ISSN: 2574-0407
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Aurore Bergamasco
France
Research Article
Mitigating the Paucity-of-Data Problem for Target Population Sizing: Exploring a Model-Based Approach for Advanced Gastroenteropancreatic Neuroendocrine Tumours
Author(s): Aurore Bergamasco, Gabrielle Nayroles, Anne-Marie Castilloux, Jérôme Dinet, Anthony Berthon, Sylvie Gabriel and Yola MorideAurore Bergamasco, Gabrielle Nayroles, Anne-Marie Castilloux, Jérôme Dinet, Anthony Berthon, Sylvie Gabriel and Yola Moride
Background: Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs) are rare neoplasms. For innovative treatments, payer recommendations frequently involve sub-populations more restricted than approved indications. Paucity of epidemiologic data specific to sub-populations is a challenge for reimbursement strategies. Objectives: To estimate the population size by site and type of GEP-NETs in the US, EU, and Australia, over a five-year period.
Methods: Two GEP-NET sub-populations, respectively approved and restricted indication for reimbursement, were considered: i) Stable/slow progressing well-differentiated, functioning and non-functioning GEP-NETs and unresectable locally advanced/metastatic disease; and ii) Stable/slow progressing well-differentiated, nonfunctioning GEP-NETs and unresectable locally advanced/metastatic disease. For both, tum.. View More»
DOI:
10.4172/2574-0407.1000131