OBJECTIVES

To identify and analyse the global early access program (EAP) landscape, assessing the different national framework’s applicability for cell and gene therapies.

METHODS

A targeted systematic literature review was conducted to identify EAPs across global markets: Africa; Asia; Europe; North America; Australia and South America. National healthcare agency websites were used to compile information relating to guidelines, laws and criteria of specific EAPs. EAP applicability for cell and gene therapies was assessed, considering potential reimbursement and individual patient access through these schemes.

RESULTS

The heterogeneity of the global EAP landscape is significant and rapidly evolving. Various EAP paradigms currently exist, utilising a variety of regulatory frameworks and criteria, but all share the goal of ensuring patient’s timely access to potentially lifesaving therapies. Of the 57 countries included in this analysis, 34 have well defined, established EAPs in place. European countries in particular have carefully defined EAPs, (present in 16 out of 25 countries, including all EU5 markets). In contrast, early access to medicines in Asian and South American markets is still in an early evolutionary phase; discerning the details and degree of reimbursement of scheme in these countries is challenging due to the paucity and uncertainty of information. 11 of the 34 countries with defined EAP frameworks offered reimbursement of the treatment. 12 countries had schemes with ambiguous or highly conditional reimbursement.

CONCLUSIONS

Manufacturers of gene and cell therapies in rare diseases may find EAPs to be unfeasible, as facilitating early access to their therapies without reimbursing can greatly diminish their cohort of patients, especially for one-time treatments. Taking a holistic view of the global EAP landscape, Europe presents the most attractive option for manufactures seeking early access for cell and gene therapies in rare diseases, as most countries have formalised pathways for reimbursed therapies prior to market authorisation.