The Right to Try legislation that was passed simply removes the FDA from the equation once a drug has passed phase 1 clinical trials. So why are the authors of the Right to Try Act so eager to exclude the FDA from the process? It seems they think doing so will improve access to investigational drugs — a nonexistent problem.
Pharmaceutical companies are not obligated to provide experimental drugs to patients, and patients do not have a constitutional right to try investigational products.6 Proponents of the bill argue that under previous compassionate use programs, the manufacturer’s obligations to monitor patients for adverse events, submit safety reports, and provide physicians with guidance was the major impediment to big pharma offering patients their investigational drugs. Yet wouldn’t any reasonable individual want this level of oversight when using an experimental drug?
The FDA does concede that the process of providing medications through an expanded access pathway does require significant human effort6 for intermediate-to-large groups of people; however, for individuals the process is fairly simple. There is evidence indicating that when companies try to regulate themselves, they incur much larger costs than when administering drugs under one of the FDA’s expanded use pathways.7 Nonetheless, recall the reasons why the FDA was established in the first place – to protect consumers and patients from harm.
Moreover, Right to Try proponents like to argue that dying patients have nothing to lose5 — that is not true. They can lose quality of life and precious time. A few more meaningful days with the people we love can be infinitely meaningful. Is that not the underlying motivation for expanded access to these experimental drugs in the first place?
Let’s get real about investigational drugs: most never get approved because they are either found to be either unsafe or not efficacious.5 The most likely outcome of using unapproved investigational drugs is either harm or no benefit at all.5,8 In fact, only 11% of drugs being tested get approved for use. So, if we really want to be honest about Right to Try, then we ought to discuss experimental therapies as likely to be harmful with a very slim chance of therapeutic efficacy. Instead, politicians and proponents of Right to Try talk about investigational drugs as if they are “promising” and “life-saving” — as if there is a high probability that these drugs will work. We know that those statements are false. Talking about these medicines as if they are guaranteed to be promising and lifesaving only contributes to the misinformation and false hopes of patients with devastating diagnoses. As James Hamblin, MD, senior editor at The Atlantic writes, Right to Try is disingenuous.
With that in mind, do we really want to offer these unproven substances to frail, terminally ill patients, knowing that those drugs are more likely to lead to a deterioration in quality of life and possibly even accelerate death? Well, maybe. If we can make a rational argument based on the limited available data that an unproven therapy may help patients and offer that intervention to them in a controlled setting with regulatory oversight, then it may be worth the expanded use of that unapproved drug. But that isn’t Right to Try —that is the status quo established by the FDA that Right to Try undermines. In fact, in about 10% of cases, the FDA makes safety recommendations to expanded use cases that would otherwise not occur under the newly approved law.5
Maybe a less obvious consequence of the Right to Try legislation is the effect it might have on our ability to obtain clinical trial data; hence the reluctance of pharmaceutical companies to participate in expanded access programs. Unless the requirement that patients who cannot otherwise qualify for a clinical trial is strictly enforced, then we might find ourselves in a situation where patients are obtaining investigational drugs outside of clinical trials, further delaying drug approvals by making it difficult to obtain sufficient participation in clinical trials.5 Imagine that your patient qualifies for a clinical trial but is told that there is a 50% chance that they may not get the investigational drug. Why would they take the chance of getting a placebo when they can obtain the drug outside of the clinical trial? This legislation may threaten future clinical trials and ultimately slow progress to safe and efficacious therapies. In fact, a Baker Institute Policy Report by Rice University found this to be the case in states that have passed right to try laws.7
Although there may be some good reasons for pharmaceutical companies to offer experimental drugs to patients outside of the FDA approval process — like tax breaks, additional revenue streams, and establishing goodwill in markets where competitors may come in with alternatives — there are also equally significant reasons why a pharmaceutical company might abstain from expanded access, including diminished future earning potential by homogenization of the drug price across markets and slowed regulatory approval resulting from reduced participation in clinical trials.8
Opponents of the law are particularly worried that the law would be misinterpreted to include chronically ill patients, and eventually, patients who are simply unsatisfied with their current treatment options.5 This would ultimately undermine the FDA’s goal of limiting broad access to medicines that have not been proven to be safe and efficacious and further exacerbate the problem of reduced clinical trial participation.
Although the Right to Try bill is emotionally appealing, it does little to truly improve expanded access programs and it weakens the FDA’s ability to monitor safety concerns and offer recommendations. Right to Try legislation also potentially opens the door for abuse by providers, patients, and pharmaceutical companies that could diminish the ability to obtain reliable and timely clinical trial data needed to make investigational products accessible to all who need them.
The concerns with the 2017 Right to Try Legislation are so significant that 38 patient advocacy groups and physician professional organizations came together to oppose the legislation before it was signed into law.3 Yet, despite their concern, it was still signed into law by the President. The sensationalized and emotionally charged arguments that appeal to our life-loving human nature have surpassed the rational concerns of individuals, physicians, and institutions who seek safe and affordable drugs that are proven to work. So, while politicians in Washington may be celebrating this “victory,” those of us who can see beyond the propaganda must follow intently to mitigate the possible consequences of this law that threaten to further destabilize our fragile healthcare system.
- Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017. S 204, 115th Cong (2017-2018).
- Hamblin J. The Disingenuousness of “Right to Try.” The Atlantic. https://www.theatlantic.com/health/archive/2018/06/right-to-try/561770/. June 2, 2018. Accessed June 29, 2018.
- History, Art, and Archives. United States House of Representatives. The Pure Food and Drug Act. http://history.house.gov/HistoricalHighlight/Detail/15032393280. June 23, 2018. Accessed June 29, 2018.
- Hamowy R. Medical Disasters and the Growth of the FDA. Independent Policy Report. http://www.independent.org/pdf/policy_reports/2010-02-10-fda.pdf. February 2010. Accessed June 29, 2018.
- Joffe S, Fernandez-Lynch H. Federal Right-to-Try legislation — threatening the FDA’s public health mission. N Engl J Med. 2018;378:695-697.
- Darrow JJ, Sarpatwari A, Avorn J, Kesselheim AS. Practical, legal, and ethical issues in expanded access to investigational drugs. N Engl J Med. 2015;372:279-286.
- Rubin MJ, Matthews KRW. The impact of Right to Try laws on medical access in the United States. The James A. Baker III Institute for Public Policy of Rice University. May 2016. Accessed June 29, 2018.
- Borysowski J, Ehni H-J, Górski A. Ethics review in compassionate use. BMC Medicine. 2017;15:136.