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Rare Drugs In the Netherland Context - A Question of Necessity

Updated: Nov 11, 2021

In this post I will reflect on my previous research which I was part of during my doctoral research at Erasmus University Rotterdam.

In the Netherlands, societal debate regularly focuses on drugs for rare diseases. The discussion often focuses on whether specific drugs for rare diseases are too expensive to be covered by the basic benefits package of the collective health insurance, which every Dutch citizen is obliged to take out. Formally, the requirements for all health care technologies that are covered is that they should be cost-effective, but also necessary.

The Dutch Health Care Institute uses these criteria to advise the Minister of Health about the contents of the benefits package. But how is the Minister of Health to establish whether a health care technology is necessary?

In my dissertation, I have examined this process of establishing a necessity at the Dutch Health Care Institute. I show that context-dependent coverage advice can, and often does, withstand pressure from outside sources.

The first step of my dissertation was to map the argumentations pertaining to necessity. As it turns out, there are many. This discussion did not only center on the medical necessity of a treatment, or even the severity of the illness but also its nature: Is this a disease, or is it just part of life? Does it affect social functioning? Or dignity? Are there other people it impacts? And is relieving this burden a societal or an individual responsibility? In various case studies from the Netherlands and other Western European countries, including drugs for rare diseases but also paracetamol tablets and maternity care, I investigated the patterned but varying use of such argumentations.

Varying use of the necessity argumentations may seem problematic, as variation would indicate inconsistency and potential inequity between decisions. Instead, I show that these variable necessity argumentations deliver tailor-made decisions; they provide the right context. As the relevant setting differs per health care technology appraised, the relevant necessity argumentations vary likewise. Well-contextualized health care coverage decisions contain necessity argumentations that are specific for this decision and carefully woven together, which makes the decision relatively able to withstand pressure from outside.

One case I examined was the CUREiHUS initiative, where doctors and patients together advocated eculizumab, an expensive medicine for a rare disease, to be covered through the benefits package. This medicine was not cost-effective enough, but it is now covered only for patients who participate in a study set up to show that lower cost-effectiveness is possible. This proposed study was a context that was vital to the decision-making process. Enabling this study to go ahead, it is argued, is a societal responsibility. It is also a clear example of SPIN, social pharmaceutical innovation.

I am excited to find out more under the SPIN research flag concerning the coming together of the CUREiHUS initiative and its institutionalization, in addition to having studied the vital moment of establishing its necessity.


Written by: Tineke Kleinhout-Vliek,


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