ArticleThe patient2018
Engagement of Canadian Patients with Rare Diseases and Their Families in the Lifecycle of Therapy: A Qualitative Study.
Article in The patient, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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
4 citing papers in PubMed.
- How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis.Orphanet journal of rare diseases · 2023Review
- Practical Considerations in Using Online Modified-Delphi Approaches to Engage Patients and Other Stakeholders in Clinical Practice Guideline Development.The patient · 2020Article
- A checklist for managed access programmes for reimbursement co-designed by Canadian patients and caregivers.Health expectations : an international journal of public participation in health care and health policy · 2018Article
- Patient Involvement in the Lifecycle of Medicines According to Belgian Stakeholders: The Gap Between Theory and Practice.Frontiers in medicine · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
introductionPatient involvement is increasingly recognized as critical to the development, introduction and use (i.e. the lifecycle) of new and effective therapies, particularly those for rare diseases, where natural histories and the impact on patients and families are less well-understood than for common diseases. However, little is known about how patients and families would like to be involved during the lifecycle.
objectiveThe aim of this study was to explore ways in which Canadian patients with rare diseases and their families would like to be involved in the lifecycle of therapies and identify their priorities for involvement.
methodsPatients with rare diseases and their families were recruited to participate in two deliberative sessions, during which concepts related to decision-making uncertainty and the technology lifecycle were introduced before eliciting input around ways in which they could be involved. This was followed by a webinar, which was used to further identify opportunities for involvement. The data were then analyzed qualitatively using eclectic coding.
resultsPatients and families identified opportunities that fell into three goals: (1) incorporation of their 'lived experience' in coverage decision making (i.e. decisions by governments on funding new therapies); (2) improved care for patients; and (3) greater awareness of rare diseases, with the first being a priority.
conclusionsOpportunities for patients and families to contribute their 'lived experience' are needed throughout the orphan drug lifecycle, but the ideal mechanisms for providing this input have yet to be determined.
Indexed as
Identifiers
29299833What OpenQuestion holds
Registered trials
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.