Webinar overview + objectives:

Therapies for rare diseases are emerging at a rapid pace yet rigorous evidence regarding their safety, efficacy, and effectiveness in practice is often sparse and is difficult to generate. High quality longitudinal patient registries may help to address this evidence gap. In this webinar, we will:

1. Describe the challenges in generating high quality evidence about treatments for rare diseases;

2. Summarize some of the strategies proposed to address these challenges, emphasizing the potential value of patient registries;

3. Describe the features of registry-based randomized clinical trials and discuss the scientific and practical challenges associated with such trials; and

4. Summarize current guidance and emerging developments in designing and improving rare disease registries to support clinical trials.

Meet the speakers!

Pranesh Chakraborty is the Chief/Chair of the Department of Pediatrics at the Children’s Hospital of Eastern Ontario (CHEO) and the University of Ottawa, where he is also a Full Professor. He is a physician certified by the Royal College in medical biochemistry and pediatrics, with a subspecialty in biochemical genetics. Dr. Chakraborty joined CHEO in 2003 as a clinician for patients with inherited metabolic diseases. In 2006, he led the establishment of Newborn Screening Ontario at CHEO and he was its Medical and Laboratory Director until taking on the Chief/Chair of Pediatrics role in 2023. Dr. Chakraborty’s research interest focuses on inherited metabolic diseases and newborn screening. He is involved in team research in newborn screening policy, the development of novel laboratory diagnostic and screening methods, translational metabolomics and disease pathophysiology research, as well as patient registries and observational outcome research.

Beth Potter is a Professor of Epidemiology and Public Health and University Research Chair in Health Services for Children with Rare Diseases at the University of Ottawa; and an Affiliate Investigator at the Children’s Hospital of Eastern Ontario Research Institute. Dr. Potter completed her PhD in Epidemiology at the University of Western Ontario in 2003 and joined the University of Ottawa as a faculty member in 2007. Her research aims to produce evidence to improve health care and outcomes for children with rare genetic diseases, particularly inherited metabolic diseases. Dr. Potter is Principal Investigator for INFORM RARE (www.informrare.ca), a Canadian CIHR-funded clinical trials network that brings together patients and their family members, clinical providers, methodologists, and policy makers to focus on research questions prioritized by those who make decisions about rare disease care.

Webinar objectives:

1. Understand the role that historical and ongoing marginalization play in research participation

2. Discuss how research findings in homogeneous populations are not generalizable to all children

3. Describe research approaches that can improve diversity and inclusion in pediatric clinical trials

Meet the speaker!
Dr Tamorah Lewis is a physician scientist, Division Head for Clinical Pharmacology and Toxicology at SickKids. In this role, Dr Lewis helps advance the hospital-wide vision for Precision Child Health and collaborates with multiple clinical Divisions for pharmacology research. Dr Lewis also continues to provide clinical care in the level IV Neonatal ICU at SickKids. The goals of Dr Lewis’ research program are to bring precision therapeutics to the NICU population. Dr Lewis' interests include PBPK and population pharmacokinetic and pharmacodynamic investigations in neonates and children, pertaining to both drugs in common use and drugs under development. Her research focuses on the intersection between ontogeny and pharmacogenetic effects on pharmacology of drugs in infants and neonates. In addition to her clinical and research roles, Dr Lewis is a dedicated advocate for Health Equity. Through publications and talks, she educates about the need for increased diversity in the biomedical workforce and the importance of diversity and equity in clinical research and clinical care.

Webinar objectives:
In this session, we will explore best practice guidelines for researchers to build more equitable, reflective, and inclusive research and clinical trials with respect to gender and sex. We will touch briefly on a variety of topics including, participant eligibility, recruitment, sampling plans and data analysis, measurement of genders and sexes, responsiveness across time, and working with validated scales. The objective is to stimulate critical thought about effective integration of sex and gender in research and clinical trials and to offer some key resources for continued learning.

Meet the speaker!
Dr. Natalie Rosen (she/her) is a Professor in the Departments of Psychology and Neuroscience and Obstetrics and Gynaecology at Dalhousie University, Halifax, Nova Scotia, Canada. Her research focuses on understanding how couples cope with sexual dysfunction and changes to their sexual relationship —such as during pregnancy and the postpartum period. She is committed to translating her research findings into novel interventions and to sharing evidence-based information with service providers and the public. Dr. Rosen has authored over 150 peer-reviewed publications and she is the current Past-President of the Canadian Sex Research Forum. Dr. Rosen is the Sex and Gender Science Lead for the CIHR-funded Maritimes SPOR Support Unit and has developed several resources to support researchers’ consideration of sex and gender in their work.

Webinar objectives:
The webinar will introduce effectiveness-hybrid designs, showcase examples, and outline important considerations for conducting these types of studies. It will also briefly summarize tensions that may occur in conducting implementation research and mention tools to support implementation planning and execution.

Meet the speaker!
Melanie Barwick, Ph.D., C.Psych, is a Senior Scientist in the Child Health Evaluative Sciences Program of the SickKids’ Research Institute and the SickKids Centre for Global Child Health. She also leads professional and resource development in Dissemination and Implementation Research and Practice as Scientific Director of the Knowledge Translation Program in the SickKids’ Learning Institute. At the University of Toronto, she is a Professor in the Department of Psychiatry, Temerty Faculty of Medicine, and the Dalla Lana School of Public Health (Social and Behavioural Health Sciences, and the Institute for Health Policy, Management, and Evaluation). Her community work includes Chairing the Governing Board for Children’s Mental Health Ontario, membership on the Editorial Board for the SIRC journal Implementation Research and Practice, and as an Associate Editor for Frontiers in Health Services – Implementation Science. An internationally recognized expert in dissemination and implementation research and practice, her health services research program spans many areas of health to improve the implementation of evidence into practice and broaden its reach to support decision-making, policy, knowledge, awareness, health, and well-being. She provides professional development in dissemination and implementation practice internationally through the Specialist Knowledge Translation Training™ (SKTT, for researchers and KT practitioners), the Knowledge Translation Professional Certificate™ (KTPC, for KT practitioners), and Planning for Implementation Practice™(PIP).

Webinar overview + objectives:
Join us for an engaging webinar aimed at enhancing understanding and implementation of diversity, equity, and inclusion (EDI) principles within clinical trial teams. Attendees will explore the significance of EDI in improving research outcomes and participant experiences, learn effective strategies for fostering an inclusive team culture, and gain practical knowledge on implementing EDI principles in clinical trial management.

1. Understand the significance of diversity, equity, and inclusion (EDI) in clinical trial teams and management practices, and how these elements contribute to improved research outcomes and participant experiences.

2. Learn effective strategies for fostering a culture of inclusion within clinical trial teams, including communication techniques, conflict resolution approaches, and decision-making processes that prioritize diverse perspectives.

3. Gain practical knowledge on implementing EDI principles when developing teams and clinical trials processes,with a focus on promoting fairness, accessibility, and representation across all phases of research.

Meet the speaker!
Munaza has 24 years of experience in the world of clinical trials. She is passionate about EDI principles, integrating them into all her work, with a special focus on the inclusion of immigrants in clinical trials. Munaza’s work experience includes leadership roles at several excellent healthcare institutions, overseeing large complex teams facilitating the start-up, execution, and close-out of clinical research projects in multiple therapeutic areas. Munaza leads the Monitoring Program at MICYRN. She is on Faculty at McMaster University, where she teaches in the Applied Clinical Research Program. She chairs the N2 Public Engagement Committee, where she champions many EDI initiatives. She is on the executive board of ACRP Canada. She also sits on the Joint Task Force for Clinical Trials Competency at the MRCT Center at Harvard, which has defined the core competency framework for clinical research professionals globally.