Final report of the Oslo Medicines Initiative. Improving access to novel, high-priced medicines in the WHO European Region

The market for pharmaceutical products has changed considerably in recent years, from a blockbuster model that targeted high volumes of patients, to therapies that are targeted to be more effective but for smaller patient groups affected by serious, often rare, low-prevalence diseases that require complex treatments.

Such medicines – advanced therapy medicinal products (ATMPs) and cell and gene therapies (CGTs) – are welcomed by patients, but they often come with challenges.

They are disruptive to health-care systems and are associated with significantly higher prices per product – often over US$  1  million per patient – despite uncertainties over longer-term risks and benefits. 

Provisions for early access for patients with otherwise incurable diseases, such as conditional marketing authorizations, also means that there are often major uncertainties about their effectiveness due to the immature evidence base.

Executive summary:
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