China medical device benefit-risk assessment guidelines have been revised and released (No.25-2023) by the Center for Medical Device Evaluation (CMDE) under the National Medical Products Administration (NMPA) on July 18, 2023. The document aims to provide guidance in evaluating the main factors for benefit-risk assessment, in order to enhance the predictability, consistency, and transparency of the pre-market review process.
Scope of application
The document elucidate the key factors that regulatory affair specialists should consider when conducting benefit-risk assessments during the medical device technical evaluation process. When determining the benefit-risk profile of a product becomes challenging, reference to these guidelines can be immensely valuable. The concepts discussed herein are applicable throughout the entire lifecycle of medical devices, spanning stages such as design, pre-clinical research, clinical evaluation, and design modifications.
Essential factors in medical device benefit-risk assessment
Here are some major factors that will be taken into consideration in the assessment which can potentially contribute to improved patient outcomes:
- Different types of benefits encompass various impacts of medical devices on clinical applications, patient health, and patient satisfaction within the target population. These impacts include enhancing patient self-care abilities, improving quality of life, facilitating functional recovery, increasing survival rates, preventing functional loss, and alleviating symptoms, among others. These endpoints that indicate clinical benefits are often directly measurable, but in some instances, validated surrogate endpoints might be needed. For diagnostic devices, a public health benefit assessment might be necessary, as such devices can identify specific diseases, prevent disease transmission, predict future disease occurrences, enable early disease diagnosis, or identify patients more likely to benefit from certain therapies.
- Evaluating the magnitude of benefits involves assessing whether specific endpoint criteria or predetermined health thresholds have been met. This assessment typically utilizes clinical scoring scales. Changes measured through these scales, such as endpoint improvements or deteriorations, participant health status changes, etc., help gauge the size of patient benefits. Consideration is also given to variations in benefit magnitude across different demographic groups.
- Considering available data, it is possible to predict which patients are likely to benefit. Data might indicate that only a small subset of patients within the target population will benefit, or a particular benefit frequently occurs among the entire patient population. Additionally, different patient subgroups might experience varying degrees of benefit, or the extent of the same benefit might differ. If identifiable subgroups can be recognized, the device can be specified for use in those subgroup patients. Furthermore, when weighing benefits against risks, both the size and probability of benefits and risks should be simultaneously considered.
- The duration of treatment effects can directly impact the determination of benefits. Therapies that necessitate long-term repetition could introduce greater risks, or the benefits might gradually diminish with repeated treatments. From a regulatory perspective, assessing the duration of effect is vital in evaluating the sustainability and long-term impact of the benefits associated with a medical device.
Evaluation of device risks
In the evaluation of potential risks and harms associated with medical devices, the following factors should be comprehensively considered:
- Severity, type, quantity and frequency of adverse events related to device use.
- Probability of adverse events.
- Duration of adverse events.
- Risks arising from false positive or false negative diagnostic results.
Other factors in the benefit-risk assessment to consider
- Consider the degree of certainty in device benefits and risks, accounting for factors like trial design issues, data quality, and potential for blinding.
- Patient-oriented measures, like quality of life indicators and patient-reported outcomes, aid treatment decisions and serve as evidence for product benefits.
- Disease attributes, clinical effects, and treatment history are essential when assessing benefits and risks.
- Patients’ willingness to accept risks relative to potential benefits, especially when enhancing quality of life, influences benefit-risk determinations.
- Evaluate approved alternatives for intended use, considering their efficacy, risks, and how they meet patient and practitioner needs.
- Employ measures to reduce harm probability, such as warnings, precautions, or scope limitations, and consider other risk control approaches.
- Real-world data collection can refine benefit-risk assessments, identifying new risks, validating risk mitigation, and understanding patient subgroup responses.
- Consider if a device uses breakthrough technology to meet unmet medical needs, even if benefits are modest or uncertain.
For specific guidance and advice tailored to your particular circumstances, please consult with legal and regulatory professionals familiar with China’s medical device regulations.
Further information
Download the revised China medical device benefit-risk assessment guidelines to read.
The appendices of the guidance document also cover a table and four case studies as reference to the public for the benefit-risk determination. Contact us to request for a copy of the English translations.
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