欧洲药品管理局（EMA）通过加速程序和在授权要求方面的灵活性，促进了对创新药物的获取，适用于罕见病和肿瘤等存在医学需求的疾病。然而，由于有条件授权和在异常情况下获得授权的药物数量的增加，导致了对药物疗效和安全性的临床不确定性相对于使用标准授权时更高。这种不确定性对临床实践和定价与报销谈判产生了重要影响，特别是因为高价格是基于高价值的假设，并得到监管优先权的支持。临床开发的负担通常会转嫁到公共医疗保健体系上，导致支出预算和机会成本的增加。有效管理不确定性，通过适当的测试和评估，并在价格中公平反映成本和风险，至关重要。然而，重要的是不能牺牲基于证据的医疗保健的基本要素，只为了获得对新治疗方法的接触。在平衡对新治疗方法的敏感和理性访问、确保它们对医疗保健系统的安全、疗效和经济可行性方面需要深思熟虑的决策。最终，负责任的及时获取创新药物的方法需要平衡患者需求与医疗保健系统关切。该方法强调了基于证据的决策和公平定价与报销的重要性。版权所有©2023 Vallano, Pontes和Agustí。

The European Medicines Agency (EMA) fosters access to innovative medicines through accelerated procedures and flexibility in the authorization requirements for diseases with unmet medical needs, such as many rare diseases as well as oncological diseases. However, the resulting increase of medicines being marketed with conditional authorizations and in exceptional circumstances has lead to higher clinical uncertainty about their efficacy and safety than when the standard authorizations are applied. This uncertainty has significant implications for clinical practice and the negotiation of pricing and reimbursement, particularly as high prices are based on assumptions of high value, supported by regulatory prioritization. The burden of clinical development is often shifted towards public healthcare systems, resulting in increased spending budgets and opportunity costs. Effective management of uncertainty, through appropriate testing and evaluation, and fair reflection of costs and risks in prices, is crucial. However, it is important not to sacrifice essential elements of evidence-based healthcare for the sake of access to new treatments. Balancing sensitive and rational access to new treatments, ensuring their safety, efficacy, and affordability to healthcare systems requires thoughtful decision-making. Ultimately, a responsible approach to timely access to innovative medicines that balances the needs of patients with healthcare systems' concerns is necessary. This approach emphasizes the importance of evidence-based decision-making and fair pricing and reimbursement.Copyright © 2023 Vallano, Pontes and Agustí.