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Identification

Identification: Drug screen

Identification: Pathogenesis targeting

Identification: Data from similar diseases

Identification: Computational

Identification: Off-label use

Validation

Validation: In vivo

Validation: In vitro

Validation: Evaluation of RWE

Clinical Use

Clinical Use: Clinical trial

Clinical Use: Off-label use

Outcomes

Outcomes: Approvals

Outcomes: Alteranatives

Outcomes: Abandoned

Home

Identification

Drug screen

Pathogenesis targeting

Data from similar diseases

Computational

Off-label use

Validation

In vivo

In vitro

Evaluation of RWE

Clinical Use

Clinical trial

Off label use

Outcomes

Approvals

Alternatives

Abandoned

Drug Repurposing ROADMAP

A subtitle of some sort

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.

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.

Welcome to the ROADMAP tool!

We encourage you to read the sections below before you begin using the tool. You can access the tool at any point by closing this window.

Roadmap Overview

Our conceptualization of the drug repurposing process consists of 5 steps:

  1. Preparation
  2. Identification
  3. Validation
  4. Clinical Use
  5. Outcome

Preparation involves laying the groundwork of the rare disease non profit and creating the vital research infrastructure to set up any future repurposing projects for success. This would mean fundraising or applying for grants that you can then provide to researchers, creating a contact registry of all your patients, creating a network of researchers and physicians who are already or potentially could be doing work related to your rare disease, and developing certain resources, such as a biobank or a natural history study/registry.

Identification is the first step in the repurposing process! It entails figuring out which existing drug can be potentially beneficial to your patient population. There are many ways this can be done (high throughput drug screens, machine learning data mining, analysis of real world off label drug use, etc.), which we explore in detail in the ROADMAP.

Validation is an important step in which your initial hypothesis about the potential of the identified drug to be useful for your patient population is tested with additional research.

Clinical use - if the drug still seems promising after additional research has been done, it can move on to being used in humans, either in an official clinical trial or in the real world, meaning it will be used “off label”.

Outcome is the final step in the drug repurposing process, meaning the repurposing has reached either success or failure. Success could be measured in many ways, such as FDA approval for the new indication, integration into treatment guidelines, improving patient quality of life with off label use, etc.

Throughout all of these steps, there are many ways that rare disease non profit organizations can play a supportive role or spearhead aspects of the drug repurposing process.

Organizational support options

Roadblocks & Advice