Clinical Trial Process NO Pics

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Clinical Trial Process: AnClinical Trial Process: AnOverviewOverview

Dennis R.Dennis R. DeRosiaDeRosia, PA, MA, PA, MADirector, Business DevelopmentDirector, Business Development

Profil Institute for Clinical ResearchProfil Institute for Clinical Research

[email protected]@profil--research.comresearch.com

Past President & COBPast President & COB

Association of Clinical Research ProfessionalsAssociation of Clinical Research Professionals

www.acrpnet.orgwww.acrpnet.org


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What Is a Clinical Trial?What Is a Clinical Trial?

Effectiveness of intervention to treat aEffectiveness of intervention to treat adiseasedisease

Safety of a new drug or deviceSafety of a new drug or device Defining dose administrationDefining dose administration

Testing drug formulationTesting drug formulation

Exploring combination therapiesExploring combination therapies

Evaluating effect of therapies on quality ofEvaluating effect of therapies on quality oflifelife


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Types of Clinical TrialsTypes of Clinical Trials

TreatmentTreatment

Test new approaches to treat a diseaseTest new approaches to treat a disease

PreventionPreventionWhat approaches can prevent diseaseWhat approaches can prevent disease

EarlyEarly--detection/screeningdetection/screening

What are new ways to find hidden diseaseWhat are new ways to find hidden disease DiagnosticDiagnostic

How can new tests or procedures ID diseaseHow can new tests or procedures ID disease


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Phases of Drug DevelopmentPhases of Drug Development

Phase 1Phase 1 Phase 2Phase 2 Phase 3Phase 3 Phase 4Phase 4

No. ofNo. of

ParticipantsParticipants

1515--3030 100100--300300 100 to100 to

thousandsthousands

SeveralSeveral

hundreds tohundreds to

severalseveral

thousandsthousands

PurposePurpose First inFirst in

humanshumans

safe dosesafe dose

POCPOC

1/3 fail1/3 fail

DetermineDetermine

efficacyefficacy

SafetySafety

50% fail50% fail

CompareCompare

new agentnew agent

withwithstandardstandard

treatmenttreatment

SafetySafety

1/3 fail1/3 fail

PostPost marketmarket

LongLong--termterm

safety andsafety andefficacyefficacy


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Evolution of RegulationsEvolution of Regulations

19381938 Food, Drug & Cosmetic ACTFood, Drug & Cosmetic ACT

19621962 KefauverKefauver--Harris AmendmentHarris Amendment

19681968 Drug Efficacy Study ImplementationDrug Efficacy Study Implementation 19811981 IRB Review RequiredIRB Review Required

19831983 Orphan Drug ActOrphan Drug Act

19971997 ICHICH--E6 Good Clinical Practice (GCP)E6 Good Clinical Practice (GCP)

19981998 Pediatric RulePediatric Rule

20002000 NIH launches www.clinicaltrials.govNIH launches www.clinicaltrials.gov


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Human Research is HighlyHuman Research is Highly

RegulatedRegulated Code of Federal Regulations (CFR)Code of Federal Regulations (CFR)

Title 21Title 21-- Food and DrugsFood and Drugs

Part 50 Informed ConsentPart 50 Informed Consent

Part 56 IRBPart 56 IRB

Part 312 INDPart 312 IND

Part 314 NDAPart 314 NDA

Part 600, 6001 BiologicsPart 600, 6001 Biologics

Part 812, 813, 814 Medical DevicesPart 812, 813, 814 Medical Devices

Title 45Title 45-- PublicWelfarePublicWelfare

Part 46Part 46 (subparts B, C, D)(subparts B, C, D) DHHS, Protection of Human subjectsDHHS, Protection of Human subjects


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What About InternationalWhat About International

Regulation?Regulation? International Conference on HarmonizationInternational Conference on Harmonization

E6 Good Clinical Practice (GCP):E6 Good Clinical Practice (GCP):

Consolidated GuidanceConsolidated Guidance International ethical and scientific qualityInternational ethical and scientific quality

standard for designing, conducting, recordingstandard for designing, conducting, recording

and reporting trial results. (US, EU & Japan)and reporting trial results. (US, EU & Japan)


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Why is Human Research HighlyWhy is Human Research Highly

Regulated?Regulated? Past transgressions lead to the need for lawsPast transgressions lead to the need for laws

that protect the rights and welfare of humanthat protect the rights and welfare of human

subjects.subjects. Nuremberg Doctors Trial of 1946 (Nuremberg Code)Nuremberg Doctors Trial of 1946 (Nuremberg Code) Thalidomide Tragedy (KefauverThalidomide Tragedy (Kefauver--Harris Amendment)Harris Amendment)

Tuskegee Experiments (Belmont Report)Tuskegee Experiments (Belmont Report)

Human Radiation ExperimentsHuman Radiation Experiments Gene Transfer ExperimentGene Transfer Experiment


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Research Protocol: RoadmapResearch Protocol: Roadmap

Detailed Research Plan that Includes:Detailed Research Plan that Includes:

ObjectivesObjectives

Background and RationaleBackground and Rationale Subject Selection CriteriaSubject Selection Criteria

Treatment PlanTreatment Plan

Study ProceduresStudy Procedures

Response Evaluation CriteriaResponse Evaluation Criteria

Statistical SectionStatistical Section


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ProtocolsProtocols A Phase I, DoubleA Phase I, Double--blind, Placeboblind, Placebo--

controlled, Dosecontrolled, Dose--escalation Study of theescalation Study of the

Safety and Pharmacokinetics of Drug A31Safety and Pharmacokinetics of Drug A31

in Subjects with Type 2 Diabetes Mellitusin Subjects with Type 2 Diabetes Mellitus A randomized, doubleA randomized, double--blind, placeboblind, placebo--

controlled study assessing the effect ofcontrolled study assessing the effect of

(study drug) Controlled(study drug) Controlled--Release Tablet onRelease Tablet on

hypoglycemia in type 1 diabetic subjectshypoglycemia in type 1 diabetic subjects

intensively treated with insulinintensively treated with insulin


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Protocol MouthfulProtocol Mouthful

A PHASE 1, RANDOMIZED, PLACEBOA PHASE 1, RANDOMIZED, PLACEBO--

CONTROLLED, SEQUENTIAL PARALLELCONTROLLED, SEQUENTIAL PARALLEL

GROUP, MULTIPLEDOSEESCALATIONGROUP, MULTIPLEDOSEESCALATION

TRIAL TO EVALUATE THESAFETY,TRIAL TO EVALUATE THESAFETY,TOLERABILITY, PHARMACOKINETICS,TOLERABILITY, PHARMACOKINETICS,

AND PHARMACODYNAMICSOF 28 DAYSAND PHARMACODYNAMICSOF 28 DAYS

OFADMINISTRATION OF NDOFADMINISTRATION OF ND-- 0025400254

TABLETS TO SUBJECTS WITH TYPE 2TABLETS TO SUBJECTS WITH TYPE 2

DIABETESMELLITUSDIABETESMELLITUS


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Study Flow ChartStudy Flow Chart


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Study Flow ChartStudy Flow Chart


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Institutional Review BoardInstitutional Review Board

(IRB)(IRB) All clinical trials must be approved andAll clinical trials must be approved and

monitored by an IRB.monitored by an IRB.

IRB is an independent committee ofIRB is an independent committee ofphysicians, nurses, statisticians, communityphysicians, nurses, statisticians, community

advocates and others.advocates and others.

The function of the IRB is to ensure that aThe function of the IRB is to ensure that a

clinical trial is ethical and the rights welfareclinical trial is ethical and the rights welfare

of study participants are protected.of study participants are protected.


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Informed ConsentInformed Consent

Learning the key facts about a trialLearning the key facts about a trial beforebeforedeciding whether to participate.deciding whether to participate.

Research study purposeResearch study purpose

Risks/BenefitsRisks/Benefits

Alternative treatmentsAlternative treatments

Confidentiality of recordsConfidentiality of records

Medical treatment available if injury occursMedical treatment available if injury occursWhom to contact for answers to questionsWhom to contact for answers to questions

Statement that participation isStatement that participation is voluntaryvoluntary


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Patient Recruitment ChallengePatient Recruitment Challenge

Poor patient recruitment is the number onePoor patient recruitment is the number one

reason that trials fail.reason that trials fail.

Only 3 to 5 percent of newly diagnosedOnly 3 to 5 percent of newly diagnosedadult cancer patients participate in a clinicaladult cancer patients participate in a clinical

trial.trial.

Reasons for this relatively low number areReasons for this relatively low number are

many.many.


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Recruitment StrategiesRecruitment Strategies

Physician trust and contactPhysician trust and contact

Study staff contactStudy staff contact

Speaking to community groupsSpeaking to community groupsNewspaper and radio AdsNewspaper and radio Ads

Internet websitesInternet websites

Physician referralsPhysician referrals


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Subject Data CollectionSubject Data Collection

Data is collected on case report formsData is collected on case report forms

(CRF)(CRF)

Much of clinical data is taken from theMuch of clinical data is taken from thesubjects medical record (source documents)subjects medical record (source documents)

Pharmaceutical and device trials, data isPharmaceutical and device trials, data is

verified by multiple playersverified by multiple players


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Serious Adverse EventsSerious Adverse Events

Events that results in any of the following:Events that results in any of the following:

Death or lifeDeath or life--threateningthreatening

Hospitalization or prolonged hospitalizationHospitalization or prolonged hospitalization

Persistent or significant disability/incapacityPersistent or significant disability/incapacity

Congenital anomaly/birth defectCongenital anomaly/birth defect

Events that are serious, unexpected, andEvents that are serious, unexpected, and

related or possibly related to participation inrelated or possibly related to participation inthe research must be reported to thethe research must be reported to theSponsor, FDA and IRB in a timely manner.Sponsor, FDA and IRB in a timely manner.


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Clinical Trial End ProductClinical Trial End Product

Ideal: Unambiguous conclusion regardingIdeal: Unambiguous conclusion regarding

the clinical outcome of the testthe clinical outcome of the test

treatment/device.treatment/device. Always strive for the ideal, but in mostAlways strive for the ideal, but in most

cases have to settle for the best comprise.cases have to settle for the best comprise.


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PositivePositive--leaning articles tend to focus onleaning articles tend to focus on

gains made in fighting particular diseases.gains made in fighting particular diseases.

Alzheimers vaccine study promising

Treatment for cancer advances in trials

A promising weapon in the fight against MS

* * * * * * * * * * * * * * * * * * * * *

Medical Miracles or Misguided Media

The Los Angeles Times

It sometimes seems as if there are Page 1 stories, television news reports and

magazine cover stories almost daily on medical breakthroughs - new treatments

for everything from the flu, obesity, AIDS and heart disease.


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News UpdateNews Update

Medical clinical research slows for lack of patients

Los Angeles Times March 14, 2009

Enrollment problems delay more than 70% of clinical trials

from one to six months

In cancer care, less than 5% of patients enter clinical trials

700 cancer therapies in pipeline


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News UpdateNews Update

Its time to speed up drug approvals

The San DiegoUnion-Tribune July 11,2008by Sally C. Pipes

FDA approved just 18 cancer drugs in past three years

CyberKnife shoots beams of radiation at tumors Every day 1,500 Americans die from cancer


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Coalition of National CancerCoalition of National Cancer

Cooperative GroupsCooperative Groups


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Pay Attention to the ClinicalPay Attention to the Clinical

Research Around YouResearch Around You

You may be the

beneficiary some day!

Thank you

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