Thursday, January 15, 2015

Social Media "Compassionate Use" Crusade Sets Unsettling Precedent

Former Chimerix CEO Ken Moch Warns Drug Makers to Prep for “Compassionate Use” Campaigns

By Marc Dresner, Senior Editor, IIR

Back in March the drug industry got a jarring demonstration of social media’s firepower when a grassroots campaign to get a seven-year-old cancer patient pre-approval access to an experimental anti-viral medicine captured national attention and made headlines worldwide.

The #SaveJosh campaign—named for patient Josh Hardy, now eight—generated sufficient pressure to force Durham, NC-based Chimerix, a small biotech, to create a 20-patient open-label study for brincidofovir (CMX001)—in Phase 3 trials—in order to accommodate this very sick little boy.
Josh Hardy

Without question, the drug saved Josh Hardy’s life and the good news is that his condition has dramatically improved.

But the case raised some very troubling ethical questions around “compassionate use” in an age when “who shall live” decisions can in effect be crowdsourced via social media.

It also introduced a new dilemma for drug manufacturers in that the advent of compassionate use crusades almost overnight and on the massive scale facilitated by social media can potentially impede development efforts.

Kenneth Moch
According to former Chimerix CEO Kenneth Moch—who received death threats and had to be placed under security detail during the emotionally-charged ordeal—had the outcome been different and Josh Hardy’s response less favorable, development of brincidofovir could’ve been delayed or even derailed, consequently preventing the larger prospective patient population from accessing a potentially life-saving drug.

“This was not just about Josh Hardy; it was about the many ‘Joshes.’” - Ken Moch


“This was not just about Josh Hardy; it was about the many ‘Joshes,’” said Moch in an interview with Inside Clinical.

“The issue is how to balance providing immediate access to a patient in dire need against the goal of making a drug available to as many patients as possible by getting it approved,” Moch emphasized.

For smaller biotechs like Chimerix—with just 50 employees and no other product in market—the financial strain and demands placed on personnel by compassionate use interventions can compound the problem by siphoning scarce resources that would’ve otherwise been dedicated to moving a drug through the development and approval process.


“This was the first time that social media had been used so intensely around a child who looked and was so needy.”

“This was the first time that social media had been used so intensely around a child who looked and who was so needy,” Moch noted.

“The decision for making a drug available pre-approval currently rests with the company developing the drug,” he added. “And smaller companies—particularly smaller biotech companies—may not have the ability to respond to these significant social media pressures.”

It’s worth pointing out that the mainstream media—Fox News, in particular—depicted Chimerix as a Big Pharma Goliath with deep pockets, an image that served to incite public outrage and ratchet up the pressure.

Helping a gravely ill child get access to a life-saving drug that had been denied him by a heartless drug giant is a compelling and dangerously irresponsible narrative to spin, and the emotions it elicited at the time appear to have made a reasoned dialogue about the complexities involved in making compassionate use decisions impossible.

According to Moch, Josh Hardy was one of hundreds of patients who had not been eligible to participate in the brincidofovir SUPPRESS trial and who had petitioned Chimerix for pre-approval access but had been denied the medication for reasons related to the company’s Phase 3 development efforts.

Social and mainstream media pressure enabled Josh Hardy to get the drug and to survive, but the event set a troubling precedent by relegating the decision to the whims of public opinion and inequitable influence.

How do you decide who can and cannot have access to an experimental drug? Contacts? Money? Social media?

“How do you decide who can and cannot have access to an experimental drug? Is it through social media? Is it through contacts? Is it through money? Is that fair and equitable? I think not,” said Moch.

The Josh Hardy case has forced the industry and regulators to scrutinize expanded access to experimental drugs. BIO and PhRMA have since responded and the FDA has formed a task force.
But Moch emphasized that companies must be prepared to address social media campaigns for pre-approval access to drugs they’re developing head on or face potentially disastrous consequences to their development efforts.
In this podcast for Inside Clinical—the official interview series of the 24th Annual Partnerships in Clinical Trials conference—Ken Moch discusses the issues around “compassionate use” and what manufacturers can learn from his experience.
Listen to the podcast and download a transcript here!

Author's note: Ken Moch co-authored a great post on this subject with NYU's Art Caplan on the Health Affairs blog. Check it out here.

Editor’s note: Ken Moch will present “Fast Tracking Clinical Development in a Moment’s Notice: Patient Advocacy, Social Media and the Implications of Compassionate Use” at the 24th Annual Partnerships in Clinical Trials Conference running April 22-24 in Boston.

For more information or to register, please visit www.clinicaltrialpartnerships.com  

ABOUT THE AUTHOR/INTERVIEWER
Marc Dresner is IIR USA's senior editor and special communication projects lead. He is the former executive editor of Pharma Market Research Report, a confidential newsletter for marketing researchers in the pharmaceutical industry. He may be reached at mdresner@iirusa.com. Follow him @mdrezz.




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