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
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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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. 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
Sorted by citations
Items 501–600 of 1,903
Bennett, D., Brayne, C., Feigin, V. L., Barker-Collo, S. L., Brainin, M., Davis, D. H. J., Gallo, V., Jetté, N., Karch, A., Kurtzke, J. F., et al. (2015) · Neuroepidemiology · doi:10.1159/000439132 · Cited by 17
Aschbrenner, K., Mueser, K., Naslund, J., Gorin, A. A., Kinney, A., Daniels, L., & Bartels, S. (2016) · Journal of the Society for Social Work and Research · doi:10.1086/686486 · Cited by 16
Palliative Care in the Management of Pain, Odor, and Exudate in Chronic Wounds at the End of Life
Kelechi, T. J., Prentice, M. A., Madisetti, M., Brunette, G. P., & Mueller, M. (2017) · Journal of Hospice and Palliative Nursing · doi:10.1097/njh.0000000000000306 · Cited by 17
“Formal a priori power calculations were not computed; instead, sample size was determined using the ‘‘pragmatics of recruitment’’ for enrollment of inpatients and outpatients, n = 50, to evaluate a signal of clinical efficacy in wound symptom outcomes of pain, odor, and exudate.(34) We did not power to detect statisti…”
MethodGoogle Glass in Nursing Education
Byrne, P. J., & Senk, P. A. (2017) · CIN Computers Informatics Nursing · doi:10.1097/cin.0000000000000339 · Cited by 17
“As a feasibility pilot study, predetermination of sample size is not necessary as inferential statistics were not performed as part of the study.(21) The study was presented to all senior baccalaureate nursing students (n = 100).”
BackgroundBrief Report: Autism Spectrum Disorder: A Comprehensive Survey of Randomized Controlled Trials
Tromans, S. J., & Adams, C. E. (2018) · Journal of Autism and Developmental Disorders · doi:10.1007/s10803-018-3569-y · Cited by 16
Consumer attitudes toward downward extensions: an exploration of Giordano’s extension in Hong Kong
Fan, Z., Lee, S., & Heung, C. M. (2019) · Fashion and Textiles · doi:10.1186/s40691-019-0185-1 · Cited by 16
Vance, D. E., Fazeli, P. L., Azuero, A., et al. (2021) · Applied Neuropsychology Adult · doi:10.1080/23279095.2021.1906678 · Cited by 16
“The sample size considerations for the study were based on feasibility and, as any pilot project (Leon et al., 2011), the study was not powered for formal statistical inference.”
MethodAn Efficient System for Eye Movement Desensitization and Reprocessing (EMDR) Therapy: A Pilot Study
Goga, N., Boiangiu, C.-A., Vasilăţeanu, A., Popovici, A.-F., Drăgoi, M.-V., Popovici, R., Gancea, I. O., Pîrlog, M. C., Popa, R. C., & Hadăr, A. (2022) · Healthcare · doi:10.3390/healthcare10010133 · Cited by 16
“[23] in recruiting and selecting participants, who emphasize that a pilot sample size is based on the pragmatics of recruitment a d the necessity for examining the feasibility of the application.”
BackgroundInfluentialvon Klinggraeff, L., Dugger, R., Okely, A. D., Lubans, D. R., Jago, R., Burkart, S. A., Weaver, R. G., Armstrong, B., Pfledderer, C. D., & Beets, M. W. (2022) · Pilot and Feasibility Studies · doi:10.1186/s40814-022-00991-8 · Cited by 17
“This perspective of interpreting feasibility markers alongside preliminary signals of efficacy is reflective of changes in the field of behavioral research where investigators suggest testing for statistical effects in pilot studies is no longer recommended [31, 32].”
Kemp, J. J., Benito, K., Herren, J. A., Brown, Z. A., Frank, H. E., & Freeman, J. B. (2023) · Frontiers in Psychiatry · doi:10.3389/fpsyt.2023.1096259 · Cited by 17
“The pilot nature of this study does not allow sufficient statistical power to conduct a non-inferiority test of group differences on EBPAS change, so comparison of descriptive statistics will provide initial evidence of specificity to be fully tested in a larger trial (61, 62).”
Hamid, N., Sanusi, S., Norizan, S., Urus, S. T., & Lestari, E. D. (2023) · Review of Economics and Finance · doi:10.55365/1923.x2023.21.6 · Cited by 16
“The goal of this pilot test is to make the questionnaire better, which will help identify if an approach that is meant to be used in a larger study is possible (Leon, Davis, & Kraemer, 2011).”
Coughlin, L. N., Bonar, E. E., Wieringa, J., Zhang, L., Rostker, M. J., Augustiniak, A. N., Goodman, G. J., & Lin, L. A. (2023) · Journal of Psychiatric Research · doi:10.1016/j.jpsychires.2023.05.012 · Cited by 16
Lau, A. S., Huey, S. J., & Baumann, A. A. (2023) · Behaviour Research and Therapy · doi:10.1016/j.brat.2023.104377 · Cited by 19
Burke, M. M., Cheung, W. C., & Kim, J. (2023) · Intellectual and developmental disabilities · doi:10.1352/1934-9556-61.5.426 · Cited by 16
A randomized, controlled pilot study of positive affect treatment adapted for anorexia nervosa
Haynos, A. F., Anderson, L. M., Askew, A. J., Liu, C., Venables, K. M., Craske, M. G., & Peterson, C. B. (2023) · International Journal of Eating Disorders · doi:10.1002/eat.24071 · Cited by 18
Macrynikola, N., Chen, K., Lane, E., Nguyen, N. S., Pinto, J., Yen, S., & Torous, J. B. (2024) · JMIR Mental Health · doi:10.2196/65222 · Cited by 19
“In line with, and guided by, guidelines for feasibility studies [67], we computed descriptive statistics to assess feasibility and acceptability, and then conducted paired samples t tests (2-tailed, with Cohen d effect sizes) to examine within-group pre-post differences as a marker of potential…”
Griffith, K. A., Royak-Schaler, R., Nesbitt, K., Zhan, M., Kozlovsky, A., Hurley, K. M., Pelser, C. B., Tkaczuk, K. H. R., & Ryan, A. S. (2012) · Nutrition and Health · doi:10.1177/0260106012459938 · Cited by 16
“Because pilot studies are not designed to test hypotheses, the sample size was estimated based on the ability to test an intervention for acceptability (Leon et al., 2011).”
BackgroundConsumer Perspectives on Involving Family and Significant Others in a Healthy Lifestyle Intervention
Aschbrenner, K. A., Bartels, S. J., Mueser, K. T., Carpenter-Song, E. A., & Kinney, A. (2012) · Health & Social Work · doi:10.1093/hsw/hls032 · Cited by 17
Airway clearance by exercising in mild cystic fibrosis (ACE-CF): A feasibility study
Ward, N., Stiller, K. R., Rowe, H., et al. (2018) · Respiratory Medicine · doi:10.1016/j.rmed.2018.07.008 · Cited by 16
Gulsrud, A. C., Carr, T., Williams, J., Panganiban, J. L., Jones, F., Kimbrough, J., Shih, W., & Kasari, C. L. (2019) · Autism Research · doi:10.1002/aur.2160 · Cited by 17
Appalasamy, J. R., Joseph, J. P., Seeta Ramaiah, S., Md Zain, A. Z., Quek, K. F., & Tha, K. K. (2020) · JMIR Aging · doi:10.2196/17182 · Cited by 16
“Moreover, a feasibility study without inferential results does not necessarily require a power analysis [29].”
BackgroundDevelopment of the Occupational Performance Inventory of Sexuality and Intimacy (OPISI): Phase One
Walker, B. A., Otte, K., Lemond, K., Hess, P. C., Kaizer, K., Faulkner, T., & Christy, D. (2020) · The Open Journal of Occupational Therapy · doi:10.15453/2168-6408.1694 · Cited by 17
Brown, R. A., Bloom, E. L., Hecht, J., Moitra, E., Herman, D. S., & Stein, M. D. (2014) · Behavior Modification · doi:10.1177/0145445514538279 · Cited by 16
“We accomplished our primary goal of establishing the feasibility and acceptability of this approach (Leon et al., 2011).”
Feasibility of online self-administered cognitive training in moderate–severe brain injury
Sharma, B., Tomaszczyk, J. C., DAWSON, D. R., Turner, G. R., Colella, B., & Green, R. (2016) · Disability and Rehabilitation · doi:10.1080/09638288.2016.1195453 · Cited by 17
A Sample Size Calculator for SMART Pilot Studies
Kim, H. (2016) · SIAM Undergraduate Research Online · doi:10.1137/15s014058 · Cited by 17
“However, because the primary aim of pilot studies center on acceptability and feasibility considerations, the sample size for pilot studies are not based on statistical power considerations [18, 19, 20, 21].”
Claborn, K., Becker, S. J., Ramsey, S. E., Rich, J. D., & Friedmann, P. D. (2017) · Addiction Science & Clinical Practice · doi:10.1186/s13722-017-0073-1 · Cited by 15
“Primary outcomes will be feasibility and acceptability of the CCI and assessment procedures to be used in a larger trial [35].”
BackgroundMethodAsfar, T., Caban-Martinez, A. J., McClure, L. A., Ruano-Herreria, E. C., Sierra, D., Clark, G. G., Samano, D., Dietz, N. A., Ward, K. D., Arheart, K. L., et al. (2018) · Contemporary Clinical Trials · doi:10.1016/j.cct.2018.02.007 · Cited by 15
McGirr, C., Rooney, C., Gallagher, D., Dombrowski, S. U., Anderson, A. S., Cardwell, C. R., Free, C., Hoddinott, P., Holmes, V. A., McIntosh, E., et al. (2020) · Public Health Research · doi:10.3310/phr08040 · Cited by 15
Trinh, L., Kramer, A. F., Rowland, K. M., Strom, D. A., Wong, J. N., & McAuley, E. (2020) · Journal of Behavioral Medicine · doi:10.1007/s10865-020-00185-8 · Cited by 17
Feasibility and Pilot Studies: Small Steps before Giant Leaps
Story, D., Leslie, K., & French, C. (2018) · Anaesthesia and Intensive Care · doi:10.1177/0310057x1804600103 · Cited by 17
Murphy, M. J., Newby, J. M., Butow, P., Joubert, A., Kirsten, L., Shaw, J., Shepherd, H. L., & Andrews, G. (2021) · Internet Interventions · doi:10.1016/j.invent.2021.100449 · Cited by 15
“This study will inform the sample size for any future RCT (Leon et al., 2011).”
BackgroundDraper‐Rodi, J., Vogel, S. K., & Bishop, A. (2021) · Pilot and Feasibility Studies · doi:10.1186/s40814-021-00901-4 · Cited by 14
“75 for the behaviour) but caution would be required if these findings were used in a main trial due to limitations in using feasibility studies to provide precise between treatment group effect size estimates [1, 14, 40, 43].”
MethodAshford, J. B., & Gallagher, J. (2019) · Criminal Justice and Behavior · doi:10.1177/0093854819835277 · Cited by 15
Early, G. J., & Cheffer, N. D. (2019) · Hispanic Health Care International · doi:10.1177/1540415319836384 · Cited by 15
The Effects of Caffeine on Voice: A Systematic Review
Georgalas, V. L., Kalantzi, N., Harpur, I., & Kenny, C. (2021) · Journal of Voice · doi:10.1016/j.jvoice.2021.02.025 · Cited by 20
“It was agreed by the review team to include pilot studies at Level 5 of evidence, as pilot studies, much like “mechanism-based reasoning” studies, explore the feasibility of larger scale RCTs and summarize information for future hypothesis testing.(48)”
BackgroundMethodNicolson, G., Hayes, C. B., & Darker, C. D. (2021) · International Journal of Environmental Research and Public Health · doi:10.3390/ijerph18179292 · Cited by 16
“Inferential statistical tests were deemed not to be appropriate due to the exploratory nature of the feasibility trial [63].”
MethodStevens, E. R., Lei, L., Cleland, C. M., Vojjala, M., Shahawy, O. E., Berger, K. I., Kirchner, T., & Sherman, S. E. (2022) · Addiction Science & Clinical Practice · doi:10.1186/s13722-021-00284-0 · Cited by 14
“This study is not meant to provide a definitive test of intervention efficacy and will be used to inform future sample sizes for a larger trial [48, 49].”
MethodMace, R. A., Greenberg, J., Lemaster, N., Duarte, B., Penn, T., Kanaya, M., Doorley, J. D., Burris, J. L., Jacobs, C. A., & Vranceanu, A.-M. (2022) · JMIR Formative Research · doi:10.2196/34654 · Cited by 15
“The phase 2 quantitative results offer preliminary evidence that GetActive-OA is sensitive to population-specific needs and that the measures are sensitive to changes in key outcomes [98,99].”
BackgroundMethodFurlano, J. A., Horst, B., Petrella, R. J., Shoemaker, J. K., & Nagamatsu, L. S. (2023) · Canadian Journal of Diabetes · doi:10.1016/j.jcjd.2023.01.004 · Cited by 16
Bteddini, D. S., LeLaurin, J. H., Chi, X., Hall, J., Theis, R. P., Gurka, M. J., Lee, J., Mobley, E. M., Khalil, G. E., Polansky, C., et al. (2023) · Addictive Behaviors · doi:10.1016/j.addbeh.2023.107637 · Cited by 15
“First, the relatively small sample size does not allow for meaningful effect size estimates and the findings should be interpreted with caution (Leon, Davis, & Kraemer, 2011).”
Waring, M. E., Pagoto, S., Simas, T. A. M., Carr, L. T. B., Eamiello, M. L., Libby, B. A., Rudin, L. R., & Heersping, G. E. (2023) · JMIR mhealth and uhealth · doi:10.2196/41545 · Cited by 16
“As recommended [56,57], we based the sample size on necessities for examining feasibility.”
Chow, W. S. E. (2023) · Journal of Research in Special Educational Needs · doi:10.1111/1471-3802.12632 · Cited by 15
Salmoirago‐Blotcher, E., Wayne, P. M., Bock, B. C., DUNSIGER, S. I., Wu, W., Stabile, L. M., & Yeh, G. Y. (2015) · Contemporary Clinical Trials · doi:10.1016/j.cct.2015.06.020 · Cited by 16
“(79) We propose to enroll 60 patients in this study (30 per dose condition).”
BackgroundZavala-Yoé, R., Ramírez-Mendoza, R. A., & Cordero, L. M. (2016) · Journal of Integrative Neuroscience · doi:10.1142/s0219635216500138 · Cited by 15
Alcohol and disadvantaged men: A feasibility trial of an intervention delivered by mobile phone
Crombie, I. K., Irvine, L. A., Falconer, D. W., Williams, B., Ricketts, I. W., Jones, C., Humphris, G. M., Norrie, J. D., Slane, P. W., & Rice, P. M. (2017) · Drug and Alcohol Review · doi:10.1111/dar.12455 · Cited by 15
“Following the guidance of methodologists [31,62 – 64], this feasibility study did not attempt to estimate the effectiveness of the intervention.”
MethodMcCormick, S. A., McDonald, K. R., Vatter, S., Orgeta, V., Poliakoff, E., Smith, S. J., Silverdale, M., Fu, B., & Leroi, I. (2017) · BMJ Open · doi:10.1136/bmjopen-2017-016801 · Cited by 15
“23 If conducted in an unblinded, non-randomised manner, the outcomes, particularly for a complex psychosocial intervention such as is being tested here, may be quite different.”
Lama, J. R., Mayer, K. H., Perez‐Brumer, A., Huerta, L., Sánchez, H., Clark, J. L., Sánchez, J., & Reisner, S. L. (2019) · JMIR Research Protocols · doi:10.2196/14091 · Cited by 15
“Nevertheless, this study will play a key role in the development or refinement of a potential future intervention that we anticipate could be tested for efficacy in a later trial [47].”
Rogers, C. R., Okuyemi, K. S., Paskett, E. D., Thorpe, R. J., Rogers, T. N., Hung, M., Zickmund, S. L., Riley, C., & Fetters, M. D. (2019) · BMJ Open · doi:10.1136/bmjopen-2019-030000 · Cited by 14
“The intraclass correlation coefficient will be estimated from our study data and inflated (due to the expected downward bias) to estimate the necessary sample size for the full trial.(80)”
MethodGorgey, A. S., Abilmona, S. M., Sima, A. P., et al. (2019) · Spinal Cord · doi:10.1038/s41393-019-0364-3 · Cited by 15
Mevers, J. L., Call, N. A., Gerencser, K. R., Scheithauer, M. C., Miller, S. J., Muething, C., Hewett, S., McCracken, C., Scahill, L. D., & McElhanon, B. O. (2019) · Journal of Autism and Developmental Disorders · doi:10.1007/s10803-019-04305-5 · Cited by 15
Langdon, K. J., Ramsey, S. E., Scherzer, C. R., Carey, K. B., Ranney, M. L., & Rich, J. D. (2020) · Addiction Science & Clinical Practice · doi:10.1186/s13722-020-00189-4 · Cited by 15
Burrell, T. L., Sharp, W. G., Criado, K. K., Berry, R. C., Luevano, C., Khan, R., & Scahill, L. D. (2020) · Seminars in Pediatric Neurology · doi:10.1016/j.spen.2020.100830 · Cited by 15
Community-Engaged Research with Vietnamese Americans to Pilot-Test a Dementia Caregiver Intervention
Meyer, O. L., Sun, M., Do, T., Ho, J., Dinh, B.-T., Nguyen, S. N., & Hinton, L. (2020) · Journal of Cross-Cultural Gerontology · doi:10.1007/s10823-020-09410-y · Cited by 15
Pratscher, S. D., Mickle, A. M., Marks, J. G., Rocha, H. A., Bartsch, F., Schmidt, J., Tejera, L., Garcia, S., Custodero, C., Jean, F., et al. (2021) · Nutrients · doi:10.3390/nu13051556 · Cited by 15
Single case designs for early phase behavioral translational research in health psychology.
Epstein, L. H., Bickel, W. K., Czajkowski, S. M., Paluch, R. A., Moeyaert, M., & Davidson, K. W. (2021) · Health Psychology · doi:10.1037/hea0001055 · Cited by 17
Aschbrenner, K. A., Mueser, K. T., Naslund, J. A., Gorin, A. A., Zawacki, S. A., Pratt, S. I., Kinney, A., & Bartels, S. J. (2017) · Community Mental Health Journal · doi:10.1007/s10597-017-0100-4 · Cited by 19
“The number of participants is consistent with the intent of pilot studies as an initial step in exploring the feasibility and potential benefits of a novel intervention (Leon et al. 2011).”
ResultWalls, H., Liverani, M., Chheng, K., & Parkhurst, J. (2017) · Health Research Policy and Systems · doi:10.1186/s12961-017-0260-2 · Cited by 15
“of an intervention, or the feasibility of an intervention in a particular context when the effectiveness of that intervention may be context dependent [75].”
BackgroundEpigenetic Modifications Following Noxious Stimuli in Infants
Hatfield, L. A., Hoffman, R. K., Polomano, R. C., & Conley, Y. P. (2018) · Biological Research For Nursing · doi:10.1177/1099800417754141 · Cited by 15
“Sample size was based on pragmatic recruitment (number of infants the primary investigator [PI] was able to enroll in 1 year) and the necessity for examining recruitment feasibility (Leon et al., 2011).”
BackgroundMechanoreceptor profile of the lateral collateral ligament complex in the human elbow
Kholinne, E., Lee, H., Lee, Y., et al. (2018) · Asia-Pacific Journal of Sports Medicine Arthroscopy Rehabilitation and Technology · doi:10.1016/j.asmart.2018.04.001 · Cited by 15
Al-Eidi, S., Mohamed, A. G., Abutalib, R. A., et al. (2019) · Journal of Acupuncture and Meridian Studies · doi:10.1016/j.jams.2019.04.005 · Cited by 15
Shi, Y., Zheng, H., Zhou, S., Zheng, Q., Zhang, L., Xiao, X., Cao, W., Liu, Y., & Li, Y. (2019) · Trials · doi:10.1186/s13063-019-3433-1 · Cited by 15
“The inclusion of a control group allows for a more realistic examination of recruitment, randomization, implementation of interventions, blinded assessment procedures, and retention in blinded interventions [42].”
BackgroundKamody, R. C., Thurston, I. B., & Burton, E. T. (2020) · Eating Disorders · doi:10.1080/10640266.2020.1731055 · Cited by 16
A New Era of Clinical Research Methods in a Data-Rich Environment
Riley, W. T. (2016) · Elsevier eBooks · doi:10.1016/b978-0-12-802115-6.00018-5 · Cited by 15
Quantifying Dental Office-Originating Adverse Events: The Dental Practice Study Methods.
Tokede, O., Walji, M. F., Ramoni, R. B., et al. (2021) · PubMed · doi:10.1097/pts.0000000000000444 · Cited by 14
“Pilot studies, however, do not provide a meaningful effect size estimate for planning subsequent studies because of the imprecision inherent in data from small samples.(32) As such, these initial estimates are neither representative nor generalizable, and we refrain from overanalyzing our findings.”
ResultMetacognitive Therapy for Individuals at High Risk of Developing Psychosis: A Pilot Study
Parker, S., Mulligan, L. D., Milner, P., Bowe, S. E., & Palmier‐Claus, J. E. (2020) · Frontiers in Psychology · doi:10.3389/fpsyg.2019.02741 · Cited by 14
“However, in line with the aims of pilot studies, we were able to examine the feasibility of processes and procedures (i.e., recruitment, retention, implementation of MCT) in preparation for a larger RCT (Leon et al., 2011).”
ResultBurkhouse, K. L., Dao, A., Argiros, A. P., Granros, M., Cárdenas, E. F., Dickey, L., Feurer, C., Hill, K. E., Pegg, S. L., Venanzi, L., et al. (2023) · Behaviour Research and Therapy · doi:10.1016/j.brat.2023.104384 · Cited by 16
Impact of Career Counseling & Vocational Guidance on Employment in TVET Sector
Arshad, M., Tahir, A., Khan, M. M., et al. (2018) · International Journal of Human Resource Studies · doi:10.5296/ijhrs.v8i1.12554 · Cited by 14
“Furthermore, on the basis of results, the researchers can sense the possible anticipated problems, resulting the changes are made before the large-scale study is undertaken (Hundley and van Teijlingen, 2002; Kim, 2011; Leon et al., 2011).”
BackgroundBeech, A. N., Haworth, S. J., & Knepil, G. J. (2018) · British Journal of Oral and Maxillofacial Surgery · doi:10.1016/j.bjoms.2018.02.018 · Cited by 14
Leyton, F., Olhaberry, M., Alvarado, R., Rojas, G., Dueñas, L. A., Downing, G., & Steele, H. (2019) · Trials · doi:10.1186/s13063-019-3310-y · Cited by 13
Schmitz, L., Ferrari, N., Schwiertz, A., Rusch, K., Woestmann, U., Mahabir, E., & Gräf, C. (2019) · Pilot and Feasibility Studies · doi:10.1186/s40814-019-0459-9 · Cited by 15
Crits-Christoph, P. F., Goldstein, E., KING, C. A., Jordan, M., Thompson, D., Fisher, J., & Gibbons, M. B. C. (2020) · Behavior Therapy · doi:10.1016/j.beth.2020.01.008 · Cited by 16
Gentile, C., Starnino, L., Dupuis, G., & D’Antono, B. (2021) · Clinical Gerontologist · doi:10.1080/07317115.2021.1887421 · Cited by 14
Daughrity, B. (2021) · American Journal of Speech-Language Pathology · doi:10.1044/2021_ajslp-20-00196 · Cited by 14
Improving the Quality of Pilot/Feasibility Trials Reporting in Pediatric Psychology
Hilliard, M. E., Modi, A. C., & Palermo, T. M. (2021) · Journal of Pediatric Psychology · doi:10.1093/jpepsy/jsab056 · Cited by 14
Traeger, L. N., Styklunas, G. M., Park, E. Y., Lee, M. T., Fricchione, G. L., & Park, E. R. (2022) · The Gerontologist · doi:10.1093/geront/gnac031 · Cited by 16
Cancer and aging: Reflections for elders- Expressive writing intervention: A pilot study
Tutino, R. C., Saracino, R. M., Kelman, J., Schofield, E., Roth, A. J. L., & Nelson, C. J. (2022) · Journal of Geriatric Oncology · doi:10.1016/j.jgo.2022.02.009 · Cited by 15
Blumen, H., Ayers, E., Wang, C.-L., Ambrose, A., Jayakody, O., & Verghese, J. (2022) · Journal of Aging and Physical Activity · doi:10.1123/japa.2022-0176 · Cited by 13
A Motion Videogame for Opioid Relapse Prevention
Abroms, L. C., Leavitt, L. E., Alstyne, J. M. V., Schindler-Ruwisch, J. M., Fishman, M. J., & Greenberg, D. (2015) · Games for Health Journal · doi:10.1089/g4h.2014.0100 · Cited by 14
Bedrosian, R. C., Striegel‐Moore, R. H., & Wang, C. (2010) · International Journal of Eating Disorders · doi:10.1002/eat.20844 · Cited by 15
McMillen, J. C., Narendorf, S. C., Robinson, D., Havlicek, J. R., Fedoravicius, N., Bertram, J. E., & McNelly, D. (2015) · Child and Adolescent Psychiatry and Mental Health · doi:10.1186/s13034-015-0057-4 · Cited by 14
“feasibility, and not to use them to gauge efficacy or calculate effect sizes for a larger trial [32, 33].”
SALVAGING PSYCHOTHERAPY RESEARCH: A MANIFESTO
Coyne, J. C., & Kok, R. N. (2014) · University of Groningen research database (University of Groningen / Centre for Information Technology) · Cited by 16
Lisenby, K. M., Carroll, D. N., & Pinner, N. A. (2015) · Hospital Pharmacy · doi:10.1310/hpj5008-700 · Cited by 14
“Readmission within 30 days 26 (40) 12 (28) 0.”
Priming Patient Safety Through Nursing Handoff Communication: A Simulation Pilot Study
Groves, P. S., Bunch, J. L., Cram, E., Farag, A., Manges, K. A., Perkhounkova, Y., & Scott‐Cawiezell, J. (2016) · Western Journal of Nursing Research · doi:10.1177/0193945916673358 · Cited by 16
Improving Self-Efficacy and Self-Care in Adult Patients With a Urinary Diversion: A Pilot Study
Merandy, K., Morgan, M. A., Lee, R. E., Jr., et al. (2017) · Oncology nursing forum · doi:10.1188/17.onf.e90-e100 · Cited by 14
“Lack of a validated continent UD self-care skills evaluation tool and validated SE scale for patients with UD were further limitations; however, one of the functions of a pilot study is refinement of study tools (Leon et al., 2011).”
MethodKraft, S., Wolf, M., Klein, T., Becker, T. M., Bauer, S., & Puschner, B. (2017) · JMIR mhealth and uhealth · doi:10.2196/mhealth.7095 · Cited by 14
“According to recommendations for reporting results of pilot studies [26,27], reporting of results focuses on descriptive statistics, that is, mean and standard deviation for continuous variables, and frequencies and percentages for categorical variables.”
Hilgeman, M. M., Uphold, C. R., Collins, A. N., et al. (2018) · Journal of Gerontological Nursing · doi:10.3928/00989134-20180614-06 · Cited by 14
Self-citationBlackwell, S. E., Westermann, K., Woud, M. L., Cwik, J. C., Neher, T., Graz, C., Nyhuis, P. W., & Margraf, J. (2018) · Pilot and Feasibility Studies · doi:10.1186/s40814-018-0325-1 · Cited by 14
Reid, J. C., McCaskell, D. S., & Kho, M. E. (2019) · Pilot and Feasibility Studies · doi:10.1186/s40814-019-0509-3 · Cited by 14
Mace, R. A., Doorley, J. D., Popok, P., & Vranceanu, A. (2020) · JMIR Research Protocols · doi:10.2196/25351 · Cited by 14
“Consistent with guidelines for early feasibility studies [86,87] and the NIA Stage Model, our mixed method analysis will not assess efficacy [23].”
ResultCumbe, V., Muanido, A., Turner, M., Ramiro, I., Sherr, K., Weiner, B. J., Flaherty, B. P., Sharma, M., Faduque, F., Xerinda, E. R., et al. (2022) · Implementation Science · doi:10.1186/s13012-022-01213-8 · Cited by 15
“No confirmatory hypothesis testing driven by p value inference will be performed, as recommended when conducting exploratory studies by Leon (2011) [78].”
MethodReed, S. G., Manz, M. C., Snipe, S. M., et al. (2015) · Journal of Oral Science · doi:10.2334/josnusd.57.55 · Cited by 14
“Feasibility was assessed by recruitment, acceptance of the FMPE and SOBT, and assessment of correlation between the SOBT and FMPE periodontal measures in this sample of pregnant women (6).”
MethodMinen, M. T., Sahyoun, G., Gopal, A., Levitan, V., Pirraglia, E., Simon, N. M., & Halpern, A. (2020) · Headache The Journal of Head and Face Pain · doi:10.1111/head.13738 · Cited by 12
Designing Psychosocial Intervention Pilot Studies: A Tutorial for Palliative Care Investigators
Winger, J. G., Kelleher, S. A., Fisher, H. M., Somers, T. J., & Samsa, G. P. (2022) · Journal of Pain and Symptom Management · doi:10.1016/j.jpainsymman.2022.02.338 · Cited by 13
Contractor, A. A., Slavish, D. C., Thornton, J., & Weiss, N. H. (2022) · Journal of Psychotherapy Integration · doi:10.1037/int0000292 · Cited by 13
Thomas, T. H., Bender, C. M., Donovan, H. S., Murray, P. J., Taylor, S. E., Rosenzweig, M. Q., Sereika, S. M., Brufsky, A., & Schenker, Y. (2023) · Cancer · doi:10.1002/cncr.34887 · Cited by 13
O'Connor, L. C., Behar, S., Tarrant, S., Stamegna, P., Pretz, C., Wang, B., Savage, B., Scornavacca, T., Shirshac, J., Wilkie, T., et al. (2025) · BMC Digital Health · doi:10.1186/s44247-024-00142-4 · Cited by 13
Zavala-Yoé, R., & Ramírez-Mendoza, R. A. (2017) · Metabolic Brain Disease · doi:10.1007/s11011-017-0036-y · Cited by 13
Chugani, C. D., Fuhrman, B. J., Abebe, K. Z., Talis, J. M., Miller, E. P., & Coulter, R. W. S. (2020) · BMJ Open · doi:10.1136/bmjopen-2020-036833 · Cited by 13
Leffler, J. M., Junghans-Rutelonis, A. N., & McTate, E. (2020) · Evidence-Based Practice in Child and Adolescent Mental Health · doi:10.1080/23794925.2020.1759471 · Cited by 13
Larsen, S. E., Hunt, J. C., Geier, T. J., Heyrman, K., Schumann, N. R., Brandolino, A. M., Timmer‐Murillo, S. C., Bergner, C., Larson, C. L., & deRoon‐Cassini, T. A. (2020) · Journal of Traumatic Stress · doi:10.1002/jts.22580 · Cited by 15
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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
A language model drew the claims from the paper on 2026-09-28, under version 3 of the claim definition (model deepseek-v4.1-flash, prompt extract-v3).
A second model labelled each claim on 2026-09-28 (model jev-1.13-20260917, definition revision gold-guideline-v0.2).
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
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