CASE STUDY TWO: A new diabetes drug

For each of the four selected issues from this case study, which VALUE, which PRINICPLE and which GUIDELINE(S) would help you to answer the question? To assist you, in addition to chapters 1 (Principles) 2.1 (Risk) and 2.2 (Consent), it is likely that chapters 3.3 (Interventions and therapies, including clinical and n on-clinical trials, and innovations) and 4.3 (People in dependant relationships) will be relevant.

You are encouraged to make some choices of your own in the free text box provided before clicking the VALUE, PRINCIPLES AND GUIDELINE(S) buttons after each question to take you to the suggested answers.

[Revise Case Study]


1.Is the trial being conducted in order to discover new knowledge or only to establish grounds for the registration and marketing of N17, the new drug?

  1. Which is the relevant VALUE?

Research merit and integrity are discussed first. Unless proposed research has merit, and the researchers who are to carry out the research have integrity, the involvement of human participants in the research cannot be ethically justifiable. (NS p. 11)

  1. Which is the relevant PRINCIPLE?

1.3 Research that is conducted with integrity is carried out by researchers with a commitment to:

  1. searching for knowledge and understanding;
  2. following recognised principles of research conduct;
  3. conducting research honestly; and
  4. disseminating and communicating results, whether favourable or unfavourable, in ways that permit scrutiny and contribute to public knowledge and understanding.

  1. Which is or are the relevant GUIDELINE(S)

3.3.3 Researchers should show that:

  1. the research is directed to answering a specific question or questions;
  2. there is a scientifically valid hypothesis being tested that offers a realistic possibility that the interventions being studied will be at least as beneficial overall as standard treatment, taking into account effectiveness, burdens, costs and risks;
  3. the size and profile of the sample to be recruited is adequate to answer the research question; and
  4. the research meets the relevant requirements of the CPMP/ICH Note for Guidance on Good Clinical Practice (CPMP/ICH-135/95), ISO 14155 Clinical Investigation of Medical Devices, and the TGA.

3.3.4 Researchers must inform the HREC of:

  1. any business, financial or other similar association between a researcher and the supplier of a drug or surgical or other device to be used in the trial;
  2. any other possible conflicts of interest; and
  3. any restrictions on publication.

3.3.5 In any clinical research, especially clinical trials, an HREC should be satisfied that:

  1. funding is sufficient to conduct and complete the trial as designed;
  2. any payment in money or kind, whether to institutions, researchers or participants, will not adversely influence the design, conduct, findings or publication of the research; and
  3. the facilities, expertise and experience available are sufficient for the trial to be conducted safely.

(NS. p. 35)

2. Will Dr A's patients feel coerced to agree to involvement because of their previous relationship with him as their doctor?
4. What information will be provided to inform their consent?

  1. Which is the relevant VALUE?

Among these values, respect is central. It involves recognising that each human being has value in himself or herself, and that this value must inform all interaction between people. Such respect includes recognising the value of human autonomy Ð the capacity to determine one's own life and make one's own decisions. But respect goes further than this. It also involves providing for the protection of those with diminished or no autonomy, as well as empowering them where possible and protecting and helping people wherever it would be wrong not to do so.

  1. Which is the relevant PRINCIPLE?

1.10 Respect for human beings is a recognition of their intrinsic value. In human research, this recognition includes abiding by the values of research merit and integrity, justice and beneficence. Respect also requires having due regard for the welfare, beliefs, perceptions, customs and cultural heritage, both individual and collective, of those involved in research.

1.11 Researchers and their institutions should respect the privacy, confidentiality and cultural sensitivities of the participants and, where relevant, of their communities. Any specific agreements made with the participants or the community should be fulfilled.

1.12 Respect for human beings involves giving due scope, throughout the research process, to the capacity of human beings to make their own decisions.

1.13 Where participants are unable to make their own decisions or have diminished capacity to do so, respect for them involves empowering them where possible and providing for their protection as necessary. (NS p. 13)

  1. Which is or are the relevant GUIDELINE(s)?


2.2.9 No person should be subject to coercion or pressure in deciding whether to participate. Even where there is no overt coercion or pressure, consent might reflect deference to the researcher's perceived position of power, or to someone else's wishes. Here as always, a person should be included as a participant only if his or her consent is voluntary.


2.2.2 Participation that is voluntary and based on sufficient information requires an adequate understanding of the purpose, methods, demands, risks and potential benefits of the research.

2.2.3 This information must be presented in ways suitable to each participant (see paragraph 5.2.16, page 84).

2.2.4 The process of communicating information to participants and seeking their consent should not be merely a matter of satisfying a formal requirement. The aim is mutual understanding between researchers and participants. This aim requires an opportunity for participants to ask questions and to discuss the information and their decision with others if they wish.

2.2.5 Consent may be expressed orally, in writing or by some other means (for example, return of a survey, or conduct implying consent), depending on:

  1. the nature, complexity and level of risk of the research; and
  2. the participant's personal and cultural circumstances.

2.2.6 Information on the following matters should also be communicated to participants. Except where the information in specific sub-paragraphs below is also deemed necessary for a person's voluntary decision to participate, it should be kept distinct from the information described in paragraphs 2.2.1 and 2.2.2:

  1. any alternatives to participation;
  2. how the research will be monitored;
  3. provision of services to participants adversely affected by the research;
  4. contact details of a person to receive complaints;
  5. contact details of the researchers;
  6. how privacy and confidentiality will be protected;
  7. the participant's right to withdraw from further participation at any stage, along with any implications of withdrawal, and whether it will be possible to withdraw data;
  8. the amounts and sources of funding for the research;
  9. financial or other relevant declarations of interests of researchers, sponsors or institutions;
  10. any payments to participants;
  11. the likelihood and form of dissemination of the research results, including publication;
  12. any expected benefits to the wider community;
  13. any other relevant information, including research-specific information required under other chapters of this National Statement. (NS pp.19-20)

3.3.13 Due to the potential complexity of information to be provided to participants, the requirements of paragraphs 2.2.2 to 2.2.6 (page 19) should be carefully considered and followed. Written information should not be unduly long or complex. Adequate time should be allowed for prospective participants to read and take in what is proposed, and they should be encouraged to ask questions.

3.3.14 Particular care should be taken in clinical trials to make it clear to participants whether there is intended to be any therapeutic benefit to them from the trial.

3.3.15 It should always be made clear to those who might be subject to a proposed intervention whether it is innovative and/or experimental. (NS. p.36)

9. What will happen if people receive only the placebo and their health suffers?

  1. Which is the relevant VALUE?

Researchers exercise beneficence in several ways: in assessing and taking account of the risks of harm and the potential benefits of research to participants and to the wider community; in being sensitive to the welfare and interests of people involved in their research; and in reflecting on the social and cultural implications of their work. (NS p.11)

  1. Which is the relevant PRINCIPLE?

1.6 The likely benefit of the research must justify any risks of harm or discomfort to participants. The likely benefit may be to the participants, to the wider community, or to both.

1.7 Researchers are responsible for:

  1. designing the research to minimise the risks of harm or discomfort to participants;
  2. clarifying for participants the potential benefits and risks of the research; and
  3. the welfare of the participants in the research context.

1.8 Where there are no likely benefits to participants, the risk to participants should be lower than would be ethically acceptable where there are such likely benefits.

1.9 Where the risks to participants are no longer justified by the potential benefits of the research, the research must be suspended to allow time to consider whether it should be discontinued or at least modified. This decision may require consultation between researchers, participants, the relevant ethical review body, and the institution. The review body must be notified promptly of such suspension, and of any decisions following it (see paragraphs 5.5.6 to 5.5.9, page 91Ð92). (NS p.13)

  1. Which is or are the relevant GUIDELINE(s)?

3.3.8 In clinical research, where patient care is combined with intent to contribute to knowledge, any risks of participation should be justified by potential benefits to which the participants attach significance.

3.3.9 The prospect of benefit from research participation should not be exaggerated, either to justify to an HREC a higher risk than that involved in the participantÕs current treatment or to persuade a participant to accept that higher risk.

3.3.10 The use of a placebo alone or the incorporation of a non-treatment control group:

  1. is ethically unacceptable in a controlled clinical trial where:
    1. other available treatment has already been clearly shown to be effective; and
    2. there is known risk of significant harm in the absence of treatment;
  2. may be considered if there is genuine uncertainty as to whether currently available treatments have a net clinical benefit.

    (NS pp. 35)

3.3.23 It may be unethical for a researcher to continue a trial if:

  1. there are or have been substantial deviations from the trial protocol;
  2. side-effects of unexpected type, severity, or frequency are encountered; or
  3. as the trial progresses, one of several treatments or procedures being compared appears to be so much better or worse than the other/s that the continuation of the trial would disadvantage some of the participants.

The clearer it becomes that one treatment is substantially better or worse than the others, the stronger the need to consider discontinuing the trial. (NS pp. 37-38)

11. Will participants have a right to receive the new drug after the trial if it proves to be better than current treatment?

  1. Which is the relevant VALUE?

At a profound level, justice involves a regard for the human sameness that each person shares with every other. Human beings have a deep need to be treated in accordance with such justice, which includes distributive justice and procedural justice. In the research context, distributive justice will be expressed in the fair distribution of the benefits and burdens of research, and procedural justice in Ôfair treatmentÕ in the recruitment of participants and the review of research. While benefit to humankind is an important result of research, it also matters that benefits of research are achieved through just means, are distributed fairly, and involve no unjust burdens. (NS p. 11)

  1. Which is the relevant PRINCIPLE?

1.4 In research that is just:

  1. taking into account the scope and objectives of the proposed research, the selection, exclusion and inclusion of categories of research participants is fair, and is accurately described in the results of the research;
  2. the process of recruiting participants is fair;
  3. there is no unfair burden of participation in research on particular groups;
  4. there is fair distribution of the benefits of participation in research;
  5. there is no exploitation of participants in the conduct of research; and
  6. there is fair access to the benefits of research.

1.5 Research outcomes should be made accessible to research participants in a way that is timely and clear. (NS p. 12)

  1. Which is or are the relevant GUIDELINE(s)?

3.3.18 An HREC should be satisfied that:

  1. . . . . . .
  2. . . . . ; and
  3. it has been made clear to participants whether they will have continued access after the trial to treatments they have received during the trial, and on what terms. (NS. p. 36)