Response to Regulations Amending the Patented Medicines … · 2020. 4. 2. · Canadian Organization for Rare Disorders 151 Bloor Street West, Suite 600, Toronto, Ontario M5S 1S4

ResponsetoRegulationsAmendingthePatentedMedicinesRegulations

Submittedby:CanadianOrganizationforRareDisorders(CORD)

February15,2018

CanadianOrganizationforRareDisorders151BloorStreetWest,Suite600,Toronto,OntarioM5S1S4

Tel:(416)969-7464Toll-free:1-877-302-7273www.raredisorders.ca

Preface:FirstThoughtsTheCanadianOrganizationforRareDisorders(CORD)welcomestheopportunitytoprovideinputintotheconsultationonproposedamendmentstothePatentedMedicinesRegulations(PMPRBAmendments)postedDecember2017.However,fortherecord,weremainextremelydisheartenedthatourcommentssubmittedfortheJune2017consultationontheproposedregulatorychangeswerenotrecognizableinthesummaryoffeedbackfrompatientorganizationsnorwereourconcernsandrecommendationsreflectedintheDecember2017proposedamendments.Thatbeingsaid,CORDagreeswholeheartedlythatthecurrentpatentedmedicinesreviewprocess,fromHealthCanadaregulatoryreviewtothePatentedMedicinesPricesReviewBoard(PMPRB)throughthepanCanadianPharmaceuticalAlliance(pCPA)andintothe(privateandpublic)drugplans,isinneedofreviewandreform.Fromthegovernment’sperspective,theproposedAmendmentsareconsiderednecessarytoredressthefailuresofthecurrentregulations,whichweredesignedtoallowthePMPRBmeetitsgoalsofassuring“reasonable”drugpricesand10%pharmaceuticalresearchinvestment.Fromourpatientandpublicperspective,reformsareurgentlyneededtoredressthefailureoftheCanadiandrugassessmentprocesstoassuringthatCanadianshavetimelyaccesstothebestmedicinesatthebestpricesfortheirindividualneedandsocietalbenefit.Thisdeficiencyintimely,appropriateandreasonablypricedprescriptiondrugaccessisaccentuatedwithrespecttoCanadianslivingwithrarediseases.Undercurrentregulatoryprocesses,especiallyintheabsenceofCanadianOrphanDrugRegulations,onlyabouthalfofallorphandrugsapprovedintheUnitedStatesorinEuropeareavailableinCanada1andoftennotlauncheduntilyearslater.Moreover,onlyaboutone-fourthoftheorphandrugsapprovedbyHealthCanadaarefundedthroughpublicdrugplanswithsignificantdiscrepanciesacrosstheprovincesandterritories.2Today,nearly35yearsafterthepassageoftheOrphanDrugActintheUnitedStatesand18yearsfollowingsimilarlegislationintheEuropeanUnion,Canadastandsaloneastheonly“highincome”countryandoneoftheveryfewcountriesintheOrganisationforEconomicCo-operationandDevelopment(OECDwithoutsomeformoforphandrugorrarediseaselegislation.3

Lacking:PatientCentredPrincipledApproachTheproposedamendmentstothePatentedMedicinesActarepresenteddevoidofthebroadercontextofaCanadianpharmaceuticalpolicyandindeedwithoutcommitmenttotheessentialroleofpharmaceuticalswithinthehealthcaresystem.Ithasbeenadecade(andmore)sincetwomajorreviewsofCanadianhealthcare,theCommissionontheFutureofHealthcareinCanada:TheRomanowCommission4andtheMichaelKirbySenatereport5,bothconcludedthatpharmaceuticals,specificallyprescriptiondrugs,areessentialhealthcareservicesandshouldbe



guaranteedintheCanadaHealthAct.Withoutthisexplicitcommitment,asingularlyfocusedagendatoreform(reduce)drugprices,liketheproposedPMPRBAmendments,willinevitablyresultinpoorerpatientaccesstomedicines,poorerpatientoutcomes,andpoorersocietalbenefits.Priortomakingregulatoryreformstoapricingmechanism,itisparamountthatCanadaengagesinacomprehensive,multi-stakeholderconsultationtoarticulateCanada’spharmaceuticalpolicy.CORDoffers,asstartingpointstothedialogue,thefollowingthreefundamentalpropositionsorprinciples.Principle1.MedicinesareessentialhealthcareandpatientshaverighttotimelyaccesstothemostappropriatesafeandqualitymedicinesfortheirindividualneedsPrinciple2.Pharmaceuticalpricesanddrugbudgetsexistinadynamicecosystem,wherebypricesarebasedon(predicted,demonstratedormostlikely)therapeuticoutcomes(relativetoothertreatmentoptions)butareadjustedinthefaceofnew(real-world)evidenceofperformanceandimpact;atthesametime,drugbudgetsmustbeforecasttosufficientlyaccommodateoldandemergingtherapieswithmechanismstosupportcost-effectiveuseandmeetsocietalvalues(forexample,equitableaccess,fairinnings,and/orruleofrescue).Principle3.Canadamustcommittoparticipatingasatop-tiercountryininnovativetherapeuticresearchanddevelopment,andtothatend,itshouldstriveforacombinationofvolumeandpricingforapharmaceuticalproductthatwillgeneratesufficient(financial)incentivestothecompanytoprovideon-goingsupportforappropriateclinicaluseandresearchforoutcomesmonitoringaswellasincentivesforfutureR&DinCanada.

Needed:PatientCentredPrinciplesforRareDiseaseDrugs

CORDadditionalproposesanarticulatedpolicyspecifictodrugsforrarediseases(DRDs)thatrecognizestheuniquenatureofrarediseasesandiscompatiblewithpoliciesandproceduresinother(OECD)countries.RareDiseasePrinciple1:Patientswithrarediseasesareentitledtoequityinaccesstohealthservices,whichmayentailinequityintheassessmentofvalueoftherapies.RareDiseasePrinciple2:Fairaccessshouldtakeintoconsiderationsocietalpreference(addedvalue)fortreatmentsforpatientswithsevereanddebilitatingconditionswheretherearenogoodalternatives.RareDiseasePrinciple3:Regulatoryapprovalofrarediseasedrugmusttakeintoconsiderationuniqueclinicaltrialsdesignedtoaccommodatepatientwithrarediseases.Forexample,CTsthataredefinedby:

• SmallpatentnumbersthatprecludestatisticalanalysesdesignedforlargeRCTswithuniquephase2andphase3patientparticipants;

• Variabilityofdiseaseandlackofnaturalhistory,whichchallengeswell-definedpatientoutcomemeasures;



• Severityofconditionsandlackofalternativetherapies,whichmayprecludecomparisonwithstandardofcareandalsoaccesstotreatmentonethicalgroundspriortoconclusionoftrial.

Essential:InvestmentinInnovationItisdistressingthatneithertheRegulatoryImpactAnalysisStatement(RIAS)northePMPRBGuidelinesScopingPaperaddressesthecausesforlackofinvestmentinresearchanddevelopment(R&D)inCanada.NotwithstandingthewidediscrepancybetweenthePMPRB’scalculatedindustryinvestmentofR&Dat4.45%(oftotalsalesrevenues)andEYanalysisof9.97%,6thereisanacknowledgedneedtocultivateaclimatethatismorecompetitiveandinducingtopharmaceuticalinvestmentinCanada.IndeedCORDisverymindfulofthestrongwarningissuedbyBIOTECanadaandco-signedbytheprovincialbiotechnologyorganizationsthattheproposedamendmentswill“negativelyimpactthewholeCanadianbiotechecosystem.”Withrespecttoinvestmentinrarediseasepharmaceuticalresearch,CORDknowsfirsthandandthroughfeedbackfromourmanypatientcommunitiesthatCanadaisnotperceivedasadesirableenvironmentforrarediseasedrugresearchanddevelopment.Comparedtoothercountries,Canadalacksincentives,infrastructure,andpoliciessupportiveofexploratoryacademic/clinicalinvestigation,earlystageclinicaltrials,andearlymarketlaunch.Comparedtothoseinothercountries,Canadianacademicresearchershaveaccesstolessstart-upfundingforrarediseases;similarlyCanadianswhodiscoverpromisingtherapiesforrarediseaseswillmovethemtotheUnitedStatesorEuropewheretheycangetorphandrugdesignationandaccesstocapitalinvestments.Canada’sreputationasa“goodplace”todoclinicaltrials7doesnotgenerallyextendtotrialsforrarediseasetherapieswhereinfrastructuresupportandpotentialmarketaccessarelimited.

RegulatoryGoal:EquippingPMPRBasEffective“PriceMonitor”GiventhePMPRB’sself-declaredfailuretomeetitsobjectivesofassuring“reasonabledrugprices”and“sufficientpharmaceuticalinvestment”,itistimelytoconsideramoresubstantiveoverhauloftheentitywithinthecontextoftheoverall(current)pharmaceuticalsystemincludingHealthCanada’srevisedregulatoryapproach(R2D2),revisedCADTHfunctionsandtimelines(earlysubmission,stream-linedreviews,newrecommendationoptions),pCPA’senhancedscopeofwork(butincreasingbacklog)andchangestopublicdrugplans.Asastartingpoint,weaskwhethertheproposedAmendmentsaddressthefundamentalreasonswhythePMPRBhasfailedtofulfillitsroleasa“pricesetting”and“pricemonitoring”agency.WillchangingthebasketofcountriesreallyreducetheoverallCanadianinvestmentinpharmaceuticals?



Moreimportantly,willthePMPRB’sfocusondriving“average”Canadianpricestoa12-referencecountrymediancreateunintendedharms,especiallytopatientaccessandresearchinvestment?Forexample,someanalystshavepointedoutthatCanada’s“higherthanOECDmedian”drugpricesarecorrelatedwith“earlierlaunches”ofnewdrugsinCanada.Fromapatientperspective,thatisaverydesirableoutcome.8,9Moreover,despitethecalculationsprovidedinthescopingdocument,wearenotconfidentthattheadditionalreportingandmonitoringrequirementswillreallybe(muchmore)effectivegiventhelackoftransparencyandpossiblevalidationofactualdomesticandinternationalprices.Wearenotawareofanyothercountrythatisadoptingthisstrategyanditcanonlyservetodisenfranchisemanufacturers.Asimportantly,CORDisconcernedthatthePMPRB,inredefiningitsprocessisalsoredefiningitsmandate,frommonitoringpotentially“excessive”pricestoaddressing“budgetaffordability”withoutclearlyspecifyingwhosebudgetitisdefending:publicdrugplans,privateinsurers,and/orindividualpayers.Clearly,excessivepricingandbudgetaffordabilityareverydifferentconcepts,wherebymonitoring“excessiveness”isclearlyalignedwithPMPRB’slegislatedmandatebutevaluating“affordability”,notreally.Moreover,wecontendthat“affordability”isnotsolelyaneconomicconceptbutapoliticalandsocialone,anchoredinspendingprioritiesandbudgetallocations,soaffordabilitycannotbemeasuredwitheconomicfactorsalone.Incontrast,determiningwhetherapriceis“excessive”requirescomparisontoabenchmarkwithdeterminantsthatarenotonlyobjective(cost,profit,return-on-investment)andbutalsosubjective(equal,equitable,outcomes-based,earlyadopter).Soeventhedeterminationofexcessivenessrequiresconsiderationoffactorsthatarebeyondtheeconomics.Overall,weareunsurewhetherPMPRB’srevisedprocedurescanimprovetheircapabilitiestoassure“non-excessive”prices;however,weareverysurethatPMPROBisnotappropriatelypositioned,structured,orresourcedtoaddress“affordability”ofnewtherapies.AnequallytroublingquestionthatpatientshaveregardingthePMPRB’srevisedprocessiswhetherthePMPRBcouldandshouldusepharmacoeconomics(PE)tosetalegallybindingceilingpricethatisbasedonastandard“costperQALY.”Indeednotalloftheothercountriesinthe12-countryreferencebasketrelyonatargeted“costperQALY”andnoneestablishaPE-basedceilingpricepriortomarketentry.WearenotawareoftheactualprocessthatPMPRBwillbeusingtoarriveatavalue-basedpriced,butwehavebeeninformedthattheeconomicevaluationwillbeconductedbyCADTH.UnderthecurrentCADTHprocess,company-submittedPEanalysesandCADTH-conductedPEanalysesoftenyieldwidelydiscrepantoutcomes,presentedasincrementalcost-effectivenessrations(ICER’s)orcostperquality-adjustedlifeyear($/QALY).Thesedifferencesareespeciallypronouncedforinnovativeandrarediseasedrugswherethereinputandoutputmeasuresaswellasotherfactorsmaybehighlyuncertain.Asaresult,CADTHoftenrecommendsapricereduction(neveranincrease)toimprovecost-effectiveness(sometimesaspecifiedamount).



WhatisdifferentfromtheproposedprocessisthattheCADTHrecommendationisusedasastartingpointfornegotiationanddoesnotconstituteaceiling(non-excessive)price.Therearemanyotherelementsthatcanbroughtintothenegotiationtoimprovecost-effectivenessand/orreducerisktothepayer,includingrisk-sharingagreements,managedaccessplans,patient-supportprograms,patientscreeningandregistries,andpost-marketmonitoring.TheCanadiannegotiationprocessparallelsthetypeofnegotiationsundertakenbetweenindustryandpayerinothercountries.UndertheproposedPMPRBamendments,companiescoulddecidenottobringaproductinCanadaortheywillwaittolaunchuntilpriceshavebeenestablishedelsewhere.Theseoutcomes,whicharehighlylikely,wouldbeextremelydetrimentaltoCanadianpatientsand,forsome,wouldmeanthedifferencebetweengettingtreatmentwhenitcouldmakeadifference,ornot.WerecommendthatthePMPRBtakeadvantageofthisproposedreviewofmandateandregulationstomorefullyexplore,withallstakeholders,alternativepathwaysthatwouldleadtothetriplegoalofmeetingpatientneeds,supportinginvestmentininnovation,andassuringnon-excessivepricing.Tothatend,CORDoffersthefollowingrecommendationstoguidethisprocess.

1. PMPRBshouldprovideincentivesoratveryleastnotputCanadianpatientsatdisadvantageforearlyaccess,includingclinicaltrialsandregulatoryfiling

2. PMPRBproceduresshouldbeasstreamlinedaspossible,thatis,minimizebureaucratichurdlesandredtape,toassurenounnecessarydelayandcostandpromotewillingnesstotrialandmarketdrugsinCanada

3. Mechanismsofsettingpricesandconditionsforaccessandmonitoringshouldbeastransparentaspossiblewithpublicaccountability

4. Pricingnegotiationsshouldallowforpatient-centredaccessapproachesthatwouldoptimizetheopportunityforallpatientswhomaypotentiallybenefitfromtherapytohaveaccess,forexample,throughrisk-sharing,managedaccess,earlyaccess,orcompassionatetrialprograms

KeyConcernswithProposedPMPRBProcess

SingularFocusonPricesMeansNegativeImpactonAccessandPricesItisunconscionablethattheRegulatoryImpactAnalysisStatementRIASdidnotexplicitlyincludeassessmentoftheimpactoftheproposedamendmentsonpatientaccesstomedicines.ItisnotasufficientdefensetoarguethataccessisnottheresponsibilityoftheregulatorortoproposethatthePMPRB’ssolemandateistopoliceexcessivepricing,nottoconsidertherapeuticvalue.Indeed,withoutafirmcommitmenttopatientaccess,whichistheonlyreasonforadrugpolicyandprogramme,thereisnocontextfordeterminingwhethertheamendmentstothePatentedMedicinesActarehelpfulorharmful.Thedrugaccessenvironment,likeathree-leggedstool,isonlyfunctionalandsteadyifallthreeobjectivesarebalanced.



Theseobjectivesare:timely(individual)patientaccesstomedicinesappropriatetopatientneeds,continuedinvestmentininnovationtodevelopbettertherapies,anddrugpricingappropriatetovalueofthemedicinesandfundingresources.

LackSearchforWin-Win-WinSolutions

Wemustconsiderhowandtowhatdegreetherevisedpoliciesandprocedures,whichputdownwardpressureoninitialdrugprices(andsubsequentdrugcosts),couldresultinpooreraccesstoneededmedicinesorlongerdelaystoclinicaltrialsandaccessandthealternativesolutionstomanageinevitablyrisingdrugbudgetswithoutblockingaccesstoimportantnewtherapiesforCanadianpatients.Wedonotbelieveweshouldsetupasystempremisedon“trade-off’s”betweenaccessandcostbutonethatseekstofindsolutionstomeetourcollectiveandfundamentalobligationtoassuringsustainableaccessto“firstinclass”medicinestotreatunmetneeds,fortherapies(includinggeneandcellular)thatwillpreventandcurechronicconditions,andfordrugsandmedicaldevicesthatwillreplaceriskierandmoreinvasiveinterventions.Weneedstrategiesthatareforwardthinkingandnota“doublingdown”onacknowledgedfailedprocessesbytighteningdown(external)reference-basedpricecontrolsandintroducing(internal)complexpharmacoeconomicevaluations(value-basedpricing)toestablishtheinitialceilingprice.

12-CountryReferenceIncludesThosew/SlowerandPoorerAccess

Wehavealotofquestionsabouthowthestepsoutlinedintheflowchartwillwork,whethersingularly,sequentially,orintandem.Intermsofthefirstpassagainstthereferencegroupof12countries,weareconcernedbecausemanynewdrugsarenotavailableinsomeofthesecountriesandinothercasessomearelaunchedconsiderablyaftertheirintroductioninCanada.Sonowwhat?Canadianpatientsobviouslyshouldnotbeobligedtowaituntilthereisa“median”benchmarkpricebeforeintroductiontoCanada.(Wewereassuredthatthiswouldnotbethecasebuthavenounderstandinghowthisrulewouldbeapplied.)

TraditionalPE:ChallengeofaSingle$/QALYThresholdUndertheproposedamendments,drugsthatmeetthefirsthurdleofreferencepricingwillundergoapharmacoeconomic(value-based)evaluationiftheyareidentifiedas“highpriority”basedonacombinationoffactors:firstinclass,havingfewornotherapeuticalternatives,providingsignificanttherapeuticimprovementoverexistingtreatmentoptions,[indicationforaconditionthathasahighprevalenceinCanada],highcostorclassifiedasahighpriority…becauseofunmetmedicalneed.Moreover,itisnotclearfromthescopingdocumentiforhowvaluesotherthaneconomiconesareintegratedintotheestablishmentoftheceilingprice.Perhapswearereadingtoomuchintoit,butCORDisverytroubledbytheuseofthemuch



olderterm“pharmacoeconomic”evaluation,whichfocusesonfactorsexpressedaseconomicvalues,ratherthenthemorecomprehensiveapproachdenotedby“healthtechnologyassessment”,which(theoretically)isamultidisciplinaryprocessthatsummarises“medical,social,economicandethicalissuesrelatedtotheuseofahealthtechnologyinasystematic,transparent,unbiased,robustmanner”10andmostimportantly,incorporatingpatientpreferences11npatients(forexample,asmeasuredbypatientreportedoutcomesandimpact.12In2013,theUKgovernmentmandatedNICEtodevelopaprocessfor“valuebasedpricing”;however,NICEsubsequentlyabandonedthetask,concludingthattheirproposed(quantitativemethodswouldnotadequatelyreflectsociety’sdifferentialvaluefornewtherapiesforconditionsofdifferentseverityorwithvaryingbenefitstowidersociety.13Indeed,evenCADTHhaswalkedbackfromthepositionthatasingle“evidence-justified”cost-effectiveness(cost-utility)thresholdacross(most)conditionsisfeasibleorevenethical.Certainly,mostothercountries,includingmostinthereference”basket”donotrelyonasingleCEthresholdorevenmultipleCEthresholds.

TraditionalPE:SoWrongforRareDiseaseDrugsWeareobviouslytroubledbytheproposedscreeningcriteria,whichwillguaranteeeverydrugforrarediseasesisincludedinthistier.Thereareseveralreasonswhy“value-based”pricing(pharmacoeconomicevaluation)atlaunchisproblematicbuttheapplicationisespeciallydetrimentalforrarediseasedrugs.First,ifitisdemonstrablydifficulttoestablishasingle“value-based”thresholdacrossallconditionsandtherapies,itisnighnearimpossibletojustifyaninitialCEthresholdforDRDs,evenifthereweredifferentiations(discountingfactors)thatwouldallowthethresholdtorise(based,forinstance,onseverityorlackofalternatives).Incontrasttoalmosteveryothercountry,highormiddleincome,Canadahasconsistentlybeenreluctanttorecognizethatdrugsforrarediseasesmustbetreateddifferentlyifpatientsaretobetreatedequitably.ThesedifferencescannotbesubsumedintoquantitativefactorstomodifyaCEthreshold.Notonlyarerarediseases,bydefinition,smallpatientpopulations,butmostarealsosevere,debilitatingorlifethreatening.Usuallyanewtherapynotonlyaddressesanunmetneed,butitisalsothefirsttherapyforthecondition,sotherearenoalternativetreatmentsforcomparisonorsubstitution.Finally,ararediseasemayalsobehighlyvariableintermsofcausation,symptomology,naturalprogression,andresponsetotherapy,allofwhichcomplicatethepredictionofdrugperformanceandlong-termoutcomes.Drugsforrarediseasesarenotthesameas“personalized”medicinesforsubgroupsofmorecommondiseases,whichmayhavewell-establisheddiseasetrajectories,severalalternativetreatments,well-documentedclinicaloutcomesorotherpredictorsoflong-termimpact.



ManyDRDsaretrulyinnovative,theresultofbreakthroughsinunderstandingofcausativepathways,innovationintechnologies,andcreativenewtrialdesigns,noneofwhichlendtoapplicationoftraditionalhealthtechnologyassessmentusingtraditionalpharmacoeconomicmodels.

AlternativestoPEandICERsforRareDiseaseDrugsItisimportanttonotethatsomeofthecountriesinreferencebasketdonotapplycost-effectivenesstoassessmentoforphandrugs.14CORDagreeswiththeopinionofmanyhealtheconomiststhattraditionalHTAmethodsappliedtoorphan(raredisease)drugs“capturethecomparativeclinicaleffectivenessornethealthbenefitof[these]newtreatments.”Withrespecttorarediseases,thepropositionthatubiquitousqualifiersof“prolonginglife”or“significantQALYgains”couldserveasamelioratingfactorsforadjustingthe$/QALYwouldbechallengingfordrugsforrarediseases.ManyDRDslackthenecessaryevidencethatwouldbederivedfromlong-termexperience,naturalhistoryorotherindicators.Asaresult,anyassessmentswouldbeshroudedbyhighuncertainty,basedonthesamereasons.Wenotethefollowingstatements,whichsummarizethechallengesforDRDs.“Orphandrugshavehighlyvariableanduniquecircumstancesspecifictoeachdiseaseandfacemethodologicaldataconstraints,includingvaryinglevelsofavailableevidence,smallstudypopulations,quantificationofqualityoflifebenefit,rarelymeasuredspillovereffectsinfamilies,variationincost-offsetsthatdeterminecost-effectiveness,highburdenofillness,lackofappropriatecomparatortreatments,etc.…heterogeneityintreatmentoptionsandcharacteristicsoforphandiseasepatientscannotbeaddressedbya‘one-size-fits-all’assessmentapproach,andattemptingtodosounderminesthefullvalueofthesetreatments.“15Manyothercountries,includingthoseinthereferencebasket,haveadoptedapproachesotherthantraditionalHTAtodemonstratethevalueofDRDs.Forexample,EnglandandScotlandinclude“diseaseandtreatmentexperiencesfromamulti-stakeholderstandpoint”,combinedwithothermeasurestodealwithuncertainty(e.g.managedentryagreements).Thismulti-stakeholderapproachisreflectedinpanEuropeaninitiatives,suchastheMechanismofCoordinatedAccesstoOrphanMedicinalProducts,16thatfostersmulti-stakeholderdialogueandconsensusaboutvaluedeterminantsthroughoutthelife-cycleof[aDRD].17SwedendoesnotusePE/CEforDRDsbutappliesthreeprinciplesforvaluedeterminations:ahumandignityprinciple(allcitizensshouldbetreatedequallydespitepersonalcharacteristicsorstandinginsociety);aneeds-solidarityprinciple(thehealthsystemshouldprovideequalaccesstocareforallandstriveforoptimizedclinicalbenefitbasedonpatientneed);andacost-effectivenessprinciple(thehealthsystemshouldstriveforbalancebetweencostsandeffect).



WhiletheNetherlandsdoesperformHTA,theyhavenofixedICERforDRDs.Interestingly,OntarioisoftennotedasajurisdictionwhereDRDsarenotsubjecttoHTAorICER;ratheraCEAisbasedonpatients’ratingsofqualityoflifemoderatedbyotherfactors,notablyseverityofdiseaseandlevelofdisability.Sadly,theOntarioDRDcommitteeandprocesshasbeeninactiveforseveralyears.Asimportantly,asapatentcommunity,wearefrightenedbytheapparentretreatundertheproposedamendmentsforanycommitmenttoinvestmentininnovation(R&D),premisedostensiblyontheinabilityofthePMPRBwithcurrentprocedurestoassureadesiredlevelofresearchanddevelopment.Withoutthecontinuedinvestmentofpharmaceuticalmanufacturersininnovativedrugdevelopment,includingclinicaltrials,Canadianpatientswillsufferfromthelackofaccesstoexperimentaltherapiesandtheinvestmentinclinicalsitesanddiseasemanagement.WearenotinapositiontosuggesttheappropriatepercentageofR&Dinvestmentbutwedonotknowthatcompaniesandinvestorsareattractedtoenvironmentsfavourabletobusinessinvestment.WesuggestthatCanadacouldgoalongwaytocreatingamorefavourableR&Denvironmentforinnovativepharmaceuticalresearch,suchastheOrphanDrugActthatwaspassedintheUSA,inEurope,inJapanandothercountries.

RecommendedStepsForwardWeareatcriticaljunctureforaction.TheproposedamendmentstothePatentedMedicinesActhavepropelledallstakeholderstoacknowledgethecurrentdrugaccesssystemisbrokenandthereisanurgentneedfortransformation.CORDbelieves,basedonthepre-submissiondialogueswehavehostedaswellasotherconversations,thatstakeholdersdonotfeeltherehasbeenadequateexplorationofalloptionsfordesigningasystemthatismeetsourmutualgoalsfortimelyappropriateaccess,investmentininnovation,andsustainablefinancing.Therefore,CORDoffersthefollowingnextsteps:

1. ImmediatelywithdrawtheproposedAmendmentstothePatentedMedicinesActandceaseallconsultationsonthem.

2. Conveneagatheringofleadersrepresentingallstakeholders(andsectors)todevelopafocused,goal-directed,andtime-limitedengagementprocessthatcouldincludeopenmeetings,requestsforwhitepapersandbriefs,expertmulti-stakeholderdialogues(includingpatientexpertsandinternationalexperts),andotherformsofdeliberationandcollaborationwiththeobjectivesofsurfacingprinciplesforpatient-centred,responsible,innovative,andsustainablepharmacare,internationalbestpractices,opinions,innovativeapproaches,collaborativewin-win-winoptions.

3. Proposeviablealternativesforfocuseddeliberationsleadingtoconsensusonaviablelifecycleapproachtoprovidingaccess,promotinginnovation,andassuringfinancialsustainableofmedicinesinCanada.



AbouttheCanadianOrganizationforRareDisorders(CORD)CORDistheCanadianOrganizationforRareDisorders,Canada’snationalnetworkfororganizationsrepresentingallthosewithraredisorders.CORDprovidesastrongcommonvoicetoadvocateforhealthpolicyandahealthcaresystemthatworksforthosewithraredisorders.CORDworkswithgovernments,researchers,cliniciansandindustrytopromoteresearch,diagnosis,treatmentandservicesforallraredisordersinCanada.1in12Canadianshasararedisorder.Manyothersareaffectedoratriskbutremainundiagnosedandunaware.CORDprovidesinformationtoindividualsandconnectionstootherraredisordersupportgroupsandorganizationsfromPrinceRupert,BritishColumbiatoSt.John’s,Newfoundland.CORDhasledthedevelopmentofCanada’sRareDiseaseStrategy,bringingtogetherexpertsfromeverysector.TheStrategydetailstheextraordinaryburdenfacedbyCanadianfamilieswithrareillnesses.TheStrategyproposesafive-pointactionplanthatwilladdressunnecessarydelaysintesting,wrongdiagnosesandmissedopportunitiestotreat.CORDisakeypartnerinthedesignandimplementationofOntario’sRareDiseaseStrategyandFramework.CORDhasbeenactiveinshaping’sCanadianOrphanDrugPolicy,includingdraftingofthefederalgovernment’sproposedCanada’sOrphanRegulatoryFramework,theProvincial/TerritorialplanforExpensiveDrugsforRareDisorders,andimplementationofCanada-widestandardsforNewbornScreening.CORDpartnerswiththeCanadianInstitutesforHealthResearchtosupportfundingforinnovativeresearchinrarediseasesandisworkingtoensureCanada’sClinicalTrialsRegistryworkseffectivelyforthosewithraredisorders.CORDispartneringtopromotetimelygeneticscreeninganddiagnosticstoreducetimetoaccuratediagnosisandsupportsdevelopmentofCentresofExpertiseforRareDiseaseslinkedtoregionalandlocalservicestofacilitatebest-practicetreatment,careandsupport.CORDlinkspatientswithoneanotherandwithsupportgroups.CORDlinkspatientgroupswithresearchers,companiesengagedindrugdevelopmentandclinicaltrials,agenciesreviewingdrugsandothertherapeuticinterventions,payersforhealthservicesincludingdrugs,andpolicymakerstoensurepatient-centredhealthservicesandallocationofresources.



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