While the Right-to-Try Act may appear to have a patient-centric focus, it will likely increase risk for seriously ill patients and weaken the FDA’s power to protect patients taking experimental medication.
President Trump recently signed the “Right-to-Try Act” which allows patients with a “life-threatening disease or condition” to try experimental drugs without the approval of the FDA. Though the administration has touted the law as a success for patient-centric healthcare, many doctors and healthcare professionals aren’t so sure.
What Right-to-Try Means for Healthcare
On its face, the Right-to-Try Act seems to be a sensible, patient-centric piece of legislation. The law gives terminally ill patients access to potentially life-saving medication that has passed Phase 1 clinical trials for safety and tolerability. Once they’ve run out of approved regimens and regulated clinical studies, these patients will now need only the permission of their physician and the drug manufacturer to begin experimental treatment.
While this measure does offer new opportunities to terminally ill patients, the FDA already has a program in place for terminally ill patients who are not eligible for clinical trials to access experimental — but regulated — drug treatments. Through this system, called expanded access or compassionate use, the FDA approves 99% of requests within one day for emergencies and four days for non-emergencies.
The difference between expanded access and the Right-to-Try Act is that expanded access drugs have passed Phase 2 clinical trials. The drugs in that program have been determined to be effective and useful, while Phase 1 drugs have only been tested for safety. Additionally, most of these treatments will not be covered by insurance, meaning that patients could spend thousands of dollars on a drug that may not have any chance of working.
A Negative Impact on Patients
By weakening the FDA’s power to regulate experimental drugs, Right-to-Try introduces potential dangers for the healthcare industry. The FDA’s role is to protect patients, and is the most knowledgeable about little-known risks or proper applications of medication. It is therefore best prepared to prioritize safety and offer guidance to terminally ill patients seeking experimental treatments. Removing the Phase 2 requirement prevents the FDA from being able to protect seriously ill patients from unknown side effects.
In addition, Right-to-Try could jeopardize the success of some clinical trials. Patients taking the drug outside of a regulated study may experience unexpected complications, affecting the public’s perception of the treatment. This could cause investors to pull their support for a potentially life-saving medication. Clinical trial participants could even withdraw from existing studies if they hear about side effects for patients outside of the trial.
The shift toward patient-centric healthcare has been a largely positive one, and most of us would agree that patients should be able to advocate for themselves. But laws like the Right-to-Try Act shift the balance too far toward the patient. When it comes to experimental drugs, the FDA and medical professionals should be determining which drugs are safe and effective – and which are not. While Right-to-Try is presented as a benefit for terminally ill patients, it could potentially do more harm than good in some cases.
Patient centricity is an important and growing trend that is vital to the success of many clinical trials, but does right-to-try ultimately serve patients’ best interests? This new legislation may, however, have the positive effect of making more people aware of experimental treatments, prompting patients to begin an important dialogue with their healthcare providers.