Ultra-Rare Disease Health Technology Assessments in Brazil - Dataset and Data Dictionary
Description
This dataset supports a retrospective observational study of all health technology assessment (HTA) reports for ultra-rare disease drugs issued by Brazil's National Commission for Health Technology Incorporation (Conitec) between 2011 and December 2025. The unit of analysis is the drug–indication pair; 63 reports covering 55 drugs across 36 ultra-rare diseases met eligibility criteria. Data were extracted in duplicate from publicly available Conitec reports using a standardized spreadsheet and data dictionary. Variables cover disease and technology characteristics, clinical evidence, economic evaluation, and appraisal process. The primary outcome was the final Conitec recommendation, classified as favorable, unfavorable, or reconsidered after public consultation. File 1 — conitec_2026 (source screening database) Complete panel of Conitec health technology assessment reports (N = 1,180) covering all technologies evaluated by Conitec, used as the source database from which ultra-rare disease drug reports were identified and screened for eligibility, as described in the Methods (Study design, eligibility criteria and data sources). File 2 — extraction_spreadsheet_DUR (extracted data) The analytic dataset extracted in duplicate from the 63 eligible Conitec reports, one row per drug–indication pair, covering all variables across the six domains described in the Methods (disease and technology characteristics, report characteristics, clinical evidence, economic evaluation, appraisal recommendation, and process characteristics). File 3 — dataset_DUR (data dictionary) Data dictionary defining each variable in the extraction spreadsheet: column letter, domain/category, variable name, description, possible values, coding criteria, and definition of missing-data codes (NA/NE) used during extraction. File 4 — analysis_script_DUR (statistical analysis code) R script (R version 4.6.0) used to reproduce all statistical analyses and figures reported in the manuscript, including normality testing, group comparisons, post-hoc pairwise comparisons, crude odds ratio calculations, and all publication figures.
Files
Institutions
- Instituto Nacional de CardiologiaRio de Janeiro, Rio de Janeiro