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.
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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 7, 2023
<a href="/education-training/short-courses">Back to all short courses</a><br /> <br /> <strong>Developing Decision-Grade Real-World Evidence</strong> <p><strong><br /> LEVEL: </strong>Intermediate<strong><br /> TRACK: </strong>Real World Data & Information Systems <strong><br /> LENGTH: </strong>4<strong> </strong>Hours | Course runs 1 day<br /> <br /> This short course is offered in-person at the ISPOR 2023 conference. Separate registration is required. <a target="_blank" href="/conferences-education/conferences/past-conferences/ispor-2023/about/registration-information">Visit the ISPOR 2023 website to register</a> and learn more.</p> <strong><strong>Sunday, 7 May 2023 | Course runs 1 Day<br /> </strong></strong>8:00AM-12:00PM Eastern Daylight Time (EDT) <br /> <h5><strong><br /> DESCRIPTION</strong></h5> <p>In this course, participants are guided through a hands-on analysis of real-world data to develop decision-grade real-world evidence (RWE) that could be used to support an indication expansion. The first section of the course focuses on what makes RWE “decision-grade.” A review is conducted of the most recent RWE frameworks and guidelines set by regulatory agencies and professional organizations, and case studies are examined in which these guidelines were used in regulatory and HTA approval. The second half of the course is an active workshop where participants use principles from the first half of the course to execute a decision-grade RWE study. Participants are guided step-by-step in using a software platform that enables them to work within a longitudinal US insurance claims database with anonymized patients. After the study has been executed, a discussion on how these results are communicated to decision makers is held. Participants should come with a laptop with Google ChromeTM installed. <strong><em>PREREQUISITE: Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data, but this course does not require specific programming skills.</em></strong> </p> <p>Registrants receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.</p> <p><strong>FACULTY MEMBERS</strong></p> <strong></strong> <div><strong></strong></div> <div><strong></strong></div> <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 and Epidemiology, Harvard Medical School </div> <div>and Chief, Division of Pharmacoepidemiology </div> <div>Brigham and Women’s Hospital</div> <div>Boston, MA, USA</div> <div> </div> <div><strong>Jeremy A. Rassen, ScD</strong></div> <div>Co-Founder & Chief Scientific Officer</div> <div>Aetion, Inc.</div> <div>New York, NY, USA<br /> <br /> <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> <div> </div> <p><strong>Basic Schedule:<br /> </strong>4 Hours | Course runs 1 Day</p>)
Short Courses & Webinars
May 7, 2023
<a href="/education-training/short-courses">Back to all short courses</a><br /> <br /> <strong>Developing Decision-Grade Real-World Evidence</strong> <p><strong><br /> LEVEL: </strong>Intermediate<strong><br /> TRACK: </strong>Real World Data & Information Systems <strong><br /> LENGTH: </strong>4<strong> </strong>Hours | Course runs 1 day<br /> <br /> This short course is offered in-person at the ISPOR 2023 conference. Separate registration is required. <a target="_blank" href="/conferences-education/conferences/past-conferences/ispor-2023/about/registration-information">Visit the ISPOR 2023 website to register</a> and learn more.</p> <strong><strong>Sunday, 7 May 2023 | Course runs 1 Day<br /> </strong></strong>8:00AM-12:00PM Eastern Daylight Time (EDT) <br /> <h5><strong><br /> DESCRIPTION</strong></h5> <p>In this course, participants are guided through a hands-on analysis of real-world data to develop decision-grade real-world evidence (RWE) that could be used to support an indication expansion. The first section of the course focuses on what makes RWE “decision-grade.” A review is conducted of the most recent RWE frameworks and guidelines set by regulatory agencies and professional organizations, and case studies are examined in which these guidelines were used in regulatory and HTA approval. The second half of the course is an active workshop where participants use principles from the first half of the course to execute a decision-grade RWE study. Participants are guided step-by-step in using a software platform that enables them to work within a longitudinal US insurance claims database with anonymized patients. After the study has been executed, a discussion on how these results are communicated to decision makers is held. Participants should come with a laptop with Google ChromeTM installed. <strong><em>PREREQUISITE: Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data, but this course does not require specific programming skills.</em></strong> </p> <p>Registrants receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.</p> <p><strong>FACULTY MEMBERS</strong></p> <strong></strong> <div><strong></strong></div> <div><strong></strong></div> <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 and Epidemiology, Harvard Medical School </div> <div>and Chief, Division of Pharmacoepidemiology </div> <div>Brigham and Women’s Hospital</div> <div>Boston, MA, USA</div> <div> </div> <div><strong>Jeremy A. Rassen, ScD</strong></div> <div>Co-Founder & Chief Scientific Officer</div> <div>Aetion, Inc.</div> <div>New York, NY, USA<br /> <br /> <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> <div> </div> <p><strong>Basic Schedule:<br /> </strong>4 Hours | Course runs 1 Day</p>)