How to better anticipate innovative anticancer drugs and their potential challenges: A European survey from the iPAAC WP9.

Abstract

e14134 Background: The European joint action iPAAC, funded by the European Commission, aims to develop innovative approaches to advances in cancer control. The field of anticancer drugs has strongly evolved over the past years and the clinical development remains very important. Horizon scanning systems (HSS) help policy makers and healthcare professionals to anticipate new medicines and their main impacts before their marketing authorization. The Work package 9 (WP9) of iPAAC, dedicated to innovative therapies in cancer, wanted to evaluate the need for more finesse in the anticipation of innovative therapies in the oncology field. Methods: A survey among organizations in charge of an HSS was conducted to point out methodologic features needed to enable a better anticipation of anticancer drugs, gene and cell therapies, biomarkers and pediatric indications. In addition, a retrospective analysis of the efficiency of HSS was conducted based on the review of 11 approved indications of checkpoint inhibitors and CAR-T cells. For each indication, organizations indicated if it was anticipated via their HSS and provided eventual challenges encountered. Results: Eleven main organizations in charge of HSS were identified in Europe, from which 9 participated to the iPAAC WP9 study. Time frames of these HSS ranged from 3 years to only a few months prior marketing authorizations. While most of the organizations included all innovative drugs in their scope, 2 were focusing only on oncology drugs and had developed specific methods to prioritize innovative medicines. To enable a good anticipation of biomarkers and gene & cell therapies, it appeared important to screen earlier phases clinical trials and to collect additional data. The retrospective analysis showed that biomarker expression threshold and centers authorized to provide gene and cell therapies or biomarker tests were some of the hardest parameters to anticipate. Having a structured database in the HSS process was seen as helpful, as it allowed the generation of ad hoc queries. Involving the expertise of practicing clinicians for prioritizing and assessing the innovative therapies was seen as an important asset for predicting clinically impacting drugs. Conclusions: Anticipating innovative drugs in oncology requires specific characteristics, especially given the variety of drugs, the earliness of data supporting marketing authorization and the dynamics of this therapeutic area. HSS is a valuable resource for the proper introduction and diffusion of innovative drugs.