J&J forms 'compassionate use' panel for experimental drugs

Drug maker Johnson & Johnson (J&J) announced Thursday that it will establish a new pilot program made up of independent medical ethicists to help evaluate whether individual patients can be given access to the company’s experimental drugs — a debate otherwise known as “compassionate use.”

The new committee, which the company says is the first of its kind, will be made up of bioethicists from the New York University School of Medicine (NYU). They will help J&J decide whether to grant patients what the U.S. Food and Drug Administration calls “expanded access” to experimental drugs — medications that are still under investigation and haven’t been approved by the FDA, but which patients hope can potentially treat their life-threatening illnesses.

Arthur Caplan, a bioethics professor at NYU, will head the panel. The group of bioethicists will merely advise J&J. The company will ultimately make the final decision on whether to grant access to a drug or not. And it will initially work on just one J&J medication, which the company declined to name. The FDA would also have to sign off on allowing access to the drug.

“If we could structure this right, this would be a chance to not just complain about what’s wrong, but maybe to suggest a way forward,” Caplan told The New York Times.

Typically, a drug has to be approved by the FDA’s Center for Drug Evaluation and Research (CDER), a process that involves three rounds of clinical trials to show that it is both safe and effective for use. But after pushback from patients’ rights groups in the last few decades, the FDA has established channels for patients to appeal for the use of drugs that aren't finished with testing.

In 1979, for example, a young woman with terminal head and neck cancer appealed to the FDA for access to a drug called Laetrile that was only approved for colon cancer, but which her doctor believed might help her. Although she died before she was able to enter a clinical trial for the drug, her father took her case all the way to the Supreme Court, which ruled that the terminally ill did not have a constitutional right to an experimental treatment.

However, starting in the 1980s, HIV-infected patients appealed to drug companies for access to experimental new treatments that they hoped could help them. That set a precedent for more formal processes of appealing to the FDA. More recently, doses of the experimental tobacco-based Ebola treatment called ZMapp were given to a handful of infected aid workers, even though it had never been tested in humans. While ZMapp appeared to cure them, its use by those lucky few Ebola patients in wealthy Western countries was debated internationally by bioethicists.

“It’s a really long-standing debate,” said Patricia Zettler, a fellow at Stanford Law School’s Center for Law and the Biosciences who formerly worked as an attorney for the FDA’s Center for Drug Evaluation and Research.

While the FDA approves most individual applications for expanded access that it receives — in 2014, for example, it granted 1,873 out of 1,882 cases (PDF) — private companies don’t release information about whether they approve compassionate use of their drugs. That could mean that patients have come to the FDA only after they are given a green light from the company.

But since the FDA must also approve expanded access, a nod from J&J doesn’t mean a patient will get the drug, according to Zettler.

Companies that are considering approval of a patient’s expanded access face a number of difficult questions, including whether there is enough supply of the drug, or whether compassionate use could delay the drug from being FDA-approved if it ends up giving the patient an adverse reaction.

In recent years, distressed patients have directed their pleas for compassionate use to media and social media, as illustrated by Josh Hardy, an 8-year-old afflicted with cancer whose family gathered so many signatures of support on Change.org that the drug company Chimerix entered him in a clinical trial.

But that introduces worries for bioethicists that the patients who are savviest about compassionate use — or social media — will disproportionately benefit.

“The squeaky wheel gets the grease,” Zettler said. “And that’s not really a fair way to decide who gets access to a limited resource.”

Seven U.S. states have passed so-called “Right to Try” laws based on legislation drafted by the Arizona-based Goldwater Institute, a conservative think tank — measures that would further streamline compassionate use in those states. And 32 more states have proposed laws, according to the National Conference of State Legislatures.

But perhaps the more pressing question for bioethicists is whether an experimental drug might instill false hope during a patient’s final days.

“We have a long history of getting overexcited about products that don’t have very much data,” Zettler said. “Some people, myself included, think ‘compassionate use’ is a bit of a misnomer because it isn’t necessarily compassionate to provide someone with a product that may or may not work.”