Scientific Rationale

Safety is a critical component of medical product development and approval. In particular, cardiac safety has received intense focus with several high-profile issues, including market withdrawals. This has stimulated debate over the best way to measure efficacy while ensuring the protection of the public health. At the center of this debate is the balance between efficacy and safety. Importantly, rapid advances in therapeutic medical product development have yet to reach full potential, in part due to limits in our ability to understand and quantitate therapeutic balance.

Unfortunately the evaluative sciences surrounding medical-product approval have not kept pace with our fundamental understanding of disease. Societal investment in research and development to improve the approval process has been lacking in contrast to the large investment, both private and public, in basic research and specific product advances. To improve the ability of regulatory agencies to promote and protect the public health, there needs to be a fundamental investment in the evaluative sciences. Specifically, the scientific basis of our understanding of medical-product safety needs to be enhanced and integrated with refined measurements of efficacy. An investment in the evaluative sciences will provide more precise, scientifically-based information, enabling healthcare professionals and patients to appropriately assess risk and benefits.

The FDA’s Critical Path Initiative (CPI) has been developed address many of these issues by establishing greater collaboration among regulators, academics, physicians, and scientists from industry. The spirit of this collaboration intends to create opportunities by sharing existing knowledge and data that will facilitate the process of enhancing, refining, and ultimately improving the process used to evaluate new medical products. A central tenet to CPI is focusing on the evaluative science of the approval process, including both efficacy and safety measures.

The Cardiac Safety Research Consortium (CSRC) is a first step in bringing together key constituencies to focus on cardiac safety issues during the new medical product development process. Utilizing the principals of CPI, the CSRC will focus on improving the evaluative sciences, specifically in relation to cardiac safety.