Martine Zimmermann | Senior Vice President, Head of Global Regulatory Affairs
Alexion Pharmaceuticals

Martine Zimmermann, Senior Vice President, Head of Global Regulatory Affairs, Alexion Pharmaceuticals

Martine Zimmermann, Senior Vice President
Head of Global Regulatory Affairs, ALEXION PHARMA GMBH
Martine is global head of regulatory affairs at Alexion Pharmaceuticals.  She is based in Zurich, Switzerland. She has over 20 years of combined R&D and global regulatory strategy experience.  She joined Alexion in 2009 and since then has been dedicated to the registration of Orphan Medicinal Products globally as well as shaping a favorable regulatory environment for orphan drug development.
Throughout her career, Martine has been directly involved in multiple regulatory approvals across the globe and most recently 2 enzyme replacement therapies for ultra-rare metabolic disease.
Martine received her Pharm D from University Louis Pasteur in Strasbourg (France).


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

Day 1 Orphan 14th Nov @ 15:15

Tailoring drug development to meet an evolving orphan drug approval landscape

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