Rare Disease Prevalence and Orphan Drug Pricing in Europe


By Claudia Graeve, Ph.D. (Vice President) and Kelly Cockerill (Director)
Health Advances GmbH


  • We analyzed the relationship between orphan disease prevalence and orphan drug pricing in Europe in order to tease out factors influencing orphan drug pricing potential.
  • According to our findings, treatments for orphan diseases with the lowest prevalence are likely to have the highest price across European countries. A clear understanding of the target population size and the potential budget impact is crucial to develop a pricing strategy for new therapies.
  • We discovered orphan drug prices differed considerably between European countries. Launch strategies can mitigate the potential negative impact of international reference pricing.

Recently, more drugs for rare diseases have become available in European markets. While offering treatment options to once untreatable patients, the high price points often commanded by orphan drugs (ODs) have sparked intense discussions about their value and how to determine a fair price.

Today, most European countries rely on national HTA methodology to assign clinical and/or economic value to new drugs, which together with international reference pricing (IRP) form the basis of pricing and access decisions. However, target patient populations for ODs are by definition small, posing challenges in collecting data on small populations and making pricing decisions.

Using epidemiologic data from HTA submissions and the literature, public list prices and HTA reports, we analyzed 13 non-oncological ODs launched since 2011 for a potential relationship between orphan disease prevalence and orphan drug pricing in seven European countries.

Figure 1: Relationship between Orphan Disease Prevalence and Orphan Drug Pricing in Belgium

Results and Implications
We found that treatments for the rarest diseases are likely to have the highest price in all analyzed countries (see example in figure 1).

Orphan drug list prices were highest in Denmark and Germany and lowest in France and Belgium (see figure 2).

Orphan drugs appear to follow the rule that catering to small populations commands a higher price – but where will this leave new OD pipeline candidates as they make their way through approval into the market? In times of elevated pricing scrutiny, orphan drugs will need to clearly demonstrate their medical (and potentially economic) benefit to ensure a favorable reimbursement outcome in Europe.

figure_23Figure 2

About The Authors
The authors are members of Health Advances European Practice Area which helps clients navigate through the diversity of European healthcare systems to optimize commercialization strategies of pipeline and in-market products.

Sladeček S, et al., “Relationship Between Orphan Disease Prevalence And Orphan Drug Pricing In Seven European Countries,” Value Health, November 2015.

Leave a Reply