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Patient reported outcome measures: researchers ask whether they are up to the job

BMJ 2010; 341 doi: https://doi.org/10.1136/bmj.c6160 (Published 01 November 2010) Cite this as: BMJ 2010;341:c6160
  1. Matthew Limb
  1. 1London

Measuring the way that patients experience their treatment is an idea whose time has come but one that still poses big challenges for scientists and clinicians, a leading expert said last week.

Stefan Cano, one of the organisers of last week’s conference of the International Society for Quality of Life Research, spoke to the BMJ about the opportunities and controversies surrounding patient reported outcome measures (PROMs).

He said it was exciting that UK ministers were now actively promoting the use of PROMs in areas such as surgery; and there was also guidance from US and European regulatory bodies on their use to support drug labelling claims.

But he acknowledged that many specialists were in “heated debate” over whether the patient level measurement scales and questionnaires developed so far were “fit for purpose.” Many researchers were also concerned about how the tools could meet standards being set by regulators.

Dr Cano said, “These questionnaires, these scales are becoming very important. And they are here to stay. They are being used to make decisions, which means they have to perform and they have to give the right information in the right way.

“But there are controversies about some of the science; there are controversies about some of the scales; and there are controversies about some of the approaches.”

Dr Cano, a senior lecturer in psychometrics at the Peninsula Medical School of the Universities of Exeter and Plymouth, helped draw up the scientific programme for the society’s 17th annual conference, held over four days in London from 27 October.

He said that the aim of the event, entitled “Translating quality of life measurement into decision-making,” was to debate views and new scientific thinking, tackle technical and practical issues to do with data collection, and ultimately develop policy positions. Participants from around the world included clinicians, researchers, health economists, representatives from drug and medical device manufacturers, and regulators and policy makers.

PROMs are questionnaires that patients complete themselves to measure their health status or health related quality of life. Since April 2009 all UK providers of NHS funded hip replacements, knee replacements, groin hernia surgery, or varicose vein surgery have had to collect these data from patients before and after the procedures.

The first study of PROMs for the four surgical procedures in NHS hospitals in England was published in September this year (BMJ 2010;341:c5143, doi:10.1136/bmj.c5143).

The data are meant to support patients’ in their healthcare choices and to be useful to health service commissioners as an indicator of the quality of healthcare.

Dr Cano told the BMJ that a key issue of debate on the validity of PROMs was that many of the measurement scales were originally developed for a different purpose: scientific and clinical research. “They were used to talk about groups, not individual people.”

There was also some concern over the stipulations being made by regulatory bodies—particularly the US Food and Drug Administration and to a lesser extent the European Medicines Agency—to ensure that drug labelling claims accurately reflected the patient’s perspective in cited studies.

Dr Cano said, “There’s tension that the scientific bars they’ve raised are so high that it’s very hard for any of the existing scales to reach the standards they want.”

A further problem was the existence of many different scales for the same condition, making it difficult for researchers to know which to use and how to ensure valid comparisons between studies.

Dr Cano said a big effort was now being expended throughout the International Society for Quality of Life Research community to get these things to work.

He explained that the momentum behind PROMs was growing and that the initiative in surgery would gradually extend into other areas, such as diabetes and other long term conditions.

One workshop at the conference looked at the challenges of using paediatric PROMs in clinical trials involving children.

Two facilitators from Mapi Values, a company developing PROMs, described how researchers needed to take particular care with questionnaires to obtain valid information that reflected the experience of children and young people accurately according to their ages and development.

Linda Abetz-Webb told participants: “You shouldn’t just take a PROM instrument developed for adults and just plug it into children.”

In conducting interviews with children about their symptoms and experiences, researchers had to make sure they used easily understood language and did not overload them with answer options, pose leading questions, or ask them to recall events too far in the past. Discussion also centred on when it was appropriate for parents to sit in on interviews and when they should be questioned separately.

Rob Arbuckle, also of Mapi Value, said, “Parents tend to rate a child’s level of health as worse than the child [does].”

The workshop heard about the effect of recent guidance from regulatory bodies, such as the FDA. Mr Arbuckle said that anecdotal evidence suggested that some drug companies were “doing the bare minimum” in trials rather than taking the time to use well designed paediatric PROMs.

Notes

Cite this as: BMJ 2010;341:c6160