Diarmuid De FaoiteSmith & Nephew, Baar, Switzerland

Correspondence to:Diarmuid De FaoiteClinical, Scientific & Medical AffairsSmith & Nephew Orthopaedics AGOberneuhofstrasse 10d6340 BaarSwitzerlandTel: +41 41 766 22 62diarmuid.defaoite@smith-nephew.com

AbstractProfessor Matthias Rose is Medical Directorof the Psychosomatic Department at theCharité University Hospital in Berlin,Germany. In this interview, I discuss with himpatient-reported outcomes and the Patient-Reported Outcomes Measurement Informa -tion System (PROMIS®) initiative, which,according to the PROMIS website(http://www.healthmeasures.net/explore-measurement-systems/promis) is “a set ofperson-centered measures that evaluates andmonitors physical, mental, and social healthin adults and children” that “can be used withthe general population and with individualsliving with chronic conditions”.

MEW: Thank you for agreeing to thisinterview, Professor Rose. What is the mostcommon mistake you see in the developmentof patient-reported outcomes (PROs)?Prof. Rose: I think the most problematic thing isthat people jump straight into it without thinkingabout what is the construct they really want tomeasure. Frequently, we are approached bydifferent parties who say they want to measure“quality of life” without really understandingwhat is meant by that. In my view, people try to

Interview withProfessor Matthias Rose on developing patient-reportedoutcomes and the PROMIS initiative

PROMIS®

In 2004, the US National Institutes of Health initiated the development of a comprehensivePatient-Reported Outcome Measurement Information System (PROMIS®). The aim of thisinitiative is to improve substantially the standards for the assessment of the self-reported healthstatus. Over 300 measures of physical, mental, and social health are available for use with thegeneral population (adults and children) and individuals with chronic conditions. The PROMISmeasures have been tested and validated in large reference populations making them suitable forresearch on different conditions.

The programme has generated a reliable and oftentimes more sensitive system, customisedto the patient, which poses fewer questions than traditional paper-based PROMs do.

Find out more at http://www.common-metrics.org/ or www.healthmeasures.net/promiswhere you can also take an online computer adaptive test demonstration.

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ProfessorMatthias Rose

Diarmuid De Faoite – Interview with Professor Matthias Rose

bypass the first steps in developing the con cep -tional measurement model much too often. Theypick out some established instrument from theliterature without questioning its appropriatenessfor their particular research question.

MEW: Given this, how important is thedevelopment of guidelines like theInternational Society for Pharma co economicsand Outcomes Research (ISPOR) steps inidentifying and evaluating an existing PROmeasure?Prof. Rose: Very important. I think that thelonger you are in the field, the more clearly yousee the need for the basics to be correct.Initiatives like the ISPOR guidelines1 are veryuseful in ensuring that the basic elements neededin a PRO are present.

MEW: Patient-reported outcome measures(PROMs) are the instruments used tomeasure a patient’s health status or health-related quality of life at a single point in time.However, are there too many PROMs?Prof. Rose: There are probably over 4,000different PROMs out there, and most of them arecarefully developed and validated. Although thisis an impressive amount of work, I believe thatthis plethora of instruments actually hinders theiracceptance. For PROMs to enjoy the same levelof acceptance like biomarkers, we need muchgreater standardisation and less confusion.

MEW: Which is what the Patient-Reported Outcome MeasurementInformation System (PROMIS®) istrying to achieve, right?Prof. Rose: Yes. Think of it like this.Today, most PROMs are likethermometers using different scales,which makes it highly complicated tocompare measurement results amongthem, even if they measure the sameconstruct. PROMIS provides a com -mon metric to allow this (Figure 1).Thus, if you score your instrument onthe PROMIS metric, scores resultingfrom different assessment tools can beinstantly compared in a meaningfulway. Just like using different ther -mometers to measure temperature.

Thus, PROMIS also addresses another olddispute in the field, which is if you favour genericor disease-specific tools. Disease-specific tools

are typically more responsive to demonstratetreatment effects, whereas generic tools allowcomparisons between different clinical popula -tions. When you look into the constructionprinciple of disease-specific tools, essentially theyare a compilation of health domains combined in

one composite score.PROMIS domains are generic, but

they can at the same time act asbuilding bricks providing a disease-specific score. Thus, the compilation ofhealth domains is specific, not theassessment itself.

Let me give you a more concreteexample. PROMIS identifies the ele -ments such as physical function, pain,anxiety, and so on, which are relevantto everyone. You can then pick andchoose the different domains whichare relevant for different diseases. Forexample, some of the PROMISdomains are relevant for both heart

disease and musculoskeletal disease (e.g.physical function), but others are only relevantfor heart disease (e.g. dyspnoea).

This is the core of the idea behind PROMIS.The combination you choose is disease-specific

but not the constructs! We have liberated thedifferent domains from being tied to specificinstruments – and diseases.

MEW: You have been involved in PROMIS sinceit began and are the Chair of PROMISGermany. You have seen a lot of progress, butwhat is the next quantum leap for PROMIS inyour opinion?Prof. Rose: PROMIS started because we hadnew methods like computer-adaptive tests(CATs), which could be employed for moreprecise measure ments. In addition, the initiativehas such political clout with the necessaryfunding behind it to make it happen. But thebigger achievement of PROMIS is that it createsa framework of health. It has the potential to setscales independent from the tools, indispensablefor standardisation. Consider it this way, for thefirst time, in the world of patient-reportedoutcomes, we would have definitions and scalesthat are as easy to understand as, for example, theCelsius scale is for temperature.

There will never be complete agreement onwhich instrument to use, that’s human nature.After all, people never want to have just one typeof car, but the advancement in PROs that

For PROMs toenjoy the same

level ofacceptance likebiomarkers, we

need muchgreater

standardisationand less

confusion.

www.emwa.org Volume 27 Number 4 | Medical Writing December 2018 | 39

Figure 1. Home page of the PROMIS website, which is part of HealthMeasures.HealthMeasures consists of four precise, flexible, and comprehensive measurement systems that assessphysical, mental, and social health, symptoms, well-being and life satisfaction; along with sensory,motor, and cognitive function.

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Interview with Professor Matthias Rose – Diarmuid De Faoite

PROMIS will bring means we are moving closerto achieving the status of biomarkers that Ipreviously mentioned.

MEW: Given that you are talking to EMWA,what country in Europe is furthest indeveloping and adopting PROMs?Prof. Rose: In my opinion, it is the Netherlands.The Dutch mindset has always been innovativeand open to adoption. If you look atdifferent research consortiums forEuropean Union funding etc., theDutch are always well represented. So,if I had to pick any one country inEurope, I would choose theNetherlands.

MEW: What should medical writerskeep in mind when they write aboutPROs?Prof. Rose: They should be careful with theterms they use. Don’t confuse outcomes withpredictions or determinants. The term patient-reported outcomes is used but people are oftennot thinking of outcomes when they write this,but are rather thinking of predictions. Anoutcome is something you expect to change orvary based on other factors. You should be clear

in what it is that you are reporting.When writing, make sure that you distinguish

between the proximal outcomes (i.e., symptomsand function) and the distal outcomes (e.g.quality of life). For example, with heart failure,shortness of breath and physical function areproximal outcomes, which are likely to changedue to medical interventions. However, a distaloutcome like quality of life might not be affected

by the intervention, as aspects alsorelevant for this construct, like level ofjob satisfaction or environmentalfactors, are not targeted by theintervention.

A conceptual model well knownwithin the German healthcare systemis the one developed by Wilson andCleary2 a couple of decades ago. It is abasic model, but one which is very

effective at classifying different measuresof health outcome. It might be useful for medicalwriters who are new to the subject of patient-reported outcomes to learn more about thismodel.

MEW: Any last comments?Prof. Rose: I have always been a missionary forpatient-reported outcomes. It is great that ajournal like Medical Writing is concentrating on

the subject and helping to get the message aboutPROs out there.

Conflicts of interestThe author is employed by Smith & Nephew.

References1. Rothman M, Burke L, Erickson P, Leidy NK,

Patrick DL, Petrie CD. Use of existingpatient-reported outcome (PRO)instruments and their modification: the ISPOR Good Research Practices forEvaluating and Documenting ContentValidity for the Use of Existing Instrumentsand Their Modification PRO Task ForceReport. Value Health. 2009;12(8):1075–83.

2. Wilson IB, Cleary PD. Linking clinicalvariables with health-related quality of life: a conceptual model of patientoutcomes. JAMA. 1995;273(1):59–65.

Author informationDiarmuid De Faoite is the guest editor ofthis issue of Medical Writing and a memberof the EMWA Executive Committee. Hisdaily work involves patient-reportedoutcomes in orthopaedics.

I have alwaysbeen a

missionary for patient-

reportedoutcomes.

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