I was reading a good blog post about ethics violations in clinical trials in India, and was reminded of an interesting case in the U.S. Several years ago a 21-year-old woman (Abigail Burroughs) with terminal cancer (who had exhausted all treatment options) was denied the option of purchasing a drug that was in the final process of receiving FDA approval. The FDA ruled that terminally ill patients should not be permitted to purchase drugs until the drugs have completed the approval process, citing safety concerns. Their arguments also included that:
1. Patients cannot make a truly informed decision regarding whether or not to try a medicine that has not yet been fully tested.
2. Allowing access to drugs before they are FDA approved would undermine the clinical trial process.
3. If terminally ill patients could buy treatments, others would soon expect health plans to cover these costs for others.
4. Allowing access to drugs before they were proven effective would add to the public’s general confusion about evidence-based medicine and promote magical thinking.
I don’t know how you feel about this, but it makes me squirm a little bit. Although I appreciate the FDA’s position (and my colleague Dr. Jim Sabin has blogged about his support for the ruling) it just seems a little heavy-handed, especially in light of the details of the Abigail case. Here’s what I wrote to Dr. Sabin:
About the Abigail case… I think they were trying to get access to Erbitux for Abigail, and it was in phase III trials at the time (so there was mounting evidence for its efficacy – their request to purchase it wasn’t scientifically unreasonable.)
I have very mixed feelings about the FDA ruling. I understand that we don’t want to 1) set a precedent that would lead to insurers having to pay for expensive experimental therapies 2) discourage people from entering clinical trials 3) allow drug makers to profit from “false hopes” in terminal cases. However, couldn’t we add enough caveats to make it reasonable to allow patients who have exhausted all other options to purchase (with their own money) drugs that have shown promise but are not yet FDA approved?
What if we said that the drugs had to be in phase III trials, with enough evidence to suggest a plausible benefit for the patient? Terminal patients are rarely good candidates for clinical trials (I don’t think we’d be poaching from the CT pool), few can afford to buy monoclonal antibodies (and the like) on their own so if there were trials for terminal patients, they’d still have great incentive to join, and I’m not sure that just because a patient can buy a treatment that insurance will HAVE TO follow suit.
Something about limiting personal choices (for those who have the means to make them) seems un-American to me. There are very low risks of harm in monoclonal antibody treatments (so the argument that the FDA must err on the side of patient safety doesn’t really fit the Abigail case). Yes, it’s sad for those who can’t afford to buy every possible therapy – but why should we deny access for those who can? With the right caveats, I think we could allow people like Abigail to try every remaining option. But of course I agree that phase I drugs are just not far enough along the research pipeline to make educated choices about them.
Maybe Abigail was a casualty of the one-size-fits-all, population-based rules that are appropriate most of the time, but fail in exceptional cases. I predict that this sort of thinking (where evidence-based protocols are applied in a cook book manner to all patients with a given disease) will dominate the healthcare landscape in the coming years as we seek to reign in costs and do the best thing for most. I just wish there were a way to bend rules on the edges a bit. What do you think?