The frequency and availability of population-specific patient reported outcome measures and minimal clinically important differences among approved drugs in Canada

Allison Soprovich,Dean T Eurich,Meghan Ingstrup

doi:10.1186/s12955-018-1070-0

Abstract

BackgroundPatient reported outcome measures (PROMs) and minimal clinically important differences (MCIDs) are included in Canada’s Common Drug Review (CDR) process to approve new drugs. Often, the measures report on the health-related quality of life (HRQoL), but can also describe the symptoms, efficacy and harms important to patients. They can be generic or population/condition specific, validated or not. We examined the frequency, availability and accessibility of validated, specific PROMs and MCIDs reported in the CDR reports.MethodsWe searched the Canadian Agency for Drugs and Technologies in Health (CADTH) on-line database for completed Common Drug Review, Clinical Review Reports (CDR-CRR) between November 2013 and February 2017. Two independent reviewers examined the reports and references for PROMs and MCIDs. Both reviewers separately categorized the PROMs and MICDs according to purpose, validation, availability and funding received. Discrepancies were rectified by consensus with a third investigator.ResultsOne-hundred and five unique PROMs were extracted from 39 CDR-CRR, 57% with a HRQoL component. 91/105 (87%) referenced a validation study and 62/105 (59%) referenced a validation study in the study population of interest. Fifty-seven MCID references were extracted from 39 CDR-CRR. 34/57 (60%) were specific to the study population of interest, and 36% had a HRQoL component. 50% of PROM and 53% of MCID references were publicly available.ConclusionsPROMs and MCIDs referenced in CDR-CRR show similar trends. The majority are validated, but not necessarily in the study population of interest. Continued critical examination is required to evaluate new drugs specific to the population of interest.

Highlights

Patient reported outcome measures (PROMs) and minimal clinically important differences (MCIDs) are included in Canada’s Common Drug Review (CDR) process to approve new drugs
If we are to advance in patient involvement in clinical care, regular patients and clinicians will need to have timely access to the research findings. In response to these three patient-related issues, we examined the incorporation of PROMs, MCIDs and public access in the CADTH Common Drug Review, Clinical Review Reports (CDR-CRR)
Of the 60 PROMs with a health-related quality of life (HRQoL) component, 55 (92%) referenced a validation study and 36 (60%) referenced validation in the study population of interest. 69% of the time, PROMs validated in the study population were in specialty drugs (p = 0.021) where CDR-CRRs including biologic agents were less likely to be validated (60%, p = 0.586)

Summary

Introduction

Patient reported outcome measures (PROMs) and minimal clinically important differences (MCIDs) are included in Canada’s Common Drug Review (CDR) process to approve new drugs. The measures report on the health-related quality of life (HRQoL), but can describe the symptoms, efficacy and harms important to patients. They can be generic or population/condition specific, validated or not. Many of the clinical trials comprising the drug reviews include Patient Reported Outcome Measures (PROMs) that describe patient perspectives on treatment and disease impacts. They aim to capture the personal and social context of the disease and treatment experiences to assess the full impact of a treatment [2]. These measures are helping to move patient centered practices forward

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