Patients with serious illnesses and their families who have run out of treatment options and do not qualify for clinical trials need to know that their requests for compassionate access to investigational medicines have been treated in as thoughtful and objective manner as possible. We understand the importance of making medicines available to people in urgent need, and have worked in the past to try to grant pre-approval access to medicines when appropriate for patients with no other treatment options. These decisions present some of the most difficult challenges for many reasons, including the limited information on safety and efficacy we often have early in development, and since supplies of investigational medicines are often limited while they are still being developed. With such important decisions, we believe that there is a continued need to ensure that we are as objective and as thoughtful as possible. As physicians, it is inspiring to see innovation in science offering such great potential for patients.
As an extension of Johnson & Johnson’s (J&J) long-standing commitment to the highest standards of scientific and ethical decision-making, we are announcing a first of its kind partnership between J&J and the New York University School of Medicine to obtain independent review of individual compassionate use requests for access to investigational medicines. This new approach brings independent advice to further ensure that individual patient requests are evaluated in the most objective, fair and ethical manner. We will begin with a pilot focused on a single Janssen investigational medicine and, if successful, envision extending the new approach more broadly across our investigational products in the future.
As part of this collaboration, an independent Compassionate Use Advisory Committee, or “CompAC,” has been developed by the NYU School of Medicine and is composed of internationally recognized ethicists, physicians and patient representatives. The CompAC is chaired by Art Caplan, Ph.D., Director, Division of Medical Ethics at the NYU School of Medicine and the Drs. William F. and Virginia Connolly Mitty Professor of Bioethics. J&J has partnered with NYU School of Medicine because of its long-standing leadership in the field of medical ethics and especially its work in the area of compassionate access.
J&J remains committed to supporting clinical trials as the most important mechanism to generate the scientific evidence needed to obtain health authority approvals and thereby help the greatest number of patients. Moreover, while we await health authority approvals, we will continue our long-standing commitment to offer pre-approval access to investigational medicines through expanded access programs (EAPs), which typically provide access for patients who have the same disease that was studied in pivotal clinical research, and for which we have full safety and efficacy information.
For individuals who do not qualify for either a clinical trial or EAPs, the CompAC will independently review requests by treating physicians on behalf of their patients. The CompAC will make independent recommendations to Janssen, taking all important factors into account, including available information on the patient, the product and available drug supply. Janssen’s physicians will make the final decisions on providing access.
The concept of using an independent, external committee to assess requests for access to our investigational medicines was modeled after our successful collaboration with the Yale School of Medicine Open Data Access Project (YODA) to obtain independent assessment of requests for access to our clinical trial data. In both situations, the external input allows for an independent, objective assessment so that every request is treated in the most thoughtful, fair and ethical manner. Our commitment is to keep patients and consumers at the heart of all we do.
To learn more about our collaboration with NYU School of Medicine, please http://www.janssen.com/compassionate-use-pre-approval-access.