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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.
 

SMi's symposium will demonstrate innovative models for translational research; the global economic environment for orphan drugs; how to bring your orphan drugs to the patient and to market successfully and other key business strategies that companies must consider when entering and working in the orphan drugs field.

Attend to hear perspectives from rare diseases trusts, patient groups, regulators & HTAs, academia, pharmaceutical and biotechnology companies as they give presentations on: health technology appraisals & market access; the process of achieving orphan drug status; reimbursement, and new patient treatments.

  • Learn about ultra-rare diseases and the very latest orphan drug developments
  • Network with regulators and policy makers of orphan medicines
  • Discover how orphan drugs can be made more accessible to patients
  • Analyse how R&D and market access costs can be reduced
  • Discuss successful funding, financial and economic models for orphan drug development and translational research
  • Consider the ethical and social factors versus drugs-to-make money

Chief Executives, Chief Scientific Officers, Vice Presidents, Heads, Directors, Principal Scientists and Managers in the following areas:

  • Rare Diseases
  • Orphan Medicines
  • Drug Policy
  • Policy and Coordination
  • Medical Advice
  • Drug Development
  • Biotherapeutics
  • Market Access
  • Government Affairs
  • Partnerships
  • Regulatory Affairs
  • Medical Science Liaison
  • External Affairs
  • Pharmaceutical Law
  • Business Development
  • Medical Technologies Evaluation
  • Public Policy
  • Outcomes and Reimbursement

 

Conference programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Tony Hall

Tony Hall, Co-Founder, Findacure Foundation

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
  • 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
  • 10:30 Morning Coffee

    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
  • 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
  • 12:20 Networking Lunch

    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
  • 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
  • 14:50 Afternoon Tea

    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
  • 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
  • 16:30 Chairman’s Closing Remarks and Close of Day One

    Tony Hall

    Tony Hall, Co-Founder, Findacure Foundation

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Donald Macarthur

    Donald Macarthur, Independent Consultant & Principal, Justpharmareports

    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
  • 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
  • 10:30 Morning Coffee

    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
  • 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
  • 12:20 Networking Lunch

    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

    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
  • 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?
  • 15:30 Afternoon Tea

    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

    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

    17:10 Chairman’s Closing Remarks and Close of Day Two

    Donald Macarthur

    Donald Macarthur, Independent Consultant & Principal, Justpharmareports

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    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

    Workshops

    Orphan Drugs - From Application to Market Access
    Workshop

    Orphan Drugs - From Application to Market Access

    Copthorne Tara Hotel
    10th 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, SMI 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@smi-online.co.uk

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