Pharma firm pioneers bioethics model to get experimental drug to myeloma patients

In early 2015, executives at Janssen Pharmaceuticals wanted help with one of the thorniest dilemmas in drug development: deciding which desperately ill patients would get the short supply of an experimental drug that hadn’t yet been approved by federal regulators.

The drug in question was Janssen’s daratumumab – dara for short – which a journal editorial would later call “a landmark advance in the treatment of multiple myeloma,” an incurable bone marrow cancer that causes pain and fractures.

Janssen executives enlisted New York University Langone Medical Center bioethicist Arthur Caplan, a leading voice on obstacles to “compassionate access” of experimental drugs.

“At first, I thought they [at Janssen] were just trying to outsource a problem, but legally they can’t do that,” recalled Caplan, who spent decades on the University of Pennsylvania faculty. “I’ve come to believe they really wanted to do something to help.”