Evaluation of orphan drugs arrangement eculizumab (Soliris®) for the treatment of atypical Haemolytic Uraemic Syndrome (aHUS)
The National Health Care Institute concludes that agreements made in 2016 about the use of the medicinal product eculizumab (Soliris®) can be continued. This is shown by an evaluation of the orphan drugs arrangement. Eculizumab is an orphan medicinal product for the treatment of patients with a rare kidney disease called atypical Haemolytic Uraemic Syndrome (aHUS). In 2016, the product was admitted to the basic health care package under strict conditions.
Haemolytic Uraemic Syndrome (aHUS) and number of patients
aHUS is a serious and rare disease; it can be fatal without proper and timely treatment. aHUS is a heterogeneous disease that is caused by defective complement regulation. This is a group of proteins that is important for the functioning of the immune system. The disorder aHUS leads to kidney failure in most patients. In the Netherlands, an average of 5 patients are diagnosed with aHUS every year.
More appropriate care thanks to the Dutch treatment guideline
The National Health Care Institute concludes in the evaluation that patients with aHUS receive more appropriate care thanks to the Dutch treatment guideline for eculizumab. The CUREiHUS study has monitored and evaluated the Dutch guideline for the treatment of aHUS since 2016. Research results show that 3 months of treatment is sufficient for many aHUS patients, while the marketing authorisation holder recommends life-long treatment with the patient receiving an infusion every 2 weeks. Treatment according to Dutch guidelines is much less invasive for the patient. The cost to society is also much lower: savings of €14 million over 4 years. Based on the evaluation, the National Health Care Institute sees no reason to revise the package advice of 2016.
2016 package advice eculizumab for aHUS
In 2009, the European Medicines Agency (EMA) admitted eculizumab to the market for the treatment of aHUS. After that decision, it was reimbursed by health insurance companies. After a few years, the National Health Care Institute selected it for an assessment in the context of risk-focused package management. On 21 November 2016, the National Health Care Institute published the package advice eculizumab for the treatment of aHUS. In this report, we advised the Minister of Health, Welfare and Sport to continue to reimburse eculizumab for aHUS from the basic health care package only after price negotiations and under strict conditions. Those 2 conditions were:
- the aHUS patient must be treated according to the Dutch guideline (discontinue eculizumab treatment after 3 months if possible), deviating from the guideline only if the indication committee unanimously agrees on the treatment recommendation;
- the treating physician and the patient must both fully cooperate in data collection for the CUREiHUS study.
Orphan drugs arrangement
Following the Minister's adoption of the advice and the price negotiations in 2016, an orphan drugs arrangement was agreed for eculizumab. This was done in consultation with the patient associations, health care insurers and the centre of expertise. The arrangement includes agreements with the occupational group on:
- start and stop criteria (according to the Dutch treatment guideline);
- setting up an indication committee (national working group aHUS);
- data collection and evaluation (register/CUREiHUS).