Kelly Franchetti | Vice President, Global Patients Insights and Engagement
Mapi Group

Kelly Franchetti, Vice President, Global Patients Insights and Engagement, Mapi Group

Kelly Franchetti has nearly two decades of direct patient experience, gained in both hospital and industry settings including CROs, healthcare
technology, patient research and hands-on patient care across multiple therapeutic areas. 
Ms. Franchetti has been involved in the development and moderation of advisory boards, patient HCP and KOL forums, and has collaborated
with Patient and Clinical Advocacy groups. 
This positions her to contribute significant insights into how patients regard their treatment options and how to best approach patient recruitment and retention strategies.


Pre-congress Workshop Day Orphan @ 13:10

Afternoon Pre-congress Workshop Monday 13 November

Assuring success from Regulatory to Global Market Access of Rare Disease Treatments

13:40 – 14:30

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
Kelly Franchetti, Vice President Global Patient Insights and Engagement, Mapi Group

14:30 – 15:30

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

16:00 – 17:00

An introduction to the science of endpoint design in the current regulatory and HTA environment in the US and 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
Rory Graham, Senior Director, EU Regulatory Services, Mapi Group
Martine Zimmermann, Global Head of Regulatory Affairs, Alexion pharma GmbH
17.00 End of Workshop Followed by Networking Drinks Hosted by Mapi Group

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