Objective To investigate the efficacy of home-based gait training using the wearable Stride Management Assist (SMA) exoskeleton in people with moderately advanced Parkinson's disease. Methods This was a single-center, open-label, parallel, randomized controlled trial. We included outpatients with idiopathic Parkinson's disease who were capable of walking independently with or without walk aids and had Hoehn and Yahr stage 2-4 in the ON state. Patients were randomly assigned (1:1 ratio) to receive either SMA gait training (SMA group) or control gait training (control group). All participants underwent gait training for approximately 30 min. These training sessions were conducted 10 times for 3 months. We measured clinical outcomes at baseline and post-intervention. The between-group difference of distance in the three-minute walk test was the primary outcome. Results Of the 15 randomly assigned participants, 12 (five in the SMA group) completed this study. The between-group difference was a mean of 13.7 meters (standard error of the mean: 7.8) in the 3-minute walk test (p = 0.109). The distance traversed increased from 141.4 m to 154.7 m in the SMA group (p = 0.023), whereas there was no marked change in the control group. In addition, although there was a decrease in the physiological cost index from 0.29 to 0.13 in the SMA group (p = 0.046), it remained unchanged in the control group. Conclusion These findings suggest that home-based SMA gait training may increase the exercise endurance in people with moderately advanced Parkinson's disease.

There is growing interest in the utilization of real‐world data (RWD) and real‐world evidence (RWE) for regulatory purposes. However, there are challenges in the practical utilization of RWD to provide RWE as a basis for regulatory decision making. This article presents the regulatory initiatives in Japan and efforts taken to promote the utilization of RWD/RWE for regulatory decision making at the pre‐ and postapproval stages of a drug. There has been a rapid increase in the number of RWD cases evaluated for drug safety assessment in Japan. Nevertheless, more regulatory experiences and considerations are necessary for the utilization of RWD in the efficacy evaluation of a drug. Based on past experiences, data reliability and appropriateness of the methodology for analysis are the major discussion points in utilizing RWD and RWE for regulatory decision making. International harmonization of regulatory requirements is another important area in utilizing RWD and sharing the RWE globally. We describe our perspective on providing RWE, which is useful for regulatory decision making throughout a drug’s life cycle.