Pmu45 Prevalence vs. Treatment Cost: Analysis of Drugs for Rare Disease in Italy

Abstract

, were collected and categorized according to: i) kind of innovativeness: full, conditional or no innovativeness; ii) orphan designation; iii) therapeutic area: oncological vs non-oncological drugs. For each single indication assessed for innovativeness, the ranking of the three criteria (therapeutic unmet need maximum to absent, added therapeutic value maximum to absent, and quality of evidences high to very low) was analyzed. Sub-analyses according to the three categories were also run. Results: Out of forty-one indications assessed by AIFA for innovativeness (and corresponding to 30 drugs), 12 (29%) were granted full innovative status, 14 (34%) obtained the conditional innovativeness and the remaining 15 (37%) were assessed as not innovative (7 orphan and 8 oncological indications). 50% of full innovative indications referred to orphan drugs (6/12) and 42% to oncological diseases (5/12); regarding conditionally innovative indications, 29% were for orphan drugs (4/14) and 79% (11/14) for oncological ones. Analysing innovativeness criteria application, AIFA have never recognized the maximum grading for added therapeutic value; moreover, focusing on fully innovative indications, 9 out of 12 (75%) did not meet all the three criteria thresholds stated by AIFA decree. Conclusions: This analysis highlighted that there is no strict matching between AIFA criteria (as reported by AIFA decree) and the granting of full innovative status. In fact, most of the analysed cases are borderline situations (full vs. conditional innovativeness) and have been assessed case-by-case, leaving a shade of subjectivity that gives innovativeness decision-making process a margin of flexibility. The limited sample does not let us make conclusions on the impact of orphan designation or oncological setting.

Variables affecting pricing of orphan drugs: the Italian case

“…The empirical evidence on variables influencing orphan drug prices is quite scarce. There is some evidence showing a price–volume trade-off, with higher prices for orphan versus non-orphan drugs [ 15 ] and ultra-orphan versus orphan medicines [ 16 ]. A recent paper investigated the determinants of orphan drugs prices in France through a regression model [ 17 ].…”

“…The aim of this paper is to investigate the determinants of orphan drugs annual treatment cost in Italy, where the empirical evidence is limited to the role played by the disease prevalence [ 15 , 16 ]. Our empirical findings shed new light on which variables are actually considered when prices for orphan drugs are set.…”