The Evaluation of Drug Innovativeness in Italy: Key Determinants and Internal Consistency

BackgroundInnovative 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.ObjectiveWe aimed to evaluate the internal consistency and drivers of the innovativeness appraisal process.MethodsAppraisal 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.ResultsAmong 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.ConclusionsInnovativeness 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.

Automated summary

In Italy, innovative medicines receive dedicated funds and immediate market access. The evaluation process considers unmet need, added therapeutic value, and quality of evidence. This study found that innovativeness status is driven by the added therapeutic value and quality of evidence.