The Food and Drug Administration (FDA) wants drugs to be more targeted to factors that affect a patient’s life in the ways they care for them. To achieve this goal, the Food and Drug Administration last month released a file Third Guidance Document on Patient Centered Drug Development It generally applies to a variety of clinical outcome assessments (COAs), including patient-reported outcomes (PRO), observer-reported outcomes (ObsRO), clinician-reported outcomes (ClinRO), and performance-based outcomes (PerfO). ).
- Patient Report Results (PRO). Reports come directly from the patient. These measures are useful for assessing symptoms (eg, pain intensity, shortness of breath), performance, events, or other aspects of health from the patient’s perspective. Oftentimes performance pros are collected through questionnaires, but increasingly they are being collected using digital health technologies (DHTs).
- Observer Reported Results (ObsRO). Reports come from someone other than the patient or a health professional (eg, a parent or caregiver) who has an opportunity to observe the patient in daily life. These measures are used when patients such as young children cannot reliably report themselves, or to assess observable aspects of patients’ health (eg, signs, events, or behaviors).
- Doctor Reported Results (ClinRO). Reports come from a trained healthcare professional using clinical judgment. This type of scale is useful when reports of signs, behaviours, clinical events, or other manifestations of an observable disease or condition benefit from clinical judgment or experience.
- Performance Result (PerfO). These measures are based on a standardized task(s) that the patient is actively performing. Examples include a grip strength test or a six-minute walk (6MWD).
The US Food and Drug Administration (FDA) recommends creating a conceptual model to represent how patients’ specific health experiences of their disease/condition relate to measures of interest. An example is below.
The context of how the measures are used is also important. especially:
- Use of COA: Clinical trial goals and how COA will be used to support COA-based endpoints (eg, calculating the average COA score at 12 weeks)
- Target population: including the definition of a disease or condition; Criteria for selecting participants for clinical trials (eg, severity of core symptoms, patient demographics, comorbidities); and patient experiences or anticipated events during the trial (for example, that some patients will need assistive devices)
- Study context: The design of the clinical trials in which COA will be used, including the type of comparison group and whether those providing responses or participating in COA tasks (patients, observers, clinicians, and trained assessors) are masked with respect to treatment allocation and/or study visit)
- timing When are COA evaluation(s) 269 . to be conducted?
- COA implementation: Including COA collection site (eg, inpatient, outpatient, home); How the COA will be collected (eg, DHT, paper form); It (eg, patient, study coordinator, investigator, parent/caregiver.)
The FDA also provides a roadmap for selecting a fit-for-purpose COA for use in a clinical trial.
Once the roadmap is followed, manufacturers need to justify the inclusion of COA in a clinical trial based on 8 key points of evidence.
For more information on the specific roadmap and evidence needed to justify inclusion of a COA in a clinical trial, do the full FDA guidelines. over here.
