Commercial Excellence in Rare Diseases and Orphan Drugs

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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 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

Content Highlights

• Orphan drugs market overview
  • The evolution of the orphan drugs market
  • The orphan drugs market is expected to undergo continued growth
• What is commercial excellence in orphan drugs?
  • Traditional measures of success are not appropriate for orphan drugs
  • One of the most important factors is the number of patients that have been helped
  • Access is seen as a critical success factor for orphan drugs
• Why is commercial excellence in orphan drugs important?
• What are the key hurdles for orphan drugs?
  • Identifying the appropriate unmet need
  • The execution of effective clinical trials
  • Improvement is still needed in regulatory processes
  • Market access is a particular issue for orphan drugs
  • Even after a successful launch, there are still challenges for orphan drugs
• Stakeholder engagement
  • Why is stakeholder engagement important?
  • The treatment of orphan diseases tends to be concentrated amongst a small number of experts
  • Case study: Birmingham Health Partners Rare Disease Centre initiative
  • Key stakeholder groups that should be engaged
    • Patients should be engaged throughout the lifecycle
      • Patient advocacy groups
      • Case study: PPMD submits patient advocacy initiated draft guidance for a rare disease
      • Patients are critical in providing insight into the disease
      • Staying close to patients can serve as a reminder of the importance of orphan drugs
      • Patient engagement must be conducted in a sensitive manner
      • Patient engagement needs to start as early as possible
      • Physicians are critical to orphan drug success
        • Rare disease KOLs communicate and work with one another
        • KOLs have a critical role to play in the development and pre-launch phase for orphan drugs
        • Post-launch, KOL engagement is vital for commercial success
        • KOL engagement should be as early as possible
  • Payers are equally as important as patients and physicians
    • Although market access is not currently a major challenge, orphan drugs still have to demonstrate value
    • Payers are frequently being engaged in order to ensure clinical trials are designed appropriately
    • When is the best time to engage payers?
  • The regulatory authorities are seen by many as an important stakeholder for orphan drugs
• Commercialisation of orphan drugs
  • Collaboration and acquisition is driving the landscape for orphan drugs
  • Big pharma is expected to dominate in orphan drugs
    • The structure of orphan drugs teams in big pharma
      • Specific rare diseases teams
      • Absorbing orphan drugs into therapy area teams
      • Maintaining acquired orphan drug specialists as separate entities
      • A separate rare diseases operation is seen as the most effective structure for big pharma
      • The benefits of being a smaller pharmaceutical company
      • Alliances and co-operation tends to be favoured
        • Reasons for collaboration in orphan drugs
        • Consolidation and co-development likely to continue in the orphan drugs space
• Best practice
  • Case study: Genzyme – an orphan drugs pioneer still leading the way
    • Factors for Genzyme’s success
  • Case study: Shire – putting rare disease at the centre
  • Shire’s acquisition activity has established its orphan drug presence
  • Shire will continue to be a leader in orphan diseases
• Case study: Roche activities in orphan disease
  • The Genentech acquisition
  • Genentech has a strong history with orphan drugs
  • Roche is building on its successful Genentech merger with other acquisitions in the orphan drugs area
• The future outlook
  • Market access is expected to become more challenging
  • Patient involvement in orphan drugs will continue to grow
    • Venture philanthropy is an area where patient advocates are increasingly involved
      • The Cystic Fibrosis Foundation courts controversy with Kalydeco
  • Smaller companies will continue to be of critical importance
    • Smaller biotech companies will have to continue to make alliances in order to succeed
  • Recommendations to companies in orphan drugs
    • Developing a “fit for purpose” orphan drug is key
    • An accurate assessment of unmet need is critical
    • Flexibility during commercialisation is needed
    • Early engagement with all stakeholders is fundamental to commercial excellence
    • Collaboration will help boost commercial excellence

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