Analysis

21st Century Cures Act and similar policy efforts: at what cost?

BMJ 2015; 351 doi: https://doi.org/10.1136/bmj.h6122 (Published 23 November 2015) Cite this as: BMJ 2015;351:h6122
  1. Diana M Zuckerman, president ,
  2. Nicholas J Jury, senior fellow,
  3. Christina E Silcox, senior fellow
  1. 1National Center for Health Research, 1001 Connecticut Avenue NW, Washington, DC 20036, USA
  1. Correspondence to: D M Zuckerman dz{at}center4research.org

Using preliminary research to approve new treatments has high costs in morbidity and healthcare dollars, as Diana Zuckerman and colleagues outline

A controversial proposed law in the United States, the 21st Century Cures Act, is described by supporters as an innovative attempt to jump start the process of finding new cures for the thousands of diseases that currently lack effective treatments. But would this bill promote new cures, or increase the availability of new medical products that do not necessarily work? Alzheimer’s disease is often cited as an example of a devastating disease with enormous costs and no cure in sight. We examine the potential harms and costs that would have been incurred by three Alzheimer’s drugs that were rejected under current Food and Drug Administration approval standards but could have been approved under the standards promoted by 21st Century Cures and similar legislation.

What the new law would do

The 21st Century Cures Act would provide $8.7bn (£5.7bn; €8bn) to the US National Institutes of Health to encourage the development of new drugs. This has generated enthusiastic lobbying from universities and researchers. However, the act would also speed the availability of new drugs and devices by urging the FDA to replace large randomized clinical trials with smaller, shorter term trials and rely on surrogate endpoints rather than on meaningful health outcomes such as patients’ survival, health, and quality of life. It also encourages the FDA to rely more on animal studies, in vitro research, clinical experience, and case histories.

The European Medicines Agency and the FDA usually approve the same drugs; however, FDA standards for expedited approval are sometimes lower than EMA standards.1 Moreover, the UK, Canada, Germany, and many other countries require higher standards for health plan coverage than for regulatory approval.2 Since Medicare and US insurance companies usually pay for FDA …

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