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Innovative Medicines Initiative
www.efpia.eu
EMA-EBERegulatoryConferenceonATMPs Salah-Dine Chibout, Novartis
Global Head Discovery & Investigative Safety/ Global Head Preclinical Safety Therapeutic Areas
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2www.efpia.eu
Mul-plecompaniesjoinforcewheretheywouldfailalone:Iden&fymissingorweaklinksinmedicinespathwaysthatholdprogress
Combine(o:en)proprietaryknowledge,dataandassetsOpenthemupforchallengebyandcollabora-onwithpublicpartnersValidateproposedsolu&onsduringprojectlife&meinR&Dprac&ce
Solu-onsfordiseaseswithhighburdenandcostforpa-entsandsociety
Solu-onsthatchallengecurrentbusinessmodelsandfocusonvalueforpa-entsandsustainablehealthcare
Trackingandaddressingsciencegapsandinefficienciesfromdiscoverytodiseasemanagement
IMIacceleratesinnova-on
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3www.efpia.eu
Publicprivatepartnership Companiesandpublicpartnersworktogether Industrycostisnotreimbursed:itisourinkindcontribu&on Publicpartners(includingcompaniesupto€500mioturnover)
costisreimbursedbyEU:grantsforcollabora&ngwithindustry
Industrydefinestheresearchagendaandprojects(butdoesnotchosewithwhomtowork) Beyondthepublica&on:impactonresearch,regulatoryand
medicalprac&ce
Managedbyaneutralbrokerthatallowspar&cipa&onofauthori&esandpa&ents
Essen-alfeaturesforresearchandpolicyagendas
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4www.efpia.eu
TheInnova-veMedicinesIni-a-ve:thelargestpublic-privatepartnershipfor
healthresearchworldwide€5,276billion
IMI1€2billionfrom2008–2014IMI2€3,276billionfrom2014-2024
PartoftheEUFP7andHorizon2020R&Dfunding
Source:hUp://www.imi.europa.eu/content/mission
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5 www.efpia.eu
EFPIA direct and indirect members
3.
1. 2.
What are the results ?
= in IMI2 consortia
1,638
3,276
From H2020
2014 - 2024
1,638 Billion € 1,638 Billion €
3,276 Billion €
+
1,425
Private Partners
Public Partners
CASH for grants for PUBLIC PARTNERS
Other sectors213
People
Consumables
Cash
Laboratories
Cash
Improve R&D
Improve outcomes & safety
Speed up patient access
The publiccontribution
The privateIndustry in-kind contribution
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Distribution of IMI funding per area
Infectious diseases
Drug discovery
Brain disorders
Metabolic disorders Drug safety
Stem cells
Cancer
Data management
Inflamatory disorders
Biologicals
Geriatrics
Lung diseases
Education & training
Sustainable chemistry
Drug delivery
Drug kinetics Relative effectiveness
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7www.efpia.eu
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8www.efpia.eu
End-to-End:Alzheimer’sExample
Ae-onomyInterven&ons
andModelling
EMIF-ADCharacterisa&
onandModelling
Pharma-CogBiomarkers
EPADTrialandModelling
ROADMAPOutcomesdefini&on
andtracking
LegalandEthicalframework,Parent
cohorts,fingerprin&ng,databrowser
Trial,Hypothesisvalida&on
Knowlegebase,novelhypothesesandbiomarkersPa&entaccess,
healthcaresystems
Combina&onoftoolstoaccuratelymeasuredisease
progression
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9www.efpia.eu
End-to-End:R&Daccelerator
Targetvalida&on
ELF
HittoLead
Enable
Biomarkeraccelera&on
SAFE-T
BigData
EMIFEHR4CR
ClinicaltrialsNetworks
EU-AIMSCOMBACTE
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10www.efpia.eu
EuropeanLeadFactory
Ra-onaleNeed
1. Accesstohigh-qualitychemicallibraryforacademics/SMEstotranslateacademicbiologicaldiscoveries/targetsintosuitablechemicalmaUer
2. Accesstootherwiseuna`ainablechemicalspaceforpharmapartnersthroughcompoundsharingandsynthesisofnovelchemicallibraries
3. Accessnewbiologyfromacademia/biotech
Aim
• Providestar-ngpointsforleaddiscoveryorhigh-qualitypharmacologicaltoolsbothforacademics/SMEsproposingtargetsandEFPIAcompanies.
• Createpartneringopportuni-esforpublicpartnersandEFPIAcompaniestoprogresshitsalongthepharmavaluechain
IMI funding: € 80 Mio Academia / Biotech cont. € 25 Mio Pharma resources : € 91 Mio TOTAL PROJECT COST: € 196 Mio Duration: 01.01.2013 – 31.12.2017
Funds
PartnershipProjectProfileShareBurdenand
Risk
Standardiza&onandRegulatoryBodyInterac&on
Collabora&onIndispensable
DirectPipelineImpact
MethodsandTools
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11www.efpia.eu
Boos-ngDrugDiscoveryEuropeanLeadFactory
SevenPharmacompaniesprovidedahigh-qualitycross-sec&onoftheirin-houselibraries
JointEuropeanCompoundLibrary
EuropeanScreeningCentre
DRUGTARGETS
LIBRARYDESIGNS
EFPIAcontribu-on(>300,000cpds)
Publiccontribu-on(200,000cpdsby2017)
50%EFPIAtargets50%publictargets
Publictargetsthroughcrowd-sourcingprocess
Chemistryconsor&umdesignsandsynthesizedPublicCompoundCollec&on(PCC)
Totalbudget:
€196million
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12www.efpia.eu
Shortandlong-termbenefitsEuropeanLeadFactory
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13www.efpia.eu
EuropeanLeadFactoryWhat’sinitforthestakeholders?
Output
Progress Public programme advanced to be developed within IMI‘s programme; a further project lead progressed as asset in ScandiCure start-up
Knowledge Training and education activities increased public knowledge on early drug discovery; establishment of a network of scientists working in drug discovery across Europe; more than 40 publication in peer-reviewed journals
Public target proposals 129 Accepted
targets 73 Public screens completed 49 Qualified hit
lists (QHLs) 43 Improved hit lists (IHLs) 12
Capabilities Access to a unique, unprecedented screening library, assay development, and state-of-the-art screening including hit validation capabilities ü
2/3 from academia 1/3 from SMEs
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14www.efpia.eu
Goal: Support the evolution towards outcomes-focused and sustainable healthcare systems, exploiting the opportunities offered by big and deep data sources
TheBigDataforBe`erOutcomesprogramme
Design sets of standard outcomes
and demonstrate value
Increase access to high quality outcomes data
Use data to improve value of HC
delivery
Increase patient engagement through
digital solutions
COORDINATION AND SUPPORT ACTION (CSA)
HEMATOLOGIC MALIGNANCIES
PROSTATE CANCER
CARDIOVASCULAR
1 2 3 4
MULTI-DISEASE / MULTI-MORBID PATIENTS
DISTRIBUTED DATA NETWORK
ROADS: ALZHEIMER'S DISEASE
Coordination and operational topics
Themes / Enablers
Disease- specific topics
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15www.efpia.eu
HARMONYprojectToimproveoutcomesinHematologicMalignancies,
weareteamingwithleadingins&tu&onsacrossEurope
HealthcareAllianceforResourcefulMedicinesOffensiveagainstNeoplasmsinHematologY
Pharma Regulators/HTAs
Medicalassocia-ons
Universi-es
Researchinst.andhospitals
Pa-entassocia-ons
Techandbiotech
LargestfundedprojectwithinIMI251partners,including44publicpartnersfrom10differentEUcountries
"IMIprojectsarebestpracJceintheindustry"–FDArepresentaJve
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16www.efpia.eu
Based on feedback from the IMI Stakeholder Forum on AdvancedTherapies,fivepoten-alIMI2topicsarecurrentlybeingconsidered:
PrecisionGenomeEdi-ng(PGE)
Clinicaldevelopmentandpa-entaccess
Clinicaldevelopmentofcelltherapiesincancer
Manufacturing
Immunogenicity
ATMPsKeychallengesandfutureIMI2topics
PrioriJsedfordevelopmentin2017
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17www.efpia.eu
Scope Addressgapsinourunderstandingofprecisiongenomeedi&ng
(PGE)biology,func&onandapplicability. Increaseconfidenceintheaccuracy,safetyandefficacyofthe
technologiesforbothresearchandtherapeu&capplica&ons. Examplesofdeliverables
Novelcharacteriza&onassaysandtoolsforthequan&fica&onofon-target/off-targeteffects,ie.NewDNAanaly&ctechnologiesoradvanced‘nextgenera&onsequencing’(NGS)plahorms. Op&miza&onofexis&ngPGEplahorms-ie.Bioinforma&ctoolsanddesignguidelinestoincreasetargetselec&vity. Developmentofnewpre-clinicalcell/animaltes&ngparadigms,ie: Developandprovideaccesstoqualifiedreagents,plahormsanddata. Definetheboundariesbetweenthecompe&&veandprecompe&&vespace,throughcon&nueddialoguebetweenresearchers,manufacturersandplahormdevelopment,throughouttheprogramme.
PrecisionGenomeEdi-ng(PGE)
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18www.efpia.eu
Scope Frameworkforthedata-enabledop&miza&onofclinicaltrialsfordifferenttypesofATMPs. Infrastructureandmethodologiesfortheefficientu&liza&onofexis&ngandnewregistriesandotherdatarepositories. Enhanceinteroperabilitybetweendatabasesandintegra&onofdata Updatepolicies,processesandqualifica&onpathwaystoassessclinicalu&lityofexis&ngdataandnewevidencerequirements.
Examplesofdeliverables Technicalcapabili&esarounddatasourcestandardsandinteroperability. Qualitystandards,accuracyandregularityofdataentry,repor&ngandanaly&cs. Developnewdatanetworkarchitecturesandlinks,aswellasdatasetqueryprotocoldesigns,toavoidfragmenta&on. Increasebuiltinflexibilitytoaccommodateemergingknowledgeandchangingrequirements. Addresschallengesindatabasesustainability Clarifystatusofpa&entleveldataprotec&on,accesscontrolsandsurveillance. ClinicaltrialregistriescouldalsoexpandtoprovideevidenceinfurthersupportofHTAevalua&ons,focusedonpa&entoutcomes.
Clinicaldevelopment
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19www.efpia.eu
Scope Capturethechallengesacrossthepathwayfromthebenchtothebedside,andacrossthedifferenttypesofATMPs. Clarifyevidencerequirementsforacomprehensiveassessmentandcommercializa&onframework. Allowsufficientflexibilitytoaccommodatethepaceofscien&ficprogress. Securetheappropriateuseofhospitalexemp&onandleverageexis&ngschemes,ie.Orphan/rarediseasefunds.
Examplesofdeliverables Analysisofpipelineprojectsandcommercialproducts,investmentdecisionsetc. Iden&fysuccess/failuredriversandkeygo/no-godecisionfactorsacrosstheproductjourneyfromR&Dtothehealthsystems(casestudies). Deviseanaly&calframeworksandperformanceindicatorstocompareEUcountries,withUSandotherglobalcompe&tors. Model/proposenovelreimbursementandpaymentschemes. TabulatethekeyHTAconsidera&onsandcontrastwithevidenceforregulatoryapprovalsandsurveillance. Analyzecaseexamplesonhospitalexemp&onacrossMemberStates. Iden&fyandevaluateexis&ngandproposenewmodellingmethodsanddatatools(ie.Registries)throughspecificprojectsandworkstreams.
Pa-entaccess
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20www.efpia.eu
Scope Addressgapsinearlyfinancingofproof-of-conceptstudies. Improvecomparabilityofclinicalbenefitofcelltherapiesincancer. Enablecombina&ontherapywithcheckpointinhibitorsandtargeted
therapies Examplesofdeliverables
Public-privatepartnershipinpre-PoCstagetoimprovenumberandqualityofclinicallytestedapproaches Earlyfocusondemonstra&ngclinicalbenefit–aloneorincombina&onwithcheckpointinhibitorsandtargetedtherapies-ofcellulartherapies Searchforbiomarkersofac&vityandmodeofac&onofcelltherapies. Defineandstandardizeproduc&onqualitystandards&specifica&ons. Analysefeasibilityofproduc&ononanadequatescale Focusinves&gatorson„affordabilityandprofitability“. Useofhistoricalandreal-worldevidencetocompareoutcomesinATMPclinicaltrials. Definetheboundariesbetweenthecompe&&veandprecompe&&vespace
Howanoverarchingprojectcouldlooklike:ClinicalDevelopmentofCellTherapiesinCancer
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21www.efpia.eu
Scope Technologicalinnova&onincelltherapyandgenetherapyproduc&on,
withspecificaUen&ontoclosedsystems,automa&onandmonitoringtechnologies
Par&cularemphasisontherapeu&cscaleproduc&onandGMPstandardsatreasonablecost,achievingregulatorycompliance.
Deliverables Twomaintypesofautoma&onprocessesshouldbedevelopedinorderto
addressbothcondi&onsofop&malcellgrowth,namelyforadherentcellsinflasksandforcellsinsuspensionorsemi-suspensioninbioreactors.Robo&sedtechnologiesandcontrolledmethodologiesforcGMPbankingofcelltherapyproducts.Large-scalebankingofclinical-gradecelltherapyend-productsrequiresincreasingappropriatelyallsafetymeasuresanddesigningfullycontrolledproceduresbothforfreezingandthawing.
Addressingtheques&onofahugediversityofcellandgenetherapyproducts.
QualitycontrolsandstandardsusingtheproceduresofQualitybyDesignandQualityRiskManagement(ICHparadigmusedforchemicalcompoundsproduc&on)
Rulesforcon&nuedengagementbetweenscien&sts/manufacturersandregulatorsbasedonriskassessmentalongtheprogramme
ATMPManufacturing
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22www.efpia.eu
Scope Immunogenicityofdifferenttypesofallogeneiccellsources. Impactofthediseaseontheimmuneresponses. Impactoftherouteandscheduleofcelladministra&on. Clinicalinves&ga&onofimmuneresponsesandtheirimpactonsafetyand
efficacyoftheATMP. Deliverables
Understandingtheinnateandadap&veimmuneresponsesagainstdifferentallogeneiccelltypes.
Insightintheintensity,specificity,kine&cs,andpersistenceofsuchimmuneresponses.
Dynamicsofmemoryresponsesuponrepeatedadministra&on. Understandingtheinfluenceofgivenpathologiesonthean&-cellimmune
responses. Clinicalassessmentoftheassociatedsafetyaspects. ClinicalassessmentoftheimpactofimmuneresponsesonATMPefficacy. KnowledgeoftheimpactofHLAmatchingandmismatchingonsafety/
efficacy.
Immunogenicity
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23www.efpia.eu
IMIisdelivering
This is a truepartnership,where companies, publicpartnersandSMEsworktogether
There is an opportunity to transform the ATMPlandscape
Conclusion