What is a patient-reported outcome measure?
Patient-related outcome measures or PROMs are questionnaires that are completed by patients that seek to measure patient perceptions about their health status after diagnosis and treatment for a disease – thus the term “outcomes”. PROMs are different from natural history studies in that they focus on outcomes from treatment. Treatment is broadly defined in this discussion. It can range from surgical intervention to rehabilitation and is specific to the disease and therapies being applied to the patient.
The British Journal of Anesthesia defines PROMs are “standardized, validated questionnaires that are completed by patients’ during the perioperative period to ascertain perceptions of their health status, perceived level of impairment, disability, and health-related quality of life. They allow the efficacy of a clinical intervention to be measured from the patients’ perspective. Questionnaires are given to patients both pre and post operatively to allow comparison of outcomes pre and post procedure.” The addition of PROMs helps medical and patient communities to better understand the ultimate value of treatment beyond the specific intervention. Quality of life (QOL) is of a key element in PROMs.
FDA Regulatory Impacts
The FDA defines a patient-reported outcome (PRO) as a measurement based on a report that comes directly from the patient (i.e., the study subject) about the status of a patient’s health condition, without amendment or interpretation of the patient’s response by a clinician or anyone else. As the definition implies, there value of gathering this type of measure can be extremely valuable in the evaluation of effectiveness medical interventions. Therefore, PROMs that are integrated into a patient registry programs can gather key measures which include elements such as clinical efficacy, side-effects and adverse events, as well as symptom control intervention in a longitude manner beyond regulatory phase 3 clinical trials.
PROMs in Phase 3 Regulatory Trials
In clinical research, the application of PROMs is not decidedly prevalent. A probable cause is the high cost and significant scientific hurdles manufacturers must encounter when developing new medical treatments. While beneficial, the addition of PROMs will likely add cost and extend timelines –which may work against regulatory approval of new therapies. As such, many PROMs are conducted post-regulatory approval in the format of a patient registry program. Patient registry software systems allow manufacturers and patient advocacy groups the opportunity to automate the collection of PROMs in a rapid and systematic that is cost-effective, auditable and reliable.
Inclusion of PROs in a Patient Registry
Including patient-reported outcomes in registries offers numerous advantages.
- Incorporation of the patient voice helps keep care and research patient-centered, acknowledging the balance and tension between traditional outcomes and PROs.
- Symptom burden, QOL, and satisfaction with care are dynamic variables that cannot be recreated accurately through retrospection; they are essentially lost if not captured “in the moment.” For this reason, routine, systematic, and longitudinal collection is recommended and should be a standard of practice1.
- Gliklich RE, Dreyer NA, Leavy MB, editors. Registries for Evaluating Patient Outcomes: A User’s Guide [Internet]. 3rd edition. Rockville (MD): Agency for Healthcare Research and Quality (US); 2014 Apr. 5, Use of Patient-Reported Outcomes in Registries. Available from: https://www.ncbi.nlm.nih.gov/books/NBK208628/