NCPA - National Center for Policy Analysis


October 2, 2007

The Orphan Drug Act has helped bring drugs to patients afflicted with diseases that affect fewer than 200,000 people in this country.  And while the number of drugs treating those diseases has grown to more than 300, the price of progress is often stunningly high, says the Wall Street Journal.

Sometimes the price is so high that it threatens patients' access to the medicines that are supposed to help them:

  • One example is Acthar, a version of a hormone known as ACTH, which has long been approved for use in patients with multiple sclerosis.
  • A company called Questcor bought the rights in 2001 and is now trying to win approval to use it for a pediatric condition called infantile spasms.
  • Since the purchase, the price has jumped to $23,000 a vial from $1,600; under the new pricing, a round of treatment costs about $100,000.

For years now, researchers have been predicting a coming era of personalized medicine, with drugs targeted to match your condition and genetic profile, says the Journal.  Of course, personalized drugs will by their nature also have a small market.  Given the high price of many orphan drugs, it might make sense to start thinking about what the era of personalized medicine will cost.

Source: Jacob Goldstein, "The Big Cost of Little Drugs," Wall Street Journal, October 1, 2007.

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