Role of patient-reported outcomes and other efficacy endpoints in the drug approval process in Europe (2008–2012)

Abstract

The present study aimed at systematically reviewing the role and extent of patient-reported outcomes (PROs) usage within the package of scientific evidence considered for marketing authorization (MA). All regulatory information published by the European Medicines Agency (EMA) for products authorized between January 2008 and December 2012 and appearing in the European Public Assessment Report (EPAR) database was examined for efficacy endpoints. The endpoints here considered included: PROs, clinician reported outcomes (CROs), and laboratory reported outcomes (LROs). LROs were the most frequently reported endpoints. Out of the 180 products here selected, 99 (55%), 67 (37%), and 30 (17%), respectively, used LROs, CROs and PROs as primary endpoints (PEs). PROs as any endpoints were used in 82 (46%) products. Out of these, PROs were documented as PE in 30 (37%), with 27 (33%) products having used PROs both as primary and non-PEs. PRO usage was most frequently identified with nervous system and antineoplastic agents. During the study period, the use of all the three types of endpoints appeared to be static. Both the regulatory bodies and the industry should ensure complete and clear reporting of all endpoints used, including PROs, to improve transparency.

Patient-reported outcome claims in European and United States orphan drug approvals

“…PROs were the primary trial endpoint for nearly 31% of products with PRO claims, which is slightly below the 37% reported for all EMA approvals in a previous study [12]. However, it is much less than the 71% and 76.7% reported for all new FDA approvals with PRO claims [7,10] and the 86% reported for FDA orphan drug approvals with such claims (Table 2) [9].…”

“…Additionally, 21.7% of orphan-designated approvals in the studied sample presented PRO claims in their SmPCs. This is significantly below the 47% reported PRO claims for all new drug approvals by the EMA from 2006 to 2010 [11] and the 46% reported from 2008 to 2012 [12]. Conversely to the above mentioned studies, which analysed the entire content of the European Public Assessment Reports (EPARs), we focused only on the SmPCs, because they include PRO claims granted by the EMA.…”

The impact of quality-of-life data in relative effectiveness assessments of new anti-cancer drugs in European countries

“…There is evidence supporting the inclusion of patient-reported outcomes, including health-reported QoL, in regulatory product approvals [ 15 , 17 – 19 ]. Vodicka et al investigated the entries in the US clinicaltrials.gov register between 2007 and 2013 and reported an increase in the collection of patient-reported measures from 2009 onwards, particularly oncology drug trials [ 25 ].…”

“…To a large extent, such acceptance has been fuelled by clinicians, patients and caregivers [ 15 – 17 ]. Two different studies found that one-third of the EMA reports included patient reported measures among which QoL data, with the latter being more frequently mentioned in reports of antineoplastic agents [ 18 , 19 ]. A similar trend is observed at the Food and Drug Administration (FDA) where a draft guidance on the use of patient-reported outcomes in industry-sponsored studies was released for public consultation by the FDA in early 2006 and later updated in 2009 [ 20 ].…”

The utility of a single simple question in the evaluation of patients with nondiabetic polyneuropathy

“…The number of patient‐reported scales has been increasing in the last several years, as neurological examination, electrophysiological, imaging, and laboratory test findings do not include significant information regarding patients' experiences, and they are inadequate to assess quantitatively several important disease components, such as pain and fatigue . However, as some of these scales are time‐consuming, they may not be feasible in situations in which time is limited.…”