BackgroundOrphan 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.MethodsUsing 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: The OMP share of total pharmaceutical expenditure was calculated from 2000 to 2017, to assess its evolution over time.The results of this analysis were compared with a 2011 forecast of OMP budget impact.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.ResultsAcross 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.ConclusionsRelative 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.

survival of ovarian cancer (OC) in the EU. The inputs were fed into the DICE model and prevalence over time was evaluated and compared with EMA submissions. Results: A stable OC prevalence of 4.1/10,000 was estimated for the EU and compared with the varying rates submitted to EMA (2.3-4.9/10,000). Multiple scenarios were tested. For example, if incidence was increasing by 2% per year post-2002, prevalence would reach the 5/10,000 cutoff in 2012. Conclusions: The DICE epidemiological model can produce a robust prevalence estimate by synthesizing available data from different sources and allowing for multiple scenarios to be tested. This model can assist sponsors and the EMA in their review by introducing a transparent and flexible tool which can be used as a standard for orphan designation submissions across multiple diseases.