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What’s in a gold standard? In defence of randomised controlled trials

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Abstract

The standardised randomised clinical trial (RCT) has been exceedingly popular in medical research, economics, and practical policy making. Recently, RCTs have faced criticism. First, it has been argued by John Worrall that we cannot be certain that our sample is not atypical with regard to possible confounding factors. I will argue that at least in the case of medical research, we know enough about the relevant causal mechanisms to be justified to ignore a number of factors we have good reason not to expect to be disruptive. I will also argue against an argument provided by Nancy Cartwright and Eileen Munro that RCTs should not be taken to deductively infer probabilistic causal claims, but ampliatively. The paper will end on a discussion of evidence hierarchies and a defence of the stance of evidence-based medicine that RCTs are the best available method to assess a treatment’s efficacy.

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Notes

  1. Since RCTs are widely used even outside medical research, I will, let the “C” stand for “controlled” rather than “clinical”.

  2. See e.g. OCEBM Levels of Evidence Working Group (2011).

  3. See e.g. Angrist and Pischke (2010).

  4. For a refutation of that claim, see Jerkert (2013).

  5. See e.g. Stegenga (2014).

  6. See e.g. Worrall (2002, 2007a, b), Papineau (1994), La Caze et al. (2012), and Howick (2011).

  7. See e.g. Worrall (2002) and Reiss (2015).

  8. A point that Worrall borrows from Lindley (1982).

  9. See e.g. OCEBM Levels of Evidence Working Group (2011).

  10. If a mysterious mode of action contradicting current physics and chemistry wasn’t enough. Homeopathy is so well tested and well refuted in RCTs that it even has been argued that is unethical to continue to waste resources on testing it any further (Smith 2012, pp. 407–408)

  11. For the biochemical properties of COX-2, see O’Banion (1999)

  12. See e.g. Stegenga (2014) and Reiss (2015).

  13. I eliminated Cartwright’s problematic use of \(K_i\) as subpopulation rather than a state description that is used to pick out a subpopulation.

  14. We will ignore that macro-dispositions such as “the power to alleviate headache” are far from unproblematic [See e.g. Bird (2016)].

  15. As does Adam La Caze in fact, but without the emphasis of it being a purely deductive method. See La Caze (2011, 91–92).

  16. See e.g. Williams (2010, 116–118), and Stegenga (2014, p. 319).

  17. If that were ethically sound, which it would not be in the case of a study where we would have to deliberately increase the risk of cardiovascular problems. We will turn to this particular problem, i.e. that t is not always feasible to conduct an RCT later.

  18. See e.g. Evidence Based Medicine Working Group (1992, p. 2421).

  19. For an overview, see Howick and Mebius (2015, pp. 2–3).

  20. For a discussion of how other types of evidence sometimes can trump the results of RCTs in scenarios where there is a risk of harm, see Osimani (2013).

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Acknowledgements

I would like to thank the members of Thomas Müller's working group in Konstanz and Utrecht, Johanna Thoma, a number of anonymous referees, and the participants of a number of conferences where I presented this paper for their helpful comments. I am also very grateful to the Deutsche Forschungsgemeinschaft (DFG) for their funding within the project “The Metaphysics of Induction”.

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  1. Konstanz University, Constance, Germany

    Marius Backmann

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Backmann, M. What’s in a gold standard? In defence of randomised controlled trials. Med Health Care and Philos 20, 513–523 (2017). https://doi.org/10.1007/s11019-017-9773-2

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