In an industry first, Johnson & Johnson ($JNJ) has collaborated with the NYU School of Medicine to create a panel of experts who will evaluate pleas for access to its experimental meds. And it's being chaired by Arthur Caplan, a nationally recognized bioethicist who has long advocated for a better approach in handling compassionate-use requests.
The group of 10--the Compassionate-Use Advisory Committee, or CompAC--will be called in to review compassionate use appeals from all around the world to help guide the company's response. Caplan likened the effort to the system used for distributing organs for people in need of transplants, substituting the often haphazard process now in place in favor of a more rational approach. And he expects that this is one plan that may well be imitated by others in the biopharma world.
"We want to establish a model that will create a structured policy of allocation based on equality, need and efficacy to ensure that the utility of our scarce resources are maximized," he said. "If successful, CompAC will serve as a model for others in industry and in government to follow."
Compassionate use requests are a sensitive issue in the industry. Developers are often leery about allowing individuals access to a drug outside of a controlled clinical trial, worried that an unexpected adverse event could prompt regulators to interfere with their study, often unwilling to bear the cost or unable to share limited supplies.
But with the advent of social media, angry patients and parents who have been stiff armed by drug developers can occasionally trigger a brutal media storm these days. Chimerix was a prime example of that. Their initial refusal to treat a young boy created an outburst on Twitter that was swiftly followed up by 24/7 coverage on cable news shows. Chimerix then scrambled to organize a special, added study and enrolled the child as its first patient.
"It used to be you would call your local news and try to beg them to cover you," Caplan told The New York Times. "Now you build this giant Twitter thing and you make the media come to you."
This is one experiment that at the very least will gain close attention from biopharma players of all sizes.
"Our goal is to ensure that compassionate use of specific investigational medicines still in development is guided by ethical principles, and that the selection process continues to be thorough, transparent and fair," said Caplan in a statement. "Compassionate use decisions are incredibly challenging, and we readily acknowledge that the current allocation system remains a work in progress."
- here's the story from The New York Times