President Obama Signs Cures Act Into Law
President Obama 12/13 signed into law the 21st Century Cures Act, which will provide a $500 million funding boost for FDA over the next 10 years. The new law also builds on the agency’s “ongoing efforts to advance medical product innovation and ensure that patients get access to treatments as quickly as possible, with continued assurance from high quality evidence that they are safe and effective,” FDA commissioner Robert Califf said shortly after the law’s signing. “Cures will greatly improve FDA’s ability to hire and retain scientific experts,” he said. “One of our ongoing challenges has been recruiting and retaining the experts we need in specialized areas to allow us to get our work done and meet our growing responsibilities. This is an especially important need given the tremendous advances in biological sciences, engineering, information technology and data science. Preventive, diagnostic and therapeutic strategies will become more complex with much greater potential for benefit and in some cases greater risk if used without adequate evidence to exclude risks that exceed potential benefits.”
Califf also noted that Cures will support FDA’s efforts to modernize and improve efficiency in clinical trial design. He said “exciting new approaches are now available, and we need to develop a common understanding of which designs should be used for which clinical issues. In cancer, for example, we’re already weighing the use of common control trials, which share a control arm, involve multiple different drugs for the same indication, and may even involve different companies. One of the benefits of using a common control arm is that the overall number of patients who need to be recruited and enrolled decreases, thereby optimizing clinical trial resources and potentially shortening the time it takes to get a new study off the ground.”
Overall, Cures provides $6.3 billion in additional funding to accelerate discovery, development, and delivery of new cures and treatments. It will provide $4.8 billion to the National Institutes of Health, including: $1.4 billion for President Obama’s Precision Medicine Initiative to drive research into the genetic, lifestyle and environmental variations of disease; $1.8 billion for Vice President Biden’s “Cancer Moonshot” to speed research; and $1.6 billion for the BRAIN initiative to improve the understanding of diseases like Alzheimer’s and speed diagnosis and treatment.
Califf also acknowledged the law’s Limited Population pathway that will help streamline the development programs for certain antibacterials and antifungals intended to treat targeted groups of patients suffering from serious or life-threatening infections where unmet need exists due to lack of available therapies. “Approvals of these antimicrobials are expected to rely on data primarily targeting these limited populations,” he said. “The statement ‘Limited Population’ will appear prominently next to the drug’s name in labeling, which will provide notice to healthcare providers that the drug is indicated for use in a limited and specific population of patients. The limited population statement, additional labeling statements describing the data, and FDA review of promotional materials, will help assure these drugs are used narrowly to treat these serious and life-threatening infections while additional evidence is generated to assess safety and effectiveness for broader use.”
Additionally, Califf said the law addresses how drug firms could provide healthcare economic information to payers and formulary committees. “This complex area will require careful delineation of principles to guide information exchange to enable these entities to appropriately assess the value of drugs,” he said.