The Economics of Orphan Drugs: Incentives and Market Dynamics

This report provides a comprehensive analysis of the evolving landscape for orphan drug development and commercialisation. It examines the impact of changing incentives, pricing pressures and access challenges in key markets such as the US and Europe and explores how these factors influence investment decisions and long-term sustainability for orphan drugs. The report addresses the increasing demands for evidence generation, the role of dynamic contracting and indication-based pricing and the importance of early and ongoing engagement with patient communities.

Gain insights into optimising internal decision making, leveraging available incentives and funding opportunities and adapting to shifting policy and reimbursement frameworks to maximise the potential of orphan drug assets in a more challenging market environment.

Key Questions Answered:

• 1. How important have orphan drug incentives been for past and future orphan drug development?
• 2. Which markets are most critical for informing orphan drug investment decisions?
• 3. What policy and incentive changes could impact future investment in orphan drugs?
• 4. What are the main challenges in pricing, access and reimbursement for orphan drugs?
• 5. How can companies optimise commercialisation strategies for new orphan drugs?
• 6. What role do patient communities play in orphan drug development and market access?
• 7. How should companies plan evidence generation to support orphan drug commercialisation?
• 8. What tactics can mitigate risks from changing payer willingness and tougher market conditions?

Key Companies:

Partial List of Participating Experts:

• Founder and Managing Partner, Dolon, based in the UK
• A Vice President, International Market Access and Pricing, at a biopharmaceutical company, based in Switzerland
• A Director, Global Market Access, at a top 20 pharmaceutical company by global sales in 2024, based in Switzerland

Methodology:

Dossier reports are developed from independent and concise analysis derived from original industry surveys and in-depth interviews with industry thought leaders. Reports analyse in detail significant developments and market trends that pharma executives need to understand if they are to manage the opportunities and challenges that lie ahead. The focus of each report is defined by a review of the secondary literature and any knowledge gaps identified. Based on this initial research, an evidence-based and expert-informed discussion guide is developed to ensure the research answers the most critical questions. Additionally, robust screening criteria are established to ensure that those interviewed are qualified in terms of their experience, knowledge and stature to speak on the topic.

What makes our reports different?

FirstWord Reports, a trusted industry leader with an exclusive focus on the pharmaceutical sector, delivers in-depth, actionable insights for biopharma professionals and decision-makers. Our deep industry knowledge enables us to provide relevant and valuable insights that help you identify emerging trends and navigate complex challenges effectively. Backed by extensive research and independent, impartial insights from leading experts and KOLs, our reports deliver the accuracy and reliability you need. With exclusive access to interviews and data unavailable elsewhere, along with ongoing market monitoring, we give you a comprehensive view of market dynamics. Covering over 40 dynamic disease areas and providing physician intelligence, including KOL insights and quantitative physician surveys, as well as industry expert views on issues in medical affairs, digital health, sales & marketing, market access, and other areas, our reports empower you to make smarter, data-driven decisions and maintain a competitive edge in a fast-moving industry.