The DOI matches its Crossref record; 16 of the 17 references it cites were found in a registry or an index; none of the 5 sources its claims cite is checked for support yet.
The role and interpretation of pilot studies in clinical research
Open accessAbstract
Pilot studies represent a fundamental phase of the research process. The purpose of conducting a pilot study is to examine the feasibility of an approach that is intended to be used in a larger scale study. The roles and limitations of pilot studies are described here using a clinical trial as an example. A pilot study can be used to evaluate the feasibility of recruitment, randomization, retention, assessment procedures, new methods, and implementation of the novel intervention. A pilot study is not a hypothesis testing study. Safety, efficacy and effectiveness are not evaluated in a pilot. Contrary to tradition, a pilot study does not provide a meaningful effect size estimate for planning subsequent studies due to the imprecision inherent in data from small samples. Feasibility results do not necessarily generalize beyond the inclusion and exclusion criteria of the pilot design. A pilot study is a requisite initial step in exploring a novel intervention or an innovative application of an intervention. Pilot results can inform feasibility and identify modifications needed in the design of a larger, ensuing hypothesis testing study. Investigators should be forthright in stating these objectives of a pilot study. Grant reviewers and other stakeholders should expect no more.
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Claims the paper makes
40 Own interpretation · 5 No basis given · 2 No full text of the sources · 1 Sources not found
None of the sources cited for this claim could be matched to a paper in Crossref, so there is nothing to check it against.
In the paper
“When event rates are reported and no adverse event is observed for a particular category, the rule of three should be applied to estimate the upper bound of the 95% CI, where the upper bound is approximately 3/n.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Tolerability and Preliminary Safety
What the sources say
- Not matchedcites Jovanovic & Levy, 1997
Couldn’t be matched to a paper in Crossref, so it can’t be checked.
- Not matchedcites Jovanovic et al., 1997
Couldn’t be matched to a paper in Crossref, so it can’t be checked.
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: unclear — It is unclear whether the paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
- Not matched
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Basing a decision to proceed or terminate evaluation of a particular intervention on pilot data is perilous because there is a very good chance that the decision will be derived from false positive or false negative results.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > DISCUSSION
boosteddirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Each aspect of feasibility could be quite different from an uncontrolled study when intervention assignment is randomized and blinded, particularly if placebo is a distinct possibility.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > WHAT A PILOT STUDY CAN DO: ASSESS FEASIBILITY > Rationale for a Control or Comparison Group in a Pilot
hedgedcorrelational- Asserted: passes — The authors put it forward as true.
- Checkable: unclear — It is unclear whether it could be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“With these issues in mind, a pilot study can better address its goals if a control group is part of the design.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > WHAT A PILOT STUDY CAN DO: ASSESS FEASIBILITY > Rationale for a Control or Comparison Group in a Pilot
plainconditional- Asserted: passes — The authors put it forward as true.
- Checkable: unclear — It is unclear whether it could be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“A case series estimate tends to be steeped in optimism, particularly if an estimate of such magnitude is seldom, if ever, seen in full scale trials for psychiatric disorders.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
hedgedcorrelational- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
The paper states this without a source or data behind it here, so there is nothing to check it against. The quote shows it is in the paper.
In the paper
“(It is the population effect size, not the sample effect size, that an RCT is designed to detect with sufficient power.)”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A claim is checked only against the cited paper’s full text, never its abstract, and none is available for its sources.
In the paper
“There has been a venerable tradition of using pilot studies to estimate between group effect sizes that, in turn, are used to inform the design of subsequent larger scale hypothesis testing studies. Despite it widespread use, it has been argued that the tradition is ill-founded. (Friedman, Furberg and DeMets, 1998; Kraemer et al., 2006)”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
What the sources say
hedged- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“A pilot sample size is instead based on the pragmatics of recruitment and the necessities for examining feasibility.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > SAMPLE SIZE DETERMINATION IN DESIGNING A PILOT STUDY
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“With no inferential statistical tests, a pilot study will not provide p-values.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > SAMPLE SIZE DETERMINATION IN DESIGNING A PILOT STUDY
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Pilot study data generally should not be combined with data from the subsequent larger scale study. This is because it is quite likely that the methods will be modified after the pilot, even if minimally. Such changes in protocol risk adding additional, perhaps unknown, source of variation.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > SAMPLE SIZE DETERMINATION IN DESIGNING A PILOT STUDY > Are Pilot Data Included in the Larger Trial?
hedgedconditional- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
- Rests on an assumption: Methods will likely be modified after a pilot study, even if minimally.
- It may state two separate claims as one. 13
- Probably not a substantial claim. 13
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Pilot results can inform feasibility and identify modifications needed in the design of a larger, ensuing hypothesis testing study.”
- Also stated in: § DISCUSSION · plain
“Pilot results inform feasibility, which in turn, is instructive in that it points to modifications needed in the planning and design of a larger efficacy trial.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > Abstract
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
- Also stated in: § DISCUSSION · plain
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“The definition of clinically meaningful is not entirely empirically-based, but instead requires input from clinicians who treat the patient population of interest and perhaps from patients with the disorder.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > DISCUSSION
hedged- Asserted: passes — The authors put it forward as true.
- Checkable: fails — It may be too vague to check against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. It may be too vague to check against evidence. The paper may give no reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“These good clinical practices include the refinement of source documentation, informed consent procedures, data collection tools, regulatory reporting procedures, and monitoring/oversight procedures, especially when multiple sites and investigators are engaged in the study.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > WHAT A PILOT STUDY CAN DO: ASSESS FEASIBILITY > Good Clinical Practices
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. The paper may give no reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Safety, efficacy and effectiveness are not evaluated in a pilot.”
- Also stated in: § DISCUSSION · plain
“a pilot study is not a hypothesis testing study and therefore safety and efficacy are not evaluated.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > Abstract
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
- Also stated in: § DISCUSSION · plain
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Pilot studies generate pilot data, their design need not be guided with the support of prior pilot data.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > SAMPLE SIZE DETERMINATION IN DESIGNING A PILOT STUDY > Pilot Data for a Pilot Study
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. The paper may give no reason why it holds. It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“It is possible, but highly unlikely, that the between group effect size (d) from a pilot study sample will provide a reasonable estimate of the population effect size (Δ), but that cannot be known based on the pilot data.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
hedged- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“A pilot study can be critical in research staff training and provide experiences that strengthen and confirm competencies and skills required for the investigation to be conducted with accuracy and precision.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > WHAT A PILOT STUDY CAN DO: ASSESS FEASIBILITY > Good Clinical Practices
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: fails — It may be too vague to check against evidence.
- Load-bearing: fails — The paper’s conclusions may not depend on it.
- Based: passes — The paper gives a reason why it holds.
Probably not a substantial claim. It may be too vague to check against evidence. The paper’s conclusions may not depend on it.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“An alternative approach is to base sample size estimates for a full scale RCT on what is deemed a clinically meaningful effect.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > An Alternative to Using Pilot Data for Sample Size Determination
plain- Asserted: passes — The authors put it forward as true.
- Checkable: fails — It may be too vague to check against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
Probably not a substantial claim. It may be too vague to check against evidence.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“A pilot study provides opportunity to develop consistent practices to enhance data integrity and the protection of human subjects.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > WHAT A PILOT STUDY CAN DO: ASSESS FEASIBILITY > Good Clinical Practices
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
Probably not a substantial claim.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“We learn little if anything about the efficacy of an intervention with data from a small sample; yet, as discussed earlier, a great deal can be learned from a pilot study.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Hypothetical Example
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“This applies to effects sizes for any type of outcome, be it a severity rating, a response status, or survival status.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
Probably not a substantial claim.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Hence, with imprecision comes a vast disparity in sample size estimates and, if sample size determination for a subsequent study is based on an imprecise estimate, there is an enormous risk of underpowered or overpowered design.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Hypothetical Example
boosteddirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“In summary, pilot studies are a necessary first step in exploring novel interventions and novel applications of interventions – whether in a new patient population or with a novel delivery system (e.g., transdermal patch).”
§ The Role and Interpretation of Pilot Studies in Clinical Research > DISCUSSION
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: fails — It may be too vague to check against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. It may be too vague to check against evidence. The paper may give no reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Although a pilot study will undoubtedly incorporate relevant outcome measures and can serve a vital role in treatment development, it is not, and should not, be considered a preliminary test of the intervention hypothesis.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
The paper states this without a source or data behind it here, so there is nothing to check it against. The quote shows it is in the paper.
In the paper
“The primary role of a pilot study is to examine the feasibility of a research endeavor.”
- Also stated in: § INTRODUCTION · plain
“The fundamental purpose of conducting a pilot study is to examine the feasibility of an approach that is intended to ultimately be used in a larger scale study.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > DISCUSSION
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
Probably not a substantial claim.
- Also stated in: § INTRODUCTION · plain
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“This does not preclude the need for a theoretical rationale for the intervention or the methodology being proposed for a pilot study.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > SAMPLE SIZE DETERMINATION IN DESIGNING A PILOT STUDY > Pilot Data for a Pilot Study
plain- Asserted: passes — The authors put it forward as true.
- Checkable: fails — It may be too vague to check against evidence.
- Load-bearing: fails — The paper’s conclusions may not depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. It may be too vague to check against evidence. The paper’s conclusions may not depend on it. The paper may give no reason why it holds.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“If a pilot study effect size is unduly large (i.e., a false positive result), subsequent trials will be designed with an inadequate number of participants to provide the statistical power needed to detect clinically meaningful effects and that would lead to negative trials.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“However, pilot studies provide an opportunity to implement and examine the feasibility of the adverse event reporting system.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Tolerability and Preliminary Safety
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: fails — The paper’s conclusions may not depend on it.
- Based: passes — The paper gives a reason why it holds.
Probably not a substantial claim. The paper’s conclusions may not depend on it.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“It is quite reasonable and expected that a pilot study is proposed with no pilot or other preliminary data supporting the proposal and that its proposed sample size is based on pragmatics such as patient flow and budgetary constraints.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > SAMPLE SIZE DETERMINATION IN DESIGNING A PILOT STUDY > Pilot Data for a Pilot Study
plain- Asserted: passes — The authors put it forward as true.
- Checkable: fails — It may be too vague to check against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. It may be too vague to check against evidence. The paper may give no reason why it holds. It may state two separate claims as one.
The paper states this without a source or data behind it here, so there is nothing to check it against. The quote shows it is in the paper.
In the paper
“A pilot study can be used to evaluate the feasibility of recruitment, randomization, retention, assessment procedures, new methods, and implementation of the novel intervention.”
- Also stated in: § INTRODUCTION · plain
“evaluate the feasibility of recruitment, randomization, retention, assessment procedures, new methods, and/or implementation of the novel intervention.”
- Also stated in: § WHAT A PILOT STUDY CAN DO: ASSESS FEASIBILITY · plain
“A pilot study can be used to evaluate the feasibility of recruitment, randomization, retention, assessment procedures, and implementation of the novel intervention and each of these can be quantified”
§ The Role and Interpretation of Pilot Studies in Clinical Research > Abstract
plain- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
- Also stated in: § INTRODUCTION · plain
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“There are two fundamental reasons that hypothesis testing is not used in a pilot study: the limited state of knowledge about the methods or intervention in the patient population to be studied and the smaller proposed sample size.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“As a consequence, pilot studies can reduce the proportion of failed trials and allow research funds to be spent on projects for which feasibility has been demonstrated and quantified.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > DISCUSSION
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: passes — The paper gives a reason why it holds.
It may state two separate claims as one.
A conclusion the paper draws from its own results — it cites no other source for it, so there is none to check. The quote shows it is in the paper.
In the paper
“Well-conceived and implemented pilot studies will reduce the risk of several problems that are commonly faced in clinical trials. These include the inability to recruit the proposed sample size and a corresponding reduction in statistical power, excessive attrition due to intolerable procedures or interventions, and the need to modify a protocol midway through a trial.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > DISCUSSION
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: unclear — It is unclear why the paper says it holds.
It may state two separate claims as one.
The paper states this without a source or data behind it here, so there is nothing to check it against. The quote shows it is in the paper.
In the paper
“Estimates become more precise with larger sample sizes.”
§ The Role and Interpretation of Pilot Studies in Clinical Research > EXCEEDING THE LIMITATIONS OF A PILOT STUDY: WHAT PILOT STUDIES CANNOT DO > Pilot Study Effect Sizes and Sample Size Determination
plaindirect- Asserted: passes — The authors put it forward as true.
- Checkable: passes — It can be checked against evidence.
- Load-bearing: passes — The paper’s conclusions depend on it.
- Based: fails — The paper may give no reason why it holds.
Probably not a substantial claim. The paper may give no reason why it holds.
The paper relies on this without support.
The true population effect size is unknown when a pilot study is designed.
“An essential challenge of therapeutic development is that the true population effect size is unknown at the time a pilot study is designed.”
The paper relies on this without support.
Methods will likely be modified after a pilot study, even if minimally.
“This is because it is quite likely that the methods will be modified after the pilot, even if minimally.”
The paper relies on this without support.
Pilot results presented to grant review committees tend to be selective, overly optimistic, and at times misrepresentational.
“Furthermore, pilot results that are presented to grant review committees tend to be selective, overly optimistic and, at times, misrepresentational.”
The paper relies on this without support.
Many pilot studies have far fewer than 18 participants per group.
“Many pilot studies have far fewer than 18 participants/group and therefore even greater imprecision.”
The paper relies on this without support.
It is not unusual for research grant applications and pilot study publications to convey optimism based on pilot data.
“Nevertheless, it is not unusual for research grant applications and pilot study publications to convey such optimism, particularly when based on pilot data.”
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The cited source doesn’t support the claim as written.
In the citing paper
“The planned sample size was 30 allowing for attrition. This study will inform the sample size for any future RCT (Leon et al., 2011).”
From A mixed methods pilot and feasibility open trial of internet-delivered cognitive behaviour therapy (iCanADAPT Advanced) for people with advanced cancer with depression and/or anxiety (2021) · cites this paper as Leon et al., 2011
Checked by a reasoning model — may be wrong.
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“This was a pilot study which like other pilot studies of MI did not have a power calculation. 31,32”
From A Pilot Randomized Controlled Trial to Assess the Impact of Motivational Interviewing on Initiating Behavioral Therapy for Migraine (2020) · cites this paper as [31]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Data analyses in the context of a pilot study are not hypothesis-driven but serve to provide information regarding feasibility and acceptability of the intervention [55].”
From Acceptance and Commitment Therapy Wellness Program for Latine Adults Who Smoke and Have Psychological Distress: Protocol for a Feasibility Study (2023) · cites this paper as [55]
Experimental — accuracy not yet measuredThe cited source says what the claim attributes to it.
In the citing paper
“A common misuse of pilot and feasibility studies has been the application of hypothesis testing for clinical outcomes in small under-powered studies.”
From Determining sample size for progression criteria for pragmatic pilot RCTs: the hypothesis test strikes back! (2021) · cites this paper as [20]
The cited source says what the claim attributes to it.
In the citing paper
“Hypothesis testing in pilot studies, particularly in the context of effectiveness/efficacy of clinical outcomes, has been widely criticised due to the improper purpose and lack of statistical power of such evaluations [2, 20, 21, 23].”
From Determining sample size for progression criteria for pragmatic pilot RCTs: the hypothesis test strikes back! (2021) · cites this paper as [20]
The cited source says what the claim attributes to it.
In the citing paper
“Since the focus was on feasibility and acceptability of the intervention and we did no hypothesis testing, no power calculation was required (Leon et al., 2011).”
From PandaMom – Feasibility and acceptability of an internet- and mobile-based intervention to enhance peripartum mental well-being and to prevent postpartum depression (2024) · cites this paper as Leon et al., 2011
The check wasn’t confident either way, so it gives no verdict — never disputed.
In the citing paper
“Our use of effect sizes was, however, deemed appropriate for this pilot trial (55, 63).”
From EMPOWER: A Multi-Site Pilot Trial to Reduce Distress in Surrogate Decision-Makers in the ICU (2024) · cites this paper as [63]
Experimental — accuracy not yet measuredIn the citing paper
“Previous work suggests early studies often test feasibility of methods (e.g., recruitment, randomization, retention, and assessment), not hypotheses [48].”
From Agile science: creating useful products for behavior change in the real world (2016) · cites this paper as [48]
In the citing paper
“In terms of statistics, a Bayesian approach might be more appropriate than Fisherian statistics [56] as the Fisher approach is not appropriate with limited data [48].”
From Agile science: creating useful products for behavior change in the real world (2016) · cites this paper as [48]
Citations
Cites
Fundamentals of Clinical Trials
Friedman, L. M., Furberg, C. D., DeMets, D. L., Reboussin, D. M., & Granger, C. B. (2015) · doi:10.1007/978-3-319-18539-2 · Cited by 2,237
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Fundamentals of Clinical Trials
Friedman, L. M., Furberg, C. D., & DeMets, D. L. (1998) · doi:10.1007/978-1-4757-2915-3 · Cited by 873
. Caution regarding the use of pilot studies to guide power calculations for study proposals.
Kraemer, H. C., Mintz, J., Noda, A., Tinklenberg, J., & Yesavage, J. A. (2006) · Archives of General Psychiatry · doi:10.1001/archpsyc.63.5.484 · Cited by 702 · cited 1×
“(Friedman, Furberg and DeMets, 1998; Kraemer et al., 2006) Pilot study results should not be used for sample size determination due to the inherent imprecision in between treatment group effect size estimates from studies with small samples.”
Self-citationWolfstein (1905) · The Journal of Nervous and Mental Disease · doi:10.1097/00005053-190502000-00009 · Cited by 346
Attrition in randomized controlled clinical trials: methodological issues in psychopharmacology
Leon, A. C., Mallinckrodt, C. H., Chuang-Stein, C., Archibald, D. G., Archer, G. E., & Chartier, K. (2006) · Biological Psychiatry · doi:10.1016/j.biopsych.2005.10.020 · Cited by 303 · cited 1×
“(Leon et al., 2006; Leon & Davis, 2009).”
Self-citationSixteen S ‐squared over D ‐squared: A relation for crude sample size estimates
Lehr, R. (1992) · Statistics in Medicine · doi:10.1002/sim.4780110811 · Cited by 253 · cited 1×
“(N/group ≈ 16/d2; Lehr, 1992) The clinical interpretation of HAMD change of 1.2 to 3.0 would drive the choice among possible sample sizes in planning a study.”
BackgroundInfluentialJovanovic, B. D., & Levy, P. S. (1997) · The American Statistician · doi:10.1080/00031305.1997.10473947 · Cited by 179 · cited 1×
“…a particular category, the rule of three should be applied to estimate the upper bound of the 95% CI, where the upper bound is approximately 3/n. (Jovanovic & Levy, 1997; Jovanovic et al., 1997) For example, consider a study with N=15 receiving medication and no suicidal ideation was reported…”
BackgroundHarvey, I. M. (2009) · Public Health · doi:10.1016/j.puhe.2009.05.009 · Cited by 173
Jovanovic, B. D., & Levy, P. S. (1997) · The American Statistician · doi:10.2307/2685405 · Cited by 50
Cited by
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Items 701–800 of 1,903
Vance, D. E., Pope, C. N., Fazeli, P. L., Azuero, A., Frank, J., Wadley, V. G., Raper, J. L., Byun, J. Y., & Ball, K. (2021) · Journal of the Association of Nurses in AIDS Care · doi:10.1097/jnc.0000000000000316 · Cited by 10
“Because of the exploratory nature of this small pilot study, the sample size was not powered for strict statistical inference (Leon et al., 2011).”
BackgroundBoyce, R. M., Muhindo, E., Baguma, E., Muhindo, R., Bwambale, S., François, R., Hawke, S., Shook‐Sa, B. E., Ntaro, M., Nalusaji, A., et al. (2022) · Malaria Journal · doi:10.1186/s12936-022-04086-w · Cited by 11
Bonar, E. E., Chapman, L., Pagoto, S., Tan, C. Y., Duval, E. R., McAfee, J., Collins, R. L., & Walton, M. A. (2022) · Drug and Alcohol Dependence · doi:10.1016/j.drugalcdep.2022.109693 · Cited by 10
Norweg, A. M., Wu, Y., Troxel, A. B., Whiteson, J. H., Collins, E. G., Haas, F., Skamai, A., Goldring, R. M., Jean‐Louis, G., Reibman, J., et al. (2023) · Journal of Integrative and Complementary Medicine · doi:10.1089/jicm.2022.0552 · Cited by 13
Martínez, O., Fernandez, M. I., Yang, P., Wu, E., Valentin, O., & Johnson, M. O. (2023) · American Journal of Men s Health · doi:10.1177/15579883231167106 · Cited by 11
“This decision was guided by the literature (Arain et al., 2010; Becker, 2008; Eldridge et al., 2016; Kistin & Silverstein, 2015; Kraemer et al., 2006; Lancaster, 2015; Lancaster et al., 2004; Leon et al., 2011; Thabane et al., 2010, 2016) and the NIH (National Center for Complementary and Integrative Health [NCCIH], 2…”
BackgroundFilippone, P., Serrano, S. E., Gwadz, M. V., Cleland, C. M., Freeman, R., Linnemayr, S., Cluesman, S. R., Campos, S., Rosmarin-DeStefano, C. L., Amos, B., et al. (2023) · Frontiers in Public Health · doi:10.3389/fpubh.2023.1167104 · Cited by 10
“” The main purpose of conducting a pilot study such as this pilot optimization trial is to examine the acceptability and feasibility of an approach that is intended to be used in a larger scale study (63, 106).”
BackgroundMethodWolf, F., Nielsen, J., Saliger, J., Hennecken, E., Kröber, P., Eschweiler, M., Folkerts, A., Karbe, H., & Zimmer, P. (2023) · BMC Neurology · doi:10.1186/s12883-023-03436-8 · Cited by 16
“Since this was a small-scale feasibility study, hypothesis testing of within-or between-group treatment effects was not performed [42, 43].”
Orchowski, L. M., Merrill, J. E., Oesterle, D. W., Barnett, N. P., Borsari, B. E., Zlotnick, C., Haikalis, M., & Berkowitz, A. D. (2023) · JMIR Formative Research · doi:10.2196/47354 · Cited by 11
“As such, outcome analyses are often included in pilot randomized trials to gauge the preliminary direction of effects and examine trends rather than to document effect sizes [51].”
BackgroundMethodSirey, J. A., Hannon, C. P., D’Angelo, D., & Knies, K. (2012) · Journal of Gerontological Social Work · doi:10.1080/01634372.2011.644029 · Cited by 10
“However, it is preliminary data and documenting clinical impact of the intervention is beyond the scope of a pilot study of this type (Leon et al., 2011).”
Congestive heart failure adherence redesign trial: a pilot study
Mangla, A., Doukky, R., Powell, L. H., Avery, E., Richardson, D., & Calvin, J. E. (2014) · BMJ Open · doi:10.1136/bmjopen-2014-006542 · Cited by 10
Woods, P., Schumacher, L. T., Sadhra, S. S., Sutton, A. J., Zarkar, A. M., Rolf, P., & Grunfeld, E. A. (2016) · JMIR Research Protocols · doi:10.2196/resprot.5300 · Cited by 10
“The study follows the best practice guidelines set out by the Medical Research Council, the UK national funding agency, in the development and evaluation of complex interventions [49] and published recommendations for pilot studies [50-53].”
Hafner, B. J., Halsne, E. G., Morgan, S. J., & Davidson, G. (2017) · JPO Journal of Prosthetics and Orthotics · doi:10.1097/jpo.0000000000000163 · Cited by 12
“While the sample in the present study (n = 7) limits generalizability of the results to a broad population, the results are informative and may inform larger prospective studies.(67) A second limitation of this study is the age and amputation etiology of the included participants.”
ResultO’Rourke, H. M., Sidani, S., Chu, C. H., Fox, M. T., McGilton, K. S., & Collins, J. (2017) · Gerontology and Geriatric Medicine · doi:10.1177/2333721417734672 · Cited by 10
Lim, A. C., Courtney, K. E., Moallem, N. R., Allen, V. C., Leventhal, A. M., & Ray, L. A. (2018) · Frontiers in Psychiatry · doi:10.3389/fpsyt.2018.00362 · Cited by 10
“Analytic Plan In accordance with NIH policies for and limitations of small feasibility trials in generating accurate and reliable effect sizes [NIH NOT-OD-16-149; (37, 38)], null hypothesis significance tests are not reported for clinical outcomes.”
MethodSong, M., Lee, C. S., Lyons, K. S., Stoyles, S. A., & Winters‐Stone, K. M. (2018) · SAGE Open Medicine · doi:10.1177/2050312118801220 · Cited by 10
Monroe, C. M., Geraci, M., Larsen, C. A., & West, D. S. (2019) · Obesity Science & Practice · doi:10.1002/osp4.352 · Cited by 9
“The target sample size (N = 36) was primarily dictated by practical considerations, including budgetary constraints (35), and corresponds with observed median sample sizes of previous pilot and feasibility randomized controlled trials (36).”
Felsted, K. F., & Supiano, K. P. (2019) · Research in Gerontological Nursing · doi:10.3928/19404921-20190702-02 · Cited by 10
Spratling, R. G., Faulkner, M. S., Feinberg, I. Z., & Hayat, M. J. (2019) · Journal of Advanced Nursing · doi:10.1111/jan.14235 · Cited by 10
Bianchi, W., George, T., McEvoy, C. S., Piehl, M. D., Manzano, A., Boboc, M., Zarow, G. J., Natarajan, R., Gaspary, M. J., Auten, J. D., et al. (2020) · Military Medicine · doi:10.1093/milmed/usz200 · Cited by 12
Santa-Cruz, D. C., Caparrós-González, R. A., Romero‐Gonzalez, B., Peralta‐Ramírez, M. I., González-Pérez, R., & García-Velasco, J. A. (2020) · International Journal of Environmental Research and Public Health · doi:10.3390/ijerph17093020 · Cited by 10
“A secondary objective of this study was to assess the methodological feasibility prior to designing a new study protocol with a larger sample [46,47].”
BackgroundKang, Y., & Ritzhaupt, A. D. (2021) · Journal of educational multimedia and hypermedia · Cited by 10
Schoenfeld, E. M., Poronsky, K. E., Westafer, L. M., DiFronzo, B. M., Visintainer, P., Scales, C. D., Hess, E. P., & Lindenauer, P. K. (2021) · Trials · doi:10.1186/s13063-021-05140-9 · Cited by 10
“Our sample size for phase I (until interim analysis 1) is based on the expert recommendation that at least 50 subjects be included to examine the pragmatics of recruitment [30].”
MethodInternet-delivered interventions for personality disorders – A scoping review
van der Boom, B., Boumparis, N., Donker, T., Beurs, D. P. D., Arntz, A. R., & Riper, H. M. (2022) · Internet Interventions · doi:10.1016/j.invent.2022.100525 · Cited by 16
Crombie, I. K., Cunningham, K. B., Irvine, L. A., Williams, B., Sniehotta, F. F., Norrie, J. D., Melson, A. J., Jones, C., Briggs, A. H., Rice, P. M., et al. (2017) · Health Technology Assessment · doi:10.3310/hta21190 · Cited by 12
Zhou, Y., Jackson, C. D., Oates, V. J., Davis, G. W., Davis, C., Takizala, Z.-M., Akatue, R. A., Williams, K., Liu, J., Hébert, J. R., et al. (2017) · Open Journal of Epidemiology · doi:10.4236/ojepi.2017.72009 · Cited by 10
“However, such pilot studies are necessary to better understand the methods and procedures applied and to improve future programs on a larger scale [56] [57].”
Machine learning in cancer imaging for enhanced precision in diagnosis and therapy
Chow, J. C. L. (2026) · Discover Computing · doi:10.1007/s10791-026-10078-0 · Cited by 12
“Moreover, prospective validation through clinical trials or pilot implementations provides the most rigorous test of clinical utility [60].”
Patel, M. L., Cleare, A. E., Smith, C. M., Rosas, L. G., & King, A. C. (2022) · JMIR Formative Research · doi:10.2196/42191 · Cited by 12
“we did not report between-group differences in any outcome, as recommended [93,94].”
Methodvon Klinggraeff, L., Ramey, K. T., Pfledderer, C. D., Burkart, S. A., Armstrong, B., Weaver, R. G., & Beets, M. W. (2023) · Pilot and Feasibility Studies · doi:10.1186/s40814-023-01345-8 · Cited by 9
“Though recommendations call for the reporting of trial and study feasibility rather than efficacy [27, 28], the perception remains that smaller efficacy studies and those not reporting efficacy are unlikely to be published [29].”
Backgroundvan Loggerenberg, F., Akena, D. H., Alinaitwe, R., Birabwa‐Oketcho, H., Méndez, C. A. C., Gómez–Restrepo, C., Kulenović, A. D., Selak, N., Kiseljaković, M., Musisi, S., et al. (2023) · BMC Family Practice · doi:10.1186/s12875-023-02197-0 · Cited by 9
“Effect sizes from pilot studies tend to be optimistic and can be unstable due to small sample sizes and this needs to be considered when using these estimates to design fully powered randomized controlled trials [52].”
BackgroundLau, N., Palermo, T. M., Zhou, C., Badillo, I., Hong, S., Aalfs, H., Yi-Frazier, J. P., McCauley, E., Chow, E. J., Weiner, B. J., et al. (2024) · JMIR Research Protocols · doi:10.2196/57950 · Cited by 9
“Exploratory efficacy will be examined using the first phase parallel RCT data with regression models [44,45].”
BackgroundMethodShankar, R., Hong, W.-Z., Bundele, A., Si, K. P., & Mukhopadhyay, A. (2024) · BMJ Open · doi:10.1136/bmjopen-2024-090550 · Cited by 10
“63 The analysis of the outcomes will focus on estimating effect sizes and variability to inform sample size calculations for a future definitive trial.”
BackgroundDang, T. H., Wickramasinghe, N., Jayaraman, P. P., Burbury, K., Alexander, M., Whitechurch, A., Dyer, M., Quinn, S., Forkan, A. R. M., & Schofield, P. (2025) · JMIR Formative Research · doi:10.2196/65302 · Cited by 9
“…the patients’ acceptance of SAMSON, measured by the Unified Theory of Acceptance and Use of Technology (UTAUT) [24,25] at 12 weeks (t 1 ), and study feasibility, measured by predefined rates of recruitment, randomization, retention, intervention adherence, and outcome assessment completion [26,27].”
Improving statistical reporting in psychology
Schubert, A., Snijder-Steinhilber, M., Kang, H., & Quintana, D. S. (2025) · Communications Psychology · doi:10.1038/s44271-025-00356-w · Cited by 20
Corones‐Watkins, K., Cooke, M. L., Theobald, K. A., White, K. M., Thompson, D. R., Ski, C. F., King‐Shier, K. M., Conway, A., & Ramis, M. (2020) · Australian Critical Care · doi:10.1016/j.aucc.2020.10.012 · Cited by 9
A feasibility study of the Redesigning Daily Occupations (ReDO TM -10) programme in an Irish context
Fox, J., Erlandsson, L., & Shiel, A. (2021) · Scandinavian Journal of Occupational Therapy · doi:10.1080/11038128.2021.1882561 · Cited by 9
“inferential statistical tests were carried out [51].”
Cardiovascular health in emerging adults with type 1 diabetes
McCarthy, M. M., Yan, J., Jared, M. C., You, E., Ilkowitz, J. T., GALLAGHER, M. P., & Dickson, V. V. (2021) · European Journal of Cardiovascular Nursing · doi:10.1093/eurjcn/zvab062 · Cited by 9
Kelleher, S. A., Fisher, H. M., Hyland, K. A., Miller, S. N., Amaden, G. H., Diachina, A. K., Pittman, A., Winger, J. G., Sung, A. D., Berchuck, S. I., et al. (2022) · Journal of Psychosocial Oncology · doi:10.1080/07347332.2022.2152519 · Cited by 11
The negativity bias predicts response rate to Behavioral Activation for depression
Gollan, J. K., Hoxha, D., Hunnicutt‐Ferguson, K., Norris, C. J., Rosebrock, L. E., Sankin, L. S., & Cacioppo, J. T. (2015) · Journal of Behavior Therapy and Experimental Psychiatry · doi:10.1016/j.jbtep.2015.09.011 · Cited by 11
Not All Studies With Small Samples Are Pilot Studies
McGrath, J. M. (2013) · The Journal of Perinatal & Neonatal Nursing · doi:10.1097/01.jpn.0000437186.01731.bc · Cited by 9
Whiteside, L., Darnell, D. A., Jackson, K., Wang, J., Russo, J. E., Donovan, D. M., & Zatzick, D. (2017) · Journal of Substance Abuse Treatment · doi:10.1016/j.jsat.2017.08.005 · Cited by 9
Wijker, C., Spek, A. A., Leontjevas, R., Verheggen, T., & Enders‐Slegers, M. (2017) · Autism - Open Access · doi:10.4172/2165-7890.1000221 · Cited by 9
“Before conducting a hypothesis testing study, it is important to examine the feasibility and implementability of an approach in a pilot study [41].”
BackgroundGhayomzadeh, M., Earnest, C. P., SeyedAlinaghi, S., Wesson, P., Taj, L., Rezaei, S., Navalta, J. W., Mohraz, M., Gharakhanlou, R., & Voltarelli, F. A. (2019) · Journal of the Association of Nurses in AIDS Care · doi:10.1097/jnc.0000000000000020 · Cited by 9
Stewart, E. M., Catroppa, C., & Lah, S. S. (2019) · Pilot and Feasibility Studies · doi:10.1186/s40814-019-0393-x · Cited by 9
“The decision to conduct an open-label pilot was guided by research by Leon and Davis [33], who state that open-label studies in which participants are recruited to known albeit experimental interventions with no risk of receiving placebo are best practise in pilot trials.”
MethodElias, R., Murphy, H. G., Turner, K. A., & White, S. W. (2019) · Journal of Cognitive Psychotherapy · doi:10.1891/0889-8391.33.4.343 · Cited by 9
“Establishing feasibility of a novel application of an intervention is often regarded as the first step in determining the appropriateness of the program for a large-scale clinical trial (Lancaster, Dodd, & Williamson, 2004; Leon et al., 2011; Wittes & Brittain, 1990).”
BackgroundGuadagni, S., Fiorentini, G., Papasotiriou, I., Apostolou, P., Masedu, F., Sarti, D., Farina, A. R., Mackay, A. R., & Clementi, M. (2020) · BMC Research Notes · doi:10.1186/s13104-020-05021-5 · Cited by 9
Kelechi, T. J., Prentice, M. A., Mueller, M., Madisetti, M., & Vertegel, A. A. (2020) · JMIR mhealth and uhealth · doi:10.2196/15015 · Cited by 9
“Similarly, no effect sizes (eg, Cohen d ) were provided because of large imprecision with small sample sizes [25].”
von Kaeppler, E. P., Donnelley, C. A., Ali, S. H., Roberts, H. J., Ibrahim, J. M., Wu, H., Eliezer, E. N., Porco, T. C., Haonga, B. T., Morshed, S., et al. (2021) · Pilot and Feasibility Studies · doi:10.1186/s40814-021-00766-7 · Cited by 9
Coulter, R. W. S., Mitchell, S. R., Prangley, K., Smallwood, S., Bonanno, L., Foster, E. N., Wilson, A., Miller, E. P., & Chugani, C. D. (2021) · JMIR Research Protocols · doi:10.2196/26554 · Cited by 9
“We calculate statistical power based on our primary outcomes (study aim 1), per the best practices for feasibility studies [64,79-84].”
MethodNiyibizi, J. B., Nganabashaka, J. P., Ntawuyirushintege, S., et al. (2021) · Frontiers in Tropical Diseases · doi:10.3389/fitd.2021.752357 · Cited by 9
Jaffar, A., Muhammad, N. A., Sidik, S. M., Admodisastro, N. I., Manaf, R. A., Foo, C. N., & Suhaili, N. (2022) · International Journal of Environmental Research and Public Health · doi:10.3390/ijerph19063574 · Cited by 11
“This study was expected to have 64 participants within two months as there was no requirement to have a powered sample size for the pilot study [40].”
BackgroundGwadz, M. V., Serrano, S. E., Linnemayr, S., Cleland, C. M., Cluesman, S. R., Freeman, R., Kellam, K., Stefano, C. D., Israel, K., & Pan, E. (2022) · Frontiers in Public Health · doi:10.3389/fpubh.2022.916224 · Cited by 9
“Following recommendations from NIH (62) and in the methods literature (63), we did not perform null-hypothesis significance testing with these pilot data.”
MethodAschbrenner, K. A., Mueser, K. T., Bartels, S. J., Carpenter-Song, E. A., Pratt, S. I., Barre, L., Naslund, J. A., & Kinney, A. (2015) · Health & Social Work · doi:10.1093/hsw/hlv006 · Cited by 9
Srikesavan, C. S., Shay, B. L., & Szturm, T. J. (2016) · Games for Health Journal · doi:10.1089/g4h.2016.0005 · Cited by 11
Woessner, M. N., VanBruggen, M., Pieper, C. F., O’Reilly, E. K., Kraus, W. E., & Allen, J. D. (2017) · JMIR Research Protocols · doi:10.2196/resprot.7596 · Cited by 9
“While the results of this pilot study will prove useful in the calculation of power, we are aware of the many risks of “playing the winner” in choosing to power a subsequent trial from pilot study results only [50,51].”
King, G. A., McPherson, A. C., Kingsnorth, S., Gorter, J. W., & DeFinney, A. (2018) · Developmental Neurorehabilitation · doi:10.1080/17518423.2018.1497722 · Cited by 9
Oakeshott, P., Kerry-Barnard, S. R., Fleming, C. F., Phillips, R., Drennan, V. M., Adams, E. J., Majewska, W., Harding-Esch, E. M., Cousins, E., Planche, T., et al. (2018) · Clinical Microbiology and Infection · doi:10.1016/j.cmi.2018.10.019 · Cited by 9
“Since this was a feasibility study, no significance testing was performed [19].”
BackgroundGallinat, C., Moessner, M., Haenssle, H. A., Winkler, J. K., Backenstraß, M., & Bauer, S. (2018) · Contemporary Clinical Trials Communications · doi:10.1016/j.conctc.2018.100315 · Cited by 9
“We follow the recommendations of Leon, Davis and Kraemer regarding sample size determination in pilot studies [22].”
MethodRanney, M. L., Patena, J. V., DUNSIGER, S. I., Spirito, A., Cunningham, R. M., Boyer, E. W., & Nugent, N. R. (2019) · Contemporary Clinical Trials · doi:10.1016/j.cct.2019.05.009 · Cited by 11
Flehr, A., Barton, C., Coles, J. Y., Gibson, S. J., Lambert, G. W., Lambert, É., Dhar, A. K., & Dixon, J. B. (2019) · BMC Complementary and Alternative Medicine · doi:10.1186/s12906-019-2642-1 · Cited by 9
“Sample size was determined bearing in mind recruitment, methodology and funding feasibility [65].”
Lavallée, A., Aita, M., Côté, J., Bell, L., & Luu, T. M. (2020) · Pilot and Feasibility Studies · doi:10.1186/s40814-020-00601-5 · Cited by 9
“Moreover, as suggested by Leon, Davis [67], pilot studies’ sample size should be based on pragmatics such as patient flow.”
BackgroundDe-implementation of detrimental feeding practices: a pilot protocol
Swindle, T., Rutledge, J. M., Johnson, S. L., Selig, J. P., & Curran, G. M. (2020) · Pilot and Feasibility Studies · doi:10.1186/s40814-020-00720-z · Cited by 10
Gruber, A. H., McDonnell, J., Davis, J. J., Vollmar, J. E., Harezlak, J., & Paquette, M. R. (2021) · Frontiers in Sports and Active Living · doi:10.3389/fspor.2021.630975 · Cited by 9
“This support is consistent with the purpose of pilot studies (Leon et al., 2011).”
ResultSomers, T. J., Blumenthal, J. A., Dorfman, C. S., Huffman, K. M., Edmond, S. N., Miller, S. N., Wren, A. A., Caldwell, D., & Keefe, F. J. (2022) · JCR Journal of Clinical Rheumatology · doi:10.1097/rhu.0000000000001793 · Cited by 13
“Given that this was a pilot feasibility study, outcome analyses were exploratory, and thus, results should be interpreted with caution.(38)”
ResultLangdon, K. J., Hitch, A. E., Collins, A. B., Beckwith, C. G., Becker, S. J., Tashima, K. T., & Rich, J. D. (2023) · Addiction Science & Clinical Practice · doi:10.1186/s13722-023-00418-6 · Cited by 8
“A sample size of 40 was selected to garner information about our study design as well as determine what modification may be needed to develop a larger, hypothesis testing, study in the future [45, 46].”
MethodExploring the perceived mental health benefits of pickleball for junior players: a pilot study
Caldarella, P., Moya, M. S., Larsen, R., Horner, J. S., & Richards, E. J. (2024) · Leisure/Loisir · doi:10.1080/14927713.2024.2332659 · Cited by 9
“Pilot studies are an important initial step in exploring novel applications of interventions or programs, with results helping to inform feasibility and potential modifications needed in large scale studies (Leon et al., 2011).”
Dwenger, K., Roy, N., Jennings, S. G., Smith, M. E., Mathy, P., Simonyan, K., & Barkmeier‐Kraemer, J. M. (2025) · Journal of Speech Language and Hearing Research · doi:10.1044/2024_jslhr-24-00476 · Cited by 8
“Thus, on the basis of guidance from the literature on proof-of-concept pilot feasibility studies (Billingham et al., 2013; Leon et al., 2011; Moore et al., 2011), we aimed for a minimum of five participants in each group to inform future larger scale studies and population size estimates.”
Feasibility of a family‐centered intervention for depressed older men in primary care
Hinton, L., Frano, E. L., Harvey, D. J., Alfaro, E. D., Kravitz, R. L., Smith, A., Apesoa‐Varano, E. C., Jafri, A., & Unützer, J. (2019) · International Journal of Geriatric Psychiatry · doi:10.1002/gps.5196 · Cited by 8
Przkora, R., Sibille, K. T., Victor, S., Meroney, M., Leeuwenburgh, C., Gardner, A. K., Vasilopoulos, T., & Parvataneni, H. K. (2021) · European Journal of Translational Myology · doi:10.4081/ejtm.2021.9932 · Cited by 8
Frawley, E., Cowman, M., Cella, M., Cohen, D. R., Ryan, E., Hallahan, B. P., Bowie, C. R., McDonald, C., Fowler, D. G., Wykes, T., et al. (2022) · Pilot and Feasibility Studies · doi:10.1186/s40814-022-01064-6 · Cited by 9
“This pragmatic approach is consistent with other feasibility studies in the area of early psychosis [28] and in line with current recommendations for pilot studies [29].”
ResultWilliamson, A. A., Soehner, A. M., Boyd, R. C., Buysse, D. J., Harvey, A. G., Jonassaint, C. R., Franzen, P. L., & Goldstein, T. R. (2022) · Frontiers in Public Health · doi:10.3389/fpubh.2022.971754 · Cited by 9
Soltero, E. G., Lopez, C., Musaad, S. M. A., O’Connor, T. M., & Thompson, D. I. (2023) · Contemporary Clinical Trials · doi:10.1016/j.cct.2023.107117 · Cited by 7
Dobbs, D., Yauk, J., Vogel, C. E., Fanfan, D., Buck, H. G., Haley, W. E., & Meng, H. (2023) · The Gerontologist · doi:10.1093/geront/gnad018 · Cited by 13
Sileo, K. M., Muhumuza, C., Wanyenze, R. K., Kershaw, T., Sekamatte, S., Lule, H., & Kiene, S. M. (2023) · Contraception · doi:10.1016/j.contraception.2023.110096 · Cited by 9
Woodworth, E. C., Briskin, E. A., Plys, E., Macklin, E. A., Tatar, R., Huberty, J., & Vranceanu, A. (2023) · JMIR Research Protocols · doi:10.2196/50108 · Cited by 18
“Consistent with guidelines for early feasibility studies, our analysis will not assess efficacy, as we are not powered to do so [68].”
MethodLloyd‐Johnsen, C., Hampton, A., Stubbs, E., et al. (2023) · BMC Health Services Research · doi:10.1186/s12913-023-10148-9 · Cited by 8
“This can include the evaluation of recruitment, retention, data collection procedures and analysis plans [7].”
BackgroundMalighetti, C., Schnitzer, C. K., YorkWilliams, S. L., Bernardelli, L., Runfola, C. D., Riva, G., & Safer, D. L. (2023) · Journal of Clinical Medicine · doi:10.3390/jcm12237402 · Cited by 8
“The choice of these definitions of feasibility were based on the research literature [46].”
MethodBeauchamp, J. E. S., Wang, M., Novelo, L. G. L., Cox, C. S., Meyer, T., Fagundes, C. P., Savitz, S. I., Sharrief, A. Z., Dishman, D., & Johnson, C. (2023) · Journal of Stroke and Cerebrovascular Diseases · doi:10.1016/j.jstrokecerebrovasdis.2023.107515 · Cited by 8
Groessl, E. J., Hafey, C., McCarthy, A., Hernandez, R. M., Prado-Nava, M., Casteel, D., McKinnon, S., Chang, D. G., Ayers, C. R., Rutledge, T. R., et al. (2023) · Global Advances in Integrative Medicine and Health · doi:10.1177/27536130231220623 · Cited by 8
“54 Thus, we provide pre-post effect sizes and 95% con fi dence intervals for the pre-post change in outcomes.”
MethodMcDermott, K. A., Levey, N., Brewer, J. R., Ehmann, M., Hooker, J. E., Pasinski, R., Yousif, N., Raju, V., Gholston, M., Greenberg, J., et al. (2023) · JMIR Research Protocols · doi:10.2196/52117 · Cited by 8
“Given the concerns regarding the use of open pilot studies to calculate effect sizes [89,90], sample size estimates and proposed analyses were determined with the primary goal of establishing feasibility, as opposed to powering for specific effects for the quantitative outcomes.”
MethodLatimer, S. L., Walker, R., Chaboyer, W. P., Thalib, L., Coyer, F. M., Deakin, J. L., & Gillespie, B. M. (2024) · Intensive and Critical Care Nursing · doi:10.1016/j.iccn.2024.103746 · Cited by 10
Using incremental science to improve inclusive educational psychology research
Chow, J. C., & Toste, J. R. (2025) · Educational Psychologist · doi:10.1080/00461520.2025.2486140 · Cited by 8
“The use of pilot or pre-liminary studies to inform larger, more costly studies is widespread across the fields of medicine, psychiatry, psychology, sociology, and education (Leon et al., 2011; Moore et al., 2011; Pfledderer et al., 2024; Smith, 2019; Thabane et al., 2010).”
The Value of Pilot Studies in Clinical Research
Wolfe, B. E. (2013) · Journal of the American Psychiatric Nurses Association · doi:10.1177/1078390313493248 · Cited by 8
“However, others note the limitations in using crude estimates to project effect size of a population (Kraemer, Mintz, Noda, Tinklenberg, & Yesavage, 2006; Leon et al., 2011; Thabane et al., 2010).”
BackgroundPfund, R. A., Whelan, J., Meyers, A. W., Peter, S. C., Ward, K. D., & Horn, T. L. (2019) · Journal of Technology in Behavioral Science · doi:10.1007/s41347-019-00123-9 · Cited by 9
Young, A., Healy, S., Silliman-French, L. M., & Brian, A. (2021) · Adapted Physical Activity Quarterly · doi:10.1123/apaq.2020-0171 · Cited by 11
Foley, J. D., Firkey, M. K., Sheinfil, A. Z., Ramos, J. M., Woolf‐King, S. E., & Vanable, P. A. (2021) · Archives of Sexual Behavior · doi:10.1007/s10508-021-02045-1 · Cited by 9
Cepni, A. B., Taylor, A., Thompson, D. I., Moran, N. E., Olvera, N. E., O’Connor, D. P., Johnston, C. A., & Ledoux, T. A. (2021) · Appetite · doi:10.1016/j.appet.2021.105608 · Cited by 8
MacKenzie, D. E., Kiepek, N. C., Picketts, L., Zubriski, S., Landry, K., & Harris, J. (2021) · The Open Journal of Occupational Therapy · doi:10.15453/2168-6408.1799 · Cited by 8
“It was not designed to estimate a sample size of a future study (Leon et al., 2011), so it was important to use the smallest sample possible to limit human and financial resources while still completing a quality study.”
BackgroundMethodLutala, P., & Muula, A. (2022) · Pilot and Feasibility Studies · doi:10.1186/s40814-022-01032-0 · Cited by 8
“As a pilot study, interpretation has to be made within the following limits: this is not a hypothesis-testing study, and does not necessarily allow for generalization beyond the inclusion and exclusion criteria [64].”
BackgroundFerrara, M., Rivera-Real, A., Hillier, R. J., Habib, M., Kadhim, M. R., Sandinha, T., Curran, K., Muldrew, A., & STEEL, D. H. (2022) · Graefe s Archive for Clinical and Experimental Ophthalmology · doi:10.1007/s00417-022-05932-y · Cited by 8
“In this regard, feasibility and proof-of-concept studies play a crucial role in investigating this area, representing a key first step in assessing specific study designs and endpoints with the intent to use them in subsequent larger-scale trials [11].”
BackgroundBoyce, R. M., Ndizeye, R., Ngelese, H., et al. (2023) · PLOS Global Public Health · doi:10.1371/journal.pgph.0001678 · Cited by 8
“Due to the pilot nature of the study, no power calculations were conducted for the primary or secondary outcomes [27,28].”
MethodMcGuier, E. A., Feldman, J. A., Bay, M., Ascione, S., Tatum, M., Salas, E., & Kolko, D. J. (2023) · Children and Youth Services Review · doi:10.1016/j.childyouth.2023.107096 · Cited by 11
VanVoorhis, R. W., Miller, K. L., & Miller, S. M. (2023) · Journal of Child and Family Studies · doi:10.1007/s10826-023-02622-z · Cited by 10
“preparation of researchers and research assistants (Leon et al., 2011).”
BackgroundMartorella, G., Hanley, A. W., Tong, H., Miao, H., Worts, P. R., Bellamy, D., & Ahn, H. B. (2024) · Mindfulness · doi:10.1007/s12671-024-02329-2 · Cited by 7
“…OCC’s validity and reliability in detecting drug-related behavior are well established despite a low internal consistency ( α between 0.19 and 0.41, which is explained not performed since a pilot study is usually designed for hypothesis generation instead of hypothesis testing (Leon et al., 2011).”
InfluentialDevasahayam, A. J., Lim, C. K. S., Goh, M. R., You, J. P. L., & Pua, P. Y. (2014) · Proceedings of Singapore Healthcare · doi:10.1177/201010581402300307 · Cited by 8
What's in a Name? Revisiting Pilot Studies in Nursing Education Research
Spurlock, D. R. (2018) · Journal of Nursing Education · doi:10.3928/01484834-20180720-02 · Cited by 9
Yoé, R. Z., & Ramírez-Mendoza, R. A. (2019) · International Journal on Interactive Design and Manufacturing (IJIDeM) · doi:10.1007/s12008-019-00601-7 · Cited by 8
Harriss, A. B., Abbott, K. C., Kimpinski, K., Holmes, J. D., Johnson, A. M., Walton, D. M., & Dickey, J. P. (2019) · Sports · doi:10.3390/sports7110229 · Cited by 8
“Accordingly, the objective of this investigation was to conduct a pilot study in female youth soccer players, in accordance with current guidelines [22], to investigate (1) the feasibility of the study design (participant recruitment, adherence, retention, and adverse events) and (2) the effect sizes associated with c…”
MethodRush, C., Strickland, J., Pike, E., Studts, C. R., & Stoops, W. W. (2019) · Drug and Alcohol Dependence · doi:10.1016/j.drugalcdep.2019.107803 · Cited by 5
McCarthy, M. J., Sanchez, A., Garcia, Y. E., Lyons, K. S., & Bakas, T. (2020) · Research on Social Work Practice · doi:10.1177/1049731520961172 · Cited by 8
Fuller, G. W., Keating, S., Goodacre, S. W., Herbert, E., Perkins, G. D., Rosser, A., Gunson, I. M., Miller, J., Ward, M., Bradburn, M. J., et al. (2021) · Health Technology Assessment · doi:10.3310/hta25070 · Cited by 9
Metadata
- Authors
- Leon, A. C., Davis, L. L., & Kraemer, H. C.
- Affiliations
- Weill Cornell Medicine (US)
- University of Alabama (US)
- Stanford University (US)
- Corresponding author
- Leon, Andrew C.
- Venue
- Journal of Psychiatric Research
- Publisher
- Elsevier BV
- Published
- 2011-05
- Volume
- 45
- Issue
- 5
- Pages
- 626-629
- ISSN
- 0022-3956 (ISSN-L)
- PMC
- PMC3081994
- OpenAlex
- W2168434488
- Cited by
- 1,917
- References
- 17
- Citations by year
- 2026: 203 · 2025: 190 · 2024: 143 · 2023: 169 · 2022: 163 · 2021: 217 · 2020: 169 · 2019: 136 · 2018: 128 · 2017: 120 · 2016: 92 · 2015: 80 · 2014: 52 · 2013: 31 · 2012: 17
- FWCI
- 14.45
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Journal of Psychiatric Research (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of Mental Health (P30MH068638, R01MH060447)
- Topics
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Social Sciences › Decision Sciences › Statistics, Probability and Uncertainty › Meta-analysis and systematic reviews
- Keywords
- pilot study, feasibility assessment, clinical trials, research design, sample size determination, effect size estimation, generalizability, recruitment and retention, intervention implementation, research funding review
- MeSH terms
- Clinical Trials as Topic, Data Interpretation, Statistical, Feasibility Studies, Humans, Pilot Projects, Biomedical Research
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