Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties.

ABSTRACT:

Background

To assess uncertainty in regulatory decision-making for orphan medicinal products (OMP), a summary of the current basis for approval is required; a systematic grouping of medical conditions may be useful in summarizing information and issuing recommendations for practice.

Methods

A grouping of medical conditions with similar characteristics regarding the potential applicability of methods and designs was created using a consensus approach. The 125 dossiers for authorised OMP published between 1999 and 2014 on the EMA webpage were grouped accordingly and data was extracted from European Public Assessment Reports (EPARs) to assess the extent and robustness of the pivotal evidence supporting regulatory decisions.

Results

88% (110/125) of OMP authorizations wer

SUBMITTER: Pontes C 

PROVIDER: S-EPMC6238348 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature