Orphan Drugs and Rare Diseases Conference (Boston, MA, United States - October 17-18, 2024) - Navigating Orphan Drugs & Rare Diseases from Regulation to Detection & Precision Treatments - ResearchAndMarkets.com

DUBLIN--(BUSINESS WIRE)--The "Orphan Drugs and Rare Diseases Conference" has been added to ResearchAndMarkets.com's offering.

The 2 day Orphan Drugs and Rare Diseases Conference takes place in Boston, Massachusetts in the United States on October 17-18th 2024.

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.

You are invited to join 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!

Key-Highlights

• 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.

Why Attend?

• 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.

Conference Agenda:

Forging Ahead: Latest Regulatory Insights into Early Access Programs

• Keynote Presentation-Regulatory Pathways for Orphan Drug Approval: Requirements & Considerations
• Early Access Programs: Facilitating Timely Access to Orphan drugs for Patients in Need

Rare Disease Perspectives: Patient Centricity, Pricing Models & Advocacy Efforts

• Patient-Centric Care: Integrating Patient Advocacy and Support Programs into Clinical Decision Making
• Panel Discussion: What strategies/models should be implemented to ensure the long-term sustainability of orphan drug development and access programs
• Towards greater health equity - new insights for measuring disease burden and patient diversity in rare diseases.
  • Adrian Kielhorn, Senior Director, Global HEOR Neurology, Alexion Pharmaceuticals, Inc

Unveiling Challenges in Orphan Drug Discovery & Disease Management

• Market Access Challenges for Orphan Drugs: Regulatory Hurdles and Reimbursement Policies
• Clinical Trial Design and Patient Recruitment: Addressing Strategies & Challenges
• Addressing Unmet Medical Needs of Pediatric RDs Patients: Pediatric Trials and Ethical Considerations
• Palliative Care & Symptom Management: Enhancing Quality of Life of Patients
• Case Studies: Clinical Presentations & Management Strategies for Selected Rare Diseases

Enhancing Care for Rare Conditions: Approaches for Improving Diagnosis and Tailored Treatments

• Keynote Presentation - Five headwinds facing rare cancer drug developers (and what to do about it)
  • Benjamin Brown, Co-Founder & Executive Director, American Society of Pharmacovigilance
• Cell and Gene Therapies: Exploring potential and challenges in clinical practice
• The Promise of Individualized Medicines for Uniquely Rare Diseases
  • Irina Antonijevic, Chief Medical Officer, EveryONE Medicines Inc.
• Diagnostic Approaches: Genetic Testing Technology, Biomarkers and Differential Diagnosis
• MRNA Therapies for Rare Genetic Metabolic Disorders
• Telemedicine & Remote Monitoring: AI & ML Applications in RDs Diagnosis and Treatment

Global Collaboration & Monitoring Safety

• National Center for Advancing Translational Sciences: Opportunities to Move Your Rare Disease Research Forward
• Karlie Sharma, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health (NIH/NCATS)
• Panel Discussion Session: Collaborative Approaches to Rare Disease Research and Clinical Development: Industry-Academia Partnerships, Patient Registries, and Data Sharing Initiatives
• Pharmacovigilance and Monitoring of Rare Disease Patients: Ensuring Safety

Conference Speakers

• Joslyn Arnon Crowe, Executive Director, National Niemann-Pick Disease Foundation
• Donna (McKenna) Sullivan, Director of Patient Advocacy, Pathways To Trust
• Zia Choudhry, Senior Medical Director Rare Autoantibody Diseases - Immunology, Johnson & Johnson
• Neha Kumar, Global Precision Medicine Strategy Leader, Johnson & Johnson
• Harpreet Ram, President, EVR Consulting LLC
• Elyssa Benedikt, Global Rare Disease Value & Access, Sanofi
• Tara Hastings, Patient Engagement Lead, Sanofi
• Arjun Channi, Director, Global Regulatory Affairs, Sanofi
• Adrian Kielhorn, Senior Director, Global HEOR Neurology, Alexion Pharmaceuticals, Inc
• Benjamin Brown, Co-Founder & Executive Director, American Society of Pharmacovigilance
• Karlie Sharma, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health (NIH/NCATS)
• Irina Antonijevic, Chief Medical Officer, EveryONE Medicines Inc.
• Victoria Arteaga, Co-founder, SHER Hispanic Society for Rare Diseases

For more information about this conference visit https://www.researchandmarkets.com/r/nyjw37

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