In the USA, a rare or orphan disease is defined as affecting fewer than 200,000 people and more than 7,000 rare diseases have been identified to date. The EU adopts a similar definition and identifies the existence of a similar number of rare diseases (see here).

Pharmaceutical companies are often reluctant to fund R, D for orphan diseases. As they affect so few people, there is no guarantee that successfully developed orphan drugs will be profitable. The Pharmaceutical Research and Manufacturers of America estimates that 95% of rare diseases do not have approved treatments (see here).

Both the US and EU have therefore sought to incentivise companies to develop orphan drugs. For example, in the US, the Orphan Drugs Act 1983 provides tax credits for clinical research into rare diseases, and the EU’s Horizon 2020 scheme provides funding of around €900 million for collaborative projects related to rare diseases.

Although more than 600 orphan drugs and biological products have now been approved in the US, they are still scarce, and often come with a high price attached. Given the relative lack of competition in this area, it’s no surprise that antitrust concerns have been raised about the potential for excessive prices to be charged.

This month’s CLIP examines antitrust enforcement actions involving orphan drugs in the US. It concludes that there has not been a special focus on orphan drug pricing compared to any other drugs.

From the European perspective, we’ve noted the attempt by the Dutch foundation for ‘pharmaceutical accountability’ to secure an investigation into orphan drug pricing in the Netherlands (here). As long ago as 2003, the OFT (as it then was) issued an abuse of dominance decision against Genzyme, in relation to a margin squeeze involving a treatment for an orphan indication. More recently, the question of excessive pricing in the pharmaceutical sector has been a focus across Europe. The UK’s CMA and CAT looked at it in Pfizer/Flynn (see here), and the European Commission is investigating Aspen Pharma for its pricing practices for cancer medicines (here).

In this current climate, whether it’s an orphan drug or any other potentially dominant product, pharmaceutical companies need to be prepared to justify their prices in case one of the competition regulators does come calling.