Ethical Drug Development for Rare Childhood Diseases: When There Are Limited But Promising Data in Adults, How to Choose Between Safety or Efficacy Studies?

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

Who cites it

2 citing papers in PubMed.

1. Flexibility Required: Balancing the Interests of Children and Risk in Drug Development for Rare Pediatric Conditions.The American journal of bioethics : AJOB · 2020
   Article
2. Clearing Muddy Waters: The Need to Reconceptualize Minor Increase over Minimal Risk in Pediatric Rare Disease Research.The American journal of bioethics : AJOB · 2020
   Article

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

Authors and funding

3 authors.

Funding

Abstract

PubMed holds no abstract for this paper.

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