World's Most Expensive Medicine: Is it Worth the Price? - (Bloomberg via NewsPoints Desk)

  • German health authorities ruled Thursday that the benefit of UniQure's gene therapy Glybera is "not quantifiable" and further safety and efficacy data are needed to begin a new assessment in June 2016, Bloomberg reported.
  • UniQure CEO Jorn Aldag said that the cost of the drug, which is around $1 million, should be compared with other orphan drugs that target rare diseases, which on average cost about $250,000 a year.
  • "It is often hard to be the first," Aldag said, adding "this is where people are learning. Not only are we learning, but so are the regulatory agencies."
  • Aldag suggested that for other gene therapies, payments may be spread out over several years and could cease if a patient dies, rather than for Glybera where the price is paid upfront.
  • According to UniQure's marketing partner Chiesi Farmaceutici, the final price in Germany will be negotiated with health insurance funds, which will use the latest assessment as a guide.
  • "Glybera, a pioneer product in gene therapy, is targeting an exceptionally small market," noted Chardan Capital Markets analyst said Gbola Amusa, adding the cost "is likely to be far above the price of future gene therapies."

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