Delays in cancer drugs that work

For decades, critics have complained that the drug approval process at the U.S Food and Drug Administration (FDA) has sometimes delayed access to lifesaving drugs to patients with little other recourse. This is partially due to the requirement that drugs be proven both safe and effective in controlled human drug trials at the end of a long experimental process.

A Wall Street Journal editorial points to another glitch in the system: drugs can be proven both safe and effective – but if they aren't effective for the whole population group specified in advance by the pharmaceutical company, the human trials must be repeated with a new target group. For example:
 

• Provenge, a late-stage prostate cancer drug that stimulates the body's immune system, was found in 2002 to delay the disease's progression in many patients, but not in the most aggressive cases – but because the drug application did not specify less aggressive cases as the target subgroup, the drug's maker must conduct new trials, delaying approval until 2005.
   
• Similarly, says the WSJ, the FDA delayed Iressa, another targeted cancer therapy, for months after an advisory panel recommended approval, and it still has not approved ImClone's Erbitux, despite confirmations of its efficacy.
  
  The coming generation of cancer drugs will likely work on certain patient subgroups not easily identifiable in advance. This suggests the agency should find a way to recognise obvious evidence of efficacy, approving these drugs quickly and monitoring data from actual clinical practice to refine its understanding of their action.
  
  Source: Editorial, New Cancer Drugs, Wall Street Journal, January 26, 2004.
  
  For WSJ text (requires subscription)
  
  For FDA Drug Approval http://www.ncpa.org/iss/reg/
  
  FMF Policy Bulletin\10 February 2004