general information, such as the number of patients treated under the law, be reported to the government annually and that that information be posted publicly online. Unfortunately, the rules, format, and instructions for reporting this information have yet to be issued.
At this point, we should know more about the use of Right to Try for the past two years. But until the government promulgates the reporting rules, which is a legal requirement, we won’t know who has been treated, for what diseases, and which treatments have been used. It should not take two years to establish a reporting framework for Right to Try.
3. The same attention and focus on COVID-19 should be provided to patients with life-threatening and terminal illness. While we do face a public health emergency today, other patients, like Matt Bellina, whose lives hang in the balance are no less deserving of treatment options.
More than half a million patients will die of cancer alone this year—most without access to the more than 700 new treatments already under consideration. Countless more suffer and die from terminal illnesses with no FDA-approved cure, such as ALS, Duchenne muscular dystrophy, and Alzheimer’s. But there are promising treatments for them already in the FDA’s pipeline.
The same enthusiasm we see being applied to accelerating treatments and vaccines for COVID-19 should be applied more broadly. Much of these reforms rely on removing bureaucratic obstacles and red tape.
The two-year anniversary of the federal Right to Try law is a good time to reflect on the importance of making healthcare more patient-centric. The Right to Try law was never a call to ignore research or undermine science, or for doctors to abandon obligations to patients, or for drug companies to disregard complex ethical questions such as how to distribute limited supplies of drugs. And obviously, Right to Try was not a guarantee that investigational medications would work, or that patients and doctors would have perfect information to inform their decisions.
Rather, Right to Try is about recognizing and protecting the fundamental principle of patient autonomy. Indeed, the law explicitly intends to “expand the scope of individual liberty and agency among patients, in limited circumstances.” It does this by recognizing that these deeply personal decisions belong in the hands of patients and their physicians.
Naomi Lopez is the Director of Healthcare Policy at the Goldwater Institute.