J&J Enlists Outside Ethicists to Dispense Early Access to Drugs

Johnson & Johnson will ask an independent panel to recommend who gets a breakthrough drug that’s still under development, a plan that could help the company contend with the phenomenon of desperate patients using social media to campaign for early access to medications.

The collaboration between the world’s largest maker of health-care products and the division of medical ethics at New York University Langone Medical Center will start with one medicine. If successful, the company will expand the initiative, said Chief Medical Officer Joanne Waldstreicher.

The approach involves creating a panel of medical experts, patient advocates and ethicists to establish a clear and fair method to distribute the drugs, said Art Caplan, director of the division of medical ethics at NYU. Medications that are under development are often in short supply and used primarily in clinical trials, making it difficult for patients who don’t qualify for the studies to get treatment.

Johnson & Johnson declined to disclose the name of the first drug that will be involved in the collaboration. The independent group, called the Compassionate-Use Advisory Committee, or CompAC, will make recommendations to J&J’s Janssen drug unit on which patients should receive treatment. The company will retain the final authority over distributing the medicine. The recommendations won’t be made public and the decisions will be conveyed directly to the patients’ doctors.

“There will be none of this, ‘Call the governor, call your rich brother-in-law’ kind of thing,” Caplan said. “I understand why people seek out these drugs, why they go online and do everything they can to help their child or themselves. But that’s not a system. This will level the playing field.”

Picking Patients

Compassionate use -- treating seriously sick patients with unproven drugs -- can involve many patients, such as deciding who got access to experimental drugs for Ebola when the outbreak was ravaging West Africa months ago. It can also impact individuals like Josh Hardy, a 7-year-old kidney cancer patient who was dying from a respiratory virus last year when his family took to social media in a successful campaign to get access to Chimerix Inc.’s brincidofovir.

J&J has several novel drugs that are being closely watched by patients and investors, including daratumumab and Imbruvica for blood cancer and ARN-509 for prostate cancer. Some medications already on the market are being studied for other ailments, including the anti-clotting drug Xarelto, diabetes medicine Invokana, the psoriasis treatment Stelara and the cancer drug Zytiga.

Requests will come in to J&J and be reviewed to see if the patient qualifies for a study or an expanded access program. For those who don’t, the company will send their information to the NYU group for a review, said Amrit Ray, chief medical officer of Janssen.

Fair Process

“This comes at a time when we are seeing innovation in science offering great potential for patients,” Ray said in a telephone interview. “There is a need that the process for requests is fair and transparent. This is very much about doing the right thing.”

The advisory committee may choose a lottery or randomized approach to recommending which patients should get treatment, Caplan said. It could also put a priority on age, other ailments, who is sickest or test results that could indicate which patients are most likely to benefit.

“Everybody can’t get the drug. There just won’t be enough of it,” Caplan said. “We have to let this experienced group have a go at it. I want people to trust the panel and believe they are honest brokers.”