final report mapping corrected march 2018 - efcca report mapping 2018.pdf · final report january...
TRANSCRIPT
1
MappingofInnovativeTreatmentsandDevices
inEFCCAMemberCountries
FINALREPORT
January2018
2
Compiledby
ProjectCoordinator:
SannaLönnfors
EFCCAWorkingGroup:
MarcDerieppe
AlejandroKhalilSamhanArias
MarkoPerovic
Thisprojectreceivedanunrestrictedmulti-stakeholdergrantbyCelltrion,Pfizer,TakedaandOtsuka.
3
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
4
3.5.1.OtsukaAdacolumn(apheresis)...........................................................................................23
4.DISCUSSION.......................................................................................................................................23
5.EFCCA’SRECOMMENDATIONS..........................................................................................................25
References............................................................................................................................................26
5
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
6
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
EFCCAworkinggroup �
March2017
Launchingthesecondphasewithmorecountries
EFCCAworkinggroupEFCCAmembercountries
�
Summer2017
Closingthesecondphase EFCCAworkinggroupEFCCAmembercountries
�
Late2017 Analyzingtheoutcomes,generatingthefinalproduct
EFCCAworkinggroup �
Early2018 Finalreportanddevelopmentofrecommendations
EFCCAworkinggroup �
2018 EventatEuropeanParliament? EFCCA Figure1.Scheduleoftheproject
7
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
8
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
9
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.
10
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
11
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
12
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.
13
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,
14
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.
15
• 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.
0
1
2
3
4
5
6
7
8
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
16
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.
17
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
18
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
19
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.
Outofthe6associationsthatreportedthemedicineisapprovedintheircountry,3(50%)reported
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.
When European and non-European countries were separated, the medicine was reported as
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
20
intheircountry,2associations(9%)thatitispartiallycoveredand7associations(32%)thatcoverage
requiresanIBDdiagnosisintheircountry.
Outofthe22associationsthatreportedthemedicineisapprovedintheircountry,9(41%)reported
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.
When European and non-European countries were separated, the medicine was reported as
approved by 21 associations in Europe (72%). Out of those 21 associations, 13 associations (62%)
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)
21
Inflectra (infliximab) was reported as approved to IBD patients in their country by 27 of the
participatingassociations(84%).Outofthe27associationsthatreportedthemedicineisapproved,
17 (63%) reported that it is fully covered in their country, 3 associations (11%) that it is partially
coveredand7associations(26%)thatcoveragerequiresanIBDdiagnosisintheircountry.
Outofthe27associationsthatreportedthemedicineisapprovedintheircountry,12(44%)reported
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.
When European and non-European countries were separated, the medicine was reported as
approved by 27 associations in Europe (93%). Out of those 27 associations, 17 associations (63%)
reported the medicine as fully covered and three associations (11%) as partially covered. Seven
22
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
participatingassociations(78%).Outofthe25associationsthatreportedthemedicineisapproved,
12 (48%) reported that it is fully covered in their country, 4 associations (16%) that it is partially
coveredand8associations(32%)thatcoveragerequiresanIBDdiagnosisintheircountry.
Outofthe25associationsthatreportedthemedicineisapprovedintheircountry,11(44%)reported
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
When European and non-European countries were separated, the medicine was reported as
approved by 24 associations in Europe (83%). Out of those 24 associations, 12 associations (50%)
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
24
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:
26
• 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.