A Review of Patient-Reported Outcome Labels in the United States: 2006 to 2010

Abstract

The FDA continues to approve PRO claims, with 24% of new molecular entities and biologic license applications being granted. Successful PRO label claims over the past 5 years have generally supported treatment benefit for symptoms specified as primary endpoints.

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

“…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]. This can be partially explained by the fact that, while primary endpoints were typically based on disease symptoms, the FDA-approved PRO claims were symptom-based.…”

“…Such a procedure allowed us to avoid including sponsors attempts to achieve such claims. Whereas a decreasing trend in PRO claims was reported for FDA approvals from 2006 to 2015 [7,10], it is unknown if a similar trend occurred at the EMA. However, it is plausible that our PRO rates are still below the more recent PRO claim rates for EMA non-orphan drug approvals; this aspect needs to be confirmed by additional research.…”

“…Only 9% of orphan drugs approved by the FDA between 2012 and 2016 had PRO claims in their labels [9] compared to 24% and 16.5% of drugs approved by the FDA in 2006–2010 and 2011–2015, respectively [7,10]. One possible explanation for the low rates of PRO claims for orphan drugs is a low number of dedicated PRO instruments available.…”

“…For instance, the EMA has encouraged the development of new PRO tools for rare cancers to guide the use of PRO measures in oncology studies, because the existing ones may not be appropriate or specific enough to measure important outcomes in this population [6]. However, PROs are rarely reported in clinical trials of drugs for rare diseases [7,8]. Only 9% of orphan drugs approved by the FDA between 1/1/2012 and 31/12/2016 contained PRO claims in their PIs [9].…”

“…Only 9% of orphan drugs approved by the FDA between 1/1/2012 and 31/12/2016 contained PRO claims in their PIs [9]. In contrast, two studies by Gnanasakthy, et al reported that 24% and 16.5% of PIs of all drugs approved by the FDA in 2006–2010 and 2011–2015 respectively, included PRO claims [7,10]. …”