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Witness appearing before the House Appropriations Subcommittee on Labor, HHS, Education, and Related Agencies

Francis S. Collins, M.D., Ph.D.
Director, 2023 ÃÛÑ¿´«Ã½

Good morning, Chairman Cole, Ranking Member DeLauro, and distinguished Members of the Subcommittee. I am Francis S. Collins, M.D., Ph.D., and I am the Director of the 2023 ÃÛÑ¿´«Ã½ (NIH). It is an honor to appear before you today to present the Administration’s fiscal year (FY) 2016 budget request for the NIH, and provide an overview of our central role in enhancing the nation’s health through scientific discovery.

Before I discuss our diverse investments in biomedical research and the exciting scientific opportunities on the horizon, I want to thank this Subcommittee for the recent FY 2015 Omnibus Appropriation bill. We are truly grateful for your leadership.

As the nation’s premier biomedical research agency, NIH’s mission is to seek fundamental knowledge about the nature and behavior of living systems, and to apply that knowledge to enhance human health, lengthen life, and reduce illness and disability. I can report to you today that NIH leadership, employees, and grantees continue to believe passionately in our mission.

NIH has been advancing the understanding of health and disease for more than a century, and many of our contributions stem from our nation’s commitment to investing in basic science research. Basic science lays the foundation for advances in disease diagnosis, treatment, and prevention by providing the building blocks for clinical applications. Basic science is generally not supported in the private sector, and NIH’s focus on understanding fundamental biological processes fosters innovation and ultimately leads to effective ways to treat complex medical conditions. Our successful investment in basic science is reflected by the awarding of no less than 145 Nobel prizes to NIH-supported scientists; the vast majority of these individuals were recognized for basic science advances.

A compelling current example of how basic science is revolutionizing the future of biomedical research is in the area of genome editing. The human genome is a three-billion letter instruction book, using the four-letter DNA alphabet. Just a single typo can lead to a devastating disease. Imagine if doctors could someday act like copy editors and quickly fix any spelling errors so that your genetic text reads flawlessly. A recent, groundbreaking discovery is the development of the genome editing tool, CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats. Despite its complicated name, the concept is rather simple—CRISPR technology acts like a precise molecular scalpel that can target genetic mutations in order to correct the genome. CRISPR was discovered by basic science investigators trying to understand how certain bacteria resist infection by viruses, and has already revolutionized genetic research in cells and animal models. Combining this approach with rapid developments in induced pluripotent stem (iPS) cell biology holds out the promise of powerful new individualized treatments for diseases like sickle cell anemia.

Another way we are trying to unravel life’s mysteries through basic science is with the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative—and we are grateful to this committee for its FY2014 and FY 2015 support. This bold, multi-agency effort to revolutionize our understanding of the human brain will enable the development and use of innovative technologies to produce a clearer, more dynamic picture of how individual cells and neural circuits interact in both time and space. By measuring activity at the scale of neural networks in living organisms, we can begin to decode sensory experience and, potentially, even memory, emotion, and thought. Ultimately, the technologies developed under the BRAIN Initiative may help reveal the underlying pathology in a vast array of brain disorders and provide new therapeutic avenues to treat, cure, and prevent neurological and psychiatric conditions such as Alzheimer’s disease, autism, schizophrenia, and addiction.

Scientific advances are also accelerating progress toward a new era of personalized medicine. Historically, physicians have had to make recommendations about disease prevention and treatment based on the expected response of the average patient. This one-size-fits-all approach works for some patients and some conditions, but not others. Technology developments, along with plummeting costs of DNA sequencing, now make it possible to develop an innovative approach to treatment that accounts for individual differences in patients’ genes, environments, and lifestyles. With this in mind, we are excited to take a lead role in the multi-agency Precision Medicine Initiative (PMI). A near term goal of the PMI focuses on cancer; cancer research has been leading the way in precision medicine for many years, by defining the driver mutations in individual tumors and using this information to design the ideal therapy for each patient. To accelerate the pace of discovery, this initiative seeks to expand current cancer genomics research to understand the development of resistance to targeted therapy, to apply non-invasive methods to track patients’ responses to treatment, and to explore the efficacy of new drug combinations targeted to specific tumor mutations.

As a longer term goal of this initiative, NIH will also launch a national research cohort of one million or more volunteers who will play an active role in how their genetic and environmental information is used for the prevention of illness and management of a wide array of chronic diseases. A project of this magnitude will lay the foundation for a myriad of new prevention strategies and novel therapeutics. There’s no better time than now to embark on this ambitious new enterprise to revolutionize medicine and generate the scientific evidence necessary to move this personal approach into everyday clinical practice.

Another way we are turning discovery into health is in our efforts to combat the problem of Antimicrobial Resistance (AMR). Because most bacteria, viruses, and other microbes multiply rapidly in a short period of time, they can evolve and develop resistance to antimicrobial drugs. Public health surveillance has documented an alarming increase in AMR, especially those strains that cause hospital-acquired infections. Each year in the United States, more than two million people acquire serious infections from bacteria that are resistant to antibiotics designed to treat those infections, and approximately 23,000 of these people die. As long as antibiotics are used indiscriminately to kill microbes in medicine and agriculture, resistance will continue to emerge. Recognizing the growing public health threat of AMR, NIH is engaged in a multi-agency venture, in collaboration with the U.S. Food and Drug Administration, the U.S. Department of Agriculture, and the Centers for Disease Control and Prevention, designed to reverse these disturbing trends. To this end, we will expand efforts to develop new antibiotics, like the teixobactin recently identified by an NIH grantee that shows great promise against the difficult-to-treat infection, Methicillin-resistant Staphylococcus aureus (MRSA) and resistant tuberculosis. We will work to develop a rapid diagnostic test for resistant organisms that will be of critical clinical and public health utility. We will also build a national genome sequence database on all reported resistant human infections. And we will create a rapid response clinical trial network that is ready to test new antibiotics on individuals infected with highly resistant strains of bacteria.

Preventing disease has always been a top priority for NIH, and influenza is one area of prevention in which we are poised for significant progress. Currently, to provide protection against the rapidly evolving influenza virus, a new vaccine must be produced each year. Despite our best efforts, some guesswork is involved, and the vaccine isn’t always ideal—as we know all too well after this particularly difficult flu season. In an average year, the flu claims up to 49,000 American lives and costs the U.S. economy about $87 billion. But it does not have to be that way. NIH-funded researchers continue to move forward on a universal flu vaccine—designed to produce broad protection against virtually all strains of the flu for extended periods of time and, thus, potentially reduce the need for annual flu shots and the risk of a global pandemic. I am happy to report that universal flu vaccine candidates have now moved into early stage clinical trials.

So far, I’ve described for you several examples of NIH’s commitment to basic, translational, and clinical research. But, none of this would be possible without a diverse and talented biomedical workforce. Recruiting and retaining the brightest minds regardless of race, ethnicity, sex, disability, and socioeconomic status is critically important not only to NIH, but to the entire American scientific enterprise. That is why we are taking new and aggressive steps to attract the most talented individuals from all groups. The Building Infrastructure Leading to Diversity (BUILD) Initiative is a bold experiment, consisting of a set of training awards designed to attract a diverse array of students into the training pipeline and encourage their futures as NIH-supported researchers. In addition to BUILD, we also established the National Research Mentoring Network (NRMN). The network establishes a nationwide, interconnected set of skilled mentors linked to mentees from a variety of scientific and social backgrounds. These efforts should strongly support our goal to enhance the diversity and maximize the talent of the biomedical workforce.

While all of these exciting research efforts and scientific opportunities are leading to a much deeper understanding of health and human disease, much more work needs to be done. To this end, the President’s FY 2016 budget request for the NIH is $31.311 billion, $1 billion or 3.3 percent above the enacted FY 2015 level. This budget request reflects the President’s and the Secretary’s commitment to improving the health of the nation and to maintaining our nation’s leadership in the life sciences. The request highlights investments in innovative research that will advance fundamental knowledge and speed the development of new therapies, diagnostics, and preventive measures to improve public health, including $200 million for the Precision Medicine Initiative and an additional $100 million to combat AMR.

The FY 2016 budget request will enhance NIH’s ability to support cutting-edge research and training of the scientific workforce. Within this budget, we will increase Research Project Grants (RPGs), NIH’s funding mechanism for investigator-initiated research. NIH expects to support 10,303 new and competing RPGs in FY 2016, an increase of 1,227 above the FY 2015 estimate. The budget request allocates resources to areas of the most extraordinary promise for biomedical research, while maintaining the flexibility to pursue unplanned scientific opportunities and address unforeseen health needs.

I have provided you with examples of how investments in biomedical research through NIH are advancing human health, spurring innovations in science and technology, and laying the groundwork for the future of the United States biomedical research enterprise. We have never witnessed a time of greater promise for advances in medicine than right now. With your support, the future of medicine can be very bright.

This concludes my testimony, and I look forward to answering your questions.