From AI to Patient Advocacy: The Comprehensive Orphan Drugs and Rare Diseases Conference: London, United Kingdom – October 9-10, 2023

11 5 From AI to Patient Advocacy: The Comprehensive Orphan Drugs and Rare Diseases Conference: London, United Kingdom - October 9-10, 2023

DUBLIN, Sept. 22, 2023 /PRNewswire/ — announces the 4th Annual Orphan Drugs and Rare Diseases Conference, scheduled for October 9-10 in London, UK. This prestigious event offers an in-depth exploration into the ever-evolving world of orphan drugs, post the landmark 1983 Orphan Drug Act (ODA).


The conference will spotlight novel therapies addressing the diverse and unique challenges of rare diseases, recognizing the vital importance of meeting the pressing needs of patients. It also seeks to address the current challenges facing the field, including constrained resources, the severity of these diseases, restricted patient access, and the prevalent knowledge gap in the realm of rare diseases.

Highlights of the Conference Include:

  • A comprehensive global overview of the current orphan drug landscape.
  • Insights into next-gen therapies targeting rare infections.
  • The role of AI and digital health in accelerating drug development and improving patient data access.
  • Strategies for pricing and marketing orphan drugs.
  • The growing importance of patient advocacy groups in market access.
  • Innovative approaches in regenerative medicine for complex rare diseases.

Key Speakers Include:

  • Lara Haidar, Rare Disease Lead, Sanofi.
  • Rudiger Schulze, VP and GM, Germany and Central/Eastern Europe, Ultragenyx Pharmaceutical Inc.
  • Dairine Dempsey, Non-Executive Director, Soleno Therapeutics, Inc.
  • And many more!

About the Conference: Day 1 of the event is themed “Current Trends and Landscape of Orphan Drugs” and “Approaches to Aid and Cure Rare Diseases”, while Day 2 will delve into “Analyzing Existing Roadblocks & Opportunities” and the future advancements in tackling rare diseases.

Who Should Attend? Industry representatives from Pharma/Therapeutic Companies, CEOs, CSOs, CMOs, COOs, Vice Presidents, Directors, Heads of Departments, Senior Managers, and Principle Scientists. Academics, Professors, Researchers, and key representatives from numerous departments such as Cell and Gene Therapy, Regenerative Medicine, Rare and Ultra-Rare Diseases, Patient Engagement, Clinical Research Organizations, and more.

With a unique blend of presentations, panel discussions, and networking sessions, attendees will be provided with invaluable insights and the latest advancements in the orphan drug and rare diseases sectors.

For those eager to make meaningful connections, understand the latest scientific developments, and share innovative solutions in orphan drug development, this is a must-attend event.


Day 1 


  • Latest Developments in Orphan Drug Regulation
  • Dairine Dempsey, Non-Executive Director, Soleno Therapeutics, Inc.
  • Enhancing Orphan Drug Innovation and clinical development to facilitate patient access 
  • Effective strategies to scale – up commercializing & of orphan drugs 
  • Patient Advocacy groups and their role in enhancing market access 


  • Cell and gene therapy for rare diseases: a promising path to treat rare diseases 
  • Development of multi-omics methods for RD diagnosis 
  • Computational Modelling: Faster Way to Predict Rare Diseases 
  • Inclusion of Artificial Intelligence tools in patient data access & identifying RD
  • Personalized Treatment For patients with RD
  • Panel Discussion Session

Day 2 


  • Major development challenges at the clinical stage 
  • Orphan Drugs: Pricing and Reimbursement & Patient access
  • Launching Orphan Drugs in Underserved Markets
  • Rudiger Schulze, Vice President and General Manager, Germany and Central / Eastern Europe, Ultragenyx Pharmaceutical Inc. 
  • Global Collaborations between big pharma and regulatory authorities for faster drug development 


  • Diagnosis of rare genetic disorders: Advances and Challenges 
  • Novel Treatment Strategies and regenerative therapies for treating multiple Rare Disorders
  • AI and machine learning impacting drug discovery in the rare disease space 
  • Closing remarks from the Chairperson

For more information about this conference visit

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rt From AI to Patient Advocacy: The Comprehensive Orphan Drugs and Rare Diseases Conference: London, United Kingdom - October 9-10, 2023

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