How patients’ input can help to select the right PROs

Using Patient and Public Input (PPI) to select patient-reported outcomes

Trial results can rest on the appropriate selection of PROs, that are both sensitive enough to detect meaningful change yet targeted enough to limit participant and site burden. Patient and Public Input (PPI) can be an opportunity to assure the effective selection of PROs while developing a protocol through scoping burden of disease with a selected sample of patients across important variables, such as disease severity or prior treatment experiences. Initial Patient and Public Input (PPI) ensures robustness, feasibility, and patient-centricity of a trial design.

Initial PPI can particularly benefit trial planning with rare diseases. In one instance, through PPI with a rare disease, Clinigma® qualitative research revealed that the proposed PRO for assessing pain was inappropriate for the primary trial outcomes. Rather than varying on a daily basis, patient insight resoundingly detailed the disease and treatment cycle rather than daily variations as determinative of a meaningful treatment impact. As such, the proposed, widely used PRO for pain would lack the sensitivity to quantitatively support treatment impact and, as with a rare disease, the sample size would preclude subgroup analyses that attend to potential cyclical variation without the specificity identified in initial PPI work. With this initial PPI work, the protocol can utilize another pain PRO with a report of patient voice to support the selection, supplement the sensitivity with qualitative interviews, and propose a mixed-methods data analysis plan using both trial and qualitative outcomes.

Clinigma® has the experience and agility to conduct and complete initial PPI projects informing the selection of PROs that can demonstrate the appropriate selection in accord with FDA Patient-Focused Drug Development Guidance 3, “Selecting, Developing, and Modifying Fit-For-Purpose Clinical Outcomes Assessments.” Additionally, Clinigma® help clients to develop and validate fit-for-purpose instruments when needed through content validity efforts like cognitive interviews, literature reviews, and expert consultation as appropriate along with mixed-methods analysis supporting the validation of instruments with patient voice.

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