Strategy for Assessing New Drug Value in Orphan Diseases: An International Case Match Control Analysis of the PROPEL Study.

ABSTRACT:

Background

Although randomized studies are designed to assess overall survival (OS) benefit, the conduct of regulatory studies in patients with orphan diseases can be timely and costly without offering the same commercial return on the investment. The peripheral T-cell lymphomas (PTCL) represent a rare group of heterogeneous lymphoid malignancies with very poor prognosis. PROPEL was a pivotal phase II study that led to the accelerated approval of pralatrexate for patients with relapsed or refractory PTCL.

Methods

An international database of 859 patients was assembled from four institutions with an interest in PTCL, of which 386 were considered eligible for matching against the PROPEL criteria. Using a rigorous propensity score matching algorithm, a unique 1:1 case match of

SUBMITTER: O'Connor OA 

PROVIDER: S-EPMC6649793 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature