PMU85 LESSONS FOR BREAKING NEW GROUND WITH FIRST-IN-CLASS THERAPIES

Abstract

To explore the access challenges faced by first-in-class treatments across therapeutic areas and identify optimal evidence and submission strategies to help secure desired reimbursement levels in the top-five European markets. We analysed products approved by the European Medicines Agency (EMA) within the last five years and filtered this list for first-in-class therapies, defined as first-to-market or on the basis of a novel mechanism of action. Our final analogue selection included treatments launched in three major therapeutic areas: rare diseases, non-rare diseases, and oncology. Using a targeted search protocol, we evaluated information on the regulatory label and examined the evidence package, including clinical trial design, inclusion and exclusion criteria, and the efficacy outcomes that supported approval. For each therapy, we compared the regulatory label with the reimbursement outcomes across markets to identify drivers for health technology assessment (HTA) decision-making and barriers to positive reimbursement outcomes. In oncology and non-rare diseases, reimbursement restrictions were imposed in therapies that failed to investigate treatment effect across the full population represented in the label. Similarly, therapies with pivotal trials that lacked inclusion of sub-populations representative of current clinical practice also faced HTA restrictions in these therapy areas. In contrast, HTA bodies were less likely to impose restrictions in treatments for rare diseases with high unmet need and lack of alternative options. First-in-class therapies with a broad regulatory label can achieve widespread unrestricted access only with an evidence package that spans the whole licensed indication. However, HTA bodies might be more lenient when evaluating treatments targeting diseases with high unmet need and lack of treatment options. Therefore, to maximize the success of submissions, manufacturers need to carefully consider the population within the clinical trials, but also ensure they have an accurate reflection of the level of unmet need in the target population.