Right to Try 2.0 doubles down on a bad idea
By Katarina Torres Radisic
Katarina Torres Radisic recently graduated from Northeastern University with a behavioral neuroscience and journalism degree. They aspire to work with people with disabilities and continue their reporting to make science understandable to everyone.
June 16, 2022
We’ve all seen headlines about “miracle drugs” and “cures right around the corner” that are in fact still early in development. For patients with terminal conditions, the physicians who care for them, and reputable drug developers, this stream of false hope can be infuriating. But for the former President of the United States to promise these experimental drugs would become easily obtainable to everyday folks and then be wrong about it is on another level.
The Trump administration signed the Right to Try Act into law on May 30, 2018, allowing patients with life-threatening conditions to access experimental treatments without the FDA’s involvement. Just two months ago, Arizona Gov. Doug Ducey expanded former President Trump’s legislation by proposing Right to Try 2.0. The new bill, also known as Right to Try for Individualized Treatments, is similar to the original law but focuses on treatments that would need to be individualized “based on [each patient’s] genetics”. At this time, this is the only information available to the public on this new version of the act - and it’s maddeningly vague.
At a White House ceremony addressing the initial Right to Try law, former President Donald Trump said he believed this bill would save a remarkable number – “hundreds of thousands” – of lives. But there is little evidence the law has lived up to that expectation, and opponents say such a law was never needed.
Under the current Right to Try bill, patients who have exhausted all other options and are unable to participate in a clinical trial of a requested drug can bypass the FDA and receive the drug for experimental use. According to the FDA, an eligible investigational drug is one that is in active development and has completed at least one Phase One trial (at which stage there is usually no reliable information as to whether the treatment works). But as much as we may feel patients deserve the “right” to try any therapy that may save or improve their life, intentionally dodging FDA control to make this happen is not the answer.
The original Right to Try Act reconfigures the existing Expanded Access program (also known as “compassionate use”) that was enacted to make experimental drugs accessible to patients. Under that program, a patient and their physician decide whether they want to try an investigational drug outside of a clinical trial. If they choose to do so, they request the drug from the manufacturer, and if the manufacturer agrees to provide the drug, they then petition the FDA for sign-off. Although Expanded Access and Right to Try are similar, Right to Try doesn’t require FDA’s okay. Instead, the patient and physician go to the manufacturer and if they agree to deliver the drug, that is the end of the process. Right to Try 2.0 works in a similar way and is considered another political attempt to weaken the FDA.
In fact, the original Right to Try Act was drafted by the Goldwater Institute, a conservative public policy research and litigation organization, with the explicit goal of cutting the FDA out of the decision-making process. The Goldwater Institute was formed in 1988 with the mission to promote “limited government, economic freedom, and individual liberty.” They believe the FDA, which they refer to as “government red tape,” should not stand between patients and potentially helpful treatments – the sole reason why they claim the Expanded Access program fails patients. The Goldwater Institute is a member of the State Policy Network (SPN), a web of right wing “think tanks” that focus on state-level policy. Of course, the institute works on behalf of whoever funds them - meaning that their anonymous donors make the decisions. Although the institute claims to be funded only by individual donations, IRS records show that some of their largest donors are out-of-state charitable organizations with political agendas, including several conservative nonprofit foundations. In 2019, the Goldwater Institute made $5,624,841 in revenue. It appears the point of Right to Try was to score political points for the Republican party and profit off the effort.
In their defense, the FDA reports that they approve 99.7% of applications within days through the Expanded Access program. In a 2017 statement, FDA Commissioner Dr. Scott Gottlieb said that “emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days.” And despite the Goldwater Institute’s view of the FDA as an obstacle to access, manufacturers are not required to provide drugs to patients, and many are reluctant to approve use outside of clinical trials. They fear that adverse outcomes could hurt their stock price or hinder their ability to obtain FDA approval and worry that if they agree to provide a drug to one patient, they must approve all patients who request it.
A study performed by Cardinal Health Specialty Solutions, an organization that supports biopharma companies through the drug development, initiation, and approval processes, found that taking the FDA out of the equation did not improve success rates in gaining access to unapproved drugs. Between 2013 and 2018, the FDA received about 9,000 Expanded Access requests and authorized 99% of them. More than 100,000 patients have received access to investigational drugs through Expanded Access over the past 30 years. The FDA continues to improve the program and, in 2015, streamlined the required supporting documentation for requests, decreasing the time it takes physicians to complete the form from eight hours to 45 minutes. And according to Pfizer, the FDA’s recent efforts to “simplify their website, provide factsheets for patients and physicians, and provide updated expanded access guidance” had already resolved many of the concerns that prompted legislators to pass Right to Try.
In most cases, the reason for the infrequent denial of unapproved drugs is to decrease serious harm to patients, something Right to Try does not address. In response to former President Trump’s praise for and promise of experimental treatments, about 40 groups that represent patients with life-threatening conditions addressed Congress regarding the potential damage that the law could cause. In a letter, the organizations reminded congresspersons that the existing regulatory guidelines that took over 50 years to establish were developed to protect patients from exploitation and injury.
Aside from safety, there are other concerns about both Right to Try laws. For patients to try an investigational drug, they must first provide written consent through documents that can be difficult to understand. After all, because these drugs haven’t been approved by the FDA, there is no official label listing side effects, dosing constraints, and other important information.
Additionally, the Right to Try Act does not require or encourage insurance companies to cover the costs of the experimental drug or treatment. Denial of coverage for an experimental therapy can leave families with significant out-of-pocket expenses and financial hardship. With so little oversight, the law limits the liability of physicians and pharmaceutical companies if there are adverse or negative results. However, although the threat of legal action is mentioned as a barrier to manufacturer participation under expanded access, such liability protection likely isn’t needed, since there has never been a single lawsuit.
Frustratingly, Right to Try 2.0 doesn’t seem to address any of the original law’s issues. While 1.0 seems to have fallen well short of expectations, instead of shoring up oversight or regulating patients’ expenses, 2.0 simply aims to expand 1.0’s sentiment to a relatively small set of bleeding-edge drugs, continuing to talk the same big game Trump did without producing results.
The original Right to Try law was sold to the public as a way to provide patients with life-threatening conditions the opportunity to access more treatments and raise awareness of experimental treatments. With limited information available on doses given and patients treated, Right to Try 1.0’s success rate remains unknown, but seems underwhelming. At a request to attain these numbers, the FDA responded by stating the data are collected, but not yet published. The reality is that no one has the “right” to try investigational drugs. Right to Try legislation simply undermines a key regulatory institution while creating false expectations for dying patients who are willing to take a risk on treatments that could do more harm than good.
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