What Does "Good" Look Like In RWE Research?

Real-World Evidence (called RWE) is research that uses real-world data (RWD). RWD is collected routinely data collected outside of a traditional clinical study that pertains to someone’s health status and health care delivery. RWD sources can include data from everyday health care settings in doctors’ offices, clinics and hospital such as electronic health records, claims databases, and patient registries. It can also be information that people collect with tools like wearable devices, apps etc.  

RWE can be used to study medical products like medicines and medical devices to better understand how they are used, assess their effectiveness, and potential benefits or risks of an intervention. RWE provides insights to support regulatory decisions, health policy, and clinical practice.  

The goal of the IDERHA policy-focused activities is to accelerate the pace of policy development by building consensus recommendations on the use of RWD and RWE in regulatory and Health Technology Assessment (HTA) decision-making for medicines and medical technologies.

The recommendations can support everyone in the health care environment. However, at this phase of the work, the key audiences for these recommendations are public decision-makers such as medical product regulatory authorities and HTA bodies and anyone that conducts research that is intended to be used for decision-making (e.g. industry sponsors, academic and clinical research organisations). The recommendations seek to align expectations and best practices between those conducting the research and those making decisions based on the research.

• What kinds are medical products are included? The recommendations apply to Real-World Evidence research for both medicines and medical devices.
• Is this for all RWE studies or just some? These recommendations specifically apply only to RWE studies intended for inclusion in submissions to regulatory authorities and/or HTA bodies to inform decision-making. RWE studies conducted for other purposes, such as product development, clinical trial design, or commercial market research are outside the scope of these recommendations.
• Do these recommendations apply to both health technology assessments (HTA) and Regulatory Decision-Making? These recommendations are for RWE studies submitted to regulatory authorities and/or organizations that conduct Health Technology Assessments (HTAs). While the types of research questions for medical product regulation and HTA submissions often differ, the recommendations focus on how studies are conducted rather than the specific study questions. Therefore, the principles of good RWE research practice should remain the same regardless of the nature of the study question. 

No, these recommendations are intended to be global and promote greater alignment across policy jurisdictions. The current lack of harmonisation in policies between different regions/countries limits access to critical clinical insights and hinders the ability to share important clinical evidence globally. Achieving greater global convergence would reduce uncertainty and inefficiencies in evidence generation, ultimately supporting more timely and equitable access to effective medicines and medical devices, and improve patient outcomes. 

This is the first iteration of the report. As a core component of the project plan and IDERHA’s commitment to public engagement and transparency, this report will be released for public consultation to seek input on the draft recommendations. Additionally, the team will convene a series of workshops with external experts and targeted discussions with key stakeholders to provide feedback. These activities are intended to: 1) solicit feedback on the recommendations, 2) collect additional information on other resources and information that should be considered for inclusion and 3) foster meaningful discussions and build consensus. 

The team has identified several areas for future expansion of the recommendations which we explicitly solicit input on as part of the public consultation to be conducted in early 2026. Based on the feedback from the public consultation and consensus building workshops, the team will refine the content for a final report with policy recommendations, which will be issued in 2027. 

In 2024, IDERHA conducted a landscape review of the current policies from multiple regulatory authorities/bodies, HTA organisations and other relevant non-decision-making organisations. The review was released for public consultation and targeted expert feedback was gathered through workshops and interviews. Based on this feedback, input from the IDERHA internal advisory boards, and findings from other similar literature in the field^{i,ii,iii,iv,v}, the IDERHA policy team decided to focus on the importance of demonstrating the rigor of RWE study conduct to counter the perception that RWE is of a lower scientific and evidentiary standard. 

For many medical products, clinical studies remain critical to establishing safety and efficacy. However, traditional clinical studies have limitations which can limit generalisability and underrepresent diverse patient populations. In certain circumstances, such as ethical and/or recruitment challenges, no established standard of care in (very) rare disease and/or pathophysiological rationale, randomised clinical trials may not be feasible. Recognising these limitations underscores the need to complement traditional clinical evidence with RWE, enabling more inclusive, applicable, and patient-centred healthcare decisions and ultimately enhancing the real-world impact of medical research. The health care ecosystem and stakeholders are increasingly advocating for more inclusive and diverse research to address gaps in the clinical evidence. The landscape review revealed that almost all the policy relevant documents acknowledged the potential for RWD and RWE to help close these gaps.

The RWE policy landscape review revealed that almost all the policy relevant documents acknowledged the potential for RWD and RWE to help close clinical evidence gaps. However, many also raised concerns about the robustness of RWE study design and conduct, as well as questions about when, and to what extent, RWE could inform regulatory and HTA decision making.

The team examined the information extracted in the landscape review to develop a simplified “process map” outlining the typical RWE research workflow. This map draws on and adapts the work of academic societies and other professional organisations such as ISPOR and ISPE Joint Task Force, Duke-Margolis Centre for Health Policy and NESTcc amongst others. Please note that while this map is intended to signal a generalised process, additional steps may be needed for different types of studies (e.g. causal inference studies).  

The proposed recommendations are mapped to the different stages in the process. These initial policy recommendations seek to build on the work of early adopters and accelerate development by other countries and organisations who have not yet developed policies and guidance documents.  

There are two primary recommendations that apply across the research map. Under each recommendation are more detailed sub-recommendations on individual steps in the process map. Each of the sub-recommendations have information categorised based on their priority level: 

Essential: Actions that are foundational and must be implemented to ensure credibility, transparency and acceptability of RWE studies. These are non-negotiable elements that directly impact the trustworthiness of the evidence. 

Important: Actions that significantly enhance the quality and robustness of RWE but may be adapted based on context, resources, or study objectives. While not mandatory, they are strongly encouraged. 

Optional: Actions that are beneficial and can add value, especially in specific contexts or for certain stakeholders. Their implementation may depend on available resources, study objectives, or stakeholder expectations. 

This prioritisation of actions and considerations is intended to help stakeholders navigate the research process, allocate resources strategically and progressively strengthen practices in line with study goals and regional/local requirements across regulatory and HTA environments.