MarketsandMarkets

Orphan Drugs and Rare Diseases Conference – US Edition

17th – 18th October 2024

Boston, USA

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 Boston, 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

CONFERENCE AGENDA

Registration

08:15 - 08:50

Welcome note from MarketsandMarkets

08:50 - 08:55

Opening Remarks from the Chairperson

08:55 - 09:00

Forging Ahead: Latest Regulatory Insights into Early Access Programs

Keynote Presentation-Regulatory Pathways for Orphan Drug Approval: Requirements & Considerations

09:00 - 09:30

Early Access Programs: Facilitating Timely Access to Orphan drugs for Patients in Need

09:30 - 10:00

Reserved for Sponsor Presentation

10:00 - 10:30

Morning Refreshments | One-to-One Networking Meetings

10:30 - 11:15

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

Patient-Centric Care: Integrating Patient Advocacy and Support Programs into Clinical Decision Making

11:15 - 11:45

Reserved for Sponsor Presentation

11:45 - 12:15

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

12:15 - 12:45

Towards greater health equity – new insights for measuring disease burden and patient diversity in rare diseases.

Adrian Kielhorn

Adrian Kielhorn, Senior Director, Global HEOR Neurology, Alexion Pharmaceuticals, Inc

12:45 - 13:15

Lunch | One-to-One Networking Meetings

13:15 - 14:15

Unveiling Challenges in Orphan Drug Discovery & Disease Management

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

14:15 - 14:45

Reserved for Sponsor Presentation

14:45 - 15:15

Clinical Trial Design and Patient Recruitment: Addressing Strategies & Challenges

15:15 - 15:45

Evening Refreshments | One-to-One Networking Meetings

15:45 - 16:30

Addressing Unmet Medical Needs of Pediatric RDs Patients: Pediatric Trials and Ethical Considerations

16:30 - 17:00

Palliative Care & Symptom Management: Enhancing Quality of Life of Patients

17:00 - 17:30

Case Studies: Clinical Presentations & Management Strategies for Selected Rare Diseases.

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

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

Benjamin Brown, Co-Founder & Executive Director, American Society of Pharmacovigilance

09:00 - 09:30

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

09:30 - 10:00

Reserved for Sponsor Presentation

10:00 - 10:30

Morning Refreshments | One-to-One Networking Meetings

10:30 - 11:15

The Promise of Individualized Medicines for Uniquely Rare Diseases

Irina Antonijevic

Irina Antonijevic, Chief Medical Officer, EveryONE Medicines Inc.

11:15 - 11:45

Diagnostic Approaches: Genetic Testing Technology, Biomarkers and Differential Diagnosis

11:45 - 12:15

MRNA Therapies for Rare Genetic Metabolic Disorders

12:15 - 12:45

Telemedicine & Remote Monitoring: AI & ML Applications in RDs Diagnosis and Treatment

12:45 - 13:15

Lunch | One-to-One Networking Meetings

13:15 - 14:15

Global Collaboration & Monitoring Safety

National Center for Advancing Translational Sciences: Opportunities to Move Your Rare Disease Research Forward

Karlie Sharma

Karlie Sharma, Program Director, National Center for Advancing Translational Sciences, National Institutes of Health (NIH/NCATS)

14:15 - 14:45

Reserved for Sponsor Presentation

14:45 - 15:15

Panel Discussion Session: Collaborative Approaches to Rare Disease Research and Clinical Development: Industry-Academia Partnerships, Patient Registries, and Data Sharing Initiatives

15:15 - 15:45

Pharmacovigilance and Monitoring of Rare Disease Patients: Ensuring Safety

15:45 - 16:15

Closing remarks from the Chairperson

16:15 - 16:20

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

16:20 - 16:20

SPEAKERS

Joslyn Arnon Crowe

Joslyn Arnon Crowe

Executive Director, National Niemann-Pick Disease Foundation

Donna (McKenna) Sullivan

Donna (McKenna) Sullivan

Director of Patient Advocacy, Pathways To Trust

Zia Choudhry

Zia Choudhry

Senior Medical Director Rare Autoantibody Diseases – Immunology, Johnson & Johnson

Neha Kumar

Neha Kumar

Global Precision Medicine Strategy Leader, Johnson & Johnson

Harpreet Ram

Harpreet Ram

President, EVR Consulting LLC

Elyssa Benedikt

Elyssa Benedikt

Global Rare Disease Value & Access, Sanofi

Tara Hastings

Tara Hastings

Patient Engagement Lead, Sanofi

Arjun Channi

Arjun Channi

Director, Global Regulatory Affairs, Sanofi

Adrian Kielhorn

Adrian Kielhorn

Senior Director, Global HEOR Neurology, Alexion Pharmaceuticals, Inc

Benjamin Brown

Benjamin Brown

Co-Founder & Executive Director, American Society of Pharmacovigilance

Karlie Sharma

Karlie Sharma

Program Director, National Center for Advancing Translational Sciences, National Institutes of Health (NIH/NCATS)

Irina Antonijevic

Irina Antonijevic

Chief Medical Officer, EveryONE Medicines Inc.

Victoria Arteaga

Victoria Arteaga

Co-founder, SHER Hispanic Society for Rare Diseases

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PARTNERS

LOCATION

Venue

Boston, USA

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