Agenda at a Glance

Orphan Drugs & Rare Diseases - Conference Day 2

Day 2: Tuesday - 4th April 2023

8:00 am - Conference Registration and Continental Breakfast

8:50 am - Chairpersons Opening Remarks and Summary of Day 1

9:00 am - Keynote Panel Discussion: Implications of Covid-19 Pandemic to Rare disease community

Major challenges that each stakeholder encountered during the pandemic
Lessons learned from Covid-19 and how we can benefit from it?
How to build specific needs, support, and recommendations for sustainable healthcare systems that provide directions for better preparedness in the future?

Moderator:

Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck

Panellists:

Rebecca Stewart, CEO, RARE Revolution Magazine
David Ross, Patient Advocate, Rare Disease Mental Health
Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd

9:45 am - The future environment of orphans in Europe

The aim is to provide an overview of the recent discussions and updates from the European Commission concerning the revision of the orphan drug legislation in Europe.

Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck

10:15 am - Spotlight Presentation: Successful Trial Delivery in Rare Indications

Every data point is precious
Optimal relationships with hospital sites are essential
Patient centricity must inform all stages of the development process

Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd

10:45 am - Morning Networking & Refreshments

11: 15 am - Spotlight Presentation: High Patient Engagement + Standard Data? Absolutely Achievable

How to improve patient engagement during studies while collecting standardized, regulatory-grade data
Clinical and hybrid studies to improve real-world data collection for drug development

Dr Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe

11:45 am - Rare and Ultra-Rare Diseases. Prioritisation and Sustainability

What is the state of play in rare and ultra-rare diseases and what needs to happen to have a
sustainable future?
Input gathered from 18 advocacy organisations/healthcare professionals
Awareness / diagnosis / access to treatment / driving research and innovation / impact of covid /
patient perspectives / health economist perspectives of EU drug policy / industry perspective
Public perception survey data around awareness of rare diseases and attitudes towards their
prioritisation

Paul Greenland, President EMEA, Amryt Pharma

12:15 pm - Spotlight Presentation: A strategic framework to guide Rare Disease Drug Development planning.

Rare Disease Overview
Framework for successful Rare Disease Drug Development
Ergomed Rare Disease Innovation Center Q&A

Zizi Imatorbhebhe, MBA, MS, PMP®,Senior Vice President, Global Strategy and Development, Rare Disease, Head Rare Disease Innovation Center, Ergomed

12:45 pm - Spotlight Presentation: The importance of patient identification in the use of precision therapies for rare cancers and blood disorders

Clinical trial design and execution
Collaboration across key stakeholders through development and regulatory processes
Blueprint Medicines has demonstrated success in Europe

Andrew Kim, VP International Strategic Operations and & General Manager, Global Distributor Markets,
Blueprint Medicines

1:15 pm - Networking Lunch Break

2:15 - 2:45, 2:50 - 3:20 pm - ROUNDTABLE DISCUSSIONS - divided between 2 sessions of 30 mins. (5 mins. to change table) - Attendees have 2 round table options to attend

TABLE 1 - Challenges and Opportunities in developing a treatment for rare oncology

Andrew Kim, VP International Strategic Operations and & General Manager, Global Distributor
Markets, Blueprint Medicines

TABLE 2 - Value of partnering with advocacy organizations to create innovative patient-driven solutions

Carole Scrafton, CEO & Co-Founder, FibroFlutters
David Ross, Patient Advocate, Rare Disease Mental Health
Paul Lim, Associate Director, Patient Advocacy, Blueprint Medicines

TABLE 3 - Patient-centric clinical development

Karen Matthews, Business Development Manager, CRNCC, NIHR Clinical Research Network

TABLE 5 - Drug repurposing studies in rare cancers in HICs and LMICs

Farhat Khanim MD, Director of Research, Institute of Clinical Sciences, University of Birmingham

TABLE 6 - Optimising rare disease clinical trial recruitment and retention in a post-COVID-19 world

Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd

3:20 pm - Afternoon Networking Break

3:50 am - FDA’s Rare Pediatric Disease Designations and Priority Review Vouchers

History
Mechanism: how it works
The increasing number of RPDDs
Past, present, and future value of Priority Review Vouchers

Christian Girard, Strategic Consultant, Orphan Drugs Industry Advisory & Intelligence

4:20 pm - Closing Panel Discussion: Global collaborations to improve diagnosis, access and care for persons living with a rare disease

Breaking down barriers for access
Strengthening health systems around the world and accelerate pathways to diagnosis
Stakeholders to address the rare diseases’ ecosystem gaps to collaboratively build a sustainable roadmap for better health and a brighter future for the patients suffering from rare diseases.
Challenges and opportunities in creating sustainable healthcare for all.

Moderator:
Daniel Lewi, Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.

Panellists:

Farhat Khanim MD, Director of Research, Institute of Clinical Sciences, University of Birmingham
David Ross, Patient Advocate, Rare Disease Mental Health
Arvin M. Gouw, PhD, VP of Research & Development, Rare Genomics Institute

5:00 pm - Chairperson's Closing Remarks