Roosevelt Institute | Cornell University

Experimental Drug Access: What Republicans Got Wrong

By Jed KaiserPublished February 17, 2019

Main Logo for the Right To Try Movement
The Right to Try Act just gave critically ill Americans access to every experimental drug that has passed Phase I FDA testing. Since only about 9.6% of drugs that pass Phase I testing will ultimately become FDA approved, what does this mean for the future of the FDA and the health and safety of the American people?

If you listened to President Trump’s State of the Union address on Wednesday, then you just may have caught Trump briefly discuss the passing of the federal Right to Try Act in May of 2018, stating that to “give critically ill patients access to life-saving cures, we passed right to try.” That statement was grossly misleading. While the argument for easing the drug approval process in the United States may seem valid in theory, as well as extremely popular among the American public, the passing of the Right to Try Act has the potential to undermine and limit the efficacy of the Food and Drug Administration (FDA), leaving millions of Americans at risk.

Access to experimental drugs before earning full approval by the FDA became a national conversation during the 1980s when the HIV/AIDS epidemic first surfaced in the United States. A diagnosis of HIV or AIDS was considered a death sentence at the time and, with no known cure and little research having been done on the subject, patients were desperate for new forms of treatment. The FDA had long been praised for the thoroughness of its drug screening process, beginning with its addition of the “Kefauver Amendment” requiring that a drug’s efficacy be proven before receiving approval. Following the HIV outbreak, however, many Americans began wondering whether such a long-drawn-out approval process was really in the best interest of the American people. Eventually yielding to public demand, the FDA established the “expanded access” program, which allowed terminally ill patients to obtain experimental medication while still incorporating FDA oversight and guidelines.

Despite the FDA’s progress in this area, many politicians have secured their constituents access to experimental drugs by passing legislation dubbed “right to try” laws, typically with significant public support. Nevertheless, the first question one should ask when evaluating such legislation is whether the drugs accessed through such laws are as safe as those approved under the FDA’s “expanded access” program. The answer? Absolutely not.

Many aspects of the Right to Try Act are extremely dangerous. Under the Act, patients and doctors can bypass FDA review and approval, bypass utilization of an oversight committee tasked with weighing the benefits of an experimental drug against its risks, and bypass an ethical review for experimental treatments. A process with so little regulation and oversight leaves consumers at risk of exposure to unsafe substances that could worsen their conditions.

Additional concerns arise because experimental drugs are only required to have passed Phase I clinical testing to be eligible for prescription under the Right to Try Act. In conjunction with lack of oversight, this presents significant risk since 70% of drugs pass Phase I, but about 90.4% of those drugs are ultimately deemed unsafe or ineffective. This means that most drugs eligible for prescription under the Right to Try Act would have never received FDA approval and any person who chooses to obtain experimental drugs in this way is thus at risk.

The legislation also contains no coercive authority over pharmaceutical companies, insurance companies, or physicians. For example, the Right to Try Act cannot require pharmaceutical companies to provide, test, or set a reasonable price for an experimental drug, leaving the legislation vulnerable to accusations of offering false hope to patients rather than actual access. Likewise, pharmaceutical companies and physicians cannot be held accountable for producing or prescribing experimental drugs unless doing so is determined to be “reckless or willful misconduct, gross negligence, or an intentional tort.”  So these industry actors may produce or prescribe experimental drugs solely for financial gain and confident in their ability to avoid any penalties or consequences, even when their conduct is otherwise negligent. Lastly, as the Right to Try Act has little power over insurance companies, patients may be left at risk of incurring crippling debt obligations because insurers may refuse to cover the costs of the experimental drugs they may need.

Possibly the most menacing potential consequence of the legislation is the undermining of the FDA’s “expanded access” program. If a patient can obtain experimental drugs under the new law they could not obtain with FDA approval, or if a patient feels they do not have time to navigate the FDA’s approval process, then they will choose to circumvent the FDA regulatory scheme, which could also lead to legislation undermining other aspects of the FDA as well.

Many of you may be sitting at home wondering to yourselves, “Okay, maybe the Right to Try Act is flawed, but do you have an alternative proposal to allow terminally ill patients to safely access necessary experimental drugs?” The answer is “Yes.”  The FDA “expanded access” program affords the same early access to drugs that have passed only Phase I testing, but also requires FDA review and approval, an oversight committee to ensure the drug’s benefits outweigh its costs, and ethical review for experimental treatments. Additionally, while the Right to Try Act leaves patients vulnerable to medical and financial harm, the FDA’s “expanded access” program requires patient protections before a drug can be approved. Lastly, while the FDA’s “expanded access” program may have stricter regulations, the FDA still accepts over 99% of requests. So why challenge the FDA’s experience and authority in drug approval?

The Right to Try Act will do more harm than good, compromising the health and safety of every American. A drug approval process so lacking in testing and oversight has no place in federal law. The FDA’s “expanded access” program has been perfected over decades in order to provide consumers with safe and effective drugs. Why fix something that isn’t broken?