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At least 649 records · Page 36

Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Abstract Background The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children. Prior to requesting a voucher, applicants can request RPD designation, which confirms their product treats or prevents a rare disease in which the serious manifestations primarily affect children. This study describes the trends and characteristics of these designations. Details of RPD designations are not publicly disclosable; this research represents the first analysis of the RPD designation component of the program. Results We used an internal US Food and Drug Administration database to analyze all RPD designations between 2013 and 2022. Multiple characteristics were analyzed, including the diseases targeted by RPD designation, whether the product targeted a neonatal disease, product type (drug/biologic), and the level of evidence (preclinical/clinical) to support designation. There were 569 RPD designations during the study period. The top therapeutic areas were neurology (26%, n = 149), metabolism (23%, n = 131), oncology (18%, n = 105). The top diseases targeted by RPD designation were Duchenne muscular dystrophy, neuroblastoma, and sickle cell disease. Neonatology products represented 6% (n = 33), over half were for drug products and 38% were supported by clinical data. Conclusions The RPD PRV program was created to encourage development of new products for children. The results of this study establish that a wide range of diseases have seen development—from rare pediatric cancers to rare genetic disorders. Continued support of product development for children with rare diseases is needed to find treatments for all children with unmet needs.

Mease, Catherine (ORCID:000900050156198X)↗

COVID-19-associated school closures and related efforts to sustain education and subsidized meal programs, United States, February 18-June 30, 2020

Pre-emptive school closures are frontline community mitigation measures recommended by the US Centers for Disease Control and Prevention (CDC) for implementation during severe pandemics. This study describes the spatiotemporal patterns of publicly announced school closures implemented in response to the coronavirus disease 2019 (COVID-19) pandemic and assesses how public K-12 districts adjusted their methods of education delivery and provision of subsidized meals. During February 18–June 30, 2020, we used daily systematic media searches to identify publicly announced COVID-19–related school closures lasting ≥1 day in the United States (US). We also collected statewide school closure policies from state government websites. Data on distance learning and subsidized meal programs were collected from a stratified sample of 600 school districts. The first COVID-19–associated school closure occurred on February 27, 2020 in Washington state. By March 30, 2020, all but one US public school districts were closed, representing the first-ever nearly synchronous nationwide closure of public K-12 schools in the US. Approximately 100,000 public schools were closed for ≥8 weeks because of COVID-19, affecting >50 million K-12 students. Of 600 districts sampled, the vast majority offered distance learning (91.0%) and continued provision of subsidized meal programs (78.8%) during the closures. Despite the sudden and prolonged nature of COVID-19–associated school closures, schools demonstrated flexibility by implementing distance learning and alternate methods to continue subsidized meal programs.

60 APPLIED LIFE SCIENCES↗

Launching a saliva-based SARS-CoV-2 surveillance testing program on a university campus

Regular surveillance testing of asymptomatic individuals for SARS-CoV-2 has been center to SARS-CoV-2 outbreak prevention on college and university campuses. Here we describe the voluntary saliva testing program instituted at the University of California, Berkeley during an early period of the SARS-CoV-2 pandemic in 2020. The program was administered as a research study ahead of clinical implementation, enabling us to launch surveillance testing while continuing to optimize the assay. Results of both the testing protocol itself and the study participants’ experience show how the program succeeded in providing routine, robust testing capable of contributing to outbreak prevention within a campus community and offer strategies for encouraging participation and a sense of civic responsibility.

59 BASIC BIOLOGICAL SCIENCES↗

2021–2022 Can Do Colorado E-Bike Full-Scale Pilot Program Study

In 2021–2022, the Colorado Energy Office conducted a full-scale pilot program study on e-bike usage as part of the Can Do Colorado initiative, providing e-bikes to low-income participants across the state. A [2020 mini pilot program study](https://www.nlr.gov/transportation/secure-transportation-data/tsdc-2020-can-do-colorado-e-bike-pilot-program.html) informed the full-scale study. Both studies used pedal-assist e-bikes, which feature an electric motor and battery to help power the bike. The motor amplifies the power behind each pedal stroke, augmenting the energy you put into the bike. #### Data Collection Agency The Colorado Energy Office conducted the study in partnership with local organizations in Adams and Broomfield counties (Smart Commute Metro North), Boulder (Community Cycles), Durango (Four Corners Office for Resource Efficiency), Fort Collins (City of Fort Collins), Pueblo (Pueblo County), and Vail (Town of Vail). #### Survey Methodology Program participants received an e-bike and accessories at no cost and manually submitted travel data and feedback via the CanBikeCO smartphone app. Developed in partnership with NLR, the app used a customized version of the open-source [NLR OpenPATH platform](https://www.nlr.gov/transportation/openpath.html). #### Survey Records and Data Survey records include 170 participants. The six datasets contain up to 18 months of partially automated travel diaries, combining sensed and surveyed travel behavior data—patterns of multimodal, end-to-end, individual human mobility—as well as demographic information from participants. The number of e-bike trips and e-bike miles traveled per location are 1,560 and 4,179 for Adams and Broomfield counties; 8,481 and 27,000 for Boulder; 2,815 and 6,307 for Durango; 3,483 and 7,080 for Fort Collins; 4,022 and 14,887 for Pueblo, and 1,206 and 3,3361 for Vail.

32 ENERGY CONSERVATION, CONSUMPTION, AND UTILIZATI↗

Fermilab Superconducting Nb₃Sn High Field Magnet R&D Program

Magnets based on the modern Nb₃Sn conductor are the main candidates for future high-energy hadron colliders. Fermilab as part of the U.S. MDP executes an extensive R&D program on these high-field magnets. This program includes basic conductor and material R&D, quench per-formance studies, and building a meter-long high-field demonstrator. This paper summarizes the current status of the program including its recent results.

43 PARTICLE ACCELERATORS↗

The LCLS-II HE High Q and Gradient R&D Program

The LCLS-II HE project is a high energy upgrade to the superconducting LCLS-II linac. It consists of adding twenty additional 1.3 GHz cryomodules to the linac, with cavities operating at a gradient of 20.8 MV/m with a Q₀ of 2.7·10¹⁰. Performance of LCLS-II cryomodules has suggested that operations at this high of a gradient will not be achievable with the existing cavity recipe employed. Therefore a research program was developed between SLAC, Fermilab, Thomas Jefferson National Accelerator Facility, and Cornell University in order to improve the cavity processing method of the SRF cavities and reach the HE goals. This program explores the doping regime beyond what was done for LCLS-II and also has looked to further developed nitrogen-infusion. Here we will summarize the results from this R\&D program, showing significant improvement on both single-cell and 9-cell cavities compared with the original LCLS-II cavity recipe.

43 PARTICLE ACCELERATORS↗

DIS physics at the EIC and LHeC and connections to the future LHC and $\nu$A programs

Deeply-inelastic scattering (DIS) stands to enter a golden age with the prospect of precision programs at the Electron-Ion Collider (EIC) and Large Hadron-electron Collider (LHeC). While these programs will be of considerable importance to resolving longstanding issues in (non)perturbative QCD as well as hadronic and nuclear structure, they will also have valuable implications for a wider range of physics at the Energy and Intensity Frontiers, including at the High-Luminosity LHC (HL-LHC) and future \nu A ν A facilities. In this plenary contribution, we highlight a number of salient examples of the potential HEP impact from the complementary EIC and LHeC programs drawn from their respective Yellow Report and Whitepaper. Interested readers are encouraged to consult the extensive studies and literature from which these examples are taken for more detail.

Hobbs, Timothy↗

Phase I - Smart Grid Data Access Pilot Program: Utilizing STEM Education as a Catalyst for Residential Consumer Decision Making and Change

Under Phase I of the Smart Grid Data Access Pilot Program, the Gulf of Maine Research Institute (GMRI) partnered with Central Maine Power (CMP), and the Maine Mathematics and Science Alliance (MMSA) and engaged key vendors Tilson Government Services, LLC (Tilson), and Image Works to demonstrate the efficacy of PowerHouse, an interactive online learning environment linking middle school students with their home electricity consumption data provided through CMP’s Advanced Metering Infrastructure (AMI). The goal of the program is to harness the power of youth to alter home energy consumption behaviors using AMI data. Successful programs aimed at smoking cessation, recycling, and seat belt use have demonstrated the power of young people to influence household behaviors. In an era of increasing concern about energy costs, availability, and human impacts on global climate, GMRI sought to demonstrate the effectiveness of a student-focused approach to understanding and managing household energy use. We also sought to contribute to a solid foundation of science-literate students who can analyze evidence to find solutions to increasingly complex energy challenges.

29 ENERGY PLANNING, POLICY, AND ECONOMY↗

Analysis of Tank 4 (FTF-4-15-22, 23) Surface and Subsurface Supernatant Samples in Support of Enrichment Control, Corrosion Control and Evaporator Feed Qualification Programs

This report provides the results of analyses on Savannah River Site Tank 4 surface and subsurface supernatant liquid samples in support of the Enrichment Control Program (ECP), the Corrosion Control Program (CCP) and the Evaporator Feed Qualification (EFQ) Program. The purpose of the ECP sample taken from Tank 4 in August 2015 was to determine if the supernatant liquid would be “acceptable feed” to the 2H and 3H evaporator systems.

12 MANAGEMENT OF RADIOACTIVE AND NON-RADIOACTIVE W↗

Analysis of Tank 38H (HTF-38-15-119, 127) Surface, Subsurface and Tank 43H (HTF-43-15-116, 117 and 118) Surface, Feed Pump Suction and Jet Suction Subsurface Supernatant Samples in Support of Enrichment, Corrosion Control and Salt Batch Planning Programs

Compositional feed limits have been established to ensure that a nuclear criticality event for the 2H and 3H Evaporators is not possible. The Enrichment Control Program (ECP) requires feed sampling to determine the equivalent enriched uranium content prior to transfer of waste other than recycle transfers (requires sampling to determine the equivalent enriched uranium at two locations in Tanks 38H and 43H every 26 weeks) The Corrosion Control Program (CCP) establishes concentration and temperature limits for key constituents and periodic sampling and analysis to confirm that waste supernate is within these limits. This report provides the results of analyses on Tanks 38H and 43H surface and subsurface supernatant liquid samples in support of the ECP, the CCP, and the Salt Batch 10 Planning Program.

12 MANAGEMENT OF RADIOACTIVE AND NON-RADIOACTIVE W↗

Insights from Smart Meters. Identifying Specific Actions, Behaviors and Characteristics that drive savings in Behavior-Based Programs

In this report, we use smart meter data to analyze specific actions, behaviors, and characteristics that drive energy savings in a behavior-based (BB) program. Specifically, we examine a Home Energy Report (HER) program. These programs typically obtain 1% to 3% annual savings, and recent studies have shown hourly savings of between 0.5% and 3%. But what is driving these savings? What types of households tend to be “high-savers”, and what behaviors are they adopting? There are several possibilities: one-time behaviors (e.g., changing thermostat settings); reoccurring habitual behaviors (e.g., turning off lights); and equipment purchase behaviors (e.g., energy efficient appliances), and these may vary across households, regions, and over time.

32 ENERGY CONSERVATION, CONSUMPTION, AND UTILIZATI↗

Laboratory Directed Research and Development Program: FY 2015 Annual Report

The Department of Energy (DOE) and the SLAC National Accelerator Laboratory (SLAC) encourage innovation, creativity, originality and quality to maintain the Laboratory’s research activities and staff at the forefront of science and technology. To further advance its scientific research capabilities, the Laboratory allocates a portion of its funds for the Laboratory Directed Research and Development (LDRD) program. With DOE guidance, the LDRD program enables SLAC scientists to make rapid and significant contributions that seed new strategies for solving important national science and technology problems. The LDRD program is conducted using existing research facilities.

99 GENERAL AND MISCELLANEOUS↗

A Survey of State and Local PV Program Response to Financial Innovation and Disparate Federal Tax Treatment in the Residential PV Sector

High up-front costs and a lack of financing options have historically been the primary barriers to the adoption of photovoltaics (PV) in the residential sector. State clean energy funds, which emerged in a number of states from the restructuring of the electricity industry in the mid-to-late 1990s, have for many years attempted to overcome these barriers through PV rebate and, in some cases, loan programs. While these programs (rebate programs in particular) have been popular, the residential PV market in the United States only started to achieve significant scale in the last five years – driven in large part by an initial wave of financial innovation that led to the rise of third-party ownership.

14 SOLAR ENERGY↗

Continuation and Enhancement of the MPOWIR Program

MPOWIR is a community-based program that provides mentoring to physical oceanographers from late graduate school through early careers. The overall goal of MPOWIR is to make mentoring opportunities for junior physical oceanographers universally available and of higher quality by expanding the reach of mentoring opportunities beyond individual home institutions. The aim is to reduce the barriers to career development for all junior scientists in the field, with a particular focus on improving the retention of junior women. Over the past five years MPOWIR has expanded significantly. This funding cycle saw the development and enhancement of MPOWIR’s programs and outreach opportunities. MPOWIR’s main programmatic offerings are: mentor groups, a biannual conference, a website and blog, and town hall meetings at national events. Since 2009, MPOWIR has hosted 15 mentor groups, three Pattullo conferences, and created a website that has attracted over 50,000 visitors. MPOWIR’s mentoring groups and Pattullo conferences have reached more than 130 unique participants. Mentor Groups Mentor groups were established in the fall of 2008, and continue to gain momentum after 5 years. Since 2009, 11 groups have formed, with participants mainly, but not exclusively, drawn from Pattullo attendees. To gauge the impact of mentor groups, participants are surveyed approximately annually. Based on an extensive 2011 survey, 100% of mentoring group participants reported that they made progress on their stated scientific, professional, and personal goals. As part of these surveys, participants are asked what they value about their mentoring group, what the benefit of the mentoring group is to their current position, and they are asked questions about the logistics and setup of the groups. Based on the survey conducted in 2012, all participants rated participation in these groups a valuable experience, with particular value placed on feedback on professional development (100% Excellent to Good) and feedback on personal matters (92% Excellent to Good). Pattullo Conferences The centerpiece of the MPOWIR program is the Pattullo Conference, named for June Pattullo, the first woman to receive a Ph.D. in physical oceanography. At each event, the agenda includes research talks, professional development sessions, small group activities, and a question-and-answer session about early-career and retention issues posed by junior scientists to senior researchers. Pattullo Conference participants are surveyed after each conference about their experience. This survey asks participants to rate their opinion on specific topics and invites comments. Combining results from three conferences, 99% of junior participants would recommend the conference to another junior scientist. Of the junior participants, 92% rated the conference as “extremely valuable” overall, with a particular value placed on networking and professional development opportunities. Overall Impact To gain a better perspective on the success of MPOWIR to date, early participants were surveyed in 2013 about their current position, and the impact of MPOWIR on their career path. Individuals were asked to rank the extent to which MPOWIR had impacted various aspects of their professional growth. These aspects included efforts to obtain their current position; exposure to professional development skills; broadening of their professional network; mentoring on work and family balance; performance in current position; and raising awareness of mentoring in career development. Participants overwhelming indicated that MPOWIR had positively impacted all of these aspects. In particular, 95% of respondents expressed that MPOWIR exposed them to useful professional development skills “to a great extent” and 89% consider MPOWIR to have positively impacted their professional network “to a great extent”. Of those surveyed, the majority of participants were 2 years from their Ph.D. at the time of initial involvement. Their current job positions are well distributed over all post-Ph.D. categories: the greatest percentages of positions held are at research institutions (non-academic) or government agencies (29.3%), followed closely by non-faculty research positions at universities (27.6%) and faculty at research universities (22.4%). The remaining positions held (4-year college faculty, profit or not-for-profit company, policy, and graduate students) collectively total 19%. These initial indications point toward mentoring, as offered by MPOWIR, as an effective means for improving retention of women in physical oceanography.

58 GEOSCIENCES↗

Bulk Electrical Cable Non-Destructive Examination Methods for Nuclear Power Plant Cable Aging Management Programs

This Pacific Northwest National Laboratory milestone report describes progress to date on the investigation of nondestructive test methods focusing particularly on bulk electrical test methods that provide key indicators of cable aging and damage. The work includes a review of relevant literature as well as hands-on experimental verification of inspection capabilities. As nuclear power plants consider applying for second, or subsequent, license renewal to extend their operating period from 60 years to 80 years, it is important to understand how the materials installed in plant systems and components will age during that time and develop aging management programs to assure continued safe operation under normal and design basis events (DBE). Normal component and system tests typically confirm the cables can perform their normal operational function. The focus of the cable test program, however, is directed toward the more demanding challenge of assuring the cable function under accident or DBE. The industry has adopted 50% elongation at break (EAB) relative to the un-aged cable condition as the acceptability standard. All tests are benchmarked against the cable EAB test. EAB, however, is a destructive test so the test programs must apply an array of other nondestructive examination (NDE) tests to assure or infer the overall set of cable’s system integrity. Assessment of cable integrity is further complicated in many cases by vendor’s use of dissimilar material for jacket and insulation. Frequently the jacket will degrade more rapidly than the underlying insulation. Although this can serve as an early alert to cable damage, direct test of the cable insulation without violating the protective jacket becomes problematic. This report addresses the range of bulk electrical NDE cable tests that are or could be practically implemented in a field-test situation with a particular focus on frequency domain reflectometry (FDR). The FDR test method offers numerous advantages over many other bulk electrical tests. Two commercial FDR systems plus a laboratory vector network analyzer are used to test an array of aged and un-aged cables under identical conditions. Several conclusions are set forth, and a number of knowledge gaps are identified.

21 SPECIFIC NUCLEAR REACTORS AND ASSOCIATED PLANTS↗

Ecological Monitoring and Compliance Program 2015 Report

The Ecological Monitoring and Compliance Program (EMAC), funded through the U.S. Department of Energy, National Nuclear Security Administration Nevada Field Office (NNSA/NFO), monitors the ecosystem of the Nevada National Security Site (NNSS) and ensures compliance with laws and regulations pertaining to NNSS biota. This report summarizes the program’s activities conducted by National Security Technologies, LLC (NSTec), during calendar year 2015. Program activities included (a) biological surveys at proposed activity sites, (b) desert tortoise compliance, (c) ecosystem monitoring, (d) sensitive plant species monitoring, (e) sensitive and protected/regulated animal monitoring, and (f) habitat restoration monitoring. During 2015, all applicable laws, regulations, and permit requirements were met, enabling EMAC to achieve its intended goals and objectives.

54 ENVIRONMENTAL SCIENCES↗

Insights from Smart Meters: Identifying Specific Actions, Behaviors, and Characteristics That Drive Savings in Behavior-Based Programs

In this report, we use smart meter data to analyze specific actions, behaviors, and characteristics that drive energy savings in a BB program. Specifically, we examine a Home Energy Report (HER) program. These programs typically obtain 1% to 3% annual savings, and recent studies have shown hourly savings of between 0.5% and 3%.1 But what is driving these savings? What types of households tend to be “high-savers,” and what behaviors are they adopting? There are several possibilities: one-time behaviors (e.g., changing thermostat settings), reoccurring habitual behaviors (e.g., turning off lights), and equipment purchase behaviors (e.g., energy efficient appliances); these may vary across households, regions, and over time.

29 ENERGY PLANNING, POLICY, AND ECONOMY↗

Comparison of State-Funded Technology Maturation Programs.

This study examines the structure and impact of state-funded technology maturation programs that leverage research institutions for economic development throughout the United States. The lessons learned and practices identified from previous experiences will inform Sandia National Laboratories' Government Relations and Technology Partnerships teams as they participate in near-term discussions about the proposed Technology Readiness Gross Receipts Tax Credit and Program, and continue to shape longer-term program and partnership opportunities. This Page Intentionally Left Blank

99 GENERAL AND MISCELLANEOUS↗