Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis.

Abstract

Objectives: This paper aims to provide insight into the challenges and opportunities of conducting an investigator-led, international, multicentre clinical trial for Inclusion Body Myositis (IBM), a rare inflammatory myopathy.

Methods: An international, multicentre, randomised, controlled trial of a repurposed drug (sirolimus) was initiated based on promising results from a mono-centric pilot study. The progress of the trial was analysed to identify key challenges encountered and solutions developed.

Results: This large, collaborative study has presented a mosaic of challenges and opportunities, many ubiquitous with investigator-led trials. Key challenges have included securing adequate funding, coordinating manufacture of placebo, negotiating international contracts, managing limited study budgets and delays linked to the COVID-19 pandemic. Alongside these challenges, the study team have found opportunities for creative and effective solutions, including the flexibility of building study databases, optimising digital data capture and harnessing patient involvement.

Conclusions: Instrumental to the progress of the trial has been the collaboration between site teams, patient partnership and adaptability.