Handelsblatt Exclusive

Drug Firms Exploit Rare Disease Loophole

many of the so-called orphan drugs are used to target cancer.
  • Why it matters

    Why it matters

    Patients and health insurers are both losing out as pharmaceutical firms use rules that were meant to encourage them to develop cures for rare diseases to generate profits from common conditions.

  • Facts


    • Diseases are categorized as rare if they affect fewer than 5 in 10,000 people.
    • Some 30 million people in the European Union suffer from a rare disease.
    • In the United States, the average treatment for a patient suffering from a rare disease in 2014 cost around €86,000. Some exceed €1 million.
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Lars and Jonas Borek are brothers living in Hamburg. Aged 19 and 12, both suffer from Fragile X Syndrome, a rare disease that leads to autistic behavior patterns.

They have trouble filtering experiences and suffer from anxiety. Their mother descibed their condition as like playing the piano “and the key doesn’t stop when you let it go.”

There’s a “chaos of sounds” in their minds.

Both of them will need life-long care. But that’s not the only problem the family faces. The pharma industry has so far shown little interest in investing in possible treatments for Fragile X.

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