In this article, we discuss why you should use validated patient reported outcomes (PROs) in your PMCF survey. Under the EU Medical Device Directive (MDD), the use of validated questionnaires in post-market surveys was not so rigidly required. However, under MDR, this has changed significantly. Notified Bodies are now expecting all studies with patient reported outcomes to generate clinical evidence using validated questionnaires. This requirement also applies to FDA submissions.
Real-World Evidence and the Role of PROs
Both FDA and competent authorities in the EU are placing greater importance on Real-World Evidence (RWE) since normal investigational studies alone are not sufficient to provide assurance of efficacy and safety of medical treatments using medical devices throughout their lifetime. One of the key tools to obtain Real-World Data (RWD) is the patient-reported outcome (PRO).
In order to generate reliable and accurate clinical data, many researchers and institutions have developed PRO questionnaires to collect consistent, reliable and accurate data from patients and validated them through extensive testing. There are numerous validated PROs available in many therapeutic areas.
What Are Validated PROs and Non-Validated PROs?
Validated PROs
Rigorously tested questionnaires that have been validated for their ability to measure psychometrics with consistency across different demographics, linguistics, and cultures. These provide reliable and standardized measurement.
Non-Validated PROs
Questionnaires developed in-house that have never been rigorously tested for validity in delivering consistent measurement. These lack the standardization and reliability required by regulatory bodies.
The Rise of Patient-Centric Research
Due to the emphasis on patient-centric clinical research and studies, patient-reported outcomes (PROs) are being used more than ever before. In fact, the FDA is recommending the use of clinical outcome assessments (COA), including PROs, as a way to reduce burdens on patients who participate in studies as a substitute to traditional study methods.
Quality Standards
The quality of data cannot be compromised by using PROs. To ensure delivery of high-quality, reliable data, researchers and clinicians in many therapeutic domains have developed PROs through rigorous testing and review processes.
Examples of Validated PROs
Here are some examples of validated PROs used across different therapeutic areas:
Common Validated PRO Instruments
- Kansas City Cardiomyopathy Questionnaire (KCCQ): Measures disease-specific health status in patients with heart failure
- Breast Evaluation Questionnaire (BEQ): Assesses patient satisfaction with their breasts before and after surgery
- FACE-Q: Assesses aesthetic devices using different subsets of scales for appearance appraisal, adverse effect checklist, process of care scales, and quality of life scales
There are numerous readily available validated PROs in most therapeutic areas. Since it takes significant time and cost to develop validated PROs or validate your own PRO, it is recommended to use readily-available PROs whenever suitable ones are available for your clinical studies, including PMCF surveys.
Why You Need to Use Validated PROs for Your PMCF Surveys
The purpose of PMCF is to collect clinical data to generate clinical evidence to fill data deficiency gaps and to update the devices' clinical safety and performance. Therefore, high-quality evidence is required.
Regulatory Expectations
From our experience, Notified Bodies will no longer accept non-validated PROs for your PMCF plan. Even if they accept the PMCF plan, it would be extremely difficult to convince them that data generated by a non-validated PRO constitutes sufficient clinical evidence.
Licensing and Permissions
In order to use readily-available validated PROs, in most cases you will need to pay a license fee to obtain permission for use from the author of the PRO as it is copyrighted. If certain PROs are not required to pay license fees, you will still need to obtain permission from the author as they are copyrighted.
Cost vs. Efficiency
Financial and Regulatory Considerations
Complying with EU MDR poses financial challenges on medtech manufacturers compared to MDD. It is therefore crucial to choose optimal PMCF activities not only from a regulatory compliance aspect but also from a corporate financial aspect. The PMCF survey is one of the most efficient options for PMCF activities. Therefore, if it is applicable to fill the data gap for your CER, the PMCF survey is a highly recommended option.
Digital Solutions: ePROs
The PMCF survey can be efficiently implemented if using electronic patient-reported outcomes (ePROs). There are a number of digital platform providers who offer ePROs and other modules such as eConsent. Digital platforms streamline data collection, improve patient compliance, and enhance data quality through automated validation and real-time monitoring.
Key Takeaways
- Under EU MDR, Notified Bodies now require validated PROs for all studies with patient-reported outcomes
- FDA also expects validated questionnaires for clinical evidence generation
- Real-World Evidence (RWE) is increasingly important for demonstrating device safety and efficacy throughout their lifetime
- Patient-reported outcomes (PROs) are key tools for collecting Real-World Data (RWD)
- Validated PROs have been rigorously tested for consistency across different demographics, languages, and cultures
- Non-validated PROs developed in-house lack the standardization required by regulatory bodies
- Patient-centric research is driving increased use of PROs as alternatives to traditional study methods
- FDA recommends clinical outcome assessments (COA) including PROs to reduce patient burden
- Quality of data cannot be compromised when using PROs
- Examples of validated PROs include KCCQ, BEQ, and FACE-Q across various therapeutic areas
- Readily-available validated PROs should be used whenever suitable alternatives exist
- Developing or validating your own PRO is time-consuming and costly
- License fees are typically required to use copyrighted validated PROs
- Permission from the PRO author is mandatory even for non-licensed instruments
- Non-validated PROs will likely result in Notified Body deficiency feedback
- PMCF surveys using validated PROs are more efficient than alternative PMCF activities
- Electronic PROs (ePROs) enhance efficiency and data quality in PMCF surveys
- Choosing appropriate PMCF activities balances regulatory compliance with corporate financial considerations
Need Expert Guidance on PROs and PMCF Surveys?
If you want to learn more about validated PROs or PMCF surveys, our team of experts is here to help you navigate the complexities of EU MDR compliance.
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If you have questions about validated PROs or PMCF surveys, please feel free to contact us at clientcare@eclevar.com. Our team is ready to support your regulatory strategy.