Innovative research methods for studying treatments for rare diseases: methodological review.

ABSTRACT:

Objective

To examine methods for generating evidence on health outcomes in patients with rare diseases.

Design

Methodological review of existing literature.

Setting

PubMed, Embase, and Academic Search Premier searched for articles describing innovative approaches to randomized trial design and analysis methods and methods for conducting observational research in patients with rare diseases.

Main outcome measures

We assessed information related to the proposed methods, the specific rare disease being studied, and outcomes from the application of the methods. We summarize methods with respect to their advantages in studying health outcomes in rare diseases and provide examples of their application.

Results

We identified 46 articles that proposed or describ

SUBMITTER: Gagne JJ 

PROVIDER: S-EPMC4242670 | biostudies-literature | 2014 Nov

REPOSITORIES: biostudies-literature