Mapi Group will be a featured exhibitor at this year’s World Orphan Drug Congress in Barcelona, Spain 13-15 November 2017. Learn more about this event at www.terrapinn.com/orphandrugeu and discover which experts will be featured speakers at this conference at http://www.terrapinn.com/conference/world-orphan-drug-congress/speakers.stm.
13.30 – 17.00: Partnering with patients for clinical success
- This interactive workshop will introduce participants to a 360° approach to obtaining patient insights and applying them for the successful design and execution of clinical studies. 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 then learn how insights are analysed and applied in order to:Design studies that appeal to the targeted patients
- Determine effective and motivating recruitment methods
- Engage and communicate with patients and caregivers
- Identify retention methods that meet the needs of a specific patient population
- Develop materials that resonate with patients and caregivers
17.00 End of Workshop Followed by Mapi Group’s Networking Drinks Event
Moderator: Dr. Will Maier, Chief Scientific Officer and Head of Patient-Centered Sciences, Mapi Group
More co-speakers to be confirmed shortly
Methodologies in developing Real World Data: Engaging stakeholders in Developing Evidence Strategies
- How is expanded access used to provide treatment for patients with rare diseases?
- Compassionate Use Programs in the window between submission and commercialization
- What are the benefits of conducting clinical studies and registries for rare diseases?
- What are the types of studies conducted during different phases of orphan drug development?
- How are patient registries used to generate real-world data for rare diseases and orphan drugs?
- Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
- What are the unique challenges of conducting rare disease/orphan drug registries?
- How can proactive study management strategies successfully address these challenges?
- Potential risk mitigation strategies, and alternative registration strategies will be discussed.
Speaker: Kelly Franchetti, RN, CCRN, CEN – Vice President Global Patient Insights and Engagement, Mapi Group
Patient Representative: To be announced
An Introduction to the Science of Endpoint Design in the current Regulatory & HTA Environment in the United States & Europe
- Regulatory strategies for early scientific advice: FDA & EMA scientific advice, PRIME & adaptive pathways link to scientific advice
- The creation, categorization, and hierarchy of endpoints and requirements of different stakeholder groups, including regulatory agencies, clinicians, HTA bodies, payers, increasingly active patient groups
- Real-life examples of endpoints used in selected therapeutic area & techniques for the development and validation of new endpoints
- Highlighting guidance and publications that will be of use to newcomers to the area
- Solving the non-alignment between different agencies and geographical differences in approach
Speaker: Rory Graham, Senior Director, EU Regulatory Services, Mapi Group
Co-speaker: Martine Zimmermann, Global Head of Regulatory Affairs, Alexion Pharma GmbH
Learn more about Mapi’s WODC 2017 event involvement: http://mapigroup.com/eventcalendar/wodc-barcelona-2017-pre-conference-workshop/ & http://mapigroup.com/eventcalendar/wodc-barcelona-2017/.