Update 3/11/14, 9:57 p.m.: In a stunning reversal, Chimerix has announced that it has found a way to provide seven-year-old Josh Hardy the drug brincidofovir. The company has said that it has reached an agreement with the FDA to immediately initiate “a pilot trial of open-label brincidofovir for the treatment of adenovirus infections in immunocompromised patients,” including Josh, who will be enrolled tomorrow.
“Josh Hardy’s story brought to public attention the often-devastating impact of adenovirus infection, and helped accelerate a discussion between the FDA and Chimerix regarding the need for additional clinical development to assess brincidofovir’s potential in adenovirus infection,” the company said in a statement.
Chimerix CEO Kenneth I. Moch said that being unable to fulfill requests for the drug earlier was “excruciating,” while the company’s clinical research vice president, Hervé Momméja-Marin, M.D., explained that the new trial will still allow the company to pursue FDA approval for the drug.
NYU bioethicist Arthur Caplan praised the pilot trial as “a good compromise.”
It “looks like the pressure to find a regulatory solution that did not set the drug development back worked — (the) FDA now has a pilot study going at (Josh’s hospital) only which gets the drug to Josh but does not open the floodgates to all uses at all institutions,” he said. Still, he cautioned, “another child with adenovirus could appear (at another hospital) and the whole scenario will be recreated. Still, it is a good outcome for now.”
To understand what initially prevented the company from releasing the drug to Josh, read below.
If doctors told you that a trial drug could save your child’s life, but a pharmaceutical company refused to provide it, would you take “no” for an answer?
The parents of seven-year-old Josh Hardy certainly didn’t. Last week, Aimee and Tom Hardy mounted what’s since become a viral social media campaign urging anyone sympathetic to their cause to reach out to Chimerix, a small North Carolina pharmaceutical company, and pressure them to provide the drug brincidofovir to their son under what’s often known as “compassionate use” — the lawful release of a drug that has yet to win FDA approval for use by a patient with a serious or life-threatening condition.
Josh, a cancer survivor, is now suffering from adenovirus after a bone marrow transplant. The doctors at St. Jude’s Children’s Research Hospital in Tennessee are confident that oral brincidofovir is his best chance at staging a recovery, CNN reports. The St. Jude’s doctors themselves have seen the drug in action in clinical studies.
As a parent, I couldn’t help but view this as a nightmare scenario — a drug could save a child’s life, and some bean counters said no? WHAT?
But as a reporter, this fascinated me: why on earth would any company — especially a pharmaceutical start-up that few have heard of — willingly endure such terrible publicity? Who would court infamy as the business that turned away a dying child?
If the company is to be believed, it’s not just about Josh — it’s about the greater good.
In a local newspaper’s early report of the Josh Hardy story, Chimerix said that while they had provided the drug for compassionate use to some patients earlier (CNN later pegged the number of patients affected at more than 400), they ultimately decided to stop and “focus resources on earning FDA approval.”
I read the word “resources” to mean money and, in fact, CNN later reported that it costs Chimerix $50,000 to fulfill a single compassionate use request. Josh Hardy’s family told me they have access to the funds and would pay Chimerix, which reportedly receives millions in government funding, if that’s what it takes to save Josh’s life.
So what’s the problem? I reached out to the company to find out.
A spokesman told me “there has been no discussion of the cost of this medicine” and also provided me with a statement similar to what ran in the local newspaper. He suggested I get in touch with bioethicist Dr. Arthur Caplan of NYU Langone Medical Center. Caplan said he has no ties to Chimerix but has followed the case closely.
Caplan elaborated on the risks facing Chimerix. He explained that if a patient receives a drug through a “compassionate use” request but still dies not long afterward, it’ll be up to Chimerix to prove to the FDA that the drug wasn’t what killed him. Doing so might take days or it could take months, derailing their efforts to win final approval for the drug. It’s the sort of setback that a small company might not survive … and if it doesn’t survive, the drug definitely wouldn’t hit store shelves in 2016, to the detriment of “all the Joshes down the road,” Caplan said.
“All the Joshes down the road, of which there could be many more, they want to be benefited,” Caplan said. “There are some really tough trade-offs here.”
Josh Hardy’s uncle, William Burns, told me his doctors are confident the drug would work for Josh. But Caplan said that the fact that Chimerix’s current clinical trials aren’t targeting adenovirus — the disease now ravaging Josh — indicates that the company isn’t as confident about the drug’s efficacy for that condition.
Even if Josh had a great outcome, many others could lodge compassionate use requests on the heels of his. Satisfying those would open up the company and the drug to even more risk.
Caplan said that, in general, compassionate use of drugs can endanger future clinical trials. If patients all clamor to receive a drug through compassionate use requests, researchers might have trouble finding enough people to participate in clinical trials (where participants risk being given just a placebo instead of the actual drug.) No clinical trials, no FDA approval. No FDA approval, no widespread distribution of potentially game-changing drugs.
Caplan isn’t the only expert defending Chimerix.
“This is a very sticky situation for both Chimerix and the Hardy family,” said Dave Dragoo, a physician who practices in Colorado. “The fact of the matter is that without some form of regulation, drug developmentwould become like the ‘wild west’ where drugs come to market without having gone through the rigors of determining there safety. The bottom line for Chimerix, despite the fact that its drug may save this one child’s life, is that they have a huge amount of liability at stake.”
Caplan argues that there needs to be a government-organized systematic approach to the distribution of trial drugs under compassionate use — one that takes into account that patients often can’t afford to pay for experimental drugs that insurance companies refuse to cover.
That system doesn’t exist right now. What does exist is a very sick little boy who has thousands in his corner praying for his recovery and for Chimerix executives to change their mind.
For now, they’ve haven’t. The execs have apparently decided that they’d rather take a hit to the company’s reputation than, as Caplan suggests, risk the survival of their company and, by extension, their much sought-after drug.
Understandably, Josh Hardy’s family isn’t thinking about where Chimerix and its drug might be two years from now.
“We’re not talking about the future,” William Burns told me. “With this particular case, if he doesn’t get this drug, he’s going to die.”
Given the greater good argument, would you take “no” for an answer? I still can’t think of a single parent, including myself, who would. There will be future Joshes out there, but right now, it’s hard to see past this one.
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Photo courtesy of Josh Hardy family via SaveJosh.