Orphan drugs are treatments for rare diseases which are difficult to acquire or extremely expensive because the lack of a profitable market discourages companies to invest in them.
The group was started by the Belgian and Dutch health ministers in 2015. They were joined by Luxembourg in 2015 and Austria in 2016. Ireland joined in June 2018. Anne Calteux, senior policy advisor at the ministry of health in Luxembourg, told Delano in December that “the accent put by the initiative on rare diseases convinced Luxembourg to join this project”.
As a coalition of countries, Beneluxa has more leverage in price negotiations with pharmaceutical companies and can demand more transparency into how the costs of new drugs are set. Working together by sharing data and policies also improves the price transparency between the countries, which ideally allows relatively unified prices and policies for patients.
The initiative also conducts joint health technology assessments and participates in “horizon scanning”. That is the practice of detecting new and groundbreaking treatments before they reach the market by analysing data and research output. This allows countries to make informed decisions for their reimbursement policies. It further allows national authorities to “better react to possible impacts on health budgets,” said Calteux.
To render this process more effective, the outfit is setting up a permanent system called International Horizon Scanning Initiative. This project is not limited to participating countries, but will be available to any country wishing to join.
Spinraza
Beneluxa’s first, and up till now only, major achievement is the successful price negotiation in 2018 with the pharma company Biogen for the orphan drug Spinraza, which is used to treat a special type of spinal muscular atrophy. Belgium and the Netherlands will reimburse the medication until at least 2020. According to the APM news service, after months of bargaining Beneluxa was able to reduce the initial list price for the drug of €83,000 per injection to a more reasonable amount, although the exact figure was not disclosed (six injections are needed the first year and three per subsequent year).
There are critical voices doubting the real impact of the initiative due to possible confidentiality issues and companies’ unwillingness to relent their country-specific pricing strategies, demonstrated by for example the failed agreement on the drug Orkambi. Yet, among member countries, optimism prevails. Belgian health minister Maggie De Block stated last summer: “The more patients we represent, the more our voices will be heard when discussing high-cost innovative medicines.”