17-year literature review shows that patient-reported outcomes consistently respond to clinically-efficacious drugs but with wide variability in rates of important quality of life improvements

WORCESTER, MA, March 17, 2014 – Some of the most debated questions regarding growing reliance on patient-reported outcome (PRO) surveys in evaluating the quality of health care were addressed in an article by a medical student and a professor today in Medical Care, a research journal published by the American Public Health Association.

• Are self-administered patient survey measures responsive enough to capture the quality of life benefits of improvements in laboratory tests and other clinical outcomes caused by drug therapies?
• How often do clinically-efficacious therapies lead to important and meaningful improvements in what patients are able to do in everyday life?

According to the most comprehensive review of quality of life outcomes reported from well-controlled clinical trials of drug therapies, the answer to the first question is “Yes,” more than 80% of the time. However, there is wide variability in the rates at which drug treatments achieved accepted thresholds for improvements in quality of life (about 58% overall; 0-100% across clinical areas). For example, drug therapies for rheumatoid arthritis, psoriatic arthritis, and psoriasis consistently achieved the largest average functional health improvements, whereas therapies for peripheral arterial disease or chronic obstructive pulmonary disease achieved much smaller benefits, often below thresholds considered important and meaningful. The reviewers note that, with the migration to electronic data capture and modern adaptive health survey methods, the efficiency and usefulness of PRO measures will increase. For the important health domains that old and new measures have in common, cross-calibration of new measures with existing measures will enable comparability of past and future PRO data.

Dan Frendl, an MD-PhD student at UMass Medical School and John E Ware, Jr.,a UMass Professor and Chief Science Officer at John Ware Research Group, conducted a systematic review of 185 randomized, double-blind, placebo-controlled drug trials published from 1995-2011 with well-documented results for primary clinical endpoints and for outcomes measured by the SF-36 Health Survey. They were assisted by Mikel Strom, MSLS. Responsiveness was defined as concordance (both statistically significant or both non-significant) between primary clinical and SF-36 outcomes. Meaningful improvements in average SF-36 physical and mental component summary scores, net of placebo, were defined as those exceeding the developer’s recommended threshold for a minimal important difference (MID) and rates were compared across clinical areas. The Medical Care publication is available here. Auxiliary documentation of findings including citations for well-controlled trials and results by therapeutic area are available at:

http://links.lww.com/MLR/A691
http://links.lww.com/MLR/A690
http://links.lww.com/MLR/A688
http://links.lww.com/MLR/A689

Frendl DM and Ware JE. Patient-reported functional health and well-being outcomes with drug therapy: A systematic review of randomized trials using the SF-36 Health Survey. Medical Care 2014 52(5):439-445.