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# FDA Has ‘Strayed from Its Mission’: White Paper
- URL: https://www.fdaweb.com/fda-has-strayed-from-its-mission-white-paper/
- Published: 2018-07-10T12:00:00.000Z
- Updated: 2026-09-15T00:18:25.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5141834

A Niskanen Center white paper says that over the years, FDA “has strayed from its mission by mandating clinical trials that require far more than evidence of safety and effectiveness.” The [paper](https://niskanencenter.org/wp-content/uploads/2018/07/Fostering-Resilience-in-the-Medical-Marketplace.pdf?ref=fdaweb.com) says this mission shift, which Congress has allowed to happen, has increased drug development times, costs, and prices.

The authors propose categories of medical evidence upon which approval decisions can be made. They say such a change “would refocus FDA on judging products by using appropriate clinical measures and on communicating to the medical marketplace the nature of the evidence supporting its safety and effectiveness decisions.”

FDA frequently has adopted the position, the paper says, that purported clinical utility and clinical benefit (as defined by the agency and not by sponsors) as well as survival and disease outcomes are required for approval. “This has greatly handicapped medical innovation and has redirected drug development away from diseases that affect millions of Americans and toward niche diseases,” the white paper says. “It has also added to the long lead time and enormous cost of developing new drugs which, in turn, drive drug prices upward.”

According to the authors, the most important element of needed reform is to provide for tiered categories of approval of products that can be labeled for safe use according to the evidence used to validate their clinical activity. The four-part effectiveness paradigm they suggest would cover biomarkers, clinical signs and symptoms, disease modification, and long-term outcomes.

“This would put FDA in the proper position of adjudicating safety and effectiveness,” the report concludes, “and it would help FDA to clearly communicate to the medical marketplace its rationale for approval and the clinical effects that doctors and patients can expect when using new drugs.”