Progressing Policy through Clinical Futures and PolicyLab Pilot Grants

The Pilot Grant Program supports CHOP researchers in launching research studies on clinical effectiveness and policy-oriented health services.

By Holly Burnside and Lindsey Klinger-O'Donnell

Awardees for the Spring 2024 cycle of pilot grants jointly administered by Clinical Futures and PolicyLab, two Centers of Emphasis at Children's Hospital of Philadelphia Research Institute, are driving change in policy and practice. From developing an expert consensus panel to improve pediatric diagnoses to exploring state legislature language around firearm violence, these researchers are conducting unique and exceptional studies on clinical effectiveness and policy-oriented health services to support families. Learn more about their projects:

Exploring State Legislator Language Around Firearm Violence and Policy Implications: Q&A with Vivek Ashok, MD, FAAP

Firearm violence is the leading cause of death among children and adolescents ages 1 to 19-years-old, disproportionately impacting children of color. Key to addressing firearm violence is an improved understanding of policymaker perceptions and framing of the causes and consequences of firearm violence, proposed policy solutions, and the sociopolitical and economic environments that influence health policy agenda setting.

With pilot grant support, CHOP researchers will conduct a content analysis of social media posts from state legislators to identify policymakers' perspectives of firearm violence, study changes in framing over time, analyze if policymakers' perspectives reflect evidence-based health and public health knowledge or reinforce bias and inequities, and interpret how these portrayals impact policy formation.

Researchers involved in this project include: Vivek Ashok, MD, FAAP, a fellow in the National Clinician Scholars Program at the Perelman School of Medicine, a fellow physician in the Academic General Pediatrics Program at CHOP and a general pediatrician and internist; Zachary Meisel, MD, MPH, MSHP, a PolicyLab faculty scholar and the Director of the Center for Emergency Care Policy Research and associate professor of Emergency Medicine at the Perelman School of Medicine at the University of Pennsylvania; Joel Fein, MD, MPH, Co-director of the Center for Violence Prevention at CHOP; Therese Richmond, PhD, RN, FAAN, affiliate research scientist in the Center for Injury Research and Prevention at CHOP; Jonathan Purtle, MSc, MPH, DrPH, assistant professor at Drexel University Department of Health Management and Policy; Katherine Kellom, BA, director of the Qualitative Research Core and an administrative manager at PolicyLab; and Isabella Ntigbu, medical student at the University of Pennsylvania School of Medicine.

What makes your research project innovative?

Our project is the first to systematically study policymaker language and analyze rhetoric through health and public health frames. Furthermore, our analysis is grounded in John Kingdon's Multiple Streams Framework for Policy Agenda Setting (MSF). Underutilized in health policy, the MSF model has been used extensively in social and political sciences to evaluate policy agenda setting and offers an innovative way to conceptualize the pathway to health policy creation.

We hope this study can be replicated when evaluating other health policy foci that overlap with political discourse, such as reproductive rights and climate change. Additionally, Quorum, a dataset of policymaker social media posts, has not, to date, been used to analyze policymaker perspectives on firearm injury and provides a rich and innovative repository of data for this study.

How could results from your project lead to future research?

Our long-term goal is to co-develop social media recommendations for policymakers that accurately reflect the health and public health crisis of firearm violence, structural inequities driving the crisis, and evidence-based policy solutions to address it. Using findings from our preliminary data, we hope to expand our analysis to include a nationally representative sample of policymakers. Lastly, we hope to produce robust policymaker-facing social media guidelines to be implemented, evaluated, refined, and validated.

Developing a Consensus Agenda for Improving Pediatric Diagnosis: Q&A with Irit Rasooly, MD, MSCE

Conservative estimates attribute 80,000 deaths and nearly 800,000 instances of serious harm every year in the US to diagnostic errors. These statistics have made clear the need for the inclusion of diagnostic safety in measures of healthcare quality. Many regulatory, research, and patient safety organizations have articulated the importance of evaluating diagnostic performance to identify opportunities for improvement.

While priorities for addressing adult diagnosis and high-risk adult conditions and demographics are emerging, there needs to be a clear analog in pediatric medicine. The absence of pediatric diagnostic improvement priorities impedes efforts to improve the quality of pediatric healthcare and, ultimately, to advance child health.

With support from Clinical Futures and PolicyLab, CHOP researchers seek to convene a multicenter expert consensus panel to identify and align pediatric priorities in this developing field. In the immediate term, expert consensus will focus on and unify diagnosis-related clinical effectiveness research and policy efforts in pediatrics. They plan to apply findings from this pilot to application for an AHRQ R13 Conference Grant and the development of a national survey of priorities guidelines for improving diagnosis in pediatric healthcare. By articulating priorities for improving diagnosis, this pilot work will provide a launching point for enhancing the quality of pediatric care and preventing harm.

Researchers involved in this project include: Irit Rasooly, MD, MSCE, a hospital medicine physician and researcher within the Department of Biomedical and Health Informatics (DBHi) and Clinical Futures at CHOP; Meghan Galligan, MD, MSHP, an attending hospitalist in the Division of General Pediatrics; Cara Jefferies, MSN, RN, program manager at the Center for Diagnostic Excellence; Anna Costello, MD, a fellow with the Division of Rheumatology at CHOP; Morgan Congdon, MD, an attending physician with the Division of General Pediatrics at CHOP; Jillian Krause, MD an attending physician in the Department of Pediatrics; and Kathy Shaw, MD, MSCE, associate chair for Patient Safety and Quality Improvement in the Department of Pediatrics at CHOP.

What makes your research project innovative?

This pilot project would define the agenda for pediatric diagnostic improvement work. While diagnostic excellence is recognized as an important safety priority by many regulatory organizations, there is limited literature and guidelines related to improving diagnosis for children. Harnessing knowledge and expertise from leaders in pediatric diagnosis across the country will enable us to create an expert agenda to further research, quality improvement, and policy development related to this emerging domain of quality and safety.

How could results from your project lead to future research?

Findings will focus and unify pediatric diagnosis-related clinical effectiveness research and policy efforts in this emerging field. The outcomes of this pilot will inform the development of a national survey of priorities and guidelines for improving diagnosis in pediatric healthcare. By articulating priorities for improving diagnosis, this pilot work will provide a launching point for research and improvement work aimed at enhancing quality of pediatric care and preventing harm.

Open-Label Treatment Trial of Sirolimus in Children with Leigh Syndrome Caused by Genetically Confirmed Mitochondrial Respiratory Chain Deficiency: Q&A with Matthew Demczko, MD

Leigh syndrome (LS) is a rare and genetically heterogeneous progressive neurodegenerative disorder affecting 1 in 40,000 live births. LS is the most common presentation of primary mitochondrial disorder (PMD) in children, associated with inherited defects involving mitochondrial oxidative phosphorylation (OXPHOS), or the process mitochondria use for energy production. The CHOP Mitochondrial Medicine Frontier Program (MMFP) follows more than 150 patients with genetically confirmed LS. Recent in vitro studies have suggested that sirolimus also known as rapamycin (Rapamune®, Pfizer), an inhibitor of the mammalian Target of Rapamycin (mTOR) signaling pathway, greatly enhanced survival and attenuated disease progression in a Ndufs4−/− animal model of complex I-associated LS. A case report of one child with NDUFS4-/- complex I deficiency manifesting as LS treated with an mTOR inhibitor showed marked clinical improvement. No clinical trials of sirolimus have been conducted in human PMD patients.

In this prospective interventional pilot study, researchers hypothesize that sirolimus will have a broad benefit in the improvement of health outcomes in pediatric LS caused by molecularly confirmed mitochondrial respiratory chain complex I disorders. The study aims to investigate the preliminary safety, tolerability, feasibility, and potential therapeutic benefit of sirolimus in children seen in CHOP MMFP with genetically confirmed LS from complex I deficiency.

Researchers involved in this project include: Matthew Demczko, MD, an attending physician in the CHOP Division of Human Genetics within the MMFP; co-investigators Amy Goldstein, MD, an attending physician with the Division of Human Genetics at CHOP; Marni J. Falk, MD, executive director of the CHOP MMFP and founder and organizer of the CHOP/Penn Mitochondria Research Affinity Group; and Kathleen Sullivan, MD, PhD, chief of the Division of Allergy and Immunology.

What makes your research project innovative?

LS, a severe form of mitochondrial disease, causes a progressive neurologic deterioration that is ultimately fatal. Despite numerous trials over the last 20 years, no therapies have been approved, and management is predominantly symptom-based. Most therapies that have gone through clinical trials have looked at ways to overcome the energy production deficit in mitochondrial disease.

However, in 2013, a group from the University of Washington showed for the first time in an animal model of LS that giving sirolimus decreased neuroinflammation and significantly attenuated disease progression. Since this study, several case reports have been published suggesting sirolimus may be safe and effective in patients with mitochondrial disease. Our project is the first clinical trial to study this objectively in a large cohort of patients with LS.

How could results from your project lead to future research?

Positive results from our study would help guide additional research into why mTOR inhibitors such as sirolimus are beneficial to children with LS, whether other means of reducing neuroinflammation would also be beneficial, and if non-neurologic symptoms commonly seen in LS also can be mitigated by this therapy.

Preparing for Change: Leveraging Intervention Mapping in Preparation of a Parent Life Coaching Program for Intimate Partner Violence Survivors and their Children at CHOP: Q&A with Ashlee Murray, MD, MPH

In this proposed project, researchers seek to address the gap in onsite support services at CHOP for parents who have experienced intimate partner violence (IPV). They will adapt the evidence-based Family Vision Program to fit the Philadelphia context in preparation for a Hybrid Type 1 effectiveness-implementation trial.

Through Intervention Mapping, the project aims to tailor the intervention to meet the specific needs and preferences of the CHOP community. The project also will involve developing a detailed implementation plan for the adapted intervention, incorporating input from stakeholders to ensure effective implementation and sustainability.

This project is guided by the Exploration, Preparation, Implementation, and Sustainment (EPIS) framework. Researchers have already completed the Exploration phase, which included a Philadelphia city-wide community needs assessment consisting of interviews with content experts and focus groups with IPV survivors. The proposed grant will enable the team to use these findings and complete the preparation phase work to adapt Family Vision, develop an implementation plan, and prepare for a large-scale Type 1 hybrid trial.

Utilizing innovative methodologies and community-engaged approaches, the project aims to promote healing and resilience among IPV survivors and their families at CHOP, ultimately enhancing the accessibility and effectiveness of support services within the community.

Researchers include: Principal Investigator Ashlee Murray, MD, MPH, director of the Intimate Partner Violence Task Force at CHOP; co-investigators Jillian Baker, DrPH, EdM, executive director of the Center for Parent and Teen Communication; and Gwendolyn Lawson, PhD, a faculty member at Clinical Futures, PolicyLab, and in the Department of Child and Adolescent Psychiatry and Behavioral Sciences at CHOP.

What makes your research project innovative?

Our project is innovative because it adapts the Family Vision Program specifically for IPV survivors in Philadelphia, integrating the EPIS framework and Community-Based Participatory Research to ensure cultural relevance and effectiveness. By embedding a strengths-based parent coaching program within CHOP's healthcare setting, we address a critical gap in onsite IPV support services. This tailored, proactive approach empowers survivors to engage in their own healing and their children's recovery, offering a novel, replicable model for IPV intervention in healthcare.

How could results from your project lead to future research?

Results from our project could pave the way for a subsequent Type 1 hybrid effectiveness-implementation trial, evaluating the adapted Family Vision Program's efficacy and feasibility. This could lead to broader research on scaling the intervention in different healthcare settings and contribute to evidence-based practices for IPV support, ultimately influencing national healthcare policies and interventions.

Within CHOP Research Institute, 15 Centers of Emphasis strengthen our scientific capacity to advance the understanding and improvement of child health. Led by faculty, they promote integration and collaboration to enhance research productivity and growth. These pilot projects highlight the diverse transdisciplinary research conducted within these Centers of Emphasis, with contributions from the Center for Injury Research and Prevention, the Center for Violence Prevention, Clinical Futures, and PolicyLab.

Editor's Note: Contact Holly Burnside with any questions about the Clinical Futures/PolicyLab Pilot Grant Program, eligibility, or the application and submission process. Please note that for the current application cycle, applicants also can indicate interest in one of two $2,000-$4,000 "Equity Enhancement" supplemental grants intended to expand engagement and recruitment to populations traditionally underrepresented in research. Projects designed to investigate disparities and improve equity of healthcare access and outcomes are especially encouraged. Applications for the fall funding cycle are due no later than 4 p.m. Oct. 1, 2024. View the full announcement here.

Learn more about the Clinical Futures/PolicyLab Pilot Grant Program.