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Chapter 4 · Access denied

Real launches. Real waits.

Celebrated medicines can still take years to reach patients in some markets. These are documented, public timelines, presented as fact, not judgment.

Cystic fibrosis

A breakthrough therapy, reaching markets years apart

Oct 2019Approved in the United States
Nov 2021Reimbursed access agreed in Spain
Apr 2022Reimbursed access agreed in Australia
Jun 2024A durable long term access deal in England, nearly five years after approval

Marketed in Europe under a different brand name. Sources: FiercePharma, 2024; Vertex company statements.

Spinal muscular atrophy

A one time gene therapy, reaching patients unevenly

One of the most talked about gene therapies of recent years gained regulatory approval across roughly 37 European countries, yet only about 32 established a reimbursement pathway. In some markets, early access ran through managed programs while price and reimbursement were still being negotiated.

Sources: company disclosures and market access reporting, 2024 to 2025.

The common thread

Approval is not access. The days between the two are real, they differ sharply by market, and they are rarely measured on purpose. Across Europe the median wait sits at 532 days.

EFPIA W.A.I.T. 2025
Final chapter

If we could measure this together, we could improve it

Why TFPA