Goal 4: Foster Sound, Sustained Advances in the Sciences
HHS is proud to be home not just to many of the world’s preeminent individual scientists, public health experts, and clinicians, but also the world’s finest scientific institutions: the largest single source of biomedical research funding in the world in NIH, the world’s premier epidemiological institution in CDC, and the world’s gold-standard food and drug safety agency in FDA.
In 2018, work continued to maximize the impact these institutions have on improving the health and well-being of Americans and the entire world, while various efforts were undertaken to improve the underlying infrastructure of our research, approval, and public health work.
In this page:
- Accelerating Biomedical Innovation
- Harnessing Real World Evidence
- Making Strides in the War on Cancer
- Prioritizing Other Breakthrough Research Areas at NIH
Accelerating Biomedical Innovation
Speeding innovation for military medical needs: In 2018, FDA established a framework for enhanced collaboration with the Department of Defense (DoD) to expedite the FDA’s review of products to diagnose, treat, or prevent serious or life-threatening diseases or conditions facing American military personnel. In support of this partnership, FDA:
- Granted an Emergency Use Authorization for a freeze-dried plasma requested by DoD, and issued a draft guidance on the development of dried plasma products intended for transfusion
- Approved, six months ahead of DoD’s product development schedule, an auto-injector device as a medical countermeasure for chemical nerve agent exposure.
- Partnered with DoD on regulatory science research to help speed potential countermeasures for Ebola and other viruses.
- Signed an MOU with DoD setting forth the framework for the ongoing partnership and the creation of a robust program that can better serve the health care needs of American military personnel
FDA and WHO working together to expedite review of HIV treatments: FDA announced a plan to work with the World Health Organization (WHO) to pilot a process to share documents on HIV drug applications that have been approved or tentatively approved by the agency under the U.S. President’s Emergency Plan for AIDS Relief. In this initial pilot, the FDA will, with the applicants’ permission, provide the WHO with minimally-redacted reviews of one or two HIV drug applications, which can then be used to produce review dossiers to be shared with regulators in resource limited countries to speed up their own regulatory review processes.
Working toward international regulatory harmonization: FDA expanded the United States’ ability to use drug inspections conducted by competent European authorities by completing capability assessments for 15 European Union (EU) regulatory authorities, which are now recognized as capable authorities under the Mutual Recognition Agreement between the FDA and EU that went into effect in November 2017. FDA also promoted future access to safe, effective and affordable generic drugs by securing the International Council on Harmonization’s endorsement of FDA’s proposal for future harmonization of scientific and technical standards for generic drugs.
Paving the way for a digital-health future: FDA made significant strides toward implementing its Digital Health Innovation Plan, which is focused on fostering innovation at the intersection of medicine and digital health technology. For instance, FDA issued three significant draft guidances to implement the 21st Century Cures Act, including clarifying the regulatory status of products that contain some functions fall outside the scope of FDA regulation, but others that do not.
Advancing science in outer space: On HHS’s behalf, Deputy Secretary Hargan signed an interagency agreement with NASA, covering cooperation on scientific research that would benefit humanity on Earth and on individuals traveling to the Moon and beyond. NASA and various HHS agencies have mutual interests in exploring autonomous portable medical capabilities for remote locations and field medical care, long shelf life and manufacturing of pharmaceuticals, and improved tools to deal with isolation and confinement. The National Institutes of Health already had its own agreement with NASA, but this new interagency agreement is an umbrella arrangement designed to cover the entire Department of Health and Human Services.
Harnessing Real World Evidence
Building real-world evidence through partnerships: In collaboration with FDA, the National Evaluation System for health Technology (NEST) Coordinating Center established agreements with twelve data partners representing over 195 hospitals, nearly 4,000 clinics and over 495 million patient records. Soon, NEST will launch pilot programs testing the use of medical records, patient registries and claims data to evaluate the safety and effectiveness of devices in many clinical areas and throughout the medical device total product life cycle. Both FDA and CMS serve on the NEST Governing Committee, as improved data collection through NEST can also help establish the evidence needed for CMS national coverage determinations.
Using real-world evidence in regulatory decisions: FDA developed guidance for the use of electronic health records and claims data as Real World Evidence to support regulatory decision making and held multiple public meetings and workshops to discuss how regulatory decision making can adapt to permit the use of complex innovative designs—such as simulations or trials without placebos—in clinical trials of drugs and biological products.
Launching the All of Us research program: In May 2018, NIH launched the All of Us Research Program, an effort to engage 1 million U.S. research volunteers to build a national resource that accelerates health research and individualized healthcare. To date, over 150,000 individuals initiated sign up to join the program, with over 64,000 of these individuals already contributing data as participants. The size of the program will enable researchers to derive insights especially for populations that are traditionally underrepresented in biomedical research.
Expanding the evidence base to increase organ donation: HRSA established a pilot registry for living organ donors tracking the long-term outcomes of living donations, to allow HRSA and NIH an opportunity to study the long-term implications of living donations.
Making Strides in the War on Cancer
NIH and VA collaborate to boost veterans’ access to cancer clinical trials: Veterans with cancer who receive treatment from the VA will now have easier access to clinical trials of novel cancer treatments, thanks to an agreement between VA and the National Cancer Institute (NCI). The NCI and VA Interagency Group to Accelerate Trials Enrollment, or NAVIGATE, which is launching at 12 VA facilities across the country, will enhance the ability of veterans to participate in trials carried out through NCI’s National Clinical Trials Network (NCTN) and the NCI Community Oncology Research Program.
Advancing the Beau Biden Cancer Moonshot: The $300 million appropriated for the Beau Biden Cancer Moonshot in FY 2018 has been invested in research focused on speeding progress against cancer in children and adults though advances in immunotherapy and the understanding of drug resistance. The moonshot efforts were enhanced by the FY 2018 launch of NIH’s Partnership for Accelerating Cancer Therapies, a five-year public-private collaboration between NIH and 12 biopharmaceutical companies to develop biomarkers to advance cancer immunotherapy.
A major initiative on drivers of cancer in black men: NIH and the Prostate Cancer Foundation launched a large study on aggressive prostate cancer in black men, the largest coordinated research effort to study biological and non-biological factors associated with this issue. The $26.5 million study, called RESPOND, or Research on Prostate Cancer in Men of African Ancestry: Defining the Roles of Genetics, Tumor Markers, and Social Stress, will investigate environmental and genetic factors related to the aggressiveness of prostate cancer in African-American men to better understand why they disproportionally experience aggressive forms of the disease compared with men of other racial and ethnic groups.
New discoveries expanding our understanding of cancer: Scientific discoveries in 2018 around cancer that were supported by NIH funding include:
- A combination of two drugs was found in an interim analysis of a Phase 3 clinical trial to be superior to the standard treatment for some patients with chronic leukemia.
- A novel approach to immunotherapy developed by researchers at NCI has led to the complete regression of breast cancer in a patient who was unresponsive to all other treatments.
- Findings from the groundbreaking Trial Assigning Individualized Options for Treatment (“TAILORx trial”) showed no benefit from chemotherapy, as opposed to hormone therapy alone, for 70 percent of women with the most common type of breast cancer.
- Interim results from a randomized clinical trial for patients with certain rare sarcomas show that a drug extended progression-free survival compared with a placebo.
Prioritizing Other Breakthrough Research Areas at NIH
The next steps in regenerative medicine: In 2018, FDA continued to make strides in advancing the field of regenerative medicine. The field of regenerative medicine may allow engineered cells and tissues to grow healthy, functional organs to replace diseased ones; allow genes to be introduced into the body to combat disease; and allow adult stem cells to generate replacements for cells that are lost to injury or disease.
To support the growth of this field, FDA issued numerous draft and final guidance documents addressing topics such as risk-based compliance and enforcement priorities regarding the premarket approval requirements for human cell and tissue-based products, as well as expedited programs for regenerative medicine therapies for serious conditions. At NIH, therapeutic strategies supported by $10 million FY 2018 funding include cell delivery for restoring the structure and function of damaged cells and tissues and enhancing the body’s own innate healing capacity.
Revolutionizing our understanding of the human brain: NIH moved forward with its inter-agency BRAIN Initiative, which seeks to develop technologies that will revolutionize understanding of the human brain in health and disease, in part by developing a dynamic map of individual cells and complex neural circuits to understand how they interact and what happens in brain diseases.
Specific goals for NIH contributions to the initiative were formulated in “BRAIN 2025: A Scientific Vision,” a 12-year roadmap. NIH used the $86 million in FY 2018 that was authorized by the 21st Century Cures Act to support, among other initiatives, the BRAIN Initiative Cell Census Network, to catalog brain cell types across species, and an interdisciplinary and integrative BRAIN Circuit Programs to link neural circuit activity to behavior. Continuing to build on early research successes, NIH also announced more than 200 new BRAIN awards totaling over $220 million.
Researching sickle cell disease: NIH’s National Heart, Lung, and Blood Institute launched the Cure Sickle Cell Initiative, a collaborative effort to accelerate the development of genetic therapies to cure the disease, which affects 100,000 Americans, disproportionately impacting African-Americans.
Researching the progression of Parkinson’s: As part of the Accelerating Medicines Partnership (AMP), NIH and partners launched AMP Parkinson’s Disease, to focus on identifying and validating promising markers of the disease to track progression and eventually develop biological targets for discovery of new drugs.
Seeking a better understanding of Down Syndrome: NIH launched the INCLUDE (Investigation of Co-Occurring Conditions Across the Lifespan to Understand Down Syndrome) project, dedicating $22.2 million in additional funding to raise NIH’s total Down Syndrome research funding to approximately $59 million for FY 2018.