Japan to accept electronic study data for new drugs from FY 2016
This article was originally published in SRA
Japan's medicines regulator will in fiscal year 2016 start to accept electronically-submitted study data from drug makers seeking market approval, as part of the agency's plan to make electronic submission for study data mandatory. The Ministry of Health, Labour and Welfare explained the new rule in a notification on electronic submission of study data for new drug applications 1,2.
Companies will not be obliged to submit their study data in electronic format from FY 2016 (that starts from April), according to the Pharmaceuticals and Medical Devices Agency, which says there will be a transition period of about two years before the requirement becomes mandatory. However, the PMDA asks companies to use the electronic data submission system as soon as possible once the system is up and running.
The PMDA says it would, in principle, require electronic data on results from all phase II and III studies (including long-term studies) that are generally regarded to be the major evidence for evaluation of efficacy, safety and dosage and administration.
For study results from Phase I studies and clinical pharmacology studies, electronic submission of data will be required from: Phase I studies of oncology drugs; Phase I studies that have been conducted on both Japanese and non-Japanese subjects (eg, in case of a strategy of global clinical trials and bridging studies); and QT/QTc studies based on the International Conference on Harmonisation E14 guideline.
The PMDA adds that in cases where integrated analyses have been conducted for multiple study results on efficacy or safety, the datasets for those analyses results (integrated summary of safety [ISS]/integrated summary of effectiveness [ISE]) may also be required to be electronically submitted.
The agency is presently considering whether it should ask companies to submit electronic data from studies other than clinical studies (eg non clinical studies) and clinical studies conducted after approval. It will issue an opinion on this matter at a later stage.
The PMDA explains that it is seeking electronic study data on new drug applications from companies because it wants to use this information to conduct its own research and analyses while reviewing the applications of individual products. This, in turn, would allow the agency to offer more objective and scientific decisions on products under its review and improve the overall quality of its review process.
The agency believes that electronic submission of study data will also benefit the industry in many ways. For example, using the results of PMDA-conducted analyses during reviews and scientific consultations may increase both the efficiency and success rate of drug development for companies.
Also, electronic submission of study data may help to reduce the overall burden on companies. This is because, at present, the PMDA often requires applicants to re-analyze their clinical study data. By having the PMDA conduct those analyses, such requests from the agency are expected to become less frequent and more specific.
Moreover, the agency believes that establishing a system of clinical data collection in Japan based on the widely and internationally used electronic format may not only allow both the PMDA and companies to conduct appropriate and the latest analyses and evaluations with the consideration of international co-operation, but may also promote multi-regional research and development.
The PMDA wants to establish uniform methods for collecting study data from various products as this will also allow product cross-sectional evaluations and may enable utilization of modeling and simulation. The agency notes that modeling and simulation is an approach that is gaining much attention and is expected to enable more accurate predictions, for example, on the relationship between pharmacokinetics and clinical effect, dose-response of clinical effect, and course between a disease and its prognosis.
Details of the new requirements are outlined in a guidance note and a Q&A document, which explain, among other things, the format and method that companies should follow for electronic submission of study data.
References
1. PMDA, Basic Principles on Electronic Submission of Study Data for New Drug Applications, 10 July 2014, www.pmda.go.jp/english/service/pdf/taskforce/140620-tsuchi_e.pdf
2. PMDA, Question and Answer Guide Regarding PFSB/ELD Notification No.0620-6, 10 July 2014, www.pmda.go.jp/english/service/pdf/taskforce/140620-QandA_e.pdf