owi white paper corporate citizenship, expanded access, and … · 2017-05-08 · corporate...

KeithWatts.April2016

WhitePaper

Corporate Citizenship, Expanded Access, and

Ethical Pre-License Drug Sales

CorporateCitizenship,ExpandedAccess,andEthicalPre-LicenseDrugSales 2

Abstract

Highpublicexpectationsforcorporatesocialresponsibility(CSR)andaccountabilityhavedirectedpharmaceuticalindustrypracticestoinfluencepositivechangelocallyandglobally.Drugcompaniesoftenrespondtoissuesaffectingpatientwell-beingfollowingcommercializationofnewproducts.Today,pharmaceuticalcompaniesareheldtohigherstandardsascorporatecitizensandmustnowconsiderhowtoimplementthisrolethroughoutaproduct’slife-cycle.Channelsthatallowaccesstomedicinestotreatserious,life-threateninganddebilitatingdisordersmustbebalancedwiththeinterestofallstakeholders.Today,thisbalancingacttakesplaceearlierintheproductlife-cycle,oftenbeforecommercialization,attheend,andeveninthoseinstanceswheremarketauthorizationisnotsought.Therearevariousethicalchannelsavailabletoprovideneededmedicationstopatientswithnoalternatives.Some

ofthesechannelsincludeEarlyandExpandedAccessPrograms(EAP),NamedPatientPrograms(NPP),CompassionateUse(CU),andPre-LicenseSales(PLS).Confusionexistsbecausethesetermscanhavevaryingmeaningsthroughouttheglobe.ItisimportantthatthesechannelsarefullyunderstoodbypharmaceuticalcompaniestoupholdCSR,lessenrisks,andpreservepatientsafety.Clearerdefinitionsofthesedifferentchannelsareneeded,asisunderstandingandexpertisetoethicallyandcompliantlyleveragethesepathwaystobenefitthosearoundtheworldwithrare,serious,andoftenlife-threateningdisorders.Whilethiswillsurelybeadifficulttask,clarifyingunderstandingofthesepathwayswilleventuallyharmonizemanufacturers,distributors,andpatients.

WhitePaper


CorporateCitizenship,ExpandedAccess,andEthicalPre-LicenseDrugSales

whichroughlyonly5%aretreatablewithmedicinesapprovedbytheUSFoodandDrugAdministration(FDA).Araredisease,asdefinedbytheOrphanDrugsActof1983,affectsnomorethan200,000patients.TheUSwasthefirstcountrytosetuplegislationthatprovidedformarketingexclusivityandpaymentincentivesfororphandrugs(Gammieetal.,2015).Inthe1990’sJapanandAustraliafollowedtheUSincraftingorphandruglegislationthatprovidedincentivestodevelopandmarketdrugsforrarediseases;theEuropeanUnion(EU)followedin2000withspecificorphandruglegislation(Gammieetal.,2015).Manycountrieshavesetuporphandruglegislationthatincludesvaryingrangesofreimbursement.Forexample,theNetherlandsorphandruglegislationallowsforpre-licenseaccesstoorphandrugsandreimburses100%ofthecostforapprovedorphandrugs(Gammieetal.,2015).Attheotherendofthecontinuum,India,acountrywithoverone-billionpeople,hasnoorphandruglegislationandpaymentoftencomesfromself-fundedfoundationsorfundedout-of-pocketbythepatient(Gammieetal.,2015).

Source:Phrmawebsite:NewReportHighlightsaDecadeofInnovationinRareDiseases.Accessed4/11/16.Availableat:http://catalyst.phrma.org/new-report-highlights-a-decade-of-innovation-in-rare-diseases

Countriesaroundtheworldhavesetupregulatorymechanismsforethicalaccesstoorphanandnon-orphandrugsatvaryingstagesoftheproductlife-cycle.Earlyandexpandedaccessareonlytwoofthemanynamesusedtodescribeaccesschannelsoutsidethemorefamiliartraditionalsupplychain.Providers,patients,andadvocacygroupsareleveragingearlyaccesschannelstoensurepotentialinterventionsforlife-threateningandlife-alteringdisordersreachthosepatientswithoutviabletreatmentalternatives.Legislationexistsforinnovationstotreatraredisordersthatprovideincentivesmakingitcommerciallyfeasibleformanufacturerstotaketheextraordinaryrisksassociatedwithdevelopingdrugsforsmallpatientpopulations.AdvocacygroupsandinternationalorganizationssuchastheNationalOrganizationforRareDisorders(NORD)intheUnitedStates(US)andtheEuropeanOrganisationforRareDiseases(EURODIS),areinfluentialdriversoforphandruglegislation(Gammie,Lu,&Babar,2015).Thereisanestimated350millionpeoplearoundtheworldwhosufferfromroughly7,000differentrarediseases("Rarediseases:Factsandstatistics,"n.d.),of


ThisarticlediscussesCSRasitconvergeswithchallengesalongvarioussupplychannelsthatexistatvaryingstagesofadrug’slife-cycle.

Consideringallrelevantstakeholdersisimportantwhendecidingwhetheraddressingglobaldemandforearlyaccessisconsistentwithacompany’sCSRinitiatives.Thedecisionismorecomplicatedandriskyduringearlystagesofaproduct’slife-cycle.Safety,efficacy,andamyriadofstrategicconsiderationsinthemidtolatedevelopmentandpost-researchphasesmustdirectresponsibledecisionmaking.Pre-approvalandpost-approvalpre-licensephasesincountrieswithestablishedethicalearlyaccesslegislation,ifcompetentlymanaged,mayprovidethegreatestsocietalandcorporatebenefit.

ThisarticlediscussesCSRasitconvergeswithchallengesalongvarioussupplychannelsthatexistatvaryingstagesofadrug’slife-cycle.ThesechannelshavemanylabelsincludingEAP,post-approvalPLS,NPP,amongothers.Thesechannelsrepresentethicalpathwayssetupbyhostcountriestoaddresstheissuesfacingthosepatientsawaitingadrug’scommercialapprovalormarketingauthorizationtotreatalife-threateningorlife-alteringdisorder.

CorporateSocialResponsibilityPharmaceuticalcompaniesarebecomingawarethatintentionalinitiativestosupportCSRcanimprovetheircompetitiveadvantage.CSRaffectsmanystakeholdersacrosssociocultural,political,economic,andregulatoryenvironments.Inthepast,amediumtolargepharmaceuticalcompanycoulddonatetovariouscharitiesandclaimtobeagoodcorporatecitizen(Matten,Crane,&Chapple,2003).Companiesarenowexpectedtoengageinprogramsthataffectthehealthandwell-beingofpatientsaswellasinfluencesustainablepositivechangeinlocalandglobalcommunities.Companyreputationandpublicopinionimproveswhendecisionsaredirectedbytransparentdeterminantsusedtoguideproductaccessthroughethicalpathwaysdesignedtomeetunmetmedicalneeds.SaltonandJones(2015)arguedthatpharmaceuticalcompaniesshouldbeheldtohigherstandardsofethical

socialbehavior.Pharmaceuticalandbiotechfirmssharethecommonpurposeofimprovingthehealthandqualityoflifeforpeoplearoundtheworld(Salton&Jones,2015).Itiscriticaltobalancethispurpose,andtheextraordinarypossibilitytopositivelyaffectlivesagainstthepossiblenegativeconsequencesflowingfrommisdirectedorpoorlyconsideredproductaccessimplementation.Ensuringthesecurityandintegrityofthesupplychainiscriticalwhenmakingtheseaccessdecisionsandtheprocessbeginswithchoosingtherightdistributionpartnerfortherightstageofaproduct’slife-cycle.

EAPexisttoprovideneededmedicinestopatientswithspecialcircumstancesincountrieswithestablishedregulatorymechanismsforaccessbeforemarketauthorization.Somecompaniessetupextensiveprogramstoprovidedrugsoutsideclinicaltrialsifconvincedtheremaybesomebenefittopatientsandcliniciansbeforeseekingmarketauthorization.Therearepharmaceuticalfirmsthathavesetuppoliciesdenyingorignoringtheneedforearlyaccesstomedicationsincountrieswheretheydonotintendtoseekmarketauthorization.Thereasonforrestrictivepoliciesincludelackofproductsupply,uncertaintyofthesupplychannelregulations,andotherreasonsthatcouldbeaddressedbycollaboratingwiththerightstrategicpartner.Thesepoliciesoftenexisteveninthepresenceofhostcountryregulatorymechanismsallowingaccessandreimbursementforneededdrugsapprovedinanothercountry.RisksofnegativepublicopinionandfailureofCSRarehighwhenacompanyrefusestoengagetheproperearlyaccesschanneltohelppatientsinneed.Therisksofnegativeperceptionishigherwhenthereisnofinancialorregulatoryrisktothecompany.Licensed,professional,anddedicateddistributioncompaniesexistwiththesolebusinessmissiontoethicallyaddressearlyaccessneedsofpatientsaroundtheworld,commerciallyorbyCU,whilesecuringthesupplychainandprotectingstakeholderinterests.Thesmallnumberofcompaniesdedicatedtothisbusinessmodel,suchasOneWorld,Inc.(OWI)canprofessionallyaddressthecommonissuesgivenforwithholdingproductfromsmallerlesscommerciallyattractiveregionsaroundtheworld.


“A small number of companies, such as One

World, Inc. (OWI), can address the common issues given for

withholding product from smaller, less commercially

attractive regions around the world.“

RisksofnegativepublicopinionandfailureofCSRarehighwhenacompanyrefusestoengagetheproperearlyaccesschanneltohelppatientsinneed.

Drugsupplyforecastsforapproveddrugseligibleforearlyaccessareusuallymorerobustandcanaccommodatechanneldemandmoreeasilythanresearchstagesupply.CompaniessuchasOWIcanhelpwithestimatesoftreatmentdemandinvariousregions,andbysecuringthesupplychainfromsecondarywholesalersandspecialtypharmacieslikelysellingintothoseregionswithneitheraccountabilityorthemanufacturer’sknowledge.Clinicalandmarketinsightsfromreal-lifeexperiencescommunicatedbyprovidersandkeynetworkpartners,whereallowedbyexistingregulatorymechanisms,canbeobtainedforstrategicusetohelpguidecommercialization.DelaysinmarketauthorizationalsocreatedisadvantagesforpatientsoutsidetheUSthatcanbegreatlyreducedbyauthorizedspecialtydistributorsknowledgeableinEAPregulations.Forexample,intheEU,onceadrugapplicationisfiled,ittakesanaverageof418daysforthedrugtoreceiveEMAapproval(Adesetal.,2014).PatientsintheUStypicallyhavemorerapidaccesstonewproducts,possiblybecauseofexpeditedreviewprocessesavailableinthestates(Adesetal.,2014).Patientswithlife-threateningandlife-alteringdisordersareplacedatextremedisadvantageandriskwhenmanufacturerswithholdavailableproductfromanapproved,licensedandcompliantspecialtydistributorthatcanethicallyaccessregulatedchannelswherethedrugisawaitingmarketauthorization,butisapprovedforearlyaccess.

Diversionanddrugcounterfeitingisanongoingconcernthatriskspatientsafetyandlives(Hintlian&Kelly,2014).Diversionappearstobemorecommonasamechanismtomeettheinternationaldemandforneededmedicationsininstanceswheremanufacturershavedecidedtowithholdproduct,pre-andpost-approval,fromterritorieswherenocommercializationisplanned.Thisisoftenthecase,evenwhenethicaldistributionpartnersspecializingincompliancedrivensupplyofunlicensedandpre-licensemedicationsusingestablishedregulatorymechanismsoffertoassumefinancialrisk.Drugdiversionbysecondarywholesalersalsoservingdomesticmarketsfurthercomplicatestheethicalaccesstoneededmedicinesandthatleadtohighercostsandriskpatientsafety(Hintlian&Kelly,2014;WorldHealthOrganization,2014).Unauthorizedcompaniesenteringtheinternationaldistributionchainwithpoorqualitymanagementalsocreaterisksfromdiversionandcounterfeitproduct(Kohler,Mitsakakis,Saadat,Byng,&Martinez,2015;Wilcock&Boys,2014).

Keith Watts


Unauthorizedcompaniesenteringtheinternationaldistributionchainwithpoorqualitymanagementalsocreaterisksfromdiversionandcounterfeitproduct.

Theserisksincludenegligentenvironmentalcontrolsandmonitoringcombinedwithpoorlyimplementedexport-importstrategiesthatruncountertoregulatoryrequirementsandriskpatientssafety(Hintlian&Kelly,2014).Thesesituationsexistwhenmanufacturers’policiesignorelegitimatepathwaysthatcanbelegitimatelyaccessedbyworkingwithcompaniessuchasOWItoreachpatientswherecommercializationisdelayedornotplanned.Providersandpatientsareforcedtosourceneededmedicationsoutsidelegitimatesupplychainchannelswhenmanufacturersrefuseforfailtouseethicalandregulatedchannelsbyworkingwithspecialtydistributorstosupplypre-licensedandunlicensedmedicines.Unauthorizeddistributioncreatesanenvironmentwherepatientseitherdonotreceivethedrug,ormustpayveryhighpricesforaccesstodrugswithnotraceability,educationalsupport,orrecallprocedureofferedbytheunauthorizedseller.Furtherriskstopatientsafetyoccurwhentherearenoassurancesthatenvironmentalrequirementshavebeenmet(Hintlian&Kelly,2014).

CSRisevaluatedtodaybasedontheGlobalReportingInitiative(GRI)thatisrobust,butdoesnottargetissuesspecificallyrelevanttothepharmaceuticalindustry.Offivemajorpharmaceuticalcompaniesevaluated,Johnson&JohnsonandRochereachedthehighestCSRscoresachieving65%and64%GRIguidelinecoverage,respectively(Salton&Jones,2015).Sanofi,GlaxoSmithKlineachieved58%and44%GRIcoverage,followedbyPfizerat28%(Salton&Jones,2015).Somecompaniesfocusmoreheavilyononeortwoofthe10GRImeasures,thereby,understatingtheircontributiontosocietyinthebroadermeasures,asreportedintheSaltonandJones(2015)study.DespitetheratingsfromGRI,Pfizerisconsideredagoodcorporatecitizenandhasdevotedsignificantresourcestosettingupregulatorycompliantearlyaccessforneededmedicinesbeforeregulatoryapproval.

SomecompanieswilldevotesignificantresourcestoengageinROWCSRinitiatives.MerckcreatedauniquesupplychainmodeltosupplycontraceptivesinSenegalthatisreplicableforotherproductsinterritoriesthatcouldproducecommercialvaluewhileaddressinghumanneed(Wright,2016).Merck’sinitiativeisagreatexampleofCSR.OtherlargepharmaceuticalcompaniesfocusondifferentCSRinitiativeswithnoattentiongiventoproductaccessincountrieswherenomarketauthorizationisplanned.Patientswithlife-threateningdiseaseslivinginregionswithnoexistingorexpectedcommercializationvaluefacesignificantdisadvantageunlessmanufacturerspartnerwithcompaniessuchasOWItoensureethicalaccesstoneededmedications.

OWIisamongafewcompaniesdedicatedtoensuringaccess,nomatterhowsmallordifficultthechallengesandcanhelpsupportCSRforcompaniesablebutunwillingtoservethesemarkets.Thereareclearlysupplychainissueskeepingneededdrugsfrompatientsinhardtoreachorcommerciallyunattractivemarkets(Wright,2016),butcompetentdistributionpartnersspecializinginEAPandPLScansupportmanagedaccessinitiativesaroundtheworld.TheEUcanbechallengingforthoseunfamiliarwithearlyaccesswherereimbursementpoliciesdifferandmarketauthorizationlagtimesvaryamongmembernations(Adesetal.,2014;Gammieetal.,2015;Hohmanetal.,2015).Companiesoftenignoretheseterritories,becausenomarketauthorizationissoughtorregulatoryexpertiseismissingtoensureproperdistribution.Merck,Pfizer,andothermanufacturerscanhelpexpandcompliantglobalaccesstoneededmedicationsthroughouttheproductlife-cycleinregionswithsmallpatientpopulationsanduniqueregulatorymechanismsbypartneringwithdedicateddistributorsspecializinginthesesupplychannels.


AproductcanreceiveregulatoryapprovalinonecountryandbeavailableforCUorPLSpriortomarketauthorizationinothercountries.

EthicalAccesstoNeededMedicinesDefiningethicalEAPandtryingtoapplythedefinitionuniformlyacrosscontinentscanbecomplicated.Itisoccasionallyunclearononecontinentacrossmultipleindustryparticipants.IntheUSthetermEAPandCUprogramscanbeusedinterchangeably,whileinEuropethetermsdonothavethesamemeaning(Iudicello,Alberghini,Benini,&Mosconi,2016).CUtypicallyreferstoaprocesswhereapharmaceuticalmanufacturertemporarilyallowsaccesstotreatmentforapatientwhohasrunoutofalternativetreatments(Hyry,Manuel,Cox,&Roos,2015).AproductcanreceiveregulatoryapprovalinonecountryandbeavailableforCUorPLSpriortomarketauthorizationinothercountries.Somecompaniesofferthedrugatnochargewhileothersseekoutpaymentprograms(Hyryetal.,2015).CUoftenrepresentsacommitmenttoCSRthatcanbuildearlymarketpresenceyieldingusefulrealworlddataassociatedwiththeexpandedaccessprogram(Hyryetal.,2015).ManufacturersworryaboutallowingCU,mainlybecausetheywanttoavoidcreatingexpectationsoffreeproduct.

TheUSandEMAdefinitionsforEAParesimilar;however,thedifferencesareenoughtorequireregulatoryspecializationwhencarryingoutEAPatvaryingstagesofdevelopmentandapproval.Open-labelextension,whichisclearlydelineatedfromEAPintheUSdefinition,butisnotsoclearwithintheEMA,cancauseconfusion(Iudicelloetal.,2016).ApharmaceuticalmanufacturermayuseCUtocarryoutspecificinternalprogramsdesignedtoprovidesomeformofearlyaccesstoaninvestigationaltreatment.CUmayalsobetiedtoapatientassistanceprogram,NPP,oranynumberofotherprogramsfittingoutsidethenormalsupplychain.Theseprogramsareoftenunfamiliartopharmaceuticalmanufacturersincertaindecisionmakingroles.FearoftheEAPandPLSchannelsoftenstemsfromdifferingregulatoryconstraintsandrequirementsforthesechannelscomparedtothoseofthecommercialsupply

chain.TheEMAallowscompaniesthatmakepromisingmedicinestorunCUstoallowearlyaccesstotheirmedicineandtoextendusetopatientswhocanbenefitfromit.TheEUregulatoryenvironmenthasmanynuancesthatvaryamongmembernations.Thesedifferencesmeritdiscussion,butarebeyondthisarticle’sscope.Futurearticleswilladdresssomeofthedifferencesbetweenopen-labelstudiesandEAPintheEU.

TheBrazilianUniversalHealthSystem(SUS),withguidancefromtheMinistryofHealth,hasauniquesystemwiththegoalofprovidingneededmedicinesfree-of-chargetocloseto75%ofitspopulation(Bertoldi,Helfer,Camargo,Tavares,&Kanavos,2012).Differentmeaningsfortermsthatdescribelegislatedaccesstounapprovedinvestigationaldrugs,post-researchpre-approvaldrugs,andunlicensedmedicinesawaitingauthorizationhaspersuadedresearchersoftheneedforclaritythroughcommondefinitionsforproperaccessmechanisms(Iudicelloetal.,2016).ClearerdefinitionsthatdistinguishEAPfromclinicaltrialsshouldreduceconfusionforpatients,providers,andcompaniesdealingwiththesecomplextopics.AglobaldefinitionisatallorderasthechallengeistocoordinatealltermsassociatedwithvariousEAPs.Claritywillhelpdirectsupportofaccesstoneededmedicationsatvaryingstagesoftheproductlife-cycle(Iudicelloetal.,2016).

Pre-LicenseSalesPre-licenseaccessallowsimportationoforphandrugsavailableinothercountriesbutcurrentlyunauthorizedinthehostcountry(Gammieetal.,2015).NPPisoftenusedtodescribepre-licensingaccessandisoftenthemostcommonmethodforpatientsaccessingorphandrugsinmanycountries(Gammieetal.,2015).NPPscanoccurthroughapprovedpre-licensedsale(PLS)ofaproductaccessgrantedtoanindividualoragroupofpatientswithaseriousorlife-threateningdisorderincaseswhereexistingtreatmentalternativeshavebeenexhausted.Pre-licenseregulatorymechanismsforproductaccesstypicallyrequireaphysician


CompanieswithdedicatedEAP/PLSmodelscansupportPLSandCUchannelsininstanceswhereapharmaceuticalmanufacturerisnotseekingmarketauthorizationinspecifiedcountries.

responsibleforthepatient’scaretoassesstheneedandopenarequestforadrug,onbehalfofthepatient(Gammieetal.,2015).Amanufacturer,orcooperationbetweenthemanufacturerandacompetentEAP/PLSdistributionpartner,willassesswhethertohonortherequestforaccess,basedonfixedguidelinesfromthemanufacturer.Aregulatorymechanismforpre-licenseaccessmaygrantethicalaccess,although,reimbursementbypublicpayersmaynotbetherule(Gammieetal.,2015).Turkeyisauniquecontraryexampleofacountrywithoutorphandruglegislationthathasanationalreimbursementprogramallowingimportationandaccesstoorphandrugsinapre-licensesettingwherethesedrugsareunavailableorunauthorized(Gammieetal.,2015).

CompanieswithdedicatedEAP/PLSmodels,suchasOWI,cansupportPLSandCUchannelsininstanceswhereapharmaceuticalmanufacturerisnotseekingmarketauthorizationinspecifiedcountries.Incountrieswheredemandforunavailablemedicinesexists,butmarketauthorizationstrategiesareunattractiveforpharmaceuticalcompanies;anexperienceddistributionpartnercansupportethicalaccesstotheseproducts.Supplychainqualitymanagementbythesespecialtydistributorsisimportanttoprotectagainstcounterfeiting,diversion,andunauthorizedproductsales(Kohleretal.,2015;Wilcock&Boys,2014).Specialtydistributorsmustreturnvaluetomanufacturersintheformofsupplychainsecurityandintegrity,RWD(inregulatorycompliance),marketinsightsandprovidereducation.Incertaincases,RWD,marketinsights,andpatientreportedoutcomes(PRO)cansupportlabelclaimsapprovedbyUSandEuropeanregulatoryagencies.TheEMAmorecommonlyallowsPRO-basedclaimsthantheUSFDA(Wilcock&Boys,2014).TheEMAgenerallywillgranthigherorderclaims,suchashealthrelatedqualityoflifeandphysicalfunctioning,fromPRO(DeMuroetal.,2013).TheFDAmainlyallowssymptomrelatedclaimsfromPRO(DeMuroetal.,2013).Thetwoagenciestendtoalignonsymptomswhere,forexample,patientreportedpainsymptomscansupportlabelclaims(DeMuroetal.,2013).Knowingwhattoexpectfromdifferentregulatorybodies

mayhelpidentifykeyRWDelementswhencollectionofsuchdataisallowed.

Manycompanieshaveastatedpolicyofwithholdingearlyaccessincountriesthatarenotconsideredformarketauthorizationorcommercialization.Thesecountriesarechallengingandsomecompanies,manufacturers,andEAPdistributors,areeithernotequippedorprefernottodevoteresourcestohandlerequestsfromthesedifficultregions.IntheUSafewspecializedcompaniesexistwhosesolebusinessasalicensedexportdistributorcentersonethicallyservingrest-of-world(ROW)pre-licenseproductneedsthroughexistingregulatorymechanisms.Specialiststendtobeexceedinglycapableandenthusiastictoservethosechallengingregions,whethertherearetwoor200patients.AnethicalpharmaceuticaldistributorwithastrongPLSmodel,havingadevelopednichepresenceandcompetenceinsecuringEAPsupplychainsinROWmarkets,canbeastrongallyofthepharmaceuticalmanufacturer.Itisimportantthedistributionpartnerthoroughlyunderstandsthehostcountryregulatoryandrelatedissuesfacingmanufacturersandpatients.TheneedsofpatientscanbeefficientlyservedbyanexperiencedspecialtyPLSdistributorthatsimultaneouslyprotectstherelatedinterestsofpharmaceuticalmanufacturers.

APLSbusinessmodeldoesnotexcludeservicingaCUorEAP,butitisbesttoseparateoneserviceproviderfromanothertoavoidconflictsofinterestandsupplychainconfusion.Interritorieswhereanavailablereimbursementorpaymentmechanismforunlicensedmedicineexists,andwheremarketauthorizationisnotpractical,acompetentspecialtydistributorcanhelpethicallysupplyneededproducts.Thesespecialistscannavigatethecomplexitiesofservingthischannelefficientlywithinavailableregulatorymechanisms,andwithoutrisktomanufacturers’globalplans.AcompanywithasoundandrobustPLSmodelcansupportsociallyresponsibledistributionofproductstoprovidersandpatientswithnoviabletreatmentalternatives.Thisisdonebyassumingfinancialandreimbursementrisksofproductacquisition,providercredentialing,shipmentsecurity,tracking,andconfirmation


withfollow-upreporting,whereindicatedandallowedbyahostcountry.Insomecountries,reimbursementoforphandrugsisbasedontherapeuticvalue,impactonclinicalpracticeandbudgetimpact(Gammieetal.,2015).

PLSchannelmanagementforethicalaccesstoneededmedicationsrequiresaspecializeddistributionpartnerwithstrongunderstandingofthischannel.Unauthorizedandunmonitoredsalesbysecondarywholesalersnotversedintheregulatoryenvironmentofmanydifferentcountriesrisksafetyandsecurityofpatients,whetheritbeoneormany(Mackey&Liang,2012;Wilcock&Boys,2014).Itisalsoanadvantagetoworkwithapartnerwhosemodelleveragesunderstandingoftheissuesfacingmanufacturers,providers,payers,andpatients.

ManufacturerCSRthatsupportsEAP,CU,andPLS,oftenreliesoninternaldepartmentsdevotedtoconnectingneededtreatmentstoprovidersofpatientswhohaveexhaustedexistingtherapeuticregimens.Settingupaninternalsystemrequiresresourcestodevelopnetworksandglobalregulatoryexpertise.Acost-effectivealternativetyingupinternalresourcesistoengageacompanylikeOWItobridgethatgapforpatientswhocannototherwiseaccessaproductbeforemarketauthorizationorwherecommercializationisnotplanned.AnethicalPLSprogramestablishedwithatrusteddistributionpartnerexperiencedinmulti-nationalregulatoryrequirementsofthischanneloffersanefficientalternativetoin-housemanagementofthisrole.CompaniesengagedinclinicaltrialsandresearchphaseEAPsshoulduseaseparateproviderforthissupplychanneltoclearlyseparatefromtheirCUandrelatedpre-approvalEAP.

PLSandCUareimportantaccesschannelsforneededproducts,butitisnotaseparatemarketingchannel.Thereisnomarketingallowedforaproductbeforemarket.Ethicalandexperienceddistributionpartnersunderstandthisandhavepoliciesinplacedefiningtheirroletoserveanexistingunmetmedicalneedandtoavoidmarketingaproductapprovedforpre-licensedsales.Onceaproductreceivesmarketauthorization,thePLSdistributionpartner’sjobistohelptransitiontoappointedaffiliatesofthemanufacturerforcommercialization.

“PLS channel management for ethical access to needed medications requires a specialized distribution

partner with strong understanding of this

channel.”


Paymentfororphanandnon-orphandrugsintheEUissubjecttoindividualcountrypolicies.

RegulatoryMechanismsItisimportantthatmanufacturersoforphandrugsandspecialtydistributorsunderstandtheregulatoryframeworkinplacetoaddressproductaccessandreimbursementaroundtheworld.TheregulatorymechanismsinplaceintheUSandEUdirectingorphandrugmarketingauthorizationandearlyaccessaresimilartonon-orphanregulatorymechanisms(Gammieetal.,2015).Asimilarregulatoryenvironmentexistsinnon-EUcountries(Gammieetal.,2015).TheCommitteeforOrphanMedicinalProducts(COMP)oftheEMAisresponsiblefordecisionstoawardanorphandruglabel,althoughmarketingauthorizationdecisionsaretheresponsibilityoftheCommitteeforMedicinalProductsforHumanUse(CHMP).

Paymentfororphanandnon-orphandrugsintheEUissubjecttoindividualcountrypolicies.IndividualEUcountriesareresponsibleforfixingearlyaccesspolicymechanismspre-andpost-marketauthorization.IndividualEUcountriesalsomustsetreimbursementmechanismsthatalignpayersandregulatorymechanismsforearlyaccesstodrugsforraredisordersthataresustainableovertime(Gutierrez,Patris,Hutchings,&Cowell,2015;Hyryetal.,

2015).Manycountriesrequirecosteffectivenessanalysistosupportreimbursementofhighcostorphandrugsandtheuseofmanagedentryagreementsarebecomingmorecommonforbothorphanandnon-orphandrugreimbursement(Gammieetal.,2015).Reimbursementpoliciesforneededmedicationsvarysignificantlyaroundtheworld.

InBrazil,25%ofthehouseholdspayforprivateinsurancewhile75%ofthepopulationrelyontheBrazilianUniversalHealthSystemeventhoughonlyroughly60%ofneededmedicinesareavailablewhenneeded(Bertoldietal.,2012).Brazilhasthreechannelsthatallowaccesstounapprovedorpre-licensepharmaceuticalandbiotechproducts,twoofwhicharesubjecttoapprovalbytheNationalHealthSurveillanceAgency(ANVISA).Humanitarianuse,whichisCUforimportingfreeproductaftermeetingseveralconditions,isacommonchannelforaccesstounapprovedmedicines.TheEAPinBrazilallowsacohortofpatientstoreceiveproductwithoutcharge,subjecttospecificconditionsandrequiressubmissionofasummaryreportonalltreatedpatients.Finally,NPPsinBrazilallowforpersonaluseofanunapprovedproductbasedoffaprescriptionfromthetreatingdoctor-noANVISAapprovalisrequired.


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Appendix:TableofAbbreviationsANVISA AgênciaNacionaldeVigilânciaSanitária

COMP CommitteeforOrphanMedicinalProducts

CSR Corporatesocialresponsibility

CU CompassionateUse

EAP Earlyandexpandedaccessprogram

EMA EuropeanMedicinesAgency

EU EuropeanUnion

EURODIS EuropeanOrganisationforRareDiseases

FDA FoodandDrugAdministration

GRI GlobalReportingInitiative

NORD NationalOrganizationforRareDisorders

NPP Namedpatientprogram

OWI OneWorld,Inc.

PLS Pre-licensesales

PRO Patientreportedoutcomes

ROW Rest-of-world

SUS BrazilianUniversalHealthSystem

US UnitedStates


OneWorld,Inc.Tucker,GA30084

Author:KeithWattsPresident/CEOKGPharmaceuticals.BoardMember,OneWorld,Inc.AdjunctFaculty,OhioChristianUniversityHealthCareAdministrationHealthcareEconomicsandBusinessStrategyLecturerCorrespondingauthorat:OneWorld,Inc.,4920NorthRoyalAtlantaDrive,Tucker,GA30084.Tel.:404-371-1745Ext312;fax:404-371-1774;email:[email protected]

owi white paper corporate citizenship, expanded access, and … · 2017-05-08 · corporate...

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