Zorginstituut Nederland, Diemen, the Netherlands.
Department of Pharmacoepidemiology and Clinical Pharmacology, University Utrecht, Utrecht, the Netherlands.
Pharmacol Res Perspect. 2021 Apr;9(2):e00742. doi: 10.1002/prp2.742.
Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value-based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patient-relevant, by comparing these to ICHOM standard sets. We conducted a cross-sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease-specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision-making.
国际卫生保健成果测量联合会（ICHOM）制定的标准结局集有助于医疗保健实践中的基于价值的医疗保健，并得到了监管机构和卫生技术评估（HTA）机构的关注，这些机构定期评估新药的价值。我们旨在通过将这些结局与 ICHOM 标准集进行比较，评估监管机构和 HTA 机构使用的结局在多大程度上与患者相关。我们对 ICHOM 标准集和公开的监管和 HTA 评估指南进行了横断面比较分析。由于许多新药正在开发中，这些治疗方法对患者的相关性存在很大的不确定性，因此我们专注于肿瘤学。监管和 HTA 评估指南与 ICHOM 标准集的比较表明，ICHOM 和监管机构都强调了疾病特异性结局的重要性。另一方面，HTA 机构更关注通用结局，以便能够在不同疾病领域进行比较。总体而言，相似的结局与市场准入、报销和 ICHOM 标准集有关。然而，一些差异是明显的，例如中间结局的可接受性。ICHOM 标准集建议使用中间结局，但监管机构比 HTA 机构更有可能接受中间结局。在接受的结局方面有更高程度的一致性可能会提高监管和 HTA 流程的效率，并增加新药物的及时获得。ICHOM 标准集可能有助于协调这些结局。然而，由于监管和 HTA 决策的不同目的，使用的结局可能仍然存在一些差异。
