Author + information
- Christopher M. O’Connor, MD, FACC, Editor-in-Chief, JACC: Heart Failure∗ ()
- ↵∗Address for correspondence:
Dr. Christopher M. O’Connor, Editor-in-Chief, JACC: Heart Failure, American College of Cardiology, Heart House, 2400 N Street NW, Washington, DC 20037.
The United States has been a leader in the discovery, development, and delivery of new therapies and cures in the biomedical field, particularly in the area of heart failure. However, this lead has slipped to our competitors across the globe, and we are losing our edge in developing new therapeutic approaches for patients with this condition. The recently passed 21st Century Cures Act is largely devoted to promoting discovery, development, and delivery, with an emphasis on research and innovation (1). One of the objectives includes increased collaboration between the National Institutes of Health, Food and Drug Administration, and other agencies along with their scientific partners in academics and industry. This initiative will ensure that the mission of our government agencies keeps pace with scientific discovery.
With respect to discovery, the National Institutes of Health, academic medical centers, and the industry have led the way over the past several decades. However, there is considerable competition. The Human Genome Project, which was completed over a decade ago in the United States, has now been surpassed by the Beijing Genomics Institute, which is now the world's largest genetic research center. This institute is leading to new opportunities for diagnostic and therapeutic advantages in patients with heart failure because of the proactive initiatives from the government. In fact, more biotechnology companies have been developed in China than in the United States over the past 5 years. This is in part because of the less restrictive regulatory policies and government investments.
The 21st Century Cures Act includes new ways to use real-world data to help accelerate heart failure and other therapies. Currently the randomized controlled clinical trial is too costly, lengthy, and inefficient, with an extreme burden to sponsors with high rates of failure. How can we do better? Can we use registry trials, smaller trials, surrogate endpoints with post-marketing approval and surveillance, as well as confirmatory trials after approval to accelerate therapies for heart failure?
The 21st Century Cures Act includes provisions for using real-world evidence, surrogate endpoints, biomarker, genomics, and patient-reported outcomes. There will be an opportunities to develop a new generation of targeted heart failure therapies, in addition to the important mission of utilizing endpoints that matter to patients. This initiative will help accelerate scientific and medical advances to improve the care of our patients with heart failure.
In conclusion, the 21st Century Cures Act has achieved broad bipartisan support from Congress, clinicians, academicians, health system, and government agencies to tackle some of our most important issues in medicine, particularly chronic diseases such as heart failure, with a goal of bringing our patients the care they deserve.
- American College of Cardiology Foundation
- ↵114th Congress (2015–2016). H.R.6—21st Century Cures Act. Available at: https://www.congress.gov/bill/114th-congress/house-bill/6. Accessed December 29, 2016.