Table 1.

Research agenda for the identification of patient phenotypes

Identify published randomized clinical trials and observational cohorts assessing the efficacy of different class of interventions on clinically relevant outcomes, divided in structural and symptomatic outcomes

Using above data, produce clinical prediction tools to quantify a patient’s risk of progression and good outcomes from treatment interventions

With available data, identify phenotypes of patients according to their outcome. Panel of (bio)markers (clinical, biochemical, imaging) should be investigated, rather than individual items

Assess the uniformity of data across clinical trials and cohorts

Proceed to a validation step on a separate validation cohort

Ensure that all new cohorts and trials use the same core dataset to allow easy integration into extant data

Possible limitations:

The availability of the data and of the biological specimens in cohorts

The high heterogeneity in the assessments methods and reporting (e.g., multiple assessment tools for pain), which would require a hierarchical/standardization of criteria