Now that the family of seven-year-old Josh Hardy has successfully obtained access to an experimental drug, aided by an explosion of social media support, lawmakers, industry and patients still are left grappling with the fundamental inequities and flaws of the U.S. system for granting compassionate access to investigational therapies.
The problems have existed for years, including the idiosyncrasies of who gets access to investigational drugs outside of clinical trials and the criteria companies use to make decisions that are often life-and-death.
The fact that individual companies have been left to make these decisions on an ad hoc basis inevitably fuels suspicions among patients, family members and the public about the motives for denying access.
The difference in perspectives between companies focused on gaining regulatory approval and individuals trying to save desperately ill loved ones can make mutual suspicion curdle into antagonism.
The rise of social media as an advocacy tool now raises the prospect that medical and regulatory decisions will be tipped by a public outcry.
As the Hardy case illustrates, patients or their relatives can generate hundreds of thousands of supporters virtually overnight, along with a wave of attention on television. In the heat of a media feeding frenzy, it is impossible for a CEO to communicate the complexities of drug development and why the integrity of the regulatory approval pathway must be protected to get a drug to as many patients as possible.
And a CEO who says "no" is no match for 100,000 angry Twitter messages and death threats from angry individuals who jump on the bandwagon. The threat of demonization has led companies to be wary of individuals and groups seeking to obtain compassionate use access to their products.
While the Hardy case reflects old problems, it also may be a game-changer. The almost immediate and apparently extraordinary effectiveness of Chimerix Inc.'s brincidofovir for Josh Hardy, coupled with the power of social media to push the company to find a way to provide access, have already led other families to pursue similar campaigns (see "The Josh Hardy Chronicles," A7).
The Hardy case also has prompted members of Congress, industry trade associations, patient advocacy groups and individual companies to reassess the need for new principles to guide compassionate access decision-making in the era of social