Compassionate use, also known as expanded access, may bring an investigational drug or device to patients with life-threatening conditions. These treatments are not yet FDA approved and little may be known about their safety and effectiveness. They may or may not help improve a patient’s condition, but these experimental treatments are used as a last resort. The FDA may grant expanded access for individuals, groups of patients who do not qualify for any clinical trial, or large groups once clinical trials for the treatment have provided sufficient information about the drug or device. In order to gain access to these treatments, patients and their families must first explore potential treatment options and search for possible clinical trials. If nothing is available then the patient’s physician must submit an application. The FDA and an IRB will then review and approve or deny the request.
Five states, including Arizona, Colorado, Louisiana, Michigan and Missouri, have approved right-to-try laws this year. The laws intend to speed up the process so patients can receive these prospective treatments quickly. The drug or device must have passed phase I in order for a patient to have access. Although these laws aim to bypass the relatively lengthy FDA expanded use approval process, the drug or device company must also agree to provide their investigational product. Interrupting the clinical trial process to provide an individual with the treatment may delay the availability of the treatment to others. A patient who suffers a serious adverse event after using the investigational treatment may also hold up the research and FDA approval process.
Companies face a tough choice when deciding whether to allow compassionate use, and face public scrutiny if they say no. Earlier this year Chimerix, a small biotech company, received death threats after denying a request to provide their experimental drug to an 8-year-old boy. Josh Hardy was suffering from cancer when his family asked to use Chimerix’s anti-viral drug brincidofovir to treat a life-threatening adenovirus infection. Although brincidofovir was currently in phase III trials, Chimerix worked with the FDA to implement a new study that would include Josh.
In October of this year Brincidofovir received emergency FDA approval to treat patients with Ebola.
Josh Hardy Going Home After Getting Chimerix Anti-Viral Drug
States Allowing Terminal Patients Access to Experimental Drugs
More States Adopt Laws To Ease Access To Experimental Treatments
