J&J, NYU Partner in New Compassionate Use Pact
Johnson & Johnson has formed a new partnership that enlists a third party to review requests made to the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen) for compassionate use of its investigational medicines. This new approach will begin as a pilot program focused on a single Janssen investigational medicine and, if successful, will become a model that will be applicable more broadly across Johnson & Johnson.
Janssen Research & Development, LLC has forged this new partnership with the Division of Medical Ethics at the NYU School of Medicine to obtain independent advice, “further ensuring that the evaluation of requests for investigational medicines prior to their approval by the US Food and Drug Administration (FDA) or other global health authorities are treated in the most fair and ethical manner,” said the company in making the announcement of the partnership.
As part of this collaboration, the NYU School of Medicine will establish the Compassionate-Use Advisory Committee (CompAC), an external group of 10 internationally recognized medical experts, bioethicists, and patient representatives to pilot the new approach. Commencing with one of Janssen's investigational medicines, the CompAC will make recommendations regarding individual patient requests from anywhere in the world. Investigational medicines are still in development and usually limited in supply, and the committee's careful consideration and recommendations based on its independent review will inform Janssen clinicians, who will make the final decision.
The FDA describes compassionate use as “a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions. Such investigational drugs have not yet been approved by the FDA, and they have not been proven to be safe and effective.”
For compassionate-use requests, Janssen has a long-established single point of contact: all interested treating physicians can contact Janssen Medical Information. Physicians outside the US may contact the Janssen office in their country. Physicians are asked to provide information about the patient that is used to evaluate the request.
If permitted by local authorities, Janssen typically establishes an expanded access program (EAP) for investigational medicines when a favorable benefit-risk profile has been reported in a pivotal trial and there is a viable regulatory path to approval confirmed with a health authority. The EAP can potentially allow the treatment to be made available to patients living with the same serious or life-threatening condition for which the medicine was originally studied and who have exhausted other treatment options. If patients do not qualify for an EAP or an existing clinical trial, their requests will be forwarded to CompAC.
Janssen has a long-standing commitment to providing expanded access to its investigational medicines prior to approval where it is appropriate to support the needs of patients with serious illnesses. This new collaboration extends existing approaches by adding an external evaluation to the process.
Source: Johnson & Johnson