Early Patient Access to Life Saving Drugs across Europe – A Review of the Characteristics and Availability of Drugs in Early Access Programs in EU4 and the UK

OBJECTIVES: To compare early access program (EAP) eligibility criteria, requirements, and drug availability across France, Germany, Italy, Spain, and the United Kingdom (UK).

METHODS: Information on EAPs and the availability of drugs in cohort-based EAPs was obtained from the websites of national regulatory and Health Technology Assessment (HTA) authorities in January 2024. Off-label use programs were outside the scope of this study.

RESULTS: EAPs are established in all assessed countries, with Italy having the highest number of schemes (n=4). Each country offers at least one cohort-based or mixed (cohort-based and named patient) EAP and one named patient EAP. Key eligibility criteria for most EAPs include serious, rare, debilitating diseases and innovative/advanced therapies. In 3 out of 5 countries, cohort-based programs are initiated by the manufacturer, while named patient programs are always initiated by clinicians or pharmacists. Formal dossier submission is a key requirement for 5 out of 12 EAPs in France, Italy, and the UK. Additionally, drugs included in 7 out of 12 EAPs across all countries are reimbursed. The number of available drugs significantly varies across countries. For instance, the cohort-based program in the UK currently includes only one drug, whereas France has a much broader coverage with 65 drugs included in their cohort-based program. In all cohort-based EAPs, oncology was the most represented disease area, with the percentage of included drugs per scheme ranging from 21% to 100%.

CONCLUSIONS: EAPs play a crucial role in ensuring early patient access to life-saving drugs. However, requirements and, therefore, drug availability were found to be differing between the countries. The importance of EAPs for equal patient access should be considered more widely by manufacturers, healthcare professionals, regulatory, and HTA authorities.