Determining the best methods for using patient registry data in clinical research / Daniel Solomon, Nancy Ann Shadick, Elisabetta Patorno.

Format:

Book

Author/Creator:

Solomon, Daniel, author.

Shadick, Nancy Ann, author.

Patorno, Elisabetta, author.{del}.

Language:

English

Subjects (All):

Patient Reported Outcome Measures.

Physical Description:

1 online resource (59 pages)

Place of Publication:

Washington, DC : Patient-Centered Outcomes Research Institute, 2019.

Summary:

BACKGROUND: Relatively little is known about the best methods for collecting and analyzing patient-reported outcomes (PROs) in clinical registries. This gap in knowledge is especially important for disease activity measures that may not be assessed contemporaneously with changes in interventions. OBJECTIVES: This series of studies addressed several issues or objectives: (1) how patients want to be involved in clinical registries and their willingness to complete frequent questionnaires (objective 1); (2) the potential effects of different methods for analyzing disease activity reported by patients at time points other than at the time of a change in intervention (objective 2); and (3) how these methods would affect a specific comparative safety example (risk of infection) when considering rheumatoid arthritis and the initiation of different biologic disease-modifying antirheumatic drugs (DMARDs; objective 3). The overarching methodologic objective is to determine the least-biased methods for estimating sparsely measured PROs in registry analyses. METHODS: The designs differed by objective. We accomplished objective 1 through both focus groups and surveys of patients with chronic diseases who did and did not participate in clinical registries. We accomplished objective 2 through simulation studies in which representative data were generated that facilitated testing different assumptions regarding when patient-reported outcomes were measured in relationship to intervention changes. We accomplished objective 3 using actual clinical registry data from a rheumatoid arthritis--the most common autoimmune arthritis--registry. We estimated the relative risk of infection for a commonly used type of biologic DMARD, tumor necrosis factor inhibitors (TNFis), compared with non-TNFi biologic DMARDs, using Cox proportional hazards regression. RESULTS: Objective 1 found that motivating factors for participating in clinical registries include altruism, provider recommendations, and ease of inclusion. Hispanic patients had many concerns and suspicions that did not overlap with other participants. The survey of participants found that many different preferred frequencies and types of contact exist; older patients prefer face-to-face or paper, while younger patients are more comfortable with frequent electronic communication. Objective 2 found that using PROs assessments before or after the start of an intervention was acceptable as long as they were conducted near the initiation date. Restricting analyses to subgroups of patients with measurement in specific time windows introduced substantial variation in results. Objective 3 found that the risk of infection was similar across TNFis and non-TNF biologic DMARDs and did not vary substantially based on the methods used to analyze the potential confounder patient-reported disease activity. CONCLUSIONS: The conclusions differed by objective. Objective 1 studies suggested that appealing to altruism and ease of participation would be effective measures for registry recruitment. However, there is a clear need to educate Hispanic patients about participation in clinical registries. As well, participation protocols should be flexible, allowing some degree of contact tailoring--face-to-face, telephone, written, electronic--based on each participant's preference. Objective 2 demonstrated the acceptability of using PROs measured proximally but after the start of an intervention as confounder adjustment. Using a variety of assumptions, objective 3 tested the findings from objective 2 and found a similar infection risk across different types of biologic DMARDs used for rheumatoid arthritis. LIMITATIONS AND SUBPOPULATION CONSIDERATIONS: The research is limited by the sample size of the focus groups and surveys as well as the example used with the rheumatoid arthritis registry. While we believe that the samples were representative, different diseases may produce different results. Also, it would be worthwhile to continue to test the acceptability of patient registries among other racial and ethnic groups.

Notes:

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