A recent report, published in Value in Health, has described a plan for making registration of hypothesis evaluating treatment effectiveness (HETE) studies routine. The report also explored current concerns about secondary data studies.
In medicine, transparency is key. Those who make healthcare decisions must evaluate and make informed decisions on the basis of high-quality, relevant evidence. In recent years, there has been a growing interest in using data from clinical practice – real-world data (RWD) – to help make informed decisions. This has created an urgency to develop processes that promote trust in the evidence-generation process and also enable decision-makers to evaluate the quality of methods used in real-world studies. As a result, the need to increase the credibility of real-world evidence (RWE) has become critical.
RWE studies based on secondary analysis of existing data are susceptible to biases. This is less of an issue for primary analysis of randomised controlled trials. While transparently reporting study methodologies is important, it does not necessarily mean that the study is of high quality. Nevertheless, transparency improves the ability of a decision-maker to assess the quality and validity of a study. It also facilitates replication of results.
Experts have proposed study registration, particularly for HETE studies using secondary data, to improve transparency and trust in RWE. These studies evaluate the presence or absence of a prespecified effect or its magnitude. Existing study registries currently only focus on studies that collect primary data.
RWE Transparency Initiative
Transparency increases the credibility of study findings. In 2017, ISPOR and ISPE created a joint task force to identify good practices for addressing concerns about ensuring high-quality RWE and to strengthen confidence in evidence derived from HETE RWE studies. This task force published recommendations for improving the transparency of HETE RWE studies. These included: declaration of conduction of HETE study, posting study protocol and data analysis plan publicly and also publishing study results.
Then in February 2019, ISPOR brought together 30 experts to explore the structural and practical challenges in successfully implementing these recommendations. This meeting led to the creation of the RWE Transparency Initiative. This initiative is focussed on establishing a culture for transparency for study analysis and reporting of HETE studies using secondary data. In particular, the use of study registrations as a tool for encouragement.
The plans for this initiative include:
- Specifying the rationale for registration of RWE studies
- Identifying the studies that should be registered and the timeframe for registration
- Analysing how and when analytic deviations should be considered
- Specifying how and when to publish results
- Creating incentives to encourage registration
The RWE Transparency Initiative has identified key steps to building on the foundation of existing study registries, identified issues that impact the practicality of the registration process and considered how to facilitate routine registration of HETE RWE studies. As the use of RWE in decision-making grows, the need to trust the evidence simultaneously builds. The authors noted that improving the culture of transparency can help shed light on HETE RWE study practices so that the individuals using the results can better determine the quality of the study themselves.
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