RESEARCH AND RESEARCH OPPORTUNITIES IN PEDIATRICS Legislative Advocacy August 14 th, 2012 Amélie Collins, MD PhD PGY-2

Translational Research and Pediatrics “Old” view of translational research: Szilagyi 2009

Translational Research and Pediatrics Szilagyi 2009 T1 (potential application): translation of basic research into a potential clinical application. -phase I and II clinical trials, pilot clinical studies Result: theoretical knowledge about a possible intervention “T1 research must be linked with a potential clinical application and should be distinguished from basic science research that investigates pathogenesis or understanding of disease process without a close link to a clinical application.”

Translational Research and Pediatrics Szilagyi 2009 T2 (efficacy studies): new therapies are tested under controlled environments to provide the link between potential clinical applications and potential evidence-based guidelines -phase III clinical trials (RCTs), population research, systematic reviews of the literature, health services studies Result: efficacy knowledge about interventions that work in optimal settings.

Translational Research and Pediatrics Szilagyi 2009 T3 (effectiveness studies): translation from recommendations or guidelines into practice -phase IV clinical studies, quality improvement research, dissemination research, diffusion research Result: applied knowledge about interventions that work in real-world settings

Translational Research and Pediatrics Szilagyi 2009 T4 (population-based): outcomes assessments at the community or population level -extension of T3 implementation research to an entire population; cost-benefit studies Result: public health knowledge about impact at the population level

AAP Support for Pediatric Research Washington Report on Academic and Subspecialty Advocacy May 2012 “The American Academy of Pediatrics is actively engaged in federal advocacy for the needs of academic and subspecialist pediatricians and the children their work benefits.” “…promote medical research for children…”

Funding for Pediatric Research Fiscal Year 2012 spending bills includes the Labor-Health and Human Services-Education Bill FY2012: $156.3 billion in regular discretionary funding for Labor, Health and Human Services, and Education programs ($1.1 billion below FY2011 levels).

Funding for Pediatric Research U.S. Department of Health and Human Services was allocated $69.7 billion; a decrease of almost $700 million from FY2011.

Funding for Pediatric Research Programs that received cuts: Programs that received increases/no cuts:

Structure of Pediatric Research National Institutes of Health ($30.7 billion) National Center for the Advancement of Translational Science -CTSAs (Clinical and Translational Science Awards) awarded to institutions (Columbia been recipient since inception in 2006) -AAP makes it a priority to advocate for adequate pediatric funding within each institution’s CTSA Agency for Healthcare Research and Quality (AHRQ) – annual budget of $300 million. Research networks: Pediatric Research in Office Settings (PROS), Continuity Research Network, Pediatric Emergency Care Applied Research Network (PECARN), National Institutes of Child Health and Human Development (NICHD) Neonatal Research Network, Collaborative Pediatric Critical Care Research Network, Pediatric Research in Inpatient Settings.

Funding for Pediatric Research FY 2002: NIH spent $22.1 billion for all research, including $9.1 billion for “translational research.” -of that $9.1 billion, $8.3 billion (91%) was for T1 research -only $787 million for T2, T3, and T4 research combined.

Current climate for funding. “Congress is currently operating in a divisive and polarizing fiscal environment.” AAP’s Federal Advocacy Resource Center released press release Feb 2012 “expressing extreme disappointment with Chairman Ryan’s budget resolution. The proposed budget would significantly reduce funding levels for vital child health programs, block grant Medicaid, and repeal Affordable Care Act. Chairman Ryan’s proposal includes even deeper spending cuts than are currently required by law.” -Budget proposal would cut programs, including defunding the AHRQ. Federal Advocacy Resource Center -AAP login -will tell you who your Senators and congressman are -pre-drafted form s urging them not to cut funds for children’s health.

Training Pediatric Researchers American Academy of Pediatrics Committee on Pediatric Research “Promoting Education, Mentorship, and Support for Pediatric Research” Pediatrics : % of all graduates of pediatric residency programs pursue traditional research careers -Funding by the NIH to support research training in pediatrics, including individual fellowship grants, is at only 10% of the level of support provided for research training in internal medicine. -recommend a research curriculum for pediatric residents and suggest that pediatric residency programs should promote research electives either as block rotations or as longitudinal protected time to allow trainees to participate in “research project” - only one quarter of pediatric residency programs do so.

Training Pediatric Researchers

Research Training in Residency Vinci et al., Research During Pediatric Residency Training: Outcome of a Senior Resident Block Rotation. Pediatrics : retrospective survey of 165 residents that participated in 3 month required “Career Development Block” during PGY-3 year at Boston Children’s. -goal of the CDB is to “develop a scholarly project” and residents are encouraged to develop an “academic product” such as an educational curriculum, review article, abstract, or manuscript -mandatory curriculum that includes ~25 hours of seminars on topics such as research methodology, etc… -support includes Rotation Directors, project mentors, opportunity to apply for intramural grants ranging from $500 to $8000 per grant, and funds to attend any national meeting to which a resident has work accepted.

Research Training in Residency Vinci et al., Research During Pediatric Residency Training: Outcome of a Senior Resident Block Rotation. Pediatrics :1126.

Research Training in Residency Mills et al., Trainee Participation in an Annual Research Day is Associated with Future Publications. Teaching and Learning in Medicine, :62 -retrospective cohort study of pediatric residents and fellows at University of North Carolina -compared trainees who participated in voluntary “Evening of Scholarship”, annual forum to present scholarly work -69% of EOS participants had publications after graduation versus 34% of nonparticipants (p <.001). -in multivariate analysis controlling for gender and publications prior to EOS, subjects who participated in EOS were more likely than nonparticipants to publish in the future (OR = 3.7, 95% CI = 2.5–5.6).

Research Training in Residency Ozuah PO, Residency Research Requirement as a Predictor of Future Publication Productivity. J Peds, :1 -matched case-controlled study of a cohort of pediatric residents who were either in the primary care pediatric residency track (has a research requirement) or the categorical pediatric residency track (no research requirement). For each case, 2 control subjects were selected. -subjects were matched according to the following criteria: 1) reputation of the medical school from which the resident graduated (on the basis of the US News and World Report) 2) memberships in Phi Beta Kappa Society and Alpha Omega Alpha 3) academic performance in medical school (on the basis of grades achieved) 4) residency interview rank score 5) sex 6) year of graduation from the residency program

Research Training in Residency Ozuah PO, Residency Research Requirement as a Predictor of Future Publication Productivity. J Peds, :1 -subgroup of cases and control subjects who pursued fellowships (45% of cases and 65% of control subjects). As compared with control subjects who pursued fellowships, cases who went on to do fellowships had a significantly greater mean number of publications during and after residency (4.0 versus 0.5, P =.035).

Research Opportunities at CHONY Each resident is expected to complete a “scholarly project.” -one or more blocks of elective set aside as research time -opt out of full day clinic in order to devote one morning a week to research -up to $500 seed money per resident Other resources: 1.Irving Institute for Clinical and Translational Research training course -2 week course dedicated to exposing residents to clinical research. Each resident will design an IRB proposal for a clinical project in conjunction with biostatisticians experienced in guiding clinical research, and will present their proposal at the conclusion of the rotation. 2.Academic Pediatric Association Resident Investigator Awards Program -up to $5000 for research by residents related to child health promotion, health services research, teaching, or patient care.

References Washington Report Academic and Subspecialty Advocacy, published May Szilagyi, Translational Research and Pediatrics. Academic Pediatrics 2009;9:71 AAP Committee on Pediatric Research, Promoting Education, Mentorship, and Support for Pediatric Research Pediatrics 2001;107:1447 Vinci et al., Research During Pediatric Residency Training: Outcome of a Senior Resident Block Rotation. Pediatrics 2009;124:1126. Mills et al., Trainee Participation in an Annual Research Day is Associated with Future Publications. Teaching and Learning in Medicine, 2011;23:62 Ozuah PO, Residency Research Requirement as a Predictor of Future Publication Productivity. J Peds, 2009;155:1