final report mapping corrected march 2018 - efcca report mapping 2018.pdf · final report january...

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MappingofInnovativeTreatmentsandDevices

inEFCCAMemberCountries

FINALREPORT

January2018

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Compiledby

ProjectCoordinator:

SannaLönnfors

EFCCAWorkingGroup:

MarcDerieppe

AlejandroKhalilSamhanArias

MarkoPerovic

Thisprojectreceivedanunrestrictedmulti-stakeholdergrantbyCelltrion,Pfizer,TakedaandOtsuka.

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Contents

1.INTRODUCTION...................................................................................................................................5

2.METHODS............................................................................................................................................5

2.1.Pilotphase....................................................................................................................................5

2.2.Secondphase...............................................................................................................................6

3.RESULTS...............................................................................................................................................7

3.1.Participants..................................................................................................................................7

3.1.1.NumberofIBDpatientsinparticipatingcountries................................................................7

3.2.Healthcaresystems.....................................................................................................................8

3.2.1.Question5:Describethehealthcaresysteminyourcountry..............................................8

3.2.2.Question6.Isahealthinsurancemandatoryinyourcountry?............................................8

3.2.3.Question7.Whopaysaperson’shealthinsuranceinyourcountry(thepersonhimself/herself;thestate;theemployer)?.....................................................................................9

3.2.4.Question8.Howdoesapatientgetcovered/reimbursedinyourcountry?.....................10

3.2.5.Question9.Arethereregionaldifferencesinyourcountryintermsorhealthinsuranceorhealthcaresystem(incaseofafederationetc.)?........................................................................11

3.2.6.Question10.CanapatientchooseGP/specialist/pointofcarefreely?..........................12

3.2.7.Question11.DoesaccesstoorreimbursementofIBDorotherchronicillnessmedicationinyourcountryrequireanofficialrecognitionofdiagnosis(e.g.amarkingonhealthinsurancecard)?............................................................................................................................................13

3.2.8.Question12.HowwouldyoudefinetheunmetneedsandprioritiesforIBDpatientsintermsofaccesstonewinnovativetherapiesinyourcountry?....................................................13

3.3.Medicines:Biologics...................................................................................................................15

3.3.1.Humira(adalimumab,anti-TNF)..........................................................................................16

3.3.2.Remicade(infliximab,anti-TNF)..........................................................................................17

3.3.3.Simponi(golimumab,anti-TNF)..........................................................................................18

3.3.4.Cimzia(certolizumabpegol,anti-TNF)................................................................................19

3.3.5.Entyvio(vedolizumab,anti-integrinagent).........................................................................19

3.4.Medicines:Biosimilars................................................................................................................20

3.4.1.Inflectra(infliximab)............................................................................................................20

3.4.2.Remsima(infliximab)...........................................................................................................22

3.5.Devices/techniques..................................................................................................................23

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3.5.1.OtsukaAdacolumn(apheresis)...........................................................................................23

4.DISCUSSION.......................................................................................................................................23

5.EFCCA’SRECOMMENDATIONS..........................................................................................................25

References............................................................................................................................................26

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1.INTRODUCTION

European Federation of Crohn’s and Ulcerative Colitis Associations (EFCCA) is an umbrella

organizationrepresenting34nationalIBDpatientassociations.EFCCAaimstoimprovethequalityof

lifeforpeoplewithIBDandgivethemaloudervoiceandhighervisibilityacrossEuropeandbeyond.

Nationalhealthsystemsandaccesstovarioustreatmentstendtobeunequal indifferentEuropean

and non-European countries with representation in EFCCA, in particular when dealing with

new/innovativetreatmentsanddevices.Insomeofthesecountries,therearenonationalregistries

of IBDpatientsavailablethatwouldassist inobtaining informationabout IBDpatients.Thisproject

aimedtoreducehealth inequalities inEuropebycarryingoutathoroughmappingofall innovative

treatments and devices (biologics, biosimilars, apheresis) available in EFCCA member countries.

Sevennational IBDassociations (Finland, France,NewZealand, Poland, Serbia, Slovenia and Spain)

participatedinthepilotphasefromOctobertoDecemberin2016totestanonlinesurveydeveloped

byEFCCA.

The pilot phasewas followed by a second phase in 2017 in which data was collected from other

member associations of EFCCA. This final report is a general overview of the situation in EFCCA

member countries, including the level of involvement of different payers and the role of national

health authorities. The outcomes of the project will support the exchange of knowledge and

experience among countrieswith representation in EFCCA andpromote a better understanding of

European healthcare systems, improve themobility of peoplewith IBD in European countries and

facilitate the access to treatment in other countries. Finally, the outcomes can be used to display

discrepanciestoEuropeanpolicymakersandtostresstheimportanceofequalaccesstotreatment.

2.METHODS

2.1.Pilotphase

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National associations from Finland, France, New Zealand, Poland, Serbia, Slovenia and Spain

volunteeredtoparticipateinthepilotphase,whichwascarriedoutinOctober-December2016.The

aimofthepilotphasewastotestanonlinesurveythatwascreatedbytheEFCCAworkinggroupand

uploadedontheLimesurveyplatform,selectedbyEFCCAITpersonnel,andtofinetunetheproject

and shape the following phases. The volunteers were asked to fill out the online survey and give

feedbackaboutittoEFCCA.Inmostpilotcountries,thesurveywasfilledbythepatientassociationin

cooperationwithgastroenterologists.Thereweresometechnicalandcontentissuesthatweretaken

careofbeforemovingontothenextphase.

2.2.Secondphase

Afteroptimizingthesurvey,otherEFCCAmembercountrieswereinvitedtoparticipate.Thesurvey

wasopenfromMarch2017untilallnationalassociationshadcompleteditinDecember2017.Apdf

fileofthesurveyquestionswassenttotheparticipantsinadvancesotheycouldlookforthe

informationneededandpreparetheiranswers.Respondentswerealsoencouragedtocross-check

theinformationwiththenationalauthoritiesintheircountry.Theprojectproceededasfollows:

Time Action Participants Summer2016

IdentifyingpilotcountriesDevelopingtheworksheet

EFCCAworkinggroup �

September2016

Launchingthepilotphase EFCCAworkinggroupPilotcountries

�

February2017

Interimreport EFCCAworkinggroup �

February2017

Optimizingthesurveybasedonthefeedbackcollectedduringthepilotphase


March2017

Launchingthesecondphasewithmorecountries

EFCCAworkinggroupEFCCAmembercountries

�

Summer2017

Closingthesecondphase EFCCAworkinggroupEFCCAmembercountries

�

Late2017 Analyzingtheoutcomes,generatingthefinalproduct


Early2018 Finalreportanddevelopmentofrecommendations


2018 EventatEuropeanParliament? EFCCA Figure1.Scheduleoftheproject

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Afactsheetconsistingofanswerstoallsurveyquestionswasgeneratedforeachparticipating

country.Furthermore,acomparisontablethatincludedallparticipatingassociationswasgenerated

foreachquestionofthesurvey.

3.RESULTS

3.1.Participants

Allinall,thirty-twonationalassociationsparticipatedinthepilotphaseandthesecondphaseofthe

survey. The participating national associations were Argentina, Austria, Belgium Flemish / French,

Bulgaria, Croatia, Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece,

Hungary, Iceland, Ireland, Israel, Italy, Malta, New Zealand, Norway, Poland, Portugal, Romania,

Serbia,Slovakia,Slovenia,Spain,Sweden,SwitzerlandandtheUnitedKingdom.

The results have been listed with all participants together as well as associations from European

countries (n=29) and non-European countries (n=3) separated. Argentina, Israel and New Zealand

wereseparatedintothenon-Europeancategory.

3.1.1.NumberofIBDpatientsinparticipatingcountries

TheavailabilityandsourceofaprecisenumberofIBDpatientsorprevalenceofIBDvariedgreatlyin

the participating countries. In very few of the countries there was a patient registry available. In

Finland,forexample,theSocial InsuranceInstitute isabletoprovideanumberofpersons(45000)

who receive medication coverage based on IBD diagnosis, whereas Serbia, for example, while a

registry of IBD patients exists, the data is incomplete and the number of patients (7000-8000) is

estimatedbythegastroenterologistsdealingwithIBD.InIreland,anestimatehasbeenextrapolated

fromthemostrecentcensus.

Someestimateswereverywide;forexample,inAustriatheestimateis40to80000IBDpatients,and

in Bulgaria some physicians estimate the number of IBD patients to be about 18 000, whereas

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accordingtotheNationalHealthInsuranceFund,about3000receiveIBDmedicationsco-financedby

theFund.OnlySwedencouldprovideanexactnumber(41361patientsasofJune30,2017)basedon

SWIBREG, the Swedish Inflammatory Bowel Disease Registry, and France could provide a number

fromanIBDobservatorywww.observatoire-crohn-rch.fr(145220patients).

3.2.Healthcaresystems

3.2.1.Question5:Describethehealthcaresysteminyourcountry.

Mostoftheparticipatingassociations(17associations,53%)reportedthatpatients intheircountry

are covered by a state insurance, same for all citizens, funded through taxation and a possible

voluntaryadditionalprivate insurance.Sixassociations, respectively, reported thatpatients in their

countryarecoveredbyastate insurance,sameforallcitizens,fundedthroughtaxation(19%),ora

combinationofstateandprivateinsurance(19%).Oneassociationreportedonlyprivateinsurancein

theircountry(3%)andoneassociationreportedasystemofseveralsicknessfunds,ofwhichacitizen

choosesone(3%).Oneassociationreportedastatutorycontributionsystem,notfittingtoanyofthe

alternativesoffered(3%).

When European and non-European countries were separated, 52% of the European associations

reportedthatpatientsintheircountryarecoveredbyastateinsurance,sameforallcitizens,funded

through taxation and a possible voluntary additional private insurance. Six associations (21%)

reported that patients in their country are covered by a state insurance, same for all and funded

throughtaxation,andfiveassociations(17%)reportedacombinationofstateandprivateinsurance.

Three associations (10%) reported another policy (see above). In the non-European countries,

ArgentinaandNewZealandreportedacombinationofstateandprivateinsuranceandIsraelastate

insurance,sameforallcitizensandfundedthroughtaxation.

3.2.2.Question6.Isahealthinsurancemandatoryinyourcountry?

Eleven of the participating associations (34%) reported that everyone is required to have a health

insurance in theircountry,either fullybythestate, fullyprivate,orpartiallybythestate/partially

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private. Fourteen associations (43%) reported that everyone in their country is by default 100%

insured by the state. Seven associations (22%) reported that health insurance is notmandatory in

their country (but two of these countries specified that it is not mandatory because everyone is

insuredbythestate).

When European and non-European countries were separated, in Europe ten associations (34%)

reported that everyone is required to have a health insurance in their country, either fully by the

state, fully private, or partially by the state / partially private, thirteen associations (45%) that

everyone in their country is by default 100% insured by the state, and six associations (21%) that

health insurance is not mandatory in their country (see above). In the countries outside Europe,

Argentina reported that everyone is by default 100% insured by the state, Israel that everyone is

requiredtohaveahealthinsuranceintheircountry,eitherfullybythestate,fullyprivate,orpartially

bythestate/partiallyprivateandNewZealandthathealthinsuranceisnotmandatory.

3.2.3.Question7.Whopaysaperson’shealthinsuranceinyourcountry(thepersonhimself/herself;thestate;theemployer)?

Twenty-twoparticipatingassociations(69%)reportedthatintheircountry,healthcareisfinancedby

employers, employees and/or the state together. In many of the countries that reported a

combination system, thepayer largelydependsonwhether theperson isemployed, inwhich case

theemployercoversapart,otherwiseitiscoveredbythestate.Twoassociations(6%)reportedthat

theemployerpaysthehealthinsuranceintheircountry,oneassociation(3%)thatthestatepays,and

oneassociation(3%)reportedanotherpolicy.

Sixassociations(19%)reportedthatintheircountry,thepersonhim/herselfpaystheinsurance.The

Bulgarianassociation,however,alsospecifiedthatalthoughbydefaultthepersonhim/herselfpays

the contribution, the state also insures a numberof citizen groups, such as students, children and

retiredpeople.Furthermore,theBulgarianandItalianassociationsalsoreported inQuestion5that

all citizens have a state insurance funded through taxation; itmay be that in these cases, the tax

contributionshavebeenseenasdirecthealthinsurancecontributions.

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WhenEuropeanandnon-Europeancountrieswere separated, inEurope twentyassociations (69%)

reported that in their country, health care is financed by employers, employees and/or the state

together. Five associations (17%) reported that in their country, the person him/herself pays the

insurance. Two associations (7%) reported that the employer pays the health insurance in their

country,oneassociation(3%)thatthestatepays,andoneassociation(3%)reportedanotherpolicy.

IncountriesoutsideofEurope,ArgentinaandIsraelreportedacombinationsystemandNewZealand

thatthepersonhim/herselfpays.

3.2.4.Question8.Howdoesapatientgetcovered/reimbursedinyourcountry?

Nineoftheparticipatingassociations(28%)reportedthat intheircountry,thepatientpaysnothing

as insurance covers everything. Denmark specified that patients pay only a part of medicines

themselves,andNorwayspecifiedthatthepatientpaysafeeofapproximately210eurosperyear,

anythingafterthatisdirectlycoveredbythestatetodoctors,hospitalsandpharmacies.

Elevenoftheparticipatingassociations(34%)reportedthatintheircountry,thepatientpaysapart

atthepointofcareandtheinsurancecoverstherest.Argentinaspecifiedthatthisisusuallythecase,

apart from certainmedicines (biologics) which are included in a “specific fund created for certain

high-costdrugs”,andifapersonhasacertifieddisability,medicinesare100%coveredbythestate.

Sloveniaspecifiedthatalmostallarealsoinsuredforthepartthatshouldbepaidatthepointofcare.

Twoassociations(6%)reportedthatintheircountry,thepatientpayseverythingatthepointofcare

andgetspartiallyreimbursedlater.Francespecifiedthatifthepatienthasadiseasethatisregistered

inthelistofchronicdiseasessetbythegovernment,thepatientwillreceivefullreimbursementfor

theexpensesrelatedtothedisease.Insomehospitalsandstateinstitutionsthepatientpaysnothing;

in private clinics or medical and paramedical institutions, the patient pays a part and private

insurancecoverstherest,inwhichcasethepatientmayhavetopayaheadandbereimbursed.Much

of the time, the institution is paid directly by the State and the private insurance plan. Portugal

specifiedthatbiologicmedicinesarealwaysfullycoveredandthereforefreeforthepatient.

Tenoftheparticipatingassociations(31%)reported“somethingelse”.Austriaspecifiedthatatsome

private doctors the patient will be reimbursed later and that for registered medicines you pay a

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certainamount,otherwisethefullprice.Finlandspecifiedthathealthserviceshaveanupperlimitper

calendaryear,beyondwhichthepatientdoesnothavetocontinuepayingfees.Germanyspecified

thattheinsurancecoversthecostsinthestatutoryhealthinsurancebutpatientshavetopayupto

10eurosinco-paymentsforprescriptionmedicinesand10eurosperdayforhospitalstays.Greece

specifiedthatforseeingadoctorregisteredinthehealthcaresystemthepatientpays10euros,for

seeinganon-registereddoctorthepatientpayswhateverthedoctorasks.Formedicationthepatient

paysacertainpercentageoftheprice(25%incaseofIBD).Irelandspecifiedthatitdependsonthe

typeofinsurance:ifthetreatmentiscovered,thepatientpaysnothing,ifnot,thepatienthastopay

upfrontandmaynotbereimbursedlater.NewZealandspecifiedthattheirpublichealthsystemis

universalandnomoneychangeshands.Swedenspecifiedthatyoupayamaximumof180eurosper

yearformedicinesandnothingabovethat.

When European and non-European countries were separated, in Europe nine associations (31%)

reported that in their country, the patient pays nothing as insurance covers everything. Nine

associations(31%)alsoreportedthatthepatientpaysapartatthepointofcareandtheinsurance

coverstherest.Twoassociations(7%)reportedthatintheircountry,thepatientpayseverythingat

thepointofcareandgetspartially reimbursed later,onenineassociations (31%)reportedanother

policy(seeabove).IncountriesoutsideEurope,ArgentinaandIsraelreportedthatthepatientpaysa

partatthepointofcareandtheinsurancecoverstherestandNewZealandreportedanotherpolicy

(seeabove).

3.2.5. Question 9. Are there regional differences in your country in terms or healthinsuranceorhealthcaresystem(incaseofafederationetc.)?

Most participating associations (26 associations, 81%) reported no regional differences in their

country.Sixassociations(19%)reportedregionaldifferencesintheircountry.Argentinaspecifiedthat

eachofthe24jurisdictionsinthecountryhavetheirownlawsregardinghealth,andAustriaspecified

thateachofthecountry’sfederationshasitsowninsurance.NewZealandreportedthatalthoughthe

country’spublichealthcareisuniversalandsameeverywhere,accesstohealthcaresystemwillvary

due to geography and distribution of specialists. Furthermore, private health insurance coverage

variesbetweencityandregionaccordingtosocio-economicfactors.Spainreportedthatthecountry

has17regionalhealthservices,andnotallof themwork in thesamewaytosupport theneedsof

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patients and their families. The United Kingdom specified that health is a devolved function in

Scotland,WalesandNorthernIreland,withdifferentstructures,policiesandpriorities;forexample,

prescriptionsarenotchargedfor inWales,ScotlandorNorthernIreland,butchargesapply,subject

tocertainexemptioncriteria,inEngland.

When European and non-European countries were separated, 25 associations (86%) reported no

regional differences in their country. Four associations (14%) reported regional differences (see

above).IncountriesoutsideofEurope,ArgentinaandNewZealandreportedregionaldifferences(see

above)andIsraelreportednone.

3.2.6.Question10.CanapatientchooseGP/specialist/pointofcarefreely?

Twenty-two of the associations (69%) reported that in their country, a patient can freely choose

his/herGP,specialist,orpointofcare.Fiveassociations (16%)reportedthat in theircountry,aGP,

specialistorpointofcare isdefinedbythesystembasedone.g.wherethepatient lives.Argentina

specified that the patient should usually go to the appropriate family doctor and as for the

gastroenterologist,thepatientshouldoftenselectfromasmallnumberofprofessionalswithintheir

health coverage and jurisdiction, and therefore it is not alwayspossible to reach an IBD specialist.

Denmarkreportedthatpatientscannotchoosethespecialist,buttheycanchoosethehospital.New

ZealandspecifiedthateachpersonhasaGPandaccesstotheGP;thiscanbechanged if required.

Whenmovingfromprimarytosecondaryortertiarycareareferralisrequired.

Five (16%) associations defined another system from their countries: in Finland, the patient can

choose anyhealth centre for non-emergency care, and also the specialmedical careunit together

withtheirdoctor.InIcelandthepatientcanchoosetheirGP,whothenrefersthemtoaspecialist.In

Ireland,theGP,specialistandpointofcareareusuallybasedonwherethepatientlives,butassome

ruralhospitalsdonot,forexample,haveIBDnurses,patientstraveltoothercountiesformorevaried

services.InSwitzerlandthechoiceofdoctordependsonthetypeofinsuranceyouchoose,andinthe

UK,patientshavealegalrighttochooseaGPpracticethatbestsuitstheirneedsandareentitledto

askforareferral forspecialisttreatment.Whethertheyget it,however,dependsonwhattheirGP

feelsisclinicallynecessary.

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When European and non-European countries were separated, in Europe 21 associations (72%)

reported that in their country, a patient can freely choose his/her GP, specialist, or point of care.

Threeassociations (10%) reported that theGP, specialistorpointofcare isdefinedby thesystem,

andfiveassociations(17%)reportedanotherpolicy(seeabove).IncountriesoutsideofEurope,Israel

reportedafreechoiceofGP,specialistorpointofcare,whileArgentinaandNewZealandreported

thatGP,specialistandpointofcarearedefinedbythesystem(seeabove).

3.2.7. Question 11. Does access to or reimbursement of IBD or other chronic illnessmedicationinyourcountryrequireanofficialrecognitionofdiagnosis(e.g.amarkingonhealthinsurancecard)?

Sixteen of the participating associations (50%) reported that access to reimbursement of IBD

medication requires an official recognition of diagnosis in their country; one association (3%)

reported“somethingelse”.Austriaspecifiedthatthediagnosis, includingtheextentandlocationof

inflammation,needstobenotedontheprescriptionifrequiredbytheregistry.Estoniaspecifiedthat

withdiagnosis,thepatientcanbuymedicationwitha90%cheaperprice.FinlandandItalyspecified

thatthereneedstobeamarkingonthehealth insurancecard.Fifteenassociations(47%)reported

that no official recognition of diagnosis is needed for access to or reimbursement of IBD or other

chronicillnessmedications.

WhenEuropeanandnon-Europeancountrieswereseparated,inEuropefourteenassociations(48%)

reportedthataccesstoreimbursementofIBDmedicationrequiresanofficialrecognitionofdiagnosis

intheircountry;alsofourteenassociations(48%)reportedthatnoofficialrecognitionofdiagnosisis

neededforaccesstoorreimbursementofIBDorotherchronicillnessmedications.Oneassociation

(3%) reported another policy. Outside of Europe, Argentina and Israel reported that an official

recognitionisneeded,whileNewZealandreporteditisnotneeded.

3.2.8. Question 12. How would you define the unmet needs and priorities for IBDpatientsintermsofaccesstonewinnovativetherapiesinyourcountry?

Participatingassociationswerealsoaskedtodefine,intheiropinion,theunmetneedsandpriorities

of IBDpatients in termsofaccess tonew innovative therapies.Notall countries listed such issues,

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and although some of the responses givenmay have been personal opinions, some of the issues

listedwereratherstriking:

• Argentina: Those with public health coverage provided by the state see their access to new

therapiesmorerestricted,ascomparedtothosewithprivatecoverage.

• Austria:Numberofspecializedhospitalsandgastroenterologistsismuchtoolow.Diagnosesare

delayedasGP’sdonotsendpatientstogastroenterologists,andwaitingtimesinoutpatientcare

arelong.

• Belgium:Sometimespatientshavetowaitverylongfortreatmentwithbiologics;theymuststart

witha3-monthperiodofconventionaltherapy.

• Bulgaria: There is a need for improved knowledge on suchmedications for both patients and

doctors.

• Croatia:Biologictherapyisonlyavailableinbiggestcities.

• CzechRepublic:Fasteraccessisneeded.

• Estonia:Notmanypatientsarewillingtoparticipatetrialsonmedicines.

• Greece:Due to the economic crisis inGreece,most innovative therapies come to the country

laterthaninothercountriesasthelawstatesthatinordertodefinetheirpricetheyhavetobe

pricedin7differentcountriesoftheEUandin3outofthe7,ariskbenefitanalysisneedstobe

doneduringthepricing.

• Hungary:It isdifficulttocomeintouchwiththenewtherapies.Youneedtobeapatientinan

IBDcenter,filllotsofrequirementsandcompletealotofothertherapies.Ifyourconditiondoes

not improve on these therapies, you can get treated with new therapies, if the insurance

companyallowsit.

• Ireland:45%ofhospitalsinIrelandhavenoIBDnurse.ComparedtootherEUcountries,Ireland

alsofacesalackofgastroenterologists.

• New Zealand: There is geographical variation in access to specialist care, due to distance and

population distribution. Therapies are managed by a central government agency which limits

accessandrangeoftherapiesavailable.

• Norway:Norwayislateinusingnewmedications.

• Poland:AccessisdifficultforpatientswithCrohn’sdiseaseandillusoryforthosewithulcerative

colitis.

• Serbia:Thereisdelayintheapplicationoflatesttherapeuticoptionsduetothelongprocessof

theirapprovalbythehealthinsuranceauthorities.

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• Slovenia:Althoughpatientshaveaccesstonewtherapies,itmaybedelayedcomparedtoother

countries.

• Spain: Innovative therapies are nowadays the last option as regional governments have the

priorityofsavingmoney.

• UnitedKingdom:NewinnovativetherapiesareappraisedbytheNationalInstituteforHealthand

CareExcellence(NICE)andtheScottishMedicinesConsortium(SCM).Theappraisalprocesscan

takesomeconsiderable time.Access todefinedpsychological support/mentalhealthservices

andspecialistdieticiansisverylimitedandvariableacrosstheUK.

3.3.Medicines:Biologics

TheavailabilityofallmedicinesinallparticipatingcountriescanbeseeninFigure2.Informationfor

allthemedicinesseparatelyislistedonthefollowingpages.

Figure2.Availabilityofinnovativemedicinesintheparticipatingcountries.

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3

4

5

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Argentina

Austria

Be

lgium

Bulgaria

Croatia

Cyprus

CzechRe

public

Denm

ark

Estonia

Finland

France

Germ

any

Greece

Hungary

Iceland

Ireland

Israel

Italy

Malta

New

Zealand

Norway

Poland

Po

rtugal

Romania

Serbia

Slovakia

Sloven

ia

Spain

Swed

en

Switzerland

UnitedKingdo

m

AvailabilityofinnovativemedicinesinEFCCAcountries

Humira Remicade Simponi Cimzia Entyvio Inflectra Remsima

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3.3.1.Humira(adalimumab,anti-TNF)

Humira (adalimumab) isapproved to IBDpatients inallparticipatingcountries.Of theparticipating

associations,17(53%)reportedthatthemedicineisfullycovered,4(13%)thatitispartiallycovered,

and11(34%)thatcoveragerequiresIBDdiagnosisintheircountry.Threeassociations(9%)reported

regionaldifferencesintheircountryintermsofavailabilityorreimbursement.

Twelveoftheparticipatingassociations(38%)reportedthatintheircountry,themedicineisavailable

toallIBDpatients;18associations(56%)reportedthatitisavailableifcertainpreconditionsaremet.

Thepreconditions criteria variedgreatly. InPolandandRomania, forexample, theCrohn’sDisease

ActivityIndex(CDAI)isused,butinPolandtheCDAIlimitis>300,whereasinRomaniaitis>220.Two

associations(6%)reportedthatintheircountry,themedicineisavailableforCrohn’sdiseasebutnot

forulcerativecolitispatients.

Mostassociations(66%)reportedthatintheircountry,themedicineisadministeredathome.Many

oftheassociationsaddedthatthemedicineisusuallystartedatthehospital,afterwhichthepatient

canadministeritathome.25%oftheparticipatingassociationsreportedthatitisadministeredinthe

hospital,andthreeassociations(9%)reportedthatintheircountry,themedicineisadministeredin

bothhomeandhospital,whichmayalsohavemeantthatitisgivenfirstatthehospitalandlaterat

home.

OnlyBulgaria,Estonia,PolandandSerbiawereabletoprovideanestimateofhowmanypatientsare

receivingthetreatmentintheircountry(approximately300patientsinBulgaria,max.150inEstonia,

475inPoland[2015],andaround150inSerbia).TheBulgarianestimateisprovidedbythenational

health statistics, thePolishestimateby theNationalHealthFund,and theSerbianestimateby the

RepublicanFundforHealthInsurance.Othercountriescouldprovidenodata.

WhencountriesinEuropeandoutsidewereseparated,inEuropethemedicinewasreportedasfully

covered by 17 associations (59%), partially covered by 14 associations (14%) and requiring an IBD

diagnosisbyeightassociations(28%).InallcountriesoutsideofEurope(n=3),coveragerequiredIBD

diagnosis.

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3.3.2.Remicade(infliximab,anti-TNF)

Remicade (infliximab) isapproved to IBDpatients inallparticipating countries.Of theparticipating

associations,19(59%)reportedthatthemedicineisfullycovered,4(13%)thatitispartiallycovered,

and9(28%)thatcoveragerequiresIBDdiagnosisintheircountry.

Two associations out of the 32 (6%) reported regional differences in their country in terms of

availabilityorreimbursement.Spainspecifiedthatinmanyregionalgovernments,youcannotaccess

this product anymore and can only have the biosimilar instead. Germany specified that in some

regions,thereisa lackofdoctor’sofficeswithinfusionequipment.WhileItalyreportednoregional

differences, itwasspecifiedthat insomeregions, it isrequestedtousethebiosimilar,andinmany

regionsnaïvepatientsmustbetreatedwiththebiosimilar.

Fourteenassociationsoutofthe32(44%)reportedthatintheircountry,themedicineisavailableto

all IBDpatients;16associations (50%) reported that it isavailable if certainpreconditionsaremet.

Most associations specified that the patient usually has to try other medications first and that a

gastroenterologist’sopinionandprescription is required.Finland specified that themedicine isnot

usedforadultpatientsanymore.

Most associations (97%) reported that in their country, the medicine is administered at hospital,

outpatientclinicordoctor’soffice.OnlyIsraelreportedthatitisadministeredathome.

OnlyBulgaria,Estonia,PolandandSerbiawereabletoprovideanestimateofhowmanypatientsare

receivingthetreatmentintheircountry(<100patientsinBulgaria,max.150inEstonia,around700in

Poland[Remicadeandbiosimilarscombined],andaround200 inSerbia). TheBulgarianestimate is

based on the national health statistics, the Polish estimate on the National Health Fund, and the

Serbian estimate on the Republican Fund for Health Insurance. Other countries could provide no

data.

WhenEuropeanandnon-Europeancountrieswereseparated,inEuropethemedicinewasreported

as fully covered by 19 associations (66%), as partially covered by four associations (14%) and as

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requiringanIBDdiagnosisbyseven(25%)associations. IncountriesoutsideofEurope, inArgentina

andNewZealandthecoveragerequiresIBDdiagnosis,whileIsraelreportedthatthemedicineisfully

covered.

3.3.3.Simponi(golimumab,anti-TNF)

Simponi (golimumab) was reported as approved to IBD patients in their country by 25 of the

participatingassociations(78%).Outofthe25associationsthatreportedthemedicineisapproved,

14 (56%) reported that it is fully covered in their country, 3 associations (12%) that it is partially

covered and 8 associations (32%) that coverage requires an IBD diagnosis in their country. No

regionaldifferenceswerereported.

Outofthe25associationsthatreportedthemedicineisapprovedintheircountry,11(44%)reported

thatitisavailabletocolitisulcerativebutnotCrohn’sdiseasepatientsintheircountry.Nineofthe25

associations(36%)reportedthatthemedicineisavailableifcertainpreconditionsaremet,andthree

ofthemspecifiedthatit isusedonlyormostlyforulcerativecolitis.Fouroutofthe25associations

(16%)reportedthatthemedicineisavailabletoallIBDpatientsintheircountry.ApartfromGreece

mentioning that sometimes, especially in rural areas themedicine does not get to the patient on

time,noregionaldifferenceswerereported.

Outofthe25associations,14associations(56%)reportedthatthemedicineisadministeredathome,

7associations(28%)thatitisadministeredinhospitaloroutpatientclinic,andoneassociation(4%)

thatitisadministeredinboth.Threeassociationscouldnotanswer.

OnlyBulgariaandEstoniacouldprovideanestimateofthenumberofpatientsreceivingthemedicine

(<100patientsinBulgaria,max.150inEstonia).TheBulgarianestimatewasbasedonnationalhealth

statistics.Othercountriescouldprovidenodata.

When European and non-European countries were separated, the medicine was reported as

approved by 23 associations in Europe (79%). Out of those 23 associations, 13 associations (57%)

reported the medicine as fully covered and 3 associations (13%) as partially covered. Seven

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associations(30%)reportedthatcoveragerequiresanIBDdiagnosis.OutsideofEurope,themedicine

isapprovedinArgentina(fullycovered)andIsrael(coveragerequiresanIBDdiagnosis).

3.3.4.Cimzia(certolizumabpegol,anti-TNF)

Sixassociations (19%) reportedCimzia (certolizumab)asapproved to IBDpatients in their country.

Threeassociationsoutofthesix (50%)reportedthatthemedicine is fullycovered intheircountry,

one (17%) that it is partially covered and two (33%) that coverage requires an IBD diagnosis. No

regionaldifferenceswerereported.


thatitisavailabletoallIBDpatients,one(17%)thatitisavailableifcertainpreconditionsaremetand

one(17%)thatithasnotradinglicenseandhasbeenusedofflabelforsomepatients.

Threeof the six associations (50%) reported that in their country, themedicine is administered at

home, two associations (33%) that it is administered at hospitals and outpatient clinics, and one

association(17%)thatitisadministeredinboth.

Noneofthesixassociationswereabletoprovideanumberofpatientsreceivingthemedicine.


approvedbyfiveassociationsinEurope(17%).Outofthosefiveassociations,twoassociations(40%)

reportedthemedicineasfullycoveredand1association(20%)aspartiallycovered.Twoassociations

(40%)reportedthatcoveragerequiresanIBDdiagnosis.OutsideofEurope,themedicineisapproved

onlyinArgentina,whereitisfullycovered.

3.3.5.Entyvio(vedolizumab,anti-integrinagent)

Entyvio (vedolizumab) was reported as approved to IBD patients in their country by 22 of the

participatingassociations(69%).Outofthe22associations,13(59%)reportedthatitisfullycovered

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intheircountry,2associations(9%)thatitispartiallycoveredand7associations(32%)thatcoverage

requiresanIBDdiagnosisintheircountry.


thatitisavailabletoallIBDpatientsintheircountry.Tenofthe22associations(45%)reportedthat

themedicineisavailableifcertainpreconditionsaremet,withGreecespecifyingthatitisonlyused

bypatientswithmoderate to severeCrohn’sandAustria,BelgiumandHungary specifying that the

medicineisusedifanti-TNFtherapyfails.Twoofthe22associations(9%)reportedthatthemedicine

isavailableforCrohn’sdiseasebutnotulcerativecolitispatients,andoneassociation(5%)that it is

available for ulcerative colitis but not Crohn’s disease patients. Three associations (14%) reported

regionaldifferences,withGermanyspecifyingthatinsomeregionsthereisalackofdoctor’soffices

with infusion equipment, and Ireland specifying that the medicine is not available in some rural

hospitals.

All22associationsreportedthatthemedicineisadministeredinhospitalsordoctor’sofficesintheir

country.

OnlyEstoniacouldprovideanestimateofthenumberofpatientsreceivingthemedicine(max.150).

Othercountriescouldprovidenodata.



reported the medicine as fully covered and two associations (10%) as partially covered. Six

associations(29%)reportedthatcoveragerequiresanIBDdiagnosis.OutsideofEurope,themedicine

isapprovedonlyinIsrael,wherecoveragerequiresIBDdiagnosis.

3.4.Medicines:Biosimilars

3.4.1.Inflectra(infliximab)

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Inflectra (infliximab) was reported as approved to IBD patients in their country by 27 of the



coveredand7associations(26%)thatcoveragerequiresanIBDdiagnosisintheircountry.


that it isavailabletoall IBDpatients intheircountry.Fifteenof the27associations (56%)reported

thatthemedicineisavailableifcertainpreconditionsaremet.Austriaspecifiedthatthemedicineis

not in the reimbursement registry, and a specialized gastroenterologist has to senda letter to the

insurancecompany,andthecompanywilldecidewhethertheyagreeornot.Hungaryspecifiedthat

patients who started infliximab therapy after 2014 only get the biosimilar. Several associations

reportedthatthepreconditionsarethesameasfortheoriginatorRemicade.Fiveassociationsoutof

the27(19%)reportedregionaldifferences,withAustriaspecifyingthatitdependsontheinsurance

andagreement,Belgiumspecifying that itdependsonwhichhospitaladministerswhichbiosimilar,

and Germany specifying that in some regions there is a lack of doctor’s offices with infusion

equipment. Spain specified that the medicine is not available in every hospital and the United

Kingdom that local arrangements are made in terms of which biosimilar is used. Greece also

mentionedthatsometimesespeciallyinruralareasthemedicinedoesnotgettothepatientontime.

Allassociationsfromcountriesinwhichthemedicineisapprovedreportedthatitisadministeredat

home or outpatient settings. Italy also mentioned that biosimilars are the first choice in naïve

patients.

OnlyBelgium,BulgariaandEstoniacouldprovideanestimateofthenumberofpatientsreceivingthe

medicine(900-1000patientsinBelgium,<100inBulgaria,max.150inEstonia).TheBelgianestimate

wasgivenbyapharmacompanyandtheBulgarianestimatewasbasedonnationalhealthstatistics.

Othercountriescouldprovidenodata.



reported the medicine as fully covered and three associations (11%) as partially covered. Seven

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associations(26%)reportedthatcoveragerequiresanIBDdiagnosis.Themedicinewasapprovedin

anyofthecountriesoutsideofEurope.

3.4.2.Remsima(infliximab)

Remsima (infliximab) was reported as approved to IBD patients in their country by 25 of the



coveredand8associations(32%)thatcoveragerequiresanIBDdiagnosisintheircountry.


that it isavailable toall IBDpatients in theircountry.Elevenof the25associations (44%) reported

thatthemedicineisavailableifcertainpreconditionsaremet.Austriaspecifiedthatthemedicineis

not in the reimbursement registry, and a specialized gastroenterologist has to senda letter to the

insurancecompany,andthecompanywilldecidewhethertheyagreeornot,andseveralassociations

reported that the preconditions are the same as for the originator Remicade. Two out of the 25

associations(8%)reportedthataccessdependsontheregionoreconomicsituationintheregion.Six

associationsoutofthe25(24%)reportedregionaldifferences.Austriaspecifiedthatthemedicineis

not prescribed in some states, Germany specified that in some regions there is a lack of doctor’s

officeswith infusionequipment.Spainspecifiedthat themedicine isnotavailable ineveryhospital

andtheUnitedKingdomthatlocalarrangementsaremadeintermsofwhichbiosimilarisused.

Apart fromBulgaria,where themedicine isadministeredathome, themedicine isadministered in

hospitalsandoutpatientsettingsinallcountrieswhereitisapproved.

Only Belgium, Bulgaria, Estonia and Poland could provide an estimate of the number of patients

receiving the medicine (350-400 patients in Belgium, <100 in Bulgaria, max. 150 in Estonia and

around 700 in Poland [Remicade and biosimilars combined]). The Belgian estimatewas given by a

pharma company, the Bulgarian estimate was based on national health statistics and the Polish

estimateontheNationalHealthFund.Othercountriescouldprovidenodata.

23



reported the medicine as fully covered and four associations (17%) as partially covered. Seven

associations (29%) reported that coverage requires an IBD diagnosis. In the countries outside of

Europe,themedicinewasonlyapprovedinIsrael,wherecoveragerequiresIBDdiagnosis.

3.5.Devices/techniques

3.5.1.OtsukaAdacolumn(apheresis)

Sevenoutofthe32participatingassociations(22%)reportedthatOtsukaAdacolumn(theapheresis

technique) isavailable for IBDpatients their country. Inallof the sevencountries the treatment is

given in hospitals. None of the seven countries could provide data on the number of patients

receivingthetreatment.ThetechniquewasnotavailableinanyofthecountriesoutsideEurope.

4.DISCUSSION

It comes as no surprise that health systems, insurance coverage and reimbursement policies vary

from country to country; thiswas clear also in this survey. Among the participants of this survey,

therewerecountrieswithauniversal tax-fundedcoverage forall citizensaswell as countrieswith

onlyprivateinsurance,withseveralcountriesinbetweenwheredifferentcombinationsofstateand

privateinsurancesapply.Insomeoftheparticipatingcountries,apartfrompayingtaxes,citizenspay

nothing more for their health insurance; on the other hand, in some countries different health

insurancefeesapplybothtocitizensaswellastheiremployers.

Reimbursementpoliciesvaryaswell,rangingfromsystemswherethepatientpaysnothingandthe

insurancecoverseverythingtothosewherethepatientpaysoutofpocketatthepointofcareand

gets reimbursed later. If there is no backup system for personswith low income, e.g. students or

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retiredpersons,payingeverythingupfront,evenwithafullreimbursementlater,canbedifficultand

canevenleadtothepersonnotbeingabletopurchasehis/herprescriptionmedicines.

Theoutcomesof this surveyalso showclearly that access tonew innovative therapies indifferent

countriesisfarfromequal.OnlyHumira(adalimumab)wasapprovedandavailableforIBDpatientsin

all participating countries, and only five of the participating countries (Czech Republic, Finland,

Hungary, Ireland and Switzerland) could offer all of the five biologics and two biosimilars in this

survey to IBD patients. Apart from the differences between countries, there are also regional

differenceswithinthesamecountry;thesemaybeduetogeographicaloreconomicissues.Although

it is understandable that access to health care services in e.g. cities and rural areas can be very

different,itputspatientsinhighlyunequalpositions.

Not being able to choose one’s health care provider, as was the case reported by 16% of the

participating associations, may have an impact on the patient’s quality of care and life; a good

patient-physicianrelationshiphasbeenassociatedwithbettercompliancetotreatment(Kerseetal.,

2004), and inapreviousEFCCA survey54%of the respondents felt that theywerenotable to tell

theirphysiciansomethingpotentially importantabouttheir illness(Lönnforsetal.,2014)–possibly

duetotrustissuesorotherdifficultiesinthepatient-physiciancommunication.Beingabletochoose

ahealthcareprovider, insteadofbeingappointedtoone,couldbehelpful indevelopingapatient-

physicianrelationshipthatpromotesthepatient’scompliancetotreatmentandqualityoflife.

AverystrikingfindingwasthefactthatmostcountrieshadnoofficialregistriesofIBDpatientsand

couldthusprovidemereestimatesonthenumberorprevalenceofIBDpatients.Francecouldgivea

number provided by an IBD observatory, Sweden provided by a national registry, and Finland

provided by the Social Insurance Institute. Other participating national associations reported that

there are no national registries or if there are, these are incomplete or not up-to-date; these

associations were only able to give an approximation. Furthermore, very few of the participating

associationshadanydataonhowmanypatientsarereceivingeachmedication.

The European Medicines Agency defines patient registries as “organized systems that use

observational methods to collect uniform data on a population defined by a particular disease,

condition,orexposure,andthatisfollowedovertime.Patientregistriescanplayanimportantrolein

25

monitoringthesafetyofmedicines”(EuropeanMedicinesAgency,2017).AccordingtotheRegistryof

PatientRegistriesworkingundertheU.S.DepartmentofHealth&HumanServices,atitsmostbasic

level a patient registry is a database containing information about patients’ medical condition or

treatments (RegistryofPatientRegistries).Patient registriescan,however,beused foravarietyof

purposes,suchasscientificandclinicalresearch,collectingpost-marketingdataofadrugordevice,

studying the natural history of disease, and health policy purposes (European Medicines Agency,

2017; Registry or Patient Registries, 2018). Registries could be coordinated andmaintainedby, for

example,patients’orphysicians’associations,academic institutionsornationalagencies (European

MedicinesAgency,2017).Unfortunately,developingsuchregistriesseemstonothavebeenapriority

withintheIBDcommunity.ThismaybeimprovingasinDenmark,forexample,anationalregistryof

IBDpatientsreceivingbiologictherapywasinitiatedin2016(Larsenetal,.2016).

Theremayhavebeenambiguityinthequestionsandthegivenreplyalternativesthatcausedsomeof

theparticipantstohavetochooseareply fromseveral fittingoptions.Thismayhavecausedsome

discrepancies in theoutcomes.Due to thecross-sectionalnatureof this survey, itmaybe that the

situationinmanyofthecountrieshaschangedsincethetimeofthesurvey.Still,theresultsdogive

anoutlookintothedifferencesintheEFCCAmembercountriesatonepointoftime,anditisunlikely

thatalldifferenceswouldhaveclearedsincethen.Afollow-upsurvey,however,shoulddefinitelybe

carriedoutafterafewyearstoseehowthesituationhasdeveloped.Furthermore,EFCCAislooking

intothepossibilityofcreatinganonlineobservatorywheremembercountriescouldupdatechanges

intheircountryandseechangeshappeninginothercountriesinrealtime.

5.EFCCA’SRECOMMENDATIONS

Basedontheoutcomesofthissurvey,EFCCAhascreatedasetofrecommendationsthatcanbe

developedfurtherandrealizedinfutureprojectswithinEFCCAorincooperationwithother

stakeholders:

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• Accesstonewinnovativetherapiesneedstobeaccelerated;nationalassociationsare

encouragedtotakeadvantageoftheoutcomesofthisprojectintheirwork.

• Inruralareasandregionswheredistancesarelong,thepossibilitiesoftelemedicineandonline

servicesshouldbedeveloped.

• DevelopingIBDpatientregistriesonnationalorEuropeanlevelneedstobeencouraged.

References

KerseN,BuetowS,MainousAG,YoungG,CosterG,ArrollB.(2004)Physician-PatientRelationship

andMedicationCompliance:APrimaryCareInvestigation.AnnFamMed2:455-461.

LarsenL,JensenMD,LarsenMD,NielsenRG,ThorsgaardN,VindI,WildtS,KieldsenJ.(2016)The

DanishNationalRegistryforBiologicalTherapyinInflammatoryBowelDisease.ClinEpidemiol8:607-

612.

LönnforsS,VermeireS,GrecoM,HommesD,BellC,AvedanoL.(2014)IBDandhealth-relatedquality

oflife–discoveringthetrueimpact.JCrohnsColitis19:1281-6.

PatientRegistries.EuropeanMedicinesAgency.Availableat

http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000658.

jspAccessedonJanuary24,2018.

RegistryofPatientRegistries.U.S.DepartmentofHealthandHumanServices:AgencyforHealthcare

ResearchandQuality.Availableathttps://patientregistry.ahrq.gov/faq/AccessedonJanuary24,

2018.

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