2019 Agenda

Pre-congress Workshops Orphan Nov 12

Dr Renato Dellamano
10:00

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?
Rick Thompson
10:00

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

Industry vs patient rep & off-label vs label use - Transparency, incentives and approaches to help industry repurpose
  • Results from an off-label study and its impact in AKU patients in parallel with the international clinical trial
  • Industry perspective
  • Update on STAMP
  • Incentivising generics manufacturers to repurpose drugs
  • Generics of ex-orphan drugs: Uses and controversies
Adam Hutchings
10:00

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

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.
13:00

Networking Lunch

Annie Hubert
14:00

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.
Farzana Malik
14:00

Workshop E: Demonstrating Treatment Value in Rare Diseases with Health Economics and Patient-Centred Outcomes

  • Introduction to Health Economics & Patient-Centred Outcomes (HE/PCO)
  • Relevance of Cost effectiveness analyses thresholds in ultra- rare diseases and HTA perspectives on what are “acceptable cost effectiveness thresholds”
  • When and how to Conduct HE/PCO studies in ultra- rare diseases & the related challenges throughout product life-cycle
  • Regulatory authorities’ (EMA & FDA) expectations on PCO assessments & steps to maximise chances of obtaining a PCO label claim in rare disease
  • Using PCO data to support HTA and market access strategies
17:00

End of Workshops and Welcome Drinks

last published: 10/Oct/19 13:15 GMT

Nov 13th Day 1

08:00

Registration + Welcome Refreshments

08:20

Chair’s opening remarks

Panel discussion
08:25

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
09:10

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
Panel discussion
09:55

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
10:40

Morning Networking Refreshment Break

11:29

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

NEW Spanish Session (moderated in Spanish) 11:30 – 13:05

  • Collaboration at regional level in orphan drug assessment
  • Learning from and scaling up best practices at regional level
  • OD assessment collaborative model engaging different stakeholder
  • HTA patient participation
11:30

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

11:30

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

11:30

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
11:30

Cell & gene therapy: Can we manufacture and commercialise in a sustainable way?

11:30

Challenges of developing an orphan drug worldwide

11:30

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

Senior representative, Syntheract
11:30

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

11:30

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

11:30

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

11:30

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

11:30

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

11:30

Regulatory & operational implementation of the direct to patient trial management mode

11:30

Sustaining launch success as orphan medicines come of age

11:30

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

11:30

Unique challenges in Brazil for orphan drug access and pricing

11:30

What’s the of role the MCDA approach in Orphan Drugs Evaluation?

12:20

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

13:05

Networking Lunch & Patient Poster Session

14:19

Patient Centricity & Trial Design

14:19

The Value of Orphan Drugs

14:19

Case Studies in Translating Science into Treatments

14:19

Discovery Diagnostics & Digital Health

14:19

Clinical Development: Cell & Gene Therapy

14:19

Improving Cell & Gene Therapy Manufacturing

Clinical Development
14:20

The use of biomarkers to support clinical success

Steve Maricich, Senior Medical Director, BioMarin
Clinical Development
14:50

Enabling drug discovery - the need for improved epidemiology in rare diseases

Manufacture
14:50

Industrialization of gene therapy manufacturing

- Overcoming manufacturing challenges faced by therapy developers- Effectively preparing for later stage trials and commercialization
Access & Pricing
15:30

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
Science & Strategy
15:35

Success factors for rare disease launches and partnering

15:49

Pitch & Partner Showcases

Manufacture
15:50

Developing robust manufacturing processes for novel gene therapies

  • Overcoming manufacturing challenges faced by therapy developers
  • Effectively preparing for later stage trials and commercialization
16:15

Afternoon Networking refreshment break

Science & Strategy
16:45

Showcase

Synthetic plasmalogen-based therapy for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP) and other rare pediatric peroxisomal disorders
Clinical Development
17:10

A partnership that lasts- key considerations when working with CROs on rare disease studies

  • 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
Access & Pricing
17:10

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
Science & Strategy
17:30

Showcase

Clinical Development
17:35

New models to run natural history studies and registries

Perspectives from patients, CROs and regulators on sustainable RWE
Lesli Nordstrom, Director Of Marketing And Communications, National Organization for Rare Disorders (NORD)
Access & Pricing
17:35

Strategies for obtaining orphan drug and other EMA designations for orphan products

Access & Pricing
18:00

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
18:25

Chair’s closing remarks per track

18:30

Evening Networking Drinks

last published: 10/Oct/19 13:15 GMT

Nov 14th Day 2

08:29

OPENING KEYNOTE PLENARY

08:30

Chair’s opening remarks

Panel discussion
08:35

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
Panel discussion
10:35

Morning Networking Break

Access & Pricing
11:35

Chair opening remarks

Science & Strategy
11:35

Chair opening remarks

Cell & Gene Therapy
11:35

Chair opening remarks

Manufacture
11:35

Chair opening remarks

11:39

Patient Centricity & Product Development

11:39

Cross-Country Collaboration on Evidence Generation

11:39

Case Studies & Toolkits for Collaborative Drug Development

11:39

Precision Medicine Approaches for Orphan Diseases

11:39

Reimbursement, Patient Access & Investment in Cell & Gene Therapy

11:39

Scale Up of Cell & Gene Therapies

Clinical Development
11:40

Improving healthcare communications in rare disease

David Edward Rose, Business Development And Sales Associate, Rare Revolution
Benjamin James, Masters Student, DMD Pathfinders
Precision Medicine
11:40

Machine learning for medical research: Mesothelioma case study

Pierre Courtiol, Chief Data Scientist, OWKIN
Clinical Development
12:10

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
Science & Strategy
12:25

Patient-led companies with alternative business models

12:40

Networking Lunch & Patient Poster Session

Science & Strategy
14:10

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
14:10

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
14:40

Valetta Declaration

Christopher Fearne, Minister, Minister For Health
Manufacture
14:40

Strategic partnerships between CDMO’s and industry to achieve commercialisation

Access & Pricing
15:10

Baltic Procurement Initiative

Panel discussion
15:30

Networking refreshment break

15:59

CLOSING PLENARY SESSIONS

Panel discussion
16:00

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?
Moderator: Denis Costello, Executive Director, CML Advocates Network
Panel discussion
17:30

Chair’s Closing Remarks & Close of congress

last published: 10/Oct/19 13:15 GMT

 

 

World Orphan Drug Congress 2019 agenda overview