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

From: Estimating the budget impact of orphan drugs in Sweden and France 2013–2020

Parameter

Value*

Peak year budget impact as% of total pharmaceutical spend (year)

Peak year budget impact as% of total pharmaceutical spend (year)

Sweden

France

1

Change in price at loss of IPP

Base

-65% (SE)

4.1% (2020)

4.9% (2020)

-60% (FR)

Best

-80%

3.8% (2020)

4.4% (2020)

Worst

0%

5.7% (2020)

6.4% (2020)

2

Pharmaceutical annual market growth rate 2012–2020

Base

2%

4.1% (2020)

4.9% (2020)

Best

4%

3.4% (2018)

4.2% (2020)

Worst

0%

4.9% (2020)

5.8% (2020)

3

Success rate for designated OMPs obtaining market authorization approval

Base

Observed trend rate 2008–2012

4.1% (2020)

4.9% (2020)

Best

50% of base case rate

3.3% (2017)

3.9% (2017)

Worst

Observed trend rate 2000–2005

9.0%

11.0% (2020)

4

Annual growth in new OMP designations from 2012

Base

y = 5.7418x - 11452

4.1% (2020)

4.9% (2020)

Best

50% < base

4.0% (2020)

4.8% (2020)

Worst

50% > base

4.2% (2020)

5.0% (2020)

5

Average total annual sales of an OMP post-launch (year 1 – year12)

Base

As per Figure 6

4.1% (2020)

4.9% (2020)

Best

-50% p. a.

2.0% (2020)

2.5% (2020)

Worst

+50% p. a.

8.1% (2020)

9.9% (2020)

  1. *‘Best’ and ‘worst’ cases are from the perspective of funders of OMPs, with ‘best’ representing the lower budget impact and ‘worst’ the higher.