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Pre-Conference Workshops

Workshop A: Effectively Utilizing Neuromuscular Biomarkers in Clinical Trials

Wednesday, October 23 | 9:00am-12:00pm

This workshop will address how to use biomarkers effectively within clinical trials to enrich the patient selection, provide evidence of target engagement and understand which mechanisms reflect an effect and identify responders. Biomarkers can provide additional data to support clinical programs, especially in CNS disorders, that have recently seen a high rate of Phase 3 failures.

By attending this workshop, you will:

  • Understand how specific biomarkers interact with your drug/treatment, including how variables may affect the results (i.e., time of day, processing protocols, gender of subject)
  • Learn what processes and training you need in your clinical trial to ensure quality assessments and biomarker results
  • Discover what the future holds for novel biomarkers and personalized medicine, including telemedicine and wearable devices and technology

Workshop Leader:

Susan Ward - Brainstorm Cell

Susan Ward
Head of Clinical Operations
Brainstorm Cell Therapeutics

Workshop B: Linking the Regulatory & Reimbursement Landscape for Neuromuscular Disorder Drug Development

Wednesday, October 23 | 12:30pm-3:30pm

With the recent approval of multiple neuromuscular therapeutics, understanding of both the regulatory and reimbursement expectations and requirements is essential. The influence of the FDA and EMA on clinical trial design and supporting early approval balanced against
the challenges of securing reimbursement will be discussed.

Attend this workshop to:

  • Explore what is needed to align your data generation for both regulatory and reimbursement
  • Assess the regulatory and economic challenges for neuromuscular therapeutic approval
  • Debate the cost of drugs, access and reimbursement

Workshop Leader:

Untitled design (97)

Elin Haf Davies
Research Fellow
Bangor University