Healthcare funding: Rare diseases, a priority? AIM's proposals

Abstract

About 5000 to 7000 rare diseases are recognised. These life-threatening or chronically debilitating diseases are complex and have low prevalence – defined as 2 not more than 5 people affected per 10000 – so general knowledge and expertise about them can be limited. The limited number of patients affected by rare diseases also makes research difficult and potentially unattractive for the pharmaceutical industry. People affected by rare diseases can have difficulties in getting local diagnoses, in referral to suitably specialised health professionals who may be abroad, and in getting good information about their disease in their own language. In some countries or health systems the socio-medical coverage may not be welladapted. These are some of the reasons why a ‘European’ approach – which includes cooperation and coordination – is needed, wanted and supported by all concerned parties and actors in the health sector. Affordability and financial access to specific treatments represent a major challenge for national health systems as well as affected patients and their families. Due to legislation in several continents to encourage the development of drugs for rare diseases, the number of registered socalled ‘orphan drugs’ is increasing and recent statistics show that the costs of such orphan drugs are increasing exponentially. Improving the financial affordability of tremendously high cost individual treatments remains a real challenge and AIM sees here a new opportunity for action at EU level.