Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) recently published an English translation of points to be considered during the evaluation process of a new drug. The original Japanese version was published in April 2008.
The five main points the document emphasized were the following:
- Has the reliability of the conducted studies and submitted documents been ensured?
- Is the efficacy in the study population considered to be more effective than placebo according to the results of properly designed clinical studies?
- Do the obtained results have clinical significance?
- Are there any unacceptable risks as compared to the benefits?
- Can the drug be supplied continuously with stable efficacy and safety from a quality assurance standpoint?
The PMDA also made suggestions such as conducting two or more randomized controlled studies and confirming superiority against a placebo. Another main section is whether there are significant differences in efficacy and safety caused by ethnic factors. The PMDA will check for such items as:
- Have an adequate number of Japanese cases been included?
- Have ethnic factors (intrinsic and extrinsic factors) described in the ICH E5 guideline been considered?
- Is the pharmacokinetic profile in the Japanese population similar to that in the foreign populations?
- Is the dose-response relationship in the Japanese population similar to that in foreign populations?
- In the case where ethnic factors (intrinsic and extrinsic factors) are considered to be different, would the factors have many major impacts on the efficacy and safety?
- Have any specific risks been recognized in the Japanese population?
Other points the PMDA will take into consideration include whether efficacy and safety are affected greatly by ethnic factors, whether efficacy has been confirmed sufficiently even in an unblended study without a control, etc.