In addition to our regular CRI Activities Update, this month’s Research Focus highlights the research and work of Steven Joffe, MD, MPH, the Founders Professor of Medical Ethics and Health Policy and Division Chief of Medical Ethics at the Perelman School of Medicine, University of Pennsylvania.

Activities Update:

The UNC Science Expo will be held on Saturday, April 6, from 11:00 am – 4:00 pm, and the Children’s Research Institute will be there! Our booth will focus on why research in children is important, spread the word about leading-edge research programs working on in various childhood diseases here at UNC, and showcase the work of some of our asthma researchers within the Department of Pediatrics. The Expo also features booths from across UNC’s campus dedicated to STEM education and activities. Please check out Morehead’s website for more details. We hope to see you there!

Our Pediatric Clinical Research Unit (Peds CRU) continues to look for opportunities to grow our human subjects research portfolio as we build our resources. Peds-CRU is meant to enhance and support all clinical research in child health for UNC researchers. We are currently in the midst of hiring an additional research coordinator and are supporting human subjects research projects from the divisions of Pulmonology; Allergy, Immunology, and Rheumatology; General Pediatrics and Adolescent Medicine; Infectious Disease; Critical Care Medicine; and Otolaryngology/Head and Neck Surgery. We are also working across divisions and the School of Medicine, and utilizing resources from NC TraCS to develop standardized processes and training for pediatric clinical research.

Renovations on the Mary Ellen Jones building are being finalized and the move-in process for inhabitants is happening. CRI investigators are scheduled to begin moving in April and May. The new location will greatly enhance and complement our virtual presence on UNC’s campus, as well as provide a place for pediatric health researchers to collaborate. In addition to being our physical hub, the Mary Ellen Jones building houses several conference rooms of various sizes; the offices and laboratories of several of our translational and basic science investigators; a small, dedicated area for the conduct of simple pediatric research study visits; the centralized CRI-Peds CRU laboratory; and our growing core of CRI common research equipment.

Reminder: Steven Joffe, MD, MPH, will be presenting “Building a Learning Health Care Culture: Lessons from Pediatric Oncology” at our next CRI luncheon seminar series. This event will be held on Thursday, April 4, 2019, from 12:15-1:30pm in MacNider 321 (**room change**). Please mark your calendars. We look forward to seeing you there.

Research Focus of the Month:

Steven Joffe, MD, MPH

We are thrilled to welcome Steven Joffe, MD, MPH, as our speaker for our April seminar, which we are co-hosting with the UNC Center for Bioethics. Dr. Joffe is the Founders Professor of Medical Ethics and Health Policy and Division Chief of Medical Ethics at the University of Pennsylvania’s Perelman School of Medicine.

Research in pediatric populations is more complex than in adults because of many factors (eg, vulnerable population, physiologic and pharmacokinetic differences, maturity, and family dynamics). However, research in this population is needed to provide evidence-based treatment. Dr. Joffe’s research addresses the complexity of ethics involved in clinical and translational studies, such as the roles and responsibilities of investigators in clinical trials, children’s ability to participate in medical research, and new challenges presented with modern techniques such as the use of whole-exome sequencing technology in clinical care.

For his talk, Dr. Joffe will be discussing the rewards and challenges of building a “learning health system,” using pediatric oncology as an example. Learning health systems integrate data from quality improvement measures and biomedical research to improve patient care. This information can also be shared to inform improvements in care at other facilities and provide a basis for further research advances. In this way, research is systematically integrated with clinical care. This model is in contrast to waiting for a research study to be completed, published, and then adapted for potential implementation in the clinic depending upon additional funding.

Pediatric oncology is frequently cited as a model for a learning health system because more than 90% of oncology patients receive care at a National Cancer Institute-funded Children’s Oncology Group (COG)-affiliated institution, for example. This high participation rate has been extremely beneficial to patients. This research has taken childhood cancer from an almost incurable disease, to one with a 5-year survival rate of 80%.[1]

While the learning health system model presents the advantage of generating research outcomes that can be translated to clinical practice, there are areas of concern, particularly as technology advances and ethical considerations become more complex. In particular, the blurring of lines between research and clinical care raises questions about therapeutic misconception and about appropriate oversight of activities intended to produce generalizable knowledge. As a pediatric research institute at an academic medical center, it is important for us to reflect on these issues, and we are excited to host Dr. Joffe for his discussion.

Recent publications from Dr. Joffe and related links are below:

Department of Medical Ethics & Health Policy (University of Pennsylvania)

Smith MA, Joffe S. Will My Child Do Better if She Enrolls in a Clinical Trial? Cancer. 2018 Oct 15;124(20):3965-3968. doi: 10.1002/cncr.31722. Epub 2018 Oct 6. PMID: 30291807.

Kilbride MK, Joffe S. The New Age of Patient Autonomy: Implications for the Patient-Physician Relationship. JAMA. 2018 Nov 20;320(19):1973-1974. doi: 10.1001/jama.2018.14382. PMID: 30326026.

Fernandez Lynch H, Joffe S, Feldman EA. Informed Consent and the Role of the Treating Physician. N Engl J Med. 2018 Jun 21;378(25):2433-2438. doi: 10.1056/NEJMhle1800071. PMID: 29924950.

McGraw SA, Garber J, Jänne PA, Linderman N, Oliver N, Sholl LM, Van Ellen EM, Wagle N, Garraway LA, Joffe S, Gray SW. The Fuzzy World of Precision Medicine: Deliberations of a Precision Medicine Tumor Board. Per Med. 2017 Jan;14(1):37-50. doi: 10.2217/pme-2016-0074. Epub 2016 Dec 15. PMID: 28757884.

Marron JM, Joffe S. Ethical Considerations in Genomic Testing for Hematologic Disorders. Blood. 2017 Jul 27;130(4):460-465. doi: 10.1182/blood-2017-01-734558. Epub 2017 Jun 9. PMID: 28600340.

Denburg A, Rodriguez-Galindo C, Joffe S. Clinical Trials Infrastructure as a Quality Improvement Intervention in Low- and Middle-Income Countries. Am J Bioeth. 2016 Jun;16(6):3-11. doi: 10.1080/15265161.2016.1170230. PMID: 27216089.