Early access schemes (EASs) can enable access to medicines prior to completion of the regulatory process. EASs allow national regulators to issue an initial positive assessment of the balance between benefits and risks for groups of patients on the basis of early clinical trial data. The main aim is to meet the needs of patients facing exceptional challenges i.e., those with seriously debilitating or life-threatening diseases and no satisfactory treatment alternative.
Single-administration (one-time) therapies present a challenge for EASs due to their one-off nature. In existing EASs, manufacturers are often required to provide the product free. For single-administration (one-time) therapies, where treatment is completed within the timeframe of the EAS, there is no prospect of reimbursement at that time or in the future for that patient. Hence, models of early access without reimbursement may not be financially sustainable for manufacturers of single-administration therapies, reducing investment into finding subsequent single administration therapies.
In this paper, we present key considerations for the design of EASs for single-administration therapies based on a multi-stakeholder, expert roundtable. The roundtable was attended by 11 experts including current and former: payers, regulators, health technology assessment (HTA) body representatives, industry body representative, politicians, ethicists, government representatives and patient advisory groups. The experts included people with expertise at EU level as well as national level experts from Spain, France, UK, Poland, Italy and Germany.
In this paper, we first set out the context for EASs based on a review of the literature. We then present the key considerations for EASs for single-administration therapies developed during the roundtable. Finally, we conclude with suggestions for further research on how to implement the key considerations in different national contexts.