LAUSR

Introduction: Several early access schemes (EAS) exist, which aim to accelerate patient access to new, potentially life-saving therapies. While some information exists on key schemes and their modalities, the determinants that drive adoption of a new medicine under an EAS remain unclear. We aimed to map eligibility criteria for inclusion of new medicines into the different EAS available across countries. Methods: Health technology assessment (HTA) stakeholders across 23 countries globally were invited via email to complete a web-survey with questions on (i) items that define product eligibility for EAS designation, (ii) standards for minimum level of evidence, monitoring, and additional evidence generation for early access products, and (iii) funding arrangements for these products across settings and types of schemes. Anonymized responses were analysed using descriptive statistics. Results: Fourteen responses from 10 countries (including Belgium, England, France, Japan and Mexico, among others) demonstrated that “unmet clinical need” was paramount for EAS designation across all countries and types of schemes. The next most important factors were “phase-III trials underway” and “serious condition” for Compassionate Use Programme (CUP) and Named Patient Programme (NPP) inclusion (21 percent and 20 percent of respondents, respectively). “Measures in place to monitor risk” was key for CUP and NPP designation (43 percent and 27 percent of respondents, respectively), followed by “innovative product designation” for CUP and “scientific opinion” for NPP eligibility (14 percent and 23 percent of respondents, respectively). “No specific monitoring requirements” exist in Germany and Austria, whereas “reporting of adverse events” is crucial in France, England, Japan and Spain. NPP eligible products are mainly funded at a negotiated price and CUP designated products are largely provided by manufacturers free-of-charge (i.e. England, Scotland, Germany). Conclusions: Eligibility criteria/requirements and funding arrangements for early access vary considerably across settings and their respective EAS. Information from a larger sample of countries is required for an all-encompassing mapping of the early access products’ characteristics.