MarketsandMarkets

Orphan Drugs and Rare Diseases Conference – US Edition

17th – 18th October 2024

Hilton Boston Logan Airport, One Drive, Boston, MA 02128, USA

EVENT OVERVIEW

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

Join us at our MarketsandMarkets Orphan Drugs and Rare Diseases Conference in Hilton Boston Logan Airport, One Drive, Boston, MA 02128, USA 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 US and beyond!

WHAT TO EXPECT

  • Overview and Regulatory Updates: Current Landscape and Regulatory Developments in US Market.
  • Emerging Therapies for RDs: Recent breakthroughs in CRISPR-Cas9 technology, Cell & Gene Therapies, mRNA 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, sustainable models & roadblocks for orphan drugs.
  • Drug Discovery & Clinical Trial design: Novel drug discoveries and clinical trial implementation & management for rare diseases focusing patient perspective.
  • Diagnostics & Care: Biomarker Discovery, Digital health/AI applications, 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.
  • Rare to Ultra Rare Diseases: Emerging Therapies and Distinctive Obstacles in the Identification, Therapy, and Sustained Care of different rare/Ultra Rare diseases in children and adults.
  • 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

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