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>Explore how real-world evidence can inform value, affordability, and access decisions for cell and gene therapies.<br /><strong></strong></h4><div><p data-start="296" data-end="955">Cell and gene therapies (CGTs) have the potential to deliver transformative, long-term benefits through one-time treatment, often for severe or rare conditions. However, these therapies are frequently supported by limited clinical evidence, such as small or single-arm trials, creating uncertainty for payers related to eligible populations, appropriate comparators, durability of benefit, and relative effectiveness. These uncertainties are compounded by the high upfront costs of CGTs and concerns about affordability and budget impact.</p><p data-start="957" data-end="986"><strong data-start="957" data-end="986">Technical Topics Include:</strong></p><ul data-start="988" data-end="1430"><li data-start="988" data-end="1049"><p data-start="990" data-end="1049">The role of RWE in supporting economic evaluation of CGTs</p></li><li data-start="1050" data-end="1147"><p data-start="1052" data-end="1147">Addressing evidence gaps related to eligibility, comparators, and natural disease progression</p></li><li data-start="1148" data-end="1221"><p data-start="1150" data-end="1221">Evaluating relative effectiveness and durability of benefit using RWE</p></li><li data-start="1222" data-end="1298"><p data-start="1224" data-end="1298">Payer archetypes focused on value, cost-effectiveness, and budget impact</p></li><li data-start="1299" data-end="1362"><p data-start="1301" data-end="1362">Assessing internal and external validity of real-world data</p></li><li data-start="1363" data-end="1430"><p data-start="1365" data-end="1430">Using RWE to inform payment models and outcome-based agreements</p></li></ul><p data-start="1432" data-end="1519"><strong data-start="1432" data-end="1519">This Course Includes Tools and Concepts That Can Be Immediately Applied, Including:</strong></p><ul data-start="1521" data-end="1792"><li data-start="1521" data-end="1583"><p data-start="1523" data-end="1583">Practical guidance on planning and generating RWE for CGTs</p></li><li data-start="1584" data-end="1655"><p data-start="1586" data-end="1655">Examples of RWE use in payer decision-making and policy development</p></li><li data-start="1656" data-end="1717"><p data-start="1658" data-end="1717">Frameworks for balancing access, value, and affordability</p></li><li data-start="1718" data-end="1792"><p data-start="1720" data-end="1792">Real-world case examples illustrating RWE-informed economic evaluation</p></li></ul><p data-start="1794" data-end="2013" data-is-last-node="" data-is-only-node="">Participants will gain a clearer understanding of how RWE can be used to address payer uncertainty and support informed reimbursement and policy decisions for cell and gene therapies across diverse stakeholder contexts.</p><p data-start="258" data-end="762"><strong fontscheme="2">PREREQUISITE: </strong>This course requires familiarity with basic economic evaluation and HTA concepts and methodologies of pharmaceuticals.</p><div></div><div><p> </p></div></div><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</a> to 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>Daniel Gladwell, PhD</strong></div><div>Chief Scientific Officer, HEOR</div><div>Lumanity</div><div>London, England</div><div><br /></div><div><strong>Antal T. Zemplenyi, MSc, MPH, PhD</strong></div><div>Health Economist and Research Assistant Professor</div><div>Skaggs School of Pharmacy and Pharmaceutical Sciences</div><div>University of Colorado</div><div>Aurora, CO, USA</div><div><br /></div><div><strong>Oriol Solà-Morales, MD, MSc, PhD</strong></div><div>Chair Fundació HiTT</div><div>Health Innovation Technology Transfer (HiTT)</div><div>Barcelona, Spain</div><div><br /></div><div><strong>Renske MT ten Ham, PhD PharmD</strong></div><div>Assistant Professor Health Economics</div><div>University Medical Center Utrecht (UMC Utrecht) </div><div>Utrecht, the Netherlands </div><div></div><div><p> </p><div><p><strong>Schedule:</strong><strong></strong></p></div><p><strong>LENGTH: </strong>4<strong> </strong>Hours | Course runs 1 day</p><p><strong>Tuesday, 17 May 2026 | Course runs 1 Day<br /></strong><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>Explore how real-world evidence can inform value, affordability, and access decisions for cell and gene therapies.<br /><strong></strong></h4><div><p data-start="296" data-end="955">Cell and gene therapies (CGTs) have the potential to deliver transformative, long-term benefits through one-time treatment, often for severe or rare conditions. However, these therapies are frequently supported by limited clinical evidence, such as small or single-arm trials, creating uncertainty for payers related to eligible populations, appropriate comparators, durability of benefit, and relative effectiveness. These uncertainties are compounded by the high upfront costs of CGTs and concerns about affordability and budget impact.</p><p data-start="957" data-end="986"><strong data-start="957" data-end="986">Technical Topics Include:</strong></p><ul data-start="988" data-end="1430"><li data-start="988" data-end="1049"><p data-start="990" data-end="1049">The role of RWE in supporting economic evaluation of CGTs</p></li><li data-start="1050" data-end="1147"><p data-start="1052" data-end="1147">Addressing evidence gaps related to eligibility, comparators, and natural disease progression</p></li><li data-start="1148" data-end="1221"><p data-start="1150" data-end="1221">Evaluating relative effectiveness and durability of benefit using RWE</p></li><li data-start="1222" data-end="1298"><p data-start="1224" data-end="1298">Payer archetypes focused on value, cost-effectiveness, and budget impact</p></li><li data-start="1299" data-end="1362"><p data-start="1301" data-end="1362">Assessing internal and external validity of real-world data</p></li><li data-start="1363" data-end="1430"><p data-start="1365" data-end="1430">Using RWE to inform payment models and outcome-based agreements</p></li></ul><p data-start="1432" data-end="1519"><strong data-start="1432" data-end="1519">This Course Includes Tools and Concepts That Can Be Immediately Applied, Including:</strong></p><ul data-start="1521" data-end="1792"><li data-start="1521" data-end="1583"><p data-start="1523" data-end="1583">Practical guidance on planning and generating RWE for CGTs</p></li><li data-start="1584" data-end="1655"><p data-start="1586" data-end="1655">Examples of RWE use in payer decision-making and policy development</p></li><li data-start="1656" data-end="1717"><p data-start="1658" data-end="1717">Frameworks for balancing access, value, and affordability</p></li><li data-start="1718" data-end="1792"><p data-start="1720" data-end="1792">Real-world case examples illustrating RWE-informed economic evaluation</p></li></ul><p data-start="1794" data-end="2013" data-is-last-node="" data-is-only-node="">Participants will gain a clearer understanding of how RWE can be used to address payer uncertainty and support informed reimbursement and policy decisions for cell and gene therapies across diverse stakeholder contexts.</p><p data-start="258" data-end="762"><strong fontscheme="2">PREREQUISITE: </strong>This course requires familiarity with basic economic evaluation and HTA concepts and methodologies of pharmaceuticals.</p><div></div><div><p> </p></div></div><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</a> to 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>Daniel Gladwell, PhD</strong></div><div>Chief Scientific Officer, HEOR</div><div>Lumanity</div><div>London, England</div><div><br /></div><div><strong>Antal T. Zemplenyi, MSc, MPH, PhD</strong></div><div>Health Economist and Research Assistant Professor</div><div>Skaggs School of Pharmacy and Pharmaceutical Sciences</div><div>University of Colorado</div><div>Aurora, CO, USA</div><div><br /></div><div><strong>Oriol Solà-Morales, MD, MSc, PhD</strong></div><div>Chair Fundació HiTT</div><div>Health Innovation Technology Transfer (HiTT)</div><div>Barcelona, Spain</div><div><br /></div><div><strong>Renske MT ten Ham, PhD PharmD</strong></div><div>Assistant Professor Health Economics</div><div>University Medical Center Utrecht (UMC Utrecht) </div><div>Utrecht, the Netherlands </div><div></div><div><p> </p><div><p><strong>Schedule:</strong><strong></strong></p></div><p><strong>LENGTH: </strong>4<strong> </strong>Hours | Course runs 1 day</p><p><strong>Tuesday, 17 May 2026 | Course runs 1 Day<br /></strong><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>)