2019 Agenda

Pre-congress Workshops Orphan Nov 12

Dr Renato Dellamano

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

  • Getting the right balance: US, EU, other geographies
  • Orphan drugs and oncology orphans: Time to access and availability in the US and EU5
    • Success stories and remarkable failures: lessons to learn
  • Paths to optimal evidence generation plans for ODs
    • Gathering payers’ and HTA agencies’ requirements as part of a company’s evidence generation plan: regulatory approval may not always mean access
  • From theory to reality: ‘Innovative’ approaches to gross to net pricing
    • US contracting and EU managed entry agreements
  • Optimal evidence vs. market access risk: Is there an optimal balance?
Adam Hutchings

Workshop C: Gene and cell therapies in rare diseases: creating a sustainable business model and pathway to patient access

Have you ever wondered…..
  • What determines which medicines get developed?
  • How are drug prices set and are they too high?
  • How much does it really cost to develop a medicine?
  • Are biopharma companies making too much profit?
….then join our session. The answer to these questions requires an understanding of the business model of the biopharma industry, and the best way to get that understanding is to start your own biopharma company! In this interactive simulation exercise you will be put in the shoes of a biopharma CEO and get to make a series of critical decisions:
  • Which potential medicines will you invest in?
  • Your trial data is in: What do you do? Go or No-Go?
  • Did your drug get approved? How will you price it?
  • If your drug failed, what are you going to tell your investors?!
Using the insight gained in the exercise, we will collectively address the biggest and most important questions about the sustainability of innovation in rare diseases.How is this working for gene and cell therapies in practice?In the second part of the workshop we explore the real world sustainability challenges for gene and cell therapies from the perspective of different stakeholders:A case study from industry perspective on the challenges to commercialisation and patient access to Strimvelis for ADA-SCIDOverview of the RARE IMPACT collaboration: how to realise the potential of gene and cell therapies in rare diseases by ensuring a pathway to patient access that is sustainable for all stakeholders.

Networking Lunch

Rick Thompson
Annie Hubert

Workshop D: Overcoming Clinical & Regulatory challenges in ATMPs

This workshop provides expert guidance and practical case studies to help address specific aspects of ATMP development in rare diseases, including:
  • Clinical development strategy & patient engagement
  • Regulatory strategy & early access
  • ATMP manufacturing & quality development
  • Planning for commercialisation & patient access
Nathalie Morgensztejn, Scientific Committee Member, A.N.S.M.

End of Workshops and Welcome Drinks

last published: 08/Nov/19 14:45 GMT

Nov 13th Day 1


Registration + Welcome Refreshments


Chair’s opening remarks

Panel discussion

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

  • 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?
Panel discussion

What guidance can we give industry when working with European Reference Networks (ERNs) and R&D?

  • 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
Moderator: Victoria Hedley, Rare Disease Policy Manager, Newcastle University
Panel discussion

Executive Insights: What technologies need to be unlocked? Discussing all the scientific aspects that are impacting the reality of gene therapy

  • 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

Morning Networking Refreshment Break


Choose 2 x Interactive Roundtables: 11:30 to 12:15 (rotation one) and 12:20 to 13:05 (rotation two).


11:30 – 12:15: Roundtable and Q&As on plenary orphan drug legislation & incentives


A data driven approach to assess the value of orphan drugs: the role of MCDA methodology and new technologies (Blockchain, AI/ML)


Balance between medical and promotional (sales force) in achieving brand objectives in rare diseases


Beyond the dossier. Engaging politicians, patients and campaigners in a conversation about value.

Crafting and communicating a compelling value narrative is not easy, particularly when talking about orphan medicines. In a world of accelerating health innovation, growing affordability concerns and the democratisation of health, we will explore how new voices are engaging publicly with orphan medicine reimbursement and shaping the outcome as a result. Key topics:
  • Crafting an orphan medicine value narrative that resonates beyond the dossier
  • Building constructive advocacy partnerships in support of a shared goal
  • Navigating the politicisation of rare diseases and its impact on reimbursement
round tables

Challenges of developing an orphan drug worldwide


How could Artificial Intelligence improve uncovering unmet needs in rare diseases and enhance patient identification


Insight into the US healthcare system: How do we ensure the right patient, the right drug at the right price?


Leveraging patient-focused technological solutions to support better diagnosis and access to new treatments for rare disease patients


Market access and orphan drugs-how we horizon scan for orphan drugs in the UK


Optimal Selection of Data and Technology for Use Throughout the Orphan Medicinal Product Life Cycle

Erwin De Cock, Principal, Late Stage, Syneos Health
round tables

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


Pricing & reimbursement for cell & gene therapies: Getting ready for timely access in Europe


Sustaining launch success as orphan medicines come of age


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


Unique challenges in Brazil for orphan drug access and pricing


Volv inTrigue. AI-based identification drives diagnosis and cost-effectiveness in rare diseases: helping you find undiagnosed and misdiagnosed rare-disease patients across 15 countries, earlier

Christopher Rudolf, Founder and CEO, Volv Global S.A.

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


Networking Lunch & Patient Poster Session

Science & Strategy

Chair opening remarks

Raghuram Selvaraju, Managing Director, h.c. wainwright

Patient Centricity & Trial Design


The Value of Orphan Drugs


Case Studies in Translating Science into Treatments


Discovery Diagnostics & Digital Health


Clinical Development: Cell & Gene Therapy


Improving Cell & Gene Therapy Manufacturing


Industrialization of gene therapy manufacturing

- Overcoming manufacturing challenges faced by therapy developers- Effectively preparing for later stage trials and commercialization

Developing robust manufacturing processes for novel gene therapies

  • Overcoming manufacturing challenges faced by therapy developers
  • Effectively preparing for later stage trials and commercialization

Afternoon Networking refreshment break


Pitch & Partner Showcases

Access & Pricing

What is the relationship between price and prevalence in non-oncology rare disease?

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
Access & Pricing

Panel: Commercial negotiations in HTA

Why, when and what do they achieve? The framework for conducting and tracking them
Amr Makady, Pharmacoeconomics Advisor, Zorginstituut Nederland
Access & Pricing

A patient-first approach to providing pre-approval treatment options for rare diseases

  • Serving patients through awareness of clinical trials and expanded access options
  • Utilizing Patient Navigators help patients access treatment options
    • Implementing pre-approval access programs that provide meaningful Real-World Data
    • Creating value for regulatory and payor-discussions

Chair’s closing remarks per track


Evening Networking Drinks

last published: 08/Nov/19 14:45 GMT

Nov 14th Day 2




Chair’s opening remarks

Panel discussion

EUnetHTA update - Showcase of early dialogue and common assessment

  • 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
Amr Makady, Pharmacoeconomics Advisor, Zorginstituut Nederland
Panel discussion

Morning Networking Break


Patient Centricity & Product Development


Cross-Country Collaboration on Evidence Generation


Case Studies & Toolkits for Collaborative Drug Development


Precision Medicine Approaches for Orphan Diseases


Reimbursement, Patient Access & Investment in Cell & Gene Therapy


Scale Up of Cell & Gene Therapies

Clinical Development

Improving healthcare communications in rare disease - Patient Community perspectives

David Edward Rose, Business Development And Sales Associate, Rare Revolution
Benjamin James, Masters Student, DMD Pathfinders
Cell & Gene Therapy

Italy’s new CAR T cell therapy reimbursement model

Clinical Development

Using ML/AI to improve access to clinical research for Rare Disease Patients: Evolving routes to engagement and identifying under-served communities

  • There is an increased needfrom both PAGSandindustry to find and connect with rare disease patients for clinical research
  • Current industry strategies often struggle to identify the optimalsites and maximise recruitment potential.Rare disease communities often voice that the clinical trial space and pooled data sources are hidden to them.
  • In this presentation, we will outline how AI/MLcanhelp harness the rare disease data ecosystem and support the creation of new opportunities,such as rare disease patient definition, disease detection and outcome prediction

Networking Lunch & Patient Poster Session

Science & Strategy

No longer an option – evolving market demands a fully integrated clinical and commercial approach for orphan drug life cycle management success

Sharing Case Example insights in terms of effective planning and execution for Rare and Orphan Medicinal Products, leveraging the strategic and operational experience from our presenters to highlight what is unique and requires different practices in progressing assets in the area of Rare Diseases.
Precision Medicine

Novel Approaches to Clinical Trials in Rare Neurological Disorders

  • Outcomes from OV101 STARS in Angelman syndrome and OV935 adults with developmental epileptic encephalopathies
Access & Pricing

Valetta Declaration

Christopher Fearne, Minister, Minister For Health
Panel discussion

Networking refreshment break



Panel discussion

The impact of digital & computational tools on diagnostics and patient care

  • 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?

Chair’s Closing Remarks & Close of congress

last published: 08/Nov/19 14:45 GMT



World Orphan Drug Congress 2019 agenda overview