OBJECTIVES: To study the impact of therapeutic need, added therapeutic value, and quality of evidence on the final assessment on innovativeness across Agenzia Italiana del Farmaco (AIFA) reviews.

METHODS: A total of 49 AIFA reviews from 06/2017 to 05/2019 were analyzed. Two-way analysis of variance (ANOVA) tests were performed in a standardized manner on factors deciding the innovativeness of the drug (therapeutic need, added therapeutic value, quality of evidence, and final assessment on innovativeness) to identify any significant differences.

RESULTS: Complete innovativeness was recognized in 42.8% (n = 21) of drugs/therapies whereas conditional (or potential) innovativeness was recognized in 28.6% (n = 14) of drugs/therapies. There was no recognition of innovativeness in 28.6% (n = 14) of drugs/therapies. The therapeutic need (p < 0.01) and quality of evidence (p < 0.01) categories significantly affected the agency’s decision on the final assessment on innovativeness. Drugs that met "important therapeutic need” criteria received maximum recognition of innovativeness, followed by drugs that met "moderate therapeutic need” criteria. Drugs in the "important added therapeutic value” category received maximum recognition of innovativeness. Quality of evidence is the most significant variable affecting drug-innovativeness status.

CONCLUSIONS: Therapeutic need is an independent variable whereas added therapeutic value and quality of evidence are not completely independent of each other, as the determination of the added therapeutic value is based on the quality of evidence. While it appears that the quality of evidence alone impacts the final assessment on innovativeness, more research into the nature of the confounding between the quality of evidence and the added benefit, given the dependency between the variables, should be conducted. Understanding the impact of these variables on the Italian Medicines Agency’s final decision would allow quicker access to innovative medicine benefits in Italy.