Abstract
Background: For years, clinical trials for the development of respiratory drugs have been reliant upon traditional outcomes like FEV1, lacking good correlation with patient-relevant outcomes. New insights into the basic mechanisms led to the need for a more patient-centred approach. Adoption of more relevant outcome measures seems rather slow.
Goal: Analysis of primary outcome parameters in interventional phase 1-3 (Ph1-3) studies during the last 10 years, by comparing 2009-2013 with 2014-2018.
Method: Clintrials.gov database was searched for interventional studies, phase 1-3, start date 1/1/2009 till 31/12/2018, for Asthma, Chronic Obstructive Pulmonary Disease (COPD), and Cystic Fibrosis (CF). Results for primary outcomes were separated from the column with outcome measures. They were classified as traditional outcomes (pharmacokinetics, adverse events, classic spirometry), pharmacodynamic outcomes (non-traditional lung function, biomarkers), and patient-relevant outcomes (questionnaires, exacerbations, mortality, other).
Results: A total of 1678 studies were included (27.3 % Ph1, 38.0 % Ph2, 34.6 % Ph3). The average number of patients per study type were 46, 168 and 721 respectively. Asthma represented 50 % of the trials, COPD 35 % and CF 15 %. Traditional primary outcomes were used in 63 and 58 % during first and second period respectively, pharmacodynamic measures in 21 % for both periods and patient-relevant outcomes in 16 and 21 % (p=0.021).
Conclusions: Over the last 10 years there is only a very limited increase in the use of other than traditional primary outcome measures. The use of more patient-relevant primary outcomes could help reduce the high attrition rate due to lack of efficacy.
Footnotes
Cite this article as: European Respiratory Journal 2020; 56: Suppl. 64, 3242.
This abstract was presented at the 2020 ERS International Congress, in session “Respiratory viruses in the "pre COVID-19" era”.
This is an ERS International Congress abstract. No full-text version is available. Further material to accompany this abstract may be available at www.ers-education.org (ERS member access only).
- Copyright ©the authors 2020