2023 Volume 40 Issue 3 Pages 187-191
Unapproved and off–label use of drugs and devices have several problems : insufficient evidence, unstable supplies, serious adverse effects not covered by the official relief system, disadvantage of physicians in the event of a lawsuit, assessment of reimbursements, etc. Moreover, clinical trials for pediatric and rare diseases are often not conducted owing to their poor profitability and the difficulty of developing drugs and devices and recruiting patients.
For these reasons, the Clinical Trial Committee of the Japanese Society of Pediatric Cardiology and Cardiac Surgery (hereinafter the Society) began a clinical trial promotion project in 2015 and renewed the initiative in 2018. The society receives compensation under contract with a sponsor, and the project team provides support at each stage of a clinical trial : 1) pre–trial (planning, survey of eligible patients and their willingness to participate, and selecting institutions for participation) ; 2) during the trial (holding meetings, publicity and provision of information to members of the Society) ; and 3) post–trial (preparing reports and articles, conducting post–marketing surveillance, etc.).
To date, the Society has facilitated the testing and approval of seven drugs and three surgical materials through this program. Clinical trials have been completed for five subjects, one of which has qualified for insurance coverage and is now available commercially. Experience has shown that clinical trials that progress quickly are characterized by the following : 1) pediatric trials are initiated before approval for insurance coverage is obtained for adult patients ; 2) strategy meetings are held frequently ; and 3) the Society is involved from the planning stage to improve feasibility.
We hope that the Society's system for supporting clinical trials under contract with sponsors will help promote clinical trials in collaboration with industry, government, and academia so that patients can receive effective and safe treatments that qualify for insurance coverage as soon as possible.
