Regulation of precision medicine is rapidly evolving. The pre-existing regulatory framework did not contemplate many of the products and technologies which are becoming available with precision medicine. FDA and other federal agencies have responded by attempting to develop a “flexible and adaptive” approach to regulation which simultaneously encourages innovation while still assuring that new precision medicine drugs and medical devices are safe and effective.
FDA has issued a series of non-binding “Guidance Documents” addressing different topics in precision medicine. While non-binding, these guidance documents explain the Agency’s current thinking on specific issues and should be viewed as recommendations.
FDA in 2017 issued a Guidance Document relating to the use of “Real World Evidence” (RWE) to support regulatory decisions involving medical devices. RWE represents a potentially faster, less expensive and more useful alternative to traditional randomized clinical trials (RCT’s) for generating data to support regulatory approvals of drugs and devices. RWE consists of clinical evidence of a product's usage, benefits and risks obtained from an analysis of real-world data sources, such as patient EHR’s, insurance claims data and disease or device registries. In 2017, FDA approved a new indication for a medical device based entirely on RWE.
FDA has recently issued Guidance Documents and draft guidances covering many other aspects of precision medicine, including NGS–based tests, genetics variant databases and companion diagnostics. Additionally, FDA and CMS have developed a parallel review process for devices aimed at allowing developers to concurrently obtain both marketing approval and eligibility for Medicare coverage.
Many of FDA regulatory guidances have issued within the last 12 months. It is likely that regulation of precision medicine will continue its evolution for the foreseeable future.