Criteria to define rare diseases and orphan drugs: a systematic review protocol.

ABSTRACT:

Introduction

Rare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal definition of RDs or ODs. This systematic review (SR) will identify the quantitative and qualitative criteria and the underlying rationale used internationally to define RDs and ODs.

Methods and analysis

This protocol follows the conventions for the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (2015 guidelines). A SR will be conducted, including a search of the following databases: PubMed, MEDLINE, EMBASE, Scopus, Web of Science, GreyLit and OpenGrey. Eli

SUBMITTER: Abozaid GM 

PROVIDER: S-EPMC9345065 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature