Study: Hundreds of rare disease drugs in testing

For the nearly 30 million Americans who suffer from a rare disease, there's heartening news: Drugmakers have been shifting much of their research away from pills for the millions to uncommon disorders that often kill prematurely because there are few or no treatment options.

Pharmaceutical and biotech companies are running patient tests of more than 5,400 potential new medicines, including many being tested for multiple conditions. Nearly 1,800 research projects are for rare diseases, and hundreds more are for disorders for which there's been no new medicine in a decade or more. That's according to a study to be released Thursday, sponsored by the trade group Pharmaceutical Research and Manufacturers of America, known as PhRMA.

The study also found that thousands of those experimental drugs could be the first in a new medicine class with a unique target or effect, including 45% of 1,100 drugs that made it to the final patient testing stage. At least 577 use new technologies, from gene therapy to cloned antibodies, to precisely target the disease site and limit side effects elsewhere. And at least 155 are part of a long-awaited vanguard of personalized medicine, in which gene variations or other characteristics help doctors determine whether a medicine will help a specific patient.

Click here for full article


CRN Newsletter Signup - Popup
  1. Stay up-to-date with the latest information from the Cystinosis Research Network by joining our email list.

  2. First Name
    Invalid Input
  3. Last Name
    Invalid Input
  4. You must enter a valid email.