4th Annual MarketsandMarkets Orphan Drugs and Rare Diseases Conference

WHAT TO EXPECT

Current landscape of Orphan Drugs across the globe.
Latest Developments in Orphan drug regulations in Europe.
Next Generation therapies treating uncommon/rare infections.
Practices to enhance access to patient data.
Clinical development & regulatory.
Digital health and Artificial intelligence.
Existing roadblocks in orphan drug development.
Patient Advocacy groups and their role in market access
Pricing and marketing plans for orphan medications.
Revolutionary changes approaching rare diseases.
Rare Genetic disorders: Novel Treatment Strategies.
Rare Neurological and oncological disorders.
Development of multi-omics methods for RD diagnosis.
Role of Personalized Treatment in rare diseases.
Regenerative Medicine Approaches to fight Complex RDs

To share and exchange the recent advances and developments in rare diseases area.
Participate in discussions with experts on how to tackle the critical issues shaping the future of orphan drugs.
The best opportunity to showcase your work and research in front of industry leaders.
Discover and brainstorm ways to advance orphan drug development and improve access to life-saving therapies.
Opportunity for networking with professionals, delegates, and speakers.

Industry: Pharma/Therapeutic Companies/Start Up’s

Designations: CEO, CSO, CMO, COO, President, Vice President, Director, Head of Department, Executive Leader, Senior Manager, Manager, Principle Scientist.

Academic: Universities, Research institutes – Professors & Researchers.

Departments:

Cell and Gene Therapy
Molecular Geneticist
Translational Science
Personalized & Innovative Medicine
External R&D Innovation
Regenerative Medicine
Rare and Ultra-Rare Diseases
Public, Regulatory, and Medical Affairs
Patient Engagement
Market Access
Patient Advocacy Groups
Program Management
Clinical Research Organizations
Health Economics Outcomes Research
Investments and Funding
Speciality Pharmacies

CONFERENCE AGENDA

Registration	08:15 - 08:50
Welcome note from MarketsandMarkets	08:50 - 08:55
Opening Remarks from the Chairperson	08:55 - 09:00
NOVEL TRANSLATIONAL APPROACHES TO AID AND CURE RARE DISEASES
Systematic Serendipity - A Novel Approach for Finding New Treatments for Unadressed Rare Diseases Ron Jortner, Founder and CEO, Aspire Biosciences	09:30 - 10:00
The hidden population: quantifying the undiagnosed burden of rare diseases Laura Webber, Chief Operating Officer, HealthLumen	10:00 - 10:30
Morning Refreshments and Poster Presentation \| One-to-One Networking Meetings	10:30 - 11:15
Developing new treatments for the ultra-rare genetic disease Alkaptonuria (AKU) Nick Sireau, Chair and CEO, AKU Society	11:15 - 11:45
Accelerate Rare Disease Diagnosis: A data driven approach to early detection Hadley Mahon , Head of Commercial & Partnerships , Mendelian	11:45 - 12:15
AI, a new pillar for the RD Lara Haidar, Rare Disease Lead, Sanofi	12:15 - 12:45
Cellular Therapy Advancements for Type 1 Diabetes and Neurodegenerative Diseases Ariel Revel, Director of medical affairs, Kadimastem	12:45 - 13:15
Lunch and Poster Presentation \| One-to-One Networking Meetings	13:15 - 14:15
FOCUSING ON DIFFERENT RARE TO ULTRA-RARE DISEASES
Focusing on diagnosis and cure of Sickle Cell Anaemia Prof. Dr. Augusta Elizabeth Koroma, CEO/founder, Sickle Cell Intervention International	14:15 - 14:45
Home Nursing a solution in DCT world Nicolas Thevenet, Director of Strategic and International Development, Euraxi Pharma	14:45 - 15:15
The European challenge to launch a rare disease product Jonathan Chapper, CEO, CHAPPER healthcare Laurent Wartelle, Business Development Manager, Intsel Chimos	15:15 - 15:45
Rare Genetic disorders: Novel Treatment Strategies. Manolo Bellotto, General Manager and Chief Strategy Officer, Gain Therapeutics	15:45 - 16:15
Evening Refreshments and Poster Presentation \| One-to-One Networking Meetings	16:15 - 16:45
Personalized Medicine: Tailoring treatments based on patient profiles	16:45 - 17:15
Advancements in Genomic diagnostics for RD diagnosis	17:15 - 17:45
Closing Remarks from the Chairperson	17:45 - 17:50
Drinks Reception & Networking	17:50 - 17:50
End of Day 1	17:50 - 17:50

Registration	08:15 - 08:50
Welcome note from MarketsandMarkets	08:50 - 08:55
Opening Remarks from the Chairperson	08:55 - 09:00
Latest Developments in Orphan Drug Regulation Dairine Dempsey, Non-Executive Director, Omnispirant Therapeutics	09:00 - 09:30
CURRENT LANDSCAPE OF ORPHAN DRUGS MARKET AND PATIENT DATA ACCESS
Market access for rare disease drugs: a mindset, not a function Pedro Borga, Senior Manager, Market Access operations, Amicus Therapeutics	09:30 - 10:00
Solution Provider Presentation	10:00 - 10:30
Morning Refreshments and Poster Presentation \| One-to-One Networking Meetings	10:30 - 11:15
The Importance of Caring for Children with Rare Diseases and Ensuring Equal Access to Orphan Drugs Alexander Tkachenko, Chairman of the Board, Circle of Kindness Foundatin	11:15 - 11:45
The value of patient involvement in Therapy Development Pathway Sheela Upadhyaya, Chair, ISPOR	11:45 - 12:15
Patient Advocacy groups and their role in enhancing market access	12:15 - 12:45
Lunch and Poster Presentation \| One-to-One Networking Meetings	12:45 - 13:45
ANALYZING THE CHALLENEGS AND OPPORTUNITIES
Launching Orphan Drugs in Small Markets Rudiger Schulze, Vice President and General Manager, Germany and Central / Eastern Europe , Ultragenyx Pharmaceutical Inc.	13:45 - 14:15
Panel Discussion: Orphan Drug Pricing and Access: Striking a Balance Between Innovation and Affordability – Views? Rudiger Schulze, Vice President and General Manager, Germany and Central / Eastern Europe , Ultragenyx Pharmaceutical Inc. Pedro Borga, Senior Manager, Market Access operations, Amicus Therapeutics	14:15 - 15:00
Navigating operational challenges in Rare Disease Simon Guiver, Senior Director - Head of Program Delivery Rare, UCB Pharma	15:00 - 15:30
Closing remarks from the Chairperson	15:30 - 15:35
End of the conference	15:35 - 15:35