5th Annual MarketsandMarkets

Orphan Drugs and Rare Diseases Conference

14th - 15th October 2024

Freiburg, Germany

Advancing Breakthroughs: Uniting Innovation and Access for Rare Disease Treatment

Experience the MarketsandMarkets 5th Annual Orphan Drugs and Rare Diseases Conference in Freiburg, Germany, where we delve into the latest advancements in life-saving therapies, cutting-edge diagnostics, and innovative strategies to advance orphan drug development. This event is set to highlight the most significant breakthroughs in the field.

Our conference will explore the vast landscape of orphan drug advancements, focusing on critical issues such as elevated pricing, reimbursement challenges, and patient access. Attendees will gain valuable insights into improving access to novel diagnostics and effective treatments for rare and ultra-rare diseases, ensuring comprehensive discussions on these essential topics.

This gathering will unite a diverse array of stakeholders, including leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors, and solution providers. It provides an unparalleled opportunity to network and exchange invaluable knowledge with key figures from the rare disease community—patient advocates, policymakers, researchers, clinicians, and healthcare professionals.

Engage in dynamic discussions about the newest findings in rare diseases and orphan drugs, addressing both current and upcoming challenges in drug development. Participants will have the chance to interact with industry peers, staying informed about the latest developments and cutting-edge research in the field of rare diseases.

Take this opportunity to push the boundaries of innovation, forge collaborative partnerships, and create a brighter future for the millions affected by rare conditions across Europe and beyond. Be part of this pivotal event and help shape the future of rare disease treatment and care!


Industry: Pharmaceutical/Therapeutic and Biotechnology Companies

Designations: Chief Executives, President, Vice President, Director, Head of Department, Executive leader, Senior Manager, Manager, Principal Scientist, Scientists.

Academic: Hospitals, Universities, Research institutes – Professors & Researchers.


  • Research and Development
  • Pricing and Reimbursement
  • Commercial Development
  • Clinical Development
  • Cell and Gene Therapy
  • Genomics & Genetics
  • Epidemiology
  • Digital Health & Artificial Intelligence
  • Rare Oncology & Neurology
  • Personalized & Innovative Medicine
  • External R&D Innovation
  • Regenerative Medicine
  • Rare and Ultra-Rare Diseases
  • Public, Regulatory, Medical   Affairs
  • Patient Engagement
  • Therapeutic Are Lead
  • Market Access/Value Access
  • Patient Advocacy and Supply Chain
  • Program Management
  • Health Economics Outcomes Research
  • Investments and Funding
  • Specialty Pharmacies


Payers, Patient Advocates, Regulators and HTA

  • Overview and Regulatory Updates: Current Landscape and Regulatory Developments in EU Market
  • Emerging Therapies for RDs: Gene Editing, Cell & Gene Therapies, Regenerative Therapies.
  • Rare Disease Diagnostics & Care: Novel Methods, Biomarker Discovery, AI/ML, and Genetic Testing Technologies for Early Detection and Monitoring.
  • Rare Disease Genetics and Genomics: Unveiling Rare Genetic Disease Mechanisms and Therapeutic Targets
  • Patient Advocacy, Engagement & Collaboration: Role Patient Advocacy and Global Collaboration in Research and Development.
  • Health Economics & Market Access: Barriers to market access for orphan drugs and strategies ensuring patient affordability and accessibility.
  • Drug Discovery & Clinical Trial design: Novel drug discoveries and clinical trial methods for rare diseases.
  • Uncommon genetic conditions: Recent Advancements in gene therapy and personalized therapies for rare genetic disorders.
  • Pricing, Reimbursement & HTA: Health Technology Assessment on decision-making, challenges on high pricing, and reimbursement negotiations.
  • Paediatric Rare Diseases: Emerging Therapies and Distinctive Obstacles in the Identification, Therapy, and Sustained Care of rare diseases in children.
  • Ethical and Legal Considerations: Ethical dilemmas, patient privacy concerns, and legal issues surrounding rare disease research, and data sharing.

  • Hear from influential keynote speakers who share their experiences, insights, and visions for the future.
  • Get access to the latest research findings, innovative therapies, and breakthroughs in the field.
  • Platform to exchange insights, discoveries, and best practices in the realm of orphan drugs and rare diseases.
  • Participate in discussions with experts on how to tackle the critical issues shaping the future of orphan drugs.
  • Discover and brainstorm ways to advance orphan drug development and improve access to life-saving therapies.
  • Gain awareness about rare diseases and the challenges associated with developing treatments for these conditions.
  • Collaboration & Networking Opportunities between academia, industry, and advocacy groups, fostering partnerships aimed at accelerating research, drug development, and access to therapies for rare diseases.



Tom Meuleman

Tom Meuleman

Business Unit Manager, UCB


Avencia Sanchez-Mejias

Avencia Sanchez-Mejias

CEO and Co-founder, Integra Therapeutics

Nigel Nicholls

Nigel Nicholls

General Manager, Egetis Therapeutics

Augusta Elizabeth Koroma

Augusta Elizabeth Koroma

Director, European Sickle Cell Federation (ESCF)




Freiburg, Germany