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.