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A Rare International Dialogue – AGENDA May 10 – 12, 2019

Sheraton Centre Toronto Hotel 123 Queen Street West | Toronto, ON

1st Time in North America! Three days of dialogue and deliberation on all things rare: from research and policy to action and better outcomes

The largest and most significant gathering of rare disease advocates from patients to policy makers, from gene manipulators to AI gurus, from local support groups to international research networks coming together to Create a Vision for Rare Disease in the 21st

Century and Develop Action Plans from Local to Global

Hosted by Rare Disease International & Canadian Organization for Rare Disorders (CORD)

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Friday,May10,2019:VisionforRareDiseasein21stCentury

8:00a.m.–8:30a.m.

RegistrationandBreakfast:GrandEastBallroom

8:30a.m.–10:15a.m.

WelcometoRareDiseaseinthe21stCenturyWelcome:WhyRareDiseaseMatters

• DurhaneWong-Rieger,Chair,RareDiseaseInternational• MaureenSmith,BoardMember,CanadianOrganizationforRareDisorders• SenatorMaryHarney,FormerMinisterofHealth(Ireland)• PeterGoodhand,CEO,OntarioInstituteforCancerResearch,BoardMember,GlobalAllianceforGenomics&Health• ChrisMcMaster,ScientificDirector,CIHRInstituteofGenetics• SébastienMartel,GlobalHeadofRareDiseases,Sanofi

AdvancesinRareDiseaseFacilitator:BillDempster,3SixtyPublicAffairs

• DavidMalkin,TheHospitalforSickChildren• GarethBaynam,WesternAustraliaHealthDepartment(Australia)• EileenTreacy,NationalRareDiseases(Ireland)• HartmannWellhoefer,TakedaPharmaceuticals

10:15a.m.–10:45a.m.

Plenary:HowAdvocatesTransformedtheRareDiseaseLandscapeIntroduction:TransformativeRoleofAdvocacyinAdvancingRareDisease-BertBruce,Pfizer1. CelebrationofRareDiseaseHeroes(andtheirimpact)• YannLeCam,Europe• MarleneHaffner,USA• Min-ChiehTseng,Taiwan• AndersOlauson,Sweden

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10:45a.m.–11:15a.m.

NetworkingBreak

11:15a.m.–12:30p.m.

2. HowRareDiseaseHelpedTransformtheHealthcareLandscapeFacilitator:CateMcCready,BIOTECanada• DiagnosticBreakthroughs:FromNewbornScreeningtoNextGenSequencing-KymBoycott,CHEO• TransformingRareDiseaseResearchthroughTransnationalFunding-DariaJulkowska,E-RareNet• UsingGenomicstoMatchRareDiseasePatientstoTherapies-JanFriedman,UniversityofBritishColumbia• ApplyingDigitalTechnologiestoRareDiseaseDiagnosis,Care,Treatment,andCure-MichaelLiebman,IPQAnalytics,LLC• PatientCommunities:HowAIisTransformingthePatientExperience-LyndaBrown-Ganzert,Curatio

12:30p.m.–1:30p.m.

Lunch

1:30p.m.–2:00p.m.

VoiceofthePatientsIntroduction:GarethBaynam,UniversityofWesternAustralia

• ToriLacey,Canada• SherryCaldwell,Canada• EdaSelebatso,Botswana• MigdaliaDenis,LatinAmerica

2:00p.m.–3:30p.m.

Patient-FocusedDrugDevelopment-JointafternoonsessionwithRE(ACT)Introduction:OlivierMenzel,BlackswanFoundationResearchersandRegulators

• MarleneHaffner,USA• NickSireau,UK• LarissaLapteva,USA

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Pleasenote:Eventsbelowareseparateticketedevents,whichareNOTincludedwiththeconferenceregistrationfee.

6:00p.m.

7:00p.m.

WineReception(outsideGrandEastBallroom)

RareDiseaseAwardsGalaDinnerCelebration:GrandWestBallroom

• CalvinHo,USA• Soo-KyungLee,USA

3:30p.m.–4:30p.m.

Patients,Parents,andPatientGroupsFacilitator:PamelaGraves-Moore,SanofiGenzyme

• TracyKirby,BattenDiseaseSupportandResearchFoundation• John-PeterBradford,Life-SavingsTherapiesNetwork• TanyaCollin-Histed,InternationalGaucherAlliance• ChristinaMutene,RareDiseaseKenya• TatianaKulesha,UkrainianAssociationofPulmonaryHypertension

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Saturday,May11,2019:EducationalForum

8:00a.m.–8:30a.m.

ContinentalBreakfast:GrandEastBallroom

8:30a.m.–10:15a.m.

Welcome:WayneCritchley,CORDOpeningPlenary:EmbracingNewOpportunitiesandChallengesforRareDiseasein21stCenturySuccessStories:Patients,Treaters,Researchers,PolicyMakers

1. Patients:DoricaDan,Romania,K.P.Tsang,HongKong2. Alliances:GailOuellette,RegroupementQuébécoisdesMaladiesOrphelines3. Industry:DonatelloCrocetta,Takeda

PanelDiscussion:BuildingonSuccessandTendingtoUnintendedConsequencesFacilitator:JaneFarhnam,CORD

1. TrendsinRareDiseaseTreatment-MurrayAitken,IQVIA2. RareDiseasebytheNumbers-DebbyLambert,Orphanet3. Researcher/Developer-MichaelHayden,BCChildren’sHospital4. RareDiseaseTreatmentAccess-JudithGlennie,PRISM

10:15a.m.–10:30a.m.

RefreshmentBreak(participantsmoveintodialoguestreams)

ThreeParallelDialogueStreams(Overview)STREAMONE(GrandEastBallroom)TranslatingResearchintoCareandTreatment

• EndingtheDiagnosticOdyssey:Genomics,AI,Databases• ToolsandSupportforPatientandParentEngagement• EmpoweringtheWholePerson

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STREAMTWO(GrandCentreBallroom)InnovationsinTherapy:TreatingMoreandCuringSome

• CurativeTherapies:NextStepforRareDiseases?• StrategiesforAccesstoInnovativeTherapies• DriversofDrugDevelopment

STREAMTHREE(GrandWestBallroom)AddressingIssuesofDiversityandEquityinRareDisease

• StrategiesforAddressingRareDiseaseinEmergingHealthcareSystems• GrassrootsStrategiesAcrosstheGlobe• ChallengesofRareDiseaseSpecifictoUnderservedPopulations

10:30a.m.–12:00p.m.

STREAMONE:ROOM:GrandEastBallroomTranslatingResearchintoCareandTreatmentWinningtheRacetoDiagnosisFacilitator:AlexMacKenzie,CHEO

1. ReducingtheDiagnosticOdysseyforChildren:HowAGlobalCommissionisHelpingShapethePathForward-PeterJones,MicrosoftHealth

2. Howstructureddataisstreamlininggenomicdiagnosisaspartofthefirstpan-Canadianrarediseasedatarepository,Genomics4RD-OrionBuske,PhenoTips

3. GenCOUNSEL:OptimizingGeneticCounsellingwiththeClinicalImplementationofGenome-Wide

STREAMTWO:ROOM:GrandCentreBallroomInnovationsinTherapy:TreatingMoreandCuringSomeCurativeTherapies:NextStepforRareDiseases?Facilitator:IanStedman,YorkUniversity

1. UsingCRISPRtoUnravelGeneNetworksinRareDisorders-KristinKantautusandAshwinSeetharaman,UniversityofToronto

2. GeneTherapyforSingleGeneDefects:ExampleofSMA-SusanManganaro,AveXis

3. HowCAR-TCellsareKillingTumoursandSavingLives-RonanFoley,JuravinskiHospital,McMasterUniversity

STREAMTHREE:ROOM:GrandWestBallroomAddressingIssuesofDiversityandEquityinRareDisease

RareAdvancesatNational,Regional,andGlobalLevelsFacilitator:LisaSafarty,NORD

1. China-ShuyangZhang,PekingUnionMedicalHospital

2. Colombia-GérmanEscobar,DirectorofHealth,ProPacífico

3. EUCountries-DoricaDan,RomanianNationalAllianceforRareDiseases

4. HongKong-K.P.Tsang-HKAllianceforRareDiseases

5. WHOUniversalHealthCoveragePoliciesandPracticestoInsure“nopatientisleftbehind”regardlessofsocial,economic,

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Sequencing-AlisonElliott,UniversityofBritishColumbia

4. CHEOmicsClinic-AlisonEaton-CHEO

5. ReflectionsfromParentWhoAlsoHappenstobeaGeneticsProfessional-MazdakBagherie,UniversityHealthNetwork

Discussion

4. AdvancingCellandGeneTherapiesforHemoglobinopathies-KevinKuo,UniversityHealthNetwork

Discussion

andpoliticalcircumstances:InclusionofRareDiseases-YannLeCam

Discussion

12:00p.m.–1:00p.m.

Lunch

1:00p.m.–2:30p.m.

STREAMONE:ROOM:GrandEastBallroom

DesigningPathwaystoPatient-CenteredCareFacilitator:SandraAnderson,InnomarStrategies

1. Diseasemodeling:RealworldpatientsandReal-worldpracticeofmedicine-MichaelLiebman,IPQAnalytics

2. EuropeanReferenceNetworks:Crosscountrycollaborationandbestpractices(Galactosaemia)-EileenTreacy,NationalRareDiseasesIreland

3. BestPracticeCase:Hemophilia-DavidPage,CanadianHemophiliaSociety

4. BestPracticeCase:CAPS-RonLaxer,TheHospitalforSickChildren

STREAMTWO:ROOM:GrandCentreBallroom

ProgramsandStrategiesforAccesstoInnovativeTherapiesFacilitator:DevidasMenon,UniversityofAlberta

1. TaniaStafinskiUniversityofAlberta

2. SuzanneMcGurn,OntarioPublicDrugPrograms

3. EdmundJessop,formeradviserHSTP,NICE

STREAMTHREE:ROOM:GrandWestBallroom

GrassrootsStrategiesAcrosstheGlobeFacilitator:FergMills,InnomarStrategies

1. TaiwanFoundationforRareDisorders-Min-ChiehTseng

2. Agrenska:ARareServiceModel-AndersOlauson

3. Community-basedRDNetworks-RachelYang,ChineseOrganizationforRareDisorders

4. RDDJapan10thAnniversary-YukikoNishimura

5. NewbornscreeningandComprehensiveCareProgramforSickleCellDiseasein

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5. BonemarrowasaVehicleforCorrectionofRareDisorders:DonnaWall,TheHospitalforSickChildren

Ghana-IsaacOdame,TheHospitalforSickChildren

2:30p.m.–2:45p.m.

RefreshmentBreak

2:45p.m.–4:15p.m.

STREAMONE:ROOM:GrandEastBallroomEmpoweringtheWholePersonFacilitator:MaureenSmith,CORD1. DesigningCareforandbyFamilies-

IsabelJordan,RareDiseaseFoundation

2. RareConnect:ConnectingandEngagingRareDiseasePatientsGlobally-MatthewOsmond,CHEO

3. Psychological,Behavioural,Social&EthicalAspectsofRareNeurologicalDisease:Ataxia-GeorgeChandu/RamaiahMuthyala,IndianOrganizationforRareDisorders

4. EngagingYoungPeoplewithRareDiseasesAroundtheGlobe-BrynneDalmao,HDYO

5. UseofClinicalPatientRegistriestoInformBestPracticesIncludingAccesstoTherapies-CraigCampbell,LondonHealthSciencesCentre

Discussion

STREAMTWO:ROOM:GrandCentreBallroom

DriversofDrugDevelopment–RegulatoryCollaborationFacilitator:OxanaIliach,IQVIA

1. Canada’sregulatoryapproachtodrugsforrarediseases-FionaFrappier,HealthCanada

2. Europeanregulatoryapproachestodrugsforrarediseases-DanielO’Connor,EuropeanMedicinesAgency/MedicinesandHealthcareproductsRegulatoryAgency,UK

3. MedicalProductDevelopmentforRareDiseases:FDAperspective-LucasKempf,USFoodandDrugAdministration,CDERLarissaLapteva,USFoodandDrugAdministration,CBER

STREAMTHREE:ROOM:GrandWestBallroom

EthnicandDiversityIssuesFacilitator:GenevièveDubois-Flynn,CIHR

1. InclusioninClinicalTrials-JeffKeefer,IQVIA

2. IndigenousPopulationsSilentGenomeProject-LauraArbour,UniversityofVictoria

3. AddressingRareacrossFrenchCanada-GailOuellette,RQMO

4. State-of-the-ArtAcrossKeyCountriesandPathwaystoProgress-EvaMariaRuizdeCastilla,EMOLUVAPartnersLLC

5. EngagingMedicalStudentsinRareDisease-JessieKulaga-YoskovitzandKristinHunt,McGillUniversity

Discussion

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Sunday,May12,2019:SkillsTrainingandCapacityBuilding

8:30a.m.–9:00a.m.

ContinentalBreakfast:SheratonE

9:00a.m.–9:30a.m.

OverviewofDay1&2DurhaneWong-Rieger,CORDPARTICIPANTSMOVEINTOWORKSHOPSESSIONS

9:30am–12:00pm

ThemeOne:DiagnosisandBeyondROOM:SheratonE-northsideWORKSHOPA:GeneticCounselling:MakingSenseofNewbornScreeningandGeneticDiagnoses-BreanneDale,AndreaDjolovic,CanadianAssociationofGeneticCounsellors,AlisonElliot,UniversityofBritishColumbia

ThemeThree:PlatformsforRareDiseaseCollaborationandCoordinationROOM:OsgoodeWestWORKSHOPC:APECRareDiseaseFramework:DesigningandDevelopingNationalPlans-CameronMilliner,SueFletcher,Co-ChairsAPECRareDiseaseNetwork

ThemeFour:AccesstoRareDiseaseMedicinesROOM:SheratonAWORKSHOPD:AlternativeAccessSchemes:ManagedAccess(Entry)ProgramswithRiskSharing-TaniaStafinski,UniversityofAlberta,JudithGlennie,PRISM

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ThemeFive:Patient-EngagementSkillsDevelopmentROOM:SheratonBWORKSHOPE:NCATSToolkitforPatientFocusedTherapyDevelopment-EricSid,NIH,NCATSOfficeofRareDiseasesResearch

ThemeSix:RareCancerNetworksandPersonalizedCareROOM:SheratonCFacilitator:EllenColeman,VOZAdvisorsWORKSHOPF:RareCancerNetworksBridgingCommonCancersandRareConditions-JohnHopper,Co-Chairman,NORDRareCancerCoalition,LisaMachado,CORD/CanadianCMLNetwork,LouiseBinder,SaveYourSkin

12:00p.m.–1:00p.m.

LUNCH

1:00p.m.–3:30p.m.

ThemeOne:DiagnosisandBeyondROOM:SheratonE-northsideFacilitators:SafiyyaGassman,Pfizer,DurhaneWong-Rieger,CORDWORKSHOPG:CellandGeneTherapyfromLaboratorytoMarket-MarkLundie,PfizerCanada,JerryTeitel,St.Michael’sHospital,JayKonduros,Advocate,DanielleRollmann,NextWaveConsulting,EdwardBenz,Dana-FarberCancerInstitute

ThemeTwo:EmpoweringthePatientCommunityROOM:SheratonE-southsideWORKSHOPH:EmpoweringandSupportingCaregivers

• SupportingthePatientEcosystem-CherylPetruk,CanadianMPNNetwork• MoveOn:DealingwithAngerandAnxiety-KarenTompkins,ParentAdvocate;JoubertSyndromeandRelatedDisorders

ThemeThree:PlatformsforRareDiseaseCollaborationandCoordinationROOM:OsgoodeWest

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WORKSHOPI:UnleashingthePowerofReal-WorldEvidence-EfficientlyDeliveringRegulatoryGradeDataatLast-FemidaGwadry-Sridhar,PulseInfoframe

ThemeFour:PatientRegistries,DataSharing,andTargetedTherapiesROOM:SheratonAWORKSHOPJ:FromGeneticResearchtoPrecisionTherapy

• CoRDSProgramandTargetedTherapyResearch-BenjaminForred,SanfordResearch• HowNORDFostersPatient-DrivenRegistries-PamGavin,NORD,JessicaBohonowych,FoundationforPrader-WilliResearch

ThemeFive:Patient-EngagementSkillsDevelopmentROOM:SheratonBWORKSHOPK:OrganizationalSkillsBuilding

• EssentialandBestPracticesforSmallOrganizations-DevonPfeil,GlobalGenes• SocialMedia1.0and2.0-RoseMaryMoegling,GlobalGenes

ThemeSix:OrphanDrugPricingforInnovationandAccessROOM:SheratonCWORKSHOPL:ProjectHercules:AUKDuchenneGlobalCollaboration-JosieGodfrey,JGZebraConsulting

3:30p.m. Conferenceconcludes

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TheCanadianOrganizationforRareDisordersandRareDiseasesInternationalacknowledgesthecontributionofallourCorporatePartnerstoimprovingthelivesofpatientsandfamilieswithraredisorders.

Weareespeciallygratefultothefollowingthathavesupportedthisconference.

WewouldalsoliketoextendanextraspecialthankyoutoInnomarStrategiesforprovidingeventprojectmanagementsupport.