Demystify Your Disease’s R&D Readiness
Research Tools
Certain research tools are developed to help scientists understand more about the disease process. Some of these same tools, along with others, are necessary to develop a therapeutic approach, determine whether a potential therapy can move forward to clinical trials, and monitor therapeutic effectiveness throughout clinical trials. It is important to understand which tools have been developed for your disease and which ones may be needed in the future. NCATS Toolkit provides more information and resources about these and other research tools as they relate to preclinical trials in Discovery: Understand Translational Research Tools
- Cell lines: Cell lines are developed from a single tissue type and are adapted to grow continuously in the laboratory. The research use of cell lines developed from specific patient populations includes:
- Isolating the genetic cause of a disease.
- Understanding gene function.
- Understanding cellular processes that may be disrupted in the diseased state.
- Testing drug metabolism and cytotoxicity.
- Disease models: A disease model is an animal or cell line displaying all or some of the pathological processes that are observed in the actual human disease. For most disease research, having more than 1 disease model is ideal. The research use of disease models includes:
- Determining in which cell types or tissues a gene is expressed and when.
- Exploring the function of the gene or its protein.
- Understanding the biological processes that lead to symptoms.
- Studying how the disease develops.
- Testing potential treatment approaches.
- Biomarker: Biomarkers are biological molecules found in blood, other body fluids, or tissues that are a sign of a normal or abnormal process or of a condition or disease.
- Biomarkers are useful as an indicator of change. Biomarkers may be used to:
- Diagnose a disease.
- Determine who is at risk to develop certain symptoms or complications.
- Track disease progression.
- Determine how well a person responds to a new medical therapy, including if certain subpopulations within the disease community respond better than others.
- Serve as an end point of a study.
- How Biomarkers Can Improve the Drug Development Process is a short slide set from the U.S. Food and Drug Administration (FDA) that provides a quick overview about biomarkers.
- Biomarkers are useful as an indicator of change. Biomarkers may be used to:
- Patient information and data: Scientific, clinical, and patient databases can guide even early stages of thearpy development by providing information on multiple members of a disease community.
- Collected patient data can help researchers learn more about:
- Causes and pathology of disease.
- Clinical picture of a disease.
- Unmet needs of patients.
- Preferences of patient population.
- NCATS Toolkit provides more information about different ways your group can help collect data in Discovery: Determine Patients’ Needs and Prepare for Clinical Trials: Perform Patient Preference Studies and highlights how information from these databases can be used throughout the therapy development process.
- Rare Diseases Registry Program (RaDaR) was developed by the National Center for Advancing Translational Sciences (NCATS) to provide information tips and resources about the development of patient registries and natural history studies. RaDaR will help you collect data from your patient group that will be useful to therapy development for your disease and in a way that will allow researchers to use the data.
- Collected patient data can help researchers learn more about: