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ISPOR 2022: Comparing And Contrasting Early Access Opportunities Across The EU4 And UK

12/10/2022

OBJECTIVES 

Early access programs (EAPs) have clear benefits for patients, healthcare providers, payers and pharmaceutical companies. However, manufacturers can struggle to identify EAPs and understand the opportunities that would best complement their pricing and reimbursement (P&R) strategies. We have scoped EAPs across the EU4 and the United Kingdom (UK) to establish the eligibility criteria and timelines, identify resource requirements and evidence generation possibilities, and ultimately assess the opportunity for manufacturers.

METHODS 

EAPs available in the EU4 and UK were identified through secondary research; extraordinary single patient requests were excluded as deemed not commercial opportunities. Eligibility criteria, resource requirements, evidence generation possibilities and timelines were extracted for each EAP. The relative opportunity value of each EAP for manufacturers was evaluated.

RESULTS 

Nine early access programs were identified across EU4 and UK. Of these, France, Germany, Italy and Spain all had two program options and the UK had one. The programs identified all provided compassionate use for a cohort of patients. Fundamental to all schemes was the condition that the therapy is to treat a debilitating or life-threatening disease and met a therapeutic need, although innovation was not a criterion for all identified programs. Both France’s Early access authorisation (AAP) and UK’s Early Access Medicines Scheme (EAMS) provided data collection opportunities that could be utilised in their respective HTA bodies’ assessment procedures. Additionally, only France’s AAP and Italy’s 648 program offered paid Early Access for manufacturers.

CONCLUSION  

There are multiple opportunities for EAPs for manufacturers across the EU4 and the UK. Through undertaking this analysis, we have been able to populate a framework for early access opportunities, showing variation in qualifying criteria and evidence collection possibilities. The limited number of EAPs that offer paid access could significantly limit the number of manufacturers able to provide early access to potentially life-saving medicines for patients.

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