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The world's first drug made for only one patient

The first ultra-personalized drug — made for one patient, the only one who will ever take it — is raising all kinds of new questions about how to handle a scenario that's likely to only become more common, the New York Times reports.

Driving the news: The drug, described yesterday in the New England Journal of Medicine, treats the neurological disorder of an 8-year-old girl.

  • The genetic presentation of her disease is unique, but one of her doctors had an idea about how to treat it, which was eventually successful.
  • It's unclear how much developing the drug cost, but the girl's mother and doctor raised $3 million through a foundation and on GoFundMe.

The big picture: This raises huge questions about how to regulate this kind of extreme precision medicine, who should get it and who should pay for it.

  • Researchers will have to decide which of the tens of thousands of patients with rare diseases to prioritize when creating custom drugs.
  • Families would likely end up on the hook to pay for such custom drugs, automatically limiting who has access to them.
  • It's also unclear how much evidence the FDA needs of such a drug's safety and how to evaluate its efficacy.

The bottom line: We now have no choice but to answer these kinds of questions as they play out in real life. Ultra-precision medicine is no longer only theoretical.

Go deeper: The struggle to evaluate drugs