Analysis of pilot and early phase studies with small sample sizes

Shih, Weichung J.; Ohman‐Strickland, Pamela; Lin, Yong

doi:10.1002/sim.1807

Cited by 35 publications

(27 citation statements)

References 14 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In particular, testing of a null hypothesis as advocated by statisticians and scientists who conduct clinical trials is almost never the correct method for reaching a triage conclusion. Shih et al 5 have pointed out that one alternative is to replace the conventional test of a difference-between-groups with a procedure that focuses on whether there is evidence that any particular patient or group of patients benefits from a treatment modality. While such an approach is frowned on in phase III studies, it seems eminently sensible for phase I studies.…”

Section: The Question Of Whether or Not To Proceedmentioning

confidence: 99%

The Importance of Early Phase Research

Aickin

2007

The Journal of Alternative and Complementary Medicine

View full text Add to dashboard Cite

Early phase research in complementary and alternative medicine (CAM) is especially important because of the large influence it has on the subsequent expenditure of money and effort in particular areas of CAM therapies. The culture of biomedical research has, however, blurred the distinction between early and late-phase research, and this has distorted the very distinct aims of these two activities. The purpose of this paper is to reaffirm the critical role of early phase CAM research and to encourage CAM researchers to value and conduct early phase studies for their proper purposes.

show abstract

Section: The Question Of Whether or Not To Proceedmentioning

confidence: 99%

The Importance of Early Phase Research

Aickin

2007

The Journal of Alternative and Complementary Medicine

View full text Add to dashboard Cite

show abstract

“…In this respect, the situation resembles early-phase clinical trials in humans. Transferring ideas from this field (e.g., Shih et al 2004) into standardized animal screening might further help to improve the yield of the existing and future phenotyping resource(s) (e.g., Brown et al 2005).…”

Section: Discussionmentioning

confidence: 99%

Power matters in closing the phenotyping gap

Meyer

Elvert

Scherag

et al. 2007

Naturwissenschaften

View full text Add to dashboard Cite

Much of our understanding of physiology and metabolism is derived from investigating mouse mutants and transgenic mice, and open-access platforms for standardized mouse phenotyping such as the German Mouse Clinic (GMC) are currently viewed as one powerful tool for identifying novel gene-function relationships. Phenotyping or phenotypic screening involves the comparison of wild-type control mice with their mutant or transgenic littermates. In our study, we explored the extent to which standardized phenotyping will succeed in detecting biologically relevant phenotypic differences in mice generated and provided by different collaborators. We analyzed quantitative metabolic data (body mass, energy intake, and energy metabolized) collected at the GMC under the current workflow, and used them for statistical power considerations. Our results demonstrate that there is substantial variability in these parameters among lines of wild-type C57BL/6 (B6) mice from different sources. Given this variable background noise in mice that serve as controls, subtle phenotypes in mutant or transgenic littermates may be overlooked. Furthermore, a phenotype observed in one cohort of a mutant line may not be reproducible (to the same extent) in mice coming from a different environment or supplier. In the light of these constraints, we encourage researchers to incorporate information on intrastrain variability into future study planning, or to perform advanced hierarchical analyses. Both will ultimately improve the detectability of novel phenotypes by phenotypic screening.

show abstract

“…As this is a feasibility study, formal power calculations are not appropriate, as the study is not designed to test for a difference between treatments. Recommendations for feasibility studies propose that the analysis dataset comprises a minimum of 30 participants for each arm in order to estimate parameters for future sample size calculations [32-34]. We anticipate that the combined total of non-compliance and loss to follow up will be no more than 35% (that is, 25% due to loss to follow up and 10% due to non-compliance) and therefore, aim to recruit 46 patients in each arm, to account for potential missing data.…”

Section: Methodsmentioning

confidence: 99%

Testing the credibility, feasibility and acceptability of an optimised behavioural intervention (OBI) for avoidant chronic low back pain patients: protocol for a randomised feasibility study

Pincus

Anwar

McCracken

et al. 2013

Trials

View full text Add to dashboard Cite

BackgroundChronic back pain continues to be a costly and prevalent condition. The latest NICE guidelines issued in 2009 state that for patients with persistent back pain (of between six weeks and twelve months duration), who are highly distressed and/or disabled and for whom exercise, manual therapy and acupuncture has not been beneficial, the evidence supports a combination of around 100 hours of combined physical and psychological treatment. This is costly, and may prove unacceptable to many patients. A key recommendation of these guidelines was for further randomised controlled trials (RCTs) of psychological treatment and to target treatment to specific sub-groups of patients. Recent trials that have included psychological interventions have shown only moderate improvement at best, and results are not maintained long term. There is therefore a need to test theoretically driven interventions that focus on specific high-risk sub-groups, in which the intervention is delivered at full integrity against a credible control.Methods/designA feasibility study of a pragmatic randomised controlled trial comparing psychologist-delivered Contextual Cognitive Behavioural Therapy (CCBT) against Treatment As Usual (TAU) physiotherapy delivered by physiotherapists for the treatment of chronic lower back pain in ‘avoidant’ patients. Ninety-two patients referred for physiotherapy will be recruited and randomised on a 1:1 basis to receive CCBT or TAU. Treatment groups will be balanced by centre and pain interference score. Primary outcomes include assessing the credibility and acceptability of the intervention, and to demonstrate proof of principle through a greater change in pain acceptance in the CCBT arm, measured by the Acceptance and Action –II and the Chronic Pain Acceptance questionnaires. In addition, the feasibility of carrying out a full trial will be explored with reference to recruitment and follow-up rates including the assessment of the burden of outcome measure completion. Secondary patient outcomes include disability, pain, fear of movement, mood, quality of life, and global recovery. Outcomes are measured at three and six months post-randomisation.DiscussionThis paper details the rationale, design, therapist training system and recruitment methods to be used in a feasibility study which will inform the design and efficient implementation of a future definitive RCT.Trial registrationISRCTN43733490

show abstract

Analysis of pilot and early phase studies with small sample sizes

Cited by 35 publications

References 14 publications

The Importance of Early Phase Research

The Importance of Early Phase Research

Power matters in closing the phenotyping gap

Testing the credibility, feasibility and acceptability of an optimised behavioural intervention (OBI) for avoidant chronic low back pain patients: protocol for a randomised feasibility study

Contact Info

Product

Resources

About