Abstract
BACKGROUND: Innovative medicines are provided with dedicated funds and immediate market access in Italy. Innovativeness evaluation considers unmet need, added therapeutic value, and quality of the evidence. OBJECTIVE: We aimed to evaluate the internal consistency and drivers of the innovativeness appraisal process. METHODS: Appraisal reports on innovativeness refer to 1997-2021. We used both a descriptive approach and probabilistic multivariate analysis, using logistic regression models to compute odds ratios and 95% confidence intervals. The dependent variable is innovativeness status (innovative vs. non-innovative; full innovativeness vs. conditional innovativeness). Explanatory variables, besides the three above-mentioned domains, are the year of evaluation, drug type, target disease and population, and the number and type of available studies. RESULTS: Among the 141 medicines scrutinized, 31.9%, 29.8%, and 38.3% were evaluated as fully innovative, conditionally innovative, and non-innovative, respectively. Added therapeutic value and the quality of the evidence were associated with the odds of receiving innovative status, and full compared with conditional innovativeness; unmet need was not a predictive variable. Other factors played a minor role: medicines for both solid tumours and rare diseases are more likely to be judged innovative; conditional innovativeness is more probable for medicines for rare diseases. CONCLUSIONS: Innovativeness status is driven by the added therapeutic value and quality of evidence. The appraisal process is internally consistent and predictable. This provides industry with a clear indication of what is needed to ensure that access to their medicines is prioritized.