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Introduction to clinical trials

Introduction to clinical trials. Definition of a clinical trial. A research activity that involves administration of a test treatment to some experimental unit in order to evaluate the treatment. Key words. Treament. Pharmaceutical, diet, procedure, diagnostic, device, program, placebo.

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Introduction to clinical trials

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  1. Introduction to clinical trials Stefan Franzén

  2. Definition of a clinical trial A research activity that involves administration of a test treatment to some experimental unit in order to evaluate the treatment.

  3. Key words Treament Pharmaceutical, diet, procedure, diagnostic, device, program, placebo. Experimental unit Subject from a target population. Evaluate Assessment of (clinical) effect, but also adverse events, lab variables, vital signs, quality of life, health economy.

  4. The first study I have found two treats that makes a letcure seem more interesting. How do I find out which one is best?

  5. The Wheel of Science Experiment Research question Data Conlusion

  6. Drug discovery/development process discovery; refinement; chemical & biological characterisation safety & toxicity in animals; formulation development volunteer studies; patient studies regulatory process marketing Lessons & development post registration monitoring Discovery=find new active structure : Development=convert it to a useful drug

  7. The path to a new medicine 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 Years First patent application Clinical trial application Product licenceapplication Drug Discovery Drug Development Target and leadidentification Leadoptimisation Developmentfor launch Product lifecycle support Concept testing Launch Clinical Development Phase I12-150people Phase II50-1000people Phase III500-5,000people Phase IV studies continue Toxicology and pharmacokinetic studies (absorption, distribution, metabolism, excretion) Pharmaceutical and analytical development Process chemistry and manufacturing Registration and regulatory affairs Sales and marketing (preparation, promotion, advertising and selling) No. of compounds Up to 10,000 10-15 1-8 1-3 1

  8. Phase I The drug seems reasonably safe in animal study, but has never been tested on humans What do we want to know? How can we find that out?

  9. Phase I trials Phase I50-150people Phase III500-15000people Phase II100-200people Phase IV studies continue • Focused on tolerability and safety • 12-30 (150) healthy people (often males) • Efficacy on biomarkers if possible • Single and repeated doses • Increase dose levels • Interaction with other drugs • Pharmacokinetics • Explorative’ • ADME (Absorption, Distribution, Metabolism Excretion) • Through QT study • Bridging , PK in other populations

  10. Phase II The drug seems to be resonably safe in humans and there is some sign of an effect on something. What do we want to know next? How can we find that out?

  11. Phase 2 trials Phase II50-1000people Phase I50-150people Phase III500-5,000people Phase IV studies continue • 50-1000 patients • Extensive monitoring • Safety and tolerability in patients • Often complicated design, explorative • Selection of optimal dose • Pharmacokinetics in patients • Effect in special populations • Explorative

  12. Phase III The drug seems resonable safe to give to patients and we have and idea of which dose to use. What do we want to know now? How can we find that out?

  13. Phase 3 trials Phase III500-15000people Phase I50-150people Phase II100-200people Phase IV studies continue • 500-20000 patients • Effect is verified in the target population • Forms the basis of the NDA, New Drug Application • Interactions between drugs start to become measurable in the larger population • sub-groups start to be established • special features and problems show up • Confirmative

  14. Phase IV Our drug is approved for use on patients. What’s next?

  15. Phase IV trials Phase I50-150people Phase II100-200people Phase III500-5,000people Phase IV studies continue • Often large 500-30000 patients • Further investigation of efficacy and safety post approval • Special populations • New indications • Marketing

  16. data collection analysis interpretation time now Observational studies Data is collected for a set of patients without any randomisation Prospective: Data is collected after the objectives are set data collection analysis interpretation time now Retrospective: Data is collected before the objectives are set

  17. Where to look for information ICH (international Conference on Harmonisation) http://www.ich.org/products/guidelines.html FDA (Food and Drug Agency) http://www.fda.gov/ EMEA (European Medicines Agency) http://www.emea.europa.eu/ Cochrane Collaboration http://www.cochrane.org/index.htm

  18. ICH • Quality • Efficacy • Safety • Multidiciplinary

  19. ICH Quality guidelines Harmonisation achievements in the Quality area include pivotal milestones such as the conduct of stability studies, defining relevant thresholds for impurities testing and a more flexible approach to pharmaceutical quality based on Good Manufacturing Practice (GMP) risk management.

  20. ICH safety guidelines ICH has produced a comprehensive set of safety Guidelines to uncover potential risks like carcinogenicity, genotoxicity and reprotoxicity. A recent breakthrough has been a non-clinical testing strategy for assessing the QT interval prolongation liability: the single most important cause of drug withdrawals in recent years. • S7A: Safety Pharmacology Studies for Human Pharmaceutical • S7B: The Non-Clinical Evaluation of the Potential for Delayed Ventricular Repolarization (QT prolongation) by Human Pharmaceutical

  21. ICH efficacy guidelines The work carried out by ICH under the Efficacy heading is concerned with the design, conduct, safety and reporting of clinical trials. It also covers novel types of medicines derived from biotechnological processes and the use of pharmacogenetics/genomics techniques to produce better targeted medicines.

  22. ICH Efficacy Guidelines • Clinical Safety E1-E2F • Clinical Study Reports E3 • Dose Reponse Studies E4 • Ethnic Factors E5 • Good Clinical Practise E6 • Clinical Trials E7-E11 • Clincal Evaluation of a Therapeutic Category E12 • Clincal Evaluation E14 • Pharmacogenomics E15-E16 • Cross Cutting Topics E17

  23. ICH Efficacy Guidelines • E7: Studies in Support of Special Populations, Geriatrics • E8: General Considerations for Clinical Trials • E9: Statistical Principles for Clincal Trials • E10: Choice of Control Group and Related Issues • E11: Clinical Investigation of Medicinal Products in the Pediatric Population

  24. The Clinical Study Process Preparation of statisticalanalysis StatisticalAnalysis Outline Clinical StudyProtocol StudyConduct Clinical Study Report StudySetup Data Capture Publications StatisticalAnalysis Plan Clean File Data base lock T i m e

  25. Chapter 1 Reading instructions • 1.1 What are clinical trials: Read • 1.2 History of clinical trials: Less important • 1.3 Regulatory process and requirements: Read page 14 • 1.4 Investigational new drug application: Read page 17–20 • 1.5 New drug application: Less important • 1.6 Clinical development plan and practise: Read • 1.7 Aims and structure of this book: Skip

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