J&J Changes ‘Compassionate’ Care

Originally posted in the Wall Street Journal by Jonathan D. Rockoff 

Under increasing pressure to expand access to experimental medicines, Johnson & Johnson has arranged for an independent panel to review requests from seriously ill patients who want to try an unapproved drug even if they aren’t participating in the drug’s testing.

The committee of doctors, bioethicists and patient representatives organized by the New York University School of Medicine will consider the hundreds of requests that J&J receives each year from patients who believe an experimental drug can help them. The panel will recommend a course of action to J&J.

Until now, the decisions have been largely left in the hands of individual physicians around the company working on particular drugs. “This really assures each request is handled in an objective and thoughtful manner,” said J&J Chief Medical Officer Joanne Waldstreicher.

The issue of early access has drawn increasing public attention while raising thorny ethical questions for regulators and the drug industry. Companies say they typically make only enough of an experimental drug to supply the clinical trials, and they fear lawsuits or a backlash against participating in their studies if a patient given access to an unapproved drug fails or suffers serious side effects.

But supporters of early access say it could make the difference between life and death for patients whose life-threatening illnesses might not give them time to wait for testing and approvals to run their course.

Under federal law, companies generally can’t give out unapproved drugs. However, the Food and Drug Administration allows companies to give medicines for “compassionate use” in patients with serious and life-threatening illnesses who aren’t part of one of the clinical trials. In recent years, increasing numbers of patients have been asking regulators and companies for such early access—up to 1,809 requests last fiscal year, according to FDA. Meantime, the agency and drug companies have been accused of resisting requests.

The FDA has allowed more than 99% of the compassionate-use requests it received during the past several years, sometimes granting permission over the telephone to move the requests along quickly, an agency spokeswoman said.

But the ultimate decision of whether to supply a drug, even with regulators’ approval, rests with the companies.

A biotech called Chimerix Inc. last year drew heavy criticism in the press and social media over the case of a 7-year-old whose family wanted access to its experimental antiviral drug. The company ultimately agreed to make the child part of a late-stage study. A Chimerix spokesman said the company doesn’t comment on individual patients.

Sixteen states now have so-called “Right to Try” laws that aim to reduce regulators’ role in the decision on whether terminally ill patients get unapproved drugs, and bills in two more states are awaiting governors’ signatures, according to the Goldwater Institute, which advocates for the legislation.

Goldwater Institute CEO Darcy Olsen doubted an independent review would make it easier and faster for seriously ill patients to get medicines in a timely manner. “What I think you have is J&J passing the buck on tough decision-making,” she said.

The institute advocates for conservative causes, and has been pushing to give patients access to experimental medicines without government permission.

Amrit Ray, chief medical officer of J&J’s Janssen pharmaceuticals unit, said the setup should be easier for patients because the company is establishing a single website and toll-free hotline for requests, rather than the current patchwork of entry points across the company and its drug trials. Dr. Ray expects the committee could issue recommendations within days for urgent requests.

In its arrangement with NYU, J&J physicians will first see if a patient could be the subject of a clinical trial or some other early-access program. If not, J&J will forward the request to the independent panel for review. J&J said it is possible the company could decide differently, but it expects to take the committee’s advice. The patient would still have to get compassionate use clearance from FDA.

Arthur Caplan, the head of NYU School of Medicine’s division of medical ethics, who will oversee the new committee, said the panel would start slowly, focusing on a single, still-undetermined experimental drug and taking a few months to figure out criteria for making decisions. He said the goal was making sure each request is reviewed on its merits, rather than influenced by media campaigns or personal connections.

“Most people will have a fair shot by having a process that is really going to be hard to lobby and manipulate,” he said.

The Compassionate-Use Advisory Committee will report to NYU School of Medicine, but J&J will compensate the committee’s nine to 12 members and subsidize expenses such as travel and telephone fees, according to Dr. Caplan, who will serve as a nonvoting member and not receive a salary himself. J&J doesn’t charge patients for any unapproved drugs given for compassionate use.