On February 29, orphan drugs have their day of action: Orphan drugs is the name for drugs that target so-called rare diseases. So this action day is actually only topical every four years — rare, that is. But don’t worry, in non-leap years, Rare Disease Day falls on February 28.
Rare is a relative term:
- A rare disease affects no more than 5 in 10,000 people.
— Depending on the source, there are more than 6,000 to 8,000 rare diseases.
— It is estimated that four million people are affected in Germany.
— There could be 30 million sufferers in the European Union.
On the medication side, the numbers look like this
- In October 2021, 132 drugs were approved as orphan drugs in the EU
— The most expensive drug in the world currently costs over 3 million euros: the orphan drug Hemgenix from the US manufacturer CSL Behring was approved in the EU in February 2023 and is effective against severe and moderate hemophilia B even in a single dose.
— By 2026, orphan drugs are expected to account for nearly one-third of global drug development, according to statistics platform Statista
So times seem to be improving for orphan drugs. According to estimates by EvaluatePharma, sales of orphan drugs will increase to $273 billion by 2026 — twice as much as in 2020.