The clinical application of Real World Evidence (RWE) has become an international trend.  It can be used to improve the design of clinical trials or to support the assessment of post-marketing drug efficacy and safety.  Many countries have established regulations to facilitate the application of RWE.  The TFDA referenced international practices and formulated the “Key Considerations in Using RWE to Support Drug Development” as a guidance for the industry.

Using Real-World Evidence (RWE) generated from Real-World Data (RWD) to support drug efficacy or safety is an emerging trend.  The TFDA reviewed international regulations and produced this Guidance for the industry.  The TFDA also invited experts and scholars to form a case study taskforce, hoping to provide better guidance on the application of RWE to supporting drug R&D and the changes of drug indications.

RWE has already been widely used in the evaluation of post-marketing drug safety.   The application is now extended to supporting the applications of drugs for rare diseases, drugs with difficulties in conducting clinical trials and drugs lacking adequate reference products for pivotal trials, as well post-approval changes of indications. 

Drug efficacy and safety used to be assessed solely through conducting controlled clinical trials.  An adequate trial design, i.e. setting subject criteria and treatment environment, helps to produce quality data for the assessment of drug efficacy and safety.  However, clinical trials are restricted to limited sample base, strict recruitment criteria, restricted treatment choices and short observation time.  Hence, the result of clinical trials may not fully reflect the actual situation in the real world. 

This gap could be bridged by the use of RWE.  RWE is not a replacement for clinical trials.  It should be regarded as supplement data to conventional clinical trial results.

However, the quality and sources of RWD and its analysis methods all affect the data credibility.   The US FDA published the “Use of Real-World Evidence to Support Regulatory Decision Making for Medical Devices” and the “Framework for FDA’s Real-World Evidence Program” in 2017.  The EU and China have also published relevant guidance.  It indicates the importance of the application of RWE and the necessity of regulatory guidelines. 

For more information about the TFDA’s guidance, please visit the TFDA’s website at https://www.fda.gov.tw/TC/newsContent.aspx?cid=3&id=26255.

【2020-07-23/ TFDA】