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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.
Gain Answers to Key Questions
- How can patients and patient advocacy groups play an enhanced role throughout a products 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?
- 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
- 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 products 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
- 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 companys 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.
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