Most people obtain a medication when their doctors write a prescription. That prescribed medication has been studied, evaluated and approved by a national regulatory authority, such as the U.S. Food and Drug Administration (FDA). However, for patients with very serious and/or life-threatening conditions that have already tried all currently available treatment options, an investigational medication – or one that is still being studied in clinical trials – may be their best option.
Clinical trials are the primary pathway for gaining access to investigational medicines, but compassionate use, also known as pre-approval access, may provide an alternative path for people with urgent medical needs who are ineligible for clinical trials and where there are no comparable or satisfactory alternative therapy options available. Doctors can request compassionate use access to investigational medicines on behalf of seriously ill patients.
However, while investigational medicines may show promise for people with serious or life-threatening diseases, evaluating compassionate use requests can be challenging. To help review these requests in the most equitable, fair and thoughtful way possible, Janssen, through the Johnson & Johnson Office of the Chief Medical Officer (OCMO), partnered with the Division of Medical Ethics at the New York University School of Medicine to launch our novel approach to compassionate use in 2015.
Called the Compassionate Use Advisory Committee, or CompAC, this is a novel approach that Johnson & Johnson employs globally to provide a fair, ethical evaluation of compassionate use requests for specific investigational medicines for patients with serious or life-threatening diseases. CompAC utilizes an external group of experts to review compassionate use requests.
CompAC began as a pilot for one investigational medicine for multiple myeloma. More than five years later, it has evolved to serve a portfolio of investigational medicines for serious and life-threatening illnesses, including oncology, infectious diseases and neurological health.
In 2019, the Reagan-Udall Foundation for the FDA recognized the work of CompAC by selecting it as the recipient of its Innovation Award. The award highlights the impact of independent groups working together using a fair and objective process – all on behalf of patients in need.