Goal 2. Objective C: Advance the regulatory sciences to enhance food safety, improve medical product development, and support tobacco regulation
Regulatory science is the development and use of scientific tools, standards, and approaches necessary for the assessment of products including medical products and foods to determine safety, quality, and performance. Without advances in regulatory science, promising therapies may be discarded during the development process simply for the lack of tools to recognize their potential; moreover, outmoded review methods can delay approval of critical treatments. Advancements in regulatory science will help to prevent foodborne illnesses, and when outbreaks of foodborne illness occur, to identify the source of contamination quickly and to limit the impact of the outbreak. Regulatory science innovations will allow for faster access to new medical technologies that treat serious illnesses and improve quality of life. These advances will benefit every American by increasing the accuracy and efficiency of regulatory review and by reducing adverse health events, drug development costs, and the time-to-market for new medical technologies.
Advancing regulatory science and innovation is an objective shared by a number of agencies within HHS. FDA and NIH are collaborating on an initiative to fast-track medical innovation to the public. Below are several performance measures that are indicative of the types of achievements that HHS and its components expect to achieve related to improving regulatory science and food and medical product safety. The Office of the Secretary led this Objective’s assessment as a part of the Strategic Review.
Objective 2.C Table of Related Performance Measures
The average number of days to serotype priority pathogens in food (Screening Only). (Lead Agency - FDA; Measure ID - 214306)
|FY 2012||FY 2013||FY 2014||FY 2015||FY 2016||FY 2017|
|Target||6.0 working days||5.0 working days||4.0 working days||4.0 working days||3.0 working days||3.0 working days|
|Result||6.0 working days||5.0 working days||4.0 working days||3.0 working days||Dec 31, 2016||Dec 31, 2017|
|Status||Target Met||Target Met||Target Met||Target Exceeded||Pending||Pending|
Complete review and action on original New Animal Drug Applications (NADAs) and reactivations of such application received during the fiscal year. (Lead Agency - FDA; Measure ID - 243201)
|FY 2012||FY 2013||FY 2014||FY 2015||FY 2016||FY 2017|
|Target||90% w/in 180 days||90% w/in 180 days||90% w/in 180 days||90% w/in 180 days||90% w/in 180 days||90% w/in 180 days|
|Result||100% w/in 180 days||99.8% w/in 180 days||98.3% w/in 180 days||Jan 31, 2017||Jan 31, 2018||Jan 31, 2019|
|Status||Target Exceeded||Target Exceeded||Target Exceeded||In Progress||In Progress||In Progress|
Develop biomarkers to assist in characterizing an individual's genetic profile in order to minimize adverse events and maximize therapeutic care. (Lead Agency - FDA; Measure ID - 262401)
|FY 2012||FY 2013||FY 2014||FY 2015||FY 2016||FY 2017|
|Target||1) Develop analytical methods to assess drug-induced heart damage
2) Identify target genes for obesity and the consequent development of metabolic syndrome diseases and heart disease
|1) Analyze urine, blood , and tumor tissues samples to identity biomarkers that will facilitate early detection in new cases and in the reemergence of pancreatic cancer.
2) Develop a new targeted therapeutic approach to improve clinical management of breast cancer.
|Determine if some drugs cause a higher incidence of liver toxicity in women than men||1) Complete pilot project that will promote women’s health by facilitating the development of personalized approaches to treat breast cancer
2) Evaluate serum metabolic biomarkers to determine whether they are correlated to acute kidney illness diagnosis and prognosis
|Identify potentially predictable drug/drug receptor combinations that can cause rare and unpredictable side effects||Complete initial phase of research to Identify drugs that have differential toxicological effects depending on age and/or sex of an individual in an effort to develop a bioinformatics-based safety assessment|
|Result||1) A model of drug-induced heart damage was developed and is being used to identify new predictive biomarkers of early stages of drug-induced cardiac tissue injury. (Target Met)
2) Research experiments have been completed and preliminary results suggest the involvement of a number of genes involved in lipid metabolism and sugar transporters. (Target Met)
|Published results that found potential for new breast cancer therapy using epigenetic approach (Target Met)||Researchers found that 18 out of 30 previously identified drug transporter genes exhibited sex differences in normal kidney tissue. Ethnicity and age also influenced gene expression levels in normal kidney tissue.||Research generated a mutational profile of Triple Negative Breast cancer to aid in personalized medicine approaches to treat breast cancer.
Initial evaluation of biomarkers completed via Nuclear Magnetic Resonance
|Dec 31, 2017||Dec 31, 2018|
|Status||Target Met||Target Met||Target Met||Target Met||In Progress||In Progress|
Analysis of Results
HHS supports an extensive set of efforts to protect and promote food and medical product safety. FDA Foods Program scientists are evaluating and integrating commercially available instrumentation into its microbiological testing workflow that is vastly improving the ability of FDA to more quickly and effectively detect and characterize foodborne pathogens such as Salmonella directly from the food supply. Improvements in sample throughput, along with the high degree of sensitivity and specificity built into new pathogen detection technologies, will dramatically improve FDA’s foodborne response and traceback capabilities. When fully deployed, technologies such as next-generation whole-genome sequencing (WGS) and others will reduce the time to conduct these analyses from 14 days originally to just a few days. One updated technology which provides highly accurate and rapid Salmonella serotype results for FDA, known as the flow cytometry/fluorescence platform, has been validated extensively and is now deployed in nearly all FDA field laboratories, as well as in CFSAN and CVM laboratories. In FY 2015, FDA exceeded the target of four working days, reducing the average number of days to serotype priority pathogens in foods to three working days, which is the minimum amount of time required.
The Animal Drug User Fee Act (ADUFA) helps FDA ensure that new animal drug products are safe and effective for animals as well as for the public with respect to animals intended for food consumption. FDA pursues a comprehensive set of review performance goals and commitments that seek to improve the timeliness and predictability of the review of new animal drug applications (NADAs). In FY 2014 FDA exceeded its ADUFA performance goal for the twelfth year in a row, completing review and action on 98.3 percent of original NADAs within 180 days.
The National Center for Toxicological Research’s goal is to define the correlations between an individual’s nutrition, genetic profile, health, and susceptibility to chronic disease in support of personalized nutrition and health. This research will provide baseline data that supports the FDA goal of providing consumers clear and timely information to help promote personalized nutrition and health. Identifying biomarkers of health, susceptibility to chronic disease, and gene-micronutrient interactions is essential to gaining a more complete scientific understanding of health. NCTR is implementing a novel research program for personalized nutrition and health that relies on the “challenge homeostasis” concept for identifying markers of health and susceptibility. Since 2008, FDA/NCTR and USDA/ARS have had an ongoing partnership with a community development center in the Mississippi Delta region of Arkansas to conduct community-based participatory research (CBPR) that studies the effects of dietary intake and its influence on the development of obesity-associated diseases. This ongoing collaboration analyzes dietary intake patterns, micronutrient levels in the blood samples of children and adults, and calories expended. In FY 2015, the FDA met its goal when 1) research generated a mutational profile of Triple Negative Breast cancer to aid in personalized medicine approaches to treat breast cancer; and 2) the initial evaluation of biomarkers was completed via Nuclear Magnetic Resonance.
Plans for the Future
In the area of food safety, HHS established a method development, validation, and implementation program for FDA and state laboratories to ensure the highest standards of analytical laboratory practices needed to support outbreak, compliance and surveillance testing; launched the 2014 FDA Food Safety Challenge, a prize competition to advance breakthroughs in foodborne pathogen detection, specifically with the goal of accelerating the detection of Salmonella in fresh produce; and in collaboration among FDA, CDC, USDA, and academic institutions, HHS created a network of 18 state and federal laboratories equipped with desktop DNA sequencers and expert staff to collect genomic data from foodborne pathogens, greatly enhancing the ability to rapidly and precisely identify patterns and isolate sources of foodborne illness.
FY 2014 Strategic Review Objective Progress Update Summary
Please note that this section summarizes the result of the FY 2014 HHS Strategic Review process, limiting the scope of content to that available prior to spring of 2015. Due to this constraint, the following may not be the most current information available.
Analysis: HHS made progress in advancing regulatory science in 2014 across a broad spectrum of food safety, medical product development, tobacco regulation responsibilities. In the area of food safety, HHS established a method development, validation, and implementation program for state laboratories to ensure the highest standards of analytical laboratory practices needed to support outbreak, compliance, and surveillance testing. The Food Safety Challenge was launched in 2014 which was a prize competition to advance breakthrough in foodborne pathogen detection, with the goal of accelerating the detection of Salmonella in fresh produce. HHS created a network of 18 state and federal laboratories equipped with desktop DNA sequencers and expert staff to collect genomic data from foodborne pathogens, greatly enhancing the ability to rapidly and precisely identify patterns and isolate sources of foodborne illness.
The medical product pre-market programs are consistently meeting or exceeding their performance goals for assuring timely access to safe and effective products. More than 30 guidance documents on topics such as Biosimilarity; expedited programs for rare diseases and serious conditions; cellular and gene therapy were published. HHS responded to the Ebola Virus in a variety of ways including rapidly evaluating investigational new drug applications, enabling access to investigational products for Ebola under appropriate regulatory mechanisms, and monitoring for fraudulent products that claim to prevent or treat Ebola.
One focus area for the Tobacco Regulatory Science Program included research on the role and impact of flavors in cigarettes, cigars, e-cigarettes and smokeless tobacco. “Tobacco Use among Middle and High School Students – United States, 2011-2014” was published in Morbidity and Mortality Weekly. The article highlighted the alarming trend of e-cigarette use among high school and middle school students which increased from 4.5 percent in 2013 to 13.4 percent in 2014.
HHS will continue to investigate the impact of e-cigarette use. It is a challenge to provide scientific evidence needed to inform tobacco regulation by conducting research at a pace that can keep up with a rapidly changing tobacco product market. Recruiting and retaining a talented scientific workforce and maintaining and modernizing aging scientific facilities, field laboratories and equipment are essential to supporting such research. The Department will perform manufacturing, production, and supply chain oversight using enhanced hand-held instrumentation, remote sensing, and well as implementation of prevention controls regulation and quality system standards. In addition, collaboration on studies that address e-cigarettes use will include measuring harmful and potentially harmful constituents in e-cigarettes vapor and e-juice; addictive compound in e-cigarette vapor; and biomarkers of these harmful addictive constituents in blood and urine of users. The Department will also explore methods to improve our measures of regulatory science progress and outcomes.