Stewart Katie D, Johnston Joseph A, Matza Louis S, Curtis Sarah E, Havel Henry A, Sweetana Stephanie A, Gelhorn Heather L
Outcomes Research, Evidera, Bethesda, MD, USA.
Global Patient Outcomes & Real World Evidence.
Patient Prefer Adherence. 2016 Jul 27;10:1385-99. doi: 10.2147/PPA.S101821. eCollection 2016.
Pharmaceutical formulation and treatment process attributes, such as dose frequency and route of administration, can have an impact on quality of life, treatment adherence, and disease outcomes. The aim of this literature review was to examine studies on preferences for pharmaceutical treatment process attributes, focusing on research in diabetes, oncology, osteoporosis, and autoimmune disorders.
The literature search focused on identifying studies reporting preferences for attributes of the pharmaceutical treatment process. Studies were required to use formal quantitative preference assessment methods, such as utility valuation, conjoint analysis, or contingent valuation. Searches were conducted using Medline, EMBASE, Cochrane Library, Health Economic Evaluation Database, and National Health Service Economic Evaluation Database (January 1993-October 2013).
A total of 42 studies met inclusion criteria: 19 diabetes, nine oncology, five osteoporosis, and nine autoimmune. Across these conditions, treatments associated with shorter treatment duration, less frequent administration, greater flexibility, and less invasive routes of administration were preferred over more burdensome or complex treatments. While efficacy and safety often had greater relative importance than treatment process, treatment process also had a quantifiable impact on preference. In some instances, particularly in diabetes and autoimmune disorders, treatment process attributes had greater relative importance than some or all efficacy and safety attributes. Some studies suggested that relative importance of treatment process depends on disease (eg, acute vs chronic) and patient (eg, injection experience) characteristics.
Despite heterogeneity in study methods and design, some general patterns of preference clearly emerged. Overall, the results of this review suggest that treatment process has a quantifiable impact on preference and willingness to pay for treatment, even in many situations where safety and efficacy were the primary concerns. Patient preferences for treatment process attributes can inform drug development decisions to better meet the needs of patients and deliver improved outcomes.
药物制剂和治疗过程属性，如给药频率和给药途径，可能会对生活质量、治疗依从性和疾病结局产生影响。本综述的目的是研究关于药物治疗过程属性偏好的研究，重点关注糖尿病、肿瘤学、骨质疏松症和自身免疫性疾病方面的研究。
文献检索重点在于识别报告药物治疗过程属性偏好的研究。研究需使用正式的定量偏好评估方法，如效用评估、联合分析或条件估值。检索使用了Medline、EMBASE、Cochrane图书馆、卫生经济评估数据库和国家卫生服务经济评估数据库（1993年1月至2013年10月）。
共有42项研究符合纳入标准：19项关于糖尿病，9项关于肿瘤学，5项关于骨质疏松症，9项关于自身免疫性疾病。在这些疾病中，与治疗持续时间较短、给药频率较低、灵活性较高和侵入性较小的给药途径相关的治疗，比更繁重或复杂的治疗更受青睐。虽然疗效和安全性通常比治疗过程具有更大的相对重要性，但治疗过程对偏好也有可量化的影响。在某些情况下，特别是在糖尿病和自身免疫性疾病中，治疗过程属性比某些或所有疗效和安全性属性具有更大的相对重要性。一些研究表明，治疗过程的相对重要性取决于疾病（如急性与慢性）和患者（如注射经验）特征。
尽管研究方法和设计存在异质性，但一些偏好的总体模式明显显现。总体而言，本综述结果表明，即使在安全和疗效是主要关注点的许多情况下，治疗过程对偏好和治疗支付意愿也有可量化的影响。患者对治疗过程属性的偏好可为药物开发决策提供信息，以更好地满足患者需求并提供改善的结局。
