a rare international dialogue – agenda€¦ · a rare international dialogue – agenda may 10...
TRANSCRIPT
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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.