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

Viral crossroads

Hardy case shows flaws in compassionate use system, provides catalyst for change

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 media and patient empowerment.

Biotech industry leaders are suggesting it may be time to shift some decisions about compassionate access off the shoulders of CEOs and onto new institutions to help adjudicate requests for pre-approval access to medicines.

All of this points to the need for public consensus on a transparent pathway for making decisions about compassionate access (see "Commentary," A9 & "Decision Tree for Compassionate Use," A11).

FDA procedures

The medical, ethical, business and logistical aspects of providing access to investigational drugs can be complex and contentious, but the regulatory pathway is straightforward.

While companies, patients and the media sometimes accuse FDA of preventing compassionate access to an investigational drug, data show the agency virtually never denies requests.

This does not mean patients always get access to unapproved drugs. Requests for access must be submitted by drug sponsors or with their permission. Patients cannot apply directly to FDA, and the agency will not process an application from a physician unless a sponsor is willing to provide access to its product.

That does not mean FDA always plays a passive role. Agency officials sometimes reach out to companies to encourage them to provide access to investigational drugs, according to Richard Klein, director of FDA's Patient Liaison Program.

The agency has initiated expanded access discussions with companies based on requests from patients, media coverage and other triggers.

Biotech executives report they've received calls from FDA relaying requests from members of Congress for family members or constituents to receive access to investigational drugs.

FDA has used the term "compassionate access" in the past, but now it uses "expanded access to investigational drugs for treatment use." According to guidance released in 2013, expanded access is intended "for patients with serious or immediately life-threatening diseases or conditions who lack therapeutic alternatives."

The guidance lists criteria FDA applies to review expanded access applications, including a determination that the "potential benefit justifies the potential risks of the treatment use with the drug and that those risks are not unreasonable in the context of the disease or condition to be treated."

FDA must determine that providing expanded access "will not interfere with development of the drug for the expanded access use, and that the...

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