Evidence map›Paper›PMID 37885599›Full record

ReviewPaediatrics & child health2023

Ensuring access to innovative therapies for children, adolescents, and young adults across Canada: The single patient study experience.

Gabriel Revon-Riviere, Leah C Young, Elizabeth A Stephenson, Kathy Brodeur-Robb, Sarah Cohen-Gogo, Rebecca Deyell, Thierry Lacaze-Masmonteil, Antonia Palmer, Rulan S Parekh, James A Whitlock and 1 more

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Paediatrics & child health, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
2.3field-weighted citation impact, top 12% of its field
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 4 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
4 · The record

Corrections and comments

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

5 · Who and what money

Authors and funding

11 authors at 4 institutions in 1 country.

Gabriel Revon-RiviereDepartment of Paediatrics, Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.ORCID https://orcid.org/0000-0001-6512-1004
Leah C YoungC17 Council, University of Alberta, Edmonton, Alberta, Canada.
Elizabeth A StephensonDepartment of Paediatrics, Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.
Kathy Brodeur-RobbC17 Council, University of Alberta, Edmonton, Alberta, Canada.
Sarah Cohen-GogoDepartment of Paediatrics, Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.ORCID https://orcid.org/0000-0002-8852-1104
Rebecca DeyellDepartment of Paediatrics, BC Children's Hospital, Vancouver, British Columbia, Canada.
Thierry Lacaze-MasmonteilDepartment of Paediatrics, Cumming School of Medicine and University of Calgary, Calgary, Alberta, Canada.
Antonia PalmerKindred Foundation, Oakville, Ontario, Canada.
Rulan S ParekhDepartment of Paediatrics, Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.
James A WhitlockDepartment of Paediatrics, Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.
Daniel A MorgensternDepartment of Paediatrics, Hospital for Sick Children and University of Toronto, Toronto, Ontario, Canada.ORCID https://orcid.org/0000-0002-4859-1108
University of Toronto · CAUniversity of Alberta · CABC Children's Hospital · CAUniversity of Calgary · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Innovative therapeutic approaches are needed to alleviate the burden of life-limiting, rare, and chronic conditions affecting children, adolescents, and young adults (CAYA). This includes a need for improved access to both clinical research and to non-approved or off-label therapies, together with, ultimately, more therapies achieving regulatory approval in Canada. The single patient study (SPS), also known as an open label individual patient (OLIP) study, was introduced by Health Canada to open access to non-marketed drugs where a clinical trial is not readily available, but the drug is considered too investigational to be managed on a standard Special Access Program. SPS is designed for patients who have a serious or life-threatening condition and have exhausted available treatment options. Our report summarizes this relatively new development in the Canadian regulatory environment and highlights the opportunities and challenges as identified by regulators, pharmaceutical representatives, academic researchers, and patient/parent advocates.

Indexed as

BioethicsClinical trialHealth policy

Identifiers

PMID37885599
PMCPMC10599489
OpenAlexW4366495712

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.