MARKET ACCESS TRENDS IN RARE DISEASE APPROVALS IN EUROPE FROM 2005 TO 2014

Author(s)

Schultz NM1, Sarangpur S1, Toumi M2, Sherman S1
1Creativ-Ceutical, Chicago, IL, USA, 2Aix-Marseille University, Marseilles, France

OBJECTIVES: Over the past decade, the European Medicines Agency (EMA) has granted regulatory approval to over 75 rare disease therapies and acknowledged hundreds of pipeline compounds with rare disease regulatory designation. In Europe, market access for new technologies is predominantly dependent on regulatory and country-specific health technology assessments. Therefore, the purpose of this analysis was to examine how the impact of United Kingdom (UK) health policies affected the market access of rare disease technologies during 2005-2014.  METHODS: A list of approved rare disease health technologies was constructed utilizing publically available data from the EMA. The technologies had to adhere to European specific rare disease designation guidelines for study inclusion. Secondly, the list was cross-referenced with the National Institute of Health and Care Excellence (NICE) repository of appraised technologies. Information extracted from the NICE repository included: complete appraisal document, incremental cost-effectiveness ratio (ICER), and final agency recommendation. Thirdly, an Excel worksheet was created and several descriptive techniques were used for trend analysis, including: calculating the proportion of NICE appraisal of EMA approvals, EMA/NICE annual approval rate, and ICER range across NICE appraisals.  RESULTS: A Positive trend in rare disease technology regulatory approvals from 2005-2014 was observed in Europe (average increase of 1.2 approvals/year), with the highest number of approvals occurring in 2014 (total approvals = 15). The trend in NICE approvals during this period remained flat at an average of 0.2/year (5 currently under review in 2014), despite the number of appraisals increasing an average of 0.5/year. Furthermore, the highest approved ICER value of all NICE appraised rare disease submissions was noted at £47,200/QALY, while 7 technologies with ICERs above £50,000/QALY were rejected.  CONCLUSIONS: ICER thresholds in the UK continue to be barriers to access therapies for rare diseases, although recent policy revisions may lead to changes in access in this market.

Conference/Value in Health Info

2015-11, ISPOR Europe 2015, Milan, Italy

Value in Health, Vol. 18, No. 7 (November 2015)

Code

PSY125

Topic

Health Policy & Regulatory, Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes, Reimbursement & Access Policy

Disease

Multiple Diseases, Rare and Orphan Diseases

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