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May
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Company Creates Bioethics Panel on Trial Drugs – Courtesy (The New York Times)
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Johnson & Johnson has appointed a nationally known bioethicist to create a panel that will make decisions about patients’ requests for potentially lifesaving medicine, responding to an emotional debate over whether companies should allow desperately ill people to have access to the drugs before they are approved.

The move, to be announced by the company on Thursday, is believed to be the first of its kind in the industry and, given the size and influence of the drug maker, could inspire other companies to follow suit. It comes as a small but growing number of patients with terminal illnesses have sought the right to obtain drugs still in the testing phase that show promise for treating their diseases.

Some of the requests have become highly publicized cases on social media, where family members and advocates have lobbied the companies on patients’ behalf — often to no avail because drug makers fear that doing so would interfere with clinical trials, or, in the case of the Ebola outbreak, that they have too little available. The issue, which involves fundamental questions of fairness and equal access to care, has become so intense that more than a dozen states have taken up legislation to speed the process.

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Nick Auden and his family failed to persuade drug makers to give him access to cancer treatments in 2013. He died that year.

Johnson & Johnson said the bioethicist, Arthur L. Caplan of New York University, who has written extensively about the issue of experimental drug availability — known as “compassionate use” — would oversee an independent panel of doctors, ethicists and patient advocates that will review requests for access to a limited array of experimental medicines and decide how Johnson & Johnson should respond.

The pilot program will be funded by the company, which will have no influence on the panel’s decisions, Johnson & Johnson said, adding that payments will go directly to the university. Dr. Caplan will not be paid for his work in the program.

Dr. Caplan, who has argued that the industry needs a fairer, more consistent system for deciding whose requests should be granted, said he was intrigued when company executives approached him about the idea. “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,” he said in an interview.

Drug companies have been granting emergency access to their unapproved drugs since the AIDS epidemic of the 1980s, when the Food and Drug Administration set up a process to help desperate patients get experimental treatments. The F.D.A. typically signs off on use of unapproved drugs, but not until the company agrees.

But saying yes is not so simple: Manufacturers often have a limited supply of such treatments, leading to anguished decisions over who should be given the products. The unproved drugs also might not work, or could even cause harm. And the time and resources involved in granting access to such drugs could delay efforts to get them approved for a much wider population of needy patients, especially at smaller companies. In the case of the Ebola epidemic, last year the F.D.A. allowed the makers of ZMapp, an experimental treatment, to be used on a handful of patients, but the company quickly exhausted its limited supply.

“There’s no stock answer — yes or no, black or white,” said Richard Klein, director of the F.D.A.’s patient liaison program. “Maybe you can take that responsibility off the company.”

There is no reliable data on how many requests pharmaceutical companies receive, but the F.D.A. grants nearly all of them. In the 2014 fiscal year, the agency approved 1,873 requests from companies to grant what it calls “expanded access,” an 85 percent increase over the 2010 fiscal year, when it approved 1,014 requests.

Some patients and their families have started campaigns on social media sites like Twitter and Facebook to try to shame companies into granting their request. In 2014, the family of Josh Hardy, a 7-year-old boy suffering from a life-threatening infection, appealed to followers on Facebook and Twitter after Chimerix, a company that was developing an experimental antiviral treatment, turned down their request.

Within days, supporters bombarded the company and its top executives with thousands of calls, emails and Twitter messages pleading for relief.

Chimerix quickly announced that it had set up a 20-patient clinical trial that Josh and similar patients would be able to enroll in. Josh responded to the treatment and is said to be doing well.

“It used to be you would call your local news and try to beg them to cover you,” Dr. Caplan said. “Now you build this giant Twitter thing and you make the media come to you.”

But that has only created a new kind of inequality. “The social media side is now driving attention, but it only does for those who know how to use it,” he said.

The problem, some say, is that drug makers have inconsistent approaches.

“Too often with these sorts of scenarios, it’s handled in different ways in different companies, and even within the same company, different people may have different results,” said Dr. Aaron S. Kesselheim, an associate professor of medicine at Harvard Medical School and Brigham and Women’s Hospital who recently wrote about compassionate use in The New England Journal of Medicine. Johnson & Johnson’s program, while limited, “might be a model for how we could move forward,” he said.

Several states have recently passed so-called Right to Try laws, which seek to sidestep F.D.A. approval of the process. But critics of the bills, which are also pending in other states, point out that the major roadblock is not the F.D.A. but the companies themselves, which still must grant permission.

Not all social media campaigns have succeeded. The family of Nick Auden, a father of three with advanced skin cancer, went public in 2013 seeking to persuade Merck and Bristol-Myers Squibb to reverse their decisions denying Mr. Auden access to then-experimental cancer treatments that were in late-stage clinical trials. Mr. Auden had been accepted into a trial for Merck’s drug, but was later disqualified after being hospitalized for a partial bowel obstruction.



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