Fit-for-Purpose RWD: An Integral Part of Evidence Planning

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...

Value in Health 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.

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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.

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Go To The Registry

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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.

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Additional Resources

Real-World Evidence Registry
Good Practices Reports and Other ISPOR Reports from Value in Health
"Unlocking the Promise of Real-World Evidence" (Value & Outcomes Spotlight, Vol. 6, No. 5)
ISPOR's Real-World Evidence Strategic Initiatives

Conferences & Summits

Fit-for-Purpose RWD: An Integral Part of Evidence Planning

September 9, 2025

<a href="/education-training/webinars">Back to all webinars</a>Title: Fit-for-Purpose RWD: An Integral Part of Evidence Planning Tuesday, September 9, 2025  11:00AM EDT | 5:00PM UTC | 7:00PM CEST<a href="https://www.timeanddate.com/worldclock/fixedtime.html?msg=Fit-for-Purpose+RWD%3A+An+Integral+Part+of+Evidence+Planning&iso=20250909T11&p1=179&ah=1" data-sf-ec-immutable="">Click here for time zone conversion</a><a href=" https://portal.ispor.org/eweb/DynamicPage.aspx?webcode=EventInfo&Reg_evt_key=f3f998ad-0d28-467f-8962-9e903ea9c9f9&RegPath=EventRegNoFees&FreeEvent=1&Event=ISPOR%20Webinar:%20Fit-for-Purpose%20RWD:%20An%20Integral%20Part%20of%20Evidence%20Planning&FundraisingEvent=0&evt_guest_limit=0" class="button primary" data-sf-ec-immutable="">Register Now</a> Description Evidence planners across the life sciences industry—including Integrated Evidence Planning teams, HEOR and Market Access teams, Clinical Development, Epidemiology and Safety, and Medical Affairs—are increasingly adopting Integrated Evidence Planning (IEP) to address the diverse and evolving evidence requirements of multiple stakeholders throughout the asset lifecycle. Real-world evidence (RWE) is one of the most critical components of the IEP, aimed at demonstrating the safety, effectiveness, and value of pharmaceutical/biotech products beyond traditional clinical trials. At the same time, there has been an evolution and emergence of new data sources globally, encompassing various types of data sources, i.e., patient registries, chart abstraction, patient surveys, electronic health records, claims (open and closed), billing, lab, genomic and molecular data, and patient-generated data from wearables and mobile apps, plus any combination thereof. Although this data has greatly expanded our ability to generate RWE, navigating this continuously evolving landscape and ensuring selection of the appropriate real-world data (RWD) sources to address the research objective during the evidence planning phase is becoming more complex.  This webinar will explore the expanding portfolio of RWD sources across a spectrum of landscapes, including US (advanced landscape), Europe (evolving landscape) and other countries (emerging landscapes). We will discuss some of the challenges with planning and designing RWE studies and aim to provide a guiding framework to develop study concepts with a high probability of success. A key focus will be on practical strategies for selecting the right data to meet specific research needs, whether it is to evaluate safety, understand healthcare utilization or assess treatment adherence. We will also address critical considerations regarding data quality, completeness, representativeness, and regulatory compliance that impact the integrity and usability of the data to ensure successful implementation of RWE generation. Learning Objectives<ul type="disc"><li>Understand the evolving RWE landscape and key data sources</li><li>Assess ‘fit-for-purpose’ RWD</li><li>Examine common limitations of using RWD and ways to solve them</li></ul> Moderator:Dana Stafkey, PharmD, PhD, Vice President, Real-World Evidence, Cencora, Raleigh-Durham, NC, USASpeakers:Tasneem Lokhandwala, PhD, Senior Director, Real-World Evidence, Cencora, Key Largo, FL, USAHenri Leleu, MD, MPH, PhD, Scientific Director, Public Health Expertise, Paris, France  Sponsored by Corporate Partner, <a href="https://www.cencora.com/our-capabilities/commercialization-support" data-sf-ec-immutable="">Cencora</a><img sf-image-responsive="true" src="/images/default-source/cti-meeting-21021-images/a464-1-png.png?sfvrsn=153a0f15_0" height="149" style="max-width:100%;" title="a464_1.png" width="163" alt="" sf-size="300568" />Please note: On the day of the scheduled webinar, the first 1000 registered participants 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">ISPOR Educational Webinar Series webpage</a> approximately 2 days after the scheduled Webinar. Reservations are on a first-come, first-served basis.)

Short Courses & Webinars

Fit-for-Purpose RWD: An Integral Part of Evidence Planning

Real-World Evidence Transparency Initiative

A partnership between ISPOR, the International Society for Pharmacoepidemiology, the Duke-Margolis Center for Health Policy, and the National Pharmaceutical Council

Establishing a Culture of Transparency for Real-World Evidence Studies...

Improving Transparency to Build Trust...

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Real-World Evidence Registry

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Shaking the Myth of Real-World Evidence

Additional Resources

Conferences & Summits

September 9, 2025

Short Courses & Webinars

September 9, 2025

ISPOR–The Professional Society for
Health Economics and Outcomes Research

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