Clearly, the costs of clinical trials and real-world studies should not be directly compared; each study type has a different purpose and distinct requirements, and will therefore have varying associated costs. The important point here is that, for a sponsor investing tens of millions in a clinical trial for a new therapeutic agent, it may be wise to consider the additional, smaller, investment in a real-world study. This could support clinical trial data, address possible gaps in the product’s evidence-base, and improve the likelihood of a positive return on investment by way of successful market authorization, pricing and reimbursement decisions, and uptake.

What drives the cost of a real-world study? 

In simple terms, the cost of a real-world study is determined by its size. Increasing the size of any of the following elements will add to the cost. 

Number of countries

For each country, study materials must be translated and localized. The study design and implementation may also require customization to account for differences in healthcare systems, cultural norms, and ethical and data protection requirements. Multiple ethics submissions must be made. In some circumstances, it is also necessary to employ an in-country study representative or a local expert to help navigate regulations and processes.  

Number of sites

For studies that rely on healthcare sites for recruitment and/or to contribute data, there are costs associated with identifying, selecting, training, and managing each site, as well as fees to compensate sites for their time.

Number of participants

A larger study population increases costs in several ways: chiefly, recruitment costs, participant compensation costs, and those associated with data handling, storage, and analysis.

Amount of data collected

The more variables that are collected about each participant, the more work that is required to research, select, or develop measurement tools, and adapt them where necessary to their final format, such as for a study website or app. If data is to be collected and linked from multiple sources – for example, a patient and their clinician – this increases complexity. Costs associated with engaging and compensating participants also increase when more data input is required from them, especially if data collection is longitudinal. A greater amount of data about each participant will also lead to more work on analysis and reporting.  

Duration of follow-up

Studies with a longer duration of follow-up do cost more. However, costs tend to reach their peak in the period leading up to study launch, before stabilizing. Costs for multi-year studies are also split by the year in which they are incurred, thus spreading the cost and providing opportunities to check in on study progress before committing to additional years of follow-up.

 If you would like to understand more about the potential cost of a study that you have in mind, please complete the form below to receive a “Study Concept”. We will provide a high-level summary of the possible study design, with accompanying ballpark costs, which we can then discuss and refine with you before moving forward to a detailed proposal if requested.

By Emma Bagshaw

References

1. Moore TJ et al. Variation in the estimated costs of pivotal clinical benefit trials supporting the US approval of new therapeutic agents, 2015-2017: a cross-sectional study. BMJ Open. 2020;10(6):e038863