WORLD ORPHAN DRUG CONGRESS USA - DAY TWO

 

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World Orphan Drug Congress USA 2017 - Conference Day Two

08:15

REGISTRATION AND NETWORKING BREAKFAST

Scott Gray
08:50

Chairperson’s Opening Remarks

GENE THERAPY

Sven Kili
09:00

Keynote address: Development of lifesaving gene therapies through collaboration between academic discovery, scientific research, medical practice and product development

  • How can cell and gene therapy programs be developed through collaborative efforts with academia and lead to more approvals?
  • Designing a trial when using a patient’s own gene modified stem cells to correct immunodeficiency’s 
  • Overcoming challenges of small population sample, skepticism of the community and high pricing reputation 
Nick Leschly
09:20

Keynote address: Transforming the life of children on a genetic death sentence by giving them a bluebird day

  • Overview of the clinical development program for cerebral adrenoleukodystrophy and beta-thalassemia
  • Overcoming challenges in the design and interpretation of the human clinical trials, immunogenicity, and the selection of the best primary efficacy end point to progress clinical development 
  • How to balance the science and medicine being developed with the expectations of payers and regulators? 
Matt Kapusta
09:40

Keynote address: Adeno-associated virus (AAV)-based gene therapies for rare, chronic and degenerative diseases

  • From restoring vision in canines to restoring vision in humans 
  • Developing safe and efficacious trials in a field tainted by Jesse Gelsinger’s death  
  • Navigating the regulatory landscape and building the value proposition to payers 
  • Preparing for commercialization of the first to be approved gene therapy in the US
Jeffrey D. Marrazzo
10:00

Keynote address: Driving towards success in gene therapy development: pioneering a different approach for differentiated outcomes in rare diseases

• Inherited Retinal Disease (IRD): a collaborative effort between scientists, patients and regulators during a decades-long journey 
• Building a fully-integrated company to prepare for a potential first-ever U.S launch
• Considering potential new value propositions for patients, payers and the health care system
• Gene therapy platform development: lessons learned
10:20

SPEED NETWORKING

10:40

MORNING NETWORKING BREAK

SCIENTIFIC INNOVATION

REGIONAL MARKET ANALYSIS

11:20

Tackling the rare disease deficit – the advent of oral mutation specific therapies

• The nonsense mutation read-through agent represents a novel platform for treating many rare diseases 
• Insights from Translarna (ataluren) studies in nonsense mutation DMD, CF and beyond 
• The alternative splicing platform and its application to SMA & Huntington’s Disease
11:20

Initiative on rare and undiagnosed diseases in Japan (IRUD): A nation-wide trigger for clinical data sharing

  • Data sharing to improve diagnosis, global collaboration and the grouping of patients with similar diseases
  • Building regional alliances by bringing together community clinics, Clinical Centers, Analysis Centers for nation-wide medical network
  • Genotypes and phenotypes in rare and undiagnosed disease patients in Japan
  • The Tohoku Medical Megabank: providing the research infrastructure for development of personalized genomic medicine 
11:40

Antisense technology: Biogen and IONIS collaboration to develop the first treatment for Spinal Muscular Atrophy (SMA)

  • Intrathecal Anti-Sense Oligonucleotides (ASOs) for CNS Diseases  
  • Clinical development program to demonstrate improvement in range of SMA patients
  • NURTURE Study in Pre-symptomatic SMA infants: multi-disciplinary collaboration
11:40

Evolving access for orphan drugs in Canada: orphan drug evaluation at CADTH

  • Access to orphan drugs in Canada
  • Assessment of orphan drugs for reimbursement at CADTH
  • Current and future challenges in the evaluation of orphan drugs for reimbursement in Canada


 
12:00

The journey towards the approval of EU's first stem cell treatment for a rare eye disorder

  • Using stem cells to reconstruct the epithelium that covers the corneal surface 
  • Demonstrating the safety and efficacy of stem cells in a clinical setting
  • Manufacturing and logistics considerations for collection of the sample and delivery of the final treatment to the patient 
  • Fostering innovation in the rare disease field through stem cell therapies
12:00

Orphan drugs and rare diseases in Latin America

• New legislations and trends addressing the health care system sustainability of the region
• From product centric-to patient-centric
• Is LATAM still a key player for rare disease companies? Yes, but…


 

12:20 ROUNDTABLES

  • Access – How can rare disease companies, insurers and patients/families work together to ensure access to transformative and curative therapies?
  • Drug repurposing – finding scientific rationale and relying on strong safety data for orphan drug repurposing
  • Enhanced collaboration – collaboration between patients, researchers and government institutions to support patient-centered research
  • Ethical considerations – patients’ rights when participating in rare disease trials
  • Gene therapy – partnering to build capabilities in Gene Therapy
  • Global procurement – effective optimization of rare disease clinical processes to improve efficiencies and support commercialization
  • Multi-stakeholder collaboration to accelerate rare disease clinical development
  • Pricing – price gouging and the dangerous new breed for pharma companies
  • RWD – Using real-world data to find rare disease patients for long-term observational studies
  • Steering committees – reaching the goal of patient committees that can drive trial design and meaningful data
  • Strategy – successfully managing, and implementing drug development programs for domestic and international commercialization of orphan drugs
  • Diane White

    Roundtable 2: Strategy – successfully managing, and implementing drug development programs for domestic and international commercialization of orphan drugs

    Edmund Pezalla

    Roundtable 3: Pricing – price gouging and the dangerous new breed for pharma companies

    Christopher Missling

    Roundtable 4: Partnerships- successful collaboration between a rare disease foundation and a pharmaceutical company to advance preclinical and clinical research in Rett Syndrome

    Dr Cynthia Jackson

    Roundtable 5 Roundtable – simplifying the complexities of rare disease drug development

    Donna Armentano

    Roundtable 6: Gene therapy – partnering to build capabilities in Gene Therapy

    Jayne Gershkowitz

    Roundtable 7: Steering committees – reaching the goal of patient committees that can drive trial design and meaningful data

    Rashmi Gopal-Srivastava

    Roundtable 8: Enhanced collaboration – collaboration between patients, researchers and government institutions to support patient-centered research

    Keith DeMatteo

    Roundtable 9: Global procurement – using your procurement function to drive innovation and not just three bids and a buy

    Claudia Hirawat

    Roundtable 10: Ethical considerations – patients’ rights when participating in rare disease trials

    Francesca Cook

    Roundtable 11: Access: It takes a Village- How can rare disease companies, insurers and patients/families work together to ensure access to transformative and curative therapies

    Professor Matthias Schönermark

    Roundtable 12: European market access- effective strategies for setting drug prices and achieving reimbursement of orphan drugs

    Horacio Plotkin

    Roundtable 13: Virtual trials - Can we make them a reality in orphan drug development?

    John Boland

    Roundtable 14: Trial optimization- Unique challenges in small population/rare disease studies vs. large population trials

    Ms Katherine Brandt

    Roundtable 15: Orphan drugs manufacturing – overcoming CMO selection, auditing strategies, risk management and other outsourcing challenges

    Moderated by AnovaFill 
    Steven Anderson

    Roundtable 17: Genomics & biomarkers – solutions for rare diseases, implications for natural history and patient recruitment

    If you are interested in sponsoring a roundtable contact André Singer at andre.singer@terrapinn.com or +1 646 619 1797

    13:00 NETWORKING LUNCH

    Richard Moscicki
    13:30

    Lunch Keynote: Scientific rationale – FDA’s updates and considerations to orphan drug approval

    • Rigorous collection of natural history data
    • Rigorous development of assets prior to start of clinical trials, particular biomarker assets
    • Adoption of randomization to control groups in trials
     

    13:50 NETWORKING LUNCH RESUMES

    DIGITAL HEALTH & DIAGNOSTICS

    Margaret Collins
    14:30

    Healthcare digitalization: Cloud technology and Telehealth revolutionizing rare disease management

    • Improving the interactions between researchers, connecting patients with clinical studies and patient groups 
    • Creating virtual video rooms with patients to evaluate biometric data and patient reported outcomes 
    • Optimized and faster diagnostics by enabling researchers at different locations to examine the same data and share real-time feedback
    Alex Sherman
    14:50

    BIG data in "small” diseases- increasing the likelihood of success of ALS clinical trials and its applicability to other rare disorders

    • Working with pharma and other stakeholders to make anonymized data available to the global R&D community
    • Big data to understand natural history, disease heterogeneity, novel biomarkers and pathways of disease
    • The ALS Prediction Prize program: using algorithms to predict the progression of the disease over time
     
    Dr Christopher Gibson
    15:10

    The value of computer vision, machine learning and robotic automation to expedite rare disease research

    • Running experiments and performing analysis on hundreds of diseases simultaneously using powerful software and analytics
    • Finding new indications for existing drugs using augmented high-throughput drug research and technology
    • Creating high-resolution, comprehensive biological data sets 
    • Academia-industry partnerships to foster rare disease research 

    BIOSIMILARS

    Kate Rawson
    15:30

    Is the orphan drug market ready to embrace the biosimilar landscape?

    • Are orphan drug biosimilars needed in order to increase patient access to costly drugs? 
    • Is biosimilar development going to foster innovation in orphan drug manufacturers? 
    • Should the focus be on biosimilar development when there’s still so many rare diseases with no cure?
    Moderator: Kate Rawson, Contributing Editor, Prevision Policy
    16:10

    END OF CONFERENCE

    last published: 24/Mar/17 14:35 GMT

     

    WORLD ORPHAN DRUG CONGRESS HAS IT ALL...

    World Orphan Drug Congress USA is bringing together the worlds of Scientific Innovation and Commercialization, with 2 tracks in the main conference. By exploring trends in Digital Health, Biosimilars, Gene Therapy, and more, we will uncover the next scientific breakthrough. Examinations of Mergers, Commercialization, Marketing and the Regional Markets will provide insight into this billion-dollar business.

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