2018 agenda

Barcelona, 6 - 8 November 2018

Schedule

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Workshop A: 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?
Co-presenting pharma partners to be announced shortly

Doug Paul, partner, Medical Marketing Economics
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Workshop B: 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
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Workshop C: Securing Success from Regulatory to Global Market Access for Orphan Drugs & Rare Disease Treatments

Keynotes
  • Partnering with patients for clinical success
  • Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies
  • An introduction tothe science of endpoint design in the current regulatory and HTA environment in the US and Europe
Led by Senior representatives, ICON
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Workshop D: 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
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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
Avril Daly, CEO, Retina International
Till Voigtlander, Vice-President of the Board of Members States on ERNs, Medical University of Vienna
Nov 709:30
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Opening 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?
Dr Greg LaRosa, SVP & CSO, Rare Disease Research Unit, Pfizer
Dr Andreas Busch, Chief Scientific Officer, Head of Research and Development, Shire Pharmaceuticals
Nov 711:30
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INTERACTIVE ROUNDTABLES

Keynotes
Day 1 ROUNDTABLES
Covering 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.
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Challenges and opportunities in the G-BA benefit assessment of new orphan drugs

Keynotes
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Common early mistakes in orphan drug development and how to avoid them

Keynotes
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Developments in Eastern Europe: Unique challenges and opportunities

Keynotes
Pawel Wozniak, Managing Director, Komtur Polska
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How a PAG can contribute to multi-stakeholder collaboration and fund clinical trials

Keynotes
More stakeholders to be announced
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Increasing availability of novel drugs for more prevalent diseases thru “orphanization”

Keynotes
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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
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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
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Regulatory & operational implementation of the direct to patient trial management model

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

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

Keynotes
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Utilising public health records: electronic data collections from patients for better diagnosis and RWE

Keynotes
Reserved for IQVIA
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What can we learn from past gene therapy companies to ensure the demand and pricing dynamics are accurate for the market?

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 714:20
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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 Commission

Followed 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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Opportunities and challenges of operationalizing biobank-based genomic research

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
Dr Jerome Rossert, Vice President, General Medicine, Global Clinical Development, Vertex Pharmaceuticals
Nov 714:45
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The impact of genomics on drug discovery and development

Precision Medicine
Nov 715:15
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Innovative strategies for clinical development for genetic rare disease

Clinical Development
Senior representative, Premier Research
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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Life-saving regeneration of the entire human epidermis by transgenic stem cells

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
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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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
Senior representative, ICON
Nov 716:35
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Application of Real World evidence to facilitate improved access to rare disease therapies

Approval & Access
Senior representative, OPEN Health
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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
Van Zyl Engelbrecht, Clinical Project Director EU, UBC
Nov 717:00
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Patient advocacy in the early access and compassionate

Approval & Access
Reserved for Clinigen Group

Nov 717:00
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Discover what payers really think of gene therapy

Cell & Gene Therapy
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?
Anne-Virginie Eggimann, Vice President Of Regulatory Science, bluebird bio
Marc Martinell, CEO, Minoryx Therapeutics
Nov 717:25
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Multi-stakeholder perspective on managed access programs, pricing and reimbursement: Benefits, challenges, and key considerations for successful implementation

Approval & Access
Darren Walsh, Senior Director - Market Access & Govt. Affairs – EU, Orchard Therapeutics
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Nov 717:25
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Nov 717:25
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Nov 717:50
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Treating patients suffering from rare and ultra-rare diseases caused by Stop Termination Codon nonsense mutations

Precision Medicine

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Nov 808:30
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Chair’s opening remarks

Keynotes
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
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 Products
Edmund Jessop, Medical Adviser, NHS England
Jonathan Lind-Martinsson, Head of Unit, Department of Value Based Pricing, TLV, The Dental and Pharmaceutical Benefits Agency
Senior representative, H-AS, France
Agency for Health Technology Assessment in Poland – invited
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
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OECD 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
Nathalie Moll, Director General, EFPIA
Rabia Khan, Health Division, Directorate for Employment, Labour and Social Affairs, O.E.C.D.
Nov 812:00
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A PARADIGM shift: Making Patient Engagement the new norm

Clinical Development
Mathieu Boudes, Coordinator of PARADIGM, European Patients' Forum
Nicholas Brooke, Executive Director, Patient Focused Medicines Development
Nov 812:00
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An update on Beneluxa partnership and a review on:

Approval & Access
Francis Arickx, Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy,, National Institute for Health and Disability Insurance RIZIV-INAMI
Nov 812:00
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Panel: Innovative payment models for cell & gene therapies

Cell & Gene Therapy
Nov 812:00
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From diagnosis to market: The business model of the Duchenne parent project, Spain

Science & Strategy
Nov 812:00
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Industrialization of gene and cell therapies manufacturing

Manufacture
Nov 812:30
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Access to ODs in central Europe – Challenges and opportunities

Approval & Access
Nov 812:30
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Should rare disease oncology be considered true orphans?

Science & Strategy
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, National Institute for Health and Care Excellence
Fleur Chandler, Therapy Area Head of Respiratory Value Evidence and Outcomes, GlaxoSmithKline Biologicals
Nov 814:30
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Is there a Nordic collaboration on the horizon?

Approval & Access
Sofie Alverlind, Project Manager, Swedish Association of Local Authorities and Regions, NLT
Sune Lindgaard, Chief of Business Intelligence, Amgros, Denmark
Nov 814:30
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Interactive discussion: Collaboration is the new competition

Cell & Gene Therapy
Individual presentations followed by discussion

More speakers to be confirmed from patient group, academia and industry
Nov 814:30
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The role of foundations in advancing precision medicine

Science & Strategy
Ana Mingorance, Chief Development Officer, Loulou Foundation
Nov 814:30
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Post approval: Next steps to the production process

Manufacture

Dr Emanuele Ostuni, Head of Europe, Cell and Gene Therapy, Novartis
Nov 815:00
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Group discussion: How will these collaborations change the way we assess the value of ODs?

Approval & Access
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Nov 815:00
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Driving commercial discovery through open science: Alternative business models that support orphan drug development

Science & Strategy
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: 07/Jun/18 16:45 GMT

 

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