ANTICIPATINGTHECLINICALDELIVERYOFREGENERATIVEMEDICINESCHALLENGES,TENSIONS&OPPORTUNITIES

23rdMay,2017

JohnGardnerSchoolofSocialSciencesMonashUniversityMelbourne,Australia

ORTHEPROBLEMOFTECHNOLOGYADOPTION

Overview

1.  ThegeneralproblemoftechnologyadopKoninhealthcare2.  TechnologyadopKoninRegeneraKveMedicine–

innovaEonniche3.  ProposedCentresforCellandGeneTherapy4.  PrecedentsandaffordancesforRMtreatmentcentres5.  Insightsfromthesocialsciences•  InsKtuKonalReadiness

ESRCGrantRef:ES/L002779/1

TechnologyadopEoninhealthcareAdopKonprocesseshavebeenoverlookedinthepast…•  e.glinearconceptualisaKonsofinnovaKon(TRLs)

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Nowrecognisedasanimportant&problemaEcaspectofinnovaEon•  High-profileadopKonfailurese.gNaKonalNHSITprogramme•  EmphasisontranslaKonalmedicine(benchtobedside,bedsidetobench)•  RecentHoCScienceandTechnologyCommi]eeReportonRegenMed.

SomecasestudiesoftechnologyadopEoninhealthcare:•  IntraoperaKveBreastLymphNodeAssay(BLNA)(Llewellynetal

2014)•  ThedaVincirobotforprostatectomy(Ulucanlaretal2013)•  E-healthpaKentrecords(Greenhalghetal2010;2013)•  Chronicwoundcaretechnologies(Madden2012)•  CytoriCeluKon®System(Gardneretal2017)•  PaediatricdeepbrainsKmulaKon(Gardner2016;2017)

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Generalfindings:complexity&heterogeneity•  NewtechnologiesmustalignwithinsKtuKonal&professional

interests•  DisKncKonbetweencosteffecKveness&affordabilityishighly

relevant•  Agoodbusinesscase!•  Whatcountsas‘evidence’isnotalwaysclear.Professional

differences,relianceoninformalnetworks…

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SpecificchallengesforregeneraEvemedicines

•  LiveKssues/cellsrequirespecialistinfrastructure&skillsfortransportaEonandpreparaKonattheclinic

•  Newmanufacturing&logisKcsarrangements.ClinicassiteofparEal

orfullmanufacturing.•  Onsitemanufacturingwillrequireexpensivebioprocessing

equipmentsuchascellseparaKon&expansionsystems,systemsfortransfecKonetc.

•  Hospitalsmayneedtoactasprocurementserviceforthirdparty.Contractarrangementsforthiscanbecomplex.HowmightQAandliabiliKesbedistributedamongparKes?

•  Someproposedrisk-sharingcommissioningschemesneedcoordinateddata-collecEoninfrastructures.

•  EmergingtherapiesarediverseandwillpresentvaryinglevelsofdistribuKon.

seeGardneretal2015

flexibleGMPfaciliKes

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ThedeliveryofregeneraEvemedicineswillrequiresignificantorganisaEonal/insEtuEonaladjustmentsConstrucKngan‘innovaEonniche’fornewtechnologies/techniques

Gardner,J.&Webster,A.(2016).TheSocialManagementofBiomedicalNovelty:FacilitaKngtranslaKoninregeneraKvemedicine.SocialScience&Medicine.156:90-7

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ProposedCentresforCellandGeneTherapyTreatmentRecommendedbyRMEGandATMTaskforce

Providestability,enablingconsolidaKonofsupplychains,datacollecKoninfrastructures&trailingandconsolidaKonofbusinessmodels.

•  Consolidateresourcesatseveralcoordinatedclinicalsites

•  ATMTrecommends£30MillionadministeredbyInnovateUK

•  RepresentpartnershipsbetweenNHS&industry

Clinicofthefuture.CentreforProcessEngineering

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Precedents&affordancesfortreatmentcentres

1.   Haematologicalservices,NHSBT,SNBTS•  Reservoirofinfrastructure&experKse(seeLowdell&Thomas2017)

•  Procurement,transportaKon,administering,paKentpreparaKon

•  FamiliaritywithHTA,MHRA,GMPetc

•  NHSBT&SNBTSalreadypartneredinRMinnovaKonalliances

2.   ExisEngrisk-sharingcommissioningarrangements•  ProvisionofsomehighcostcancerdrugsinScotland,enabledby

integratedEPRinfrastructure

•  ElsewhereinEurope,egItaly.GSK‘money-backguarantee’onStrimvelisforADA-SCID

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Precedents&affordancesfortreatmentcentres3.   EstablishmentofaUKProtonBeamTherapyService

•  Highcost,requiringnewinfrastructures,paKent-pathways,&associatedskillsandtrainingwww.england.nhs.uk/•  Considerableinvestmentrequired:£250million•  CoordinatedacKonfromvariousagencies–NHSEngland,FoundaKonTrusts,HealthEducaKonEngland,etc

Similarlevelofinvestment&coordinaEonmayberequiredforimplemenEngsomehighlydisrupEveregenmeds

Fromh]ps://www.england.nhs.uk/commissioning/spec-services/highly-spec-services/pbt/

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Also…meaningfulpaKentengagementinservicedesign

•  RegionaldistribuKonofRegeneraKvemedicinetreatmentcentres

•  AreoutcomemeasuresrelevanttopaKentsandtheirfamilies?Infrastructureforthesemeasures?

•  Psycho-socialdimensions–appropriatesupport?Appropriatelinkswithcommunityservices?

RMtechnologies/techniquesshouldnotbeseenasatechnologicalfix–theyshouldbeapproachedaspartofanongoingregimeofcare

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InsEtuEonalReadiness(IR)InparallelwithTechnologyReadiness

thedegreetowhichorganisa7onsorgroupsarewillingtodeploy,andarecapableofimplemen7ng,anoveltechnologyorprac7ce.

•  A(novel)technologyisarelaKonalproduct,embeddedinamaterial-semioKcnetwork.

•  AdopKonistheresultofacKve,ongoingworkbycreaKveagentswithboundedraKonalityandlimitedresources.

•  DiverseagentsneedtobeacKvelyenrolledintheembeddingprocess-alignworkloadsandexpectaKons.

•  RequiresregularopportuniKesforreflexiveevaluaKon,monitoringSeealsoMay2013

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InsEtuEonalReadiness&RegeneraEveMedicineWhatparametersmightberelevant?

RMTechnology/Technique Context(ClinicalSebng)

PrevalenceofindicaKon Appropriatecapacity

OpportuniKesformeaningfulpaKent/publicinvolvement&collaboraKon?

Place&modeofmanufacturing(e.gcentralised?on-site?)

GMPcleanroomaccess;Bioprocessingcapacity

PreparaKonrequired(product,paKent…) AppropriatelytrainedQP?InfrastructureforQA

Clinicalskillrequiredforadministering Appropriatereservoirofskills,opportuniKesfortraining

Monitorable Infrastructuresforassessment&monitoring

Timeformonitoring&collecKveevaluaKon

QualityofsupporKngevidence InsKtuKonalStrategy/PrioriKes

Stakeholderalignment(e.gmanagers,frontlinestaff,paKents&families)

Affordability Paymentstructures(e.g.tariffs),otherrevenuesources(e.gcharitablegrants).

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IR-L InsEtuEonalReadinessCategoriesinhealthcare1 InsKtuKonshaveoperaKonalgroupstaskedwithengagingandidenKfyingnew

technologies

2 InsKtuKonhasidenKfiedpotenKalnewtherapeuKctechnologies.

3 InsKtuKonhasanoperaKonalgrouptaskedwithassessinginsKtuKonalcapacity/readinessfornewtherapeuKctechnologies

4 AssessmentsofcurrentinsKtuKonalarrangementsfornewtechnologyhavebeenmade

Formalguidelinesexistfor‘readying’thoseinsKtuKonalstructuresinwhichthetechnologywillbeused/produced/assessed

5 Keyindividuals/groupstaskedwithreadyinginsKtuKons(inwhichtechnologywillbeused/produced,assessed)accordingtoguidelines.

6 NovelinsKtuKonalstructuresexist,inanKcipaKonofexpectedchallenges/affordancespresentedbynoveltechnology.Thesestructuresresultfromretrainingofstaff,construcKonofnewspacesetc

7 Noveltechnologyisbeingproduced/used/assessedwithininsKtuKon.TeethingproblemsandunanKcipatedchallenges/affordancesarenoted.

8 NoveltechnologyisrouKnelyproduced/used/assessedwithininsKtuKon.CurrentinsKtuKonalarrangementsaresufficientforrouKneproducKon/assessment/deployment.

InsEtuEonalReadinessLevels…

(Moreworktobedoneonthis…watchthisspace)

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InsEtuEonalReadinessLevels ExamplesClinicalinsKtuKonalcontext GovernanceinsKtuKonalcontext CommercialinsKtuKonalcontext

1 InsKtuKonshaveoperaKonalgroupstaskedwithengagingandidenKfyingnewtechnologies

Clinician(s)constantlylookingfornovelintervenKonsforexisKngcondiKons/illnesses.

Governmentbodies/policymakersconcernedwithcostofhealthcareandnaKonaleconomicperformance.Thesebodies/policymakersacKvelypromoteinnovaKoninhealthcare.

EmployeesorR&Ddivisionofcompanies/academicgroupsacKvelylookingfornewproducts.Or,individuals/groupswithincompaniesacKvelyscouKngforsmallcompanieswithnovelproductstoalignwith.

2 InsKtuKonhasidenKfiedpotenKalnewtherapeuKctechnologies.

Clinician(s)becomeawareofnewintervenKon–perhapsindevelopmentalstageand/orusedinotherhealthcareserngs

Governmentbody/policymakersidenKfyandpromoteparKculartechnologies–suchasRM-aspartoftheirpushforinnovaKon.

PotenKalnewtechnologyidenKfiedandisbeingdevelopedwithincompany,oronecompanyhasidenKfiedpotenKaltechnologyproducedbyanother,andthusestablishesanalliance.

3 InsKtuKonhasanoperaKonalgrouptaskedwithassessinginsKtuKonalcapacity/readinessfornewtherapeuKctechnologies

FormulaKonofspecialistgroup(whichmayonlyinclude‘pioneering’clinicianandclinicaldirector)taskedwithexamininghowwellexisKngNHShospitalworkflows/structureswillaccommodatenewtechnology.Inevitablythiswillinvolveassessingwhethersuchaclinicalservicewouldbefinanciallyself-sustainingwithinthetariffsystem.

Government-appointedgrouptaskedwithassessingthesuitabilityofcurrenthealthcaresystemarrangementsforfacilitaKngofnewtechnologies.Thismayincludeanassessmentofcurrentgovernancestructures(regulatoryagenciesandappraisalbodies).

Groupwithincompanyassessescurrentcapacityoffurtherdevelopment/producKonofnoveltechnology.Thisincludesassessingcurrentmanufacturingplasorms,experKse,componentsuppliers,possiblemarketetc.

4 AssessmentsofcurrentinsKtuKonalarrangementsfornewtechnologyhavebeenmade

Formalguidelinesexistfor‘readying’thoseinsKtuKonalstructuresinwhichthetechnologywillbeused/produced/assessed

EstablishmentofproposalforadjusKngNHSclinicalsitessothatthenewtechnologycanbeintegratedintoanew/exisKngclinicalservice.Suchproposalswilloutlinethevariousresourcesrequired:staff,supporKngtechnologies,clinicalarchitectureetc

Governmentappointedgroupproposesformalchangestocurrentgovernancestructures.SuchproposalsmayincludenewregulatoryclassificaKonsystem,newformsofcost-effecKvenessanalysis,theappointmentofindividuals/groupswithparKcularexperKsetoregulatoryagenciesandappraisalbodies.

Companyhasbusinessplan/viableeconomicmodelfordevelopingandproducingnewtechnology.

5 Keyindividuals/groupstaskedwithreadyinginsKtuKons(inwhichtechnologywillbeused/produced,assessed)accordingtoguidelines.

Pioneeringclinician(orclinicalleadsareappointed)and/ormanagertaskedwithbringingtheseresourcestogether.

Individuals/groupswithingovernancestructures(suchasEMA,NICE,MHRA)aretaskedwithbringingaboutrequiredchanges.Theseindividuals/groupsmayengageinpublicconsultaKonaspartoftheir‘readyingacKviKes’.

Keymanager(s)aretaskedwithenacKngbusinessplan.ThismayincludesecuringaddiKonalcapital.

6 NovelinsKtuKonalstructuresexist,inanKcipaKonofexpectedchallenges/affordancespresentedbynoveltechnology.Thesestructuresresultfromretrainingofstaff,construcKonofnewspacesetc

Appropriateclinicalspace/nicheiscreatedforthenoveltechnology,accordingtothespecialistgroupproposal.ThisclinicalspacemaybeparKcularlynovel(suchastheformulaKonofanewmulKdisciplinaryteam,ortheconstrucKonofanewmaterialenvironmenttohousethetechnology),orerelaKvelyminor(retrainingone/severalstaffmembersofexisKngclinicalteam).

Newgovernancearrangementsareformallyestablished.Thismayincludetheestablishmentofnewclassificatorysystemfornoveltechnologiesoranewcommi]eewithspecificexperKse.

Sufficientinvestmentissecuredandbusinessplanisenacted-thisinvolvessecuringappropriatesuppliersofcGMP-compliantsupplies,construcKngmaterialinfrastructurefortechnologyproducKon,hiring&trainingstaff.

7 Noveltechnologyisbeingproduced/used/assessedwithininsKtuKon.TeethingproblemsandunanKcipatedchallenges/affordancesarenoted.

Newtechnologyisdeployedinanactualclinicalserng.Whileworkingwiththetechnology,cliniciansbecomeawareof(inevitable)problems/affordancesrequiringsmall-scale,ad-hocalteraKonstoexisKngstructures/rouKnesetc.

Thegovernancestructuresare‘processing’thenewtechnology:regulatoryagenciesandappraisalbodiesareassessingthenew‘class’oftechnologies.However,keyindividuals/groupshavenotedthatfurtherminoradjustmentsmaybenecessary.Forexample,itmightbenotedthattheclassificatorysystemisunclearandrequiresfurther‘tweaking’.

Noveltechnologyisproducedbythecompany.Furtherad-hocadjustmentsarenecessarybeforeefficientproducKoncanberouKnized.

8 NoveltechnologyisrouKnelyproduced/used/assessedwithininsKtuKon.CurrentinsKtuKonalarrangementsaresufficientforrouKneproducKon/assessment/deployment.

NewtechnologyisrouKnelydeployed.StaffhaveappropriateexperKse,trainingandtacit/embodiedknowledgerequiredtooperaKonalisenewtechnology.

GovernancestructuresrouKnely‘process’thenewclassoftechnology.Whiletheremaybesomeindividualsorgroups(suchasindustry)thatbelievesthecurrentstructuresareinadequate,suchviewpointsdonotcarrysufficientweighttopromptchangewithingovernancestructures.

NoveltechnologyisrouKnelyproducedbythecompany.Companyhasaprovenmanufacturingplasormandaviable,proveneconomicmodel.

(Moreworktobedoneonthis…watchthisspace)

THANKYOU

JohnGardnerSchoolofSocialStudiesMonashUniversityMelbourne,Australia

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