Patient Empowerment and the Collective Action Problem

In an insightful paper with human interest but also public policy implications Jasmin Barman-Aksözen writes:

My parents and I searched throughout my entire childhood for an explanation of why I frequently had unbearable burning pain after spending even short periods of time outdoors on a sunny day. This pain was incapacitating and often left me in agony for days, during which I was unable to go to school, to sleep, to tolerate even weak light exposure, or the body heat of my parents as they tried to comfort me. Not a single pain killer provided any relief, and the only option for me was to wait alone in a darkened and cooled room until the pain sub-sided. Of course, we tried everything that physicians recommended; still, not even high sun protection factor sunscreens helped prevent the symptoms despite the fact that they were obviously caused by sunlight. It must have been hard for my parents to see me in such a painful state without being able to alleviate or prevent it. What’s more, the worst thing was that classmates, teachers, and even physicians did not believe me when I told them about the symptoms; I even brought photographs showing myself with swollen and burnt hands and face. Yet, this didn’t stop some from making fun of me when I wore long clothing, hats, or used an umbrella on sunny days to protect myself from the sun’s rays. Eventually, after I was sent to see a psychologist for my “made-up symptoms,” I could no longer tolerate the derision and being treated with such condescension, and decided to stop sharing my experiences with healthcare professionals altogether.

Finally, a full 26 years after the first symptoms, Dr Google provided the answer! In April 2006, I found myself yet again unable to sleep because, despite all precautionary measures taken, I had burnt myself in the strong sunlight of spring. I entered the combination of my symptoms in the Google search mask and, surprisingly, there was a new link in Wikipedia with an expression I had not encountered before “Erythropoietic Protoporphyria.”

The made-for-tv aspect continues as Barman-Aksözen earns a PhD, moves to Switzerland to join the world’s leading lab studying these issues and, yes, develops the first effective treatment!

Afamelanotide was approved for the treatment of adult EPP patients in the European Union (EU) at the end of 2014.

But now is where reality and public policy step back in.

In April 2019, most EPP patients in Europe, however, still do not have access to the only treatment for their condition and are still unnecessarily suffering from fre-quent excruciating pain, social isolation, and impaired life choices. What went wrong? Before a newly approved medicine reaches patients, most European countries per-form a Health Technology Assessment (HTA) to evaluate the benefits in relation to the costs of the new product in order to support decisions on whether it should be reimbursed by the respective national health systems.

Getting the drug approved is only the first step. Now they have to get the medical authorities to pay for it and that means they have to show the drug is not only effective but cost effective given the disability. Barman-Aksözen goes on to describe her efforts to get the drug approved for actual use. She doesn’t put it this way but essentially she has to solve the collective action problem and form a lobbying group to make the case that patients with her disease, EPP, face a serious disability. It’s easy to measure death, however, but hard to measure the “disability weight” on say blindness. The WHO says blindness has a disability weight of .195 today, but in 2004 they gave it a weight of 0.594. Hmmm. One study of Afamelanotide suggests it has a cost of £373,000 per DALY averted, which is high, even though the article recommends adoption. Many meetings ensue in which the case for and against Afamelanotide is made. The process is slow. Years go by. Much depends on seemingly minor choices in how to present the data.

I was reminded of Mancur Olson’s discussion in the Rise and Decline of Nations:

Distributional coalitions make decisions more slowly than the individuals and firms which they comprise [and] tend to have crowded agendas and bargaining tables…The accumulation of distributional coalitions increases the complexity of regulation, the role of government, and the complexity of understandings, and changes the direction of social evolution.

In other words, socializing health care means socializing decisions about how to allocate health care. A difficult tradeoff.

Addendum: The FDA has yet to approve Afamelanotide.

Hat tip: Joe P.


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