Rare Disease Day: Orphan Drugs are changing the structure of the pharmaceutical industry

28.02.2022 14:20

In the past 20 years, 200 medicines for rare diseases (orphan drugs) have been approved in Europe. This means that those affected have more treatment options available to them every year. Orphan drugs are drivers of innovation: on average over the past five years, they accounted for a good third of new annual launches. And they are changing the structure of the pharmaceutical industry: around half of the suppliers are smaller and highly specialized companies with only one active ingredient on the market.

Of the orphan drugs approved in Europe, the vast majority (around 94 percent) are available to German patients. What is striking is the provider structure for orphan drugs and the unusually low concentration of the market: of the 112 companies with orphan drugs in 2020, 61, more than every other company, were on the market with only one active ingredient, and a further 20 with only two.

“The high number of developers of orphan drugs, often with only a few products, shows that the legal rules for orphan drugs have reduced market entry barriers for innovators. Smaller companies are focused on a medical field where there is a lot of catching up to do for patients,” says the head of the IGES Institute, Prof. Bertram Häussler. “The development of orphan drugs remains economically very risky for companies. However, the large number of innovators increases the chances of finding a treatment option for the still numerous untreatable orphan diseases. This should not be overlooked when designing the framework conditions,” says Häussler