OBJECTIVES: To understand the impact accelerated regulatory approval (RA) has on the timelines of Health Technology Assessment (HTA) of orphan drugs (ODs) across four markets (Canada, England, France, and Germany).

METHODS: ODs in receipt of accelerated RA in the four markets from May 2018 to May 2023 were reviewed. A targeted research analysis focusing on time taken to reach a final HTA decision was conducted. Key outcomes and willingness to pay threshold applied to assess cost-effectiveness were also analyzed.

RESULTS: Five ODs (Tegsedi, Verkazia, Onpattro, Evrysdi and Takhzyro) were identified. Verkazia was submitted for HTA in Canada and France, the other ODs were submitted in all analyzed markets.

The median time to final HTA decision in England was similar to the overall median time reported in literature (277 days* vs 266 days). Canada and France showed shorter timelines (Canada: 183 days^† vs 216 days; France: 92 days^‡ vs 155 days). Germany showed longer timelines (305 days^§ vs 170 days); however, new ODs are immediately reimbursed following RA as long as annual revenue is maintained below threshold, possibly impacting HTA resolution timelines.

All ODs assessed were reimbursed, some under agreements (e.g., Evrysdi through a managed entry agreement in England, and on a per patient basis in France; Takhzyro in a specific sub-population in France).

The ICER thresholds for these ODs in Canada and England were in line with other drugs that received RA via the conventional non-accelerated route.

CONCLUSIONS: ODs granted accelerated RA may have faster HTA outcomes in Canada and France, while this status does not seem to impact HTA in England and Germany. No relationship was found between accelerated approval and the applied threshold for willingness to pay.

*From evaluation consultation to recommendation. ^†From submission to recommendation. ^‡From first HTA session to recommendation. ^§From start of procedure to resolution.