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Being More Compassionate about Compassionate Use Drugs

Johnson & Johnson has created an independent review board to evaluate patient requests for new drugs that have not yet been approved by regulators. 

In creating the 10-person board, J&J, and its pharmaceutical arm Janssen Pharmaceutical, seeks to tackle an ethically difficult and emotionally charged issue that drug manufacturers are being forced to face at an increasing rate, according to The Wall Street Journal, which says that the US Food and Drug Administration received 1,809 requests from patients in Fiscal Year 2014 for so-called "compassionate use" drugs. 

The new panel is led by Arthur Caplan, a professor of medical ethics at New York University's Langone Medical Center, who will not be paid for his work for the panel, The New York Times says, adding J&J's lead could spur other pharmaceutical firms to establish similar panels.

The concept of compassionate use applies to drugs that have not yet been approved for use by the general public, but which a drug company is making available to patients who may otherwise die without access to the drugs. Drug firms have struggled to decide when to grant requests for such drugs — in addition to ethical concerns, there may be a shortage of supply of the drugs. They also worry that providing a drug for compassionate use may jeopardize clinical trials. 

While the FDA has typically signed off on patient requests for compassionate use drugs, the decision whether to grant such requests essentially rests with the drug firms, and according to NYT, they have been inconsistent in approaching the matter. 

Meantime, patients have gone to social media to pressure the companies to provide the drugs. While such efforts are not always successful, last year the family of Josh Hardy was able to get the 7-year-old boy access to an investigational antiviral drug made by Chimerix. 

Hardy had a life-threatening infection, and after the company initially rejected the family's request for the drug, they went on Facebook and Twitter and asked followers to contact Chimerix. They did and overwhelmed Chimerix with emails, phone calls, and Twitter messages. The company subsequently enrolled Hardy in a 20-patient clinical trial for the treatment, NYT says. 

Some states have also gotten into the act, and more than a dozen have passed "Right to Try" laws that seek to reduce FDA's role in the approval process while a handful of other states are considering similar legislation. 

In its announcement, J&J says, "Compassionate use decisions are incredibly challenging, and we readily acknowledge that the current allocation system remains a work in progress. This new initiative hopes to create a more robust model to help guide these decisions." 

The conservative-leaning Goldwater Institute CEO Darcy Olsen, however, is skeptical that such panels would deliver compassionate use drugs more quickly to patients. "What I think you have is J&J passing the buck on tough decision making," she tells WSJ.