The Leaflet: Patients Gain Access to Life-saving Medicines

Published June 1, 2018

After years of advocating for increasing patient’s freedom to access treatments that may save their lives choice President Donald Trump signed into law the Right to Try Act (RTT). Thankfully, RTT allows people with life-threatening diseases or conditions who have exhausted FDA-approved treatment options to finally access drugs still undergoing clinical trials. The trial drugs still must pass initial safety tests but do not have to complete the decade-long approval process before becoming accessible to patients thanks to RTT.

Heartland Institute President Dr. Tim Huelskamp was present at RTT’s signing ceremony, along with Senator Ron Johnson (R-WI) who was the bill’s primary sponsor and champion. When the Trump administration reached out to Dr. Huelskamp earlier this year for possible policy objectives, Huelskamp suggested RTT, which Trump mentioned positively during his State of the Union Address.

Widely supported by Americans across the political spectrum, RTT legislation has already passed in 40 states. The Right to Try Act mirrors the state-level laws, both of which were crafted by the Goldwater Institute who has led the national RTT effort. The latest state to pass this commonsense legislation is Rhode Island, where Neil Fachon, a 20-year-old student, was diagnosed with an aggressive pediatric brain stem tumor and had to go to court to access a trial drug the FDA halted in clinical studies. Although Fachon eventually got access to the investigational drug, it was too late. Fachon died almost a year after the diagnosis.

Similar stories of patients with life-threatening conditions having to go to extreme measures simply to obtain a drug or treatment that could potentially save their lives are far too widespread. For example, some patients have had to go to court to access drugs, travel to another country to get non-FDA-approved treatments, or create their homebrew versions of investigational drugs. These unnecessary obstacles terminally ill patients face have generated understandable outrage over the bottleneck drug-approval process that gives the FDA monopoly control over which drugs become available to patients. The drug-to-patient pipeline has become clogged with unnecessary rules and restrictions—resulting in a process spanning more than a decade. Even the most promising drugs cannot be streamlined and must adhere to the overly rigorous three-phase clinical trials, not to mention the preclinical trial phase, which typically takes up to six years.

Some critics of RTT are worried that experimental drugs may accelerate patients’ symptoms. However, not allowing terminally ill patients to take experimental drugs essentially guarantees the patients will pass away within the estimated time frame. Drugs still undergoing clinical trials could extend the lives of these patients, giving them and their loved ones a renewed sense of hope.

With the passage of the Right to Try Act, Americans have more autonomy in making important health care decisions that often make the difference between life and death. Policymakers should weigh the risks and rewards of increasing patients’ access to clinical trial drugs. Legislators in states without an RTT law should continue to push for one at the state level. The passage of RTT is not the end but rather the beginning of an era in which patients finally have access to the newest life-saving drugs sooner and at a lower cost. The Heartland Institute is building on RTT’s momentum by promoting an even more robust agenda to increase patients’ freedom: Free to Choose Medicine.


What We’re Working On

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