Abstract
BackgroundRegistration authorities evaluate effects of new medicines for chronic obstructive pulmonary disease (COPD) on airway obstruction, dyspnea, health status and exacerbations. To establish clinical relevance, minimal clinically important differences (MCIDs) are used. The aim of this study was to investigate which efficacy endpoints and MCIDs healthcare professionals consider clinically relevant for new COPD medicines.Materials and Methods7,731 Healthcare professionals received an electronic questionnaire. Participants were asked for: 1) preferred efficacy endpoints for new COPD medicines and 2) cut-off values defining clinical relevance for forced expiratory volume in 1 sec (FEV1), Transition Dyspnea Index (TDI) and St. George’s Respiratory Questionnaire (SGRQ). Those cut-off values were compared to the MCIDs used by registration authorities, namely 100 ml for FEV1, 1 unit for TDI and 4 units for SGRQ.Results227 Healthcare professionals responded to the questionnaire. Most preferred efficacy endpoints were exacerbations (51.0%), airway obstruction (46.9%) and health status (44.9%). Mean cut-off values for TDI and SGRQ were significantly higher than the corresponding MCIDs, mean differences 1.5 (95%CI = 1.3–1.8, p < 0.001) and 7.0 (95%CI = 5.1–8.8, p < 0.001), respectively. The mean cut-off value for FEV1 was comparable to the MCID (mean difference 2.2, 95%CI = -19.9–24.3, p = 0.84).ConclusionsHealthcare professionals largely agree with efficacy endpoints used for the evaluation of new COPD medicines. However, they seem to prefer higher cut-off values for clinical relevance for TDI and SGRQ than the registration authorities. Effects of new medicines on TDI and SGRQ that are considered clinically relevant by registration authorities do, therefore, not necessarily reflect healthcare professionals’ perspectives on clinical relevance.
Highlights
New medicines are evaluated by registration authorities in order to assess their benefit-risk balance
Healthcare professionals working with chronic obstructive pulmonary disease (COPD) patients in daily practice were included in this analysis
The final population consisted of 227 healthcare professionals (88 physicians, 107 pharmacists, and 27 practice nurses), resulting in a response rate of 2.9% (Figure 1)
Summary
New medicines are evaluated by registration authorities in order to assess their benefit-risk balance. The assessment of clinical relevance of new medicines by the registration authorities depends heavily on clinical trials. To establish the clinical relevance of a specific improvement on an endpoint minimal clinically important differences (MCIDs) are used. Establishing whether an improvement on a clinical endpoint exceeds the MCID is a way to evaluate the clinical relevance of a (new) pharmacological treatment (Make, 2007; Koynova et al, 2013; Bartels et al, 2017). Registration authorities evaluate effects of new medicines for chronic obstructive pulmonary disease (COPD) on airway obstruction, dyspnea, health status and exacerbations. Minimal clinically important differences (MCIDs) are used. The aim of this study was to investigate which efficacy endpoints and MCIDs healthcare professionals consider clinically relevant for new COPD medicines
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