RARE Toolkits

Below is a list of all of our current RARE Toolkits.




Patients with rare diseases often look to new treatments for help. These treatments are often new drugs but may also be devices, vaccines, or biologics such as cell-based treatments or gene therapy. Sometimes, these drugs or biologics are still being tested to learn how well they work and how safe they are. These tests are called clinical trials or clinical research.

This Global Genes Toolkit will offer information surrounding the types of clinical trials, as well as how to find them. It is part of a series on drug development, From Molecules to Medicine, with others in the series providing more detail on each step of the process. Details about how patients and their families may share their voices to improve clinical trials can found in From Molecules to Medicine: Sharing Patient Voices. For more about understanding the drug development process, take a look at From Molecules to Medicine: How Are New Drugs & Therapies Developed.