RWR Insights | What’s in a name? The humble (confusing) non-interventional study and the anti-definition
RWR CONTEXT The FDA definition for non-interventional studies is intuitively actionable. We, as researchers, can (confidently) classify our study based on the parameters provided, which in turn allows us to identify the applicable regulations and guidelines and build our study playbook to describe what we need to do [...]
RWE 201 | A Landscape Analysis of Regional RWE Frameworks – The European Health Data Space and DARWIN-EU
RWR CONTEXT The European Union (EU) is making significant strides in advancing healthcare through a series of interconnected initiatives. The EU4Health Program aims to bolster health systems, focusing on crisis preparedness and disease prevention. The European Health Data Space is an effort to ensure secure access and exchange [...]
RWR Insights | Regulatory Considerations for Non-Interventional Study Protocols
RWR CONTEXT Both RWE and clinical trials play critical roles in healthcare research. While clinical trials provide the highest level of evidence for determining a treatment's efficacy, RWE studies complement this by providing evidence on real-world effectiveness and long-term safety. Real-world evidence (RWE) study protocols and clinical trial [...]
RWR Insights | GDPR and the Secondary Use of Existing Data
RWR CONTEXT GDPR is a facilitator of the secondary use of large healthcare data in the EU. However, there are currently limitations and challenges at a national level due to differences in the interpretation, for example, of the requirements for explicit consent. Further work is needed on issues [...]
RWR Insight | The Difference Between De-Identified and Pseudo-Anonymised Data
RWR CONTEXT A tangible example of how real world evidence (RWE) can be used to support label extensions for existing drugs. Note the FDA’s emphasis on: “This approval reflects how a well-designed, non-interventional study relying on fit-for-purpose real-world data (RWD), when compared with a suitable control, can be [...]
RWR Insights| Real World Evidence (RWE) 101 – Primary Data vs Secondary Data
RWR CONTEXT This is the first in our new RWE 101 series in which we explore and explain the fundamentals of real world evidence, specifically the differences, advantages, disadvantages and limitations of primary data versus secondary data. Primary data and secondary data are two types of [...]
RWR Insights | USA – Lessons Learned from FDA Reviews of External Control Arms
RWR CONTEXT In February 2023, the FDA published a draft guidance document on considerations for the design and conduct of externally controlled trials and external control arms. The aim being to help address and prevent the common mistakes and methodological design flaws that limit the acceptability of external [...]
EU | EMA 2025 Vision for the Regulatory Use of RWE – Connecting the Pieces
RWR CONTEXT In the past 12 months, the EMA has been busy…publishing strategies and frameworks, creating coordination centres, and requesting proposals from training vendors! The question is…how does this all fit together? Here we provide a a high-level overview of what has happened and what role each of [...]
RWR Insights | Using RWE to Support Regulatory Decisions – Evidence Considerations and Opportunities
RWR CONTEXT The healthcare real world evidence (RWE) regulatory framework has rapidly evolved over the past few years (see below), such that RWE is increasingly being used to support and inform the clinical development of medicinal products. Despite this rapid evolution, the regulatory acceptance of RWE is limited, [...]
RWR Insights | EU – Applicability of GVPs to the Conduct of Non-Interventional Studies
RWR CONTEXT The EMAs Good Pharmacovigilance Practice guidelines (GVPs) supplement the requirements of Directive 2001/83/EC (Article 107m – 107q) for non-interventional studies and provide additional detailed guidance on (1) the classification of non-interventional post-authorisation safety studies (PASS) (GVP Module V), (2) guidance on the design, registration, conduct and [...]
RWR Insights – What GxPs are Applicable to Non-Interventional (Observational) Studies?
RWR CONTEXT In this series of ‘RWR Insights’ we lay out what GxPs are globally applicable to observational studies with the aim of providing you confidence that you know what is applicable and what you need to consider and/or comply with. With knowledge comes understanding…with understanding comes confidence… [...]
RWR Insights | Quality Standards for Registries – EUnetHTA REQUEST Tool
RWR CONTEXT This is the third (3rd) and last in the series exploring current quality standards for registries and registry-based studies. This month we focus on quality standard expectations when using real world data (RWD) from registries to support Health Technology Assessment. Specifically, we’ll look in detail at [...]
Quality Considerations when Using RWD from Registries to Support Regulatory Decisions in the EU
RWR CONTEXT EMA has published a comprehensive guideline, which provides recommendations on key methodological aspects that are specific to the use of patient registries when planning to conduct registry-based studies to support regulatory decision making on medicinal products within the European Union (EU). In October 2021, [...]
RWR Insights | Quality Considerations when Using RWD from Registries to Support Regulatory Decisions – USA
RWR CONTEXT Study sponsors should ensure they have documented policies and procedures in place that enable them to address these FDA recommendations, so that they can systematically assess and use (appropriate quality) registry data as a source of real world data (RWD) to support their drug development strategies, [...]
RWR Insights | USA – Overview of the Current (draft) FDA Guidance and Considerations for Sponsors who are Intending to Use Non-Interventional Study Data to Support Regulatory Submissions to the FDA GalleryRWR Insights | USA – Overview of the Current (draft) FDA Guidance and Considerations for Sponsors who are Intending to Use Non-Interventional Study Data to Support Regulatory Submissions to the FDA
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RWR Insights | USA – Overview of the Current (draft) FDA Guidance and Considerations for Sponsors who are Intending to Use Non-Interventional Study Data to Support Regulatory Submissions to the FDA
RWR CONTEXT We have created a consolidated version of these three (3) FDA Guidance documents to help provide an overview of the current (draft) FDA guidance and considerations for sponsors who are intending to use non-interventional study data to support regulatory submissions to the FDA. There is a [...]
Insight | 2021 End of Year Summary
RWR CONTEXT A tangible example of how real world evidence (RWE) can be used to support label extensions for existing drugs. Note the FDA’s emphasis on: “This approval reflects how a well-designed, non-interventional study relying on fit-for-purpose real-world data (RWD), when compared with a suitable control, can be [...]
Insights | Patient Centricity and the Patient Voice
RWR CONTEXT The “patient voice” is recognised as an essential asset in the development of medicines that matter to patients. In the context of the design and conduct of NIS, the solution is to listen to the viewpoint of patients and caregivers’ by using patient advocacy groups, [...]
Insights – Document Management and Archiving Requirements for Observational Studies
RWR CONTEXT The TMF Reference Model’s specially designed Study Master File will not have an impact on the regulatory requirements for conducting real world research studies in the country of interest…but if used…it will make your real world research studies much easier to manage and less resource intensive [...]
Insights | Bulgaria – ‘How To’ Guide for Non-Interventional Studies
RWR CONTEXT Summary of the requirements for conducting prospective non-interventional studies (NIS) in Bulgaria. Includes the updates implemented by the recent NIS Ordinance (Ordinance № 8 of March 31, 2021). June 2021 - Our three (3) step ‘How To’ guide for conducting prospective voluntary NIS in [...]
Insights | Real World Research in Mental Health
RWR CONTEXT RWR, when done properly, is a low-cost / high-quality alternative to traditional research. Improving mental health across the board needs to focus not just on pharmaceutical interventions. Strengthening the implementation of research evidence and good practice into a range of settings where people seek support is [...]
Insights | EU – Quality Standards for Registry Studies
RWR CONTEXT “Quality RWE can’t be Built without Quality RWD” EMA checklist for evaluating the suitability of registries for registry-based studies = Data quality guideline addressing quality expectations of real world data sources (i.e., disease registries) used to generate real world evidence (RWE) for regulatory purposes [...]
Good Clinical Practice for Medical Device Clinical Trials (ISO 14155:2020) – Applicability to Non-Interventional Studies
RWR CONTEXT The take home point for post-market observational (non-interventional) clinical investigations of medical devices is that you should assume that all elements of GCP (ISO 14155:2020) are applicable unless you justify why they are not. This is worlds apart/ completely different to non-interventional studies of medicinal products, [...]
