NATIONAL RARE DISEASE STRATEGIES- THE CURRENT STATE FOR ORPHAN DRUG MARKET ACCESS IN EUROPEAN UNION (EU) MEMBER STATES
Author(s)
Scholten J, Lie X, Kalbasko A, Maervoet J
Quintiles Consulting, Hoofddorp, The Netherlands
Presentation Documents
OBJECTIVES By 2013 all European Union (EU) member states were recommended to elaborate and adopt a national strategy for rare diseases. This study provides insights into the national rare disease strategies, in particular about the status of the programmes, recent developments, and the congruencies and differences between the programmes regarding market access. METHODS A literature and Internet search was performed to identify national strategies for rare diseases published by EU member states. Both policies and recent orphan drug introductions have been analysed to compare the rare disease strategies. RESULTS All member states had a variety of approaches already in place before developing a national strategy. France is the frontrunner in implementing rare disease policies as well as the member state with the highest number of marketed orphan drugs. Over the past five years, most member states finalised their national plans with a peak in publications late 2013. Plans include similar methods on increasing patient access such as off-label use, compassionate drug use and utilising cross-border healthcare. Member states with a decentralised market access model (e.g., Spain and Italy) commonly use national funds and decision-making to provide equity in treatment levels across the nation for rare diseases. There are few orphan drug specific pricing policies; however special reimbursement criteria are common especially in countries with cost-effectiveness criteria. CONCLUSIONS Increasing patient access to orphan drugs has been a focus point in the national plans for rare diseases. Congruencies in methods will aid the EU’s ambition to align policies at European level. However the implementation phase has only recently been initiated for most plans and actual policies are yet to be developed. As member states such as France are introducing cost-effectiveness criteria in their health technology assessments, one can expect that tailored criteria need to be developed for orphan drug assessments.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PHP81
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Health Disparities & Equity
Disease
Multiple Diseases