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
September 3, 2025
<p><a href="/education-training/webinars">Back to all webinars</a></p><h4>Open to all ISPOR Members and Non-members</h4><p><em><strong><br /></strong></em><strong>Title: </strong>Evolving RWE Standards Under IRA & JCA: Implications for Asia's HTA </p><p><strong>Wednesday, September 3, 2025<br /></strong>8:00AM EDT | 12:00PM UTC | 2:00PM CEST<em></em><a href="https://www.timeanddate.com/worldclock/fixedtime.html?msg=ISPOR+Educational+Webinar%3A+Distributional+Cost-Effectiveness+Analysis+to+Inform+Healthcare+Decisions&iso=20220623T11&p1=12&ah=1" data-sf-ec-immutable=""></a></p><p><a href="https://www.timeanddate.com/worldclock/fixedtime.html?msg=Evolving+RWE+Standards+under+IRA+%26+JCA%3A+Implications+for+Asia%27s+HTA&iso=20250903T08&p1=179&ah=1" data-sf-ec-immutable="">Click here for time zone conversion</a><br /></p><p><a href="https://portal.ispor.org/eweb/DynamicPage.aspx?webcode=EventInfo&Reg_evt_key=da699401-af65-4ba1-bd27-4563a397cfe1&RegPath=EventRegNoFees&FreeEvent=1&Event=ISPOR%20Webinar:%20Evolving%20RWE%20Standards%20Under%20IRA%20&%20JCA:%20Implications%20for%20Asia%27s%20HTA&FundraisingEvent=0&evt_guest_limit=0" class="button primary" data-sf-ec-immutable="">REGISTER NOW</a></p><p><p=""><strong>Description</strong></p=""> </p><div><div><p paraid="1352163533" paraeid="{6957254d-d418-4c65-8925-da7b6048a683}{141}">The U.S. Inflation Reduction Act (IRA) grants the Centers for Medicare & Medicaid Services (CMS) the authority to directly negotiate drug prices with pharmaceutical companies. This process incorporates drug- and indication-specific real-world evidence (RWE) to assess clinical value and cost-effectiveness for selected innovative therapies that have been on the U.S. market for several years. While this approach marks a paradigm shift in U.S. policy, there is currently no published documentation detailing how CMS has operationalized RWE in its initial pricing negotiations. </p></div><div><p paraid="1490112662" paraeid="{3736cf7e-a7a8-49dc-8f0c-acbe022378a3}{60}">In parallel, the European Union is implementing the Joint Clinical Assessment (JCA) under the new EU HTA Regulation, which aims to harmonize clinical value assessments across Member States. Although the JCA currently focuses on randomized clinical trial evidence, there is growing interest in how RWE—particularly at the indication level—may be incorporated in future evaluations, especially for oncology and advanced therapies. </p></div><div><p paraid="722714471" paraeid="{3736cf7e-a7a8-49dc-8f0c-acbe022378a3}{164}">By contrast, many HTA agencies in Asia continue to assess evidence primarily at the therapeutic-area level at the time of product launch, with limited use of drug- or indication-specific RWE. The growing emphasis on such evidence under both the IRA and JCA frameworks could have significant ripple effects in Asia—potentially influencing the timing, granularity, and methodological expectations for RWE generation and submission. </p></div><div><p paraid="212224371" paraeid="{dc58a326-ec3f-4ebf-bc2b-e1047a776b77}{9}">This webinar will explore how these transatlantic shifts in evidence standards—driven by the IRA and JCA—may reshape HTA practices, pricing negotiations, and reimbursement policies in Asia.</p></div><p paraid="1248192904" paraeid="{0a3627a6-0ed2-4bc2-9669-bfb8dd615211}{150}"><br /></p></div><div><p paraid="314464852" paraeid="{4c2834d2-a80a-4788-8a3a-aadb901ebbc6}{19}"><strong>Learning Objectives</strong></p></div><div><p=""><p=""><ul><li>Understand the emerging use of indication-specific real-world evidence (RWE) under the U.S. IRA and EU JCA frameworks. <br /></li><li>Assess the potential influence of these global standards on HTA policies and reimbursement strategies in Asian countries. <br /></li><li>Compare evolving RWE practices in China, South Korea, and Japan and evaluate regional readiness for adopting indication-level standards. <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></li></ul></p=""> </p=""> </div><div><p><strong>Speakers:</strong></p><p><strong> </strong></p><p><strong>Hong Li, PhD, </strong>Adjunct Associate Professor, University of Cincinnati, Winchester, MA, USA</p><p><strong>Jing Wu, PhD,</strong> Professor, Pharmaceutical Economics and Policy, Tianjin University, Tianjin, China</p><p><strong>Hae Sun Suh, PharmD, PhD,</strong> Professor, College of Pharmacy, Kyung Hee University, Seoul, South Korea</p><p><strong>Daisuke Sato, PhD, </strong>Professor, Fujita Health University Graduate School of Medicine, Toyoake, Japan</p><p> </p><p><br /></p><p><strong>Brought to you by:</strong> <a href="/member-groups/global-groups/consortia/asia-consortium">ISPOR Asia Consortium </a></p></div><p paraid="271259179" paraeid="{8d771561-2af9-432a-af6d-681df4398e6a}{103}"><strong><br />Please note:</strong> On the day of the scheduled webinar, the <strong>first 1000 registered participants</strong> will be accepted into the webinar. For those who are unable to attend, or would like to review the webinar at a later date, the full-length webinar recording will be made available at the <a href="/education-training/webinars" rel="noopener noreferrer" target="_blank">ISPOR Educational Webinar Series webpage</a> approximately 2 days after the scheduled Webinar.</p><div>Reservations are on a first-come, first-served basis.</div>)
Short Courses & Webinars
September 3, 2025
<p><a href="/education-training/webinars">Back to all webinars</a></p><h4>Open to all ISPOR Members and Non-members</h4><p><em><strong><br /></strong></em><strong>Title: </strong>Evolving RWE Standards Under IRA & JCA: Implications for Asia's HTA </p><p><strong>Wednesday, September 3, 2025<br /></strong>8:00AM EDT | 12:00PM UTC | 2:00PM CEST<em></em><a href="https://www.timeanddate.com/worldclock/fixedtime.html?msg=ISPOR+Educational+Webinar%3A+Distributional+Cost-Effectiveness+Analysis+to+Inform+Healthcare+Decisions&iso=20220623T11&p1=12&ah=1" data-sf-ec-immutable=""></a></p><p><a href="https://www.timeanddate.com/worldclock/fixedtime.html?msg=Evolving+RWE+Standards+under+IRA+%26+JCA%3A+Implications+for+Asia%27s+HTA&iso=20250903T08&p1=179&ah=1" data-sf-ec-immutable="">Click here for time zone conversion</a><br /></p><p><a href="https://portal.ispor.org/eweb/DynamicPage.aspx?webcode=EventInfo&Reg_evt_key=da699401-af65-4ba1-bd27-4563a397cfe1&RegPath=EventRegNoFees&FreeEvent=1&Event=ISPOR%20Webinar:%20Evolving%20RWE%20Standards%20Under%20IRA%20&%20JCA:%20Implications%20for%20Asia%27s%20HTA&FundraisingEvent=0&evt_guest_limit=0" class="button primary" data-sf-ec-immutable="">REGISTER NOW</a></p><p><p=""><strong>Description</strong></p=""> </p><div><div><p paraid="1352163533" paraeid="{6957254d-d418-4c65-8925-da7b6048a683}{141}">The U.S. Inflation Reduction Act (IRA) grants the Centers for Medicare & Medicaid Services (CMS) the authority to directly negotiate drug prices with pharmaceutical companies. This process incorporates drug- and indication-specific real-world evidence (RWE) to assess clinical value and cost-effectiveness for selected innovative therapies that have been on the U.S. market for several years. While this approach marks a paradigm shift in U.S. policy, there is currently no published documentation detailing how CMS has operationalized RWE in its initial pricing negotiations. </p></div><div><p paraid="1490112662" paraeid="{3736cf7e-a7a8-49dc-8f0c-acbe022378a3}{60}">In parallel, the European Union is implementing the Joint Clinical Assessment (JCA) under the new EU HTA Regulation, which aims to harmonize clinical value assessments across Member States. Although the JCA currently focuses on randomized clinical trial evidence, there is growing interest in how RWE—particularly at the indication level—may be incorporated in future evaluations, especially for oncology and advanced therapies. </p></div><div><p paraid="722714471" paraeid="{3736cf7e-a7a8-49dc-8f0c-acbe022378a3}{164}">By contrast, many HTA agencies in Asia continue to assess evidence primarily at the therapeutic-area level at the time of product launch, with limited use of drug- or indication-specific RWE. The growing emphasis on such evidence under both the IRA and JCA frameworks could have significant ripple effects in Asia—potentially influencing the timing, granularity, and methodological expectations for RWE generation and submission. </p></div><div><p paraid="212224371" paraeid="{dc58a326-ec3f-4ebf-bc2b-e1047a776b77}{9}">This webinar will explore how these transatlantic shifts in evidence standards—driven by the IRA and JCA—may reshape HTA practices, pricing negotiations, and reimbursement policies in Asia.</p></div><p paraid="1248192904" paraeid="{0a3627a6-0ed2-4bc2-9669-bfb8dd615211}{150}"><br /></p></div><div><p paraid="314464852" paraeid="{4c2834d2-a80a-4788-8a3a-aadb901ebbc6}{19}"><strong>Learning Objectives</strong></p></div><div><p=""><p=""><ul><li>Understand the emerging use of indication-specific real-world evidence (RWE) under the U.S. IRA and EU JCA frameworks. <br /></li><li>Assess the potential influence of these global standards on HTA policies and reimbursement strategies in Asian countries. <br /></li><li>Compare evolving RWE practices in China, South Korea, and Japan and evaluate regional readiness for adopting indication-level standards. <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></li></ul></p=""> </p=""> </div><div><p><strong>Speakers:</strong></p><p><strong> </strong></p><p><strong>Hong Li, PhD, </strong>Adjunct Associate Professor, University of Cincinnati, Winchester, MA, USA</p><p><strong>Jing Wu, PhD,</strong> Professor, Pharmaceutical Economics and Policy, Tianjin University, Tianjin, China</p><p><strong>Hae Sun Suh, PharmD, PhD,</strong> Professor, College of Pharmacy, Kyung Hee University, Seoul, South Korea</p><p><strong>Daisuke Sato, PhD, </strong>Professor, Fujita Health University Graduate School of Medicine, Toyoake, Japan</p><p> </p><p><br /></p><p><strong>Brought to you by:</strong> <a href="/member-groups/global-groups/consortia/asia-consortium">ISPOR Asia Consortium </a></p></div><p paraid="271259179" paraeid="{8d771561-2af9-432a-af6d-681df4398e6a}{103}"><strong><br />Please note:</strong> On the day of the scheduled webinar, the <strong>first 1000 registered participants</strong> will be accepted into the webinar. For those who are unable to attend, or would like to review the webinar at a later date, the full-length webinar recording will be made available at the <a href="/education-training/webinars" rel="noopener noreferrer" target="_blank">ISPOR Educational Webinar Series webpage</a> approximately 2 days after the scheduled Webinar.</p><div>Reservations are on a first-come, first-served basis.</div>)