BACKGROUND Orphan medicinal product (OMP) prices are considered by some to be a challenge to the sustainability of healthcare expenditure. These concerns are compounded by the increasing number of OMPs receiving marketing authorisation (MA) annually. The aim of this study was to explore the sustainability of OMP expenditure within the context of total European pharmaceutical expenditure. METHODS Using historical IQVIA data, an analysis was conducted on total pharmaceutical and OMP expenditure in eight countries (using values / volumes) in the branded, non-branded and overall pharmaceutical market. Country level and aggregated data was considered for EU5 countries, Austria, Belgium and Ireland. Three key analyses were conducted: 1.The OMP share of total pharmaceutical expenditure was calculated from 2000 to 2017, to assess its evolution over time.2.The results of this analysis were compared with a 2011 forecast of OMP budget impact.3.The evolution of the total pharmaceutical market and its different segments (branded OMPs, non-OMP branded and unbranded) were assessed by estimating the compound annual growth rate (CAGR) and percentage of pharmaceutical expenditure for each market segment from 2010 to 2017. RESULTS Across countries, OMP share of total pharmaceutical expenditure has increased each year since 2000, rising to 7.2% of total pharmaceutical expenditure in 2017. OMP expenditure has increased at a CAGR of 16% since 2010. The number of OMPs receiving MA each year showed a CAGR of 11% since 2001, four percentage points greater than the CAGR for all medicines receiving MA over the same period. OMP share of total pharmaceutical expenditure is higher than forecasted in 2011 due to slower than expected growth in the non-OMP market. OMP growth has been offset by reduced expenditure in the general market and increased use of generics and biosimilars. CONCLUSIONS Relative spending on OMPs has increased over the last 20 years, but this has been largely compensated for within the current allocation of total pharmaceutical spending by flat expenditure for non-OMPs and increased volumes of (lower-priced) generics/biosimilars, reflecting a shift towards expenditure in higher cost, lower volume patient populations and a shift in drug development towards more specialised targeting of diseases.

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对欧洲孤儿药支出的分析：是否可持续？

背景技术一些人认为孤儿药品（OMP）价格对医疗保健支出的可持续性构成挑战。每年收到营销许可（MA）的OMP数量不断增加，使这些问题更加复杂。这项研究的目的是在欧洲药品总支出的背景下探讨OMP支出的可持续性。方法利用IQVIA的历史数据，对品牌，非品牌和整体药品市场中八个国家（按价值/数量）的药品和OMP支出总额进行了分析。考虑了EU5国家，奥地利，比利时和爱尔兰的国家/地区和汇总数据。进行了三个关键分析：1.计算了2000年至2017年OMP在药品总支出中的份额，以评估其随时间的变化。OMP在药品总支出中所占的份额高于2011年的预测，原因是非OMP市场的增长低于预期。OMP的增长已被一般市场支出减少以及仿制药和生物仿制药的增加使用所抵消。结论在过去20年中，OMP的相对支出有所增加，但是在当前总药品支出分配中，非OMP的固定支出和（低价）仿制药/生物仿制药的销量增加，在很大程度上抵消了这一支出。转向花费更高的支出，更低的患者数量，药物开发也转向更加专门的疾病目标。OMP的增长被普通市场上支出的减少以及仿制药和生物仿制药的增加使用所抵消。结论在过去20年中，OMP的相对支出有所增加，但是在当前总药品支出分配中，非OMP的固定支出和（低价）仿制药/生物仿制药的销量增加，在很大程度上抵消了这一支出。转向花费更高的支出，更低的患者数量，药物开发也转向更加专门的疾病目标。OMP的增长被普通市场上支出的减少以及仿制药和生物仿制药的增加使用所抵消。结论在过去20年中，OMP的相对支出有所增加，但是在当前总药品支出分配中，非OMP的固定支出和（低价）仿制药/生物仿制药的销量增加，在很大程度上抵消了这一支出。转向花费更高的支出，更低的患者数量，药物开发也转向更加专门的疾病目标。