China drug real-world study design and protocol framework guidelines (2022 Draft) has been released by the Center for Drug Evaluation (CDE) on July 7, 2022, for industry stakeholder comments. Feedback can be submitted until September 7, 2022 (see below for further information).
The guideline applies to clinical evidence of drug evaluation obtained through real-world studies.
Main types of China drug real-world study design
- Observational study design: Although there are many types of observational studies, this guideline refers to cohort studies. Cohort studies can be divided into retrospective, prospective, and retrospective prospective cohort studies.
- Pragmatic Clinical Trial (PCT): The study refers to a clinical trial that is as close as possible to real-world clinical practice, and is something between randomized controlled clinical trial (RCT) and observational studies, which is interventional study.
- Single-arm study design: If the one-arm research is interventional, it is a one-arm test; If non-intrusive, it is a one-arm observational study. Whether interventional or non-interventional, one-arm study designs should generally have external controls in the form of historical or parallel controls based on natural history cohort data for disease or other external data, or target value controls.
Main framework of the real-world research protocol
The main framework of real-world research protocols of different design types are basically the same. The guideline suggests that the framework should cover the protocol summary, research background, research purpose, overall design, study population, treatment or intervention, study endpoints, baseline variables and important covariates, data governance / data management plan, bias consideration, statistical analysis plan, quality control, etc., however, special consideration for additional contents may be needed depending on each individual research project.
How to submit comments
The deadline for comments is September 7, 2022. Cisema customers may submit their feedback to their regulatory affairs project manager otherwise feedback can be sent to: email@example.com.
The guideline further describes in detail the key points for consideration of the above real-world designs, and the technical requirements for the feasibility of real-world research paths, representativeness of target population, mixed study designs, virtual control methods, estimated targets, etc. If you would like to learn more about the details, please contact Cisema.