Introduction to clinical trials Magnus Kjaer

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1 Introduction to clinical trials Magnus Kjaer

2 One Definition of Clinical Trial NIH 2014 A research study in which one or more human subjects are prospectively assigned to one or more interventions to evaluate the effects of those interventions on healthrelated biomedical or behavioral outcomes One of many definitions NIH: National Institutes of Health (US)

3 Key words Intervention Treatment Experimental unit Human subject Pharmaceutical, diet, procedure, diagnostic, device, program, placebo. 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 Wheel of Science Experiment Research question Data Conlusion

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

6 The path to a new medicine Years First patent application Clinical trial application Product licence application Drug Discovery Target and lead identification Lead optimisation Drug Development Development Concept testing for launch Launch Product life cycle support Clinical Development Phase II Phase I Phase III 500-5,000 Phase IV studies continue Toxicology and pharmacokinetic studies (absorption, distribution, metabolism, excretion) Pharmaceutical and analytical development No. of compounds Up to 10, Process chemistry and manufacturing Registration and regulatory affairs Sales and marketing (preparation, promotion, advertising and selling)

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

8 Phase I trials Phase I Phase II Phase III Phase IV studies continue Explorative Focus on: Tolerability Safety Pharmacokinetics (how the body affect the drug) Efficacy if possible (biomarkers) Interaction with other drugs Food interaction

9 Phase I trials Phase I Phase II Phase III Phase IV studies continue healthy (often males) Increase dose levels ascending doses Single dose Single ascending dose (SAD) study Repeated dose Multiple ascending dose (MAD) study ADME (Absorption, Distribution, Metabolism Excretion) QT studies Bridging studies, Pharmacokinetics in other populations

10 Phase II The drug seems to be reasonably 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 II trials Phase I Phase II Phase III 500-5,000 Phase IV studies continue Show some type of efficacy biomarker Selection of dose or doses optimal dose Safety and tolerability in patients Pharmacokinetics in patients

12 Phase II trials Phase I Phase II Phase III 500-5,000 Phase IV studies continue patients Longer studies 2 week to one year Often complicated design Extensive monitoring Effect in special populations or target population

13 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?

14 Phase III trials Phase I Phase II Phase III Phase IV studies continue Clinical relevant effect is verified Study population is equal to the target population Forms the basis of the NDA, New Drug Application Confirmative Lot of regulations

15 Phase III trials Phase I Phase II Phase III Phase IV studies continue Large trials patients Study duration 6 months to 3-5 years Sub-groups start to be established Special features and problem can be visable

16 Phase IV Our drug is approved for use on patients for a specific indication and target population. What s next?

17 Phase IV trials Phase I Phase II Phase III 500-5,000 Phase IV studies continue New populations New indications Marketing Often large patients Further investigation of efficacy and safety post approval

18 Planning of Clinical Programs Line of Sight Planning of phase I Understanding of what to do in phase II and III Planning of phase II Understanding of what to do in phase III

19 The Clinical Study Process Outline Clinical Study Protocol Study Setup Statistical Analysis Plan Preparation of statistical analysis Study Conduct Data Capture Statistical Analysis Clinical Study Report Publications Clean File T i m e Data base lock

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

21 Where to look for information ICH (international Conference on Harmonisation) FDA (Food and Drug Agency) EMEA (European Medicines Agency) Cochrane Collaboration

22 ICH Quality Efficacy Safety Multidiciplinary

23 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.

24 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

25 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.

26 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

27 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

28 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 Investigational new drug application: Read page 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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