Module 5: Clinical Trials and Therapy Development

In Module 1, you learned about what patient-centricity means in research. In Modules 2-4, you learned about the biology of SDS and the drivers of leukemia risk in SDS, and why new treatments are urgently needed. This module is about putting these concepts together and applying them to the framework for how new therapies are actually developed — from early laboratory work all the way to an approved therapy available to patients. Clinical trials are how therapies come to life. They are also where patient communities have the most direct opportunity to shape what gets developed, how it gets tested, and who can access it. Understanding how this process works is one of the most practical forms of capacity for patient-centered research. What you will be able to do after this module: * Describe the phases of drug development — from pre-clinical research through post-market surveillance * Explain what clinical trial phases 1, 2, 3, and 4 each do and why they matter * Understand key clinical trial concepts: endpoints, randomization, blinding, placebo controls, inclusion and exclusion criteria * Know what expanded access, compassionate use, and right-to-try programs are and when they apply * Find SDS-relevant clinical trials on ClinicalTrials.gov * Identify the ways patients and patient organizations can contribute throughout the drug development lifecycle