OBJECTIVES:

Time-to-reimbursement (TtR) is crucial in the context of Rare diseases, where therapeutics alternatives are limited. Conventional appraisal methods may be unsuitable for assessing the value of rare disease treatments which may cause a delay in TtR. To evaluate TtR and identify potential key factors influencing TtR of Rare vs Non-Rare drugs in Italy, we performed an analysis of AIFA Scientific Technical Committee (CTS) and Price Reimbursement Committee (CPR) assessment process.

METHODS:

The analysis was carried out using an MA-Provider database, populated with data on new drugs approved by EMA which started national evaluation from October 2018 and reimbursed until April 2022. The median duration of CTS/CPR evaluation phase was detected for Rare vs Non-Rare drugs; sample descriptions and sub-analyses were completed also considering the orphan and innovative status. The number of CTS/CPR postponed procedures and investigations were examined as potential influencing factors on the process.

RESULTS:

88 drugs (39 Rare vs 49 Non-Rare disease drugs) completed the P&R process. The median duration of the CTS assessment for Rare and Non-Rare disease drugs was 70 and 33 days, respectively, while, for CPR assessment, the median duration was 122 and 97 days. Among rare disease drugs, 74% (29/39) were designed as orphan, with a median duration of 84 and 177 days for CTS and CPR procedure. The non-innovative rare orphan drugs showed an increase in TtR compared to the innovative ones (419 vs 357 days) with a relevant difference in CPR time (194,5 vs 148 days). The longer time of evaluation for rare and rare orphan drugs could be ascribed to increased number of CTS and CPR investigations rather than the number of postponed procedures.

CONCLUSIONS:

Our analyses indicate that the TtR is longer for Rare disease, probably due to an increased number of CTS and CPR investigations.