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Orphan Drug Launch Strategies: Case Studies

Product Code:
s_596200854
Publication Date:
November 2017
Format:
PDF
Price:
£595

Achieving commercial success for orphan drugs requires specific strategies to be adopted, not least because market barriers are very different from those encountered when launching more common disease therapies. Lack of awareness, greater scrutiny of value, and the difficulty of gathering clinical evidence from a relatively small patient population are just some of the pertinent issues.

By comparing the experiences of 8 market forerunners, Orphan Drug Launch Strategies: Case Studies uncovers 13 key success factors for launching a new orphan drug, and gives practical insight into the commercial strategies adopted by established orphan drug manufacturers.

Discover on this page…

  • Key themes covered in the report
  • What you will learn from the report
  • Detailed table of contents
  • Research objectives and methodologies employed producing in the report
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Reasons to buy this report

Compared to mainstream drug launches, it's usually a much smaller team with fewer resources that are tasked with bringing a new orphan drug to market. And the market itself is smaller too – fewer specialists to talk to, fewer physicians to educate, and fewer patients to engage with. But smaller doesn't mean less important. Every activity is so much more visible, which makes the pressure to get it right first time even more intense than usual. And the higher price tag associated with rare disease treatments means that the commercial risks and the potential benefits are far from small either. Launching an orphan drug is clearly not for the faint-hearted.

This report will enable you to:

  • Compare the experiences of 8 pharma companies who have successfully launched at least one orphan drug, including Celgene, GlaxoSmithKline, Roche, Boehringer Ingelheim and Pfizer.
  • Understand which factors should be considered when setting orphan drug prices and find out about the specific pricing strategies adopted for each case study drug.
  • Assess the stakeholder engagement strategies needed to address the barriers to success that exist for rare disease therapies.
  • Gain practical insight into the internal elements that constitute an effective launch including launch team roles and responsibilities, leadership and KPIs.
  • Find out how to leverage partnerships and collaboration strategies to achieve maximum market impact.
  • Learn about the 13 individual success factors required to achieve an optimal orphan drug launch – taking account of pricing, stakeholder engagement and implementation.
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Table of Contents

Executive summary

Research objectives

Experts interviewed

Orphan drug launches – unique challenges requiring unique strategies

  • Key insights
  • Current landscape of orphan drug launches
  • The nature of orphan drug launches
  • The unique challenges of orphan drug launches
    • Lack of disease awareness
    • Finding patients
    • Demonstrating efficacy and cost-effectiveness
    • Geographical challenges
  • Elements of an orphan drug launch strategy
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies

Case studies: Orphan drug launches in action

  • Key insights
  • Case Study 1 – Roche's (previously InterMune) Esbriet (pirfenidone)
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 2 – GSK's Strimvelis
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 3 – Boehringer Ingelheim's Nintedanib (OFEV)
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 4 – Johnson & Johnson's Drug A
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 5 – Celgene's Revlimid (lenalidomide)
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 6 – Anonymous Company's Drug B
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 7– Pfizer's Drug C
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies
  • Case Study 8 – Vertex Pharmaceutical's Kalydeco (ivacaftor)
    • Pricing strategies
    • Stakeholder engagement strategies
    • Implementation strategies

Commonalities and Key Success Factors

Conclusion

Appendix

  • Expert Biographies
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Research Methodology and Objectives

This report offers practical insight into how 8 market players successfully launched treatments that target rare diseases. 8 separate case studies include details of strategies used for pricing, stakeholder engagement and implementation. 13 common success factors are also identified.

The insights presented in this report are based on in-depth interviews conducted from August to October 2017 with 8 experts from leading pharma companies responsible for orphan drug launches and medical affairs. Three interviewees chose to remain anonymous.

  • Markus Leyck Dieken, SVP, Global Head of CNS at Teva Pharmaceuticals
  • Allan Hillgrove, Corporate Board Division Human Pharma at Boehringer Ingelheim
  • Sven Kili, VP, Head of Cell & Gene Therapy Development, GSK
  • Ralph Torbay, Global oncology and marketing expert, leading several launches for a top 10 large pharmaceutical company
  • Michael Zaiac, Vice-President of Medical Affairs Haematology/Oncology EMEA, Celgene
  • Anonymous, Global Marketing Director, Johnson & Johnson
  • Anonymous, Vice-President, Medical Affairs, speciality pharmaceutical company
  • Anonymous, Rare Disease Medical Director at a large pharmaceutical company

Key questions explored in this report include:

  • What factors are considered when deciding the price of an orphan drug?
  • How is the on-going reimbursement landscape viewed?
  • What approaches are used for economic modelling, negotiation and price optimisation?
  • How is stakeholder engagement addressed during clinical development and over the drug lifecycle?
  • What strategies for acquiring physician input are employed?
  • What is the importance of advocacy groups and education and how is it provided?
  • Which internal roles should be involved in orphan drug launches and what specific capabilities are needed?
  • Where does leadership, collaboration, partnerships and/or outsourcing fit?
  • What monitoring systems should be put in place to evaluate launch success?
  • How is intelligence on competitor products acquired and utilised?
  • What contingency plans were developed, if any?
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