Here’s a health policy idea, let’s listen to patients

Earlier this month I was a presenter and leader of a panel session at the Washington State of Reform Health Policy Conference in Seattle titled, “A Policy Framework for New Medicine.” The other two panelists and I were asked to present our points-of-view about policy related to “personalized medicine, miracle drugs, and genome-specific therapies.” The other two panelists were gene therapy patients. Ashanthi DeSilva is the first person in the world to undergo an approved gene therapy, which she did at the age of 4 in 1990. Toby Willis was “the first adult to undergo the first gene therapy approved in the U.S. for treatment of an inherited disease,” in March 2018.

Gene therapy is a topic of particular interest to me, naturally because of the work that I do, but also because I wrote my thesis to complete an M.A. in Philosophy at Boston College on the ethical use of gene therapy, in 1994. Those were the early days of gene therapy as a practical treatment, and I encountered a great deal of resistance for my thesis proposal from the hallowed halls of the esteemed Jesuit institution. My advisors were just not convinced there was any real-world application for gene therapy, and therefore they didn’t see the value in developing a framework for navigating the ethical considerations that I was sure were on the cusp of driving innovative patient care. What an incredible experience for me, then, to meet the woman who had undergone the world’s first gene therapy trial as a young child in roughly the same time period that I was pushing for approval from the chair of the philosophy department to start a conversation on how to decide who should receive (and for what reasons) gene therapies.

Both Ms. DeSilva and Mr. Willis are remarkable people and they were able to shine a light on the policy problems that still exist decades after gene therapy was first used to treat a patient. For example, Ms. DeSilva must still argue with her insurance company to have her ancillary medications approved because they are subject to prior authorization requirements despite the fact that she needs them because of her primary diagnosis – and will likely need them for the rest of her life. Mr. Willis’s policy story focused on the core questions before the group: Should we pay for innovative treatment? For whom? Under what circumstances? And, of course, who should pay? Individuals? Employers? Insurers? Taxpayers?

These sessions can sometimes be dry presentations where speakers do their thing without any regard for the audience. What happened at this session was different. It was a free flowing discussion that used the panelists as touchpoints, but drew out the expertise and ideas of audience members – including a health plan, a specialist provider, a policymaker, and pharmaceutical manufacturer. Some policy concepts offered were easily agreed upon, for example, trying to spread risk across a broader pool of people or spreading the costs of treatments over time. But I was the most impressed with the policy ideas offered by the patients in the room who were recipients of life-changing treatment. The ideas were at once simple, and nearly impossible. To close the session I asked the other panelists to give one recommendation for people in the audience to do when they left the room.

Ms. De Silva recommended that when thinking about health care policy, we should all try to think of someone besides ourselves.

Mr. Willis, who claimed not to be a policy expert, deftly explained what seems to be the core of the problem with the U.S health care system today. He said, “the system treats health plans as the customer, but I think patients should be the customer.”

So, as we begin an already busy health care policy 2019, I will be trying to heed this great advice. Maybe policymakers will, too.