The Yale Collaboration for Research Integrity and Transparency has just released French, Spanish, and Portuguese versions of What's in Your Medicine Cabinet? Ensuring the Safety and Efficacy of Prescription Drugs, Biologics and Medical Devices in the United States, now in its third edition.
We are excited by the opportunity to participate in a larger, international discussion about improving drug safety and efficacy worldwide. Although our paper focuses on regulation of medical products by the U.S. Food and Drug Administration (FDA), medical product regulation in the U.S. is part of an interconnected global regulatory framework. The U.S. market share of the global medical product market is larger than that of any other nation, giving outsize influence to the decisions of U.S. regulators.
Our paper examines challenges currently facing the U.S. Food and Drug Administration, and proposes solutions in seven areas:
- The current expedited framework for pre-market regulation of new medical products and the potential for new products to be approved without adequate scrutiny to establish their safety and efficacy;
- The problems with post-market follow-up of medical products where required additional studies to confirm the safety and clinical efficacy of new products are not completed or completed in a timely manner;
- The difficulty in getting manufacturers to participate in expanded access programs for pre-approval access to experimental therapies;
- The problems with using less rigorous forms of real-world evidence to make key regulatory decisions;
- The use of the expansion of corporate speech rights by the courts to expand off-label marketing under a standard of “truthful and non-misleading” outside of any evaluation of the off-label indication being safe and effective by the FDA;
- The substantially weaker pre-market and post-market evaluation and surveillance for medical devices, and;
- The need for the FDA and other federal agencies to take further proactive measures to enhance access to clinical trial data, including patient level data, summary level data and meta-data.
We address concerns about a growing erosion in the evidence base supporting the use of new drugs, devices and biologics, in the context of political pressure to further relax evidentiary standards for these products’ approval. The need for improvement to medical product regulation worldwide was a key topic of discussion at our conference, held in June 2017. Read more blog posts from the conference.