Demystify Your Disease’s R&D Readiness
Tips for Success
- Scientists performing research to understand a disease process may be associated with the government, a university (academic institutions), a non-profit organization,or pharmaceutical industry.
- If you don’t have anyone with a scientific background in your group as staff or volunteers, consider hiring a graduate student, medical student, postdoc, or fellow who can do the literature search for you and report what they found in an understandable format.
- Consult with your Scientific/Medical Advisory Board about which research or research tools may still be needed. Remember, not everything has to be known before the search for new treatments or a cure begins.
- Developing more than 1 disease model may be necessary and optimal. Again, relying on your scientific and medical advisory board to evaluate this need rather than a researcher asking to be funded to do the research is important.
- Not all emerging therapeutic approaches or technologies will be applicable to your disease and popular press may exaggerate the readiness of a new development. Therefore you may want to review any new developments with your Scientific/Medical Advisory Board or talk to the leading medical researchers working on your disease(s).
How Biomarkers Can Improve the Drug Development Process U. S. Food and Drug Administration (FDA) (link)
Global Genes LIVE!: Gene to Therapy: How Individualized Therapies Are Becoming Reality Global Genes (link)
Understanding the Gene Therapy Process and Aftercare National Organization for Rare Disorders (NORD) (link)
CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers National Cancer Institute (NCI) (link)
Finding Disease and Technology Info
Institutional Review Boards Frequently Asked Questions U.S. Food and Drug Administration (FDA) (link)
Research Portfolio Online Reporting Tool (RePORTER) National Institutes of Health (NIH) (link)
Keeping Up with Advances
What's New Related to Drugs (CDER New) and other drug-related topics U. S. Food and Drug Administration (FDA) (link)
Accomplishments in Genomic Medicine National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG) (link)
2019 Genomic Medicine Year in Review National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG) (link)
News and Events from NCI-funded Research and Programs National Cancer Institute (NCI) (link)