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.
- 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.
We are so grateful to the 147 organizations who’ve partnered with us on the ROADMAP project!
Our Super Star Volunteers (past and present, in order of onboarding)
Meg Zuccato, Anna Nguyen, Derek Ansel, Martin Lukac, Annalise Jear, Mitav Nayak, Panchatapa Baul, Samatha Hood, Sabina Grigorian, Jacob Lowy, Justin Wong, Sydney Grisham, Yuan (Abby) Feng, Leanna Chen, Megan Shieh, Bryan Aguilar, Marcy Spiker, Rose Weathers, Katherine Fang, Veikko Toikka, Robert Parillo, Lindsay McBride, Sara Barrett, Robert Parillo, Dallas Ryan, Veikko Toikka, Benita Balogun, Robert Parillo, Penny Deremer, Miti Patel, Emma Roemer, Neda Pazuki, Erikka Chowdhury, Stephanie Hage, Matt Scott, Geetha Turlapati, Jessica Xiang, Jada Watkins, Jazmin Loughlin, Justin Crawmer, Ferzana Niazi, Anaheit Arathoon, Michael Zhang, Vee Suresh, Mahima Sangtani, Sara Cronin, Rita Aberbach, Kayleigh Nicole Murray, Diane Baynes, Andrew Zhu, Anastasia Kakurina, Susanna Hunanyan, Jade Bondy, Simarsukh Dhillon, Angela Perry, Carolyn Canterbury and Owen Yu.
Special thank you to our amazing CDCN ROADMAP team:
|Dr. David Fajgenbaum||Ania Korsunska||Mileva Repasky|
|Mary Zuccato||Sarah Bolden||Trae Boyd|
And prior team members Johnson Khor and Ruth-Anne Langan Pai.
The CDCN was awarded a grant from the Chan Zuckerberg Initiative, whose science program supports the science and technology that will make it possible to cure, prevent, or manage all diseases by the end of the century.