Interventional trials: A review. Study designs, Trends.

Prezentace na téma: "Interventional trials: A review. Study designs, Trends."— Transkript prezentace:

1 INTERVENTIONAL TRIALS: A REVIEW Quality of Study Designs | Trends Studies & Enrollment Source: ClinicalTrials.gov Data Download Date: December 26, 2016 Publication Date: January 7, 2017 Analytical tool: IBM Watson ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

2 Interventional trials 187,607 studies Participants are assigned to receive one or more interventions, or none at all, as assigned in the protocol, so that researchers can evaluate their effects on biomedical or health-related outcomes. Interventions can be diagnostic, therapeutic, or behavioral. January 5, 2017

3 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Priority Information Requirements How are study designs changing over time? Is the level of evidence produced improving? What are the trends in quality parameters of study designs? Can quality of studies be measured using the available data? What are the trends for number of studies? How trends in number of trials compares to enrollment? Which interventions are studied the most? Who are the sponsors of clinical trials? Where the funding comes from? What are the main reasons to conduct clinical trials? What are the endpoints? What are the trends, are they changing? What is the primary purpose? Are the trends changing? How? Are the results available? In what form?

4 INTERVENTIONS Drugs | Biological | Device | Procedure | Radiation Behavioral | Dietary Supplement | Genetic | Other ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

5 Clinical trials by Interventions Number of trials Enrolled patients Behavioral Biological Device Dietary supplement Drug Genetic Other Procedure Radiation

6 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Interventions Breakdown by phase (Number of Studies) Behavioral Biological Device Dietary supplement Drug Genetic Other Procedure Radiation

7 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Interventions Breakdown by phase (Enrollment) Behavioral Biological Device Dietary supplement Drug Genetic Other Procedure Radiation Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

8 Clinical trials by Interventions Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Behavioral Biological Device Dietary supplement Drug Genetic Other Procedure Radiation

9 PHASES Phase 0 | Phase 1 | Phase 2 | Phase 3 | Phase 4 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

10 Clinical trials by Phases Phase 0 Phase 1 Phase 1|2 Phase 2 Phase 2|3 Phase 3 Phase 4 Blank Number of trials Enrolled patients Phase 0: Exploratory study involving very limited human exposure to the drug (screening, microdose studies). Phase 1: Studies conducted with healthy volunteers to find most important safety issues see how the drug is metabolized and excreted. Phase 2: Studies that gather preliminary data on effectiveness. Safety continues to be evaluated. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages. Phase 4: Studies occurring after FDA has approved a drug for marketing to gather additional information about a drug's safety, efficacy, or optimal use.

11 Clinical trials by Phases Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Phase 0 Phase 1 Phase 1|2 Phase 2 Phase 2|3 Phase 3 Phase 4 Blank

12 DESIGN: PRIMARY PURPOSE Basic Science | Diagnostic | Educational | Prevention | Screening Health Science Research | Treatment | Supportive Care ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

13 Clinical trials by Primary Purpose Basic Science Diagnostic Educational/Counseling/Training Health Services Research Prevention Screening Supportive Care Treatment Blank Number of trials Enrolled patients

14 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Primary Purpose Breakdown by phase (Number of Studies) Basic Science Diagnostic Educational/Counseling/Training Health Services Research Prevention Screening Supportive Care Treatment Blank

15 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Primary Purpose Breakdown by phase (Enrollment) Basic Science Diagnostic Educational/Counseling/Training Health Services Research Prevention Screening Supportive Care Treatment Blank Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

16 Clinical trials by Primary Purpose Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

17 FUNDING Industry | NIH | Other | U.S. Federal Agencies Combined Funding ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

18 Clinical trials by Funding Number of trials Enrolled patients Industry Industry + other entities NIH NIH + other entities Other Other + other entities U.S. Federal Agencies U.S. Fed + other entities

19 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Funding Breakdown by phase (Number of Studies) Industry Industry + other entities NIH NIH + other entities Other Other + other entities U.S. Federal Agencies U.S. Fed + other entities

20 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Funding Breakdown by phase (Enrollment) Industry Industry + other entities NIH NIH + other entities Other Other + other entities U.S. Federal Agencies U.S. Fed + other entities Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

21 Clinical trials by Funding Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Industry Industry + other entities NIH NIH + other entities Other Other + other entities U.S. Federal Agencies U.S. Fed + other entities

22 DESIGN: ENDPOINT CLASSIFICATION PK/PD | Safety | Efficacy | BA/BE ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

23 Clinical trials by Endpoint Classification Number of trials Enrolled patients Bio-availability study Bio-equivalence study Efficacy study Pharmaco-dynamic study Pharmaco-kinetic study PK/PD Safety study Safety/Efficacy study Blank

24 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Bio-availability study Bio-equivalence study Efficacy study Pharmaco-dynamic study Pharmaco-kinetic study PK/PD Safety study Safety/Efficacy study Blank Clinical trials by Endpoint Classification Breakdown by phase (Number of Studies)

25 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Endpoint Classification Breakdown by phase (Enrollment) Bio-availability study Bio-equivalence study Efficacy study Pharmaco-dynamic study Pharmaco-kinetic study PK/PD Safety study Safety/Efficacy study Blank Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

26 Clinical trials by Endpoint Classification Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

27 QUALITY ATTRIBUTES Allocation | Intervention Model | Masking ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

28 QUALITY of EVIDENCE LEVEL ILEVEL IILEVEL IIILEVEL IVLEVEL V High quality randomized trials Lesser quality RCTCase control studyCase seriesExpert opinion High quality prospective studies Prospective or comparative study Retrospective comparative study Case control study (diagnostic studies) Testing previously developed diagnostic criteria on consecutive patients Retrospective study, lesser quality prospective study Study of non- consecutive patients inconsistently applied reference gold standard Poor reference standard Sensible costs and alternatives Untreated controls from an RCT Analyses with no sensitivity analyses. Values from many studies with multiway sensitivity analyses Development of diagnostic criteria on consecutive patients, Analyses based on limited alternatives, costs & poor estimates Systematic review of Level I studies Systematic review of L2 studies or L1 studies with inconsistent results. Systematic review of L3 studies. Source: ElsevierElsevier

29 DESIGN: ALLOCATION Randomized | Non-Randomized | Single-Arm Trials ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

30 Clinical trials by Allocation Number of trialsEnrolled patients Allocation: A clinical trial design strategy used to assign participants to an arm of a study. Randomized Controlled Trial: Participants are assigned to intervention groups by chance Nonrandomized Trial: Participants are expressly assigned to intervention groups through a non-random method, such as physician choice Number of Enrolled Patients: Results skewed by three studies with unlimited number of participants (99,999,999) Non-randomized Randomized Single arm study

31 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Allocation Breakdown by phase (number of studies) Non-randomized Randomized Single arm study

32 Clinical trials by Allocation Trends by Start Date & Completion Date Non-randomized Randomized Single arm study ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

33 DESIGN: INTERVENTION MODEL Parallel | Cross-Over | Factorial | Single-Group ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

34 Clinical trials by Intervention Model Parallel Study Design: Two or more groups of participants receive different interventions "in parallel“. Cross-over Study Design: Groups of participants receive two or more interventions in a particular order. So during the trial, participants "cross over" from one drug to the other. Single Group Study Design: All participants receive the same intervention. Factorial Study Design: Groups of participants receive one of several combinations of interventions so during the trial, all possible combinations of the two drugs are given to different groups of participants. Number of trialsEnrolled patients Crossover Factorial Parallel Single Group Blank

35 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Intervention Model Breakdown by phase (number of studies) Crossover Factorial Parallel Single Group Blank

36 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Intervention Model Breakdown by phase (Enrollment) Crossover Factorial Parallel Single Group Blank Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

37 Clinical trials by Intervention Model Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Crossover Factorial Parallel Single Group Blank

38 DESIGN: MASKING Double-Blind | Single-Blind | Open Label ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

39 Clinical trials by Masking Open Label: Describes a clinical trial in which masking is not used, and all parties involved in the trial know which participants have been assigned which interventions. Single Blind: A type of Masking in which one party involved in the clinical trial, typically the investigator or participants, does not know which participants have been assigned which interventions. Double Blind: A type of Masking in which two or more parties do not know which participants have been assigned which interventions. Typically, the parties include the investigator and participants, but sometimes also the assessor or caregiver.. Double Blind Open Label Single Blind Blank Number of trialsEnrolled patients

40 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Masking Breakdown by phase (Number of studies) Double Blind Open Label Single Blind Blank

41 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Masking Breakdown by phase (Enrollment) Double Blind Open Label Single Blind Blank Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

42 Clinical trials by Masking Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Double Blind Open Label Single Blind Blank

43 RECRUITMENT Recruiting | Enrolling by Invitation |Active, not Recruiting Completed | Suspended | Terminated | Withdrawn ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

44 Clinical trials by Recruitment Number of trials Enrolled patients Active, not recruiting Completed Enrolling by invitation Not yet recruiting Recruiting Suspended Terminated Unknown status Withdrawn

45 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Recruitment Breakdown by phase (Number of Studies) Active, not recruiting Completed Enrolling by invitation Not yet recruiting Recruiting Suspended Terminated Unknown status Withdrawn

46 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Recruitment Breakdown by phase (Enrollment) Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999) Active, not recruiting Completed Enrolling by invitation Not yet recruiting Recruiting Suspended Terminated Unknown status Withdrawn

47 Clinical trials by Recruitment Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

48 DEMOGRAPHICS: GENDER Male | Female | Both ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

49 Clinical trials by Gender Number of trials Enrolled patients Both Female Male Blank

50 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Gender Breakdown by phase (Number of Studies) Both Female Male Blank

51 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Gender Breakdown by phase (Enrollment) Both Female Male Blank Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

52 Clinical trials by Gender Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Both Female Male Blank

53 STUDY RESULTS Availability of study results for completed studies Reporting format ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

54 Clinical trials by Results Has results No results available Number of trials Enrolled patients

55 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Results Breakdown by phase (Number of All Studies) Has results No results available

56 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Clinical trials by Results Breakdown by phase (Enrollment) Has results No results available Number of Enrolled Patients: Results skewed by several studies with very high number of subjects including three studies with unlimited number of participants (99,999,999)

57 Clinical trials by Results Trends by Start Date & Completion Date ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/ Has results No results available

58 ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

59 Future Considerations Published and registered trials have to be reconcilable (publication bias) Standard format of reporting for completed trials to allow accurate assessment Enforceable publication of results of clinical trials including negative studies Assessment of level of evidence each trial provides ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/

60 ARETE-ZOE, as a consultancy, provides solutions to complex problems in the high stakes and high consequence environment of Global Pharmaceuticals, including clinical research, healthcare informatics, and public health. We blend established, Pharma sector methodologies, innovation, and adaptations/transfers from other sectors to identify and resolve consequential practices that pose risk and often result in avoidable patient casualty. However, we are specifically, not a patient advocacy group but believe in optimizing organizational effectiveness and that smart business is agile, competitive and profitable, while intrinsically safe, secure, and resilient. We work within a global context because transnational interests influence national circumstances and choices at point of prescription. ARETE-ZOE, provides full spectrum organizational and operational risk management consultancy. Our published materials provide a glimpse of some aspects of our services to demonstrate both knowledge and ongoing participation within the Pharmaceutical Industry. Our analysis and consultancy includes all channels of misuse, diversion, counterfeiting and illicit exploitation of pharmaceuticals, medical devices, and precursor chemicals. Our advisement is to manufactures, jurisdictional entities, insurers, legislators, litigators, patients, and health care providers. ​ This scope also frequently segues into the nexus of crime and terrorism as significant influencers that undermine sector integrity differentiated from other criminal activity. Obviously, vulnerability assessment, information collection management and intelligence production supporting decision-making for risk reduction and interventions are routinely within the scope of our services as well as design and implementation of operational control measures. ARETE-ZOE, LLC: 1334 E Chandler Blvd 5A-19, 85048 Phoenix, AZ, USA | T:+1-480-409-0778 (24/7) | website: http://www.aretezoe.com/