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Commercial Excellence in Rare Diseases and Orphan Drugs


Developing therapies for orphan diseases is attracting much industry interest with the promise of profits. But what are the specific R&D, regulatory, stakeholder and market access issues you need to understand and practically address?

Commercial Excellence in Rare Diseases and Orphan Drugs is a highly-detailed report for industry management who must evaluate, plan, execute, manage and deliver profit from orphan drug programmes. Enriched with case studies, the report reveals the real-world experience and actionable insights of eight senior industry experts who possess a deep knowledge of the challenging and highly-demanding operating environment for orphan drug development and commercialisation.

Reasons to Purchase

Gain Answers to Key Questions

  • How can patients and patient advocacy groups play an enhanced role throughout a product’s development cycle from research funding to market access?
  • Why is early engagement with payers and KOLs critical in ensuring wide market access and uptake?
  • To what extent is the accurate assessment of unmet clinical need and establishing potential patient population critical and what are the benchmarks?
  • With approximately 7,000 identified orphan diseases, how do you select research targets for development?
  • Clinical trial design and recruitment are challenges for orphan drug developers: what needs to be taken into account and how have companies approached this?
  • Orphan drug regulation is relatively relaxed, but what more could regulators do and how can you influence them?
  • Small/Big pharma collaborations: when is the optimum time for engagement and what can each learn from each other?
Top Benefits
  • Understand how patient support is a must-have for industry and identify the areas where they are most influential
  • Formulate communication strategies to ensure wide clinical buy-in and support
  • Address effectively the anxieties of payers who must justify orphan drug spend in their budgets
  • Examine critical orphan drug candidate selection and overcome challenges of designing and conducting clinical trials
  • Understand what is considered to be commercial excellence for orphan drugs and what are the hurdles for success
  • Gain insight into current company structures for orphan drugs and the way in which rare disease teams should be structured in order to maximise commercial success
  • Understand the regulatory structures that determine orphan drug research/approval and learn where and how regulators can be influenced
Key Takeaways
  • Orphan drugs offer a positive opportunity for Pharma, but companies must be realistic about their potential and the challenges – old models of assessment used for drugs targeting large patient populations are not fit for purpose
  • Engagement with key stakeholders at an early stage is critical to ultimate commercial success
  • Patient Groups, more than any other area, are critical to success and hugely influential in realising a product’s commercial potential
  • Orphan drugs are often very expensive: make your case to payers, and make it early to ensure buy in
  • Identifying appropriate research candidates for late phase investment requires a clear understanding of unmet needs and patient population – and how you will access them
  • Regulators may look positively on orphan drug applications but there are still challenges to overcome
  • The internal organisation of orphan drug teams varies and the assessment of different models is critical
Report Features – Not Available Elsewhere
  • Knowledgeable and detailed insights and opinions of eight industry experts working daily in developing and commercialising orphan disease drugs in the US and Europe
  • Extensive case studies demonstrating different approaches and experiences for ensuring commercial effectiveness
  • Critical insights for corporate, research, planning, marketing and communications management
Experts Interviewed
  • Business Unit Head, Rare diseases, Top 10 pharmaceutical company
  • Senior Director, Top 10 pharmaceutical company
  • Marketing Director, Mid-size European pharmaceutical company
  • Sales Director, European pharmaceutical company
  • Senior Product Manager, Top 10 pharmaceutical company
  • Director, Rare disease pharmaceutical company
  • Marketing Director, Top 10 pharmaceutical company
  • Marketing Director, Top 10 pharmaceutical company
About FirstWord Reports

FirstWord is an innovative industry intelligence leader serving over 240,000 Pharma and MedTech professionals worldwide. FirstWord offers a range of products and services designed to help your company gain a competitive edge by making key business decisions with speed and confidence
  • FirstWord Pharma PLUS is a personalised and comprehensive intelligence service delivering up-to-the-minute pharma news, insight, analysis and expert views of importance to your company’s success.
  • FirstWord Reports deliver timely, need-to-know intelligence about your products, your competitors and your markets. Covering biosimilars, market access, medical affairs, sales & marketing, technology and therapy areas, FirstWord Reports provide expert views and intelligence on the challenges facing pharma today.

1.Executive summary
2.Research objectives and methodology
4.Orphan drugs overview
4.1.The evolution of the orphan drugs market
4.2.The orphan drugs market is expected to undergo continu

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