RWE Transparency Position Statement

As leaders in the generation and evaluation of scientific evidence to inform regulatory, clinical, and policy decisions, our societies recognize a growing challenge: public trust in science has eroded at the very moment when the use of real-world evidence (RWE) studies in decision making is increasing globally. Against this backdrop, scientific rigor must be both applied and seen. Transparency enables the public and decision makers to trust the science that underpins RWE.

ISPOR—The Professional Society for Health Economics and Outcomes Research and the International Society for Pharmacoepidemiology (ISPE) are major global RWE scientific authorities. By developing and promoting the best scientific practices and policies for RWE, both organizations recognize their critical role in upholding science and rigor. With this position statement, our organizations affirm that transparency is fundamental to the reproducibility, credibility, and usefulness of RWE.

Recent experience in the health policy landscape has made this clear. Misleading evidence from RWE studies can create confusion, weaken confidence, and leave decision makers, journal editors, patients, and caregivers uncertain about how to interpret the research. When methods, assumptions, and analytic decisions are not transparent, strong studies can be ignored, while weaker studies may be given more credibility than warranted. Misinformation thrives in these blind spots, and public trust in our field suffers as a result.

Our organizations therefore assert that transparency is essential to advancing the impact and societal value of RWE studies. Our field must learn from the history of randomized controlled trials (RCTs), which adopted preregistration and public posting of protocols with tracked amendments only after years of scrutiny and external pressure.¹ We have the responsibility to require transparency proactively, before crises force reactive change or lack of trust prevents RWE from reaching its full potential for positive impact on patient wellness and public health.

As we issue this joint position statement, we also wish to clearly distinguish transparency from validity. Transparency does not guarantee that a study is valid, nor does a lack of transparency imply flawed methods. What transparency does provide, is the information that stakeholders need to differentiate between robust versus weak studies.

Validity depends on rigorous design, appropriate data, and sound analytic choices. Transparency is what allows others to evaluate those elements. It provides a clear, traceable account of what was planned, what was implemented, what was amended, and why. Without transparency, it is impossible to fully understand methodological choices, evaluate potential sources of bias, and draw appropriate conclusions. With it, however, opportunities arise to discuss differences between studies and to interpret them constructively.

Our organizations endorse intentional documentation throughout the study lifecycle using established tools and processes such as preregistration of protocols before research is initiated, use of structured protocol templates, shared analytic code, detailed evaluation of data quality, instructions on how to acquire data use agreements, and adherence to guidance for making reporting sufficiently clear so that others can follow the chain of scientific reasoning.

Our field is well positioned to achieve this by leveraging the tools that have been developed by our stakeholders:

• The RWE Registry², HMA-EMA Catalogue³, ClinicalTrials.gov⁴, and other platforms now enable RWE protocol preregistration and full reporting of results beyond the constraints of scientific publications.
• The HARmonized Protocol Template to Enhance Reproducibility (HARPER) protocol template, developed through an ISPE–ISPOR collaboration^5,6, provides a shared framework for describing intended study design and implementation. Its incorporation into recent guidance from organizations such as the International Council on Harmonization (ICH)⁷, European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP)⁸, Council for International Organizations of Medical Sciences (CIOMS)⁹, National Institute for Health and Care Excellence (NICE)¹⁰, and the Centers for Medicare and Medicaid Services (CMS)¹¹ reflect global recognition of its value.
• Data quality assessment tools, guidance, and resources such as the Data Quality Framework for the EU Medicines Regulation¹², DataSAT from NICE¹³, QCARD from the FDA Oncology Center of Excellence¹⁴, the HMA-EMA Catalogues³ of real-world data sources, and others, which are essential to achieving  scientific rigor.
• Tools and community standards for code sharing, phenotyping logic, and reproducible workflows are increasingly available.
• Transparency statements with open science badges should be used to provide a concise and visible account of what was planned, what was shared openly, and how others can access key study materials. ISPOR and ISPE journals now encourage transparency statements with open science badges, and moving forward, our conferences will recognize open science practices in oral and poster presentations.

As organizations firmly committed to advancing high-quality RWE, we take the position that transparency must become a shared expectation across our community.

But these steps alone are not enough. Widespread cultural change requires participation from every member of the RWE research community.

We therefore issue a call to action to each of our members and collaborators to build greater transparency into everyday practice for RWE generation.

Specifically, we ask that: 

1. Investigators preregister all future study protocols
2. Research teams use structured protocol templates like HARPER
3. Data providers publish quality metrics for their data
4. Authors include structured transparency statements with open science badges in manuscripts, posters, and presentations
5. Academic institutions include transparent research practices as part of the standard curriculum
6. Trainees and early-career researchers adopt transparency as a default element of scientific practice—not because it is mandated, but because it strengthens the credibility of their work and the field they are joining
7. Senior leaders model and reward these practices, and journals and peer reviewers require them
   

We invite key stakeholder organizations to join us in promoting transparency and setting field-wide expectations.

RWE can influence high-stakes decisions and shape patient care and outcomes, but without public trust in science, its impact may not be fully realized. We stand together in endorsing a future in which transparency is woven into every stage of our scientific work and call on our members and collaborators to work together to help build that future.

Signed by: