The Future Pediatric Subspecialty Physician Workforce Meeting the Needs of Infants, Children, and Adolescents (2023) / Chapter Skim
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6 Trends in the Pediatric PhysicianScientist Workforce
6 Trends in the Pediatric PhysicianScientist Workforce
Pages 203-260
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scientists who study pediatric subspecialty conditions and treatments) are discussed as relevant, the focus of this chapter is on the pediatric physician–scientist workforce.
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Success stories serve as testimony to the transformative impact of scientific discovery on clinical care, but such discoveries require ongoing efforts and partnerships among medical schools, hospitals, payers, advocates, clinicians, and researchers to address rising challenges and to optimize access of therapeutics to children as early as possible. Boxes 6-2 and 6-3 highlight two specific case studies of pediatric research success: spinal muscular atrophy (SMA)
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; • Improved survival rates for childhood acute lymphoblastic leukemia to 90 percent through clinical trials for combination chemotherapies (Hunger et al., 2012) ; • Improved life expectancy and quality of life for children with cystic fibrosis and sickle cell disease due to screening panels, effective medications, preventative care, and standardized medical manage ment (CFF, 2022; SCDAA, 2021)
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BOX 6-3 Pediatric Research Success Case Study: COG The Children's Oncology Group (COG) is a National Cancer Institute supported clinical trials network that has created a robust research infrastructure to design, study, and rapidly implement proven therapies to improve children's survival and quality of life (COG, 2023)
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, and "while as much as 65 percent of funding for studies in adult populations is provided by the pharmaceutical industry, nearly 60 percent of pediatric clinical trials are sponsored primarily by government and nonprofit organizations" (Rees et al., 2021, p.1237) , showcasing the lack of financial incentives for robust industry participation in pediatric research.
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, especially among minoritized groups. A review of pediatric studies in ClinicalTrials.gov2 from 2008 to 2019 found a total of 36,136 clinical trials and 16,692 observational studies, with the number of pediatric clinical trials nearly doubling over this period (from 7,000 to almost 12,000)
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"authorize NIH…to prioritize needs in various therapeutic areas and sponsor clinical trials of off-patent drug products that need further study in children, as well as training and other research that addresses knowledge gaps in pediatric therapeutics" (NICHD, 2022c)
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. As with adult research, pediatric clinical trials have not always been appropriately inclusive of populations experiencing health disparities (Aristizabal et al., 2015; Faulk et al., 2020; Lund et al., 2009; Walsh and Ross, 2003; Winestone et al., 2019)
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. For the purposes of this report, the committee focused on the pediatric physician–scientists, namely those with M.D., D.O.,6 M.D./Ph.D., or M.D./MPH degrees who perform biomedical, behavioral science, health services research, or public health research of any type as their primary professional activity.
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Additionally, international medical graduates may be a resource for increasing the number of physician–scientists. Following M.D./D.O.
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. Following fellowship training, the most common pathway to an independent, academic career for pediatric subspecialty fellows is the transition from fellowship to junior faculty by way of a mentored physician–scientist award, usually in the NIH K series.
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graduates in pediatrics may have implications for the overall pediatric research workforce. 7 An NIH T32 award is an Institutional Training Grant that is made to institutions to support groups of pre- and/or postdoctoral fellows, including trainees in basic, clinical, and behavioral research.
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due to data limitations and little to no coordination between the funders and other parties involved in the pediatric research workforce. For example, data from NIH on the number of physicians supported on T32 institutional training grants, receiving K01 Mentored Research Career Development Awards,8 and awarded R019‐ Equivalent research grants are currently unavailable (Garrison and Ley, 8 "K awards provide support for senior postdoctoral fellows or faculty-level candidates.
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will be critical for retention of successful pediatric physician–scientists. During one of the committee's public webinars, Ericka Boone, director for the Division of Biomedical Research Workforce, Office of Extramural Research, NIH, highlighted the need for data on the workforce and barriers to the pathway: The only way we can have a clear understanding of what these barriers are is if we ask the individuals that are engaging in these research careers, especially those individuals who have exited out of the research career.
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The duration of training to achieve this goal, coupled with the burden of debt from medical school, the work–life balance expecta tions of current trainees, the low funding rate for grant submissions, and annual income for the additional training years to pursue a career as a physician–scientist are all factors that have driven some of the best and the brightest to clinical practice or positions with industry." – Faculty Member, Houston, TX "Pediatric subspecialty training allows for the leaders in the specialty to drive the important knowledge that disseminates to the pediatric work force. It is imperative for excellent quality care and separates the [United States]
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. For many, early encounters with the medical or the health field, formative research experiences, patient encounters that informed research questions, and inspiring mentors helped fuel and define interest in medicine and/or research careers.
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This is especially true for physician–scientists from populations underrepresented in the extramural scientific workforce (including the biomedical, clinical, behavioral, and social sciences workforces)
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More than 30 medical schools have Physician–Scientist Training Programs (PSTPs) that integrate residency, fellowship, and postdoctoral training for trainees that commit to a physician–scientist career path (Garrison and Ley, 2022; Muslin et al., 2009)
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In a report of early outcomes, PIs endorsed institutional commitment of new resources to support the program and viewed the program positively regarding enhancing research opportunities and recruitment, although there is a need to increase the pool and appointees from populations underrepresented in the extramural scientific workforce (Price Rapoza et al., 2022)
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Mid-Career While most formal mentoring programs are targeted towards earlycareer researchers and junior faculty, mentorship is also important in the mid-career space, particularly for female researchers and other populations underrepresented in the extramural scientific workforce (Bora, 2023; Lewiss et al., 2020; Sotirin and Goltz, 2023; Teshima et al., 2019)
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(See Chapter 8 for more information.) During one of the committee's public webinars, Mary Leonard, Arline and Pete Harman Professor and Chair of the Department of Pediatrics at Stanford University, director of the Stanford Maternal and Child Health Research Institute, and physician-in-chief of Lucile Packard Children's Hospital, highlighted how financial considerations play a role in pursuing a pediatric research career: "Going the physician–scientist route defers compensation even further.
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NIH Loan Repayment Programs (LRPs) and Impact on Research Careers Loan repayment for pediatric researchers is one approach to overcome financial barriers to entering or remaining in pediatric subspecialty research careers.
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. From FY2013 to FY2022, there were 3,105 LRP awards in pediatric research, with a 52 percent success rate overall (40 percent for new awards and 71 percent for renewal awards)
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T32 institutional training program grants are made to institutions to support groups of pre- and/or postdoctoral fellows and K12 institutional career development institutional grants (e.g., for PDSPs) aim to prepare newly trained physicians who have made a commitment to independent research careers by providing support to institutions that mentor clinical fellows and scientists (Garrison and Levy, 2022; NICHD, 2023a)
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(2021) found that having a T32 training grant doubled the probability that pediatric subspecialty fellows published during their fellowship.
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. However, structural, systemic, and cultural barriers exist for trainees and faculties from populations underrepresented in the extramural scientific workforce that limit entry or reduce retention in this career path (Behera et al., 2019)
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. Strategies that have been suggested to promote representation in the research workforce include institutional antiracism policies; support for trainees and faculty from populations underrepresented in the extramural scientific workforce; encouraging diversity in public engagements and institutional leadership; providing child/elder care subsidies; tracking diversity outcome measures; and developing "diversity aware" training curricula (Williams et al., 2022)
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Priorities include supporting career pathways, research resources, and capacity at minorityserving institutions,15 promoting equity at extramural institutions in regard to environment and culture, and encouraging equity in policies and procedures at NIH. Mid-Career Concerns and Retention This chapter largely focuses on the early phases of the pediatric physician–scientist pathway, emphasizing that early career mentorship, protected research time, and funding are critical supports for entry into a research career, and these can lay a foundation to promote longevity over time.
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This is a substantial financial investment to which pediatric departments often cannot commit due to financial pressures, so pediatric physician–scientists may be particularly vulnerable during the K to R transition period. Given the current data limitations, it is important to collect more data on the rates of attrition across the pediatric 16 For examples, see https://ictr.johnshopkins.edu/education-training/seminars-courses/k-to r/ (accessed July 18, 2023)
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The federal government -- particularly NIH -- is a significant funder of child health research in the United States. Other major sources of funding for pediatric research include other federal agencies, private foundations, state and local governments, pharmaceutical companies, device manufacturers, and biotechnology firms.
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ARRA = American Recovery and Reinvestment Act (American Recovery and Reinvestment Act of 2009, Public Law No. 111-5, 111th Congr., H.R.
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. NIH Research Funding by Subspecialty NICHD funding for principal investigators with pediatric subspecialty fellowship training has risen steadily over time, with the largest increases coming in the past 4 years (see Figure 6-4)
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SOURCE: Provided to the committee by Rohan Hazra, NICHD, on September 9, 2022. NICHD's Child Health Information Retrieval Program (CHIRP)
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from enrollment in investigational efforts, such as natural history studies, interventions, and clinical trials. NIH funding to pediatric institutions and programs continues to be increasingly concentrated in a few sites.
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. Other current priorities of the group include the COVID-19 pandemic and its impact on children and youth, pediatric medical devices, and aligning pediatric clinical trial and other research networks.
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. These other federal funders are critical for pediatric research, but it is difficult to quantify the amounts specific to pediatric subspecialty research.
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. In 2020, there were more than 2,100 industry-sponsored pediatric clinical trials underway,22 testing 580 investigational medicines23 in infants, children, and adolescents (PhRMA, 2020)
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Act of 2021,25 and the Pediatric Subspecialty Loan Repayment Program. Other large foundations that support pediatric research include the Annie E
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What this leaves us with is a therapeutic pipeline for children that's really threatened by not having enough bright new minds going into the field and really leaves out children when it comes to early adoption of novel therapies…we're really limiting the therapeutic opportunities that we bring to children by not attracting more and speaking to not only the enthusiasm of these pediatric subspe cialists, but their earning potential for the long term.27 KEY FINDINGS AND CONCLUSIONS Key Findings Finding #6-1: Unique aspects of pediatric clinical trials can affect recruitment, retention, and trial success, including ethical consider ations, logistical and technical factors in administering interventions, smaller population size (especially for subspecialty care) , and financial disincentives related to the limited commercial market potential for pediatric drugs compared with adult drugs.
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Finding #6-8: Receipt of an NIH Loan Repayment Program award is associated with higher levels of persistence in research over a decade after initial application.
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This is espe cially true for physician–scientists from populations underrepresented in the extramural scientific workforce. Conclusion #6-5: Partnerships between government, public, and indus try organizations can play a key role in building career pathways for pediatric researchers to help push the frontiers in clinical trials, drug discovery, and health services research.
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Intentional efforts are needed to encourage and facilitate entry into research careers and foster the early phases of career development for pediatric physician–scientists, especially those from populations underrepresented in the extramural scientific workforce. The current system for producing and nurturing pediatric physician–scientists has been inadequate and needs to be improved.
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RECOMMENDATION 6-2 The National Institutes of Health and the Agency for Healthcare Research and Quality should increase the num ber of career development grants in pediatrics, particularly institutional training awards (e.g., the Pediatric Scientist Development Program) , the Pediatric Loan Repayment Program, and K awards, with attention to providing such grants to physician–scientists from backgrounds that are underrepresented in the scientific workforce29 and for high-priority subspecialties in pediatric research.
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https://www.aamc.org/professional-development/affinity-groups/ gfa/mentoring (accessed July 17, 2023)
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2014. Association between pediatric clinical trials and global burden of disease.
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2016. Children in clinical trials: Towards evidence-based pediatric pharmaco therapy using pharmacokinetic-pharmacodynamic modeling.
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upfront therapeutic clinical trials 2004–2015.
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: Impact on career development and professional society diversity. Academic Medicine 96(4)
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2023. Maternal and Child Health Bureau (MCHB)
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2009. Challenges in conducting clinical trials in children: Approaches for improving performance.
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2009. Racial/ethnic diversity in children's oncology clinical trials: Ten years later.
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. NICHD (National Institute of Child Health and Human Development)
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2022b. Populations underrepresented in the extramural scientific workforce.
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Academic Medicine 97(9)
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2018. Funding pediatric subspecialty training: Are T32 grants the future?
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2018. Association of National Institute of Child Health and Human Development career development awards with subsequent research project grant funding.
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Academic Medicine 90(7)
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