Release: Consumer groups, health experts call for changes to accelerated drug approval in FDA reform bill

Media Contacts
Taran Volckhausen

Former Communications Associate, The Public Interest Network

WASHINGTON – The Senate Health, Education, Labor and Pensions (HELP) Committee will mark up a discussion draft on the Food and Drug Administration’s user fee legislation next week. In response, U.S. PIRG joined over 35 health experts and other advocacy groups in a letter released on Friday recommending stronger enforcement of post-market clinical trials for accelerated approval drugs to better protect patients.  

The accelerated approval pathway is an alternative review process used by the FDA to approve some new drugs. When conducting clinical trials required for FDA approval, drug companies are allowed to substitute a “surrogate endpoint” which is thought to prove a clinical benefit. Changes in lab test values or changes in size of a tumor are examples of surrogate endpoints. By using these substitutes, rather than requiring a longer clinical trial to prove, for example, that the patient is healed or has a longer life, new drugs can come to market faster. The accelerated approval law does require that drugs that win accelerated approval must undergo a full post-market trial, after the drug goes on the market, to prove they are truly effective at treating the condition or disease. 

The letter points out that drug companies have not consistently started or completed these required post-market trials, meaning “too many products have lingered on the market and potentially endangered patients who continue to take these medications.” The coalition letter makes several recommendations to strengthen the accelerated approval process:

  1. Manufacturers must complete the post-market studies to confirm the drug’s predicted clinical benefit. 

  2. If the drugs approved under the accelerated approval pathway are not shown to offer clinical benefit after five years, they should be automatically removed from the market.

  3. The FDA should require that the clinical trials already be underway when the approved drug comes to market to lessen the time between approval and the end of the full clinical trial.

“It is time to fix the proven problems that have arisen under the accelerated approval pathway,” said Patricia Kelmar, U.S. PIRG’s health care campaigns director. “Swifter approval is great for patients if the drug is as safe and effective as hoped. But the post-market trials must be conducted without delay to ensure we aren’t being given false hope or being exposed to medications that could have harmful effects.”