• Deep dive into the continued evolution of expanded access
• Analyze real-world data as a viable opportunity to showcase value for all stakeholders
• Benefit from an in-depth case study tying expanded access to real-world data collection
• Assess key considerations for moving forward and advancing the industry

Speaker:

Dennis Akkaya
Corporate Development
myTomorrows

• Revisit the 2019 meeting by regrouping and reviewing last years ‘calls to action’
• Discover progress that has been made while uncovering key goals and areas for improvement
• Remain forward-looking to continuously strive for innovations that streamline patient access

Speakers:

Greg Tuyteleers
Senior Director, Global Operations Head - Managed Access
Johnson & Johnson

• Discuss key areas for development, regulatory and access challenges in rare diseases
• Explore the unique collaboration between different stakeholders during the journey to bring innovative medicines to rare disease patients
• Understand the main features of orphan regulatory frameworks in major jurisdictions
• Bridge approval and access to orphan medicines from a market access perspective

Speakers:

Martine Zimmermann
Global Head of Regulatory Affairs
Alexion Pharmaceuticals

María Pascual
Chief Regulatory Officer
Minoryx Therapeutics

Gabrielle Nayroles
Director of Market Access
Voisin Consulting Life Sciences

• Understand the key elements in a successful transition from early access to post approval and commercial launch, while ensuring seamless patient supply
• Consider unmet global patient demand and discuss strategies for continued access following a targeted commercial launch

Speaker:

Neil Watson
Director, Business Development
Clinigen

• Stay on the pulse of processes, stakeholder perspectives and initiatives being made in different European nations regarding accelerated access
• Compare and contrast managed access programme design and development dos and don’ts from a former EMA perspective

Speaker:

Pedro Franco
Director of Europe for Global Regulatory and Scientific Policy
Merck; Formerly Scientific Administrator, EMA

• Take an in-depth look at the creation of an Early Value Proposition (EVP), an Evidence Generation Plan and an Orphan Medicinal Product/Advanced Therapy Medicinal Product Innovative Pricing Strategy that meet the requirements of all healthcare stakeholders
• Discuss how industry can better understand the crucial role of patients and caregivers in rare diseases to promote optimal care
• Navigate various stakeholder perspectives and benefit from a case study spotlight on Inherited Retinal Dystrophies (IRDs)

Speakers:

David Schwicker
Principal
ORPHA Strategy Consulting

Orla Galvin
Director of Research and Policy
Retina International

• Examine European regulatory frameworks and guidelines for the collection and application of RWD in EAPs
• Hear examples of RWD mobilization transforming the patient journey
• Discover how RWD can be beneficial to optimizing the patient experience

Speakers:

Naomi Litchfield
Patient Advocacy Manager
Bionical Emas

Graham Sidorowicz
Vice President, Business Development, Early Access Programs
Bionical Emas

• Prepare to advocate for patient access while understanding and adhering to the practical aspects of enrolling in managed access programmes
• Strive to increase education and awareness for patients with critical conditions while communicating the opportunities accelerated pathways can provide
• Effectively advocate and serve as a liaison to provide tangible improvements to meeting patient needs

Speakers:

Theodora Weisz
Senior Director, Public Affairs and Patient Engagement – Europe
Akcea Therapeutics

Mimi Choon-Quinones MBA, Ph.D
Founder and Board Chairman
Partners for Patients (PfP.NGO)

• Delve into how companies address the setup of a managed access programme and what components are essential to a successful deployment
• Investigate key considerations for stakeholder buy-in to ultimately lead to a successful company-wide roll-out of early access programmes
• Map out the responsibilities from all parties involved to ensure smooth delivery to patients in need

Speakers:

Diana Schuhbauer
Community of Practice Leader, Pre-Approval Access/Compassionate Use, Global Product Development Medical Affairs
Roche

Ramona Reichenbach
Head, Managed Access Center of Excellence, Global Drug Development – Chief Medical Office
Novartis