Is Consent to Research Necessary in Comparative Effectiveness Trials? Collin O’Neil, PhD Department of Philosophy Lehman College, City University of New York

1. Is Consent to Research
Necessary in Comparative
Effectiveness Trials?
Collin O’Neil, PhD
Department of Philosophy
Lehman College, City University of New York
MEDICRES CONGRESS 2014

2. Types of Randomized Controlled
Trials
• Placebo-controlled
– Investigational Therapy versus Placebo
• Active-controlled
– Investigational Therapy versus Proven Therapy
– Proven Therapy versus Proven Therapy

3. Comparative Effectiveness Trials
(CETs)
• Why are they important?
– Improve clinical decision-making
– Lower health care costs
– Incentivize pharma to make more socially
productive R&D investments

4. Recent Argument
• Comparative effectiveness trials count as human
subjects research
• But Robert Truog, Ruth Faden, Nancy Kass, and others
have argued that some of these trials are so close to
standard clinical care that:
– No need for IRB review
– No need for written consent
– No need to inform patients they are involved in research

5. Consent
• We have a general right to bodily integrity
– A right against bodily interventions
• The function of consent is to waive rights
• But consent is not always successful or
“valid.”

6. Consent
• To be successful (or “valid”), consent must be
adequately informed
– Consent is adequately informed only if the
consent is not based on ignorance or mistake
• I.e, when it is not the case that the subject would not
have consented had they been better informed

7. Consent
• Can consent to the interventions in
comparative effectiveness trials be
adequately informed when the subject is
ignorant of the fact there is a research
purpose behind them?

8. Clinical Care Expectations
• Non-maleficence: no risks or burdens except
those necessary to prevent greater harm to
the patient
• Beneficence: the patient will be offered
whatever available treatment is, in the
physician’s judgment, best for the patient

9. CETs may disappoint these
expectations
• Non-maleficence
– Some CETs involve non-therapeutic interventions
• E.g., blood draws for purely research purposes
• Beneficence
– Some CETs randomize between X and Y even when in
the physician’s judgment, X or Y would be better for
the patient

10. Problem
• If the patient does not know they are involved
in research, they will assume these clinical
care expectations are satisfied
– When this assumption is mistaken, their consent
will often be based on this mistake, and so invalid

11. But not all CETs disappoint these
expectations
• Consider a trial that:
– Involves no non-therapeutic interventions
– Randomizes patients only with physician assent

12. • Will their consent to the interventions be valid,
even though they have not been informed they
are involved in research rather than standard
clinical care?
– The care they are receiving in the trial is in no respect
inferior to the care they would receive in standard
clinical care

13. • But the research purpose itself might matter
to them, even if it does not compromise the
care they receive
– May have conscientious objections to serving
certain research goals
– May be suspicious of research

14. Best Candidate
• No non-therapeutic interventions
• Randomized only with physician assent
• Uncontroversial research goal
• Patient population unlikely to be mistrustful
of research

15. • In this trial, although the patients are unaware
of the research purpose, their consent is not
based on this ignorance
• Is that enough to secure the validity of their
consent to the interventions?

16. • In order to successfully waive a certain right,
one needs to waive it intentionally
• So one needs to know which right or right one
needs to waive

17. • There is a general right of bodily integrity, a right
against bodily interventions
• But there are two distinct types of rights of bodily
integrity
– A right against being intervened upon for one’s own
benefit
– A right against being intervened upon for the benefit of
others

18. • To successfully waive the right against being
intervened upon for the benefit of others, one
must know this is the right one needs to waive
• And one can know this is the right one needs
to waive only if one is informed of the
research purpose

19. • Respecting the rights of subjects in CETs always
requires that the research purpose be disclosed
• But disclosure is not required for all kinds of research
– Research that merely collects data on outcomes of clinical
care
– Research on samples or specimens originally extracted for
therapeutic purposes

20. • Why the reluctance to disclose the research
purpose?
– Not impossible
– Not even difficult

21. • The main worries (I suspect) are that, if the
research purpose is disclosed, there will be
insufficient or imbalanced enrollment
– These are not legitimate reasons for not disclosing
the research purpose
– But there may be other ways to avoid enrollment
problems

22. • The requirement to obtain research consent requires
only that the patient not be randomized if they refuse
– It is commonly assumed that, if they refuse
randomization, they must be offered the treatment of
their choice
– But this requirement says nothing about what they should
be offered if they refuse

23. Proposal
• Make it a condition on their receiving any
treatment at all at the hospital that they
accept randomization
• Potential objection: coercive?

24. • Consent is coerced only if obtained by
threatening to otherwise withhold something
they have a prior right to
– NIH Clinical Center
– Private Hospitals

25. Conclusion
• However, at public hospitals, like the VA,
conditioning treatment on their acceptance of
randomization may be coercive
– Their patients do have a prior right to treatment
from the VA
• Patient education is the key here