Professor Maarten Postma:

I thank the committee for this opportunity. What I am going to say links to the previous discussion. My address is on health technology assessment, HTA, and its role in rare diseases. Health technology assessment differs between countries in Europe, although we now have the joint clinical assessment which tries to bring them together a little. The Netherlands and Ireland work together. Zolgensma is an example of a drug for spinal muscular atrophy that was assessed between the Netherlands and Ireland jointly in the Beneluxa Initiative. The Netherlands is strict on health technology assessment so I hope we did not force that on Ireland too much in the Beneluxa Initiative. For example, I have a list of drugs, including tecartus, carvykti and casgevy, which were all denied access. They are for rare diseases. There are more on my list but I will not bother the committee too much. These are innovative drugs. It illustrates the difficult time that drugs have in getting through the health technology assessment and getting reimbursed in the Netherlands. The Netherlands is a country with very strict negotiations and we tend to achieve relatively low prices and big discounts for orphan drugs, which means that if there are supply shortages globally, they often soon emerge in the Netherlands because other countries seem to pay higher prices.

In health technology, as Members of Parliament may have heard, it is often about the cost per quality. There is a value on human life and a maximum of what we pay for the life years that we gain from orphan drugs. We take a set quality of life into account. That comes in the end to the metric of cost per quality-adjusted-life-year, QALY. The cost per QALY maximum in the UK is £30,000. It is €50,000 in my country. The maximum for severe disease is €80,000. Many rare diseases qualify as severe disease, unfortunately for the patients. Drugs for rare diseases unfortunately cannot make the threshold of €80,000. Whether we are in a country which relies heavily on economic assessments or less on economic assessments, such as Germany, all over Europe, you will see that drugs for rare diseases have problems getting through HTA and cost-effectiveness is just one of them.

Together with Professor Michael Barry, in the International Society for Pharmacoeconomics and Outcomes Research, we have-----