Tappenden Paul, Sadler Susannah, Wildman Martin
ScHARR, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, England, UK.
Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, England, UK.
Pharmacoeconomics. 2017 Jun;35(6):647-659. doi: 10.1007/s40273-017-0500-x.
Cystic fibrosis (CF) negatively impacts upon health-related quality of life and survival. Adherence to nebulised treatments is low; improving adherence is hypothesised to reduce rates of exacerbation requiring intravenous antibiotics and lung function decline.
A state transition model was developed to assess the cost effectiveness of an intervention aimed at increasing patient adherence to nebulised and inhaled antibiotics compared with current CF care, in advance of the forthcoming CFHealthHub randomised controlled trial (RCT).
The model estimated the costs and health outcomes for each option from the perspective of the UK National Health Service and Personal Social Services over a lifetime horizon. Health gains were valued in terms of quality-adjusted life-years (QALYs) gained. Forced expiratory volume in 1 second (FEV) trajectories were predicted over three lung function strata: (1) FEV ≥70%, (2) FEV 40-69% and (3) FEV <40%. Additional states were included to represent 'post-lung transplantation' and 'dead'. The model was populated using CF Registry data, literature and expert opinion. Costs were presented at 2016 values. Uncertainty was assessed using deterministic and probabilistic sensitivity analyses.
If effective, the adherence intervention is expected to produce an additional 0.19 QALYs and cost savings of £64,078 per patient. Across all analyses, the intervention dominated current care. Over a 5-year period, the intervention is expected to generate cost savings of £49.5 million for the estimated 2979 patients with CF with Pseudomonas aeruginosa currently aged ≥16 years in the UK. If applied to a broader population of adult patients with CF receiving any nebulised therapy, the expected savings could be considerably greater.
If effective, the adherence intervention is expected to produce additional health gains at a lower cost than current CF care. However, the economic analysis should be revisited upon completion of the full RCT. More generally, the analysis suggests that considerable gains could be accrued through the implementation of adherence interventions that shift care from expensive hospital-based rescue to community-based prevention.
囊性纤维化（CF）对健康相关生活质量和生存率产生负面影响。雾化治疗的依从性较低；据推测，提高依从性可降低需要静脉使用抗生素的急性加重发生率和肺功能下降。
在即将开展的CFHealthHub随机对照试验（RCT）之前，开发一种状态转换模型，以评估与当前CF治疗相比，旨在提高患者对雾化和吸入抗生素依从性的干预措施的成本效益。
该模型从英国国家医疗服务体系和个人社会服务的角度，在终身范围内估计了每种方案的成本和健康结果。健康收益以获得的质量调整生命年（QALY）来衡量。在三个肺功能分层中预测1秒用力呼气量（FEV）轨迹：（1）FEV≥70%，（2）FEV 40-69%，（3）FEV<40%。纳入了额外的状态来代表“肺移植后”和“死亡”。该模型使用CF注册数据、文献和专家意见进行填充。成本以2016年的价值呈现。使用确定性和概率敏感性分析评估不确定性。
如果有效，依从性干预预计每名患者可额外产生0.19个QALY，并节省64,078英镑的成本。在所有分析中，该干预措施优于当前治疗。在5年期间，对于英国目前年龄≥16岁的估计2979例患有铜绿假单胞菌的CF患者，该干预措施预计可节省4950万英镑的成本。如果应用于接受任何雾化治疗的更广泛成年CF患者群体，预期节省的成本可能会显著更高。
如果有效，依从性干预预计能以低于当前CF治疗的成本产生额外的健康收益。然而，在完整的RCT完成后应重新进行经济分析。更普遍地说，该分析表明，通过实施依从性干预措施，将治疗从昂贵的基于医院的抢救转向基于社区的预防，可获得可观的收益。
