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) | ||