Archives: Agenda

INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape

  • Battling site competitiveness to reduce delays on study start-up: what can you do differently to tackle these operational challenges?
  • When there are immense pressures on the trial ecosystem, how can we build and ensure quality in an innovative way
  • Developing an optimized strategy as a small to medium-sized biotech with limited resources to stay afloat
  • Discussing the importance of protocol optimization and specialized statistical support to reduce the pressure on sites and relieve bottlenecks
  • Global health inequalities: Are rare diseases rare everywhere?

Real World Data/ Evidence in translational medicine and rare disease clinical trials

  • AMR, Evolution and history of the natural world
  • The innate immune system – a billion years Proof of concept
  • RWD RWE – surgical sites and improved outcomes in oral surgery
  • RWD RWE – Serious burns and a survival benefit
  • COVID pandemic RWD RWE pulmonary administration
  • Safety studies and tolerability
  • The evolutionary successor to antibiotics is a multi-modal anti-inflammatory via TNF-a, Il6 and Il-10
  • Key milestones and next steps: CHMP and Orphan designation

Placing fear reduction at the core of the patient experience strategy: assessing the impact of diminished anxiety and increased sense of control for rare disease clinical trials

  • Improving the sense of security to reduce unnecessary suffering and distress for patients
  • Understanding the different types of patients and their individual response behaviours
  • Being hyper aware of cultural differences and biases to ensure sensitivity and effective outcomes for all
  • Sharing best practice on effective strategies with case studies and examples to help reduce patient drop out rate

Bringing impactful data-driven leadership to all phases of drug development and successful regulatory interactions regarding new drug application

  • Discovery and preclinical phase: Target identification using big data analytics, predictive modelling for candidate selection (e.g., QSAR models) and case study for successful preclinical data-driven strategies
  • Optimizing clinical trial design: Adaptive trial designs, real-world data integration and patient recruitment and retention strategies
  • Biomarkers and precision medicine: Role in patient stratification and targeted therapies and precision medicine case study in oncology
  • Case studies and real-world examples: Success stories and lessons learned in data-driven drug development