Real-world evidence on treatment outcomes can be an important aspect of the evidence basis for decision making if it is seen as credible. For real-world studies that are meant to test hypotheses about comparative-effectiveness or safety, a key aspect of credibility is that they are conducted transparently with tests that follow a prespecified analytic protocol. Preregistration of such study protocols on a public website would help build trust that their results can be used for decision-making purposes.
Establishing a Culture of Transparency for Real-World Evidence Studies...
The Real-World Evidence Transparency Initiative Partnership is a joint collaboration and ongoing effort between ISPOR, the International Society for Pharmacoepidemiology, the Duke-Margolis Center for Health Policy, and the National Pharmaceutical Council. The objective of this initiative is to establish a culture of transparency for study analysis and reporting of hypothesis evaluating real-world evidence studies on treatment effects.
Improving Transparency to Build Trust...
The Real-World Evidence Transparency Initiative published a plan to encourage routine registration of noninterventional real-world evidence studies used to evaluate treatment effects. The report, “Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative,”
was published in the September 2020 issue of Value in Health.
More...
The report, “Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative,” was published in the September 2020 issue of Value in Health. The plan includes specifying the rationale for registering hypothesis-evaluating treatment effectiveness real-world evidence studies, the studies that should be registered, where and when these studies should be registered, how and when analytic deviations from protocols should be reported, how and when to publish results, and incentives to encourage registration.
Real-World Evidence Registry
The Real-World Evidence Registry provides researchers with a fit-for-purpose platform to register their study designs before they begin work to facilitate the transparency needed to elevate the trust in the study results.
More...
Real-world evidence studies can be used for hypothesis evaluation of treatment effects including safety (HETE studies). However these studies can also be perceived as less rigorous than clinical trials especially when not preregistered in a public setting such as ClinicalTrials.gov or the EU-PAS register.
ISPOR and its partners ISPE, NPC, and Duke Margolis have developed a simplified registration site especially for RWE HETE studies using secondary data. This searchable site provides a place for preregistration of studies that may not require registration for regulatory purposes but benefit from the rigor of transparent study methods and also provide a reference (such as a URL or doi) to share with peer reviewers, assessors, or other decision making bodies. Researchers can get started ‘here’ by creating a profile on the Open Sciences Framework and registering their study on the RWE Registry.
Shaking the Myth of Real-World Evidence
On-Demand Webinar
Learn more by watching the on-demand webinar, “Shaking the Myth of Real-World Evidence: Updates from the RWE Transparency Initiative.” This session provides updates from the initiative including a walk-through of the study registration site and updates on the special task force developing a standardized RWE protocol template.
Additional Resources
- Real-World Evidence Registry
- Good Practices Reports and Other ISPOR Reports from Value in Health
- HARmonized Protocol Template to Enhance Reproducibility of Hypothesis Evaluating Real-World Evidence Studies on Treatment Effects: A Good Practices Report of a Joint ISPE/ISPOR Task Force
- "Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative"
- "Good Practices for Real-World Data Studies of Treatment and/or Comparative Effectiveness"
- "Reporting to Improve Reproducibility and Facilitate Validity Assessment for Healthcare Database Studies V1.0"
- "Unlocking the Promise of Real-World Evidence" (Value & Outcomes Spotlight, Vol. 6, No. 5)
- ISPOR's Real-World Evidence Strategic Initiatives
Conferences & Summits
May 17, 2026
<h4>Learn the principles that make real-world evidence decision-grade for regulatory and HTA use.<br /></h4><p data-start="161" data-end="565">This course introduces key frameworks and guidelines that define high-quality, decision-grade real-world evidence (RWE), along with case studies demonstrating how RWE has been used in regulatory and HTA approvals. Participants will then walk through an extended interactive example exploring how study design and implementation choices influence the meaning, quality, and interpretability of RWE results.</p><p data-start="567" data-end="596"><strong data-start="567" data-end="596">Technical Topics Include:</strong></p><p data-start="598" data-end="637">• Current RWE frameworks and guidance</p><p data-start="639" data-end="684">• Regulatory and HTA case studies using RWE</p><p data-start="686" data-end="762">• Key design and implementation considerations for decision-grade evidence</p><p data-start="764" data-end="851"><strong data-start="764" data-end="851">This Course Includes Tools and Concepts That Can Be Immediately Applied, Including:</strong></p><p data-start="853" data-end="899">• Practical interpretation of RWE frameworks</p><p data-start="901" data-end="957">• Interactive discussion of a real-world study example</p><p data-start="959" data-end="1076">Participants will gain a clear understanding of how to evaluate and design RWE to support real-world decision making.</p><p data-start="196" data-end="750"><strong fontscheme="2">PREREQUISITE: </strong>Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data.<span style="background-color:transparent;color:inherit;font-family:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;"></span></p><p><em fontscheme="2"></em><strong>LEVEL:</strong> Intermediate<strong><br />TRACK: </strong>Real-World Data & Information Systems<br /><br />This short course is offered in-person at the ISPOR 2026 conference. Separate registration is required. Registration coming soon. <a href="/conferences-education/conferences/upcoming-conferences/ispor-2026">Visit the ISPOR 2026 Program page to register</a> and learn more.<strong><strong></strong></strong></p><p><strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;">FACULTY MEMBERS</strong></p><div><strong></strong></div><div><strong></strong></div><div><strong></strong></div><div><strong></strong></div><div><strong>Sebastian Schneeweiss, MD, ScD</strong></div><div>Professor of Medicine, Harvard Medical School</div><div>Division of Pharmacoepidemiology, Brigham and Women’s Hospital</div><div>Boston, MA, USA</div><div bold-block="true"><br /></div><div><strong>Jeremy A. Rassen, ScD</strong></div><div>Consultant</div><div>New York, NY, USA</div><div><br /></div><div><strong>Shirley Wang, PhD</strong></div><div>Associate Professor of Medicine </div><div>Division of Pharmacoepidemiology and Pharmacoeconomics</div><div>Department of Medicine, Brigham and Women’s Hospital</div><div>Harvard Medical School</div><div>Boston, MA, USA</div><div></div><div><p><strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;"></strong></p><p><strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;">Schedule:</strong></p></div><div><p><strong>LENGTH: </strong>4<strong> </strong>Hours | Course runs 1 day</p><p><strong>Sunday, 17 May 2026 | Course runs 1 Day<br /></strong>1:00pm-5:00pm Eastern Daylight Time (EDT)<strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;"></strong></p></div><p><strong><em>ISPOR short courses are designed to enhance knowledge and techniques in core health economics and outcomes research (HEOR) topics as well as emerging trends in the field. Short courses offer 4 or 8 hours of premium scientific education and an electronic course book. Active attendee participation combined with our expert faculty creates an immersive and impactful learning experience. Short courses are not recorded and are only available during the live course presentation.</em></strong></p>)
Short Courses & Webinars
May 17, 2026
<h4>Learn the principles that make real-world evidence decision-grade for regulatory and HTA use.<br /></h4><p data-start="161" data-end="565">This course introduces key frameworks and guidelines that define high-quality, decision-grade real-world evidence (RWE), along with case studies demonstrating how RWE has been used in regulatory and HTA approvals. Participants will then walk through an extended interactive example exploring how study design and implementation choices influence the meaning, quality, and interpretability of RWE results.</p><p data-start="567" data-end="596"><strong data-start="567" data-end="596">Technical Topics Include:</strong></p><p data-start="598" data-end="637">• Current RWE frameworks and guidance</p><p data-start="639" data-end="684">• Regulatory and HTA case studies using RWE</p><p data-start="686" data-end="762">• Key design and implementation considerations for decision-grade evidence</p><p data-start="764" data-end="851"><strong data-start="764" data-end="851">This Course Includes Tools and Concepts That Can Be Immediately Applied, Including:</strong></p><p data-start="853" data-end="899">• Practical interpretation of RWE frameworks</p><p data-start="901" data-end="957">• Interactive discussion of a real-world study example</p><p data-start="959" data-end="1076">Participants will gain a clear understanding of how to evaluate and design RWE to support real-world decision making.</p><p data-start="196" data-end="750"><strong fontscheme="2">PREREQUISITE: </strong>Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data.<span style="background-color:transparent;color:inherit;font-family:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;"></span></p><p><em fontscheme="2"></em><strong>LEVEL:</strong> Intermediate<strong><br />TRACK: </strong>Real-World Data & Information Systems<br /><br />This short course is offered in-person at the ISPOR 2026 conference. Separate registration is required. Registration coming soon. <a href="/conferences-education/conferences/upcoming-conferences/ispor-2026">Visit the ISPOR 2026 Program page to register</a> and learn more.<strong><strong></strong></strong></p><p><strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;">FACULTY MEMBERS</strong></p><div><strong></strong></div><div><strong></strong></div><div><strong></strong></div><div><strong></strong></div><div><strong>Sebastian Schneeweiss, MD, ScD</strong></div><div>Professor of Medicine, Harvard Medical School</div><div>Division of Pharmacoepidemiology, Brigham and Women’s Hospital</div><div>Boston, MA, USA</div><div bold-block="true"><br /></div><div><strong>Jeremy A. Rassen, ScD</strong></div><div>Consultant</div><div>New York, NY, USA</div><div><br /></div><div><strong>Shirley Wang, PhD</strong></div><div>Associate Professor of Medicine </div><div>Division of Pharmacoepidemiology and Pharmacoeconomics</div><div>Department of Medicine, Brigham and Women’s Hospital</div><div>Harvard Medical School</div><div>Boston, MA, USA</div><div></div><div><p><strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;"></strong></p><p><strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;">Schedule:</strong></p></div><div><p><strong>LENGTH: </strong>4<strong> </strong>Hours | Course runs 1 day</p><p><strong>Sunday, 17 May 2026 | Course runs 1 Day<br /></strong>1:00pm-5:00pm Eastern Daylight Time (EDT)<strong style="background-color:transparent;color:inherit;font-size:inherit;text-align:inherit;text-transform:inherit;word-spacing:normal;caret-color:auto;white-space:inherit;"></strong></p></div><p><strong><em>ISPOR short courses are designed to enhance knowledge and techniques in core health economics and outcomes research (HEOR) topics as well as emerging trends in the field. Short courses offer 4 or 8 hours of premium scientific education and an electronic course book. Active attendee participation combined with our expert faculty creates an immersive and impactful learning experience. Short courses are not recorded and are only available during the live course presentation.</em></strong></p>)