Applicability and added value of novel methods to improve drug development in rare diseases.

ABSTRACT:

Background

The ASTERIX project developed a number of novel methods suited to study small populations. The objective of this exercise was to evaluate the applicability and added value of novel methods to improve drug development in small populations, using real world drug development programmes as reported in European Public Assessment Reports.

Methods

The applicability and added value of thirteen novel methods developed within ASTERIX were evaluated using data from 26 European Public Assessment Reports (EPARs) for orphan medicinal products, representative of rare medical conditions as predefined through six clusters. The novel methods included were 'innovative trial designs' (six methods), 'level of evidence' (one method), 'study endpoints and statistical analysis' (four met

SUBMITTER: Mitroiu M 

PROVIDER: S-EPMC6233569 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature