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Health technology assessment (HTA) agencies assess the value of innovative therapies and publish recommendations for practice. However, is publishing HTA products sufficient to generate value in the real world? The objectives of our work were to: (i) determine whether innovative therapies for lung cancer produce the expected results in the real-world setting; and (ii) assess whether recommendations are followed in real-world practice.
Methods
Clinical administrative data were used in this two-phase project. In the first phase, a descriptive portrait of the use of epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) for treating lung cancer was produced. Their value was assessed by comparing overall survival of treated patients observed in the province of Québec to the published literature. The second phase focused on the initial evaluation of patients diagnosed with lung cancer and treated first by surgery. The delay between first evidence of cancer and surgery was assessed, and the utilization of 27 healthcare services was analyzed and assessed according to our recommendations (algorithms) for lung cancer management.
Results
From the date the first EGFR-TKI was listed, it took about five years before these drugs were fully integrated into clinical practice. The median overall survival of patients in Québec who used an EGFR-TKI (three indications) was similar to that in most published studies, supporting previous reimbursement decisions. The median delay between first evidence of cancer and surgery was longer than the 60-day consensus target. Utilization of most healthcare services was heterogeneous between regions. Bronchoscopy on its own seemed overused in many regions, whereas non-surgical approaches as a first method for invasive mediastinal evaluation should have been more systematically applied.
Conclusions
At a relatively low cost, real-world evidence can serve as a powerful tool to validate reimbursement decisions and measure the state of clinical practice. By sharing results with stakeholders, it will enable clinical teams to reflect upon their practice and implement local improvement strategies.
In 2019, the Québec provincial health technology assessment body (INESSS) recommended that lung cancer screening with low-dose computed tomography (LDCT) be accessible in Québec only within the context of an evaluation in the ‘real-world’ care setting. Based on this recommendation, the ministry of health (MSSS) decided, in 2020, to implement a screening pilot project and to conduct a formal evaluation, partnering with a clinical leader (principal investigator), participating hospitals, the provincial public health agency (INSPQ) and INESSS. The goal of this evaluation is to facilitate decision-making regarding the implementation of a province-wide screening program.
Methods
To support the implementation of the pilot project, algorithms and recommendations were developed to guide management of screening program participants. This material, based on Lung-RADS (Lung Computed Tomography Screening Reporting and Data System of the American College of Radiology), was developed by reviewing the literature and by consulting clinical experts. The evaluation plan proposes various indicators, focusing on six main topics: (i) costs, (ii) screening and investigation processes, (iii) clinical effectiveness and other effects on health, (iv) effects on smoking cessation, (v) organizational impact and (vi) implementation issues.
Results
INESSS has developed 12 algorithms and close to 50 recommendations for lung cancer screening and investigation, a tool for assessing lung cancer risk and a benefits/risks table. For the evaluation of the pilot project, MSSS, INSPQ and INESSS developed more than 100 indicators; short-term indicators are currently being measured and others will follow in the longer term. Since starting in June 2021, the pilot project is progressing well (as of November 28, 2021): 2,365 people have been referred, 1,272 were eligible for screening, 678 have had their first LDCT and 19 were Lung-RADS 4B or 4X. Results on indicators will help the ministry decide on the feasibility of scaling up screening to the provincial level and will highlight aspects to be improved.
Conclusions
This project shows how health technology assessment products can elicit changes in the health system, and how multi-stakeholder collaboration can actively support practice implementation and inform decision-making.
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