BUSINESS DAY

Company Creates Bioethics Panel on Trial Drugs

By 
Arthur L. CaplanRichard Perry/The New York Times 
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.
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.
The family gathered 500,000 signatures on an online petition, thousands of followers on Facebook and even the support of celebrities like the British comedian Ricky Gervais.
But their efforts were not successful and Mr. Auden died in November 2013. ”It was horrendous to fight the cancer, as well as to fight for compassionate use,” said Amy Auden, Mr. Auden’s wife. Ms. Auden said she was aware of the complex ethical issues in approving such requests, but added: “I think each of them can be addressed if companies were to turn their mind to it, and address it as part of their business model. But I know they don’t want to play God.”
She applauded Johnson & Johnson’s move and said she hoped other companies would follow suit.
Four months after Mr. Auden’s death, Merck approved an expanded-access program for patients who could demonstrate a critical need for its drug, now called Keytruda, and it was approved by the F.D.A. last September and hailed as a breakthrough for melanoma patients.
Merck said it had expanded access to the drug only after the company could provide adequate supply to patients — before that, it said, all supplies were allocated to the clinical trials then underway. Merck said it provided early access to Keytruda to more than 3,800 patients worldwide. Its policy for approving any requests for unapproved drugs is based on several factors, including the severity of a patient’s condition, whether the treatment is likely to help and whether the patient has exhausted all other options.
“Merck is dedicated to making our medicines available to all patients who may benefit from them as soon as possible,” a spokeswoman, Pamela Eisele, said in a statement.
Officials at Johnson & Johnson said that they had not kept track — until now — of how many compassionate use requests the company received, but that it probably fielded at least 100 requests a year for unapproved drugs to treat everything from cancer to infectious diseases.
Dr. Joanne Waldstreicher, the chief medical officer at Johnson & Johnson, said patients were responding to the fact that her company — and others — were developing breakthrough drugs with a real potential to save lives.
New cancer drugs like Keytruda, for example, are harnessing the body’s immune system to attack cancer cells and yielding encouraging results. Johnson & Johnson’s experimental treatment for multiple myeloma, daratumumab, is also generating excitement. It is currently in late-stage clinical trials.
“This comes at a time when innovation in science offers great potential for patients, great promise,” Dr. Waldstreicher said. “We believe there is a continued need for processes that are both fair and objective and, most important, patient-centered.”
The company has not yet said which treatments will be included in the program, but Dr. Caplan said the list would include no more than two or three drugs initially, although it could be expanded if it is deemed a success. Patients would receive the treatment free if their request was approved.
Drug companies often waive payment for drugs in such cases because they are permitted to charge only manufacturing and other direct costs of the product. Because the market prices of drugs are often many times higher, some companies pay for a drug rather than make its cost public, Dr. Caplan and others have said.
Even if drug costs are covered, patients could be responsible for other expenses, such as the cost for a doctor to administer the drug or provide other care related to the treatment. Insurance companies do not always cover those costs for experimental drugs, said Mark Fleury, a policy expert at the American Cancer Society Cancer Action Network.
A version of this article appears in print on May 7, 2015, on page A1 of the New York edition with the headline: Company Creates Panel for Access to Trial Drugs. Order Reprints|  Today's Paper|Subscribe