Rare disease EU patients treated unequally
access to treatments for patients with rare disorders can vary by
as much as 70 per cent within individual EU member states.
The conditions for marketing orphan drugs with European status in the EU are poorly understood and are a source of concern for the pharmaceutical industry, whether they are member states, patient associations or industry executives. Among the problems raised are differences in prices between countries and the fairness of prices practiced.
These differences arise more from different distribution and taxation policies between countries rather than differences in original manufacturer's prices, which is more uniform between countries and which reflects the desires of pharma companies to harmonize their prices.
The study also revealed that the rarity of the disease was a primary factor in treatment cost when compared to "conventional" products. While the costs for researching and developing a product are in the same order as those for a more widespread condition, the rarity, and hence the smaller patient population, resulted in a higher cost-per-patient.
The results of the study are apparent despite the fact that most product prices set by manufacturers are broadly the same from country to country. The difference depends on the healthcare systems of individual EU Member States.
The study, conducted by Alcimed, was conducted in order to map prices of orphan medicinal products and to develop effective tools to judge whether an individual product was profitable enough to warrant losing its regulatory incentives. Currently, member states, decide on a case-by-case basis, which products will be on the lists for healthcare coverage and, in turn, who will or will not pay for them.
Alcimed's study could not determine whether or not a medicine could qualify for incentives since the EU's Orphan Medicines Regulation has only been in force since mid-2000, while developing a new medicine takes about 10-12 years on average.
Pharmaceutical companies developing therapies for rare diseases called on the member states and the EU institutions to improve availability of orphan medicinal products to patients, and a supporting framework for them. They have jointly developed a "White Paper" with proposals for a framework of measures, which will be presented publicly in the near future.
Alcamed commented that the absence of a sustainable substitute for access through regular coverage by the health systems was having a serious knock-on effect. The many "fill-in" temporary solutions that have been created for individual patients suffering from rare diseases was not a viable option in the long run.
The analysis concluded that an estimated potential annual turnover would be somewhere between € 100 million and 1.5 billion, depending on the medicine. Even if some Orphan Drugs could become blockbusters, the maximal sales of these medicines would remain below real sales of the top ten medicines, which have an annual turnover of between €2 and 7 billion.
