We need a "made in Canada" orphan drug framework.

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

5 citing papers in PubMed.

1. Ensuring access to innovative therapies for children, adolescents, and young adults across Canada: The single patient study experience.Paediatrics & child health · 2023
   Review
2. Clinical characteristics of patients from Quebec, Canada, with Morquio A syndrome: a longitudinal observational study.Orphanet journal of rare diseases · 2020
   Observational
3. When are Pharmaceuticals Priced Fairly? An Alternative Risk-Sharing Model for Pharmaceutical Pricing.Health care analysis : HCA : journal of health philosophy and policy · 2020
   Article
4. A compilation of national plans, policies and government actions for rare diseases in 23 countries.Intractable & rare diseases research · 2018
   Review
5. Access to new drugs for rare disorders in Canada.CMAJ : Canadian Medical Association journal = journal de l'Association medicale canadienne · 2018
   Article

Corrections and comments

• Commented on by
  Access to new drugs for rare disorders in Canada.2018

Authors and funding

2 authors.

Funding

No grant is acknowledged in the PubMed record.

Abstract

PubMed holds no abstract for this paper.

Indexed as