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The orphans of medi­cine — are they worth it after all?

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.