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n underpowered trial is not a pilot study. Let’s just get that were available in the literature8; how-
out there to begin. But a pilot study is an underpowered trial. ever, recent years have seen some useful
Feasibility and pilot studies are preliminary studies conducted guidance become available.2,13 The TABLE
provides a summary of the objectives,
specifically for the purposes of establishing whether or not a
outcomes, and interpretation of feasibili-
full trial will be feasible to conduct, and is between such preliminary studies and ty and pilot studies. Again, the distinction
J Orthop Sports Phys Ther 2014.44:555-558.
that all the necessary components of a a proper RCT (TABLE). between them is not critical, as in prac-
trial will work properly together. A ran- Unfortunately, however, feasibility tice, often only one or the other is con-
domized clinical trial (RCT) is conducted and pilot studies are very often misused ducted, they are combined, or only the
to compare 2 (or more) treatments, or a or misrepresented.8 The most common latter gets published.11,13 However, there
treatment to a control or comparison transgressions are the characterization of is a strong case, particularly in complex
group. Preliminary studies should not a too-small RCT as a pilot study, and/or interventions and multicenter trials, for
attempt this, as they will almost always reporting treatment effectiveness results far greater attention to preliminary stud-
be woefully underpowered, and thus are from a pilot or feasibility study in the ies than is currently the case.6
very likely to give misleading answers. same way that those from RCTs are re- The purpose of a feasibility study is to
Feasibility and pilot studies play an ported.2,11 Although it is not an appropri- assess whether or not it will be feasible
important role in the preliminary plan- ate purpose of either a pilot or feasibility to conduct an RCT of a particular in-
ning of a proposed full-size RCT. In es- study to compare the efficacy of an inter- tervention in a particular setting. Some
sence, feasibility studies are used to help vention with a comparator,2,11 more than of the key feasibility issues that must be
develop trial interventions or outcome 80% of all pilot studies published from considered (TABLE) include the willing-
measures, whereas pilot studies replicate, 2007 to 2008 reported hypothesis testing ness of clinicians to recruit participants,
in miniature, a planned full-size RCT.2,4 of treatment effect using inferential sta- the willingness of participants to be
However, the terms used for these pre- tistics,2 and more than 80% of nondrug randomized to the proposed treatment
liminary studies are sometimes consid- pilot or feasibility trial articles published groups, whether the intervention can be
ered synonymous,13 and in practice may from 2000 to 2009 made statements delivered as intended within the health
overlap considerably or be combined.11,13 about efficacy in their abstract.11 care system and clinical setting, the ad-
The important issue is not the distinction Until 2004, no formal methodologi- herence/compliance of participants to
between these: the important distinction cal guidelines for preliminary studies the proposed treatments, response rates
journal of orthopaedic & sports physical therapy | volume 44 | number 8 | august 2014 | 555
• x % of eligible participants are willing to be randomized • x % of potential participants meet inclusion criteria
• Identified barriers to participation are... • x % of eligible participants are willing to be randomized
• Problems encountered with assessment procedures • x % of consenting participants complete all baseline
were... assessments within (benchmark) days
• Etc... • x % of consenting participants commence their ran-
domly allocated treatment within (benchmark) days
• x % of consenting participants complete their randomly
allocated treatment with a minimum of (benchmark)
adherence
• x % of participants complete all follow-up assessments
within (benchmark) days of target date
• Etc...
Interpretation: Interpretation: Interpretation:
(a) A full study is not feasible: stop (a) Insurmountable problems with necessary processes: In the case of a superiority trial:
(b) Problems identified are surmountable: a full stop (a) Treatment A is probably more effective than the
study may be feasible with modifications (b) Problems identified are surmountable: modifications comparison
(c) Few or no major problems, but some unknowns: will be necessary in full study protocol, and further (b) Treatment A is probably not more effective than the
a pilot study without modifications that aims to pilot testing may be necessary comparison
complete the information needed to plan a full (c) Few or no major problems: a full study can proceed
study can proceed without modifications or further testing
(d) Few or no major problems: a full study can NOT: treatment A is effective/not effective
proceed without modifications or further testing,
provided all study processes are known to be
fail safe
Abbreviations: α, the prespecified probability of making the type I error of concluding that there is a difference when in truth there is not; MID, minimal impor-
tant difference.
556 | august 2014 | volume 44 | number 8 | journal of orthopaedic & sports physical therapy
In both feasibility and pilot studies, in the first instance, and the data from fectiveness of an intervention (or inter-
the objectives and specific aims should the pilot study should then be used to ventions) compared with a comparison
be restricted to those practicality issues confirm whether these estimates hold up group; the aims of preliminary studies
(TABLE). Criteria for success should be in the population and setting intended are to assess whether and how a full RCT
specified a priori. Ribeiro et al10 provide for the full trial. Considerable cau- can be done (TABLE).
an excellent example of a preliminary tion is advised in the use of pilot study The results of preliminary studies
study addressing feasibility issues. They outcomes data to generate sample size should, therefore, be mostly descriptive.8
tried 2 variations of a novel, untried in- calculations, as the estimates may be They should focus on important practi-
tervention; multisite recruitment; man- biased due to factors that may not be calities such as recruitment, ability to
agement of the time and equipment reproduced in a full trial, or may be un- deliver the treatment properly in the in-
J Orthop Sports Phys Ther 2014.44:555-558.
necessary to run the intervention at realistic due to chance factors related to tended setting, treatment acceptability,
multiple sites; and participant accept- the small sample size.9,13 treatment fidelity, adherence, partici-
ability, adherence, and retention. They If there is genuinely no indication of pant retention, and completeness and
established that their outcome measure whether or not a treatment might plausi- adequacy of outcomes data.10 They may
was able to detect change within and dif- bly be effective, then some form of proof- include within-group treatment effects,
ference between groups, and that at least of-concept is necessary. In drug trials, such as effect size, but these should not
1 of their treatment protocols showed an this takes the form of phase 1 or phase be the basis of sample-size calculations
effect size promising enough to indicate 2 trials, conducted to provide some evi- unless the MID for the outcome measure
that it would be worthwhile developing dence of treatment effects or efficacy. is genuinely not known.9,10 Statistical
and testing that intervention further.10 These are often not randomized, and of- comparisons between groups are likely
There is some debate in the literature ten use a surrogate marker of physiologi- to be misleading due to the many poten-
regarding whether or not it is appropri- cal effect rather than a patient-reported tial problems arising from small samples
ate to assess for treatment effect in a outcome.13 In nondrug studies, such as and low power. Any results from statisti-
feasibility or pilot study, or to estimate physical therapy interventions, it might cal comparisons between groups should
the size and variability of differences take the form of a case series or a small be treated with great caution, and re-
between groups.2,8,11 The estimation of trial with a short-term, physiological ported only with explicit reference to
sample size is generally considered a le- outcome, such as within-group changes statistical power. Ideally, no statements
gitimate purpose of a pilot study7; how- in range of motion or strength,1 rather should be made regarding treatment ef-
ever, this should only be the case when than a patient-reported outcome such ficacy, particularly in the abstract, and
there are no data available from previ- as a functional outcome questionnaire the primary interpretation made from
ous studies to indicate the likely base- or recurrence,5 or a surrogate outcome the trial should be with regard to the
line status of, and variability among, a like a risk score shortly after interven- conduct of a fully powered RCT. t
journal of orthopaedic & sports physical therapy | volume 44 | number 8 | august 2014 | 557
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558 | august 2014 | volume 44 | number 8 | journal of orthopaedic & sports physical therapy