希少疾病用医薬品 (オーファンドラッグ) の経済学：インセンティブおよび市場力学

当レポートは、希少疾病用医薬品 (オーファンドラッグ) の開発および商業化において進化する状況を包括的に分析するものです。米国や欧州などの主要市場におけるインセンティブの変化、価格圧力、アクセスの課題がもたらす影響を検討し、これらの要因が投資判断や希少疾病用医薬品の長期的な持続可能性にどのように影響するかを探ります。また、エビデンス創出への需要の高まり、動的契約 (ダイナミックコントラクト) や適応症別価格設定 (インディケーションベースドプライシング) の役割、患者コミュニティとの早期かつ継続的な関与の重要性にも焦点を当てています。

本レポートを通じて、社内意思決定の最適化、利用可能なインセンティブや資金機会の活用、変化する政策や償還制度に適応することで、より厳しい市場環境下でも希少疾病用医薬品の資産価値を最大化するための洞察を得ることができます。

主な質問

• 1.過去および将来の希少疾病用医薬品開発において、インセンティブはどれほど重要か？
• 2.希少疾病用医薬品への投資判断に最も重要な市場はどこか？
• 3.今後の希少疾病用医薬品投資に影響を与える政策やインセンティブの変更とは？
• 4.希少疾病用医薬品の価格設定、アクセス、償還における主な課題とは？
• 5.新しい希少疾病用医薬品の商業化戦略を最適化するにはどうすべきか？
• 6.患者コミュニティは、開発および市場アクセスにおいてどのような役割を果たすか？
• 7.商業化を支えるエビデンス創出はどのように計画すべきか？
• 8.保険者の対応姿勢の変化や市場環境の厳格化によるリスクをどのように軽減できるか？

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.