World Orphan Drug Orphan Congress Agenda

 

 

 

Barcelona, 12 - 14 November 2019

Schedule

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Nov 1210:00
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Workshop A: Balancing on the Edge – Value Evidence and Market Access for Orphan Drugs in the US and EU

Keynotes
  • Getting the right balance
    • Drug development decisions are primarily based on commercial opportunities; US, EU and other geographies tend to have different requirements and offer different opportunities
  • Orphan Drugs and Oncology Orphans: Time to access and availability in the US and EU5
    • Success stories and remarkable failures may tell us interesting lessons
  • ​​​​​​​Paths to Optimal Evidence Generation Plans for ODs
    • ​​​​​​​Gathering payers’ and HTA agencies’ requirements as part of a company’s evidence generation plan, designed to achieve access and not just regulatory approval
  • ​​​​​​​From theory to reality: ‘Innovative’ approaches to gross to net pricing
    • US contracting and EU managed entry agreements may allow to achieve an acceptable balance between access and budget constraints
  • ​​​​​​​Optimal evidence vs. Market Access risk: Is there an optimal balance?
    • ​​​​​​​Industry and HTA Bodies’ have different perspectives, but a compromise is needed for patients to access new promising OD therapies
Nov 1210:00
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Workshop B: Drug Repurposing Use for Orphan Drugs Industry vs patient rep & off-label vs label use: Transparency, incentives and approaches to help industry repurpose

Keynotes
  • Off-label Prescribing: Justifying Unapproved Medicine
  • Results from an off-label study and its impact in AKU patients in parallel with the international clinical trial
  • Update on STAMP
  • Industry perspective
  • Incentivising generics manufacturers to repurpose drugs
  • Generics of ex-orphan drugs: Uses and controversies
Nov 1214:00
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Workshop C: Overcoming Clinical & Regulatory challenges in ATMPs

Keynotes
This workshop provides expert guidance and practical case studies to help address the major challenges of:· Drug candidate early development· Regulatory strategy & Trial design· Real patient engagement & Market access· Unique challenges in the manufacturing and commercialization of gene therapies for rare diseases
Nov 1214:00
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Workshop D: Gene and cell therapies in rare diseases: creating a sustainable business model and pathway to patient access

Keynotes
The full potential of innovative new gene and cell therapies will only be realised through the creation of a sustainable business model and a clear pathway to access for rare disease patients. Dolon’s interactive workshop provides you with the opportunity to explore the complexities of pricing an orphan drug, using a series of exercises that explore the orphan drug business model, how pricing and investment decisions are made, and how policy impacts innovation.The RARE-IMPACT initiative, chaired by Eurordis and supported by Dolon, is a consortium of rare disease manufacturers that is engaging with HTA agencies, regulatory bodies, payers, patient groups, clinicians and other experts across Europe, to identify and validate the challenges to patients’ access to gene and cell therapies.

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Nov 1308:25
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What incentives are available for developing orphan drugs? Is the current orphan drug legislation & incentives enough?

Keynotes
  • Findings from the commission’s value assessment evaluation report – should guidelines be amended?
  • Feedback and alignment from regulators and industry
  • Considerations in the unaccounted costs for industry, society values of orphan drugs and the role of competition
  • Are our policy makers direction in line with what society wants?
Nov 1309:10
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What guidance can we give industry when working with European Reference Networks (ERNs) and R&D?

Keynotes
  • Roadmap for industry: How should industry be engaging with ERNs and what does each have to offer to improve clinical and research aspects?
  • Company and ERN perspectives, experiences so far
  • What can we learn from US CRNs?
  • How can ERNs & CRNs collaborate?
  • Stakeholder engagement with ERNs – industry and patients beyond academic and clinical networks
Nov 1309:55
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Executive Insights: What technologies need to be unlocked? Discussing all the scientific aspects that are impacting the reality of gene therapy

Keynotes
  • What is the science driving your business model, how do we overcome the technological constraints?
  • Challenges in creating robust pipelines sustainable to patients (selecting indications, tissue targeting/distribution, immunogenicity, limitations of vectors)
  • How have gene therapies in the past influenced our current pipelines?
  • Realities of manufacturing & commercialisation
Nov 1311:30
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11:30 – 12:15: Roundtable and Q&As on plenary orphan drug legislation & incentives

Keynotes
Nov 1311:30
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Balance between medical and promotional (sales force) in achieving brand objectives in rare diseases

Keynotes
Nov 1311:30
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Best practices and tools to get patients groups to fully engaged

Keynotes
Nov 1311:30
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Building a new infrastructure for long term evidence generation with use of registries

Keynotes
Nov 1311:30
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Expediting drug development in rare disease: Are big data and biomarkers an option for the future?

Keynotes
Nov 1311:30
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Gene editing revolution: Hope for rare diseases, but should there be limits?

Keynotes
Nov 1311:30
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How can we overcome the clinical development challenges facing advanced therapies for rare diseases?

Keynotes
Nov 1311:30
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Leveraging patient-focused technological solutions to support better diagnosis and access to new treatments for rare disease patients

Keynotes
Nov 1311:30
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Market entry strategies for orphan drugs in Central Eastern Europe

Keynotes
Nov 1311:30
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Patient organizations as partners: Including “resource” issues from support for education to support for supporting patient communities

Keynotes
Nov 1311:30
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The impact of Brexit and Europe-wide effects from market access to the UK, patient collaborations across Europe

Keynotes
Nov 1311:30
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The Italian experience: How has our experience changed how we manage ODs?

Keynotes
Nov 1311:30
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The patient voice: An important consideration not only for late stage reimbursement, but also for early stage regulatory affairs

Keynotes
Nov 1312:20
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12:20 – 13:05 Roundtable and Q&As on plenary ERNs

Keynotes
Nov 1312:20
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Bringing ATMPs to the patients: How to take an integrated approach towards solving regulatory and market access hurdles

Keynotes
Nov 1312:20
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Cell & gene therapy: Can we manufacture and commercialise in a sustainable way?

Keynotes
Nov 1312:20
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Challenges of developing an orphan drug worldwide

Keynotes
Nov 1312:20
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Developments in Eastern Europe: Unique challenges and opportunities

Keynotes
Nov 1312:20
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From Endpoints to Endgame: Incorporating the diagnostic odyssey and patient journey in your clinical development plan

Keynotes
Nov 1312:20
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How to build an ecosystem for rare diseases, from private to public

Keynotes
Nov 1312:20
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Inclusion of “nontraditional” as well as “low and middle” income patient populations in all aspects of drug development

Keynotes
Nov 1312:20
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Pricing & reimbursement for cell & gene therapies – Are the models really that different from normal rare disease models?

Keynotes
Nov 1312:20
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Regulatory & operational implementation of the direct to patient trial management mode

Keynotes
Nov 1312:20
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The US healthcare model: Increasing patient affordability and access for oncology treatment

Keynotes
Nov 1312:20
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Unique challenges in Brazil for orphan drug access and pricing

Keynotes
Nov 1312:20
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Use of registries on OMPs post launch registration: Experience from Germany

Keynotes
Nov 1314:15
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Nov 1314:20
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Pre-approval access, a key component of your strategic drug development and market access plan in rare and orphan disease – Why? When? How?

Access & Pricing
Nov 1314:20
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Case study: How a biotech can successfully raise funds for rare disease drug development

Science & Strategy
Nov 1314:20
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Artificial Intelligence: Promise & Reality

Precision Medicine
Nov 1314:20
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Novartis: CAR T Innovation Journey

Cell & Gene Therapy
Nov 1314:20
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Industrialization of gene therapy manufacturing

Manufacture
Nov 1314:45
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Panel: Perspectives from the industry in early access strategies

Access & Pricing
Anant Murthy, Vice President Of Market Access And Pricing, Alnylam Pharmaceuticals
Nov 1314:45
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Case study: Systematic drug re-innovation and clinical evidence in orphan diseases

Science & Strategy
Nov 1314:45
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The use of artificial intelligence and machine learning to drive innovation for rare disease treatments

Precision Medicine
Nov 1314:45
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Regulatory issues in ATMPs

Cell & Gene Therapy
Nov 1314:45
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Developing robust manufacturing processes for novel gene therapies

Manufacture
- Overcoming manufacturing challenges faced by therapy developers- Effectively preparing for later stage trials and commercialization
Nov 1315:10
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Patient reported outcomes measures: An interpretable measure of patient benefit to demonstrate clinical effectiveness

Clinical Development
Thomas Morel, Director, UCB Pharma
Nov 1315:10
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How can we get high quality data from patients for precision medicine?

Precision Medicine
Nov 1315:10
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Treating Fabry disease: Phase 2 clinical updates and expansion of clinical activities into other indications

Cell & Gene Therapy
Nov 1315:30
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Panel: Commercial negotiations in HTA

Access & Pricing
Sheela Upadhyaya, Associate Director Hst, National Institute for Health and Care Excellence
Nov 1315:35
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How can technology and patient-focused approaches accelerate clinical development? orted outcomes measures: An interpretable measure of patient benefit to demonstrate clinical effectiveness

Clinical Development
Nov 1315:35
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Advancement of analytical tools for testing and characterization of AAV gene therapy products

Manufacture
Nov 1316:45
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The RUDY study to inform novel biomarkers and therapeutic targets with patients

Clinical Development
Nov 1316:45
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Tool for reducing uncertainties in evidence generation for specialised treatments for rare diseases: TRUST4RD

Access & Pricing
Tackling uncertainties for rare diseases - The use of RWE to improve access for patients
Karen Facey, Senior Research Fellow, University of Edinburgh
Nov 1316:45
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Showcases

Science & Strategy
Synthetic plasmalogen-based therapy for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP) and other rare pediatric peroxisomal disorders
Nov 1316:45
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Newborn screening for rare diseases: Early diagnosis, treatment essential

Precision Medicine
Nov 1316:45
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Defining effective manufacturing & regulatory standards

Manufacture
Nov 1317:10
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A partnership that lasts- key considerations when working with CROs on rare disease studies

Clinical Development
  • Mapping out the road to success (Sponsor/CRO relationship, operational success, and ethical considerations)
  • The importance of the patient: Patient advocacy
  • Reducing patient burden
  • Partnering and Trust
  • Roles of CRO and Sponsor and defining scope
  • Setting clear expectations for enrollment and retention: Site (KOls)/Patient identification, and patient recruitment
  • Understanding evolving regulatory climate
  • Ethical considerations
Nov 1317:10
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What is the relationship between price and prevalence in non-oncology rare disease?

Access & Pricing
In the past, payers have always valued rarity in their pricing decisions. However, the environment is changing as the collective budget impact of rare diseases is increasing. Despite provision of alternative mechanisms for orphan drugs, payers are tougher on prices, negotiating rebates & setting budget impact thresholds; they are also scrutinising the patient population as a means to reduce uncertainty. So, with a recent flurry of headline-grabbing high prices in the non-oncology rare disease space, we have set out to investigate what is the relationship between prevalence and price
Nov 1317:10
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Improving patient outcomes across the globe with the 100,000 genomes project rare disease programme

Precision Medicine
Nov 1317:10
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Homologous recombination: A precise and nuclease-free approach to gene editing

Cell & Gene Therapy
Nov 1317:35
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New models to run natural history studies and registries

Clinical Development
Perspectives from patients, CROs and regulators on sustainable RWE
Nov 1317:35
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Decentralised digital trials in populations with high unmet need - Next generation approaches

Precision Medicine
Nov 1317:35
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Translating a CRISPR platform into gene-edited cell therapies - the future is allogeneic

Cell & Gene Therapy
Nov 1317:35
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Strategies and advances in lentiviral vector manufacturing and scale-up

Manufacture
Nov 1317:45
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Panel: Critique of showcases & how to attract investment and progress your pipeline?

Science & Strategy
Private investment sources for orphan drugsInvestigating the funding opportunities available in Europe and globallyHow to attract larger players to collaborate
Nov 1318:00
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Coming to America? How to position your orphan and rare product for launch in the United States

Access & Pricing
Nov 1318:00
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What type of smartphone Apps are useful for patients? Empowerment through digital disease education created for patients with patients

Precision Medicine
Lucilla Franchetta Ortelli, Senior Manager, Alexion Pharma Germany Gmbh
Nov 1318:00
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Harnessing the body’s natural DNA repair process to treat paediatric rare diseases

Cell & Gene Therapy
Nov 1318:00
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Advances in cryopreservation technology to support the scalable supply requirements for cell and gene therapy

Manufacture

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Nov 1408:35
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EUnetHTA update - Showcase of early dialogue and common assessment

Keynotes
  • Hear from EMA’s scientific advice or accelerated approval and early consultation with EUnetHTA
  • How does this route differ?
  • Should companies continue with the usual EMA and access paths in parallel?
  • Perspectives from industry who have engaged in early dialogue and their experience
  • Perspectives from patients
Nov 1409:35
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Transatlantic synergies between innovative payment models

Keynotes
  • A global perspective on how split-cost and performance-based models work
  • What are the quality criteria used?
  • Investigating real-world learnings from European pricing models to incorporate into program designs
  • What do payers think of transformative therapies like gene therapy?
Francis Pang, Vice-President Global Market Access, Orchard Therapeutics
Sheela Upadhyaya, Associate Director Hst, National Institute for Health and Care Excellence
Nov 1411:40
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Improving healthcare communications in rare disease

Clinical Development
Gavin Jones, Director Of Rare Diseases, OPEN Health
Nov 1411:40
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Are Europe’s collaborative efforts working? Beneluxa & Horizon scanning

Access & Pricing
Nov 1411:40
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Novel Approaches to Clinical Trials in Rare Neurological Disorders

Precision Medicine
  • Outcomes from OV101 STARS in Angelman syndrome and OV935 adults with developmental epileptic encephalopathies
Nov 1411:40
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Germany and the CAR T cell therapy reimbursement model: Germany’s first outcomes-based deal

Cell & Gene Therapy
Nov 1411:40
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Scaling manufacturing to deal with the demands of commercial cell & gene therapy products

Manufacture
Nov 1411:55
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Patient-led organisation to successfully repurpose drugs

Science & Strategy
Nov 1411:55
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Nov 1412:10
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Update on V4+ initiative and ODs in Poland

Access & Pricing
MCDA as a tool in P&R decision making
Nov 1412:10
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The promise of RNA interference (RNAi) therapeutics for orphan diseases

Precision Medicine
Nov 1412:10
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Contrasting In-House and External Manufacturing Options

Manufacture
Nov 1414:10
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The role of patient advocacy in ensuring access to effective and transformative therapies

Clinical Development
Nov 1414:10
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Nordic HTA collaborations

Access & Pricing
Nov 1414:10
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Genetic surgery: Allele-specific gene editing using engineered CRISPR nuclease

Precision Medicine
Nov 1414:10
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Discover what payers really think of gene therapy

Cell & Gene Therapy
Nov 1414:40
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Industry panel: How are we incorporating the patients in a meaningful way?

Clinical Development
Gail Moore, Director Global Patient Advocacy, Horizon Pharma
Sonali Chopra, Associate Director, Genentech
Nov 1414:40
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Panel from our companies/organisations: what are the long-term implications of these alternative business models?

Science & Strategy
What are the differences to the more traditional model of pharma?What are the long-term implications?How do you collaborate, especially with very rare diseases to be sustainable?
Nov 1414:40
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Panel: European Investment landscape for cell & gene therapy

Cell & Gene Therapy
Nov 1415:10
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Therapeutic genome editing of Charcot-Marie-Tooth 1A disease

Precision Medicine
Nov 1415:10
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How can advanced therapy companies make their manufacturing process more cost effective and sustainable?

Manufacture
Nov 1416:00
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The impact of digital & computational tools on diagnostics and patient care

Keynotes
  • How will new diagnostic technologies in genomics, sequencing and AI change the way patients access diagnostics?
  • What impact will it have to physicians and at a hospital level?
  • Looking into the future, will these advances mean that treatments will become more accessible for all?
Nov 1416:45
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What are the real impacts & benefits of cross-border collaborations?

Keynotes
  • Measurable effectiveness to patient access to orphan medicines
  • How to navigate through Europe’s complex HTA landscape?
  • Bringing together perspectives from multiple collaborations
last published: 27/Jun/19 15:45 GMT