罕見疾病用醫藥品 (孤兒藥) 的經濟學:獎勵及市場動態

本報告全面分析了孤兒藥開發和商業化不斷發展的模式。報告檢視了美國和歐洲等主要市場不斷變化的激勵措施、定價壓力和可及性挑戰的影響，並探討了這些因素如何影響投資決策和孤兒藥開發的長期可持續性。報告還強調了對證據生成日益增長的需求、動態合約和基於適應症的定價的作用，以及與患者群體進行早期和持續互動的重要性。

本報告提供了一些見解，幫助您優化內部決策，利用現有的激勵措施和融資機會，並適應不斷變化的政策和報銷制度，從而即使在更具挑戰性的市場條件下也能最大限度地提高孤兒藥資產的價值。

關鍵問題

• 1.激勵措施在過去和未來的孤兒藥開發中發揮了多大作用？
• 2.哪些市場對孤兒藥投資決策最為重要？
• 3.哪些政策和誘因的改變會影響未來孤兒藥投資？
• 4.孤兒藥在定價、可近性和報銷方面面臨的主要挑戰是什麼？
• 5.如何優化新型孤兒藥的商業化策略？
• 6.病患群體在研發和市場進入中扮演什麼角色？
• 7.如何規劃支持商業化的證據生成？
• 8.如何降低付款人態度轉變和市場條件趨於嚴格帶來的風險？

主要企業

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.