罕见疾病用医药品 (孤儿药) 的经济学:奖励及市场动态

本报告全面分析了孤儿药开发和商业化不断发展的模式。报告检视了美国和欧洲等主要市场不断变化的激励措施、定价压力和可及性挑战的影响，并探讨了这些因素如何影响投资决策和孤儿药开发的长期可持续性。报告还强调了对证据生成日益增长的需求、动态合约和基于适应症的定价的作用，以及与患者群体进行早期和持续互动的重要性。

本报告提供了一些见解，帮助您优化内部决策，利用现有的激励措施和融资机会，并适应不断变化的政策和报销制度，从而即使在更具挑战性的市场条件下也能最大限度地提高孤儿药资产的价值。

关键问题

• 1.激励措施在过去和未来的孤儿药开发中发挥了多大作用？
• 2.哪些市场对孤儿药投资决策最为重要？
• 3.哪些政策和诱因的改变会影响未来孤儿药投资？
• 4.孤儿药在定价、可近性和报销方面面临的主要挑战是什么？
• 5.如何优化新型孤儿药的商业化策略？
• 6.病患群体在研发和市场进入中扮演什么角色？
• 7.如何规划支持商业化的证据生成？
• 8.如何降低付款人态度转变和市场条件趋于严格带来的风险？

主要企业

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.