Conference Day Two
Thursday 14th September 2023
8:55 am Chair’s Opening Remarks
Reimagining Endpoints to Demonstrate Efficacy in Shorter & More Efficient Clinical Trials
9:00 am Tackling the Challenges: Outlining a Novel Molecule for the Treatment of FSGS
Synopsis
- Detailing a multicenter phase III FSGS trial and addressing the timeline challenges
- Improving the recruitment of patients and encouraging the involvement of paediatric patients
- Navigating the route towards surrogate endpoint validation
9:30 am Developing a Roadmap to Address Increasing Patient Awareness, APOL1 Testing & Study Participation: Lessons for Genetic Kidney Disease Drug Development
Synopsis
- Increasing disease awareness and genetic testing for APOL
- Enhancing patient participation and awareness in clinical studies
- Bringing the kidney community together to advance kidney health and address disparities
10:00 am Panel Discussion: A Global Initiative: Increasing Collaborative Work to Unite Towards a Streamlined Trials Process
Synopsis
- Agreeing on samples and linked clinical data to decipher the relationship between the biomarker and clinical outcomes
- Collecting patient databases by improving the global repository
- Including study coordinators to aid in prioritising measurements
10:30 am Morning Break & Networking
Better Recapitulating Kidney Diseases & Elucidating Next-Generation Diagnostic Techniques to Expedite Translational Success
11:30 am Panel Discussion: Deliberating the Benefits & Challenges of Current Preclinical Models to Move Towards Translational Success
Synopsis
- Increasing the relevance and accessibility of animal models to ease and shorten the drug discovery process
- How are rats and mice used in rare and genetic kidney diseases?
- Assessing the ability to recapitulate more than one aspect of disease
12:00 pm Unravelling the Opportunities Genomics Present in Delivering Precision Medicine in the Treatment of Rare Kidney Disorders
Synopsis
- Summarizing the progress in genomic discovery in nephrology
- Tackling the translational challenges including both diagnosis and therapy
- An overview of the emerging platforms that may enable unexpected innovation and increased opportunity for patient level benefits
Innovating Clinical Trial Design by Ensuring the Evolvement of Registries & Enrolment to Improve Patient Access & Treatment
11:30 am Pooling Observational & Clinical Trial Data Sets to Evaluate Long-Term Outcomes
Synopsis
- Combining data from a myriad of different studies and creating a cohort which can evaluate long-term outcomes across various subgroups of patients with rare conditions
- Unravelling its clinical implications in rare and genetic kidney diseases
12:00 pm Using Real-World Evidence to Increase Patient Diversity in Rare Disease Early Development Trials
Synopsis
- Racial and ethnic minority populations have historically been underrepresented in rare disease early phase trials
- Reducing barriers to clinical trial diversity is essential for health equity
- Incorporation of real-world evidence into trial planning has the potential to increase recruitment and enrolment of diverse patient populations
12:30 pm Lunch Break and Networking
Discovering Novel Approaches to Trial Designs to Transform Current Studies for Rare & Genetic Kidney Disease
1:30 pm Harnessing Advanced Genome Sequencing to Diagnose Genetic Syndromes & Uncovering Underlying Pathophysiology
Synopsis
- Incorporating genomic and molecular profiling in routine clinical practice to improve diagnosis and a new era of tailored therapies
- Elucidating clinical and genetic characteristics of patients with rare diseases that manifest differently
- Interrogating genome-wide association studies to advance understanding of pathophysiology and disease mechanisms