Abstract
Objectives: Starting from April 2017, the Italian Medicine Agency (AIFA) has approved new criteria for defining any new medicinal product with an innovative indication. The purpose of the study is to analyze the activity of innovativeness evaluation according to the new approach, to estimate the weight of each criterion considered for innovativeness definition, and to evaluate how the new approach works in terms of consistency and reproducibility.Methods: A retrospective analysis was performed on the final reports evaluating the drug innovativeness assessment published on the AIFA's website between April 2017 and January 2021. Descriptive statistics, chi-square test, whether the conditions were respected, or Fisher's exact test was used to explore the association between characteristics of drugs and the innovativeness status and the association between the three criteria. Profiles of the decision process and their relationship with innovativeness response were described. In order to evaluate the weight of each criterion in predicting the innovativeness status, a Classification Tree (CT) algorithm was applied.Results: Overall, of the 109 published drugs reports, 37 (33.9%) were recognized as fully innovative, 29 (26.6%) were considered conditionally innovative, while for 43 (39.4%) reports innovativeness was not recognized. Considering the three criteria of the decision process, the added therapeutic value was the only criterion statistically associated with a drug's degree of innovation (p < 0.001). The therapeutic need and the quality of clinical evidence were statistically associated (p = 0.008) even if only a mild association was observed. The added therapeutic value was the most important variable in predicting the innovativeness status according to the classification tree (CT) model applied, achieving an accuracy of 89.4%. No difference was found between orphans and non-orphan drugs or oncological and non-oncological drugs.Discussion: The added therapeutic value is the most important criterion of the multidimensional approach for the innovativeness status definition of a new medical product. A mild association was found between the therapeutic need and the quality of evidence. Overall, similar decision profiles bring the same evaluation of innovativeness status, indicating a good consistency and reproducibility between decisions.
Highlights
The assessment of the innovativeness of a new medicine and the transparent disclosure of the information on the decision-making process are a challenge for many regulatory agencies and health organizations worldwide [1, 2].In April 2017, new criteria to define the innovativeness of a medicine were approved by the Italian Medicine Agency (AIFA)
This study represents the most updated analysis of the AIFA’s assessment reports of drug innovativeness, since the new approach using GRADE methodology was adopted by AIFA in order to evaluate the innovativeness of medications in Italy
Our study showed that the type of medicine, oncological or orphan drug, does not influence the final decision on drug innovativeness, suggesting that these characteristics cannot be considered as determinants of drug innovativeness
Summary
The assessment of the innovativeness of a new medicine and the transparent disclosure of the information on the decision-making process are a challenge for many regulatory agencies and health organizations worldwide [1, 2]. In April 2017, new criteria to define the innovativeness of a medicine were approved by the Italian Medicine Agency (AIFA). According to the new approach, as described in our previous paper [3], the decision process used to define the innovativeness of a drug takes into account three criteria, the therapeutic need, the added therapeutic value, and the quality of clinical evidence, which is assessed based on the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) methodology (Box 1). The overall assessment process resulted in a new medicinal product being awarded one of the following three innovative statuses by a specific therapeutic indication: “fully innovative,” “conditionally innovative,” or “non-innovative.” The assessment process was performed by the Scientific and Technical Committee (Commissione Tecnico-Scientifica, CTS) of AIFA, which adopted the final decision when deciding on reimbursement and price of a new medicine (or new therapeutic indication); for each assessment, a full report explaining the rationale for the final decision is made publicly available on the Agency’s website [4]
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