A Dolon Institute Publication
Paid early access (PEA) can provide an important bridge between regulatory approval and full pricing and reimbursement (P&R), allowing patients to access promising treatments sooner. This World EPA 2023 roundtable brought together stakeholders from industry, consulting, academia and implementation services to explore whether paid early access is needed in Europe, where it can add value, and how it could be structured effectively.
The discussion highlights that PEA may be particularly relevant for rare diseases and advanced therapy medicinal products (ATMPs). These treatments often have small eligible patient populations and, in the case of some ATMPs, a single administration, meaning that providing treatment free of charge can have a disproportionate impact on the manufacturer’s potential return on investment. A reimbursed early-access route can allow patients to receive treatment sooner without requiring manufacturers to absorb the full financial burden of free supply.
However, the roundtable also identifies important barriers and trade-offs. Fragmented decision-making, administrative complexity, challenging pricing dynamics and the potential duplication of formal P&R processes could all limit the effectiveness of PEA. There is also a broader equity consideration: if countries adopt different approaches based on their ability to fund early access, PEA could potentially increase disparities between higher-income and more financially constrained healthcare systems.
A key discussion centred on where decisions and funding should sit. Participants favoured national-level decision-making and funding as a way of accounting for country-specific willingness to pay while reducing the risk of fragmented regional or local approaches creating “postcode healthcare”. The roundtable also considered whether access should be granted to individual patients through Named Patient programmes or to defined cohorts, with cohort approaches potentially offering greater efficiency and equity if robust eligibility criteria are established.
The discussion identified several potential criteria for determining eligibility, including high disease burden, significant unmet need, sufficient evidence of therapeutic effect and safety, substantial improvement over the standard of care, and situations where free-of-charge provision would have a substantial impact on the economics of innovation.
The roundtable also explored the tension between speed and price negotiation. Negotiating a price can provide dialogue between payers and manufacturers but may introduce delays that undermine the purpose of early access. Free pricing could provide a faster route to patients, potentially supported by mechanisms such as rebates, although these would need to be carefully calibrated to balance incentives and risk while ensuring that the formal P&R process can subsequently be completed.
Finally, participants considered whether evidence generated through PEA could contribute to subsequent P&R decisions. While real-world data may provide useful supplementary insights into effectiveness and safety, patient numbers may be too small to generate robust evidence or satisfy payer requirements. The roundtable therefore highlighted the need for agreed protocols between industry and payers governing how evidence generated through early-access programmes should be collected and subsequently used.
The overarching message is that paid early access could play a valuable role in accelerating patient access, but its design will determine whether it genuinely complements – rather than duplicates or delays – formal P&R processes.