ROADMAP Project - ONGOING
About the Project
There are ∼7000 rare diseases affecting approximately 30,000,000 individuals in the U.S.A. and 95% of these rare diseases do not have a single Food and Drug Administration-approved therapy. Since the incentives for new drug development for rare diseases are limited, drug repurposing provides a promising way to identify effective treatments for rare diseases faster and cheaper than developing new drugs. Still, many challenges exist in the drug repurposing space, namely:
lack of a consensus of the roles that various stakeholders play (patient advocacy organizations, researchers, physicians, government, pharmaceutical companies, etc.)
lack of a “roadmap” of how rare disease organizations should go about pursuing drug repurposing,
lack of support (data, finances, resources, guidelines, etc.) during the drug repurposing process.
The Repurposing Of All Drugs, Mapping All Paths (ROADMAP) project proposes to fill some of these knowledge gaps through document analysis, surveys and interviews grounded in a participatory design approach. Ultimately, this project aims to not only answer some fundamental questions about the experience of drug repurposing for various stakeholders, but to also design a solution to some of the challenges rare disease organizations are facing through the creation of an interactive tool.
Project Goals
In this research, we seek to:
Answer fundamental questions about the experience of various stakeholders (rare disease organizations, physicians, researchers, patients and loved ones) in drug repurposing.
Understand the information and knowledge sharing practices of rare disease non profit organizations (RDNPs) within the rare disease space in regards to drug repurposing
Build a network of RDNPs and their collaborators in order to better understand the rare disease space in general.
Produce a “roadmap” for RDNPs to support the pursuit of drug repurposing.
We will utilize a combination of methods to pursue our research questions as follows: document analysis, surveys, network analysis, interviews, and participatory design sessions.
For this project, we are looking to collect data from rare disease organizations, patients, loved ones, researchers, and physicians treating rare disease patients, as well as representatives of pharmaceutical companies and government organizations.
Check it out at https://cdcn.org/roadmap/