2020 Volume 57 Issue 5 Pages 372-378
Various tumor-profiling multiplex gene panels are being developed, some of which are covered by National Health Insurance in June 2019. Some patients cannot receive targeted therapy even when actionable alterations are detected, because agents for targeted therapy of their disease are not approved. The first option for such patients is to participate in a registration-directed trial for drug approval; however, it is impossible for them to join when they do not fit the eligibility criteria. Therefore, a new trial called “BELIEVE”, which aims to improve drug access, has been planned. The BELIEVE trial is designed as a basket/umbrella trial and conforms to patient-proposed healthcare services—a system within the mixed billing system to apply a combination of treatments covered and not covered by the public health insurance. Patients aged 16 or older with solid tumors showing actionable alterations detected by multiplex gene panel tests are eligible. Patients are assigned to receive treatment on the basis of genetic changes found in their tumors. Treatment is administered in accordance with the directions in the package insert. In each drug cohort, the primary endpoint is response rate. Some collaborating pharmaceutical companies provide drugs for free to reduce the costs borne by the patients. The BELIEVE trial is a multi-institutional study organized by the National Cancer Center Hospital and started in October 2019. This is a novel approach for improving drug access. With the knowledge and experience gained from the BELIEVE trial, we wish to further discuss the use of a similar platform for pediatric patients.
