This post is an addendum to a previous post “What is a control group?”, inspired by a recently published new paper (“Control conditions for randomized trials of behavioral interventions in psychiatry: a decision framework” Early View, Lancet Psychiatry, March 2017). Following a brief review of the literature on effect sizes associated with different types of control conditions, a framework for choosing an appropriate control condition in behavioral trials is offered. The types of control conditions discussed are as follows:
- Active comparator
- Minimal treatment control
- Nonspecific factors control
- No-treatment control
- Patient choice
- Pill placebo
- Specific factors component control
- Treatment as usual
- Waitlist control
The considerations for choosing one of these control conditions for testing a behavioral intervention are (1) participant risk; (2) trial phase; and (3) available resources. With respect to participant risk, more active interventions should be provided as the control condition when the risk of withholding treatment (especially when known effective treatments are available) is high. Therefore, when making this decision characteristics of the participant population and characteristics of the available treatments will play a role in the decision making process.
Regarding trial phase, early stage exploratory trials should be concerned with the risk of Type II error; in other words the researcher will want to maximize the chances of finding a benefit of a potentially helpful new intervention. Therefore, a waitlist control group might be appropriate at this stage of the research process given that waitlist controls are associated with large effect sizes in behavioral trials. In the later stages of the research program, the researcher should strive to minimize Type I error; in other words it is important to guard against concluding that an ineffective treatment is helpful. In this case an active comparator would be a logical choice although the sample size would need to be large given that the effect size is likely to be small in this case.
Finally, the resources available to the researchers will influence the choice of control condition. For example, in a late stage trial an active comparator provided by trained and monitored study personnel would be the best choice in most circumstances; however, in this case the provision of the control may be at least as expensive as the provision of the experimental treatment. When sufficient resources are lacking, the cost effective alternative might be to ask the usual community provider to administer treatment as usual although every effort should be made to describe the control intervention in detail.
A very nice graphic is provided (Figure 2) to illustrate the decision framework and can be applied to speech therapy trials. There are a number of interventions that have been in use or are emerging in speech therapy practice with a minimal evidence base. We can consider the choice of appropriate control condition for the assessment of these interventions.
Ultrasound intervention for school aged children with residual speech errors has been examined in quite a number of single subject studies but is now overdue for a randomized control trial. Given that the exploratory work has been completed in single subject trials I would say that we could proceed to a phase 3 RCT. The risk to the participant population is more difficult to conceptualize. You could say that it is low because these children are not at particular risk for poor school outcomes or other harmful sequels of non-intervention and the likelihood of a good speech outcome will not change much after the age of nine. The cost of providing an active control will be high because these children are often low priority for intervention in the school setting. Therefore, according to Figure 2, a no-treatment control would be appropriate when you make this assumption. On the other hand, you could argue that the participant risk of NOT improving is very high-all the evidence demonstrates that the residual errors do not improve without treatment after this age. If you consider the participant risk to be higher, especially considering community participation and psychosocial factors, then the appropriate control condition would be something more vigorous: patient choice, an active comparator, a nonspecific factors component control or a specific factors component control. Given the relatively early days of this research, small trials utilizing these control conditions in order might be advisable.
Metaphon as a treatment for four-year-olds with severe phonological delay and associated difficulties with phonological processing has not, to my knowledge, been tested with a large scale RCT. The population would be high risk by definition due to the likelihood of experiencing delays in the acquisition of literacy skills if the speech delay is not resolved prior to school entry. Effective treatment options are known to exist. Therefore, the appropriate control condition would be an active comparator-in other words, another treatment that is known to be effective with this population. Another option would be a specific factors component control that examines the efficacy of specific components of the Metaphon approach. Therefore, the meaningful minimal pairs procedure could be compared directly to the full metaphon approach with speech and phonological processing skills as the outcome variables. Similar trials have been conducted by Anne Hesketh and in my own lab (although not involving Metaphon specifically).
PROMPT has still not been tested in good quality single subject or parallel groups research. If a Phase 2 trial were planned for three-year-olds with suspected apraxia of speech, treatment as usual would be the appropriate control condition according to Figure 2. The speech condition is too severe to ethically withhold treatment and the research program is not advanced enough for a specific factors components control although this would be the next step.
Finally, an RCT of the effectiveness of Speech Buddies to stimulate /s/ in 3-year-olds with speech delay could be implemented. In this case, the participant group would low risk due to the likelihood of spontaneous resolution of the speech delay. Given a phase 2 trial, either no treatment or waitlist control could be implemented.
The authors of this framework conclude by recommending that researchers justify their choice of control condition in every trial protocol. They further recommend that waitlist controls are only acceptable when it is the only ethical choice and state that “no behavioral treatment should be included in treatment guidelines if it is only supported by trials using a waitlist control group or meta-analytic evidence driven by such trials.” To me, this is eminently sensible advice for speech and language research as well.
And this I believe concludes my trilogy of posts on the control group!
What is a control group? Developmental Phonological Disorders blog post, February 5, 2017
Using effect sizes to choose a speech therapy approach, Developmental Phonological Disorders blog post, January 31, 2017
Gold, S. M., Enck, P., Hasselmann, H., Friede, T., Hegerl, U., Mohr, D. C., & Otte, C. Control conditions for randomised trials of behavioural interventions in psychiatry: a decision framework. The Lancet Psychiatry. doi:10.1016/S2215-0366(17)30153-0
Hesketh, A., Dima, E., & Nelson, V. (2007). Teaching phoneme awareness to pre-literate children with speech disorder: a randomized controlled trial. International Journal of Language and Communication Disorders, 42(3), 251-271.
Rvachew, S., & Brosseau-Lapré, F. (2015). A Randomized Trial of 12-Week Interventions for the Treatment of Developmental Phonological Disorder in Francophone Children. American Journal of Speech-Language Pathology, 24(4), 637-658. doi:10.1044/2015_AJSLP-14-0056