Orphan Market Exclusivity

Orphan drugs are drugs intended for the treatment of a rare disease (a disease occurring in 5 or fewer patients per 10,000 EU inhabitants). Under normal market conditions, there is low economic incentive for pharmaceutical companies to develop drugs intended for such a small number of patients. Consequently, only 5% of rare diseases get appropriate treatment. The 7000 rare diseases lacking treatment is an important public health issue.

The European Orphan Medicinal Products Regulation (2000) aims to stimulate the development of products to diagnose or treat rare diseases, by awarding 10 years of market exclusivity to new orphan medicines. This protects pharmaceutical companies from competition from similar medicines with similar indications, which cannot be marketed during the exclusivity period, by granting them a 10-year monopoly. Read more about this here.

As a result of the Orphan Medicines Regulation the number of orphan drugs registered in the EU increased from 8 in 2000 to 169 in 2019. However, there are still huge access problems stemming from poor availability and affordability of orphan drugs, and thousands of rare diseases still lack an effective cure. The Regulation was intended for new orphan drugs, but companies also use the system to ‘repurpose’ old drugs, for which they also get 10 years of market exclusivity in the EU. As a result, companies can charge exorbitantly high prices, raising the cost of orphan drugs constantly. Read more about this here.

As a result, drugs approved through orphan market exclusivity policies are often inaccessible due to their high price, as a result of pharma companies’ abuse of their dominant market positions. Entry to the market of more affordable generic drugs is further delayed by ‘evergreening’ and ‘repurposing’ old drugs by pharma companies to extend market exclusivity for as long as possible.

What is FTV/PAF doing about this topic?

FTV/PAF have studied the misuse of the Orphan Drug Regulation, and has raised a case with the Dutch Competition Authority (ACM) about the 500x price increase of CDCA Leadiant. See also the mexiletine case.

What can we do about it?

The EU is currently reviewing its Orphan Drug Regulation. This opens an opportunity for fixing some of the issues in the Regulation.

Further reading


Technopolis report 2019