Regulatory approval of new drugs

Most regulatory bodies, concerned with the approval of new drugs for clinical use, do not accept the results of uncontrolled studies as stand-alone evidence of efficacy. There are, however, circumstances under which data from uncontrolled studies can provide acceptable evidence of efficacy (a) the treatment results in consistent and clinically significant improvement in a disease with a well-established natural course and remission rate, (b) the treatment consistently results in significant improvement in all or almost all patients. 

The decision to approve a new drug is made by a national regulatory body (Food Drug Administration in the U.S.A., the Health Protection Branch Drugs Directorate in Canada, UK Europe, Australia) to which manufacturers are required to submit their appUca-tions. AppUcants must document by means of appropriate test data (from predinical and clinical trials) that the criteria of efficacy and safety have been met and that product forms (tablet, capsule, etc.) satisfy general standards of quality control. 

The Pharmaceuticals and Cosmetics Division (Koseisho) of the Pharmaceutical Affairs Bureau of the Ministry of Health and Welfare (MHW) is the regulatory body in Japan. Also in Japan there have been clear changes in the drug approval system, mainly inspired by ICH. One of the most important recent changes is that, under certain conditions, it is now possible to use also foreign data for the approval of new dmgs in Japan. 

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