Advancing early access policies for innovative cancer drugs: a scoping review and explorative analysis in the Italian setting

Considering the clinical impact of innovative cancer therapies, policy makers strive to balance timely access and thorough value-assessment. While some European countries promoted early access schemes, Italy does not yet display a consolidated strategy for innovative drugs or for medicines targeting pathologies with a high unmet need. To better understand the risks and opportunities of early access strategies that could be applied in the Italian setting, we performed a scoping review, searching the PubMed and Web of Science databases and interviewing two field experts. The review results were complemented with an exemplificative quantitative analysis for a subset of innovative oncology drugs, to assess the clinical and economic impact of the price and reimbursement negotiation. Our study suggests that early access schemes developed in Germany and France, combining a free-pricing period, pay-back mechanism, and arbitration, could serve as a basis for developing a feasible strategy in Italy. The quantitative analysis indicated that timely access to innovative drugs could have potentially prevented many cancer progressions, associated with a significant healthcare expenditure. Albeit not allowing to express a conclusive assessment, this study proposes a potential early access strategy for Italy and highlights the need for opening a debate on the opportunities and risks of such schemes.