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A 2016 US law that funded the Cancer Moonshot, created the RMAT designation for cell and gene therapies, told the FDA to work out how real-world data could support approvals, and made companies publish how patients can request unapproved drugs.
United States, federal statute. The 21st Century Cures Act was signed on 13 December 2016 (Public Law 114-255). Primary text: the Congress.gov record of H.R.34.
What it changed: it authorised 1.8 billion dollars over seven years for the Cancer Moonshot through the National Cancer Institute; created the regenerative medicine advanced therapy (RMAT) designation, which brought breakthrough-style benefits to CAR-T and other cell and gene therapies; required the FDA to publish a framework for using real-world evidence to support new indications and post-approval requirements (published December 2018); allowed sponsors to submit summary-level data for supplemental indications; and required manufacturers of investigational drugs for serious conditions to post their expanded access policies publicly. It also funded the NIH, reformed mental health programmes and, in its health IT title, banned information blocking and mandated patient access to electronic records through application interfaces.
The arguments: supporters call it the biggest biomedical research law in a decade; critics, including several former FDA officials, argued that pushing real-world and summary data toward approvals risked lowering the evidence bar, and that the funding was appropriated only in part. Its information-blocking rules are why patients in the United States can now pull their own oncology records into apps.
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