N-of-1 Trials: Not Just for Academics

Federman, Daniel G.; Shelling, Michael L.; Kirsner, Robert S.

doi:10.1007/s11606-010-1554-z

Cited by 4 publications

(3 citation statements)

References 2 publications

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“…For example, one possibility is the “ n ‐of‐1” design, a form of the crossover design study, in which each patient serves as his or her own control by switching back and forth between the drug and a control (eg, placebo) or different dose levels of the drug (eg, low dose and high dose) in pre‐defined treatment periods . Results of a series of n ‐of‐1 trials in multiple patients may then be combined in order to better understand the impact of treatment . The feasibility of such an approach in NBD is unknown.…”

Section: Discussionmentioning

confidence: 99%

Drug development for pediatric neurogenic bladder dysfunction: Dosing, endpoints, and study design

Momper

Karesh

Green

et al. 2014

The Journal of Clinical Pharma

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Pediatric drug development is challenging when a product is studied for a pediatric disease that has a different underlying etiology and pathophysiology compared to the adult disease. Neurogenic bladder dysfunction (NBD) is such a therapeutic area with multiple unsuccessful development programs. The objective of this study was to critically evaluate clinical trial design elements that may have contributed to unsuccessful drug development programs for pediatric NBD. Trial design elements of drugs tested for pediatric NBD were identified from trials submitted to the U.S. Food and Drug Administration. Data were extracted from publically available FDA reviews and labeling and included trial design, primary endpoints, enrollment eligibilities, and pharmacokinetic data. A total of four products were identified. Although all four programs potentially provided clinically useful information, only one drug (oxybutynin) demonstrated efficacy in children with NBD. The lack of demonstrable efficacy for the remainder of the products illustrates that future trials should give careful attention to testing a range of doses, using objectively measured, clinically meaningful endpoints, and selecting clinical trial designs that are both interpretable and feasible. Compiling the drug development experience with pediatric NBD will facilitate an improved approach for future drug development for this, and perhaps other, therapeutic areas.

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Section: Discussionmentioning

confidence: 99%

Drug development for pediatric neurogenic bladder dysfunction: Dosing, endpoints, and study design

Momper

Karesh

Green

et al. 2014

The Journal of Clinical Pharma

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“…The placebo effect also underscores a role for diligent patient examination to corroborate symptom changes with objective data 13 . A definitive solution is to undertake more placebo controlled treatments on individual patients in daily practice, and yet such trials are underutilized even among advocates [14][15][16][17] .…”

Section: Placebo Effectsmentioning

confidence: 99%

Determining Whether a Patient is Feeling Better: Pitfalls from the Science of Human Perception

Redelmeier

Dickinson

2011

J GEN INTERN MED

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Human perception is fallible and may lead patients to be inaccurate when judging whether their symptoms are improving with treatment. This article provides a narrative review of studies in psychology that describe misconceptions related to a patient's comprehension, recall, evaluation and expression. The specific misconceptions include the power of suggestion (placebo effects), desire for peace-of-mind (cognitive dissonance reduction), inconsistent standards (loss aversion), a flawed sense of time (duration neglect), limited perception (measurement error), declining sensitivity (Weber's law), an eagerness to please (social desirability bias), and subtle affirmation (personal control). An awareness of specific pitfalls might help clinicians avoid some mistakes when providing follow-up and interpreting changes in patient symptoms.

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“…In addition to drug holidays, n‐of‐1 treatment trials might also be considered in cases where the effects of treatment are difficult to distinguish from the natural history of waxing and waning headaches, or where disease progression or placebo effects seem possible. With n‐of‐1 trials, periods of active drug administration are interspersed with periods during which an identical or similar‐appearing inert pill is given, so that the patient is uncertain when active drug is being used 42 . Careful records of treatment response are kept, and compared with use of active and inert medications in order to distinguish the effects of natural history, placebo, and true drug effect.…”

mentioning

confidence: 99%

Tolerance and Loss of Beneficial Effect During Migraine Prophylaxis: Clinical Considerations

Loder¹,

Rizzoli

2011

Headache: The Journal of Head and Face Pain

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A familiar situation in migraine treatment is the patient with an initial positive response to prophylactic drug therapy who later experiences relapse. The goals of this paper are to provide a theoretical framework to help doctors think about this problem, to evaluate factors and response patterns that may be associated with different causes of relapse, and to suggest clinical strategies that may aid in its management. Six key explanations for loss of benefit from prophylactic therapy are: (1) pharmacokinetic, pharmacodynamic, and behavioral drug tolerance; (2) non-specific or placebo effects; (3) natural variability in disease activity; (4) disease progression; (5) inaccurate recall of treatment effects; and (6) drug delivery problems. Current options for patients who experience loss of benefit from prophylactic therapy include traditional techniques such as switching, re-trying, rotating, or combining drugs. Selected behavioral and environmental treatment techniques might also be useful. We describe a practical, structured approach to evaluation and management of relapse with migraine prophylaxis.

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N-of-1 Trials: Not Just for Academics

Cited by 4 publications

References 2 publications

Drug development for pediatric neurogenic bladder dysfunction: Dosing, endpoints, and study design

Drug development for pediatric neurogenic bladder dysfunction: Dosing, endpoints, and study design

Determining Whether a Patient is Feeling Better: Pitfalls from the Science of Human Perception

Tolerance and Loss of Beneficial Effect During Migraine Prophylaxis: Clinical Considerations

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