The New York Times reports “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 Times continues:
The move, to be announced by the company … 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.
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.
This decision may be more complex that it appears. Here’s a commentary on the topic by my dear friend, David Stevens, MD, MA (Ethics), who serves as the CEO of the Christian Medical Association:
Often companies hire bioethicists to provide justification for what they are already planning to do as we saw frequently during the ‘stem cell wars.’
In this case, it is more likely that Johnson & Johnson created its panel for public relations purposes since the issues involved in providing desperate patients investigational drugs have little to do with ethics and much more to do with practical and legal issues.
The FDA has guidelines for expanded access for individuals, “intermediate-size patient populations” (tens to hundreds to get a drug early in its development) and “widespread use” (a treatment protocol for larger groups after a successful trial but before FDA approval).
These guidelines are reasonable but impose burdens on patients, physicians, pharmaceutical companies and the FDA. Rarely are requests that meet the guidelines denied.
Guidelines require that the disease or the condition is serious and life threatening, that there is no other satisfactory therapy option, that the benefit expected justifies the potential risk, that the patient can’t obtain the drug in a clinical trial and that providing the drug will not hamper the initiation, conduct or completion of a clinical investigation.
These guidelines have put the “hot potato” squarely in the laps of drug companies. The heat on them has been turned up through emotional pleas by families and advocacy groups that have gone viral on Twitter, Facebook and other social media channels.
It is not that all pharmaceutical manufacturers are hard-hearted. They assume a substantial administrative burden to create protocols and provide oversight.
That is especially onerous for small companies. They may not yet have enough manufacturing capacity to meet extra demand and that could impact their ongoing clinical trials.
They are able to charge direct costs to individual patients but rarely do so since it reveals proprietary information that can lead to bad publicity when the drug goes on the market after approval at a much higher cost.
These and other issues revealed in an excellent article in the New England Journal of Medicine in January will better help you understand this complex issue.
I hope this panel will do more than ensure better PR (It’s not our fault, the independent panel made the decision!), but also lead to safer, better and reasonable use of new drugs to desperate patients during their development.