The much reported story of Josh, a 7 year old child who needed an antibiotic drug not yet FDA approved to save his life, had a happy ending where his family was successful in getting the drug through a public campaign. The FDA worked with the pharma to set up a clinical trial that included Josh. Contrast this with the story of an adult, Nick Auden, 41, a lawyer, father of three, who needed a promising new therapy for melanoma, but couldn’t get into the clinical trial due to a technicality. He lobbied with an equally large social media campaign, unsuccessfully, until his life ended without a chance to try the therapy.
Arthur Caplan, director of Medical Ethics at NYU had this to say about Josh’s case, “From the perspective of the public and future patients, it’s best for the (pharmaceutical) company to focus on getting the drug approved as soon as possible so that the largest number of people can be helped. From a patient’s point of view, though, getting immediate access to the drug is what’s important.”
Why not do both? The solution is to ease the access to the clinical trials which will speed up approval.
What slows the process down? Clinical trial design has not changed for 50 plus years. Investigators and pharmaceuticals alike often hold the science, data, and expenditures more important than the outcome for patients. How about clinical trials that have a promising therapy randomized to a placebo, or a comparator that we know is ineffective? Patients drop out of those trials and/or accrual is difficult. No data is achieved then. Or if the patient progresses on the placebo or bad comparator, most trials don’t allow crossover to the promising drug. It is well known that only with psychological drug trials will we see the placebo effect and a poor comparator only helps to speed the demise of a patient. When a drug works for melanoma or other cancers, we know it!
So, my answer to this dilemma is to create a humane structure of clinical trial design. Put risky patients (such as those with brain mets, etc) in a parallel study, and who knows what valuable data might come from that study? Their data would be isolated from the rest of the study and would not affect outcome. We are seeing the start of that design in some institutions.
The economics of opening up trials to be inclusive will be questioned as will the cost of using good comparators. But there has been much worse waste reported with as much of three quarters of industry budget going to marketing! The resources it would take to make clinical trials easier for participation to a wide spectrum of patients will speed the process to approval and therefore to access. And the bottom line is the pharmaceutical industry would still profit.