Patient-Reported Outcome (PRO)
Clinical Trials
“What matters is this – will it help patients?” ~ Bill Cook
Introduction
To ensure that patients stay at the center of clinical research, patients’ perspectives should be captured. Patient-reported outcome (PRO, or ePRO if electronic) data collected from patient-reported outcomes measures (PROM) are reported directly from the patient without interpretation of the patient’s response by a healthcare provider or anyone else. PROs are a subset of clinical outcome assessments (COA or eCOA if electronic). A COA can be completed by anyone (e.g., site staff, caregivers, patients) whereas PROs are completed only by the patient.
FDA encourages the collection of PROs. Over 75% of FDA investigational device exemptions in 2017 included a PRO (reference: FDA Newsroom). An FDA guidance describes how the agency reviews and evaluates PRO data to support labeling claims.
Types of PROs
Diaries and questionnaires are the most common types of PROs. When using PRO data to support a labeling claim, FDA reviews the PRO instrument’s measurement properties including reliability, validity, and ability to detect change.
The validation can be based on exact wording of the question, the order of questions, and mode of administration (e.g., electronic, verbal, written). For example, the Quality of Life Scale consists of a 7-point scale ranging from “delighted” to “terrible”. Since these words may not translate well into other languages, translations for each language have been validated separately.
Data Collection Methods: Paper vs Electronic
The data collection method should fit the needs of the study, site, and patients. The table below lists some considerations in choosing paper PRO or ePRO. The Society for Clinical Data Management’s Good Clinical Data Management Practices further describes considerations of each approach
| Consideration | Paper | Electronic |
|---|---|---|
| Cost | Pro: Printing and mailing costs are less expensive. | Con: ePRO modules in EDC systems often have additional fees. |
| Con: More personnel time may be needed to enter into a database. | Pro: Data are automatically integrated in EDC in real time. | |
| Accessibility | Con: Paper forms may not be feasible for larger studies. | Pro: Good for large-scale studies. |
| Pro: Preferred by patient populations not familiar with or who distrust digital technology. | Pro: Easier for people with motor or reading disabilities. Con: Rural populations may not have an adequate internet signal. |
|
| Pro: Little to no training required. | Con: Requires training and technical support. | |
| Data Quality | Con: If patients forget to complete a paper form, they might fill it out retrospectively, which can lead to recall bias. | Pro: electronic methods offer an automatic audit trail of completion time. Pro: Some systems can send automatic reminders to the patient. |
| Con: Paper forms can get lost or damaged. | Pro: Data are backed-up. | |
| Con: Handwriting my be illegible. | Pro: Data have a standard format. | |
| Timeliness | Con: Potential adverse events or treatment futility recorded on paper forms may not be seen until the patient brings them in at the next office visit r sends them in the mail. | Pro: Automatically integrated in the EDC system in real time for site personnel to review. |
Barriers to PRO
A barrier for integrating PROs in a study may be the validation of the method of collection. Even if a questionnaire was validated previously for paper-based administration, if the questionnaire is to be used for electronic administration then it would need additional validation.
A common perception of PROs at the site level is that the data are not as meaningful as other objective measures. Similarly, patients may also view PROs as irrelevant or overly burdensome. Training of the relevance of the specific PRO may increase compliance at both the patient and site level.
Conclusion
Patient reported outcomes offer valuable insight into the patient’s experience. Consideration of what PRO to use and how (electronic or paper) should be made on a per-study basis. With proper validation and training, barriers to compliance can be overcome.
Wondering how to include PROs in your study? MED offers a variety of clinical trial services and is a full-service CRO. We have over 40 years of experience designing and executing clinical trials, ranging from early feasibility studies to multinational, controlled pivotal trials to post-market registries.
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