A pipeline for patient groups too small to analyse
The setting
Methods in population health assume enough people to average over. When a disease affects a handful of people in a country, the statistics built for thousands stop working, and the usual answer is that the question cannot be asked.
What we did
Testing what can still be learned from a cohort that small, starting with propionic acidemia in Saudi Arabia.
What exists now
Work in progress. There is no result yet and nothing has been published.
What it does not do
- It produces no diagnosis, no prognosis and no output about an individual patient.
- It is a methodological question about what small-cohort analysis can support, not a tool anyone uses in care.
What has not been established
Everything. No method has been validated, no result has been produced, and nothing has been peer reviewed.
Where it goes next
The question is which inferences a cohort of that size can actually support, and which ones only look supported because the method was borrowed from a larger population.
What we are looking for
Collaborators with rare disease cohorts, and statisticians interested in what is inferable at that scale.
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