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J&J takes external advice on compassionate use

Drugmaker will run a pilot for a review of one late-stage project

Johnson & Johnson headquarters 

Johnson & Johnson (J&J) has become the first big pharma company to take independent advice on making its experimental drugs available for compassionate use.

The drugmaker is running a pilot with the Division of Medical Ethics at the New York University School of Medicine for an external review of just one late-stage project, suggesting it may be expanded to other drugs in its pipeline if successful.

The move comes at a time when it is increasingly recognised that there is an ethical imperative to improve pre approval access to experimental medicines for desperately ill patients, particularly if they are ineligible for enrolment into clinical trials.

"We have a responsibility to patients and their families to ensure their wellbeing in everything we do," said Amrit Ray chief medical officer of J&J's Janssen Pharmaceuticals unit.

"By ensuring compassionate use requests for our investigational medicines are evaluated by a well-informed, external committee, we can better weigh what we know about these therapies against the patient's condition and risk factors to make the most responsible decision for each patient," he added.

At the moment, patients in the US can get early access to pipeline drugs provided they have the support of their physician, the consent of the FDA and a pharma company willing to supply the drug. The US regulator unveiled draft guidance earlier this year on the 'right-to-try' debate in the face of state-level initiatives that threaten to sidestep the current process.

For drugmakers however requests for compassionate use can be something of a minefield, sometimes complicated by supply issues and an incomplete picture on safety and efficacy. 

There are also often unspoken concerns that making drugs available during the development phase could slow clinical development by discouraging trial enrolment and - if the treatment is unsuccessful - throw a development programme off course.

Under the terms of the pilot NYU will create a 10-member Compassionate-Use Advisory Committee (CompAC) consisting of medical experts, bioethicists and patient representatives that will deliver recommendations to Janssen clinicians, who will have the final say.

CompAC chairman Art Caplan, who is director of the medical ethics division at NYU School of Medicine, acknowledged that "compassionate-use decisions are incredibly challenging" and that the current system "remains a work in progress."

"We want to establish a model that will create a structured policy of allocation based on equality, need and efficacy," he added, noting that it could in time serve as a model for wider roll out across the drug industry.

Article by
Phil Taylor

8th May 2015

From: Healthcare



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