癌癥臨床試驗(英文版)醫(yī)學課件

1、癌癥臨床試驗(英文版)醫(yī)學癌癥臨床試驗(英文版)醫(yī)學The Drug Development and Approval Process1. Early research and preclinical testing2. IND application filed with FDA3. Clinical trials (phases 1, 2, and 3)4. NDA filed with FDA5. FDA validates claim and approves drug2The Drug Development and ApproPhases of Clinical TrialsPha

2、se 1: 15-30 peopleWhat dosage is safe?How should treatment be given?How does treatment affect the body?Phase 2: Less than 100 peopleDoes treatment do what it is supposed to?How does treatment affect the body?3Phases of Clinical TrialsPhasePhases of Clinical TrialsPhase 3: From 100 to thousands of pe

3、opleCompare new treatment with current standardPhase 4: From hundreds to thousands of peopleUsually takes place after drug is approvedUsed to further evaluate long-term safety and effectiveness of new treatment4Phases of Clinical TrialsPhaseTypes of Clinical TrialsTreatmentPreventionScreening and ea

4、rly detectionDiagnosticGeneticsQuality-of-life / supportive care5Types of Clinical TrialsTreatmTreatment TrialsTest safety and effectiveness of new agents or interventions in people with cancer Possible benefit:Early access to new treatmentsPossible risk:Occurrence of unknown side effects6Treatment

5、TrialsTest safety anPrevention TrialsFor people at risk of developing cancerAction studies vs. agent studiesPossible benefit:Early access to new interventionsPossible risk:Unknown side effects and effectiveness7Prevention TrialsFor people atScreening and Early-Detection TrialsAssess new means of det

6、ecting cancer earlier in healthy peoplePossible benefit:Detecting disease at an earlier stage, resulting in improved outcomesPossible risks:Discomfort and inconvenienceIf imaging technique is studied, exposure to x-rays or radioactive substances8Screening and Early-Detection Diagnostic TrialsDevelop

7、 better tools for classifying types and phases of cancer and managing patient carePossible benefits:New technology may be better and less invasiveEarlier detection of recurrencesPossible risk:May require people to take multiple tests9Diagnostic TrialsDevelop betteGenetics TrialsThese trials seek to:

8、Determine how ones genetic makeup can influence detection, diagnosis, prognosis, and treatmentBroaden understanding of causes of cancerDevelop targeted treatments based on the genetics of a tumor10Genetics TrialsThese trials seQuality-of-Life / Supportive Care TrialsAim to improve quality of life fo

9、r patients and their familiesPossible benefit:Early access to new treatmentPossible risk:May not benefit from participation11Quality-of-Life / Supportive CClinical Trial ProtocolA written, detailed action plan that:Provides background about the trialSpecifies trial objectivesDescribes trials design

10、and organizationEnsures that trial procedures are consistently carried out12Clinical Trial ProtocolA writtInvestigational Drug UseOutside of a Clinical TrialGroup C drugsTreatment Investigational New Drug applicationCompassionate use program13Investigational Drug UseOutsiClinical Trial DesignEligibi

11、lity criteria: Can range from general (age, sex, type of cancer) to specific (prior treatment, tumor characteristics, blood cell counts, organ function); eligibility criteria also vary with trial phaseVaries with protocol and phases Endpoint: Measurable outcome that indicates an interventions effect

12、iveness 14Clinical Trial DesignEligibiliClinical Trial DesignRandomization: A method used to prevent bias in research; a computer or a table of random numbers generates treatment assignments, and participants have an equal chance to be assigned to one of two or more groups (e.g., the control group o

13、r the investigational group) 15Clinical Trial DesignRandomizaRandomization16Randomization18Clinical Trial DesignStratification: Categorizing subjects into subgroups by specific characteristicsEnables researchers to look into separate subgroups to see whether differences exist17Clinical Trial DesignS

14、tratificStratification18Stratification20The Final Step: FDA ApprovalReview of New Drug Application (NDA) or Biologics License Application (BLA)LabelingContinued monitoringFeedback19The Final Step: FDA ApprovalReReleasing the Results of Clinical TrialsPeer-reviewed journalsPublic announcementsResults

15、 not made public until end of trial20Releasing the Results of ClinImproving Cancer Prevention, Detection, and TreatmentOnce proven safe and effective in a clinical trial, an intervention may become the new standard of care21Improving Cancer Prevention, DAdvancing Cancer CareGleevec for chronic myelo

16、genous leukemiaCervical cancer: improved survival ratesBreast cancer: less extensive surgeryIntron-A for melanomaBiological therapyMonoclonal antibodiesCancer vaccines22Advancing Cancer CareGleevec fEvolution of Participant ProtectionNurembergTuskegee Syphilis StudyNational Commission for the Protec

17、tion of Human Subjects of Biomedical and Behavioral ResearchBelmont ReportNational Research Act23Evolution of Participant ProteGovernment Oversight of Safeguards for ParticipantsOffice for Human Research Protections (OHRP)The Common RuleFDA Regulations24Government Oversight of SafeguProtecting Parti

18、cipants Before a TrialScientific review by sponsoring organizationInstitutional review board approvalInformed consent25Protecting Participants BeforProtecting Participants During a Clinical TrialInstitutional review boards (IRBs)Data and safety monitoring boards (DSMBs)Minimize risksEnsure integrity

19、 of dataCan stop study if necessary26Protecting Participants DurinBarriers to, Benefits of, and Risks of Participation in Clinical Trials27Barriers to, Benefits of, and Barriers to Adult Participation in Clinical TrialsPhysicians and other health professionals may:Be unaware of appropriate trialsBe

20、unwilling to lose control of patients careBelieve that standard therapy is bestBelieve that clinical trials are more workHarbor concerns about the patients care or how the person will react to suggestion of clinical trial participation28Barriers to Adult ParticipatioBarriers to Adult Participation i

21、n Clinical TrialsPatients may:Be unaware of clinical trialsLack access to trialsFear, distrust, or be suspicious of researchHave practical or personal obstaclesFace insurance or cost problemsBe unwilling to go against their physicians wishes29Barriers to Adult ParticipatioBenefits of Participating i

22、n Clinical TrialsEarly access to new treatmentsActive role in own health careParticipation in advancing medical knowledge30Benefits of Participating in CRisks of Participating in Clinical TrialsNew treatments are not always better than standard careUnexpected side effectsMay not work for everyoneAdditional cost31Risks of Participating in CliNCI-Sponsored C