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WODC Barcelona 2017
November 13 @ 08:00 - November 15 @ 17:00 UTC+0
Pre-Congress Workshops - 13 November 2017
- Afternoon Workshop Hosted by Mapi Group: 13 November 2017 @ 13:00 - 17:30
Main Event - November 14 - 15, 2017
- Conference Day 1: 14 November 2017 @ 09:00 - 18:00
- Conference Day 2: 15 November 2017 @ 09:00 - 18:00
Avenida Diagonal 661 671, Barcelona, Spain, 08028
Join Mapi's experts @ WODC Barcelona 2017! Visit us at Booth #5
The 8th annual World Orphan Drug Congress is the marketplace for orphan drug professionals looking at the complete value chain of orphan drug development, from clinical development and R&D to corporate development and market access. Being Europe’s largest Orphan Drug Congress, this event will provide a platform for you to showcase your thought leadership and expertise pharma, biotechs, payers, regulators and patient advocates.
Join us in November and make sure you are at the forefront of the orphan drug industry. Regardless of whether your interest lies in research, clinical development, patient access, global pricing and reimbursement or just to engage with patient advocacy groups more intimately, we have content, networking and potential partners for you. Take just three days out of the office to meet with 400 potential customers who need to find solutions to challenges around the commercial, regulatory and scientific issues in orphan drugs.
Through strategic keynote plenaries, themed tracks, and dedicated networking sessions, you have the chance to really tailor the event to the needs of your day-to-day role. Book your place today via the show website at www.terrapinn.com/orphandrugeu.
Mapi's Pre-congress Workshop Agenda:
Assuring success from Regulatory to Global Market Access of Rare Disease Treatments
Presentation #1: "Partnering with Patients for Clinical Success"
Kelly Franchetti, RN, CCRN, CEN - Vice President, Global Patient Insights and Engagement (PI&E), Patient-Centered Sciences (PCS), Mapi Group
This interactive workshop will introduce participants to a 360° approach to obtaining patient insights and applying them to 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 analyzed 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
Presentation #2: "An Introduction to the Science of Endpoint Design in the Current Regulatory & HTA Environment in the US & Europe"
Rory Graham - Senior Director, EU Regulatory Services, Mapi Group
Martine Zimmermann - Global Head of Regulatory Affairs, Alexion Pharma GmbH
- 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
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.
Dr Will Maier, Chief Scientific Officer and Head of Patient-Centered Sciences, Mapi Group
17.00 End of Workshop Followed by Networking Drinks Hosted by Mapi Group
Featured Mapi Presentations/Speakers @ #WODCEurope's Main Event (2-Day Congress):
14th November 2017 @
standalone presentation / 14:45 (* Presentation time is subject to change): Making sure your patients understand: A review of translation methods for consistency of patient-facing documents for rare disease research in international trials - Compliance, Adherence, Recruitment, and Retention are critical to the success in rare diseases research is an issue and research is often led in multiple countries and culture. Most studies invest insufficient attention to the details presented through the patient journey creating inconsistencies and miscommunications. Documents provided to the patients must be consistent across languages and cultures and respect the intent of the original including Consent, PRO measures and adherence instructions while taking into consideration the specificities of each target country. The presentation of today will introduce methods of translating patient-faced documents, e.g., informed consent forms, or patient-reported outcome measures, and will provide a rationale for these methods in developing best practices, and selecting the right cost-effective method for the content; as well issues that may be encountered if not followed rigorously. Examples will be provided.
Register to attend: