Comparative Analysis of Hta Decisions, Price and Reimbursement Level of Orphan Drugs In France and Italy

Abstract

A539of 16 criteria. Methods: 85 experts were interviewed to estimate the importance of each criterion in the decision-making on financing MT for rare diseases. We used 10-point scale, where 10 points mean major importance to the priority indicator, and 1point means minor importance. Mean estimates were calculated using descriptive statistics, then means were normalized. Results: Respondents were 41 years on average (ranging from 23 to 64 years), and included 20 public servants, 16 health administrators, 32 practitioners, and 14 researchers. 44 respondents had a scientific degree. The most important criteria were characteristics of treatment -"Effect of treatment on quality of life" and "Effect of treatment on life expectancy" with 1 points each. The least important criteria were both characteristics of the disease -"Cognitive disorders as manifestations of the disease and "Additional burden on the daily lives of care-givers" with 0.28 and 0.1 respectively. ConClusions: Characteristics of treatment turned out to be more important for respondents than characteristics of disease, therefore characteristics of treatment should be given consideration when evaluating rare diseases to determine priority financing.

HTA decision-making for drugs for rare diseases: comparison of processes across countries

“…Three hundred documents were selected for inclusion in the review (Additional file 1: Appendix 1 describes the results using the Preferred Reporting Items for Systematic Reviews and Meta-analyses [PRISMA] flowchart). Collectively, they described reimbursement and pricing processes for DRDs in the following international jurisdictions: Australia [5][6][7][8][9][10][11], France [12][13][14][15][16][17][18][19][20][21][22][23][24][25][26], Germany [12, 13, 19-21, 23, 26-42], Italy [12,13,24,29,[43][44][45][46], New Zealand [7,[47][48][49][50][51], Spain (Catalonia) [12,29,[52][53][54][55][56][57], Sweden [12,19,…”

“…(2) may bring a clinically significant advance compared to the means available; or, (3) meets a need that is not sufficiently covered [15,16,19]. Similarly, in Italy, orphan drugs, as well as those of "exceptional therapeutic and social importance" or used only in hospitals, are eligible for accelerated review (i.e., completed 100 days from filing an application, instead of the standard 180 days) [29].…”