NAPSRX CHAPTER 11 (CLINICAL TRIAL) QUESTIONS AND ANSWERS WITH SOLUTIONS 2024

NAPSRX CHAPTER 11 (CLINICAL TRIAL)
QUESTIONS AND ANSWERS WITH
SOLUTIONS 2024
ClinicalFTrialF-
FANSWERFAFstudyFofFhumanFsubjectsFthatFisFintendedFtoFascertainFanFinvestigationalFproduct'sFsafetyFandF

efficacyFbyFanalyzingFits...

-clinical

-pharmacologicalFand/orFotherFpharmacodynamicFeffects

-absorption

-distribution

-metabolism

-excretion

-adverseFreactions



EthicalFConsiderationsF-FANSWERF1.)FSocialFValue



2.)FScientificFValidity



3.)FFairFSubjectFSelection



4.)FInformedFConsent



5.)FFavorableFRisk-BenefitFRatio



6.)FIndependentFReviewFBoard/IndependentFEthicsFCommitteeF(IRB/IEB)



7.)FRespectFforFHumanFSubjects

,SocialFValueF-
FANSWERFClinicalFtrialFisFjustifiedFbasedFonFscientificFresearch,FandFwillFresultFinFimprovementsFinFhealthFor

FadvancementFofFscientificFknowledge




-FThisFrequirementFhelpsFensureFresourcesFareFnotFdirectedFatFnon-
meaningfulFclinicalFresearchFandFthatFhumanFsubjectsFareFnotFexploited



ScientificFValidityF-
FANSWERFtheFclinicalFtrialFshouldFbeFconductedFmethodicallyFwithFclearFobjectivesFandFoutcomesFthatFareF

statisticallyFverifiable



-
theFpreclinicalFandFtoxicologicalFdataFshouldFhaveFbeenFcarefullyFanalyzedFandFshouldFconfirmFtheFscientifi
cFfinding

-theFtrialFshouldFnotFbeFbiased,FandFshouldFbeFexecutedFwithoutFunreasonableFcaveatsFandFconditions



FairFSubjectFSelectionF-
FANSWERFsubjectsFshouldFbeFselectedFbasedFonFscientificFobjectivesFandFnotFonFwhetherFtheFsubjectFisFpri

vilegedForFvulnerable,ForFbecauseFofFconvenience



-inclusionFandFexclusionFcriteriaFareFwellFthoughtFoutFandFdesignedFtoFsatisfyFtheFscientificFdataF

-mustFbeFdocumentedFevidenceFtoFsupportFtheFchoiceFofFselectionFcriteria



InformedFConsentF-
FANSWERFsubjectsFshouldFbeFinformedFaboutFtheFaims,Fmethods,Frisks,FandFbenefitsFofFtheFtrial




-theFavailableFalternativesFshouldFbeFexplained

-subjectsFshouldFnotFbeFpressuredFtoFenrollingFinFtrial,FandFshouldFbeFallowedFtoFleave

-forFyoungFandFincapacitatedFpeople,FproxyFdecisionFfromFtheirFrepresentativesFmustFbeFobtained



FavorableFRisk-BenefitFRatioF-
FANSWERFwhetherFpossibleFclinicalFtrialFsubjectsFshouldFbeFsubjectedFtoFminimalFriskFandFmaximalFbenefit

,-shouldFbeFbasedFonFprovenFscientificFdataFgatheredFfromFpre-clinicalFstage

-shouldFnotFbeFconductedFifFthereFisFanyFdoubtFaboutFtheFrisk-benefitFration



IndependentFReviewFBoard/IndependentFEthicsFCommitteeF(IRB/IEC)F-
FANSWERFinsuresFthatFtheFindependentFpartyFassessesFtheFclinicalFtrialFtoFaddressFtheFquestionFofFconflictFi

nterests



-
actsFasFaFthirdFpartyFtoFoverseeFwelfareFofFtheFtrialFsubjectsFandFensureFtrialFisFconductedFinFaccordanceFtoF
theFstudy

-hasFaFrightFtoFstopFtrialForFrequireFproceduresFandFmethodsFbeFchanged

-members:Fclinicians,Fscientists,Flawyers,FreligiousFpeople,FandFlayFpeople



RespectFforFHumanFSubjectsF-
FANSWERFsubjectsFshouldFbeFprotected,FtheirFprogressFinFtheFtrialFmonitoredFcloselyFandFprovidedFwithFap

propriateFtreatment



-
newFdevelopmentsFinFtheFtrialFmushFbeFrelayedFtoFsubjectsFwithoutFprejudice,FandFsubjectsFdecisionsFsho
uldFbeFhonored

-outcomesFmustFbeFcommunicatedFtoFtheFsubjectsFpromptlyFandFinFanFunbiasedFway



PhaseF1F-FANSWERFtheFfirstFexperimentFinFwhichFaFdrugFisFtestedFonFtheFhumanFbody

-primaryFaimFisFtoFassessFtheFsafetyFofFtheFnewFdrug,Fpharmacokinetics,FandFpharmocodynamics

-10-100FhealthyFvolunteersFrecruited

-dosesFincreasedFasFtrialFprogresses,FmonitoredFcloselyFtoFcheckFtheirFtoleranceFandFsideFeffects

-samplesFtakenFtoFevaluateFabsorption,Fdistribution,Fmetabolism,FandFevaluation

-observationsFonFhowFtheyFfeel,FvitalFsigns,FandFbehavioralFmatters



PhaseF2F-FANSWERFexaminesFtheFsafetyFandFeffectivenessFofFtheFdrugFinFaFtargetedFdiseaseFgroup

-50-500Fpeople,F1-2Fyears

-hasFaFcontrolFgroup,FrandomizedFandFblindedFtoFsubjects

, -helpsFdetermineFeffectiveFdoseFandFtheFdosingFregimenFofFfrequencyFandFduration

-specificFclinicalFendpointsForFmarkersF(definitiveF&Fsurrogate)FassessFinteractionFofFdrugFandFdisease

-
statisticalFanalysisFconductedFtoFevaluateFtheFinfluenceFofFtheFdrugFonFdifferentFpatientFgroupsFandFdeter
mineFoptimumFconditions



PhaseF3F-FANSWERFconfirmsFtheFefficacyFofFtheFdrugFinFaFmushFlargerFpatientFgroup



-severalFhundredFtoFtensFofFthousandsFofFpeople,F2-5Fyears

-multi-siteFtrial:FconductedFinFseveralFlocations

-pivotalFtrial:FitsFresultsFdetermineFtheFeffectivenessFofFtheFdrug

-theseFresultsFhelpFsetFdrug'sFdosage,FtreatmentFfrequency,Fduration,FandFtargetFpatientFgroup

-resultsFsubmittedFtoFregulatoryFauthoritiesFtoFseeFapprovalFtoFmarketFtheFdrug



PhaseF4F-FANSWERFpost-
marketingFapprovalFtrialsFtoFmonitorFefficacyFandFsideFeffectsFofFtheFdrugFinFanFuncontrolledFreal-
lifeFsituationF



-collectFinformationFtoFobserveFdrugsFefficacy,FsideFeffects,FpatientsFqualityFofFlife,FandFcost-effectiveness



InvestigatorF-FANSWERFTheFpersonFwhoFconductsFtheFtrial

-FIfFthereFisFaFteam,FthenFthereFisFaFPrincipalFInvestigator



-
FResponsibilityFisFtoFensureFthatFGCPFisFbeingFimplementedFinFtheFcourseFofFtheFtrial,FthatFtheFsubject'sFrigh

tsFandFwelfareFareFrespected,FthatFtheFstudy'sFtransparencyFisFmaintained,FandFthatFthereFareFnoFconflictsF
ofFinterest



-
FShouldFbeFableFtoFmaintainFimpartiality,FmeaningFthatFtheyFshouldFbeFneitherFemployedFbyFtheFSponsor,Fn

orFotherwiseFexpectedFtoFbenefitFfinanciallyFfromFtheFdrug'sFsuccess