As reports predict the global biomarker test market will exceed $16bn in coming years, the ability to identify and utilize predictive, diagnostic and prognostic biomarkers is a priority for drug developers. In rare disease, affecting over 400 million worldwide and where clinical insights exist for only around 400 of the 7000+ disease identified, biomarkers are a valuable tool to accelerate drug development where there is current great unmet need. With this, we were excited to announce the launch of the inaugural Biomarkers for Rare Diseases Summit.
inaugural Biomarkers for Rare Diseases Summit.
Uniting world leaders developing novel therapeutics for rare disease, we discussed learnings, challenges and future directions at the only summit dedicated to approaches to identify, test and proof clinical biomarkers in rare disease for regulatory approval. From identifying disease driving biomarkers, robust assay validation, harnessing latest technologies and demonstrating therapy efficacy, this was the definitive platform to accelerate bench to bedside rare disease development towards regulatory filing and patient populations.
Explore the Characterization and Identification of Disease Driving Biomarkers
Considering the genotypic and phenotypic variance of disease, attendees discussed approaches and hurdles to identify and discover biomarkers, and how this informs future data collection and study design with Vifor Pharma, Center for Genomic Interpretation, NIH, Regulus Therapeutics & Boston Children’s Hospital
Design Robust Biomarker Validation Plans for Meaningful Conclusions
With the variability of biomarkers over time and assay analytical validation a significant hurdle, we heard progress and strategies to better define biomarkers and support their use as a drug development tool from the likes of Novo Nordisk & Foundation for the National Institutes of Health
Demonstrate the Clinical Utility of Biomarkers for Rare Disease Drug Development
From retrospective and prospective data informing patient identification for clinical trials to novel natural history study design, exclusive clinical case studies and insights were revealed from Diamyd, Triplet Therapeutics & CHDI Foundation.
Show Therapy Efficacy Demonstration in Preparation for Regulatory Filing
As regulatory agencies endorse the need for flexibility in the review process, the attendees considered how biopharma is approaching the definition of acceptable endpoints and discussed navigating regulation pathways and a potential future roadmap for the approval of their pipelines with Takeda & Foundation for the National Institutes of Health
Detect & Diagnose Disease Earlier Harnessing Emerging Techniques and Technologies
Breakthroughs in technology are driving industry’s ability to diagnose disease earlier, investigate pathophysiology and identify new drug targets, we heard expert opinions on technologies you should consider as well as their cost and practical application from Rady Children’s Institute for Genomic Medicine, Johnson & Johnson & Passage Bio
The Expert Speaker Faculty Included:
Global Clinical Development TA Lead Nephrology & Orphan Disease Area
National Cancer Institute, NIH
Vice President & Senior Medical Officer, Global Chief Medical Office
President & Chief Executive Officer
Director, Hematology Lab Professor of Pathology
Harvard Medical School
Co-Founder & Chief Executive Officer
Center for Genomic Interpretation
Associate Director, Quantitative Sciences
President & Chief Executive Officer
Rady Children’s Institute for Genomic Medicine