Pre-Conference Workshop Day
8:00 am Registration & Morning Coffee
Workshop A: 8:30 - 10:30 am
Discovery & Translational Workshops
Workshop D: 8:30 - 10:30 am
Clinical & Market Access Workshops
Investigating Rare Kidney Diseases Through Genetic Studies, -Omic Approaches & Epidemiology
Synopsis
- Powering up the rare kidney disease drug development by leveraging genetic and -omic patient profiles from natural history of disease studies in FSGS, MCD, MGN, and IgAN to identify and validate therapeutic targets
- Mapping models and response profiles in models to rare and genetic kidney disease patients to predict mechanistic markers of patient response
- Matching gene profiles to biomarkers and predictors of response to therapies to strategize patient selection approaches
Reaching, Enrolling, & Retaining Participants in Rare Disease Clinical Trials
Synopsis
- Dissecting real-world examples to prepare for the length and ambition of studies
- Evolving strategies to identify patients for rare disease clinical trials: Using electronic health records (EHRs) to identify computable phenotypes and conduct life-course research
- Discussing friction points and strategy for pharmacological interventions, as well as combination trials alongside preexisting patient care, and gene therapies
- Working with private physicians and Centres of Excellence for patient recruitment
10:30 am Morning Refreshments & Networking Break
Workshop B: 11:00 - 13:00
Discovery & Translational Workshops
Workshop E: 11:00 - 13:00
Market Access Workshops
11:01 am Pinpointing Molecular Mechanisms Underlying Podocytopathies
Synopsis
- Understanding capacity for podocyte protection in the context of rare and genetic kidney diseases, such as nephrotic syndrome
- Exploring molecular predictors and regulators of steroid resistance in nephrotic syndrome
- Identifying novel approaches and potential future therapies that reduce podocyte injury and proteinuria in nephrotic syndrome
Integrating Patient-Centric Outcome Measures to Enable More Holistic Assessments
Synopsis
- Developing tools to study improving symptom experience to be incorporated in tandem with traditional endpoints during clinical decision making
- Collaborating to ensure that evidence-based approaches capture patient perspectives of the clinical trial experience and more effectively evaluate drug efficacy
- Discussing development of a data dictionary in collaboration with clinicians in the context of rare disease
1:00 pm Lunch & Networking Break
Workshop C: 14:00 - 16:00
Discovery & Translational Workshops
Workshop F: 14:00 - 16:00
Clinical & Market Access Workshops
Leveraging In Vitro, In Vivo, & In Silico Tools to Recapitulate Genetic & Non-Genetic Renal Disorders
Synopsis
- Following guidance that the FDA no longer requires animal models for drug development, what preclinical approaches are being harnessed to study rare and genetic kidney diseases?
- Exploring tissue-bioengineering approaches for modelling rare kidney diseases
- Understanding considerations for preclinical investigation of rare and genetic kidney diseases
- Applying computational tools alongside preclinical modelling to mitigate preclinical development risk
Commercialization & Communication Through the Clinical Experience in the Rare & Genetic Kidney Disease Space
Synopsis
- Discussing pricing, reimbursement and market access in the context of standard of care and prescriber burden
- Understanding payer concerns around rare disease coverage for therapeutic interventions
- Communicating the clinical value of orphan drugs in the rare and genetic kidney disease setting to a breadth of stakeholders
- Outlining stories of value demonstration to private insurers to incentivise prescription and enrolment