Recently, news outlets have reported on the story of Josh Hardy, a seven-year-old cancer patient in desperate need of an antiviral drug called Brincidofovir.
Manufactured by the pharmaceutical company Chimerix, Brincidofovir has not yet been approved by the FDA; consequently, Josh could not obtain the drug through normal means.
With exceptional cases like Josh’s in mind, Congress authorized the FDA—in the Prescription Drug User Fee Act (PDUFA)—to allow “compassionate use” of drugs before they are approved for marketing. The compassionate use program is an attempt to balance the need for both speed and thoroughness in the FDA drug review process. It allows companies to provide patients with life-threatening conditions drugs that may help them, but are still in the “investigational” stage and not yet approved by the FDA for marketing.
However, the compassionate use program presents pharmaceutical companies with a difficult choice: either they can enroll patients under compassionate use at the risk of slowing the drug’s approval process, or they can focus on the clinical studies that might get the drug approved sooner but leave other patients without needed medication.
But do not be fooled.
Compassionate use is not as compassionate in practice as it sounds in theory. I know, because my family has a story very similar to Josh’s.
Fifteen years ago, my brother John, then age five, was dying from a rare cancer. His only hope was a drug (now marketed as Gleevec) that had shown near 100 percent effectiveness in clinical trials but had not yet been approved by the FDA. Although the FDA compassionate use rules allowed manufacturers to provide the drug in cases like John’s, Novartis, Gleevec’s manufacturer, initially refused to comply. Like Josh’s parents today, my parents had to mount a campaign doing everything in their power to apply personal and public pressure to obtain the drug for my brother.
It took a year of lobbying congressmen and struggling to convince Novartis that our family understood the risks of using a drug that had not yet entered FDA clinical trials. After appearing on the ABC News program “20-20,” however, my parents created enough publicity that Novartis relented.
Even though the battle was won, the war—the race to save John’s life—was lost. We gained a few additional precious years with my brother, but had his treatment not been delayed while my parents lobbied Congress and the media, there is a good chance he would still be with us today.
Over a decade later, Gleevec has proven itself to be a revolutionary cancer drug. Indeed, the positive results from compassionate use of Gleevec helped speed FDA’s approval of the drug. Its developer, Dr. Brian Druker, has earned worldwide recognition for this medical breakthrough.
Yet dying patients are still battling to obtain other treatments not approved by the FDA.
Chimerix previously participated in the compassionate use program with over 400 patients. However, Chimerix CEO Ken Moch believed that continuing to do so would delay regulatory approval, stating that Chimerix “made the decision two years ago to stop the [compassionate use] program and focus resources on earning FDA approval.”
The Gleevec experience shows that sometimes compassionate use leads to quicker FDA approval. And this isn’t a conversation about statistics and percentages; real lives are at stake, and those patients—mothers, fathers, sisters, brothers—cannot wait another day.
So what is the alternative to family-based campaigning? What options can pharmaceutical companies like Chimerix and Novartis provide for patients and their families?
Sadly, there is no simple answer.
After the Hardys mounted a grassroots campaign for access to Brincidofovir, Josh was finally given the medication he needed—not through compassionate use, but through a pilot trial. The Hardy family gained access to this potentially life-saving drug, but their struggle for treatment is an all-too-common story. The current system is a bad deal for patients and a bad deal for companies. In this case Chimerix’s CEO Ken Moch resigned in the wake of the Josh Hardy P.R. blitz to “pursue other interests.”
My family may have been too late winning our own battle, but let us hope that Josh is receiving his medication just in time. Perhaps in the future there will be other options besides last-minute public campaigns. If drug companies like Novartis and Chimerix have more incentives they will bring better drugs to the market. What patients need is a faster drug review and approval process. There are few dying patients who can afford the money—or time—to mount a campaign for access to life-saving treatments.
Furthermore, they shouldn’t have to. Families, doctors, policy advisors, and pharmaceutical companies should find a solution to save children like Josh and John. PDUFA is due for reauthorization by Congress in 2017. Perhaps this will be policymakers’ opportunity to finally create a drug-approval system that works—one that reaffirms these children’s futures by offering appropriate incentives for pharmaceutical companies’ enrollment under compassionate use.