OBJECTIVES : To analyse Innovation status (IS) assessment of medicinal products by the AIFA Technical Scientific Committee (CTS).

METHODS : IS applications assessed by CTS whose outcome has been published on AIFA website between July 2017 and May 2020 were selected. The database created includes IS assessment for each product/therapeutic indication, and assigned level of innovation for the three criteria assessed by CTS. Analyses were performed on the full database, and on the subgroup of orphan (ODs) and non-orphan drugs (non-ODs) (based on EU orphan designation).

RESULTS : During the period considered, CTS evaluated 76 applications for 62 products; for 9 products the assessment was performed on two or more therapeutic indications. Overall, 50 applications (65.8%) were granted IS. Forty-eight applications for 39 non-ODs were submitted; 29 (60,42%) received IS, of which 15 full (12 with Important “Incremental Therapeutic value” –ITV – and 3 with Moderate ITV) and 14 conditional (all with Moderate ITV). Only one non-OD was granted IS even though CTS assessed the “Quality of evidence” criteria as “Low”. Concerning orphan drugs, twenty-eight applications for 23 products (of which 5 applied for two indications), were assessed. Twenty-one applications (75%) received positive outcome; 12 applications for 11 orphan drugs were granted full IS (all with Important ITV); while 9 were granted conditional IS (all with Moderate ITV). Eight OD applications obtained the innovation status although CTS assessed the “Quality of evidence” criteria as “Low”, and 1 as “Very low”.

CONCLUSIONS : The analysis showed that the majority of applications for Innovation status achieve a positive outcome; granting of status is more frequent for orphan drugs, for which CTS appears to apply a more flexible approach in the presence of lower “Quality of evidence” criteria assessment. Impact of the Incremental Therapeutic Value criteria is critical for Innovation status recognition, particularly for orphan drugs.