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USFDAExpeditedProgramsandExpandedAccess
KeLiu,MD,PhDChief,OncologyBranchDivisionofClinicalEvalua;on,PharmacologyandToxicologyOfficeofTissuesandAdvancedTherapiesCenterforBiologicsEvalua;onandResearchOp;misingthedevelopmentofATMPstomeetpa;entneedsLondon,UnitedKingdomDecember16,2016
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Disclosures
Ihavenofinancialrela;onshipstodisclose.Iwillnotdiscussoff-labeluseofproducts.
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Outline
• USFDAExpeditedPrograms– PriorityReviewDesigna;on:1992– AcceleratedApproval:1992– FastTrackDesigna;on(FTD):1997– BreakthroughTherapyDesigna;on(BTD):2012
• USFDAExpandedAccess
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FDAExpeditedPrograms:Goalsv Fordrugsthataddressanunmetmedicalneedinthe
treatmentofaseriousorlife-threateningcondi;onv Intendedtohelpensurethatdrugsforthesecondi;ons
areapproved&availabletopa;entsassoonasitcanbeconcludedthatthetherapies’benefitsjus;fytheirrisks
v Allowforearliera]en;ontodrugsthathavepromiseintrea;ngsuchcondi;ons§ Earlyconsulta;onwithFDA
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FDAExpeditedProgramsGuidance
v GuidanceforIndustry:ExpeditedProgramsforSeriousCondi;ons–DrugsandBiologics(2014)
§ Singleresourceforinforma;ononFDA’spolicies&proceduresforfourexpeditedprograms
§ Describesthresholdcriteriaapplicabletoconcludingthatadrugisacandidateforanexpediteddevelopmentandreviewprogram
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Non-Clinical
Early Clinical
Phase 3 Trial(s)
NDA/BLA Submission APPROVAL
IND Submission
FDA Review
Priority Review
Fast Track Accelerated Approval
Breakthrough Therapy
FDAExpeditedPrograms
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v Qualifyingcriteria
v Features
v Breakthroughvs.FastTrack
BreakthroughTherapyDesigna;on(BTD)
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BTD:QualifyingCriteria
v A drug that
§ Is intended to treat a serious condition AND
§ Preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints
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BTD:QualifyingCriteriav Serious Condition
§ “condition” : A disease or illness § Including life-threatening conditions § A clinical judgment, based on the condition’s
impact on factors, such as: o Survival o Day-to-day function, OR o The likelihood that the condition, if left untreated, will
progress from a less severe condition to a more serious one
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BTD:QualifyingCriteriav Intended to have an effect on a serious
condition or a serious aspect of a condition
§ A direct effect on a serious manifestation or symptom of a condition
§ Other intended effects, such as
o A product intended to improve or prevent a serious treatment-related side effect
o A product intended to prevent a serious condition or reduce the likelihood that the condition will progress to a more serious condition or a more advanced stage of disease
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BTDQualifyingCriteria
v Preliminaryclinicalevidenceofsubstan;alimprovementoveravailabletherapyononeormoreclinicallysignificantendpoints
§ IsapprovedorlicensedintheUnitedStatesforthesameindica;on,AND
§ IsrelevanttocurrentUSstandardofcare(SOC)fortheindica;on
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BTDQualifyingCriteria
v Preliminaryclinicalevidence§ Notsufficient(qualityand/orquan;ty)toestablishsafetyand
effec;venessforpurposesofapproval§ GenerallyderivedfromPhase1or2trials§ Shouldinvolveasufficientnumberofsubjectstobeconsidered
credible§ Ideallyderivedfromastudycomparingthedrugtoanavailable
therapy(orplacebo,ifnoavailabletherapy),orfromastudycomparingthedrug+SOCtotheSOCalone
§ Single-armstudiescomparingthestudysubjects’clinicalcoursewithwell-documentedhistoricalexperience,ifthemagnitudeofdifferenceislarge
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BTDQualifyingCriteria
v Substan;alimprovement§ Ama]erofjudgment§ Dependson:
o Themagnitudeofthedrug’seffectonaclinicallysignificantendpoint(includingdura;onoftheeffect) AND
o Theimportanceoftheobservedeffecttothetreatmentoftheseriouscondi;onorseriousaspectofthecondi;on
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BTDQualifyingCriteria
v Approachestodemonstratesubstan;alimprovement§ Directcomparisonofthedrugtoavailabletherapyshowsamuch
greaterresponse
§ Ifthereisnoavailabletherapy,thedrugshowsaclinicallymeaningfuleffectonanimportantoutcomewhencomparedtoplacebo
§ Thedrugplusavailabletherapyresultinamuchgreaterresponsecomparedtoavailabletherapyalone
§ Thedrugreversesorinhibitsdiseaseprogression,incontrasttoavailabletherapythatprovidesonlysymptoma;cimprovement
§ Thedrughasanimportantsafetyadvantagecomparedwithavailabletherapy,andhassimilarefficacy
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BTDQualifyingCriteriav Clinicallysignificantendpoint
§ Anendpointthatmeasuresaneffectonirreversiblemorbidityormortality(IMM)oronsymptomsthatrepresentseriousconsequencesofthedisease.
§ AnendpointthatsuggestsaneffectonIMMorserioussymptoms,including:o Aneffectonanestablishedsurrogateendpointthattypicallywould
beusedtosupporttradi;onalapprovalo Aneffectonasurrogateendpointorintermediateclinicalendpoint
consideredreasonablylikelytopredictaclinicalbenefit(i.e.,theacceleratedapprovalstandard)
o Asignificantlyimprovedsafetyprofilecomparedwithavailabletherapy(e.g.,lessdose-limi;ngtoxicityforanoncologyagent),withevidenceofsimilarefficacy
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Benefits/FeaturesofGrantedBTD
v All benefits of Fast Track designation § FDA takes actions to expedite development and
review § Eligible for rolling review of NDA or BLA (submission
and review of portions of an application before submission of the complete application)
v Intensive guidance on efficient drug development during IND, beginning as early as Phase 1
v Organizational commitment involving FDA senior managers
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FTDvsBTD:Similari;es
v Natureofprograms:Designa;onv TimelineforFDAresponse:Within60calendardaysv Intendtotreatseriouscondi;onv Benefits:FDA’sAc;onstoexpeditedevelopmentandreview
§ Frequentinterac;onswiththereviewteam§ Maybeeligibleforpriorityreviewifsupportedbyclinicaldataatthe
;meofBLA/NDAsubmission§ Mayqualifyforrollingreview
v Designa;onmayberescindedifnolongermee;ngthequalifyingcriteria
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FTDvsBTD:Differences
FTD BTD
RequirementsforDesigna>on
SourceofData
Non-clinical or clinical data
Preliminary clinical evidence
Strength
ofEvidence
The potential to address unmet medical need
Substantial improvement on a clinically significant endpoint(s)
over available therapy
DevelopmentPlan
Specify how this potential will be evaluated in the drug
development program (e.g., a description of the Phase 3
trials)
Not required
Benefits
FDA takes actions to
expedite development and review
• All benefits of FTD • Intensive guidance on an
efficient drug development program
• Involvement of FDA senior managers
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BreakthroughDesigna>onExperience
inCBEROTAT
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BTRequestsinOCTGT(12/2012–06/2016)
v TotalRequests:54§ 47products§ 7Repeatedrequests
37
15
2
Requests Denied
Requests Granted
Requests withdrawn
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BTDsbyProductTypes
Products Requested BTDs
Granted BTDs
Gene 26 12
Cellular 16 1
Tumor Vaccine 4 1
Oncolytic Virus 1 1
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BTDsbyIndica;ons
Indications Requests Granted Oncology 33 9
Non-oncology 21 6 Hematology 4 3
Ophthalmology 3 1
Cardiology 3 1
Neurology 4 1 Transplantation 2 0
Nephrology, Peripheral Vascular,
Burn, Hepatology
1 in each specialty
0
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CommonReasonsforBTDDenial
v Evidenceistoopreliminary(quan;tyand/orquality)tobeconsideredreliable§ Smallsamplesize§ Lackofappropriatecontrol§ Post-hocanalysesoffailedstudiesthatiden;fyasubsetthatmaybenefit
v Improvementoveravailabletherapydoesnotappeartobe“substan;al”
v Modifica;onofproduct
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WhatOTATHasLearned
v BTDdecisionsarecomplex.§ Thereisnoone-size-fits-allcharacteriza;onofaBTproduct.
v Thereliabilityandpersuasivenessofclinicalevidenceiscri;caltomakingtheBTDdecision.§ Thereisnotadefini;vethresholdforsubstan;al
improvement.
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ExpandedAccesstoInves>ga>onalDrugs
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WhatisExpandedAccess?
• Useofaninves;ga;onaldrugtotreatapa;entwithaseriousdiseasewhohasnoothersa;sfactoryop;ons
• IntentisTREATMENT;alsocalled“CompassionateUse”
• Contrastwithusinganinves;ga;onaldruginaclinicaltrial,wheretheprimaryintentisRESEARCH
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TypesofExpandedAccessPrograms(EAPs)
TherearethreetypesofEAPsdefinedinthecodeoffederalregula;ons:
Individual Intermediate Treatment
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RequirementsforallEAPs21CFR312.305
• Seriousorimmediatelylife-threateningillnessorcondi;on
• Nocomparableorsa;sfactoryalterna;vetherapy
• Poten;albenefitjus;fiesthepoten;alrisksofthetreatment(risksarenotunreasonableinthecontextofthedisease/condi;onbeingtreated)
• Providingdrugwillnotcompromiseproductdevelopment
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HumanSubjectProtec>onsApplytoAllEAPs
DrugsinEAPsareinves*ga*onaldrugs,andtheyaresubjecttothefollowingrequirementsfrom21CFR:
– Part50-Protec;onofHumanSubjects(informedconsent)– Part56-Ins;tu;onalReviewBoard
– Part312-includingClinicalHoldsbasedonsafetyandrepor;ngrequirements(adverseeventreports,annualreports)
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IndividualPa>entEAPs21CFR312.310
• Physicianmustdetermineprobableriskfromdrugdoesnotexceedthatfromdisease
• FDAmustdeterminethatthepa;entcannotobtainaccessunderanothertypeofIND
• Proceduresforemergencyuse(whenthereisnot;metomakeawri]enINDsubmission)– FDAmayauthorizeaccesswithoutsubmission,withveryquickturn-around(F/Uwri]ensubmissionrequiredwithin15workingdaysofauthoriza;on)
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ObtainingaSinglePa>entIND
PhysicianandPa>ent/FamilyDiscussRisks&
Benefits
ApprovalFromIRB
AgreementFromDrugCompany
SubmitForm3926toFDA,forapproval
TreatPa>ent
To provide drug, and for FDA to reference
commercial IND
30-45 minutes!! Turn around time generally < 48h,
99.4% approval rate
• Form 3926 is 2 pages and includes: o Brief medical history and rationale for trying drug o Proposed treatment plan with safety /efficacy monitoring
• Also submit: o Letter of authorization from sponsor o Investigator qualification statement / form 1571
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IntermediateSizePopula>on21CFR312.315
• Intended for situations where multiple patients with the same condition might benefit from a particular investigational product
• No set numerical parameters – meant to be practical – more than a few, and less than a lot
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TreatmentIND21CFR321.320
• Drugisbeinginves;gatedinclinicaltrialdesignedtosupportmarke;ng,ortrialsarecomplete
• Companyisac;velypursuingapproval• Sufficientevidenceofsafety&effec;veness
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Cau>onsforEAPUse• Riskhasnotbeenestablishedforinves;ga;onaldrug
– Confidenceinsafetymoreimportantthanconsidera;onofefficacy
– Forachildwithanimmediatelife-threateningcondi;on,evidenceburdenislow
• Poten;albenefitisomenoveres;mated– DruggivenunderEAPwithinten;ontoprovidebenefit– Anecdotalevidenceofevenoverwhelmingefficacymay
holduponlyinaverysmallsubsetofpa;ents,buthavetoxici;esthatincreasesufferingand/orhastendeathineveryoneelse
• Poten;alfornega;veimpactonclinicaldevelopmentplan
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BenefitsandBarriersBENEFITS:• Provideaccessandhopetopa;entswithno
alterna;ves,willingtoacceptpoten;allygreaterrisk
• Mayprovidepa;entswithameasureofautonomyovertheirownhealthcaredecisions
• Can be a foothold into marketplace for sponsors
BARRIERS: • Paperwork/time (New! Form 3926) • Manufacturing (drug availability) • Fear that adverse events may disrupt clinical product
development
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CBEROTATDCEPTOncologyBranchMembers
KeLiu,MD,PhD(BC)
LaronnaColbert,MD
SadhanaKaul,MD
PeterBross,MD(TL) Ching-Hsien(Jessica)Lee,MD,PhD
LydiaMartynec,MD
AdnanJaigirdarMD
Open
Posi;on(M.O.)
ChaohongFan,MD,PhD
Open
Posi;on(M.O.)
Open
Posi;on(TL)
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OTATContactInforma>on
RegulatoryQues;ons:• ContacttheRegulatoryManagementStaffinOTAT:[email protected]@fda.hhs.govorbycalling(301)827-6536
• OCTGTLearnWebinarSeries:
h]p://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ucm232821.htm