Planning a clinical trial programme for medicinal products for chronic constipation: An analysis of the European Medicines Agency Guideline

Abstract

INTRODUCTION. Chronic constipation is a widespread condition associated with substantial direct and indirect costs for diagnosis and treatment and a significant reduction in the quality of life of patients. There is a need for the development and clinical studies of novel medicinal products for chronic constipation due to the limited availability of effective treatment options and concerns regarding the long-term safety and tolerability of these options. Currently, the Russian Federation and other Member States of the Eurasian Economic Union (EAEU) lack guidelines governing the design and conduct of clinical trials of new medicinal products for chronic constipation.AIM. This study aimed to analyse the relevant international approaches and methods as a potential basis for drafting a regional guideline for planning clinical trial programmes for novel medicinal products for chronic constipation.DISCUSSION. This study analysed the main provisions of the European Medicines Agency (EMA) outlined in the Guideline on the evaluation of medicinal products for the treatment of chronic constipation (including opioid-induced constipation) and for bowel cleansing. The authors identified special considerations for conducting pharmacology studies and confirmatory clinical trials (selection of the clinical trial population, design, and duration; selection of primary and secondary efficacy endpoints; and safety assessment). Additionally, the authors highlighted special considerations for confirmatory clinical trials in paediatric and geriatric populations.CONCLUSIONS. The EMA guideline covers all the requirements that are necessary for designing a clinical trial programme for a novel medicinal product for chronic constipation. Therefore, the EMA guideline can inform the ongoing development of the corresponding guideline for the Russian Federation and other EAEU Member States.

Keywords

• chronic constipation
• clinical trial
• medicinal product
• efficacy
• safety
• guidelines
• european medicines agency
• ema
• laxatives