2018 agenda
Barcelona, 6 - 8 November 2018
Schedule
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Nov 609:01
Conference pass
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?
- 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
Doug Paul, partner, Medical Marketing Economics
Nov 609:01
Conference pass
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
Nov 614:01
Conference pass
Workshop C: Ensuring Success of Orphan Disease Drug Development and Market Access
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 and market access.Endpoint design and selection, and conditional approval pathways in the current regulatory and HTA environment for Orphan medicines in the US and Europe. Discussions on the use of registry studies to facilitate ,enhance and maintain product approvals.Rory Graham, BPharm, MBA (Senior Director, Regulatory Affairs, Europe & International Region, Mapi SRS, an ICON plc. Company)The Orphan medicines development environment is challenging in terms of endpoint design, statistical validation, patient recruitment, and regulatory pathways. Rory will review endpoint strategies for Orphan diseases, highlight current trends with the major regulators, and highlight the opportunity for earlier approvals that can be afforded by early and thoughtful integration of registry studies into the development plan, with reference to both FDA an EMA initiatives in this area.Engaging Patients and Advocacy Groups for Patient-Centric Trial Designs and the Promotion of Rare Disease Clinical TrialsKelly Franchetti (VP, RN, CCRN, CEN Patient Insight and Engagement, ICON)This interactive session will introduce participants to a 360° approach to obtaining and applying patient insights, and for engaging advocacy groups to improve trial design, operations, promotion, and recruitment. Through engaging activities, we will address how to get to the heart of the patient’s decision-making process, assess their physical/emotional/social needs, and identify their motivators, barriers and influencers as they relate to clinical study participation. Participants will also learn best practices for engaging with patient advocacy groups throughout the product development lifecycle.Approaches to successful execution of rare disease clinical trialsCynthia Dukes (VP, Drug Development, Rare Disease, ICON)This session will discuss the elements related to successful execution of rare disease studies. Rare disease studies require different work attitudes and different approaches to site selection, site support, patient support, and team requirements.They are not business as usual!Market Access and Supporting Research for Rare Disease TreatmentsWill Maier, PhD, MPH (Chief Scientific Officer, Commercialisation and Outcomes, ICON)This session will discuss the reimbursement and market access for rare disease treatment including regulations and additional research needed for a successful application and reimbursement profile.
Nov 614:01
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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
Delphine Roulland, Senior Manager Public Affairs chez, EUCOPE
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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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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?
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
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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Challenges and opportunities in the G-BA benefit assessment of new orphan drugs
Keynotes
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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Developing and improving access and data sharing in rare disease registries and biobanks worldwide
Keynotes
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
Dmitry Mordvintsev, Head of Specialty Care BU, Genzyme
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 a PAG can contribute to multi-stakeholder collaboration and fund clinical trials
Keynotes
More stakeholders to be announced
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 thru “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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Reducing barriers to early access
Keynotes
IQVIA
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
Gabriele Schlosser-Weber, Clinical assessor, Bfarm
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
Denis Horgan, Executive Director, EAPM (European Alliance for Personalised Medicine)
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 714:20
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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
Angi Robinson, Executive Director, Strategic Development, Rare Disease & Pediatrics, Premier Research
Nov 714:20
Conference pass
A one stop shop for HTA? The commission proposal and impact on rare diseases
Approval & Access
- The future of healthcare technology assessment in Europe
- The likelihood of the proposal materializing and perspectives from the commission, HTAs and industry
- Are we working collaboratively enough?
Nov 714:20
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Combating rare diseases with Gene Therapy
Cell & Gene Therapy
Nov 714:20
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The genetics of new sequence diversity
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?
Misha Votruba, Director, Gradus R.S.J. Life Sciences
Nov 714:45
Conference pass
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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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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The road to restoring sight in patients with rare inherited retinal diseases
Cell & Gene Therapy
Nov 715:15
Conference pass
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
Nov 715:40
Conference pass
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
Conference pass
Application of Real World evidence to facilitate improved access to rare disease therapies
Approval & Access
Sam Oliver, Joint Managing Director, pH Associates
Nov 716:35
Conference pass
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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Gene editing revolution: Hope for rare diseases, but should there be limits?
Precision Medicine
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
Van Zyl Engelbrecht, Clinical Project Director EU, UBC
Nov 717:00
Conference pass
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, Patient Advocacy Manager, Clinigen Group Plc
Nov 717:00
Conference pass
Organ on chip technologies to model rare diseases
Precision Medicine
Nov 717:00
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Pitch & Partner 1: Targeting orphan CNS diseases with high unmet medical need
Pitch & Partner
Marc Martinell, CEO, Minoryx Therapeutics
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?
Anne-Virginie Eggimann, Vice President Of Regulatory Science, bluebird bio
Marc Martinell, CEO, Minoryx Therapeutics
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
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 717:30
Conference pass
The Disruptive Potential of Gene Therapy: When Will It Fully Realize Its Commercial Promise?
Cell & Gene Therapy
Marcio Souza, Chief Operating Officer, PTC Therapeutics
Daan Kranenburg, VP Commercial Operations Europe, bluebird bio
Nov 717:30
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Pitch & Partner 3: Mezzion Pharmaceuticals: Advancing treatements for single Ventricle Heart Disease
Pitch & Partner
Erik Emerson, Mezzion Pharma
Nov 717:35
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Pitch & Partner 4: Novel peptides to treat orphan diseases
Pitch & Partner
Nov 717:50
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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
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Nov 808:30
Conference pass
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?
Karen Facey, Senior Research Fellow, University of Edinburgh
Sophia Brodin, Deputy Head of Department, Department of Value Based Pricing, The Dental and Pharmaceutical Benefits Agency T.L.V.
Nov 809:25
Conference pass
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
Alastair Mackinnon, CMO, Mereo BioPharma
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?
- 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 811:55
Conference pass
Nov 811:55
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Chair’s opening remarks
Approval & Access
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
Conference pass
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
Conference pass
Panel: Innovative payment models for cell & gene therapies
Cell & Gene Therapy
Leo Strican, Head of Access Value & Evidence Strategy, Europe, Bluebird Bio
Nov 812:00
Conference pass
The role of Foundations in advancing precision medicine
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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From diagnosis to market: The business model of the Duchenne parent project, Spain
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
Conference pass
Is there a Nordic collaboration on the horizon?
Approval & Access
Sofie Alverlind, Project Manager, Swedish Association of Local Authorities and Regions
Sune Lindgaard, Chief of Business Intelligence, Amgros, Denmark
Nov 814:30
Conference pass
Interactive discussion: Collaboration is the new competition
Cell & Gene Therapy
Individual presentations followed by discussionMore speakers to be confirmed from patient group, academia and industry
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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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
Conference pass
Driving commercial discovery through open science: Alternative business models that support orphan drug development
Science & Strategy
Nov 815:00
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Creating sustainable process development strategies for gene therapy products
Manufacture
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
last published: 24/Sep/18 16:35 GMT
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