Right to Try and Expanded Access to Investigational Therapies
On May 22nd, Congress passed H.R. 878, the Right to Try (RTT) Act of 2017. Although SNO is enthusiastically in favor of the expansion of life-saving treatments, many patient and physician advocacy groups have come out in opposition of RTT due to potential unintended consequences. SNO, too, is concerned that the legislation will create a riskier environment for patients and could put the progress of clinical trials in jeopardy.
Due to patient safety concerns and a belief that physicians should receive dosage and side effect information from the FDA, SNO recommends that physicians continue to guide patients to utilizing the compassionate use program administered through FDA.
Below you will find “Frequently Asked Questions” which run through what RTT seeks to accomplish and why supporters believe it’s necessary.
Frequently Asked Questions
Q: What is the Right to Try Act?
A: Right to Try (RTT) is legislation that attempts to allow terminally ill patients access to investigational treatments that have passed Phase 1 of FDA testing but are not yet available on pharmacy shelves. Patients with life-threatening diseases who have exhausted all approved treatment options and cannot participate in a trial of an experimental drug will be allowed to take an unproven medicine without the permission of the FDA. Only the patient, his or her physician, and the drug manufacturer would have to agree to administer the treatment.
Q: What options have these patients had until now?
A: Through their physicians, patients could access experimental drugs though an FDA system called compassionate use or expanded access. In the past, this process was criticized for being too time consuming, but FDA now states their application process only takes 45 minutes to complete. The agency approves more than 99 percent of requests within four days, and emergency requests can be granted over the phone.
Q: If these patients can apply through FDA, then why is federal action needed?
Previously, drug companies have been reluctant to allow patient access to unproven treatments due to limited resources to handle compassionate-use requests or limited quantities of the drug. Companies also fear this will cause patients to avoid trials, or that adverse effects could compromise efforts to continue development. The act encourages drug companies to issue approval because FDA scrutiny is taken out of the process, and the legislation protects manufacturers from any liability or increased regulatory review should a critically ill patient be harmed. However, it is important to note that even with passage of the bill, pharmaceutical companies cannot be compelled to provide anyone a treatment.
Q: Who are the vocal supporters and why?
In addition to a small number of patient groups, libertarian groups have been the driving force behind the legislation led by the Goldwater Institute. They argue that because compassionate use exceptions are only granted to about 1,200 patients a year, which is fewer than 3 percent of terminally ill patients, an act was needed to address the remaining 97 percent. Their goal is to give people with terminal illnesses the legal right to use investigational medicines years before they might otherwise be available on the market. Ultimately, they believe RTT returns choice and control over treatment options to the patients and doctors, and limits government involvement.
Q: Who has opposed this effort and why?
Many patient and doctor groups have come out in opposition to RTT, including the American Society of Clinical Oncology and the National Brain Tumor Society. On May 21, 2018, 70 patient groups signed a letter to Speaker Paul Ryan (R-WI) urging Congress not to move forward. They maintain that RTT laws lack important safeguards compared to the expanded access program by leaving physicians without guidance on critical aspects of patient care, such as appropriate dosing and managing side effects, since doctors likely have no clinical experience with the investigational drug a patient might receive. There is also the concern that patients will assume greater financial risk as treatment under RTT is not required to be covered by insurance companies. Opponents also argue that tending to the urgent needs of one patient, no matter how compelling, can slow the development of medicines for everyone else as patients who may experience negative side effects can jeopardize clinical studies.
Q: What does this mean for patients?
RTT laws were already passed by 38 states, but Congress’s passage creates a federal standard allowing involvement with just the doctor and the manufacturer, without regulatory review. The President signed the bill on May 30th, 2018, with FDA Commissioner Scott Gottlieb present. Although patients can now work with their doctors to reach out to pharmaceutical companies directly, SNO recommends emphasizing to patients that the FDA’s process is becoming more efficient and should be relied upon for safety purposes.
(Approved by the SNO board of directors 6/21/18)