PCN322 - Opportunities with Accelerated Approval Times, but Stumbles at Hta? the Impact of a Limited Evidence Package

Abstract

Accelerated approval pathways can shorten time to market for therapies treating diseases, particularly those with significant morbidity and mortality and few effective alternatives. However, little is known about payers’ reactions to submissions based on a Phase 2, single-arm trial. This study aimed to identify market access challenges for products with accelerated marketing authorization based on this limited data package. Oncology products receiving European marketing authorization since 2012 based on a Phase 2, single-arm study were identified and the associated EPARs and HTA submissions in France, Germany and the UK (NICE) reviewed. Six products were identified (Adcetris, Bosulif, Erivedge, Iclusig, Imbruvica, and Zydelig); four had orphan designation. No pivotal trials included survival as the primary endpoint, none reported HRQL and no comparative data were cited in the regulatory packages. Only one product was reviewed by NICE (England) and was not recommended (another did not submit). All six G-BA (Germany) reviews resulted in either additional benefit not proven, not quantifiable (or minor in one subgroup). In France, the ASMR ratings ranged from III to V (moderate, to no improvement compared with existing treatments). Common themes identified which could be addressed by additional evidence generation included: (1) clarity on the target patient population and unmet need; (2) establishing the current standard of care and positioning within the treatment pathway; (3) determining relative efficacy given the focus on survival benefit and the increasing weight of additional value drivers. Although marketing authorization is granted, products supported by Phase 2, single-arm trial data face significant HTA challenges; payers are being asked to make decisions based on limited data. Some evidence gaps can be addressed with additional evidence generated alongside the pivotal trial. However, uncertainty about a product’s value may result in poor HTA outcomes, and a poor position from which to negotiate price.