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Authors
Advisor(s)
Abstract(s)
O Acesso ao Mercado (MA) no contexto farmacêutico desempenha um papel crucial na disponibilização de Tecnologias de Saúde (HT) inovadoras aos pacientes, navegando por complexos cenários regulamentares, económicos e clínicos. Esta tese explora as estratégias de MA com foco na Avaliação de Tecnologias de Saúde (HTA), que é um processo fundamental para avaliar o benefício terapêutico, a segurança e a relação custo-eficácia das tecnologias, influenciando as decisões de preços e reembolsos, impactando assim o MA das HT. Através de uma análise comparativa das estratégias de MA em Portugal e na Alemanha, a pesquisa explora modelos de HTA nos dois países, utilizando o Forxiga (dapagliflozina) como estudo de caso para avaliar as diferenças nos resultados regulatórios e económicos. O estudo destaca o papel do benefício terapêutico adicional na definição do acesso aos tratamentos, revelando algumas disparidades entre os modelos de HTA na Alemanha e em Portugal. Estas diferenças estruturais afetam os prazos de comparticipação, a acessibilidade e as estratégias gerais de entrada no mercado.
Os dados do mundo real (RWD) e os resultados reportados pelos pacientes (PROs) surgem como ferramentas relevantes, complementando os ensaios clínicos na justificação de preços baseados no valor e acordos de entrada controlada. Os principais resultados sublinham a importância de alinhar considerações clínicas, económicas, sociais e até políticas para responder às diversas expectativas dos intervenientes nos sistemas de saúde. Além disso, a integração da Inteligência Artificial (AI) e de ferramentas digitais em evolução promete otimizar as estratégias de MA e melhorar a eficiência dos processos. Este estudo contribui para a compreensão do papel em evolução da HTA no MA, com implicações para melhorar a consistência nas práticas regulamentares europeias. Ao ligar a avaliação clínica às considerações económicas e às perspetivas dos pacientes, esta tese sublinha a necessidade de estratégias de MA dinâmicas e adaptáveis que satisfaçam as diversas necessidades de saúde na Europa.
Adicionalmente, este trabalho inclui um resumo alargado em português (Appendix I), oferecendo uma descrição mais detalhada dos objetivos, métodos e resultados para leitores de língua portuguesa.
Market Access (MA) in the pharmaceutical context plays a pivotal role in delivering innovative Health Technologies (HT) to patients by navigating complex regulatory, economic, and clinical landscapes. This thesis explores MA strategies with a focus on Health Technology Assessment (HTA), which is a critical process for evaluating therapeutic benefit, safety, and cost-effectiveness. HTA influences pricing and reimbursement decisions, thereby affecting the MA of HT. Through a comparative analysis of MA strategies in Portugal and Germany, the research explores their HTA frameworks using Forxiga (dapagliflozin) as a case study to assess differences in regulatory and economic outcomes. The study highlights the role of added therapeutic benefit in shaping access to treatments, revealing some disparities between the HTA model in Germany and Portugal. These structural differences affect the timelines for reimbursement, accessibility, and overall market entry strategies. Real-world data (RWD) and patient-reported outcomes (PROs) emerge as influential tools, complementing clinical trials in justifying value-based pricing and managed entry agreements. Key findings underline the importance of aligning clinical, economic, social, and even political considerations to meet diverse stakeholder expectations across healthcare systems. Additionally, the integration of Artificial Intelligence (AI) and evolving digital tools promises to optimize MA strategies and improve the efficiency of the processes. The study contributes to the understanding of HTA’s evolving role in MA, with implications for improving consistency in European regulatory practices. By bridging clinical evaluation with economic considerations and patient perspectives, this thesis underscores the need for dynamic, adaptive MA strategies that meet diverse healthcare needs across Europe.
Market Access (MA) in the pharmaceutical context plays a pivotal role in delivering innovative Health Technologies (HT) to patients by navigating complex regulatory, economic, and clinical landscapes. This thesis explores MA strategies with a focus on Health Technology Assessment (HTA), which is a critical process for evaluating therapeutic benefit, safety, and cost-effectiveness. HTA influences pricing and reimbursement decisions, thereby affecting the MA of HT. Through a comparative analysis of MA strategies in Portugal and Germany, the research explores their HTA frameworks using Forxiga (dapagliflozin) as a case study to assess differences in regulatory and economic outcomes. The study highlights the role of added therapeutic benefit in shaping access to treatments, revealing some disparities between the HTA model in Germany and Portugal. These structural differences affect the timelines for reimbursement, accessibility, and overall market entry strategies. Real-world data (RWD) and patient-reported outcomes (PROs) emerge as influential tools, complementing clinical trials in justifying value-based pricing and managed entry agreements. Key findings underline the importance of aligning clinical, economic, social, and even political considerations to meet diverse stakeholder expectations across healthcare systems. Additionally, the integration of Artificial Intelligence (AI) and evolving digital tools promises to optimize MA strategies and improve the efficiency of the processes. The study contributes to the understanding of HTA’s evolving role in MA, with implications for improving consistency in European regulatory practices. By bridging clinical evaluation with economic considerations and patient perspectives, this thesis underscores the need for dynamic, adaptive MA strategies that meet diverse healthcare needs across Europe.
Description
Tese de mestrado, Regulação e Avaliação do Medicamento e Produtos de Saúde , 2025, Universidade de Lisboa, Faculdade de Farmácia.
Keywords
Pharmaceutical market access Health technology assessment Pricing and reimbursement Real-world evidence Added benefit Teses de mestrado - 2025