Today marks an important day for America. It was just one year ago that the revolutionary 21st Century Cures Act was signed into law – a piece of legislation crafted to accelerate the discovery, development, and delivery of new cures and treatments. And while it’s not every day in Washington that Republicans and Democrats agree, the reality of providing patients with access to world-class medicines and cutting-edge technologies to combat life-threatening and debilitating disease came into sharp focus last December. This bigger-than-politics achievement has the potential to benefit current and future generations for decades to come, so let’s take a look at what we can expect from the Cures Act:
It was a big year for drug approvals – just look at the numbers. New data show that the Food and Drug Administration (FDA) approved 40 new drugs as of Nov. 30 – nearly double the total number from 2016 – and we’re likely to see this number grow over the next few years as more and more of the provisions included in 21st Century Cures, new PDUFA VI and BsUFA II are implemented.
Funding increase to support medical research will impact millions of lives. Included in the 21st Century Cures Act was a 10-year, $4.8 billion boost to the National Institutes of Health (NIH) to carry out initiatives like the “moonshot” against cancer, the BRAIN Initiative, and the Precision Medicine Initiative. By giving biomedical researchers the tools and resources they need, we’re on the brink of an exciting time for the development of new medicines and cures.
In addition, Cures will designate $500 million over 10 years to the FDA, giving the agency the ability to recruit and retain the best and brightest scientists, doctors, and engineers to help accelerate the discovery, development, and delivery of new cures and treatments. This funding will also be used to modernize clinical trials and study how safety and efficacy data is collected and analyzed.
The bill also included $1 billion allocated over two years for prevention and treatment activities in response to the opioid crisis. By bringing together the best minds in the public and private sectors, we have an opportunity to end this public health crisis for current and future generations.
Developing a patient-centric system. Since the passage of the Cures Act, the FDA has increased its efforts to build upon the PDUFA V’s commitment to incorporating the patient’s voice into the drug development process. Better known as Patient-Focused Drug Development, this unique and valuable initiative will allow FDA staff to better understand what improvements patients might benefit from most, and require these findings to inform regulatory decision making moving forward.
Incorporation of new data types in drug development and review. Often overlooked, the FDA is directed to develop a regulatory process to evaluate how Real-World Evidence can be used to support the approval of new indications for approved drugs or to support post-approval study requirements under the Cures Act. This framework may include what types of data that could be used and how, methods of study design and conduct, human subject protections, and methods of analysis.
We’re entering an exciting and important time for medical innovation. The next generation of medical breakthroughs including gene therapy, immunotherapy and RNAi therapy has arrived, and smart policy like 21st Century Cures will help America maintain its leadership in innovation.