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Effective Sales and Marketing Strategies for Orphan Drugs
[Published by FirstWord Pharma]

Published by FirstWord Pharma: 01 Jun 2012 | 18 | In Stock
Related Topics: GlaxoSmithKline , Orphan Drugs



The real potential for orphan drug pharma marketing

With drug pipelines moving from essential products to niche therapies, large pharma companies are increasingly attracted to the prospective high demand and low competition of orphan drug markets. However, the ‘sales and marketing’ models for orphan drugs are distinctly different to conventional models for common medications. So the transition to this specialist field is a lot more challenging than it appears.

A new report from FirstWord Dossier – Effective Sales and Marketing Strategies for Orphan Drugs – examines the effectiveness of strategic business models that big pharma are using to market these products. It draws upon the in-depth opinions of 13 experts from the orphan drugs industry, patient organisations, and regulatory authorities in rare diseases.


Report Overview

Effective Sales and Marketing Strategies for Orphan Drugs summarises the main issues, challenges and tactics involved in launching new pharma products for rare diseases. The report compares the critical differences between marketing orphan vs non-orphan drugs – from initial drug development through to commercialisation. It also discusses the huge potential of this field and the key factors driving new product awareness and approval.


Key Report Features

  • Overview of pharma’s current role in the rare diseases market

  • Orphan drug business models: specialist biotech vs big pharma

  • Distinct differences in orphan drug marketing vs common drugs

  • Clinical and medical challenges in developing orphan drugs

  • Practical ways to gain valuable patient organisation support

  • Rare disease indication strategies to maximise commercial return

  • Benefits of Managed Access Programmes (MAPS) for drug developers

  • Case studies of various pharma companies’ orphan drug launches

  • Puchase Reasons

    Key Benefits

  • Review the evolving rare diseases marketplace

  • Identify the potential of different business models

  • Manage the challenges specific to orphan drug marketing

  • Recognise the key factors that influence commercial success

  • Assess the pharmacoeconomic benefits of orphan drugs

  • Build key stakeholder alliances across the entire drug lifecycle

  • Use social media and websites as marketing tools

  • Key Questions Answered

  • How do orphan drug business models differ from standard models?

  • What are the US and European incentives for orphan drug development?

  • What strategies did GSK use to enter the rare diseases market?

  • Which issues should you consider before orphan drug investment?

  • How can you initiate and encourage corporate-patient collaborations?

  • Can an orphan drug ever become a blockbuster?

  • What can MAPs offer orphan drug developers?

  • How can you effectively target physicians about rare diseases?

  • Who Would Benefit From This Report?

    This report will be of value to pharma directors and managers with responsibilities in the following areas:

  • Market access

  • Marketing management

  • Business development

  • Brand marketing

  • Sales management

  • Pharmacoeconomics

  • Clinical research development

  • Corporate communications

  • Key Opinion Leader Liaison teams

  • Patient Advocacy Liaison teams

  • Medical affairs

  • This report will also benefit patient group organisations and charities, as well as healthcare legislators and health policy researchers.

    Expert Views Include:

  • Robert Derham, Founder of CheckOrphan

  • Abdul Mullick, Head of Global Marketing, Genetic Diseases, Genzyme

  • Craig Kephart, President, CEO of Centric Health Resources

  • Marc Dunoyer, Head of Rare Diseases Unit, GlaxoSmithKline

  • Steve Aselage, Executive VP and Chief Business Officer, BioMarin

  • Dirk Moritz, VP Global Strategic Marketing, Shire Human Genetic Therapies

  • Stephen C. Groft, Director, Office of Rare Diseases, National Institutes of Health

  • Hans Schikan, Chief Executive Officer of Prosensa

  • Nick Sireau, Chairman of the AKU Society

  • Simon Estcourt, Senior VP Strategy and Corporate, Idis

  • Maryze Schoneveld van der Linde, founder of Patient Centered Solution

  • Theresa Heggie, Senior VP of Global Commercial Operations, Shire Human Genetic Therapies

  • Tony Hall, Chief Medical Officer at PSR

  • Key Quotes

  • “If you’re not doing the pre-marketing activity for orphan drug launches you’re going to fail. It will cost you a lot more and you’re not going to have a real defined patient population,” – Robert Derham, founder of CheckOrphan.

  • “There’s an advantage to being first in your orphan drug market if you do it right. Other products may seem to work as well but the difference is in your programme,” – Craig Kephart, CEO of Centric Health Resources.

  • “Some big pharma are trying to make a business out of orphans but this isn’t something that comes naturally to them…they have to change their mind set to go into a market like this,” – Steve Aselage, executive VP, BioMarin.

  • Orphan drugs appear superficially as a very lucrative space: rare diseases, few competitors …but this takes time and persistence,” – Abdul Mullick, head of global marketing, genetic diseases, Genzyme.

  • “More than any other area, the advocacy that patient organisations bring to this area is really important, and these relationships start very early in drug development,” – Theresa Heggie, senior VP, global commercial operations, Shire Human Genetic Therapies
  • Table of Contents
    for Effective Sales and Marketing Strategies for Orphan Drugs [Published by FirstWord Pharma]

    • 1.Executive summary



      3.1. Legislation and incentives

      3.2 The rare diseases market

      3.3. Orphan drug indications

      3.4. Patient focus

      4.How do pre-marketing development strategies contribute towards later commercial success?

      5.Is the potential of the orphan drug market enough to attract investment?

      6.Regulations and incentives

      6.1. Market exclusivity in the EU

      6.2. Protocol assistance

      6.3. Fee reductions

      6.4. EU-funded research

      6.5. Orphan drug centralized procedure at European level

      7.Evolution of the orphan drug market

      7.1. Development of drugs for rare diseases compared to drugs for more common diseases

      7.2. Optimizing the orphan drug networks to maximize commercial return

      7.3. Orphan drug business models: specialist biotech versus big pharma

      8.What are the major challenges to the orphan drug development process and business model?

      8.1. Medical and clinical challenges to orphan drug development

      8.2. Diagnosis

      8.3. Disease understanding

      8.4. Case study 1: Fabry Outcome Survey

      8.5. Clinical trial challenges

      8.6 Case study 2:

      9.Endpoints stall AKU clinical trial

      10.Aspects of the orphan drug business model that may benefit from change

      10.1. Harmonization of clinical trial processes between the EU and US

      10.2 Pricing and reimbursement

      10.3. Openness between pharma and patients

      11.‘Sales and marketing’ strategy: will an orphan drug ever become a blockbuster?

      11.1. Optimizing the company-physician relationship in the orphan drug space

      11.2. Case study 3: Prolastin - An integrated healthcare management model of sales and marketing

      11.3. Disease and product familiarization amongst physicians and patients

      11.4. Diagnosis

      11.5. ‘Sales and marketing’ infrastructures

      11.6. Targeting physicians in rare diseases

      11.7. Competition in the orphan drug market

      11.8. Social media in communications and the marketing mix

      12.Rare disease indication strategies

      12.1. New molecular entity (NME) for unmet medical need

      12.2. Drug for a rare disease applied to a common disease

      12.3. Re-purposing

      13.How can companies collaborate with patient organizations across the orphan drug lifecycle to the mutual benefit of both?

      13.1. Motivated patients and patient organizations

      13,2. The interface between patient organizations and industry

      13.3. Patient organizations

      13.4. EURORDIS and NORD

      13.5. Mutual benefits

      13.6. Patient Registries

      13.7. Natural history registry

      13.8.Product registry

      13.9. The Global Rare Disease Registry and Data Repository (GRDR)

      13.10. European Centres of Expertise and EUROPLAN

      13.11. How patients can drive research and drug development in rare diseases

      14.What can Managed Access Programmes offer companies developing orphan drugs?

      14.1. When to use a MAP

      14.2. Benefits of a Managed Access Programme

      14.3 Access to medicines

      14.4. Data

      14.5. Stakeholder engagement

      14.6, Market development

      14.7. Market testing pre-approval (between post-Phase III and first submission) 14.8. Ensuring continuity of patient supply

      14.9. Access in certain countries post-launch

      15.Regulations and national plans for Managed Access Programmes

    Additional Details


    FirstWord Pharma

    Publisher Information


    18 |

    Number of Pages


    Report Format


    FirstWord Pharma Reports

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