Prospective Observational Studies: Whether, When and How to Use

Prof Adrian Towse Prof Towse


OHE’s Adrian Towse is co-author on the recent report by the ISPOR Good Research Practices Task Force on assessing comparative effectiveness using prospective observational studies.

The demand for comparative data, and in real-world conditions, will only increase as both public and private payers seek to ensure that the use of medical technologies is as cost-effective as possible.  At the same time, however, timely access to new treatments  remains crucial.  For a number of very practical reasons, it is infeasible to rely primarily on randomised clinical trails (RCTs) for comparative effectiveness data.  This clearly is recognised by policy makers in many arrangements for “coverage with evidence development” or “risk sharing”, for example. Moreover, as the report notes, “Generating evidence about new pharmaceuticals, including biological entities, is increasingly being seen as an activity that occurs throughout  the entire product life cycle, rather than pre-launch for a one-off ‘at-launch’ review”.

Prospective observational studies as an alternative to RCTs, post-launch, are the primary focus of this ISPOR Task Force Report.  It examines in depth whether, when and how this approach reasonably may be used.  A detailed recounting of the critical issues involved in designing, conducting and reporting such research is accompanied by solid recommendations for addressing them.

The report is available for download from the ISPOR website.

Berger, M.L., Dreyer, N., Anderson, F., Towse, A., Sedrakyan, A. and Normand, S-L. (2012) Prospective observational studies to assess comparative effectiveness:  The ISPOR Good Research Practices Task Force report. Value in Health. 15, 217-230. Available at [Accessed 15 June 2012].

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