Pre-congress Workshops

Pre-congress Workshops Nov 6


Morning Pre-congress Workshops

Dr Renato Dellamano

Workshop A: Pricing, Reimbursement and Market Access Challenges for Orphan Drugs and Cell and Gene Therapies

  • Orphan Drugs and Advanced Therapy Medicinal Products (ATMPs) in the EU: Pricing, reimbursement and market access challenges
  • Guided panel discussion – What pricing, reimbursement and market access models for Orphan ATMPs?
  • Unique challenges in the manufacturing and commercialization of gene therapies for rare diseases
  • Building a value dossier for personalized gene / cell therapies for rare diseases
Daniel O'Connor

Workshop B: Drug Repurposing – A Sufficient Business Model to Drive Orphan Drug Development?

  • What is a sustainable approach to repurposing generic drugs for rare diseases?
  • Can repurposing truly deliver a transformational therapy?
  • How can this be incorporated into a drug development business model and strategy?
  • How to get national healthcare providers and regulators on-board? Are there gaps in the regulatory frameworks
  • Considerations: How does drug repurposing differ? The more obvious benefits and lesser known challenges
  • What about off label repurposing?

Networking Lunch Break


Afternoon Workshops

Kelly Franchetti

Workshop C: Ensuring Success of Orphan Disease Drug Development and Market Access

Orphan drug development has several unique challenges. This workshop provides expert guidance and practical case studies to help address the major challenges of drug candidate early development, regulatory strategy, trial design, patient engagement and market access.Endpoint design and selection, and conditional approval pathways in the current regulatory and HTA environment for Orphan medicines in the US and Europe. Discussions on the use of registry studies to facilitate ,enhance and maintain product approvals.Rory Graham, BPharm, MBA (Senior Director, Regulatory Affairs, Europe & International Region, Mapi SRS, an ICON plc. Company)The Orphan medicines development environment is challenging in terms of endpoint design, statistical validation, patient recruitment, and regulatory pathways. Rory will review endpoint strategies for Orphan diseases, highlight current trends with the major regulators, and highlight the opportunity for earlier approvals that can be afforded by early and thoughtful integration of registry studies into the development plan, with reference to both FDA an EMA initiatives in this area.Engaging Patients and Advocacy Groups for Patient-Centric Trial Designs and the Promotion of Rare Disease Clinical TrialsKelly Franchetti (VP, RN, CCRN, CEN Patient Insight and Engagement, ICON)This interactive session will introduce participants to a 360° approach to obtaining and applying patient insights, and for engaging advocacy groups to improve trial design, operations, promotion, and recruitment. Through engaging activities, we will address how to get to the heart of the patient’s decision-making process, assess their physical/emotional/social needs, and identify their motivators, barriers and influencers as they relate to clinical study participation. Participants will also learn best practices for engaging with patient advocacy groups throughout the product development lifecycle.Approaches to successful execution of rare disease clinical trialsCynthia Dukes (VP, Drug Development, Rare Disease, ICON)This session will discuss the elements related to successful execution of rare disease studies. Rare disease studies require different work attitudes and different approaches to site selection, site support, patient support, and team requirements.They are not business as usual!Market Access and Supporting Research for Rare Disease TreatmentsWill Maier, PhD, MPH (Chief Scientific Officer, Commercialisation and Outcomes, ICON)This session will discuss the reimbursement and market access for rare disease treatment including regulations and additional research needed for a successful application and reimbursement profile.
Sheela Upadhyaya

Workshop D: Managed Access…Are they the holy grail?

  • How will the development of the EUnetHTA initiative support the managed entry agreement development and data collection, can it become a joint European venture too?
  • Success stories and learning from a variety of jurisdictions, discuss best practices from multiple perspectives and provide a hands-on opportunity to craft MEAs for specific therapies

End of workshops and welcome drinks Hosted by ICON

last published: 10/Aug/18 11:05 GMT



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