Assessment of Patient-Reported Outcome Instruments to Assess Chronic Low Back Pain

13 Pages·2017·0.22 MB·English

PainMedicine2017;18:1098–1110
doi:10.1093/pm/pnw357
METHODOLOGY, MECHANISMS & TRANSLATIONAL
RESEARCH SECTION
Original Research Article
Assessment of Patient-Reported Outcome
D
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Instruments to Assess Chronic Low Back Pain w
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ARHbNilth,riuMladPsALhi,a†edRSgateemvnaessn,aPIm.hByD,l,uMMmSa,cHM,EMB,§ASC,,*hMMaArol,n‡esaAL.rgMoafrf,tin, PTahnaarmbeacPehuatircmaals,CIonrcp.o;risataionc;oinssaunltainntvefosrtigMaittsourbfoisrhi https://a
MD,¶ RainerFreynhagen,MD,kMark Wallace, MD,kj Gru¨nenthalGmbH;and is aspeaker forAstellas ca
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KellyP.McCarrier,PhD,MPH,† Pharma, Inc., Pfizer,Inc.,Eli Lilly andCompany, em
Donald M.Bushnell, MA,†Noe€lV. Hatley,MPH,† Gru¨nenthal, andMerck &Co., Inc.M. Wallaceserves ic
and DonaldL. Patrick,MPH,PhD** onadvisoryboardsforDepomed,Inc., Janssen .ou
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Pharmaceuticals,Inc., ZynerbaPharmaceuticals,Inc., .c
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*Forest ResearchInstitute, JerseyCity,NewJersey; Daiichi SankyoCo., Ltd., andCoronadoBiosciences, /p
†Health ResearchAssociates,Mountlake Terrace, Inc.D.L. Patrickhasnoconflictstodeclare. ain
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Washington;‡GlaxoSmithKline,Collegeville, ed
Pennsylvania;§Grunenthal,GermanyGmbH,Aachen, icin
Germany; ¶AlbanyMedical College,Albany,New Abstract e/a
York, USA; kBenedictus KrankenhausTutzing and rtic
Objective. To identify patient-reported outcome le
Technische Universita€t, Mu¨nchen,Germany; -a
(PRO) instruments that assess chronic low back b
kjUniversity ofCalifornia SanDiego, San Diego, pain (cLBP) symptoms (specifically pain qualities) stra
California; **UniversityofWashington,Seattle, c
and/orimpactsforpotentialuseincLBPclinicaltri- t/1
Washington,USA alstodemonstratetreatmentbenefitandsupportla- 8/6
Correspondenceto:MonaL.Martin,RN,MPA, 6505 belingclaims. /10
9
216thSt.SW,Suite105,MountlakeTerrace,WA 8
Design.LiteraturereviewofexistingPROmeasures. /3
98043,USA.Tel:[phone]Ext.202;Fax:425- 0
6
2
775-6734;E-mail: [email protected]. 3
Methods. Publications detailing existing PRO mea- 8
4
Funding source:Thisstudywas supportedbyre- sures for cLBP were identified, reviewed, and sum- b
y
search fundsfrombothForest ResearchInstituteand marized. As recommended by the US Food & Drug gu
GruenenthalGmbH. Astdamndinairsdtratmioenas(uFrDeAm)enPtROchdaeravcetleorpismtiecnst gwueirdeancree-, est o
n
Disclosureand conflictsofinterest:A.Ramasamy is viewed,includingdevelopmenthistory,psychometric 2
7
anemployeeand stockholderofActavis,Inc.M. L. properties (validity and reliability), ability to detect M
a
Martin,K.P. McCarrier, and D.M. BushnellI are change,andinterpretationofobservedchanges. rc
h
employeesofHealth ResearchAssociates,Inc.,which 2
0
received fundstoconductthe studyfromForest Results. Thirteeninstrumentswereselectedandre- 19
viewed: Low Back Pain Bothersomeness Scale,
ResearchInstituteandGru¨nenthalGmbH.S. I.Blum
Neuropathic Pain Symptom Inventory, PainDETECT,
servesonthe advisoryboardforthe Patient-Centered
Pain Quality Assessment Scale Revised, Revised
OutcomesResearchInstitute(PCORI),is anemployee
Short Form McGill Pain Questionnaire, Low Back
ofGlaxoSmithKline, andis ashareholder for
Pain Impact Questionnaire, Oswestry Disability
GlaxoSmithKline andActavis. H.Liedgensis anem-
Index,PainDisabilityIndex,Roland-MorrisDisability
ployeeofGru¨nenthalGmbH.C.Argoffhasnorelevant Questionnaire, Brief Pain Inventory and Brief Pain
conflictstodisclose. R.Freynhagenservesonadvi- Inventory Short Form, Musculoskeletal Outcomes
soryboardsforAstellas PharmaInc., PfizerInc., Eli Data Evaluation and Management System Spine
Lilly and Company,Gru¨nenthalGmbH,and Janssen Module,OrebroMusculoskeletalPainQuestionnaire,
VC 2017AmericanAcademyofPainMedicine.Allrightsreserved.Forpermissions,pleasee-mail:[email protected] 1098
ReviewofLow Back PainPRO Instruments
and the West Haven-Yale Multidimensional Pain reliable and valid data collection and establish the end
InventoryInterferenceScale.Theinstrumentsvaried point(s)asfitforpurpose[11].
in the aspects of pain and/or impacts that they as-
sessed, and noneofthe instrumentsfulfilled all cri- Several questionnaires have been designed to measure
teria for use in clinical trials to support labeling cLBP symptoms and symptom-related impacts, but
claims based on recommendations outlined in the their validity and symptom comprehensiveness may not
FDAPROguidance. fully satisfy the FDA guidance for PRO instruments to
support claims in approved medical product labeling.
Conclusions.Thereisanunmetneedforavalidated Although the FDA has accepted a single patient-
reported item of worst pain severity for the approval of
PROinstrumenttoevaluatecLBP-relatedsymptoms D
products for pain conditions in the past, the symptom- o
andimpactsforuseinclinicaltrials. w
based characterization of the patient’s pain experience nlo
with a single item is limited. The objective of this review a
Key Words. Patient-Reported Outcome Measure; d
ChronicLowBackPain;FDA;Instrument Review wexaisstintogsinusmtrmumareiznetsktehyatcahsasreascstecriLsBticPssoyfmaptsoemlesctaionnd/oorf ed fro
m
impacts to determine which have the strongest mea-
h
Introduction surement properties for potential use in cLBP clinical tri- ttp
Low back pain (LBP) is usually defined as pain, muscle atols.sTuhpisposretarecahrlyanddeicnisstiorunm-menaktinregvieawbowuatsacpopnrdoupcritaetde s://ac
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tension, or stiffness localized below the costal margin measures to consider for use in therapeutic clinical trials d
e
and above the inferior gluteal folds, with or without leg bytwopharmaceutical companies. m
ic
pain (sciatica). While acute and subacute episodes (last- .o
u
ing up to three months) are the most common presen- Methods p
tations of LBP, chronic low back pain (cLBP) causes .co
m
greater disability (physical limitations and psychological Selectionof Candidate Instruments /p
effects)[1]. ain
The goal of this review of PRO instruments was to iden- m
e
Painhasbeendescribedasoneofthecardinaldomains tify those that have the best measurement properties, d
ic
to be assessed in cLBP [2–4] and is a typical reason for provide the best coverage of the selected concepts in
e
consultation. Pain is one of the best determinants of (LBP symptoms and impacts), provide the best fit for /a
disability due to LBP [5,6] and is predictive of work re- clinical study design and measurement strategy to sup- rtic
le
sumptionwithintheyearfollowingrelatedshort-termab- port a label claim that has been used in LBP studies, -a
sence [7]. Within the biomedical model, the level of pain and offer any other additional benefits. To be selected bs
a patient perceives has been traditionally regarded as a for review, instruments were in English, were cLBP- tra
c
subjective measure. Correlations between self-reported specific or had been used in cLBP clinical trials, and t/1
pain and bothself-reported disability and objectivemea- measured the symptoms and/or impacts from cLBP. 8/6
sures of function are invariably weak [4,5,8], highlighting Generic pain measures were also included for review if /1
0
the need to measure pain in a more direct way. the measure had been used in a cLBP clinical trial. 98
Measurement of pain consistently captures much Instruments were excluded if they were general instru- /3
0
greater treatment effect sizes than physical variables ments (excluding general pain measures) not specific to 62
3
[5,9] or condition-specific instruments [10]. Subjective LBP, were cLBP-specific instruments that did not mea- 8
4
patient assessment of pain and physical limitations due sure pain symptoms and/or pain impacts, were cited in- b
y
topain areessentialendpoints forclinicalstudies. frequently (fewer than three citations per year since g
u
development), were developed prior to the 2009 FDA e
s
Patient-reported outcome (PRO) instruments can be PRO guidance and did not demonstrate compliance t o
n
used to support claims in medical product labeling with the guidance, or were not developed or used for 2
7
where subjective assessments from the patient are im- cLBP. Other possible measures that were very new but M
portant to assessing treatment benefit. The US Food had no published data yet for use in cLBP populations a
rc
and Drug Administration (FDA) has published a guid- were excluded (i.e., the Patient-Reported Outcome h
2
ance for industry on the development and validation of Measurement Information System [PROMIS] pain mea- 0
1
PRO measures that outlines the evidentiary support sures [12]). The generic PROMIS pain measures were 9
needed by the FDA to review a measure for its ade- not selected in our review because the measures were
quacy to support labeling claims [11]. When reviewing a not developed for use by patients with cLBP [13] and
PRO instrument, the FDA considers the PRO instru- because item banks were deemed too broad for use in
ment’s conceptual framework and its measurement a cLBP trial. Generic quality of life measures were ex-
properties (including content validity, reliability, construct cluded, along with measures that assessed more distal
validity, and ability to detect change). The guidance also concepts unlikely to be considered end points in clinical
emphasizes the need for adequate documentation of trials to support product labeling (e.g., effects on social
patient input to support the content validity of PRO in- life or relationships and emotional impacts). Any pub-
struments to be used in evaluating medical outcomes, lished previous versions of the existing measures were
specifically ensuring comprehensibility is vital to ensure alsoexcluded.
1099
Ramasamy etal.
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Figure 1 PRO search strategy and outcomes. IMMPACT ¼ Initiative on Methods, Measurement, and Pain e/a
Assessment inClinical Trials;PRO¼ patient-reported outcome. rtic
le
-a
b
s
tra
Systematic Assessmentof Selected Instruments assess cLBP in addition to clinician-based outcomes ct/1
and physiological outcome measures. Additional instru- 8
/6
The quality of each selected PRO instrument was as- ments were identified in a review of the Initiative on /1
sessed in terms of its appropriateness in reflecting pa- Methods, Measurement, and Pain Assessment in 09
8
tients’ perspectives in a given study and context of use, Clinical Trials (IMMPACT) group recommendations (four /3
0
as described by the FDA PRO guidance document [11]. instruments) [15], a search in PROQOLID [16] and 6
2
Instrumentswereevaluatedforthefollowingcharacteris- ISPOR [17] databases (nine instruments), a parallel liter- 3
8
tics: name/acronym, general description (purpose, con- aturereviewonpatient perspectivesofcLBP(fiveinstru- 4 b
tent, versions, number of items), development history ments), and experience by the research team in the y g
(patient involvement, development population, cross- conduct of pain clinical trials (five instruments). In the ue
s
cultural input, known use in studies), scoring (response 2015 search, a total of 137 measures and scales were t o
scale, recall period, method of scoring, patient burden, identified in the Medline (PubMed) database. Of all of n
2
interpretation of scores), measurement properties (reli- the PRO instruments identified in both searches, 163 in- 7
M
ability,validity,abilitytodetectchange),andanoverview struments were excluded for relevance (Supplemental a
of strengths regarding potential use in a phase III clinical Table S1) and a total of 13 instruments were included in rch
trial. thefinal analysis. 2
0
1
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Results The 13 PRO instruments to be evaluated were grouped
by domain: 1) symptom descriptions (pain qualities), 2)
Selected Instruments pain-related impacts, and 3) instruments addressing
both symptoms and impacts. The instruments and cita-
A literature search for articles describing PRO measures tions for key publications that describe the development
for cLBP was conducted in 2011 and updated in 2015 and measurement properties are briefly described be-
using Medline (Figure 1). In the 2011 search, a system- low. Table 1 describes the PRO instruments, scoring,
atic review by Chapman and colleagues [14] was found and interpretation of scores, and Table 2 summarizes
that identified 16 PRO instruments commonly used to therelevance ofeachselected measuretocLPB.
1100
ReviewofLow Back PainPRO Instruments
Interpretation ND Totalintensityscoreisthesumofthe10de-scriptoritems5NPSIsubscalesarescoredbasedonascor-ingalgorithmMaximumscore:10Minimumscore:0Higherscoresrepresentgreaterpain Totalscoreisthesumofall7itemsMaximumscore:38Minimumscore:1(cid:2)Score12:neuropathicpainunlikely(cid:3)Score13–18:unclear,neuropathicpainmaybepresentScore19:neuropathicpainispresent(cid:4)Totalscoreisthesumofthe20descriptoritemsPQASsubscalescalculatedusing15oftheitemsMaximumtotalscore:200Minimumtotalscore:0HigherscoresrepresentgreaterpainTotalscoreisthemeanofthe4subscalescores;eachsubscaleisscoredbycalculat-ingthemeanratingsforsubscaledescriptorsNocutoffpointsaredefined;itisinferredthathigherscoresindicatemoreseverepain NA Totalscoreisthepercentageofthetotalpointsfromtheitemsanswered (continued) Downloaded from https://a
c
a
Description 1tomultiplesingle-itemratingscales 5dimensions/subscales(10descriptoritemsand2tem-poralitems):deep,spontaneousongoingpain;parox-ysmalpain;abnormalsensations;superficial,spontaneousongoingpainEachitemisratedonan11-pointNRS(0lowestpain,¼10highestpain)¼5NPSIsubscalesarescoredbasedonthemethodpre-sentedinthedevelopmentarticle7items:5itemsonsensoryresponsesratedfrom0(never)to5(verystrongly),2itemsontemporal/spa-tialcharacteristicsofpainpatternratedfrom1to4(cid:2)þ 20itemsgroupedinto3painqualityfactors:paroxysmalpain,superficialpain,deeppainEachitemratedon11-pointNRS(0nopain,¼10most[descriptor]painsensationimaginable)¼ 22itemsdescribingdifferentqualitiesofpainandrelatedsymptoms4subscales:3sensorydescriptors,1affectivedescriptorEachitemratedon11-pointNRS(0none,10worst¼¼possible) 26impactitems:18itemsratedon11-pointNRSfrom0(nodifficulty)to10(extremedifficulty);8itemsratedfrom0(never)to4(veryoften)10items:painintensity,personalcare,lifting,walking,sitting,standing,sleeping,sexlife,sociallife,traveling demic.oup.com/painmedicine/article-abstract/18/6/1098/3062
3
8
4
assessments Fullname n(painqualities)Lowbackpainbother-somenessscaleNeuropathicPainSymptomInventory painDETECTQuestionnaire PainQualityAssessmentScaleandRevisedPainQualityAssessmentScale(self-administered) RevisedShortFormMcGillPainQuestionnaire sChronicLowBackPainImpactQuestionnaire OswestryDisabilityIndex by guest on 27 March 2019
PRO criptio mpact
ed des R edi
ct m S- at
e o A el
Sel mpt PQ n-r
able1 cronym omain:syBP-BS PSI D-Q QASand F-MPQ-2 omain:paiLBP-IQ DI
T A DL N P P S DC O
1101
Ramasamy etal.
Interpretation 0–20%:minimaldisability20–40%:moderatedisability40–60%:severedisability60–80%:patientiscrippled80–100%:patientisbedboundorexaggeratingTotalscoreisthesumofall7itemsMaximumscore:70Minimumscore:0Highertotalscoreindicatesgreaterdisabilityduetopain Totalscoreisthesumofall24itemsMaximumscore:24(maximumdisability)Minimumscore:0(nodisability) Painseveritysubscaleisscoredasacompos-itemeanscoreofthe4itemsrelatingtoseverityPaininterferencesubscaleisscoredasacom-positemeanscoreofthe7itemsrelatingtointerferenceThehigherthescore,thegreaterthepainse-verity/interferenceND Totalscoreisthesumofthe21scoreditemsMaximumscore:210 (continued) Downloaded from https://a
c
a
escription achitemratedona6-pointscalefrom0(nodisability)to5(disability) categoriesoflifeactivitiescoveredin7items:family/homeresponsibilities,recreation,socialactivity,occu-pation,sexualbehavior,self-care,lifesupportactivityachitemratedonan11-pointNRSfrom0(nointerfer-ence)to10(totalinterference)emsareratedontheoverallimpact4itemsassessingphysicaldisabilityduetoLBPinthefollowingaspects:painintensity,self-care,sociallife,walking,sitting,standing,sleeping,bending,stairs,generalactivity,appetite,householdchoresachitemisscored1pointfora“yes”answerand0pointsfora“no”answeratedimpacts5items(11scoreditems)withinthefollowingsub-scales:painseverity,paininterferenceachitemisratedona11-pointNRSfrom0(nopain/doesnotinterfere)to10(painasbadasyoucanimagine/completelyinterferes) heSpineModuleincludesseparatequestionnairesforcervicalspine,scoliosis,andlumbarspineemsareratedona1-to6-pointscoredditionaldetailsontheinstrument(numberofitemsandscoring)werenotdescribed 5items(21scored)containing:demographics,locationofpain,misseddaysfromwork,painsymptoms,painimpacts,psychosocialfactors demic.oup.com/painmedicine/article-abstract/18/6/1098/30
D E 7 E It2 E n-rel1 E T ItA 2 6238
Fullname PainDisabilityIndex RolandMorrisDisabilityQuestionnaire ons(painqualities)andpaiBriefPainInventoryandBriefPainInventory–ShortForm(InterferenceScale) SpinemoduleoftheMODEMS(MusculoskeletalOutcomesDataEvaluationandManagementSystem)outcomeinstrumentOrebroMusculoskeletalPainQuestionnaire 4 by guest on 27 March 2019
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Table1Conti Acronym PDI RMDQ Domains:symptoBPIandBPI-SF MODEMS-Spine OMPQ
1102
ReviewofLow Back PainPRO Instruments
Measures ThatAssess Symptom Descriptions(Pain
Interpretation Minimumscore:00–105lowriskfordevelopmentofpersistent¼backpainproblemsinthefuture105–130moderaterisk¼130highrisk(cid:4)¼Totalscoreisthesumofallitemsansweredanddividedbythenumberofitems(either52forthecompletemeasureorthenumberofitemsinagivensubscale)HigherscoresindicatemorepainandmorepaininterferencewithlifeNocutofforcriticalscorehasbeenestablished sviwsdfcQTews(odLoeciponnhinifaeBurtlfaLecueimoghenssaPlceoleplrdtlea-emiisiiLwsffmtctB,tiieni,ecnBiLeoetmSecdouBBtPasnssneh)tr.)sp-aPyntewas[bumeic1.inonpSvreceko8eapgfeoBirt]vfgietchilPtmwceeaohuewiefracbmaeienpdachailsdelenel.soliumlirevLeniwasHlonBea[vBe1elncielocolozao9Piltlkweutsotppet]ohh.hddupraeefeobaee.reNmdcvreftordteskuo,sshetnrruhcoe,inaeienpnsaepmtn,coicualrniaoedesnuallebugi’uosncrnspnldnirsmocdeoroaeamranepbesedrntvsiyetsnviuopeoiiesiaeltmeownaiShtwnsoswepteuuafcsifdnooursrladtcbstirnelsiahmplmioye,mdecotelmiceaufoweeoantsvnaeiniefnuioosastntetsentruseefiidmpeede,rprsiedelrdetessaoostneschnfptsntthaieiderfcfstueieibaicarcafsdtfysotabaelieiheeneulllrldeeeassiy---tt Downloaded from https://ac
a
d
se n s,m em
Description Threescoreditemshavecategoricalresponseoptionandtheremaining18areansweredona0–10scal 52itemsand12subscales:interference,support,paiseverity,self-control,negativemood,punishingre-sponses,solicitousresponses,distractingresponsehouseholdchores,outdoorwork,activitiesawayfrohome,socialactivitiesItemsaresplitinto3parts:painintensityandpainimpacts scale;PROpatient-reportedoutcome.¼ TdatitdrtvbnTodhieiaopnhpoheeeenolNPnedehsteetincderdisenassegtPyriwitrNipmufn,ynaaiid[aneeare2,eiDtlinnoonedcsdu0oDGEadcpseir]frdn.uotiETdaebtc-coledmpTtEriryMtmheeirtEeanCioeainvtsertCcpaenhTaestepanparsTtstidPnaacsyeuttaeilsut(adertPrfsitiieierriPentenosnDeoinwlvtnajimumrsa-tidSttietiQsnrtshbyeeyvhyw)eittw.nilwmeehiStitisttCotciwyeshityhpt,htyahoemptccmcnoonrvnsclphoNoitenmacnpeeLatpnurProitunBncieevropomSIrotegaenPrmoauptInlevrv,i.ipsgneaisaeenIabnstelwxgnhictqduovnphithiceuotiftttoiteohrcnyafrvotrnop,ythilaeinpeoltatteilhrinniutiariecdnehyetcrisinaeiaontecptytr(psNmmnNdo,awtoaarmPuiPdoeftnualehSnisSpmsvtcithItcoIceto)deoenorownhengpewnfsapvatteenadeieavuesnrtiiinnleesss-----t ic.oup.com/painmedicine/article-abstract/18/6/1098/30623
g 8
n incLBPpatients[21–26].Morethan300,000patientshave 4
Fullname WestHaven-YaleMultidimensionalPainInventory(InterferenceScale) done;NRSnumericalrati¼ bohpocdacLdoafseaetBsneivenetehnqvPfobuleeuo.aaelrapgrseTtltpmaehesnhronregeeilttsyvrneiisnesnavst,etleatiedowdulirinaddfnraoftwiaalttseapyhtlidamt,aihnstcbmoihsnotmuyehantp,tepetssaodaainstPrsitifdeyetioueDernrvnrnn-aetetQcorcs.tiynt,hhtT,wvrrrppahoooirrtuetnpleoevhmiadevcsmtitmipadhc-per,oitineeeaasngsttLsitern,theBus.tearhPdtaertTeepn,mhrsdedebveictseliinairscammisbtcibnieenoserislerdiecntatfsyruhsolu,pirtanrrmaopnalaongatitecnenhsiinnaaddeeet,,l by guest on 27 March 2019
ot tousualcareincLBPpatients.Therearenoreportsofpa-
n tient burden for this measure or time required to complete
¼
d the measure. The stability of pain descriptors in the PD-Q
e D
u N hasbeenshowntobeaccurateforonetothreeweeks.
n
nti ble;
o a
C ail Pain QualityAssessmentScaleand RevisedPain
v
a
1 m PI ot QualityAssessmentScale (Self-administered)
e y M n
Tabl Acron WHY NA¼ TtiohnenPaiareindQeusaiglintyedAstsoesassmseesnst pSacianleq(uPaQlitAieSs) aisssaocqiuateesd-
1103
Ramasamy etal.
andrelevancetocLBP Comments ThereisnoevidencetorecommendtheinstrumentasanoutcomemeasureforcLBPTheNPSIisnotspecifictocLBPNoevidenceonthepsychometricpropertiesoftheEnglishversion;theitemsdonotspecifyarecallpe-riod,andonestudysuggestsitdoesnotpredicttreat-mentresponseTheinstrumenthasgoodcontentvalidity,butithasnotbeenvalidatedforthecLBPpopulationTheSF-MPQ-2seemstobeanadequatesymptomout-comemeasurethatneedstobevalidatedinthecLBPpopulation TheCLBP-IQisacLBPoutcomemeasurethatusedpa-tientinputduringthedevelopmentprocess;theinstru-mentneedstobevalidatedandpsychometricallytestedinacLBPpopulationbeforebeingconsideredforuseinacLBPclinicaltrialTheODIisacLBPoutcomemeasurethatfulfillsFDAre-quirementsforuseincLBPclinicaltrials;itseemstobebetterthanotherinstrumentsatdetectingchangeinchronicandmoreseverelydisabledpatientsTheinstrumentdoesnothaveaclearrecallperiodItisreportedtobemostsensitiveforpatientswithmildtomoderatedisabilityduetocLBP;theRMQDwouldbesuitableforuseincLBPclinicaltrialsifitcoveredtherequirementofpatientinvolvement TheBPI-SFisanoutcomemeasurethatfulfillsFDAre-quirementsforuseinpainclinicaltrials,includingthosespecifictoLBP;theBPIhasbeenvalidatedinpatientswithLBPbutnotspecificallyforthosewithcLBPThereisnoevidencetorecommendtheinstrumentasanoutcomemeasureforcLBP (continued) Downloaded from https://academic
s .o
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pa inati .co
edim onsivepopul m/pain
n-relat RespcLBP N/A YesNo No No No Yes YesYes Yes No medicin
ndpai cLBP e/article
paina abilityinulation -abstrac
uate Relipop N/A YesYes No No No Yes YesYes actsYes No t/18/6
al mp /1
v P 0
PROinstrumentsthate ofValidatedincLBputpopulation ualities)N/A NoYes PQAS-RNo No No Yes YesYes qualities)andpain-relatediYes No 98/3062384 by guest on 27 M
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evidencefo Evidencpatienti cription(painNo NoNo Onlyfor Yes mpactsYes Yes NoNo scriptions(paiYes uleNo rch 2019
yof des R edi mde mod
able2Qualit strument omain:symptomBP-BS PSID-Q QASandPQAS- F-MPQ-2 omain:pain-relatLBP-IQ DI DIMDQ omains:symptoPI-SF ODEMS-Spine
T In DL NP P S DC O PR DB M
1104
ReviewofLow Back PainPRO Instruments
with both neuropathic and non-neuropathic pain condi-
n tions [27–33]. The instrument was originally validated in
paia or patients with carpal tunnel syndrome. No patient in-
ofin sf volvement was reported in the development of the
entuse ment PQAS,butpatient inputwassoughtduringthe cognitive
easuremndedfor require tvceahslaitdinnitggye,s,tainigttee.rhnAaasllthocnuoognthsisbthteeeennicnys,tvraualmidneadntetdahbaisfloitgyrootthdoecodcneLtteeBncPtt
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omments heOMPQwasnotdesignedfinclinicaltrials;itisnotrecocLBPclinicaltrialheinstrumentdoesnotcoveruseinaclinicaltrial TtoQcpoioohfrauiRngmteehniensppilcaovtaariinioerlsienagnhei.nnnewdvdsnaeetisSTrrrriuenshevhimoeooe(dnSnremet-FnnvaFR-eteenMoaeuldowrsvPrmpuoiatsQehrpseedM-ead2vtocffh)aoifrGilwscriSdptitlahaalhpssotiPehnaeraodidtanqirenstuviscnFvaeQeoeollsiountrprsyspmedamieositftiodtneieoironnnMottntbossfcnootGapft[hw3hiraiirlso4iletllnhv]q.teidyuPudTpeeariheoasineas--- ownloaded from https://academ
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dated in populations with diverse chronic pain .o
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ponsiveinPpopulation otavailable. ccathoonednndtcesiLtneiBotnnPsvsiatp.ilvioditTpiytyhut,eloainticotSehnFra.n-nMaglPec,Qob-n2ustisihtteannseceydd,escmotonovnbesertgrvaeatneltiddvaatgelidodiotyidn, p.com/painm
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RecL No No ¼ Measuresthat AssessPain-RelatedImpacts dic
A in
N/ e
LBP ain; OswestryDisabilityIndex /artic
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Reliabilityinpopulation No No Plowback¼ TdrOtieohiDcsneatIsrtiwO,ectasientwshdceelousbrdeitygrxinyitndegaDinslLiltasyBabtvobPilaiit.lwlyiitdTyhaihnictIenehddaOdeaixunDplIt(pseOharDsatwioesI)itnnhwd’tsseaLmsfwBuoinPdthncests[si3toirpg5anni–ntaee3eldd7lec].tvgooeonTldoihnidies-- -abstract/18/6
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ValidatedincLBPpopulation No No DrugAdministration;L rrceehlsPirapoaboniinnilcitsyDip,viaestinaenebsasti-lsnirtdeyttoesInsedctvheeraxreenliagdbeisi,laitpby,ailirtctyioc.unlvaerrlygeinntpvaatliiednittys, wanitdh 098/3062384 by gues
d The Pain Disability Index (PDI) was designed to measure t o
an the extent to which chronic pain interferes with a per- n 2
Evidenceofpatientinput No Yes n;FDAFood¼ Tswcspohhitneaehc’nsPigfgiDeeao,dIbo)wibd.lituaTytschotheovnaaPvdleieDdnrvaIggatwaeerigndaatesbinlvefinoapultievadnastidtitrey-iontroeutastsnebdwselitfietiwnhretaaelciscarhntbriavroileilintltysiyeipcscaoopnn[na3dssi8invis–det4(one1eotno]stt. 7 March 2019
ai nothaveaclear recallperiod.
p
ed ack
u b
n w Roland-MorrisDisability Questionnaire
nti lo
o c
C ni The Roland-Morris Disability Questionnaire (RMDQ) is a
o
2 ent PI chr measure that assesses physical disability due to LBP
e m Q M ¼ [42–47]. This instrument was originally validated in pa-
Tabl Instru OMP WHY cLBP ttieennctsy,wteitsht-rLeBtePs.tTrehleiabRilMityD,Qcohnavderggeonotdvainlitdeitryn,alancdonwsiass-
1105
Ramasamy etal.
moderately sensitive to change. The RMDQ would likely [59–63]. Measurement properties of the OMPQ have
be suitable for use in cLBP clinical trials to support a been assessed in patients with acute or subacute back
product label, except that there was no documentation orneck pain, but the instrument has not been validated.
of patientinvolvement duringinstrumentdevelopment. The developer does not report any patient involvement
in the development of the instrument. The OMPQ has
been shown to have good test-retest reliability and abil-
Chronic Low BackPainImpact Questionnaire itytodetect change,but variablepredictive validity.
The Chronic Low Back Pain Impact Questionnaire
(CLBP-IQ) is a new instrument that has been designed WestHaven–Yale Multidimensional PainInventory D
o
to assess the impact of cLBP for use in clinical trials (InterferenceScale) wn
[48].The CLBP-IQwasdeveloped usinginputfromsub- lo
a
jects taking daily analgesicmedications for at least three The West Haven–Yale Multidimensional Pain Inventory de
months for their cLBP. Concept elicitation interviews d
were conducted to gather information about the symp- (WHYMPI; also known as the MPI) was developed to fro
assess several dimensions of the chronic pain experi- m
toms and impacts experienced by subjects. Cognitive ence and was founded in cognitive-behavioral theory h
interviews were conducted to evaluate the clarity and [64–72]. The WHYMPI has not been validated. Patient ttps
comprehensivenessofthedraftinstrumentitemsandin- involvement during the development process differed for ://a
structions. No published clinical trials or studies have c
each of three parts of the measure. The WHYMPI was a
used the CLBP-IQ. The development article is the only reported to have good internal consistency, test-retest de
m
published literature on the CLBP-IQ. This instrument reliability, convergent validity, and ability to detect ic
hasnotbeen validatedinanypatientpopulation. .o
change. u
p
.c
Measures ThatAssess Symptom Descriptions(Pain om
Qualities) andPain-Related Impacts Discussion /p
a
in
We identified 13 instruments that have the potential to m
TheBrief PainInventory andBrief PainInventory e
assess key patient-reported concepts surrounding cLBP d
ShortForm ic
symptoms and impacts. Several of these instruments in
e
havestrongpsychometricproperties,butnoneofthein- /a
The Brief Pain Inventory and Brief Pain Inventory Short struments fulfilled criteria for use in clinical trials to sup- rtic
Form (BPI/BPI-SF) measure the intensity of pain and in- le
terference of pain in the patient’s life [49–53]. This in- port labeling claims based on FDA PRO guidance. -a
strument has been validated in patients with LBP, but Wsehairlechseuschweerveidneontcaebmleigtohtloecxaistteinpudbelvisehloepdedr’oscfuilmese,notau-r bstra
not specifically for patients with cLBP. The BPI showed c
ggoenotdvinatliedrintya,l acnodnsissetennscityiv,ittyestto-rectheastngreel,iabanilidty,dcisocnrvimeri-- tdiounctetdo indduicriantge ththaet thdiseveclroitpicmalensttepprhoacdessb.eenIdecaollny-, t/18/6
identification or development of an appropriate PRO in- /1
nated amonglevelsofpain severity. 0
strument should be done early in a clinical development 98
program, but our purpose was to evaluate PRO instru- /3
0
ments that would be suitable for the purpose of pursu- 62
MusculoskeletalOutcomesData Evaluationand ingalabelclaim,not justtobeusedinaclinicaltrial. 38
4
ManagementSystem–SpineModule b
y
PRO instruments can be developed for a specific mea- g
The Musculoskeletal Outcomes Data Evaluation and surement strategy, or if acceptable candidate instru- ue
s
Management System (MODEMS)–Spine Module was ments can be identified or potentially modified to t o
originally developed to measure spine-related musculo- adequately assess a clinical trial end point, it may be n 2
skeletal diseases and injuries [54–58]. The MODEMS- possible to avoid the burden of new instrument devel- 7 M
Spine Module has not been validated in a patient popu- opment. The FDA requires documentation of several a
lation. There is no available documentation of patient in- key aspects of a PRO if the goal of the sponsor is to rch
volvement in the development of this measure. There is use the PRO for a drug label [11]. Evidence of patient 20
1
also no evidence of reliability data or ability to detect input during the PRO development is important to en- 9
change. sure that the instrument is based on concepts relevant
to the patient’s experience. When using or modifying an
existing instrument and the development history is not
Orebro MusculoskeletalPain Questionnaire available, sponsors should provide documentation of
content validity, including open-ended patient input from
The Orebro Musculoskeletal Pain Questionnaire (OMPQ) the appropriate patient population. Seven of the 13 in-
was designed as a screening tool for people who have struments in this review had no documentation of pa-
suffered back injuries to determine which patients are at tient involvement, and of the six instruments that did
risk of developing persistent back pain or disability and have documented patient input, only three were vali-
which patients are most in need of early intervention dated inpatientswith cLBP.
1106
ReviewofLow Back PainPRO Instruments
The PRO developer should also provide documentation wefindthatthePROMISinstrumentsarerecommendedby
supporting the choice of recall period(s). The optimal re- theTaskForceasusefultocollectvaluableandmeaningful
call period will depend on the purpose and intended informationonthepatientexperience[74].AsPROsarenot
use of the instrument, including the variability, duration, a major emphasis of the Task Force report, our findings
frequency, and intensity of the concept measured, char- could potentially supplement the information in the report
acteristics of the disease, and the treatment [11]. A criti- by providing a summary of how well various PROs assess
cal aspect of the recall period is the patient’s ability to thesymptomsandimpactsofcLBP.
accurately recall the information requested. Four of the
13 instruments examined had no recall designated, one This early step in assessing the appropriateness of ex-
instrument used various recall periods ranging from isting measures is only a first start in the selection pro-
D
weeksto months,three instrumentsusedarecallperiod cess. The steps outlined here need to be completed in o
w
of the “past week,” and two instruments had a recall order to clarify whether these measures are the most nlo
period ofthelast24hours. appropriate pathway forward, whether a modification of a
d
any of them might be advisable, or if a new measure ed
The PRO developer must also provide documentation needs to be developed to address the symptoms and fro
m
supporting the choice of response options. Possible re- impacts of cLBP from the patient perspective. Any se-
h
sponse options for PROs include visual analog scales, lection or development of a cLBP-specfic measure ttp
Lreikceorrtdsincgaleosf,reavtienngtsscaaslest,hpeiyctoorciaclusrc.alRese,spcohencseklisotsp,tiaonnds wevoaululdatendeedintoeagrloy-pthhraosueghtritahlse upsriuoarl tporoucseessinofpbiveointagl s://ac
a
should be clear and appropriate for the intended popula- studiestosupportlabelclaims. d
e
tion, offer a clear distinction between choices, avoid po- m
ic
tential ceiling or floor effects, and avoid bias in the In summary, this review describes existing PRO instru- .o
u
direction of responses [11]. The number of options should ments that have the potential to be used for patients with p
be empirically justified [11]. A variety of response options cLPB. None of the instruments reviewed met the FDA’s .co
m
werenotedinthePROinstrumentsreviewedhere. criteria for use in clinical trials of cLBP to support labeling /p
claims, leaving the next step to be decisions on whether ain
Content validity, reliability, and the ability to detect an existing measure might be modified or if a new PRO m
e
change are critical elements that are used by the FDA instrumentneedstobedevelopedforthispurpose. d
ic
to evaluate an instrument to support potential labeling in
e
claims. Content validity, the degree to which the instru- /a
ment measures the concept of interest, should be sup- rtic
ported by qualitative studies to evaluate the items and Acknowledgments le-a
domains of an instrument in the target population. bs
According to the FDA, content validity should be estab- Julia R. Gage of Gage Medical Writing, LLC, provided tra
c
lished before other properties of the instrument can be editorialsupport. t/1
assessed. Reliability of an instrument (ability to provide 8/6
consistent, reproducible estimates of a treatment effect) /1
0
and the ability to detect change are important to docu- References 98
ment improvement or worsening of PROs in patients /3
1 Manek NJ, MacGregor AJ. Epidemiology of back 0
participatinginclinicaltrials.Oftheinstrumentsreviewed disorders: Prevalence, risk factors, and prognosis. 623
here, most had been tested for these properties, al- 8
Curr OpinRheumatol 2005;17:134–40. 4
thoughmostwere notevaluatedinpatients withcLBP. b
y
g
2 Bombardier C. Outcome assessments in the evalua- u
In recognition of the importance of PROs and to support e
the development of PRO instruments, the Patient-Reported tion of treatment of spinal disorders: Summary and st o
general recommendations. Spine (Phila Pa 1976) n
Outcome Measurement Information System was initiated 2
2000;25:3100–3. 7
andfundedbytheNationalInstitutesofHealthin2004(12). M
ThegoalofPROMISistodevelopuniversallyrelevantmea- a
3 Mannion AF, Elfering A, Staerkle R, et al. Outcome rc
sures rather than disease-specific measures [13]. The h
assessment in low back pain: How low can you go? 2
PROMISsystemincludesfourinstrumentsthatwerepoten- 0
tial candidates for further review: pain behavior, pain inter- Eur SpineJ2005;14:1014–26. 19
ference, pain intensity, and physical function mobility
questionnaires [73]. These generic measures were not se- 4 Ferrer M, Pellise F,EscuderoO, et al. Validation ofa
lectedinourreviewbecausethemeasureswerenotdevel- minimum outcome core set in the evaluation of pa-
oped for use by patients with cLPB and because item tients with back pain. Spine (Phila Pa 1976) 2006;31:
banks were deemed too broad for use in a cLBP trial. A 1372–9.
suitable measure could be constructed using specific items
from the PROMIS item banks; however, such a measure 5 Mannion AF, Junge A, Taimela S, et al. Active ther-
would still need to undergo further testing and validation apy for chronic low back pain: Part 3. Factors
with cLBP patients. In reviewing the National Institutes of influencing self-rated disability andits change follow-
Health Task Force standards for clinicians who treat cLBP, ingtherapy. Spine (PhilaPa 1976)2001;26:920–9.
1107