Abstract 2726
Background
Neuroendocrine tumours (NETs) consist of a heterogeneous group of neoplasms arising from a variety of neuroendocrine cell types. According to criteria of the European legislation, some may be orphan indications. However, epidemiologic data on specific sub-types of NETs are scarce. This study aimed at assessing the prevalence of gastroenteropancreatic (GEP) and lung NETs in the European Union.
Methods
Systematic literature reviews were conducted using Medline and Embase bibliographical databases. Search strategies combined the following concepts, using MeSH and Emtree terms: Disease of interest, epidemiologic? data (incidence or prevalence) and countries of interest. The search period covered 1st Jan 2010 to 15 Feb 2016, inclusively. Pragmatic searches of web sources and conference proceedings were also conducted. Using data identified in the search, prevalence rates were estimated using the relationship between prevalence (P), incidence (I), and disease duration (D), where applicable. Size of the EU population as of 1st January 2015 was used to estimate number of patients with GEP and lung NETs. As incidences rates and survival of patients vary across countries and settings, sensitivity analyses were also performed.
Results
A total of 87 and 23 sources were identified through the literature search and pragmatic search, respectively for GEP-NETs and lung NETs. Following in-depth review, 8 and 6 data sources were retained for epidemiologic data extraction on GEP and lung NETs, respectively. Considering, the highest estimated prevalence rates of GEP-NETs and lung NETs (3.8 and 1.9 per 10,000, respectively), number of prevalent cases in Europe was estimated to be 195,314 and 97,657. In addition, results from the sensitivity analyses demonstrated that the prevalence rates of these conditions remained well below the threshold of 5 per 10,000 in all scenarios considered except the worst case including highest estimates of incidence rates and disease duration.
Conclusions
According to these results, GEP-NETs and lung NETs could be considered separately as orphan indications according to the European legislation.
Clinical trial identification
Legal entity responsible for the study
Yolarx Consultants
Funding
Ipsen Pharma
Disclosure
A. Bergamasco, Y. Moride: Affiliated with Yolarx Consultants, which received a grant from IPSEN Pharma to conduct this research project
J. Dinet, A. Berthon, S. Gabriel, G. Nayroles: Employee of Ispen Pharma