본 보고서는 희귀의약품 개발 및 상업화에 있어서 진화하는 상황을 종합적으로 분석하는 것입니다. 미국 및 유럽과 같은 주요 시장에서 인센티브 변화, 가격 압력, 접근 과제로 인한 영향을 검토하고 이러한 요인이 투자 판단과 희귀의약품의 장기적인 지속가능성에 어떻게 영향을 미치는지 알아봅니다. 또한 증거 창출에 대한 수요 증가, 동적 계약(동적 계약) 및 적응증별 가격 설정(표시 기반 가격) 역할, 환자 커뮤니티와의 조기 및 지속적인 참여의 중요성에 초점을 맞추었습니다.
본 보고서를 통해 사내 의사결정 최적화, 이용 가능한 인센티브 및 자금 기회 활용, 변화하는 정책 및 상환 제도에 적응함으로써 보다 어려운 시장 환경 하에서도 희귀의약품의 자산 가치를 극대화하기 위한 인사이트를 얻을 수 있습니다.
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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.
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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.
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.