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SMi are pleased to announce its second annual Orphan Drugs & Rare Diseases conference due to meet on the 14 & 15 October 2013, in London. Recent years have been a time of huge growth in orphan drug development, but also a time of increased pressure as many leading blockbuster drugs soon come off patent. SMi's orphan drugs conference will allow a specific focus on the current rare diseases drug development landscape, and where recent research will be touching upon going into the future.


Join the world leading expert speaking faculty and gain a wide ranging, highly inclusive view on the orphan drug industry as it currently stands. Speakers will highlight cutting edge research via case studies taking place in previously untreatable patients. Current regulatory policies involving the FDA & EMA, new drug discoveries and partnerships in clinical trials and drug development with patient groups.

A 'big pharma spotlight session' will be held on the morning of the 14th highlighting where current pipelines stand and where exactly the next succesful drug will be treating rare diseases. We will also be discussing HTA and pricing issues that are currently being implemented after recent reforms in Europe.
 

  • Use a unique 'Big Pharma Spotlight' sessions to understand current pipelines
  • Discover how research & development and market access costs must be controlled
  • Review optimal targeting of your new drug with patient groups to engage rare diseases
  • Learn from case studies of new molecules in clinical trials
  • Analyse current funding options; including financial and economic reimbursement for the clinical development of drugs
  • Intellectual Property
  • Legislation and Policy Advice
  • Business Development
  • Licensing
  • Manufacturing
  • APIs
  • Bioequivalence
  • Drug and Safety Assessment
  • Market Strategy
  • Regulatory Affairs
     

 

Ablynx; Armengaud-Guerlain; Arnold and Porter; AstraZeneca; Atheln Biomed; BRG; Bristows; Brockmeyer Biopharma GmbH; Business Insights; Crown CRO; Edenbiodesign Ltd; Eli Lilly; Flamel Technologies; Fresenius Kabi Deutschland GmbH; Frontline Pharma Consulting Ltd; Genentech; GenericsWeb; GlaxoSmithKline; Glycotope GmbH; Harvest Moon Pharmaceuticals; I B R Inc Institute For Biopharmaceutical Research; IDDI; IMS Health; Interpharm Consultancy; JustPharmaReports; Medical Industry Week; Medimmune; Menarini; Merck; Pinsent Masons; PolyTherics Ltd.; Roche; Ropes & Gray; Sanofi-Aventis; Takeda Pharmaceuticals; Technology Networks; TRISKEL Integrated Services S.A; UCB;

 

Conference programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Tony Hall

Tony Hall, Co-Founder, Findacure Foundation

9:10 Rare disease research at Pfizer - A spotlight

Gregory LaRosa

Gregory LaRosa, Senior Director, Rare Disease Research Unit, Pfizer

  • Overview of research strategy
  • Pipeline highlights
  • Case studies of Pfizers current work
  • 9:50 De-risking rare diseases drugs development and commercialisation

    Adrien Lemoine

    Adrien Lemoine, Director, Strategy & Operations, GlaxoSmithKline Rare Diseases

  • Misconceptions about the rare disease industry
  • Innovative business models to minimize development and commercial risks
  • Franchises
  • Value-based pricing
  • Partnerships   
  • Case study: venture capital fund dedicated to rare diseases
  • 10:30 Morning Coffee

    11:00 Orphan drugs and healthcare systems sustainability: Myths, challenges & opportunities

    Fernando Royo

    Fernando Royo, VP, Intl. Government Affairs, Genzyme

  • Current and foreseeable expenditure in orphan drugs
  • Compounding factors
  • The different perspectives on the value of orphan drugs
  • Initiatives for sustainable yet equitable access
  • 11:40 HTA Assessment in Orphan Drugs

    Panos Kanavos

    Panos Kanavos, Reader in International Health Policy, London School of Economics

  • HTA Reviews in Orphan Drugs
  • Value based pricing in a regulatory setting
  • Platforms for collaboration and harmonization
  • Case study
  • 12:20 Networking Lunch

    13:20 Improving service delivery for rare diseases and delivering new therapies: what is the role of the EUCERD?

    Kate Bushby

    Kate Bushby, Professor of Neuromuscular Genetics, University Of Newcastle

  • The process of national plans for RD
  • Centres of expertise and European Reference Networks
  • How these structures may support therapy development and delivery
  • Case study
  • 14:00 Early access for orphan drugs: An essential part of market access strategy

    Dominic Atkins

    Dominic Atkins, Global Business Development Director Clinigen Global Access Programs (GAP), Clinigen GAP

    Patients suffering from orphan diseases are very aware of products in development and demand early access. This presentation will give examples of successful global access programs, as well as addressing the challenges, pitfalls and opportunities of early access.

    14:40 Afternoon Tea

    15:10 Where European Payers Push Back on Prices and Funding for Orphan Drugs

    Donald Macarther

    Donald Macarther, Global Pharmaceutical Business Analyst, Justpharmareports

  • HTA and payer bodies views on orphan drugs
  • Why reimbursement is sometimes refused
  • How market entry agreements and patient registries can help
  • Case studies from across Europe
  • 15:50 Orphan Drugs in Germany

    Bertram Häussler

    Bertram Häussler, Chairman of the Board of Management, IGES Institut

  • Regulatory and Political Environment
  • Market and Budget Impact
  • Future Developments
  • 16:30 Chairman’s Closing Remarks and Close of Day One

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Tony Hall

    Tony Hall, Co-Founder, Findacure Foundation

    9:10 How regulatory strategy can help to optimize Orphan drugs development?

    Camille Métais

    Camille Métais, Sr Regulatory Affairs Manager, Alexion Pharmaceuticals

  • What are the available procedures to optimize drug development and in particular orphan drugs development?
  • What are the regulatory hurdles to develop and register an orphan drug?
  • What is coming next? Will this address the current hurdles
  • 9:50 Regulatory challenges of ex-vivo gene therapy medicinal products for rare diseases

    Estelle de Barbeyrac

    Estelle de Barbeyrac , Regulatory Affairs, Genethon

  • Pharmaceutical requirements for genetically-modified cell-based medicinal products and constraints associated with very rare patients’ populations
  • Flexibility versus standardization of the manufacturing process for small scale productions
  • Possible impact on the data set available for registration
  • 10:30 Morning Coffee

    11:00 Global developments in orphan drug and rare disease legislation

    David King

    David King, Director, Regulatory Policy and Intelligence, Novo Nordisk

  • Recent trends in orphan drug legislation
  • Future perspectives over the next few years
  • Case study
  • 11:40 Lynovex®, a novel multi-acting therapeutic orphan drug candidate for the treatment of cystic fibrosis

    Deborah A. O'Neil

    Deborah A. O'Neil, Chief Executive & Scientific Officer,, NovaBiotics Ltd

  • Lynovex® as a first-in-class, wholly novel, differentiated approach to tackling cystic fibrosis
  • Mucoactive, antibacterial & antibiofilm properties
  • Repositioning an existing therapeutic agent for CF against a background of proven safe historical use in other conditions
  • Lynovex’s interactions with and potential clinical impact on the existing armoury of CF antibiotics
  • One tri-functional active compound, two (or more) potential routes of delivery
  • 12:20 Networking Lunch

    13:20 Findacure Foundation

    Tony Hall

    Tony Hall, Co-Founder, Findacure Foundation

    Fundamental diseases
    Lack of commercial viability for many rare diseases
    Can a non-commercial approach address this unmet need?

    14:00 Opportunities and challenges for biotech company to develop Orphan Drugs

    Anders Waas

    Anders Waas, Chief Executive Officer, Tikomed

  • Strategy and tactics on the way – building a pipeline
  • Effective drug development and research
  • Partnering focus and timing of deals
  • 14:40 Afternoon Tea

    15:10 A Patient Group's perspective on patient recruitment and retention

    Oliver Timmis

    Oliver Timmis, Communications Project Manager, AKU Society

  • Patient recruitment in Europe
  • How to maximise patient retention
  • Why patient groups can be an effective may to manage the patient input of clinical trials
  • 15:50 Regulatory Protection for Orphan Medicinal Products

    Genevieve Michaux

    Genevieve Michaux, Counsel, Hunton & Williams

  • Market exclusivity
  • Data exclusivity
  • Paediatric incentives
  • 16:30 Chairman’s Closing Remarks and Close of Day Two

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    FEATURED SPEAKERS

    Adrien Lemoine

    Adrien Lemoine

    Director, Strategy & Operations, GlaxoSmithKline Rare Diseases
    David King

    David King

    Director, Regulatory Policy and Intelligence, Novo Nordisk
    Estelle de Barbeyrac

    Estelle de Barbeyrac

    Regulatory Affairs, Genethon
    Panos Kanavos

    Panos Kanavos

    Reader in International Health Policy, London School of Economics
    Tony Hall

    Tony Hall

    Co-Founder, Findacure Foundation

    Adrien Lemoine

    Director, Strategy & Operations, GlaxoSmithKline Rare Diseases
    Adrien Lemoine

    Anders Waas

    Chief Executive Officer, Tikomed
    Anders Waas

    Bertram Häussler

    Chairman of the Board of Management, IGES Institut
    Bertram Häussler

    Camille Métais

    Sr Regulatory Affairs Manager, Alexion Pharmaceuticals
    Camille Métais

    David King

    Director, Regulatory Policy and Intelligence, Novo Nordisk
    David King

    Deborah A. O'Neil

    Chief Executive & Scientific Officer,, NovaBiotics Ltd
    Deborah A. O'Neil

    Dominic Atkins

    Global Business Development Director Clinigen Global Access Programs (GAP), Clinigen GAP
    Dominic Atkins

    Donald Macarther

    Global Pharmaceutical Business Analyst, Justpharmareports
    Donald Macarther

    Estelle de Barbeyrac

    Regulatory Affairs, Genethon
    Estelle de Barbeyrac

    Fernando Royo

    VP, Intl. Government Affairs, Genzyme
    Fernando Royo

    Genevieve Michaux

    Counsel, Hunton & Williams
    Genevieve Michaux

    Gregory LaRosa

    Senior Director, Rare Disease Research Unit, Pfizer
    Gregory LaRosa

    Karl Petrovsky

    Senior Manager - Industry Affairs, Baxter
    Karl Petrovsky

    Kate Bushby

    Professor of Neuromuscular Genetics, University Of Newcastle
    Kate Bushby

    Oliver Timmis

    Communications Project Manager, AKU Society
    Oliver Timmis

    Panos Kanavos

    Reader in International Health Policy, London School of Economics
    Panos Kanavos

    Tony Hall

    Co-Founder, Findacure Foundation
    Tony Hall

    Workshops

    Applications for development of Orphan Drugs
    Workshop

    Applications for development of Orphan Drugs

    Marriott Regents Park
    16th October 2013
    London, United Kingdom

    Marriott Regents Park

    128 King Henry's Road
    London NW3 3ST
    United Kingdom

    Marriott Regents Park

    This 4 star north London hotel in zone 2 is the perfect destination for the astute business traveler as well as the leisure guest that knows how convenient north London hotels are, as a base from which to explore the city .Bond Street is just 3 stops from Swiss Cottage underground station on the Jubilee Line, so you can be shopping, exploring the sights and taking in one of London’s world-renowned West End shows in less than 15 minutes when you stay at this hotel near central London. At the same time, the hive of activity that is Camden Town, the chic shops, cafes and restaurants of Primrose Hill and ZSL’s London Zoo in Regents Park are all just a short walk from this hotel in north London.

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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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