Sep 1, 2007, 00:00 AM

10.1111/j.1524-4733.2007.00236.x

https://www.valueinhealthjournal.com/article/S1098-3015(10)60467-6/fulltext

Section Title :

Section Order : 13

First Page :

The report of the ISPOR Real World Data Task Force [1] is very timely, in that a number of reimbursement bodies worldwide are now expressing a need for “real-world” data. With the exception of safety data, the requirements to collect such data are rarely mandatory, so discussion needs to take place about precisely what data are required and who will collect them.

The Task Force report makes a number of important contributions. First, it provides some clear definitions of what constitutes “real-world data” and the difference between “data” and “evidence.” Second, it contains helpful characterizations of real-world data, by type of outcome (e.g., clinical, economic, patient-reported outcomes), by hierarchy of evidence (e.g., randomized controlled trial, observational study), and by source of data (e.g., supplements to trials, registries). Third, it makes a sound intellectual case for the need for real-world data (see the section of the report on “Recognizing the importance of RW data”).

Nevertheless, as the report progresses, it becomes clear that several methodological and practical questions need to be answered before we can make much progress in the collection and use of real-world data. Two statements in the report point to this need. First, in the section on “The level of evidence required depends on the circumstance,” the authors state that “Decisions typically rely on multiple sources, and are best thought of as conditional––to be revisited as additional evidence is generated.” Then, in the “Conclusions,” they state that “It is critical that policy makers recognize the benefits, limitations, and methodological challenges in using RW data, and the need to carefully consider the costs and benefits of different forms of data collection in different situations.” The danger, in my view, is that these issues will not be adequately debated. Either a requirement for real-world data will be added (without much thought) to the existing list of data requirements, or there will remain a lack of guidance on which data to collect in a given situation.

Therefore, having made the intellectual case for the collection of real-world data, there is an urgent need for a discussion of some of the detail. First, regarding methods, which types of data can be considered acceptable forms of evidence for answering a particular question? For example, can the comparative effectiveness of therapies only be determined through an RCT, or can it be determined through an observational study, if accompanied by an appropriate analytic strategy? Second, regarding the practical issues, how can appropriate incentives be given for the collection of real-world data? Are there occasions where it should be made mandatory (beyond the collection of safety data)? Alternatively, can incentives be provided by a commitment, by decision-makers, to revisit certain decisions if certain categories of data are collected? Would the collection of real-world data be best integrated with formal risk-sharing schemes, where these exist?

In my view, the collection of real-world data represents an important way forward. However, for it to proceed in a comprehensive fashion, we need a reality check on several important methodological and practical issues. Detailed methodological guidance for the collection of real-world data needs to be produced, to mirror that we have for modeling studies and “piggyback” economic evaluations undertaken alongside clinical trials. Also, reimbursement agencies need to engage in more discussions about the types of data required and the decisions that will result.

Reference
1 Garrison LP, Neumann PJ, Erickson P, et al. Using real-world data for coverage and payment decisions: the ISPOR real-world data task force report. Value Health 2007; DOI 10.1111/j.1524-4733.2007.00186.x.

https://www.valueinhealthjournal.com/action/showCitFormats?pii=S1098-3015(10)60467-6&doi=10.1111/j.1524-4733.2007.00236.x

HEOR Topics :

• Decision & Deliberative Processes
• Health Policy & Regulatory
• Health Technology Assessment
• Reimbursement & Access Policy

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Regions :

• Global