Viewpoint: Working with drug companies to help terminally ill patients gain access to experimental drugs

| | June 13, 2019
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Cassidy Hempel waved at hospital staff as she was being treated for a rare disorder. Her mother Chris, left, fought to gain permission for an experimental drug. Image: AP Photo/Marcio Jose Sanchez
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My colleagues and I at New York University’s Division of Medical Ethics quickly recognized that right to try could not succeed in meeting the hopes or hype vested in it. For doctors to be willing and able to seek access to experimental drugs, they need education about how to find new drugs and how to work with their sponsors. And for the pharmaceutical or biotech companies fielding these requests — who are the primary gatekeepers to these products — convincing them to be willing to grant access to unapproved drugs means addressing their concerns.

We worked with Janssen to form the first of what became several Compassionate Use Advisory Committees (CompACs). These are international advisory groups composed of bioethicists, physicians, and patient advocates who were educated about Janssen’s investigational compounds (under a disclosure agreement with information that was confidential and proprietary), who offer feedback on when and in what contexts specific drugs ought to be made available to whom and provide rapid consultations when emergency cases arise.

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By supporting Janssen, the CompACs have helped give access to hundreds of people during the time that right-to-try laws have provided access to almost no one.

Read full, original post: ‘Right-to-try’ laws provide little access to investigational drugs. We created a process that does

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