Pragmatic Studies for Real World Evidence – Lifting the veils

Regulators, Health Technology Assessment (HTA) agencies and health care payers are increasingly demanding Real World Evidence (RWE) to support both initial product approval/reimbursement as well as continuing approval/reimbursement over a product’s life cycle.

Clinical trials are frequently conducted to demonstrate safety and efficacy, Non-Interventional Studies (NIS), patient and product registries to generate RWE, but increasingly the industry is coming across more ‘pragmatic studies’. So, why is interest growing in discussion around this ‘new’ type of study?

RWE is a broad area including clinical, economic, and patient reported outcomes, epidemiological and comparative setups, and various external stakeholders as well as the manufacturer’s internal organization. Within the RWE area, comparing products’ performance in the real world has been receiving special attention: Relative or comparative effectiveness and benefit/risk are key words in today’s conferences and communication.

HTA agencies specifically look into comparative RWE: Observational methods cannot fully eliminate allocation bias, as treatments are assigned at the discretion of the treating physician. Agencies thus look to combine real world data collection with control of treatment allocation bias, preferably by study designs including subject based randomization i.e., pragmatic studies. Although the term ‘pragmatic’ has been used increasingly for such real life, randomized studies, it is not fully clear what pragmatic studies are, and for which purposes they should be performed: There are veils to lift around pragmatic studies, strategically as well as operationally.

This webinar describes and explains pragmatic studies and discusses why key Late Stage stakeholders are increasingly interested in pragmatic studies – and why, nevertheless, pragmatic approaches are still relatively rare in the area of RWE. The panel outlines operational modifications and adaptations of the main pragmatic study approach, such as community based studies, cluster randomized approaches, and ‘stepped wedge’ designs.

The panel also discusses both current and future regulatory considerations such as the new EU Clinical Trials Regulation. An outlook into the future of pragmatic study types will be provided, and strategic options for potential design modifications will be assessed. We will close our evaluation of pragmatic studies and their nuances by assessing evidence validity from pragmatic approaches with others, primarily analytic methods minimizing bias in observational settings.

Learning Objectives

• What do we mean by ‘Real World Evidence’?
• Understand RWE needs of Late Stage stakeholders
• Understand why stakeholders are asking for pragmatic approaches and why they are still rarely performed
• Understand internal / external validity and efficacy vs. effectiveness
• Discuss nuances of pragmatic approaches such as cluster randomization, community based designs, or stepped wedge approaches
• Learn about the regulatory considerations when planning and conducting pragmatic studies
• Understand analytic considerations for evidence validity in pragmatic designs, versus non-interventional approaches using analytic techniques such as regression methods, propensity score matching, instrumental variables.

Speakers

Stuart McCully, PhD,,Vice President, Late Stage Support Services, inVentiv Health

Stuart McCully is an internationally recognized expert on NIS practices and country-specific submission processes, and the founder and owner of CHCUK Ltd, an on-line service specializing in non- interventional studies and clinical research compliance. Stuart has more than 15 years in biomedical research regulatory compliance and is responsible for regulatory compliance, training, standard operating procedures (SOPs), and metric reporting. He manages a team of non-interventional study regulatory specialists. Stuart has a Degree and PhD in human mechanistic pharmacology and toxicology.

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Florian Eichmann, PhD, Principal, Scientific Affairs and Real World Evidence, Late Stage, inVentiv Health

Florian is based in Germany and consults on Late Stage and RWE topics such as defining the optimal study design for a research purpose, aligning operational strategy with the selected study design, and ensuring outcome validity.
He has more than 20 years of experience in the conduct and management of Late Stage studies ranging from Phase IIIb/IV clinical trials to preference/opinion based studies. His focus has been the design and implementation of retrospective and prospective non-interventional studies for Access, Health Outcomes and Risk Management.
Florian holds a Master’s and Doctoral degree in Biology specializing in Behaviour, Endocrinology and Immunology.

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