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Critical appraisal of arguments for the delayed-start design proposed as alternative to the parallel-group randomized clinical trial design in the field of rare disease

Overview of attention for article published in Orphanet Journal of Rare Diseases, August 2017
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Title
Critical appraisal of arguments for the delayed-start design proposed as alternative to the parallel-group randomized clinical trial design in the field of rare disease
Published in
Orphanet Journal of Rare Diseases, August 2017
DOI 10.1186/s13023-017-0692-3
Pubmed ID
Authors

Loukia M. Spineli, Eva Jenz, Anika Großhennig, Armin Koch, Loukia M. Spineli, Eva Jenz, Anika Großhennig, Armin Koch

Abstract

A number of papers have proposed or evaluated the delayed-start design as an alternative to the standard two-arm parallel group randomized clinical trial (RCT) design in the field of rare disease. However the discussion is felt to lack a sufficient degree of consideration devoted to the true virtues of the delayed start design and the implications either in terms of required sample-size, overall information, or interpretation of the estimate in the context of small populations. To evaluate whether there are real advantages of the delayed-start design particularly in terms of overall efficacy and sample size requirements as a proposed alternative to the standard parallel group RCT in the field of rare disease. We used a real-life example to compare the delayed-start design with the standard RCT in terms of sample size requirements. Then, based on three scenarios regarding the development of the treatment effect over time, the advantages, limitations and potential costs of the delayed-start design are discussed. We clarify that delayed-start design is not suitable for drugs that establish an immediate treatment effect, but for drugs with effects developing over time, instead. In addition, the sample size will always increase as an implication for a reduced time on placebo resulting in a decreased treatment effect. A number of papers have repeated well-known arguments to justify the delayed-start design as appropriate alternative to the standard parallel group RCT in the field of rare disease and do not discuss the specific needs of research methodology in this field. The main point is that a limited time on placebo will result in an underestimated treatment effect and, in consequence, in larger sample size requirements compared to those expected under a standard parallel-group design. This also impacts on benefit-risk assessment.

Twitter Demographics

The data shown below were collected from the profile of 1 tweeter who shared this research output. Click here to find out more about how the information was compiled.

Mendeley readers

The data shown below were compiled from readership statistics for 40 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 40 100%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 8 20%
Researcher 8 20%
Student > Master 5 13%
Student > Bachelor 4 10%
Other 3 8%
Other 8 20%
Unknown 4 10%
Readers by discipline Count As %
Medicine and Dentistry 11 28%
Mathematics 5 13%
Social Sciences 5 13%
Psychology 3 8%
Nursing and Health Professions 2 5%
Other 8 20%
Unknown 6 15%

Attention Score in Context

This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 18 August 2017.
All research outputs
#9,297,600
of 11,626,228 outputs
Outputs from Orphanet Journal of Rare Diseases
#1,069
of 1,266 outputs
Outputs of similar age
#193,567
of 264,243 outputs
Outputs of similar age from Orphanet Journal of Rare Diseases
#17
of 21 outputs
Altmetric has tracked 11,626,228 research outputs across all sources so far. This one is in the 11th percentile – i.e., 11% of other outputs scored the same or lower than it.
So far Altmetric has tracked 1,266 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 6.8. This one is in the 8th percentile – i.e., 8% of its peers scored the same or lower than it.
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We're also able to compare this research output to 21 others from the same source and published within six weeks on either side of this one. This one is in the 1st percentile – i.e., 1% of its contemporaries scored the same or lower than it.