Do orphan drugs depend on specific conditions of a region? an international comparison based on official lists

FONTANA, D; MIANA, G; TOGNONI, G; MAZZIERI, MR

Conferencia; International Conference on Rare Diseases and Orphan Drugs (ICORD).; 2009

INTRODUCTION: Despite the increasing development of attention given over the last several years to orphan drugs (OD) and rare diseases (RD), a great variability still exists with respect to registration policies and availability to the concerned populations. Moreover, other pharmacotherapeutic issues remain unsatisfied such as drugs for neglected diseases (ND), orphan dosage forms, among others. OBJECTIVE: To analyze major international OD lists, and to compare with the situation in Argentina. METHODS: Observational, descriptive, transversal study. On-line sources: latest update (as July 2008)of OD lists of regulatory agencies of USA (FDA), Europe (EMEA), Australia (TGA), and Japan (MHW). Database of approved drugs by the Argentinian regulatory authorities (ANMAT). In each country OD were grouped according to their status: designated (OD-D), approved (OD-A), with current marketing authorization (OD-C). In Argentina an ad-hoc enquiry (via a direct contact with the producer) was needed to better clarify the OD availability. RESULTS: The number of OD-C was: 104 in USA (1536 OD-D, 325 OD-A); 43 in Europe (479 OD-D, 43 OD-A); 33 in Australia (84 OD-D, 63 OD-A); 59 in Japan (167 OD-D, 95 OD-A). ATC description of all OD-C was: L(37%), A(12%), B(11%), V(10%), H(8%), N(7%), other(15%). Surprisingly, only 1 OD-M (imatinib) was common to all lists for the same indication. The range of indications for OD-A in each list was: 1-15 in USA, 1-2 in Europe, 1-4 in Australia, 1-6 in Japan. Somatropin, for example, was approved for 15 RD in US, for 3 in Australia and 2 in Japan. Some indications of the OD-A coincide with medicine for ND. Of the 146 OD-C cumulatively found in the four official lists, the 60% (88) was registered by ANMAT and the 51% (75) was available in the Argentinian market. DISCUSSION AND CONCLUSION: The great variability of OD status was observed in countries with well established drug policies and it is accentuated in Argentina where a national Agency has been more recently established. A close verification of the impact of this variability on the needs of patients should be mandatory in each country.