Orphan Drugs and Rare Diseases Conference: Life-saving Therapies, Critical Issues, and Strategies to Enhance Orphan Drug Development in near future (London, United Kingdom – October 9-10, 2023) – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Orphan Drugs and Rare Diseases Conference” conference has been added to ResearchAndMarkets.com’s offering.

The 4th Annual Orphan Drugs and Rare Diseases Conference, to be held on October 9-10 in London, UK, aims to provide a valuable and broad platform to discuss recent advances in the development of life-saving therapies, critical issues, and strategies to enhance orphan drug development in near future.

With the enforcement of the 1983 Orphan Drug Act (ODA), manufacturers are obliged to scale production to treat rare/orphan diseases, leading to a drastic advancement in the landscape of orphan drugs. Novel therapies aimed at treating these uncommon diseases are paramount to fulfilling the unmet needs of patients.

The present-day challenges, including constrained resources, disease severity, limited patient access, and a lack of available knowledge in rare diseases, necessitate the discovery of innovative solutions to hasten orphan drug development.

The conference will bring together scientists, researchers, entrepreneurs, academicians, medical officials, and technologists across the globe to discuss the newest findings in the areas of rare diseases and orphan drugs, helping to shape present and upcoming challenges in drug analysis.

It gives participants the chance to interact with industry peers and participate in this scientific conference while staying informed about the most recent developments in the study of rare diseases.

WHAT TO EXPECT

Key Highlights

• 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

Why Attend?

• 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.

Who Should Attend:

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

Agenda:

Day 1 Mon, 09-Oct-2023

• Registration – 08:15 – 08:50
• Welcome note – 08:50 – 08:55
• Opening Remarks from the Chairperson – 08:55 – 09:00
• Keynote Presentation – 09:00 – 09:30

CURRENT TRENDS AND LANDSCAPE OF ORPHAN DRUGS

• Latest Developments in Orphan Drug Regulation
• Dairine Dempsey
• Dairine Dempsey, Non-Executive Director, Soleno Therapeutics, Inc. – 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
• Enhancing Orphan Drug Innovation and clinical development to facilitate patient access – 11:15 – 11:45
• Effective strategies to scale – up commercializing & of orphan drugs – 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

APPROACHES TO AID AND CURE RARE DISEASES

• Cell and gene therapy for rare diseases: a promising path to treat rare diseases – 13:45 – 14:15
• Development of multi-omics methods for RD diagnosis – 14:15 – 14:45
• Computational Modelling: Faster Way to Predict Rare Diseases – 14:45 – 15:15
• Inclusion of Artificial Intelligence tools in patient data access & identifying RD – 15:15 – 15:45
• Personalized Treatment For patients with RD – 15:45 – 16:15
• Evening Refreshments and Poster Presentation – One-to-One Networking Meetings – 16:15 – 17:15
• Panel Discussion Session – 17:15 – 18:00
• Closing Remarks from the Chairperson – 18:00 – 18:05
• Drinks Reception & Networking – 18:05 – 18:05
• End of Day 1 – 18:05 – 18:05

Day 2 Tue, 10-Oct-2023

• Registration – 08:15 – 08:50
• Welcome note – 08:50 – 08:55
• Opening Remarks from the Chairperson – 08:55 – 09:00
• Keynote Presentation – 09:00 – 09:30

ANALYZING EXISTING ROADBLOCKS & OPPORTUNITIES

• Major development challenges at the clinical stage – 09:30 – 10:00
• Orphan Drugs: Pricing and Reimbursement & Patient access – 10:00 – 10:30
• Morning Refreshments and Poster Presentation – One-to-One Networking Meetings – 10:30 – 11:15
• Launching Orphan Drugs in Underserved Markets
• Rudiger Schulze
• Rudiger Schulze, Vice President and General Manager, Germany and Central / Eastern Europe, Ultragenyx Pharmaceutical Inc. – 11:15 – 11:45
• Solution Provider Presentation – 11:45 – 12:15
• Global Collaborations between big pharma and regulatory authorities for faster drug development – 12:15 – 12:45
• Lunch and Poster Presentation – One-to-One Networking Meetings – 12:45 – 13:30

HEADING A WAY TOWARDS FIGHTING RARE DISEASES: ADVANCES IN NEAR FUTURE

• Diagnosis of rare genetic disorders: Advances and Challenges – 13:30 – 14:00
• Novel Treatment Strategies and regenerative therapies for treating multiple Rare Disorders – 14:00 – 14:30
• AI and machine learning impacting drug discovery in the rare disease space – 14:30 – 15:00
• Closing remarks from the Chairperson – 15:00 – 15:05
• Afternoon Refreshments and Poster Presentation – One-to-One Networking Meetings – 15:05 – 15:05
• End of the conference – 15:05 – 15:05

Speakers

Lara Haidar

Rare Disease Lead, Sanofi

Pedro Borga

Senior Manager, Market Access operations, Amicus Therapeutics

Rudiger Schulze

Vice President and General Manager, Germany and Central / Eastern Europe , Ultragenyx Pharmaceutical Inc.

Giovanna Barcelos

Senior Manager, Value & Evidence Global HEOR (Rare Diseases), Pfizer

Senior Representative, Takeda

Dairine Dempsey

Non-Executive Director, Soleno Therapeutics, Inc.

Kristin Smedley

Co – Founder, Thriving Blind Academy

Katarina Piponi

Pre-clinical Translational Research Associate, Orchard Therapeutics

For more information about this conference visit https://www.researchandmarkets.com/r/rm2pau

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