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Table 1 Results from one-way sensitivity analysis on key model parameters.

From: Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

Parameter Value Peak year budget impact as % of total pharmaceutical spend (year)
1 Lag between orphan drug designation and marketing approval Base 4 yr 4.6% (2016)
   Best 6 yr 4.2% (2016)
   Worst 2 yr 4.9% (2015)
2 Eurozone + UK pharmaceutical market value in 2010 (ex-factory) Base €140 bn 4.6% (2016)
   Best €170 bn 3.8% (2016)
   Worst €110 bn 5.8% (2016)
3 Total pharmaceutical market growth per year Base 6.6% 4.6% (2016)
   Best 10% 4.0% (2014)
   Worst 3% 5.9% (2020)
4 % of products with orphan designation that achieve market authorisation Base 10.92% 4.6% (2016)
   Best 7% 4.2% (2015)
   Worst 20% 6.6% (2016)
5 Growth in new designations per year after 2010 Base 10% 4.6% (2016)
   Best 5% 4.6% (2016)
   Worst 50% 4.7% (2020)
7 Average cost of orphan disease per year (Yr 1 - Yr 10) Base As per Figure 2 4.6% (2016)
   Best -25% p.a 3.4% (2016)
   Worst +25% p.a 5.7% (2016)
8 Drop in drug prices at the point that drugs lose patent protection or marketing exclusivity Base 25% 4.6% (2016)
   Best 50% 4.2% (2014)
   Worst 0% 5.1% (2019)
  1. ('Best' and 'worst' cases are from the perspective of funders of orphan medicines, with 'best' representing the lower budget impact and 'worst' the higher)