2018 agenda

Barcelona, 6 - 8 November 2018

Schedule

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Nov 609:01
Conference pass

Workshop A (9am-1pm): Pricing, Reimbursement and Market Access Challenges for Orphan Drugs and Cell and Gene Therapies

Keynotes
  • Orphan Drugs and Advanced Therapy Medicinal Products (ATMPs) in the EU: Pricing, reimbursement and market access challenges
  • Guided panel discussion – What pricing, reimbursement and market access models for Orphan ATMPs?
  • Unique challenges in the manufacturing and commercialization of gene therapies for rare diseases
  • Building a value dossier for personalized gene / cell therapies for rare diseases
Nov 609:01
Conference pass

Workshop B (9am-1pm): Drug Repurposing – A Sufficient Business Model to Drive Orphan Drug Development?

Keynotes
  • What is a sustainable approach to repurposing generic drugs for rare diseases?
  • Can repurposing truly deliver a transformational therapy?
  • How can this be incorporated into a drug development business model and strategy?
  • How to get national healthcare providers and regulators on-board? Are there gaps in the regulatory frameworks
  • Considerations: How does drug repurposing differ? The more obvious benefits and lesser known challenges
  • What about off label repurposing?
Daniel O'Connor, Medical Assessor, Medicines and Healthcare Products Regulatory Agency
Dr Pan Pantziarka, Program Director, Anticancer Fund
Nov 614:01
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Workshop C (2pm-5pm): Ensuring Success of Rare Orphan Disease Drug Development

Keynotes
Orphan drug development has several unique challenges. This workshop provides expert guidance and practical case studies to help address the major challenges of:
  • Drug candidate early development
  • Regulatory strategy
  • Trial design
  • Patient engagement
  • Market access
Nov 614:01
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Workshop D (2pm-5pm): Managed Access…Are they the holy grail?

Keynotes
  • How will the development of the EUnetHTA initiative support the managed entry agreement development and data collection, can it become a joint European venture too?
  • Success stories and learning from a variety of jurisdictions, discuss best practices from multiple perspectives and provide a hands-on opportunity to craft MEAs for specific therapies

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Nov 708:35
Conference pass

European Reference Networks (ERNs) – What are they expected to deliver?

Keynotes
  • Why ERNs were established, how do they work and what kinds of ERNs are already in place for rare diseases?
  • Creating a clear governance structure for knowledge sharing and coordination across the EU to improve access to diagnosis, treatment and care
  • What resources do ERNs have to reach their goals, at a European and Member State level
Nov 709:30
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Keynote discussion: Executive industry insights - Why are we pursuing the same diseases?

Keynotes
  • What is it that companies need in order explore a new orphan indication or therapy area?
  • How to approach developing and launching treatments in as-yet untreated diseases that are not well-characterized
  • Insights into individual pipelines – what have we learnt that will make orphan drug research and development sustainable?
Nov 711:30
Conference pass

INTERACTIVE ROUNDTABLES

Keynotes
Day 1 ROUNDTABLESCovering a number of hot topics and delivered by patient groups, sponsors and leading solution providers, choose which two roundtable discussions you would like to join, for ‘off the beaten track’ learning and information you can’t find online. Each session will last 45 minutes so you can join two discussions in the allotted time.
Nov 711:31
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Common early mistakes in orphan drug development and how to avoid them

Keynotes
Nov 711:31
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Nov 711:31
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Developments in Eastern Europe: Unique challenges and opportunities

Keynotes
Pawel Wozniak, Managing Director, Komtur Polska
Nov 711:31
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Eastern Europe and Asia: Local legislation and pitfalls, sharing experience

Keynotes
Nov 711:31
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Expediting drug development in rare disease: Are big data and biomarkers an option for the future?

Keynotes
IQVIA
Nov 711:31
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From Endpoints to Endgame: Incorporating the diagnostic odyssey and patient journey in your clinical development plan

Keynotes
Nov 711:31
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Gene therapies : are HTA agencies and healthcare systems in Europe ready for their market adoption?

Keynotes
Nov 711:31
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How can expanded access combined with real-world data to enhance drug development

Keynotes
  • Key challenges of recruiting patients for drug development trials
  • Overcoming failure to treat and the failure to learn
  • Combining expanded access with Real-world data.
Nov 711:31
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How patient-led technological solutions are helping diagnosis, treatment and care and paving the way for patient-centric medicines development

Keynotes
Arlete Monteiro, IT Director, Ministry Of Health Of Portugal
Tapani Piha, Head Of Unit, European Commission
Nov 711:31
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Increasing availability of novel drugs for more prevalent diseases through “orphanization”

Keynotes
Nov 711:31
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Innovative ways patient groups can contribute to the drug development process

Keynotes
Alastair Kent Obe, Former Director, Genetic Alliance UK, Retired
Nov 711:31
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Market entry strategies for orphan drugs in Central Eastern Europe

Keynotes
Nov 711:31
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Patient empowerment in access negotiations: The case of “afamelanotide” for the treatment of the ultra-rare light intolerance erythropoietic protoporphyria

Keynotes
Jasmin Barman, Scientific Advisor, SGP
Nov 711:31
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Regulatory & operational implementation of the direct to patient trial management model

Keynotes
Nov 711:31
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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

Keynotes
Nov 711:31
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Status of access and power of advocacy in more difficult markets: Quality of life and collaborations with neighbouring countries

Keynotes
Dorica Dan, President, Romanian Pader Willi Association
Nov 711:31
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The challenges of developing an orphan drug worldwide

Keynotes
Pedro Franco, Director for Europe for Global Regulatory and Scientific Policy, Merck Serono
Nov 711:31
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The current European precision medicine landscape and how the Innovation Medicines Initiative is advancing innovation in the field, through public private partnerships

Keynotes
Nov 711:31
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The virtuality of rare disease clinical trials: Where are we heading?

Keynotes
Nov 714:15
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Chair’s opening remarks

Cell & Gene Therapy
Dr Michael Binks, VP, Rare Disease Clinical Research, Pfizer
Nov 714:15
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Chair’s opening remarks

Precision Medicine
Nov 714:20
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Strategies for obtaining orphan drug and other EMA designations for orphan products

Clinical Development
Nov 714:20
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A one stop shop for HTA? The commission proposal and impact on rare diseases

Approval & Access
  • The future of healthcare technology assessment in Europe
Speaker:European CommissionFollowed by panel:
  • The likelihood of the proposal materializing and perspectives from the commission, HTAs and industry
  • Are we working collaboratively enough?
Senior representative, EUnetHTA WP4 (pharmaceutical Joint Assessments)
Nov 714:20
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Combating rare diseases with Gene Therapy

Cell & Gene Therapy
Nov 714:20
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The impact of genomics on drug discovery and development

Precision Medicine
Nov 714:20
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Investor panel: Hear how different investors decide on who and what they invest in, and why

Pitch & Partner
  • Compare and contrast different VC methodologies
  • What other types of innovative funding is there?
  • Unorthodox ways to get money
  • What do pharma look for when in-licensing?
More co-speakers to be confirmed shortly
Nov 714:45
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What does a patient-focused clinical trial really look like?

Clinical Development
Co-presenting patient representative to be announced
Nov 714:45
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Translating the CRISPR platform into a gene-edited therapy, finally a reality?

Cell & Gene Therapy
Nov 714:45
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Global commission to end the diagnostic odyssey: Eliminating barriers to an accurate and early diagnosis

Precision Medicine
Yolanda Ludena, Foundation twenty-nine
Nov 715:15
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Strategies for clinical development in genetic rare disease: Study designs, operational challenges, advanced therapies & Patient finding

Clinical Development
  • The definition of genetic rare disease and relatedness
  • Study design considerations :Inclusion and exclusion criteria &Siblings and other relatives
  • Operational:Genetic confirmation- historical proof, timing of assays,Central labs: specialty labs and academic labs &Special populations
  • Advanced therapies:Consent, communication,Long-term follow-up,Logistics
  • Finding patients:Screening studies,Patient registry
Nov 715:15
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Patients, pricing and profits: Is the orphan drug business model fair and sustainable?

Approval & Access
Nov 715:15
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The road to restoring sight in patients with rare inherited retinal diseases

Cell & Gene Therapy
Nov 715:15
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Combining artificial intelligence and genomics to advance treatment and diagnostics

Precision Medicine
Nov 715:15
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Multi-perspective discussion: How to partner with public/private organisations

Pitch & Partner
  • How to approach big pharma/government organisations for support
  • How can companies correctly identify a fit and interest?
  • Can partnering prevent biotech start-ups from getting stuck before Ph1?
  • Do and don’ts with partnerships
More co-speakers to be confirmed shortly
Jami Ellis, Executive Director of Commercial Development, Horizon Pharma
Nov 715:40
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Integrated real word evidence to support rare disease clinical development and commercialization

Clinical Development
Nov 715:40
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Innovative approaches to patient finding in rare diseases

Approval & Access
Nov 715:40
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Discover what payers really think of gene therapy

Cell & Gene Therapy
Nov 715:40
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Using Artificial Intelligence to accelerate repurposed drugs access to patients

Precision Medicine
Nov 716:35
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Communicating clearly and effectively with your patient community: A review of translation methods for consistency of patient-facing documents for rare disease research in international trials

Clinical Development
Nov 716:35
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Demonstrating value with disruptive therapies

Approval & Access
Nov 716:35
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Overview of current gene therapies in the market: What are we working on?

Cell & Gene Therapy
  • Creating pipelines that are robust enough for gene therapy to become a reality
  • Preparing for later stage trials
Nov 716:35
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Nov 716:35
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Success factors for rare disease launches and partnering

Science & Strategy
Nov 717:00
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Successfully managing and implementing drug development programs for domestic and international commercialisation of orphan drugs

Clinical Development
Title TBC
Nov 717:00
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We need to talk about patients: The patient view on putting the P in ‘PRO’ centre-stage

Approval & Access
Reserved for Clinigen Group
Romina Oxborough, Director of Clinigen Consulting, Clinigen Group
Lucia Von Bredow, Public Affairs & Patient Policy Manager, Clinigen Group
Nov 717:00
Conference pass

Life-saving regeneration of the entire human epidermis by transgenic stem cells

Precision Medicine
Nov 717:00
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Pitch & Partner 1: Targeting orphan CNS diseases with high unmet medical need

Pitch & Partner
Nov 717:15
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Nov 717:25
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Debate: Tailoring drug development to meet an evolving orphan drug approval landscape

Clinical Development
How does the small clinical trial populations, different types and quantities of evidence, accelerated approval pathway and use of surrogate outcomes influence development plans and assessment of effectiveness?
Nov 717:25
Conference pass

Multi-stakeholder perspective on managed access programs, pricing and reimbursement: Benefits, challenges, and key considerations for successful implementation

Approval & Access
Nov 717:30
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The Disruptive Potential of Gene Therapy: When Will It Fully Realize Its Commercial Promise?

Cell & Gene Therapy
Nov 717:30
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Should rare disease oncology be considered true orphans?

Precision Medicine
  • Is it too early to classify the majority of orphan cancers by molecular characteristics?
  • Assessing the value of immuno-oncology treatments
Nov 717:30
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Pitch & Partner 3: Mezzion Pharmaceuticals: Advancing treatements for single Ventricle Heart Disease

Pitch & Partner
Erik Emerson, Executive Vice President, Commercial Operations, Mezzion Pharma
Nov 717:35
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Pitch & Partner 4: Novel peptides to treat orphan diseases

Pitch & Partner

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Nov 808:35
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A pan European study: The realities of orphan drug access

Keynotes
  • How do European countries differ in their value assessment to new orphan drugs?
  • What next steps are needed for more harmonized, comparable and rational evidence based decision-making in Europe?
Followed by live feedback from HTAs:Dr Karen Facey, Member, Scottish Health Technologies Group & Impact HTA - Co-Lead Investigator for WP10 - Appraisal of Orphan Medicinal ProductsEdmund Jessop, Medical Adviser, NHS EnglandSophia Brodin, Deputy Head of Department, Department of Value Based Pricing, TLV, The Dental and Pharmaceutical Benefits AgencySenior representative, H-AS, France
Nov 809:25
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Speaking from experience, expedited regulatory pathways in gaining EMA PRIME examples – Where do we go from there?

Keynotes
  • Paving the way for biotechs to gain approval and access
  • Recent changes and future amendments to regulatory issues surrounding orphan drugs approval
  • How are companies interacting the payers and how to close the gap to allow treatments to reach patients
Discussion with payer and PRIME designated companies
Nov 810:10
Conference pass

Recommendations on sustainable access to innovative therapies

Keynotes
Presentation:
  • Ensuring the sustainability of health spendingas well ascontinued innovation that meets patient needs
  • How does the current pricing system need to change to compliment this?
  • Are novel pricing & reimbursement mechanisms for gene therapy products necessary?
Followed by feedback from global stakeholders on value vs pricing and affordability
  • Are we paving the way to a fair, inclusive and on-going multi-stakeholder approach with the potential to generate sustainable, affordable and actionable improvements in patient access to rare disease therapies?
  • Balancing between recouping costs for R&D and incentives to support investment in future treatments that ultimately access to patients
Nov 811:55
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Chair’s opening remarks

Clinical Development
Nov 811:55
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Nov 811:55
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Nov 811:55
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Nov 812:00
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Aligning the priorities between the healthcare community and the European Parliament: Where we are now and the necessary next steps for a regulatory framework for HTA

Approval & Access
Nov 812:00
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The role of Foundations in advancing precision medicine

Science & Strategy
Nov 812:00
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Transformative impact of TALEN-mediated gene editing on cell and gene therapies and their manufacturing

Manufacture
Nov 812:30
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From diagnosis to market: The business model of the Duchenne parent project, Spain

Science & Strategy
Nov 812:30
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Post approval: Next steps to the production process

Manufacture
Nov 814:30
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Hercules one year on: how Duchenne UK have developed HTA evidence and tools through collaboration

Clinical Development
Pharma collaborators TBC
Josie Godfrey, Director, JG Zebra Consulting
Fleur Chandler, Therapy Area Head of Respiratory Value Evidence and Outcomes, GlaxoSmithKline Biologicals
Nov 814:30
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Nov 814:30
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A non-profit mechanism for moving proof-of-concept studies to clinical therapies for rare disorders

Science & Strategy
Nov 814:30
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Creating sustainable process development strategies for gene therapy products

Manufacture
Nov 815:00
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Group discussion: How will these collaborations change the way we assess the value of ODs?

Approval & Access
Nov 815:00
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Driving commercial discovery through open science: Alternative business models that support orphan drug development

Science & Strategy
Nov 815:00
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Capacity crunch? The future of manufacturing within cell & gene therapy

Manufacture
Bastiaan Leewis, MSAT Manager Industrialization, MeiraGTx
Nov 816:00
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IRDiRC: Enabling all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention. How do evolve to achieve target?

Keynotes
Presentation:
  • The IRDiRC goals, a community effort for a better future in rare disease research and clinical management and a call for international collaboration to an unprecedented scale
  • How are we going to achieve quicker diagnosis for 7000+ diseases and to solve unsolved diagnostic cases?
  • How are we going to deliver the best possible care and give access to all available therapies to rare disease patients?
  • What change in pace do we need to develop new treatments for thousands of rare diseases?
  • Summary of learnings from the congress and targets for next year
Followed by panel discussion on; how far we are from the IRDiRC vision and how much ambitious its goals are& what we need to change in the system to achieve these goals and vision
last published: 04/Nov/18 13:45 GMT

 

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