Finding and getting access to clinical trials can be challenging for patients. But for those who don’t qualify for a study, but still want to gain access to the medicine, there are a few newer options: compassionate use and right to try.
“This is a very exciting time for patients to be considering clinical trials,” says Ken Getz, the founder of The Center for Information and Study on Clinical Research Participation (CISCRP), a non-profit dedicated to educating the public and patients. “Clinical research professionals are looking for new ways to engage patients as partners in clinical research.”
Getz, who’s also an associate professor at the Center for the Study of Drug Development at Tufts University School of Medicine, writes extensively about clinical trials. The third edition of his book, “The Gift of Participation,” will soon be released. The book updates patients and participants about trends in and changes to the clinical trial industry. The book also discusses ways that people can gain access to investigational drugs when they don’t qualify for a clinical trial.
Right to try
In 2018, a federal legislative act, The Right to Try Act, granted terminally ill patients in every state the “right to try” experimental treatments if they’ve exhausted all other options.
The patient and the physician petition the pharmaceutical company to provide the investigational drug to the patient. Still it can be a bit more complicated, since individual states and pharmaceutical companies often have additional rules. Patients need to accept risks and, in some cases, they may need pay the cost of the investigational drugs.
“They may lose their insurance coverage, they have to provide their complete consent that they understand what they’re getting into and then the pharma company sends them the study drug,” says Getz, noting that only relatively small numbers of patients have already tried “right to try” medicines.
Another option is compassionate use, also called expanded access, which is for people who are facing a life-threatening, but not necessarily terminally ill, condition. In that case, the doctor has to reach out to the drug company, then fill out a special form asking the FDA to grant permission for the patient to receive the investigational drug.
While a doctor’s request is typically granted, the patient will also need to get approval from the Institutional Review Board (IRB), the ethics oversight committee for clinical trial participant
While there’s promise for patients to be able to try new therapies, there are a few downsides too.
“When you’re dealing with a really debilitating and challenging illness, having to jump through some of these hoops takes some time,” says Getz.
His book also addresses precision medicine and the growing role of biomarkers and genetic data in clinical trials. By sharing their personal genetic information, a patient’s privacy is vulnerable.
“What are the issues you need to know about when you agree to make your biomarker and genetic data available, not only for your own trial, but that might be accessed in the future to support other clinical research?” he says. “How is that data archived and stored and how is your privacy protected?”
Overall, he’s encouraged that so many pharmaceutical companies and doctors are focused on engaging patients in clinical trials.“You learn more about a disease by just listening to patients,” he says, encouraging patients to enroll in trials, be active partners in the process, and provide input to help researchers design the studies.
For more information visit ciscrp.org.