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
    • 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
Rick Thompson
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

Ioana Kloos
14:00

Workshop D: Overcoming Clinical & Regulatory challenges in ATMPs

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
17:00

End of Workshops and Welcome Drinks

last published: 13/Sep/19 10:35 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
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

11:30

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

11:30

Building a new infrastructure for long term evidence generation with use of registries

11:30

Discussion on experiences and ideas: How could Artificial Intelligence improve uncovering unmet needs in rare diseases and enhance patient identification

Senior representative, Syntheract
11:30

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

11:30

Gene editing revolution: Hope for rare diseases, but should there be limits?

11:30

How can we overcome the clinical development challenges facing advanced therapies for rare diseases?

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

Market entry strategies for orphan drugs in Central Eastern Europe

11:30

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

11:30

The impact of Brexit and Europe-wide effects from market access to the UK, patient collaborations across Europe

round tables
11:30

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

Nicola Allen
Spanish Session
11:30

Patient Access: Working with a national health service and HTA patient’s participation

12:20

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

round tables
12:20

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

12:20

Challenges of developing an orphan drug worldwide

12:20

Developments in Eastern Europe: Unique challenges and opportunities

12:20

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

12:20

How to build an ecosystem for rare diseases, from private to public

12:20

Inclusion of “nontraditional” as well as “low and middle” income patient populations in all aspects of drug development

12:20

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

12:20

Pricing & reimbursement for cell & gene therapies – Are the models really that different from normal rare disease models?

12:20

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

12:20

The US healthcare model: Increasing patient affordability and access for oncology treatment

Melissa Paige, Uva Cancer Center, Oncology Patient Access Principal Coordinator, Patient Advocate, U.V.A. Health System
12:20

Unique challenges in Brazil for orphan drug access and pricing

13:05

Networking Lunch & Patient Poster Session

Access & Pricing
14:15

Chair opening remarks

Science & Strategy
14:15

Chair opening remarks

Precision Medicine
14:15

Chair opening remarks

Cell & Gene Therapy
14:15

Chair opening remarks

Manufacture
14:15

Chair opening remarks

14:19

Patient Centricity & Trial Design

14:19

The Value of Orphan Drugs

14:19

Case Studies

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

Clinical Development
14:45

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

Manufacture
14:45

Developing robust manufacturing processes for novel gene therapies

- Overcoming manufacturing challenges faced by therapy developers- Effectively preparing for later stage trials and commercialization
Manufacture
15:10

Capacity crunch? The future of manufacturing within cell & gene therapy

Science & Strategy
15:35

Success factors for rare disease launches and partnering

Precision Medicine
15:35

Europe: Taking the lead on ethical AI

Cell & Gene Therapy
15:35

Clinical updates on Pfizer’s gene therapy pipeline

16:15

Afternoon Networking refreshment break

16:44

Showcases Pitch & Partner

Science & Strategy
16:45

Showcases

Synthetic plasmalogen-based therapy for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP) and other rare pediatric peroxisomal disorders
Cell & Gene Therapy
16:45

LentiGlobin Gene Therapy in Transfusion Dependent β-Thalassemia

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
Manufacture
17:10

Deciding on an effective manufacturing strategy: Choosing to outsource your manufacturing

Science & Strategy
17:15

Showcases

Suzanne Saffie-Siebert, Chairwoman And Chief Executive Officer, SiSaf Ltd
Science & Strategy
17:30

Showcases

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

Science & Strategy
17:45

Panel: Critique of showcases & how to attract investment and progress your pipeline?

Private investment sources for orphan drugsInvestigating the funding opportunities available in Europe and globallyHow to attract larger players to collaborate
18:25

Chair’s closing remarks

18:30

Evening Networking Drinks

last published: 13/Sep/19 10:35 GMT

Nov 14th Day 2

07:59

OPENING KEYNOTE PLENARY

08:00

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

Clinical Development
11:35

Chair opening remarks

Access & Pricing
11:35

Chair opening remarks

Science & Strategy
11:35

Chair opening remarks

Precision Medicine
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

Alternative business models for ODs: Collaboration

11:39

Advanced Therapies for Orphan Diseases

11:39

Reimbursement, Patient Access & Investment in Cell & Gene Therapy

11:39

Scale Up of Cell & Gene Therapies

Science & Strategy
11:40

The emerging business models for commercializing ODs and gene therapy treatments

Precision Medicine
11:40

Novel Approaches to Clinical Trials in Rare Neurological Disorders

  • Outcomes from OV101 STARS in Angelman syndrome and OV935 adults with developmental epileptic encephalopathies
Dr Amit Rakhit, Chief Medical And Portfolio Management Officer, Ovid Therapeutics
Clinical Development
12:10

A landmark case of a multi-stakeholder collaborative clinical trial delivering a repositioned drug for an ultra-rare condition

Science & Strategy
12:10

Patient-led companies with alternative business models

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.
Manufacture
14:10

Overcoming bottlenecks in AAV manufacturing for gene therapy

Access & Pricing
14:40

Valetta Declaration

Science & Strategy
14:40

Panel from our companies/organisations: what are the long-term implications of these alternative business models?

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?
Precision Medicine
14:40

Developing precision genetic medicines through base editing

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?
Panel discussion
17:30

Chair’s Closing Remarks & Close of congress

last published: 13/Sep/19 10:35 GMT

 

 

World Orphan Drug Congress 2019 agenda overview