World Orphan Drug Congress Europe 2021 Agenda

Barcelona, 15 - 18 November 2021

Schedule

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Nov 1510:30
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Aligning the healthcare system across agencies: Can we apply any of the experiences from COVID to streamline the drug availability?

Keynotes
The global pandemic has led to an unprecedented level of activity in research and development in one disease. In the absence of treatments available for COVID-19 the race to develop effective therapeutics provided a platform for the repurposing of existing therapies to be considered for use. Many drugs used to treat different diseases are already in researchand some have been successfully repurposed and approved for new indications in COVID-19.In the race to deliver these treatments to patients in the context of the pandemic RAPID-C19 (Research to Access Pathway for Investigational Drugs for COVID-19) emerged to address this need, a multi-agency initiative collaboration to survey the COVID trial landscape and accelerate marketing approval and assessment in the UK.What has this initiative taught us about repurposing, the hurdles to overcome, the gains to be made and how does this approach guide and support drug repurposing for rare diseases.
Sheela Upadhyaya, Accelerated Access Collaborative Relationship & Delivery Lead & HST Specialist, National Institute for Health and Care Excellence
Daniel O'Connor, Expert Medical Assessor in Licensing Division, Medicines and Healthcare products Regulatory Agency
Nov 1510:30
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Looking through orphan drug treatment and diagnosis though a patient lens: Experience vs Journey

Keynotes
  • Patient centricity and experience – what happens to patients – how does that change the value proposition?
  • Challenging the notion of value from patients vs HTAs: the need for earlier diagnosis and subsequently earlier access to the right treatments
  • Taking a whole system thinking approach
  • Evidence based data reflecting the patient experience from a clinical setting: the “Patient Experience Explorer”
  • How can we make industry and our healthcare systems truly more patient centric?
Christopher M de M Rudolf, CEO, volv global
Nov 1514:00
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Responding to the EU OMP review - how to strengthen the EU rare disease ecosystem

Keynotes
  • Do we now have the right incentives in place to stimulate orphan drug development and address unmet medical needs?
  • How can we ensure more sustainable and equitable access for people living with rare diseases?
  • Following the launch of the European Expert Group on Orphan Drug Incentives at the WODC last year, this workshop will review the 14 proposals to further strengthen the EU rare disease eco-system put forward by the cross-disciplinary multi-stakeholder group.
  • This workshop will include an interactive panel combined with breakout discussions with workshop participants to discuss the 14 proposals along the medicine lifecycle and patient journey
Moderator: Sheela Upadhyaya, Accelerated Access Collaborative Relationship & Delivery Lead & HST Specialist, National Institute for Health and Care Excellence
Daria Julkowska, Assistant Director, Thematic Institute of Genetics, Genomics & Bioinformatics, Inserm
Toon Digneffe, Head of Public Affairs And Public Policy, Takeda

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Nov 1608:40
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Keynote Address: The European Commission’s review of the Orphan legislation – What does this mean?

Keynotes
  • How will revisions impact the development and availability of products for people living with rare diseases?
  • The complex challenges that ensure research is appropriately directed to areas of unmet medical need
  • Do we now have the right incentives are in place to stimulate the market, looking at areas of unmet need beyond cancer, alimentary conditions and metabolic disorders, which make up a large percentage of orphan medicines
  • Does the OMP regulation address unmet medical need, provide a better economic balance and affordability/accessibility?
Nov 1609:00
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Keynote panel: Sustainable Innovation and Equity of orphan drug access in Europe

Keynotes
  • The impact assessment and the evaluation of the public consultation around the review of EU Regulation on OMPs
  • The evolution around the Regulation on European Cooperation in HTA as well as the multi-stakeholder dialogue access, value and differential pricing
  • Are different areas treated different and if so, why?
  • EMA-EUnetHTA pilot
  • EFPIA “wait indicator“
Moderator: Dirk Vander Mijnsbrugge, Vice President and Medical Affairs Lead Rare Diseases International Developed Markets, Pfizer Manufacturing Belgium
Momir Radulovic, Executive Director, JAZMP
Giles Platford, President, Europe & Canada, Takeda Pharmaceutical Company
Yann Le Cam, Chief Executive Officer, EURORDIS
Nathalie Moll, Director General, EFPIA
Marcus Guardian, Chief Operating Officer, EUnetHTA
Nov 1609:45
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Keynote panel: The European Expert Group on Orphan Drug Incentives – How do we develop a sustainable European ecosystem?

Keynotes
  • What are the report recommendations?
  • The impact on the RD community, sustainability and existing treatment commitments
  • Success factors for rare disease launches
  • Regulatory review and on the evolution of the competitive environment in Europe for OMP developers
Moderator: Sophie Schmitz, Managing Partner, Partners4Access
Daria Julkowska, Assistant Director, Thematic Institute of Genetics, Genomics & Bioinformatics, Inserm
Toon Digneffe, Head of Public Affairs And Public Policy, Takeda
Anne-Sophie Chalandon, Head Of Rare Diseases, Public Affairs And Patient Advocacy, Sanofi Genzyme
Vittoria Carraro, Associate Director Government Affairs, EUCOPE, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Nov 1611:30
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Interactive Roundtables

Keynotes
Activating patients to shorten the time to diagnosis
Benefits of adaptive stimulation workshops in developing competitive strategies
Akshay Kumar, Partner, Partners4Access
Making a Successful Transition from Orphan Drug Designation to Orphan Drug Exclusivity
Market access and orphan drugs-how we horizon scan for orphan drugs in the UK
Nov 1614:10
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IMPACT HTA: Special processes to appraise rare disease treatments – Developing feasible, consistent approaches

Access & Pricing
  • Do we need more drive more radical changes and how?
  • Expanding the determination of value
  • Collecting the best evidence possible for all aspects of value
  • Engaging patient and clinical experts iteratively to resolve decision-relevant uncertainties
Moderator: Karen Facey, Senior Research Fellow, University of Edinburgh
Elena Nicod, Research Fellow, Bocconi University
Simone Boselli, Director, Public Affairs, EURORDIS
Vinciane Pirard, Senior Director Government Relations, Orchard Therapeutics
Nov 1614:10
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Improving clinical trial design for rare diseases

Cell & Gene Therapy
  • Tailoring clinical design models to reflect the uniqueness of the gene therapy modality
  • Using real world evidence studies to add value throughout the gene therapy development lifecycle
  • Managing patient expectations and establishing patient support systems
Moderator: Samantha Parker, Chief Patient Access Officer, Lysogene
Jean-Philippe Combal, Co-Founder And Chief Executive Officer, Vivet Therapeutics
Jose F. Gomez, SVP, Global Market Access & Value, AVROBIO
Josie Godfrey, Co-Founder and CEO, Realise Advocacy
Birgitte Volck, Non Executive Director, Soleno Therapeutics
Virginia Haurigot, Ocular Research Lead, Spark Therapeutics
Nov 1615:10
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Enabling patient access and engagement and the potential impact on commercialisation

Clinical Development
Yazi Al'joboori, Business Lead, University College London
Neil Watson, Business Development Director, Clinigen Group
Nov 1615:10
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Update on Lumevoq gene therapy development and regulatory advances for the treatment of LHON patients

Cell & Gene Therapy
Magali Taiel, Medical Chief Officer, Gensight Biologics
Nov 1615:40
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Are HTAs ready to receive info from wearables?

Clinical Development
  • How is HTA responding to the pandemic?
  • How good is the data from wearables, is it deemed reliable enough?
  • What do HTA think and expect?
  • What are the routes?
Nov 1615:40
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ILAP - Innovative Licensing and Access Pathway

Access & Pricing
  • A new pathway supporting innovative approaches to the safe, timely and efficient development of medicines to improve patient access
Daniel O'Connor, Expert Medical Assessor in Licensing Division, Medicines and Healthcare products Regulatory Agency
Carla Deakin, Programme Director - Commercial And Managed Access, NICE
Nov 1615:40
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The Syneos Health Cell and Gene Therapy Consortium

Cell & Gene Therapy
  • Improving cell & gene therapy preclinical/clinical development and commercialization
Nov 1616:40
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Rarified thinking: Patient Centricity at the Time of Decentralized Trials

Clinical Development
  • Covid-19 pandemia has facilitated a quick adaptation of professionals and patients to telemedicine, one of the strategies used in decentralized trials
  • Telemedicine is one classical tool used to facilitate participation of patients with rare-diseases in clinical trials
  • We present facts and figures on potential impact of decentralized trials in clinical research on rare diseases, during the last 2 years
Esther Mahillo, Executive Director Operational Strategy, Precision for medicine
Nov 1616:40
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Panel

Access & Pricing
i) How will the new Pharmaceutical Strategy from the EU Commission impact HTA?
  • Will it improve the access to orphan drugs?
  • How can orphan drug manufacturers demonstrate value and improve access to drugs?
ii) The EUHTA from the national perspectiveMore speakers TBC
Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, Riziv-Inami
Wim Goettsch, Special Hta-Advisor, Zorginstituut
Niklas Hedberg, Head Of Pharmacy, Tandvards och Lakemedelsformansverket
Nov 1616:40
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Access for rare diseases – How to navigate conflict, compromise, and collaboration

Cell & Gene Therapy
Moderator: Karl Freemyer, Head of Business Development, FIECON
Martin Schuchardt, Director Pricing and Market Access, FIECON
Nov 1617:10
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Remote endpoints - How the pandemic accelerated their development

Clinical Development
Nov 1617:25
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Panel: Overcoming the CMC bottlenecks that are hindering cell & gene therapy commercialization

Cell & Gene Therapy
More panellists to be announced
Frederic Revah, Chief Executive Officer, Genethon
Brendon Noble, Chief Scientific Officer, UK Stem Cell Foundation
Manuel Carrondo, Director, iBET: Instituto de Biologia Experimental e Tecnológica
Nov 1617:40
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Update: The gene therapy market access landscape in Germany

Access & Pricing
  • HTA evidence
  • Reimbursement
Axel Boehnke, Director Market Access EU North, PTC Therapeutics

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Nov 1708:30
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What new digital transformations and innovations have come from 2020? Ensuring we remain at the forefront of innovation for global access to treatments

Keynotes
  • Advancements in R&D, trial designs, regulatory affairs, patient engagement and new forms of partnerships
  • Continuing to close the gap between the number of treatments available and the vast amount of rare diseases
  • Bridging the gap between US and Europe
Vanessa Wolfeler, Global Franchise Head, Rare Blood Disorders, Sanofi Genzyme
Siân Smethurst, Head of Global Clinical Operations, Biogen
Adam Zaeske, Head of Portfolio & Pipeline, Europe & Canada, Takeda
Nov 1709:20
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The value of genetic testing in rare diseases: Ensuring an accurate diagnosis and timely access to treatment

Keynotes
  • The success of many transformative therapies currently in development depends on the capacity to identify patients in a timely and accurate way
  • Obtaining a confirmed genetic diagnosis is the only way to identify the exact gene mutation(s) that is the underlying cause of several rare diseases
  • Via genetic testing patients gain an exact prognosis, the understanding of the inheritance pattern of the condition, and are able to access treatment when available or participate in clinical trials
  • What role can EU and national policy-makers play to address existing barriers: from pathways to securing reimbursement to testing infrastructure, from clinical practice guidelines to availability of genetic counselling?
More speakers to be announced shortly
Avril Daly, Vice Chair, EURORDIS, Chief Executive Officer, Retina International
Angela Genge, Executive Director CRU, Montreal Neurological Institute
David Nestor, Head of Neuromuscular Disease, Europe, Canada and Partner Markets, Biogen
Nov 1711:10
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How do we as an industry, play our part in helping to bring down the cost of getting treatments to patients?

Access & Pricing
Geoff Fatzinger, Mging Director, CCPS UK, & Global VP of Quality, Regulatory & Strategic Services, Caligor Coghlan Pharma Services
Nov 1711:10
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Revolutionizing drug discovery for genetic diseases

Science & Strategy
James Doyle, CEO, Modelis Inc
Nov 1711:10
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XEN496 as a Novel Precision Medicine for KCNQ2-DEE

Precision Medicine
Sarah Weckhuysen, Neurologist, University Of Antwerp
Nov 1711:25
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Targeting rare CNS diseases with high unmet medical need

Science & Strategy
Marc Martinell, CEO, Minoryx Therapeutics
Nov 1711:40
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Cell & gene therapy readiness in specific markets (more advanced markets vs. less advanced in EU)

Access & Pricing
Akshay Kumar, Partner, Partners4Access
Nov 1711:40
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Leveraging Moderna’s mRNA platform to advance medicines for rare disease patients

Science & Strategy
Nov 1711:40
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Digital Twin-Clinical Trials supporting Orphan Drugs

Precision Medicine
Raj Pallapothu, Managing Director, Bio 9 Ventures
Nov 1712:10
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RWE practical considerations in early access, importance overall global approach

Access & Pricing
  • Frameworks to capture, what should you do to prepare
  • How they have been used to help regulatory submissions in EU & US
John Lagus, EVP, Business Development, Tanner Pharma Group
Nov 1712:10
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The use of AI-based identification for earlier disease diagnosis and subsequently earlier access to the right treatments

Precision Medicine
Christopher M de M Rudolf, CEO, volv global
Nov 1714:10
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Panel: Expanded Access / Compassionate Use in Europe

Access & Pricing
  • European EA / CU frameworks, what is important to know
  • How the pandemic has impacted the discussion around EA/CU
  • The increasing use of Real-world data for both regulatory and payers
  • What can we expect looking forward?
More panellists to be announced
Nov 1714:40
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Practical Considerations for Designing Your Clinical Trial

Clinical Development
  • Unique challenges that can hinder complex, hospital-based, clinical studies, such as those for rare disease and oncology trials
  • How to flex resources to shorten timelines and mitigate risk
Nov 1714:40
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Venture Capital insights: A global perspective on rare disease investments

Science & Strategy
  • How do private equity investment firms oversee investment in the rare disease space?
  • How does this fit into the overall biotech capital investment landscape?
More panellists to be announced
Moderator: Juan Garcia, Managing Partner, Biotechnology Business Institute
Raj Pallapothu, Managing Director, Bio 9 Ventures
Guillem Masferrer, Investment Director, Asabys Partners
Guillem Laporta, Principal, Ysios Capital
Nov 1714:40
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Panel: Digital health and cognition in rare disease trials

Precision Medicine
Moderator: Rich Cooper, Chief Commercial Officer, Cambridge Cognition Ltd
Francesca Cormack, Director, Research And Innovation, Cambridge Cognition Ltd
Arne Müller, AD & Senior Principal Data Scientist in Clinical Digital Endpoints, Novartis
Laura Germine, Director, Laboratory for Brain and Cognitive Health Technology, Harvard Medical School
Nov 1715:10
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How to run successful rare and orphan disease studies

Clinical Development
  • What are potential challenges?
  • Considerations in clinical monitoring, partnerships and relationships with patient advocacy groups
Nov 1716:10
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Multi-stakeholder panel: At which point is it “reasonable“ to approve and reimburse a drug from a regulatory standpoint?

Keynotes
  • The long-lasting debate regarding conditional approval vs full approval
  • How different stakeholders view uncertainties for potential treatments for rare diseases?
  • In the context of regulatory flexibility, how much uncertainty are regulators willing to accept when assessing risk vs benefit?
Martine Zimmermann, Global Head Of Regulatory Affairs, Alexion Pharmaceuticals

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Nov 1809:40
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Putting things into perspective: How well are GT’s performing?

Cell & Gene Therapy
  • Evaluation of patient access in the EU member states
  • What is the role of innovative payment models?
  • How can RWE help payers make an informed decision?
  • Are payers really valuing long term effects?
  • How can long term effects be measured?
  • A look at the first outcomes of price negotiations
Moderator: Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Pietro Sternini, Market Access & Marketing Lead EMEA, Astellas Gene Therapies
Tamir Singer, Head of Commercial Development, NHS England
Detlev Parow, Head Pharmaceutical Department, DAK-Gesundheit
Francis Pang, Global Head of Market Access, Orchard Therapeutics
Steven M Altschuler, Managing Director, Ziff Capital Partners
Oswald Bentinck, Vice President, Head Of Market Access EMEA, Novartis Gene Therapies
Paolo Morgese, EU Director Market Access & Member Relations, Alliance for Regenerative Medicine
Nov 1809:40
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Effective engagement in rare disease: playing the odds to turn hope to reality

Science & Strategy
Jonathan Morton, Agency lead, Comradis, Comradis
Nov 1809:40
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Access pathways outside of HTAs for ATMPs in Europe

Patient Centricity
  • Given the challenges in receiving reimbursement for ATMPs across Europe via HTAs, companies should also explore other potential access pathways such as named patient programmes
  • What are the key advantages / challenges of these schemes?
  • Available schemes in European
  • Do these schemes apply to cross-border healthcare?
  • Case studies
Nov 1810:10
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Patient case studies

Patient Centricity
  • Change of the natural history model
  • Lessons learned
  • Capture quality of life
  • How to make good engagement with patients (industry and physicians) how PAGs should initiate this
Nov 1810:40
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How the standard of care in rare diseases can be affected by regional challenges

Science & Strategy
  • Rare diseases overview
  • A case study of DMD in the MEA region
Richard White, Chief Operating Officer, Oxford PharmaGenesis Ltd
Nov 1811:40
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Panel: Evaluating cross border access to treatment and healthcare in Europe

Cell & Gene Therapy
  • EU cross-border healthcare still requires improvement; What other solutions can we implement?
  • Commission consultation – what should we convey to the commission?
  • Suggestions for regulation revision
  • What should be the roles of ERN’s in cross border health
  • Can we shift the system to collaborate between local jurisdictions?
  • Can we create a sustainable system for patients and a profitable model for the industry?
Moderator: Matt Bolz-Johnson, ERN & Healthcare Advisor, EURORDIS
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Michela Gabaldo, Head Of Alliance Management, Fondazione Telethon
Thomas Bols, Head of Government Affairs and Public Policy, EMEA & APAC, PTC Therapeutics
Vinciane Pirard, Senior Director Government Relations, Orchard Therapeutics
Maurizio Scarpa, Director, Coordinating Center For Rare Diseases, MetabERN
Martin Dorazil, Deputy Head of Unit, European Reference Networks and Digital Health, European Commission
Nov 1811:40
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How is Real World Data used to provide additional evidence for conditional regulatory approvals and initial reimbursement decisions?

Science & Strategy
  • European initiatives exploring the potential for RWD to generate real-world evidence (RWE) that can inform key healthcare decisions about treatment efficacy, value and optimization of patient care
  • The potential for better collaboration and joint learnings
Toon Digneffe, Head of Public Affairs And Public Policy, Takeda
Nov 1811:40
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Panel: The Importance of Natural History Study to Rare Disease Drug Development

Patient Centricity
Activating the Patient Voice to Drive Clinical Development SuccessMore speakers to be announced
Nov 1812:10
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Panel: Creating successful business models for ultra-rare diseases

Science & Strategy
  • How do we create profitable business models for ultra-rare diseases and translate these scientific discoveries into reality?
  • Company case studies: Exploring how to successfully launch an ultra-rare disease drug/ gene therapy in a patient-centric world
  • Overcoming the unique challenges of the ultra-rare space; Innovation, patient group identification, patient engagement, pricing, navigating through approval processes and ensuring patient access
Angela Columbano, Head Of Business Development And Partnership, Genethon
Robin Kenselaar, Senior Vice President And General Manager EMEA, Orchard Therapeutics
Sander Van Deventer, Operating Partner, Forbion Capital Partners
Nov 1812:40
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The Duchenne parent project's model and the use of online programs to help patients during the pandemic

Patient Centricity
Marisol Montolio Del Olmo, Scientific Director, Duchenne Parent Project Spain
Nov 1814:00
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How to expand and harmonize newborn screening programs across Europe?

Keynotes
  • How do we see NBS – what are the benefits to the baby and the whole family?
  • Actionable rare diseases to be included NBS
  • How can we make NBS more equitable across Europe?
  • How much further do we need to go and where are the gaps?
  • Building a new multi-stakeholder roadmap at a national level to provide scientific and ethical evidence for better NBS and quality of life
  • How do we bridge our efforts across different initiatives?
  • How could be use the latest technologies to streamline, improve and expand processes on a technical level
Moderator: Virginie Bros-Facer, Scientific Director, EURORDIS
Giancarlo La Marca, Head, Newborn Screening, Clinical Chemistry and Pharmacology Lab, University Of Florence And Meyer Children's Hospital
Nicolas Garnier, Director of Patient Advocacy, Global Product Development, Pfizer
Alessandra Ferlini, Director of the Medical Genetics Section & Unit, University of Ferrara
Charlotte Chanson, Director Global Diagnostics, Orchard Therapeutics
last published: 16/Sep/21 13:45 GMT

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