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 who have exhausted all currently available treatment options and are not eligible to participate in a clinical trial, compassionate use may be an option.
While clinical trials are the primary pathway for gaining access to investigational medicines, compassionate use may provide an alternative path for people with urgent medical needs where there are no comparable or satisfactory alternative therapy options available.
Treating physicians may request compassionate use access to investigational medicines from companies on behalf of seriously ill patients. Evaluating these requests can be challenging.
An innovative mechanism for evaluating compassionate use requests
In 2015, Janssen, through the Johnson & Johnson Office of the Chief Medical Officer (OCMO), collaborated with the Division of Medical Ethics at the New York University School of Medicine to launch our novel approach to compassionate use in 2015. It is called the Compassionate Use Advisory Committee, or CompAC.
CompAC is a novel approach that Johnson & Johnson employs globally to provide an equitable, fair and 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, including physicians, ethicists and patient representatives selected independently by the NYU School of Medicine.
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.
