HJAR Sep/Oct 2020

18 SEP / OCT 2020 I  HEALTHCARE JOURNAL OF ARKANSAS THE RIGHT TO TRY Can you please explain the difference between Right to Try, expanded access, and compassionate use? Dr. Kamal Patel While they are separate laws, at the most basic level they are truly the same thing. To put these programs into perspective, one needs to understand that there is a standard of care that dictates the recommended diagnostic and treatment process for any given malignancy. As part of their care plan, patients will undergo various forms of therapy. Once all FDA-approved available treatments have been exhausted, and the patient still has a good performance status, wemay consider enrolling the patient in an expanded access program or Right to Try for additional investigational treatments. Expanded access, also known as compas- sionate use, provides patients diagnosed with a life-threatening disease or condition access to an investigational medical prod- uct (drug, biologic, or device) for treatment outside of a clinical trial. This route is taken when the patient has exhausted all other available options. The Right to Try Act, signed into law on May 30, 2018, allows patients who have been diagnosed with a life-threatening disease or condition to have access to certain eligible drugs. These experimental therapies have completed Phase 1 testing, but have not yet been approved by the Food and Drug Ad- ministration (FDA). Right to Try sounds like a gift to terminal patients who do not qualify for a clinical trial, and in some cases might be better for an individual patient than a trial. Do you agree? Patel Both of these avenues of additional treatment options are a gift to patients, es- pecially for patients who are unable to par- ticipate in a clinical trial due to their inability to travel to a large academic facility who is hosting the trial. Financial and location bar- riers are frequent eliminators for patients when exploring available clinical trials, so to be able to make these investigative means available to patients through individual or intermediate compassionate use means is a blessing. While both the physician and the patient know the risk associated, and that it may not be the cure-all needed, at least it gives the patient one more option to try when they’ve exhausted all other available treatments. Gaining access to it at their local cancer care provider makes it easier on the patient—physically, mentally, and emotion- ally. As part of our mission, CARTI supports and participates in clinical trials as a way to provide the best treatment options to our patients, and to expand our knowledge of the most advanced cancer treatments. The clinical trials we participate in primarily look at investigational products that may extend a patient’s chance for survival with fewer ad- verse effects. CARTI’s clinical research department is currently enrolling patients in clinical trials through the expanded access program. Ad- ditionally, on occasion we have been able to secure an investigational product for com- passionate use. “When Congress passed the Right to Try Act, similar laws were already on the books in a number of states, including Arkansas. This precedent at the state level paved the way for the federal law to create a uniform approval process. I supported this effort because I believe any patient with a terminal diagnosis deserves the freedom to pursue their options, and the hope that potential treatments could offer to extend or save their lives.” -U.S. Senator John Boozman Co-sponsor of Right to Try Act