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Did Chimerix, Inc. (CMRX) Set A Bad Example For Biopharma?

3/25/2014 1:43:53 PM

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March 26, 2014

Ethics don't always feel good.

By Karl Thiel for

Chimerix generated some pretty negative publicity when it denied a dying seven-year old boy access to its experimental drug brincidofovir. Josh Hardy had developed an adenovirus infection following a bone marrow transplant related to past cancer treatment. Since Chimerix has a drug that has demonstrated some efficacy (albeit much of it anecdotal) in exactly this indication, doctors recommended the parents see about getting the medicine on a "compassionate use" basis. The company, however, demurred.

A "Save Josh" campaign waged on Facebook and across Twitter caught up thousands of followers and led to over 20,000 signatures on an online petition—even Washington Redskins quarterback Robert Griffin III got involved, retweeting the #savejosh message to his followers.

And finally the company relented, saying it would supply the medicine. While that will hopefully turn out to be a victory for Josh and his family, it's not actually great news for anyone else, and raises a number of thorny ethical issues.

There is, of course, the simple issue of how drugs are approved. Adenovirus infection is fairly common but usually not serious—unless the patient is immunocompromised. In such cases, there is no existing treatment, so any new therapy must of course establish its efficacy versus placebo. That means there are patients who get the treatment and those who don't. Getting that placebo-controlled data is vital so that future patients—not just present ones—can get the drug. Or, if it turns out not to work, so that future patients don't get a pointless therapy that may expose them to further risk. While it seems easy enough to give a sick boy some medicine that might help, it undermines everything that went into creating that medicine in the first place.

Yes, this seems obvious, yet it's anything but for much of the media. Over on Fox News, an article derided a "snarky" comment from a Chimerix board member over the eventual decision to launch a new clinical study with Josh as the first patient. It went on to say that brincidofovir (which is a prodrug of the AIDS drug cidofovir, aka Gilead's Vistide) "has been proven to clear up adenovirus in children within two weeks." No, that's not been proven. That's exactly wrong, and the thinking behind the mistake perfectly exemplifies why this kind of medicine-by-community-action is a problem.

A similar situation happened just a few months ago with BioMarin Pharmaceuticals and its decision to deny Andrea Sloan, a woman with advanced ovarian cancer, access to BMN-673. It also came up a few years ago when a drug called PLX4032 was in late development for malignant melanoma. A September 2010 article in the New York Times told the heartbreaking tale of two cousins with almost identical cancers enrolled in a clinical trial--one of whom wound up in the control arm (receiving a notoriously ineffective chemotherapy cocktail) and eventually dying, while the other got the drug--now approved as Zelboraf--and saw a miraculous improvement.

It was an anguishing situation, but one in which there was only a limited window in which to discover whether the drug, which plainly shrank tumors, actually helped patients survive. Opening the flood gates to compassionate use would have meant no more controls, and no chance of ever getting that comparative data. Such data was critical not just for Zelboraf but for future efforts to improve on it. As a sad side note, Thomas McLaughlin—-the cousin from the NYT article who survived—passed away just last month from his melanoma. It would have been great if his cousin could have won the extra time that he did, but it will be better still if future melanoma patients can buy extra decades...or a cure.

Back to Josh Hardy's case, it's worth noting that in the only study to date of brincidofovir for adenovirus, there were some dose-limiting toxicities and efficacy didn't achieve statistical significance. Of course, this was only a small exploratory study, so that doesn't mean much—but that's exactly the point. The company still desperately needs to gather more data if the drug is to help more people.

Making emotional, unscientific decisions about access leads to emotional, unscientific reactions to the outcomes—like if the patient recovers, to decide this is a cure or "proof" of efficacy; or if the patient gets worse, to decide the drug is to blame—something that could derail further development of an effective therapy.

Chimerix's solution, to begin an open-label trial in adenovirous patients is actually a great solution both in terms of PR and, hopefully, advancing scientific knowledge—it allows the company to explore a potentially important new avenue while also, hopefully, helping out a child. But it may also encourage more and more increasingly tenacious social media campaigns that vilify drug companies for doing what is sometimes the ethical thing and denying therapy.

-Karl Thiel

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