Orphan Drug Approvals In Europe: Historical Review and Trends

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.

Using 5 consecutive years of NICE guidance to describe the characteristics and influencing factors on the economic evaluation of orphan oncology drugs

“…It is shown that the number of orphan drug designations assigned by the United States Food and Drug Administration (FDA) significantly increased from 1983 to 2019, most prominently in oncology (1910, 37%) ( 2 ). Another study on orphan drug approvals in Europe found that 39% of all orphan drugs that were approved by the European Medicines Agency through a centralized process were cancer-related ( 3 ). Nine of the top 10 indications for orphan drugs as specified by the European Medicines Agency/European Mes Aedicingency (EMA) were for cancer (including acute myeloid leukemia, non-Hodgkin's lymphoma, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, renal cell carcinoma, liver cancer, and chronic lymphocytic leukemia) ( 4 ).…”

Determinants of orphan drugs prices in France: a regression analysis

“…Orphan drug market remains very heterogeneous in term of drugs revenue with a very small number of molecules representing the major part of the market [7]. Moreover, most of authorised products target specific therapeutic areas with high potential profitability, such as oncology or metabolic diseases [8]. Some authors voiced concerns regarding highly lucrative opportunity for manufacturers which orphan drugs represent.…”