OBJECTIVES: The accelerated approval of orphan drugs is based on public health concern and therapeutic innovation. While these drugs should be equitably accessible to everyone, Latin America (LATAM) patients often face higher access struggles than European patients. This study aims to compare the time from marketing authorization until health technology assessment (HTA) decision and coverage restrictions, identifying the related equity gap, for these drugs in these regions.

METHODS: We identified orphan drugs with accelerated assessment approval by the European Medicines Agency (EMA) and Medicines and Healthcare Products Regulatory Agency in the last five years. The marketing authorization dates and HTA decisions and dates were collected for Germany, Sweden, the UK, Brazil and Chile. The median time from marketing authorization to HTA decision was calculated.

RESULTS: Over the 5-year period, nine orphan drugs received accelerated approval by the EMA. Of these, only four and six received marketing authorization in Chile and Brazil, respectively. One additional drug received approval in the UK but not in the other countries, which was excluded from the research. In total, 24 HTAs were identified (Europe: 19, LATAM: 5). Differences were observed in coverage scope, with higher restrictions in LATAM. Additionally, the median time from marketing authorization until HTA decision in Europe was 10.75 months (Germany: 6.85 months, Sweden: 15.84 months, UK: 11.54 months), compared with 20.39 months in LATAM (Brazil: 20.39 months, Chile: decisions not available). This represents an equity gap factor of 1.90 when comparing time to HTA decision between regions.

CONCLUSIONS: There is a considerable inequity gap in the HTA of orphan drugs that represents a public health concern and therapeutic innovation between Europe and LATAM, which is further increased by differences in launch sequences. Policymakers should work closely with manufacturers to increase the attractiveness of the LATAM market without altering the financial stability of the region.