Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.

ABSTRACT:

Introduction

For many rare diseases, strong analytic study designs for evaluating the efficacy and effectiveness of interventions are challenging to implement because of small, geographically dispersed patient populations and underlying clinical heterogeneity. The objective of this study was to integrate perspectives from published literature and key rare disease stakeholders to better understand the perceived challenges and proposed methodological approaches to research on clinical interventions for rare diseases.

Methods

We used a meta-narrative literature review and focus group interviews with key rare disease stakeholders to better understand the perceived challenges in generating and synthesizing treatment effectiveness evidence, and to describe various research methods

SUBMITTER: Tingley K 

PROVIDER: S-EPMC6022712 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature