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Orphan Drugs & Rare Diseases
8 October - 9 October 2012
Orphan Drugs & Rare Diseases

The call to treat rare diseases has become louder around the world in recent years than ever before. Supportive organisations including patient groups, national health authorities, health trusts, academia, HTAs, pharmaceutical and biotechnical companies are now progressively growing in number and sophistication in their approaches to medicinal care for those with rare and ultra-rare diseases. This conference will present briefings from key opinion leaders, those with hands-on experience of regulating new drug discoveries, companies who have already developed advanced orphan drugs & successfully been granted orphan medicine status and selected experts in the field. Speakers will offer unique insights into cutting-edge international developments taking place in: expanding the reach of medicine to previously untreatable and unreachable patients with rare diseases; different regulatory and policy environments; new drug discoveries; innovative business strategies & funding & financing models, and the importance of partnerships with patient groups and those at the point-of-care.

Pharmaceutical companies, biotechnology companies, academic research units and other stakeholders organisations are increasingly merging their research, resources and expertise in the critical process of discovering new biopharmaceutical therapies to improve the lives of rare disease sufferers. Orphan Drugs & Rare Diseases will provide an exclusive opportunity to network with these leading experts on not only rare diseases research but ultra-rare diseases and the medicinal solutions currently being invented to treat them.
 

FEATURED SPEAKERS

Carlos  Camozzi

Carlos Camozzi

Vice President & Chief Medical Officer, uniQure
Gary Clements

Gary Clements

Senior Director, Business Development, Shire
Geraldine Honnet

Geraldine Honnet

Head of Clinical Development, Genethon
Martine Zimmermann

Martine Zimmermann

Executive Director Regulatory Affairs, Alexion Pharmaceuticals
Richard Philipson

Richard Philipson

Disease Area Head for Immuno / Inflammation, GlaxoSmithKline, Neurosciences MDC

Anders Waas

Chief Executive Officer, Tikomed
Anders Waas

Carlos Camozzi

Vice President & Chief Medical Officer, uniQure
Carlos  Camozzi

Donald Macarthur

Independent Consultant & Principal, Justpharmareports
Donald Macarthur

Elizabeth Hernberg-Stahl

Senior Consultant, Late Phase Solutions Europe
Elizabeth Hernberg-Stahl

Gary Clements

Senior Director, Business Development, Shire
Gary Clements

Geraldine Honnet

Head of Clinical Development, Genethon
Geraldine Honnet

Josie Godfrey

Head of Policy and Coordination, Advisory Group for National Specialised Services (AGNSS)
Josie Godfrey

Karen Aiach

Chief Executive Officer and Founder, Lysogene
Karen Aiach

Martine Zimmermann

Executive Director Regulatory Affairs, Alexion Pharmaceuticals
Martine Zimmermann

Oliver Timmis

Communications Project Manager, AKU Society
Oliver Timmis

Olivier Menzel

President & Founder, Blackswan Foundation
Olivier Menzel

Rafael Yanez

Senior Lecturer, Director of Planning and Resources , Royal Holloway College, University of London
Rafael Yanez

Richard Philipson

Disease Area Head for Immuno / Inflammation, GlaxoSmithKline, Neurosciences MDC
Richard Philipson

Segolene Ayme

Chair, EUCERD
Segolene Ayme

Stephen Nutt

Chief Executive, Rare Disease UK
Stephen Nutt

Tony Hall

Co-Founder, Findacure Foundation
Tony Hall

Conference agenda

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8:30

Registration & Coffee

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9:00

Welcome – introduction to orphan drugs

  • Background
  • Regulations
  • Definitions
  • Incentives
  • Relevant regulatory bodies
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    9:45

    Guide to writing an orphan drug application (EU & US)

  • Procedure
  • Sections of application
  • Sub-setting
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    10:45

    Morning Coffee

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    11:15

    Designing clinical trials for orphan indications

  • Choice of endpoint
  • Ethical issues
  • Protocol assistance
  • Predictors of success or failure
  • Surrogates
  • Guidance documents
  • Case studies
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    12:15

    Registries for rare diseases

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    13:00

    Networking Lunch

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    14:00

    Rare diseases from the patient’s perspective

  • Problems with diagnosis
  • Isolation
  • Lack of medical expertise
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    14:45

    Patient advocacy groups

  • Roles & achievements of EURORDIS
  • NORD & individual patient groups
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    15:30

    Afternoon Tea

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    16:00

    Market access and reimbursement for orphan drugs

  • Value of orphan drugs
  • Variations in patient access between countries
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    17:00

    Conclusions and end of workshop

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

    Tony Hall

    Tony Hall, Co-Founder, Findacure Foundation

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    9:10

    Shire’s orphan drug philosophy: creating stakeholder value

    Gary Clements

    Gary Clements, Senior Director, Business Development, Shire

  • The past: the early years of orphan drugs and Shire’s emerging leadership
  • The present: delivering value, growth and consolidation
  • The future: embracing innovation – imagining the possibilities:leading through the challenges
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    9:50

    Orphan drugs and rare disease policy in the UK: perspectives from a patient group

    Stephen Nutt

    Stephen Nutt, Chief Executive, Rare Disease UK

  • Towards a successful patient-focused national strategy
  • Impact of policy and regulations on access to orphan drugs
  • What are the obstacles and limitations for access to treatment?
  • Partnership working to develop personalised treatment and care
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    10:30

    Morning Coffee

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    11:00

    The BLACKSWAN Foundation: a unique organisation supporting orphan disease research

    Olivier Menzel

    Olivier Menzel, President & Founder, Blackswan Foundation

  • Promoting and supporting research into rare disease
  • Research priorities
  • The RE(ACT) Congress: Bringing together researchers and their knowledge
  • The RE(ACT) Community: an online platform to strengthen synergies between people involved in rare and orphan disease research
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    11:40

    Highly specialised drugs and technology: appraisals and selection

    Josie Godfrey

    Josie Godfrey, Head of Policy and Coordination, Advisory Group for National Specialised Services (AGNSS)

  • Health Technology Assessment requirements for highly specialised drugs and technologies
  • Assessing the effectiveness, costs and impact of new treatments
  • How to demonstrate comparative value
  • Developing a productive relationship between the health authority and industry
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    12:20

    Networking Lunch

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    13:30

    Innovative models for transferable R&D

    Karen Aiach

    Karen Aiach, Chief Executive Officer and Founder, Lysogene

  • Combining complex scientific expertise to improve rare disease therapy
  • Achieving Business & Research relationships to progress orphan drug discovery
  • What are the financial parameters for rare disease treatment and orphan drug development?
  • Partnership with patients' families, communities and patient groups: the scientific and ethical commitments
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    14:10

    Building a sustainable pipeline in rare diseases: from discovery to commercialisation

    Richard Philipson

    Richard Philipson, Disease Area Head for Immuno / Inflammation, GlaxoSmithKline, Neurosciences MDC

  • Rare disease prioritization: how to maximize R&D potential
  • Fuelling the R&D machine: internal discovery engines and external partnerships
  • Maximising the opportunities for new technologies in rare diseases
  • From discovery to treatment and commercialization
  • Case study: an innovative model for developing treatments
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    14:50

    Afternoon Tea

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    15:10

    Orphan drugs clinical trials

    Geraldine Honnet

    Geraldine Honnet, Head of Clinical Development, Genethon

  • Global Clinical Development
  • Specific gene therapy trials challenges: regulatory submissions, design, recruitment, statistics
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    15:50

    Is reimbursement for gene therapy different from other treatments?

    Carlos  Camozzi

    Carlos Camozzi, Vice President & Chief Medical Officer, uniQure

  • Conventional reimbursement for medicines in Europe and variations between nation states
  • Expectations for a new treatment modality
  • Impact on pharmacoeconomic parameters
  • Regulatory considerations
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    16:30

    Chairman’s Closing Remarks and Close of Day One

    Tony Hall

    Tony Hall, Co-Founder, Findacure Foundation

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

    Donald Macarthur

    Donald Macarthur, Independent Consultant & Principal, Justpharmareports

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    9:10

    Achieving orphan medicine status and regulation of specialised technologies: an industry perspective

    Anders Waas

    Anders Waas, Chief Executive Officer, Tikomed

  • Incentives for industry to develop rare disease medicines and technologies
  • Working with partners to enable regulatory and clinical development
  • Orphan status and how to approach intellectual property issues
  • Developing innovative and entrepreneurial strategies in the orphan drug business
  • clock

    9:50

    Working with regulators: a market overview

    Martine Zimmermann

    Martine Zimmermann, Executive Director Regulatory Affairs, Alexion Pharmaceuticals

  • The current European and global regulatory environment for orphan drugs
  • An industry overview on achieving global orphan designation Status
  • Comparisons between international country regulations
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    10:30

    Morning Coffee

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    11:00

    The benefits of patient groups, pharmaceutical companies and health institutions working together

    Segolene Ayme

    Segolene Ayme, Chair, EUCERD

  • Learning from each other
  • Political and practical achievements so far due to this partnership
  • Points on the agenda for the years to come
  • Areas for public/private partnership
  • clock

    11:40

    Partner with ePatients to maximize the value of real world data

    Elizabeth Hernberg-Stahl

    Elizabeth Hernberg-Stahl, Senior Consultant, Late Phase Solutions Europe

  • State-of-the-art late phase drug development
  • ePatients- the empowered patients
  • Regulations, social media and future perspectives
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    12:20

    Networking Lunch

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    13:30

    Regulations, incentives and technology assessments for gene therapy in rare diseases

    Carlos  Camozzi

    Carlos Camozzi, Vice President & Chief Medical Officer, uniQure

    • Research and development far ahead from regulations adaptation
    • Value of scientific advice and protocol assistance
    • Fees, grants and tax reduction
    • How to assess innovative, breakthrough technology with past knowledge

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    14:10

    Orphan drug policy and its results in Asian countries

    Donald Macarthur

    Donald Macarthur, Independent Consultant & Principal, Justpharmareports

  • Japan, Korea, Taiwan
  • Incentives to orphan drug sponsors
  • Role of HTA and risk sharing in reimbursement
  • Special funding for orphan drugs
  • Case studies
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    14:50

    Is the business model for orphan drug development sustainable and are there any alternatives?

    Tony Hall

    Tony Hall, Co-Founder, Findacure Foundation

  • Efficacy of the incentives for orphan drugs
  • Pricing & reimbursement issues
  • The sustainability of the current business models
  • Social enterprise – an alternative to the traditional approach?
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    15:30

    Afternoon Tea

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    15:50

    Consortium Cooperation in Rare Disease Research and Orphan Drug Development: Two Case Studies

    Oliver Timmis

    Oliver Timmis, Communications Project Manager, AKU Society

    • An International Consortium: Research, resources and expertise sharing
    • Collaborative rather than competitive working for the end result
    • An innovative model of drug developments and business strategy: a not-for-profit approach: the AKU Society
    • Future developments and trends in rare disease treatments

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    16:30

    Gene therapy: an overview and selected clinical studies

    Rafael Yanez

    Rafael Yanez, Senior Lecturer, Director of Planning and Resources , Royal Holloway College, University of London

    clock

    17:10

    Chairman’s Closing Remarks and Close of Day Two

    Donald Macarthur

    Donald Macarthur, Independent Consultant & Principal, Justpharmareports


    Communications Project Manager
    AKU Society
    Senior Lecturer, Director of Planning and Resources
    Royal Holloway College, University of London
    Executive Director Regulatory Affairs
    Alexion Pharmaceuticals
    Disease Area Head for Immuno / Inflammation
    GlaxoSmithKline, Neurosciences MDC
    Senior Director, Business Development
    Shire
    Chair
    EUCERD
    Chief Executive
    Rare Disease UK
    Chief Executive Officer
    Tikomed
    President & Founder
    Blackswan Foundation
    Co-Founder
    Findacure Foundation
    Senior Consultant
    Late Phase Solutions Europe
    Vice President & Chief Medical Officer
    uniQure
    Independent Consultant & Principal
    Justpharmareports
    Chief Executive Officer and Founder
    Lysogene
    Head of Policy and Coordination
    Advisory Group for National Specialised Services (AGNSS)
    Head of Clinical Development
    Genethon

    Workshops

    Orphan Drugs - From Application to Market Access

    Orphan Drugs - From Application to Market Access

    Copthorne Tara Hotel
    10 October 2012
    London, United Kingdom

    Copthorne Tara Hotel

    Scarsdale Place
    Kensington
    London W8 5SR
    United Kingdom

    Copthorne Tara Hotel

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.

     

    HOTEL BOOKING FORM

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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