Oxford Outcomes Ltd, Oxford, UK.
Patient. 2011;4(4):247-57. doi: 10.2165/11592150-000000000-00000.
Attempts to estimate the cost effectiveness of attention-deficit hyperactivity disorder (ADHD) treatments in the past have relied on classifying ADHD patients as responders or non-responders to treatment. Responder status has been associated with a small gain in health-related quality of life (HR-QOL) [or utility, as measured using the generic QOL measure EQ-5D] of 0.06 (on a scale from 0 being dead to 1.0 being full health).
The goal of the present study was to develop and validate several ADHD-related health states, and to estimate utility values measured amongst the general public for those states and to re-estimate utility values associated with responder status.
Detailed qualitative interview data were collected from 20 young ADHD patients to characterize their HR-QOL. In addition, item-by-item clinical and HR-QOL data from a clinical trial were used to define and describe four health states (normal; borderline to mildly ill; moderately to markedly ill; and severely ill). ADHD experts assessed the content validity of the descriptions. The states were rated by 100 members of the UK general public using the time trade-off (TTO) interview and visual analog scale. Statistical mapping was also undertaken to estimate Clinical Global Impression-Improvement (CGI-I) utilities (i.e. response status) from Clinical Global Impression-Severity (CGI-S) defined states. The mapping work estimated changes in utilities from study baseline to last visit for patients with a CGI-I score of ≤ 2 or ≤ 3.
The validity of the four health states developed in this study was supported by in-depth interviews with ADHD experts and patients, and clinical trial data. TTO-derived utilities for the four health states ranged from 0.839 (CGI-S state 'normal') to 0.444 (CGI-S state 'severely ill'). From the mapping work, the change in utility for treatment responders was 0.19 for patients with a CGI-I score of ≤ 2 and 0.15 for patients with a CGI-I score of ≤ 3.
The present study provides utilities for different severity levels of ADHD estimated in a TTO study. This approach provides a more granular assessment of the impact of ADHD on HR-QOL than binary approaches employed in previous economic analyses. Change in utility for responders and non-responders at different levels of CGI-I was estimated, and thus these utilities may be used to compare health gains of different ADHD interventions.
过去，评估注意缺陷多动障碍（ADHD）治疗的成本效益的尝试依赖于将 ADHD 患者分类为对治疗有反应者或无反应者。反应者状态与健康相关生活质量（HR-QOL）[或使用通用 QOL 测量 EQ-5D 测量的效用]的小幅度提高相关，提高了 0.06（0 为死亡，1.0 为完全健康）。
本研究的目的是开发和验证几种与 ADHD 相关的健康状况，并估计普通人群对这些状况的效用值，并重新估计与反应者状态相关的效用值。
从 20 名年轻的 ADHD 患者中收集详细的定性访谈数据，以描述他们的 HR-QOL。此外，还使用临床试验的逐个临床和 HR-QOL 数据来定义和描述四种健康状况（正常；边缘至轻度患病；中度至明显患病；严重患病）。ADHD 专家评估了描述的内容有效性。100 名英国普通公众使用时间权衡（TTO）访谈和视觉模拟量表对这些状况进行了评分。还进行了统计映射，以根据临床总体印象改善（CGI-I）定义的状态估算临床总体印象严重程度（CGI-S）的效用（即反应状态）。映射工作估计了 CGI-I 评分为≤2 或≤3 的患者从研究基线到最后一次就诊时的效用变化。
本研究中开发的四种健康状况的有效性得到了 ADHD 专家和患者的深入访谈以及临床试验数据的支持。四种健康状况的 TTO 衍生效用范围从 0.839（CGI-S 状态“正常”）到 0.444（CGI-S 状态“严重患病”）。从映射工作中，CGI-I 评分≤2 的患者的治疗反应者的效用变化为 0.19，CGI-I 评分≤3 的患者的效用变化为 0.15。
本研究提供了 TTO 研究中不同 ADHD 严重程度的效用值。与以前的经济分析中采用的二元方法相比，这种方法提供了对 ADHD 对 HR-QOL 影响的更细致评估。估计了不同 CGI-I 水平的反应者和无反应者的效用变化，因此这些效用值可用于比较不同 ADHD 干预措施的健康获益。
