Pennsylvania has just joined 37 other states in enacting “right-to-try” (RTT) legislation. RTT laws allow patients to seek access to investigational drug treatments without oversight from the Food and Drug Administration if they meet certain conditions. These conditions vary somewhat by state, but generally include that a patient has a terminal disease; has considered, but not necessarily received, FDA-approved options; has a prescription from a physician; and has signed an informed-consent document.
The Pennsylvania Society of Oncology and Hematology and American Society of Clinical Oncology represent the state’s oncology care providers, so we understand that Act 33, “an act providing for the use of investigational drugs, biological products, and medical devices by terminally ill patients,” was advanced into law with a sincere desire to help Pennsylvanians who have few other treatment options. Like our lawmakers, we strongly support access to investigational drugs outside of clinical trials for patients with life-threatening illnesses such as cancer. The FDA provides this opportunity through its expanded-access program known as “compassionate use.”
We have concerns about Act 33, because, unlike the FDA program that has important patient protections in place, the Pennsylvania measure fails to protect patients adequately and does not actually address obstacles that patients face in accessing investigational drugs outside of clinical trials.
When considering a nontraditional treatment program, patients should be aware that RTT laws bypass the government’s responsibility to monitor and protect the safety of patients who seek access to investigational therapy. Furthermore, these laws place no legal obligations on insurers to pay for the routine care costs associated with delivery of investigational treatment — unlike the coverage requirements that exist for patients in clinical trials. And, if complications arise from an investigational treatment, patients may lose their health-insurance coverage altogether.
Patients already have the right to request an experimental therapy from a drug company, but that company is under no obligation to provide it. Notably, RTT laws, including Act 33, do not require drug manufacturers to provide a requested investigational drug and, therefore, fail to address a frequent barrier to patient access. Moreover, these laws often interfere with the already streamlined and effective protocols in the FDA compassionate-use program.
Certainly, there is room to improve how terminally ill patients access investigational treatments, but we don’t believe that such improvement comes by circumventing the existing framework. We support a provision in the federal “21st Century Cures Act” that will simplify access to investigational drugs in the FDA’s expanded access program by requiring drug manufacturers to make information about their expanded access policies and processes readily available to patients and physicians.
A new online Expanded Access Navigator — developed by the Reagan-Udall Foundation in collaboration with patient advocacy groups, the pharmaceutical industry, FDA, and others in the federal government — has been launched as a user-friendly tool to help guide patients and physicians through the expanded access process and identify available treatment options. If no clinical trial or current expanded access program is available or accessible, the Navigator also serves as a clearinghouse for the single-patient expanded access policies from several leading biopharmaceutical companies.
We appreciate the General Assembly’s compassion for patients struggling with terminal illness, as we all share a goal of increasing access to novel and potentially effective treatments. We hope patients and physicians will work together to find the best course of treatment for each patient’s unique circumstances.
Marilyn J. Heine, M.D., is legislative chair of the Pennsylvania Society of Oncology and Hematology. email@example.com
Bruce E. Johnson, M.D., is president of the American Society of Clinical Oncology. Bruce_Johnson@dfci.harvard.edu