OBJECTIVES: To understand the frequency of cases where products are not launched in international markets following FDA approval and identify potential rationale driving the decisions.

METHODS: We evaluated specialty medicines approved by the FDA between 2019 and July 2023. The list was matched against regional / local regulatory and access authority decisions in a set of to identify any medicines that are not launched in some regions.

RESULTS: For 191 unique US FDA approved branded specialty medicines, following observations were made:

1. 102 products have been approved by the EMA post FDA approval out of which 12 have not been filed for reimbursement in FRA and DEU
   1. Manufacturers did not seek reimbursement for 21 products in the GBR
   2. Out of 86 EMA approved products which sought reimbursement, 11 withdrew from DEU (such as Rybrevant, Tabrecta) mostly due to challenges in price negotiations
2. Remaining products are either in the process of being evaluated by the EMA (N=15), had been approved by the EMA before FDA (N=20), are in the trial stage (N=25), have been abandoned (N=8) or have not yet sought EMA submission / approval (N=20)
3. 58 products have been approved in AUS out of which 19 have not sought reimbursement
4. 69 products have been approved in CAN out of which 8 have not sought reimbursement

CONCLUSIONS: This assessment identifies the key clinical and economic reasons behind the gaps in commercialized medicines in international markets vs. the US. Alignment between global payer communities and pharmaceutical manufacturers on evidentiary expectations and potential novel methods of generating evidence can accelerate access to innovative medicines globally.