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."

Caplan set up an independent board, the Compassionate Use Advisory Committee, or CompAC, to review requests for dara from physicians and make recommendations to Janssen. While not a panacea for compassionate-access challenges, the panel of 10 doctors, ethicists, and patient representatives — many, including Caplan, unpaid — enabled hundreds of terminally ill patients around the world to get dara through a fair, fast mechanism.

The first step was to let doctors and patients know about it. Fewer than 20 percent of drugmakers put their compassionate-use policies on their websites, according to a recent market report. Janssen not only explained  — in dozens of languages — how the CompAC works; it also created a YouTube video, a phone line, and assigned round-the-clock staff to screen physician requests as they came in by email.

To avoid possible bias, the CompAC directed Janssen to remove each patient's name, location, race, nationality, and even gender. During conference calls, committee members primarily considered whether patients were too sick to benefit from the drug ­— for example, if they were suffering organ failure.

"We realized the drug takes two weeks to work, so we ruled out people who wouldn't live that long," Caplan said.

So far, the CompAC has published data for patients in the U.S., where the product, Darzalex, received approval in November 2015. Of 76 compassionate-use requests it reviewed, the committee recommended 60, giving decisions within five days. Janssen approved those, plus two more from physicians who went through an appeals process built into the program.

Although the Food and Drug Administration allows companies to charge for compassionate-access drugs, Janssen covered the costs, including shipping.

Ceri Bygrave, a myeloma specialist at University Hospital of Wales in Cardiff, found the process "quite simple." Her patient, in his early 70s, was out of treatment options.

"We knew if we didn't treat him, the disease would take off," she said last month. "He had about six months of stable disease [on dara]. Now, he's on a different drug through a different compassionate-use program. It bridged him to a new drug. He might not be here otherwise. He feels fortunate, for sure."

Janssen, part of Johnson & Johnson, is now expanding the program to other drugs in development.

"We concluded this offered great potential to patients and made the important decision to expand it across Janssen's portfolio," said Amrit Ray, Janssen's chief medical officer.

Caplan would like to see the experiment become a national model. "Little companies are still at a disadvantage," he conceded, "but maybe we could work through a consortium."