Abstract
different therapeutic areas (classified according to the ICD-10; see Table 1) and 67 different indications. The most represented therapeutic areas were neoplasms (28 products, 40.6%) and endocrine, nutritional and metabolic diseases (21 products, 30.4%). Out of the 69 products, 14 had a PRO claim (20.3%); see Table 2. By comparison, a review performed in 2011 [1] showed that 22% of all EMA products approved between 2005 and 2010 had a PRO claim in their labeling (in particular in the 5.1. section of the approved labeling). PROs identified in the claims were assessing function [e.g., physical functioning ; 2 products (14.29%)], symptoms [e.g., pain; 12 products (85.71%)] and quality of life [(QOL) – 4 products (28.6%)]; with some products cumulating different types of claims. Corresponding therapeutic areas and indications were neoplasms (trabectedin, ofatumumab, ruxolitinib), endocrine diseases (aztreonam lysine, ivacaftor, tafamidis), nervous system diseases (stiripentol, rufinamide), blood diseases (eculizumab, icatibant), pulmonary arterial hypertension (bosentan monohydrate, ambrisentan), cryopyrin-associated periodic syndromes (rilonacept), and severe pain (ziconotide). More than one product out of four (4/14) had a QOL claim: two for neoplasms [trabectedin (ovarian cancer) – no differences between treatment in QOL; and ruxolitinib (myeolofibrosis) improvement of QOL], one in diseases of the blood [eculizumab (two indications: paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome improvement of QOL], one in endocrine disease [tafadimis (amyloidosis) – improvement of QOL]. Patient-reported outcome (PRO) claims in products indicated for the treatment of rare diseases and approved by the European Medicines Agency (EMA) Benoit Arnould*1, Laure-Lou Perrier2, Catherine Acquadro2 1Mapi, HEOR and Strategic Market Access, Lyon, France; 2Mapi Research Trust, Lyon, France
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