1. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Introduction Clinical trials

2. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Introduction Clinical Trials are the best way to test whether a treatment is effective and better than other treatments (or no treatment). New treatments or drugs for diseases‘ prevention, early diagnosis or cure are examined in a safe and efficient way.

3. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Introduction Clinical trials are the fastest and safest way to find and evaluates new treatments.

4. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Introduction As a patient, you might be considering enrolling in a clinical trial or you might be offered to by your physician. If you are interested in the development of new treatments, you can also be proactive and increase your knowledge about clinical trials by yourself.

5. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Introduction This tutorial will teach you the most important facts about clinical trials. Finally we hope to answer any questions you might have about this topic.

6. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Introduction Let‘s agree on words! The process we are going to describe is more typical of new drugs development, but also applies to any other type of intervention, such as diagnostic or surgical procedure, lifestyle changes (diet, exercise, etc.) or comparison between new and existing therapies in terms of effectiveness and safety (with the opportune difference when discussing doses, placebo, etc which may not be pertinent). From now on we will use the word “treatment” for making things easier! Let‘s agree on words! The process we are going to describe is more typical of new drugs development, but also applies to any other type of intervention, such as diagnostic or surgical procedure, lifestyle changes (diet, exercise, etc.) or comparison between new and existing therapies in terms of effectiveness and safety (with the opportune difference when discussing doses, placebo, etc which may not be pertinent). From now on we will use the word “treatment” for making things easier!

7. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials?

8. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Clinical trials aims to improve existing treatments or to replace them with new and better ones. Before a new treatment is made available for patients, it must be tested in a clinical trial for safety and efficacy.

9. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Clinical trials are designed for learning more about the positive and negative effects of a treatment. Treatment‘s efficacy and safety, however, can be totally assessed only by long-term use on patients in the daily clinical practice.

10. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Before being tested on patients in a clinical trial, new treatments must be carefully evaluated in the laboratory. If labs results are promising, a clinical trial can be designed for studying efficacy and safety of the new treatment.

11. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Clinical trials are the only effective and safe way to test a treatment after it has been carefully evalutated by laboratory analyses. In fact, clinical trials aim to assess not only treatments‘ efficacy but also all the possible risks.

12. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? New treatments need to be statistically evaluated by clinical trials on very large group of similar patients (differences among patients affected by the same disease can quite big and could affect the results of the clinical trial). Results must be systematically reviewed and carefully analysed.

13. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Clinical trials conducted on large number of patients are essential for proving the safety and the efficacy of a new treatment. TrueFalse

14. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Clinical trials conducted on large number of patients are essential for proving the safety and the efficacy of a new treatment. TrueFalse Your choice is correct!

15. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? Clinical trials conducted on large number of patients are essential for proving the safety and the efficacy of a new treatment. TrueFalse Your choice is not correct!

16. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? New treatments that have been tested in a laboratory have no risks whatsoever when used in clinical trials. TrueFalse

17. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? If a new treatment has been previously tested in laboratory, there is no risk whatsoever when used in clinical trials. TrueFalse Correct! Laboratory tests help designing new treatments but cannot reveal risks for patients.

18. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What are clinical trials? If a new treatment has been previously tested in laboratory, there is no risks whatsoever when used in clinical trials. TrueFalse Wrong! New treatments can always have unknown and unpredictable risks.

19. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Why are clinical trials important?

20. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Why are clinical trials important? Clinical trials help developing new and better treatments for patients. Assessing safety and efficacy of a new treatment is a very rigorous and long process. For this reason, new treatments usually take a lot of time before becoming available for the public.

21. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Why are clinical trials important? Clinical trials aim to enhance new or already existing treatment strategies or to test new therapeutic/diagnostic methods.

22. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Why are clinical trials important? Clinical trials are important because they investigate and compare new and already existing treatments, therefore enhancing the medical progress.

23. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Are you considering enrolling in a clinical trial?

24. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in Clinical Trials? Patients enroll in clinical trials for several reasons. Many people hope to benefit from having access to a new treatment.

25. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? In order to avoid disappointment you should remember that new treatments are not automatically better than standard ones in terms of efficacy and safety (and that‘s why a clinical trial is needed!).

26. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Patients should be selected for a clinical trial if they are likely to benefit the the most from the new treatment. Clinical studies give them the opportunity to get new and experimental treatments not yet available on the market.This is especially true for diseases with no- known cure.

27. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Some other participants wish to contribute to research in medicine and to help other patients by voluntarily participating to a clinical trial.

28. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Clinical trials‘ results always represent an improvement of the standard available treatment. TrueFalse

29. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Clinical trials‘ results always represent an improvement of the standard available treatment. TrueFalse No! Even if many clinical trials result in an improvement there is no guarantee for success. A new treatment could be as good as or even worse the standard treatment.

30. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Clinical trials‘ results always represent an improvement of the standard available treatment. TrueFalse Yes! Most but NOT all of the treatments have turned out to be better than the standard treatment.

31. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Researchers conducting a clinical trial hope that their new treatment is better than the standard one already available. TrueFalse

32. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Researchers conducting a clinical trial hope that their new treatment is better than the standard one already available. TrueFalse Your choice is not correct! Reseachers always conduct a clinical trial to improve a treatment

33. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Enrolling in a clinical trial? Researchers conducting a clinical trial hope that their new treatment is better than the standard one already available. TrueFalse Correct! Although disappointment is possible, researchers always intend to achieve an improvement

34. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Change on the right!

35. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Every clinical trial is designed to answer a series of scientific questions about the tested treatment. The quality of a clinical trial and its results heavily depend on a well chosen clinical trial design.

36. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process When conducting a clinical trial, researchers must always follow a written study plan also called study protocol. The protocol both ensures patients‘ safety and is essential for achieving high quality and reliable results.

37. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Some clinical trials are conducted on one group of patients only, whilst others compare two groups. Patients of different groups must be as much as possible similar e.g. they must have the same medical conditions and the same stage of the disease.

38. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Patients are assigned to one group or another (therefore receiving the new treatment or the standard one) by a method called randomisation, i.e. the choice is made by chance through a random generator. This type of clinical trial is called randomized trial.

39. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process When the study is conducted on two groups of patients, the first group is called “controI group“ and often receive the standard available treatment, while the other one is called “active group“ and is given the new treatment. This type of clinical trial is called controlled trial.

40. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process The control group in a clinical trial is treated with the same care as the active group. If possible, neither the researchers nor the patients should know which group every participant has been assigned. This special type of randomised trial is called blinded or masked randomised trial and ensure equal treatments for both groups.

41. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process For many diseases there are no standard treatments available yet. In this case the control group receives a dummy treatment called placebo such as a sugar pill. Important For ethical reasons, placebo control groups are not allowed if a treatment already exists and patients can benefit from it. Therefore the best standard available treatment must be used.

42. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Clinical trials firstly consider patients‘ safety and well being when testing a new treatment. TrueFalse

43. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Clinical trials firstly consider patients‘ safety and well being when testing a new treatment. TrueFalse Correct!

44. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Clinical trials firstly consider patients‘ safety and well being when testing a new treatment. TrueFalse Incorrect! Clinical trials are firstly committed to patients‘ safety and only in second instance to scientific results.

45. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process The control group always receives the less effective treatment. TrueFalse

46. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process The control group always receives the less effective treatment. TrueFalse Incorrect! Before the clinical trial is completed, it is not possible to tell which treatment will be better, whether the standard therapy or the new treatment. Both treatments could be equally good for the patient.

47. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process The control group always receives the less effective treatment. TrueFalse Correct! When starting a clinical trail, researchers cannot know yet whether the standard therapy or the new treatment will be better. Both treatments could be equally good.

48. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process For ethical reasons, if during a clinical trial, one treatment clearly shows to be better than the other one, the study is stopped and all the participants are given the same treatment.

49. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary The clinical trial process Patients should only enroll in a clinical trial after having clearly understood the benefits they could get and risks they might take. It is therefore essential that patients are given clear and exhaustive information before they agree to participate.

50. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent

51. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent The informed consent gives you all the important information about any aspects of the clinical trial before deciding to enroll.

52. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent As a first step, every patient meets one of the clinical trial researcher who will explain in detail the possible benefits but also risks in one-to-one talks.

53. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent After the meeting, the researcher will hand you out detailed information about the clinical trial in written form, because you should carefully think about a participation. If you decide to enroll in, you must return the informed consent signed and personally dated. Of course you always have the right to refuse taking part in the clinical trial.

54. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent Informed consent ensures that nobody enrolls in a clinical trial without having received detailed information and having personally given the consent to take part. TrueFalse

55. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent Informed consent ensures that nobody enrolls in a clinical trial without having received detailed information and having personally given the consent to take part. TrueFalse Correct! The informed consent protects the patient‘s right for information and ensures the consent to the clinical trial.

56. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent Informed consent ensures that nobody enrolls in a clinical trial without having received detailed information and having personally given the consent to take part. TrueFalse Incorrect! The informed consent protects the patient‘s right for information and ensures the consent to the clinical trial.

57. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed Consent When you are participating in a clinical trial, you will be continuosly updated with all the relevant information which may influence your decision to remain in the trial. The informed consent does not obligate you to remain in the clinical trial by any means. You can leave the clinical trial at any time without giving any explanation!

58. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed Consent Once a participant has signed the informed consent form, it is not possible to leave the clinical trial anymore. True False

59. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent Once a participant has signed the informed consent form, it is not possible to leave the clinical trial anymore. TrueFalse Correct! Participation is voluntary and can be quit at any time.

60. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent Once a participant has signed the informed consent form, it is not possible to leave the clinical trial anymore. TrueFalse Incorrect! Despite a signed informed consent, it is possible to quit a clinical trial at any time.

61. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Informed consent You should ask any questions or express doubts you might have about the clinical trial to the study team. It is essential that you understand the key statements in the informed consent about the treatment, benefits and risks.

62. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary What protection do you have as a participant in a clinical trial?

63. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Patient safety Basic ethical principles and legal codes which rule the medical practice are also valid for clinical trials! In addition clinical trials are governed through further international guidelines and legislations to protect participants and to ensure their safety.

64. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Patient safety Every clinical trial is checked for patients safety and scientific contents by an ethics committee and the competent authority before it can start. Every good study undergoes strict quality controls by the competent research body.

65. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Patient safety Results from progressing clinical trials are published on scientific conferences, in journals and reported to the authorities. The names of the participants are subject to data privacy. It is not allowed to publish names of study participants.

66. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Patient Safety It is standard practice to keep secret the names of clinical trial participants. TrueFalse

67. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Patient safety It is standard practice to keep secret the names of clinical trial participants. TrueFalse Incorrect! The names of study participants cannot be revealed for privacy reasons.

68. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Patient safety It is standard practice to keep secret the names of clinical trial participants. TrueFalse Correct! It is standard in clinical trials that names of participants are subject of data privacy.

69. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Where can I find clinical trials? -Trials Register - Change on the right!

70. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Trials Register If you are looking for a particular clinical trial you can directly ask your doctor. In addition you can search the website of the National Health Service for relevant trials http://www.nhs.uk/Conditions/Clinical- trials/Pages/clinical-trial.aspx/ or the website of the ISRCTN http://www.controlled-trials.com/ukctr/

71. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Trials Register Registers contain public information about clinical trials such as venues, aims, designs and patients‘ selection criteria. The trials registers should also contain contact information for patients interested in participating.

72. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary

73. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary A clinical trial is a research study conducted in order to test new treatments on patients. Clinical trials have precise and well defined aims whose details are described by the study plan (protocol).

74. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Clinical Trials are the fastest and safest way to make new treatments, which are better than the standard ones, available to the public. Clinical studies must be approved by ethics committees and competent authorities which review the study plan and give their authorization.

75. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary Participating to clinical trials is always voluntary. You can quit a clinical trial at any time – despite having signed the informed consent. Your personal rights and data are protected.

76. Introduction Clinical trials Why clinical trials? Process of a clinical trial Informed consent Patients‘ interests Rights and protection Study Registers Summary We hope you liked this short tutorial and found it useful. Keep exploring the ECRAN website and learn even more about clinical trials! Thank you for your kind attention!