12:00 AM
Mar 31, 2014
 |  BioCentury  |  Regulation

Josh Hardy chronicles

How Chimerix, FDA grappled with providing compassionate access to Josh Hardy

The Josh Hardy story puts a human face on both the lifesaving potential of compassionate use programs and the wrenching decisions company executives must make about who receives access to compounds in clinical development.

The Hardy family's social media campaign to gain access to an experimental therapy has apparently saved the seven-year-old boy's life.

But along the way, television news programs depicted the situation as a simple case of corporate bad behavior that was corrected by the righteous attention of the media combined with the power of millions of people who became aware of Josh Hardy and joined the campaign to save him on Twitter.

If that were the whole story, there would be no need for new policies. But in fact, the situation was more complex.

Chimerix Inc. had been providing compassionate access to brincidofovir, an antiviral to prevent cytomegalovirus and other viral infections in stem cell transplant patients, prior to beginning pivotal trials. Following dramatic results for some patients, the demand for compassionate access to the compound far outstripped the company's ability to administer an access program.

With demand so large and too few human resources to address it, Chimerix felt it had no way to adjudicate the requests. The company also could not ethically divert all or most of its resources away from the formal development programs that are required to provide access to brincidofovir to thousands of future patients.

In this case, FDA and the company worked together quickly to craft a development program that could allow Josh Hardy and a finite number of other patients to participate in a new study, one which could lead to approval of the molecule in a second indication of adenovirus infection.

The story could just as easily have been about any of thousands of companies working on potentially lifesaving medicines. And Josh Hardy could easily have been any of thousands of patients dying from a disease with no treatment options and no possibility of enrolling...

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