Chimerix, a small and unprofitable biotechnology company, will make an experimental drug available to a young Virginia boy who is suffering from an infection he contracted while being treated for cancer.
Chimerix originally said it could not ethically provide the drug to one patient without opening the floodgates to others, presenting it with a huge operational burden that might prevent the drug, brincidofovir, from ever reaching patients. The story of the boy, Joshua Hardy, was shared on CNN, USA Today, The Huffington Post, and generated a large volume of posts on the social networking site Twitter under the hashtag #savejosh. This evening, Chimerix, based in Durham, North Carolina, issued a press release saying that it was going to start a new, 20-patient clinical trial for the treatment of adenovirus (the virus brincidofovir treats) in immunocompromised patients. The first immunocompromised patient, dosed tomorrow morning, will be Josh Hardy.
"This 20-patient open-label study underscores Chimerix’s mission to develop innovative antiviral therapies in areas of high unmet need - for everyone," said Chimerix Chief Executive Kenneth I. Moch in his company's press release. “Being unable to fulfill requests for compassionate use is excruciating, and not a decision any one of us ever wants to have to make. It is essential that each individual in a health crisis be treated with equal gravity and value, a principle we have upheld by pursuing further clinical study of brincidofovir that will inform its use in adenovirus and other serious DNA viral infections.”
Update: In an interview, Moch said that the trial was being planned as the starting phase of a larger study that could eventually lead to the approval of the drug for patients like Hardy. ""The plan and stategy here is a pathway that could lead to approval," he says. "If you look back at everything that we said publicly, it had to be about the many. As much as we cared about a specific individual, it had to be equitable and it had to be associated with the [needs of the] many and that's what we've accomplished here."
The decision on Chimerix's part could become precedent-setting for the pharmaceutical industry. Usually companies don't make their products available in compassionate use for precisely the reasons Chimerix had stated, as well as others, according to Arthur Caplan, director of the Division of Medical Ethics at NYU Langone Medical Center. They fear that when a sick patient gets sicker, their drug will be blamed, potentially leading regulators to halt their clinical studies. They worry that patients won't enter clinical trials because with compassionate use, they won't get placebo. Production problems can mean there isn't enough drug to go around. And startup companies that have no profits and a handful of employees say they can't handle the burden of sifting through patients to figure out who should get a medicine and who should not.
Just last year, a larger biotechnology firm, Biomarin, came under pressure from an ovarian cancer patient who wanted its drug. The company did not relent, and Sloan died in January. Last year, The New York Times Magazine published a long feature on how dying patients get access to cancer drugs, citing examples where
One issue raised by Caplan in a phone conversation yesterday is that it's not ethical to just give a medicine to the person who has the saddest story, or the biggest social media presence. When I emailed him about this announcement, he wrote back that it was a good thing that the FDA and Chimerix had found a way to thread the needle between compassionate use and a clinical trial. But he worried about the consequences.
“The fact that this flood of public sentiment pushed this to happen shows that we are now firmly in the age of patient appeals through old and social media,” Caplan wrote me. In a column he writes for MSNBC, he'd written: 'Save Josh, but what about the rest?' A 67-year-old would still be out of luck, and that's not necessarily a fair way to make experimental medicines that may not even work available. He calls for Congress to create a fund to pay for getting experimental medicines to patients, and to change the law to make it easier for that to happen.
Tomorrow morning, it's going to be time to think very hard about how our system of getting experimental drugs to patients is broken, and how we fix it. But tonight it's good to know that Josh Hardy will get a medicine that may kill the virus that is spreading in his body. Here's hoping and praying that the drug does what it's supposed to do and that Josh gets to go home.
