5th Annual MarketsandMarkets

Orphan Drugs and Rare Diseases Conference – Germany, Europe

14th – 15th October 2024

Germany, Europe

Empowering Progress: Navigating Orphan Drugs & Rare Diseases from Regulation to Detection & Precision Treatments

Join us at our MarketsandMarkets 5th Annual Orphan Drugs and Rare Diseases Conference in Germany, Europe, for a groundbreaking exploration of the recent advances in developing life-saving therapies, technologies in diagnosing, and strategies to enhance orphan drug development.

The conference will focus on the wide scope of orphan drug developments, critical issues of elevated pricing, reimbursement & access to patients, and strategies to enhance access to novel diagnostics and effective therapies for rare to ultra–rare diseases.

The event will bring together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors, and solution providers. It is an unrivalled opportunity to network and exchange invaluable knowledge with the expert leaders in the rare disease community – patient advocates, policy makers, researchers, clinicians, healthcare professionals, healthcare industry representatives, academics, payers, regulators across the globe. 

Have a chance to discuss the newest findings in rare diseases and orphan drugs, helping to shape present and upcoming challenges in drug analysis, allowing participants 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.

We invite you to join us in pushing the boundaries of innovation, forging collaborative partnerships, and creating a brighter future for the millions living with rare conditions across Europe and beyond!

WHAT TO EXPECT

  • Overview and Regulatory Updates: Current Landscape and Regulatory Developments in EU Market.
  • Emerging Therapies for RDs: Recent breakthroughs in CRISPR-Cas9 technology, Cell & Gene Therapies, Regenerative Therapies & Precision Treatments.
  • Rare Disease and Genetics: Rare Genetic Disease Mechanisms and Therapeutic Targets.
  • Patient Advocacy, Engagement & Collaboration: Critical Role of Patient Advocacy and Global Collaboration in Research and Development.
  • Global Perspectives and Market Access: Global market dynamics and market access strategies & roadblocks for orphan drugs.
  • Drug Discovery & Clinical Trial design: Novel drug discoveries and clinical trial implementation & management for rare diseases.
  • Diagnostics & Care: Biomarker Discovery, AI/ML Models, and Genetic Testing, NGS Technologies for Early Detection and Monitoring.
  • Pricing, Reimbursement & HTA: Health Technology Assessment on decision-making, challenges on high pricing, reimbursement & Commercialization.
  • OD Manufacturing and Supply Chain: Specialized manufacturing and supply chain solutions for orphan drugs.
  • 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.

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.

Departments:

  • 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

Others:

Payers, Patient Advocates, Regulators and HTA

CONFERENCE AGENDA

Registration

08:15 - 08:50

Welcome note from MarketsandMarkets

08:50 - 08:55

Opening Remarks from the Chairperson

08:55 - 09:00

Keynote Presentation - European Orphan Medicinal Product Regulations: Whats Changing?

09:00 - 09:30

CURRENT LANDSCAPE, CHANGING OD REGULATIONS TO EARLY ACCESS PROGRAMM

How do we Navigate the Regulatory Framework of Expanded Access Programs?

09:30 - 10:00

Real-world evidence (RWE) case studies and their importance

10:00 - 10:30

Morning Refreshments | One-to-One Networking Meetings

10:30 - 11:15

Innovation vs. Repurposing: Which is better approach for rare diseases?

11:15 - 11:45

Fight for accurate patient data: Rarity of Patient Population

11:45 - 12:15

Impact of Health Technology Assessment (HTA) Frameworks on orphan drug evaluation and reimbursement decisions

12:15 - 12:45

Does orphan drug incentives prioritize innovation and patient needs effectively in Europe?

12:45 - 13:15

Lunch | One-to-One Networking Meetings

13:15 - 14:15

ANALYSING THE ROADBLOCKS IN ORPHAN DRUG DEVELOPMENT & ACCESS

Market Access Challenges for Orphan Drugs in Europe: Regulatory Hurdles and Reimbursement Policies

14:15 - 14:45

Innovating Rare Disease Clinical Trials: From Challenges to Success

14:45 - 15:15

Panel Discussion: What strategies/models should be implemented to ensure the long-term sustainability of orphan drug development and access programs?

15:15 - 15:45

Evening Refreshments | One-to-One Networking Meetings

15:45 - 16:30

Orphan Drug Pricing and Affordability in European Markets

16:30 - 17:00

Emerging markets: Challenges & Opportunities for RDs Treatment

17:00 - 17:30

Post-market surveillance systems: Monitoring OD safety and efficacy

17:30 - 18:00

Closing Remarks from the Chairperson

18:00 - 18:00

Drinks Reception & Networking

End of Day 1

18:00 - 18:00

Registration

08:15 - 08:50

Welcome note from MarketsandMarkets

08:50 - 08:55

Opening Remarks from the Chairperson

08:55 - 09:00

Keynote Presentation – Development of Innovative Therapies in Treating Rare and Ultra-Rare Genetic Disorders

09:00 - 09:30

EXPLORING ACTIONS TO IMPROVE RARE DISEASE DIAGNOSTICS AND PRECISON TREATMENTS

Gene and Cell Therapies: Exploring potential and challenges in clinical practice

09:30 - 10:00

AI’s Impact and Potential: Role of Digital Health Technologies

10:00 - 10:30

Morning Refreshments | One-to-One Networking Meetings

10:30 - 11:15

Personalized Medicine: Tailoring Treatments for Rare Disorders

11:15 - 11:45

Genetic Testing Technologies for Rare Diseases Diagnosis

11:45 - 12:15

Paediatric Rare Diseases: Diagnosis, Treatment, Long-Term Management

12:15 - 12:45

AI’s Impact and Potential: Role of Digital Health Technologies

12:45 - 13:15

Lunch | One-to-One Networking Meetings

13:15 - 14:15

PATIENT ADVOCACY & ENGAGEMENT TO COLLABORATION

Patient Advocacy Groups: Efforts in Shaping OD Development

14:15 - 14:45

OD Manufacturing and Supply Chain Solutions

14:45 - 15:15

The Integral Role of Expert Patients in Advancing Rare Disease R&D

15:15 - 15:45

Panel Discussion: Fostering Global Partnerships for Rare Disease Diagnosis and Care: work together for future OD Developments.

15:45 - 16:15

Closing remarks from the Chairperson

16:15 - 16:30

....End of the conference....

16:30 - 16:30

CONFERENCE SPEAKERS

ADVISORS

Pedro Franco

Pedro Franco

Director Europe for Global Regulatory & Scientific Policy, Merck

Anne Sophie Chalandon

Anne Sophie Chalandon

Global Rare Public Affairs - Rare Diseases Policy Head, Sanofi

Tom Meuleman

Tom Meuleman

Business Unit Manager, UCB

SPEAKERS

Daniel Scherman

Daniel Scherman

General Director, Foundation For Rare Diseases FFRD, France

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LOCATION

Venue

Germany, Europe

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