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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).

Resources

Disease Knowledge
The 10 Things You Need to Pursue Research For Your Disease: What Are the Options for Research and Why is it so Hard? Global Genes (link)
Research Tools
How Biomarkers Can Improve the Drug Development Process U. S. Food and Drug Administration (FDA) (link)
Rare Diseases Registry Program (RaDaR) National Center for Advancing Translational Sciences (NCATS) (link)
Genetic Therapies
What is Gene Therapy? U. S. Food and Drug Administration (FDA) (link)
Human Gene Therapy for Rare Diseases U. S. Food and Drug Administration (FDA) (link)
Global Genes LIVE!: Is Gene Therapy Right for Your Disease? Global Genes (link)
Global Genes LIVE!: Gene to Therapy: How Individualized Therapies Are Becoming Reality Global Genes (link)
Global Genes LIVE!: Harnessing the Power of New Genomic Frontiers Global Genes (link)
A Guide to Gene Therapy Global Genes (link)
Gene Therapy Yesterday, Today and Tomorrow National Organization for Rare Disorders (NORD) (link)
The Science Behind Gene Therapy National Organization for Rare Disorders (NORD) (link)
The FDA's Role in Gene Therapy National Organization for Rare Disorders (NORD) (link)
Understanding the Gene Therapy Process and Aftercare National Organization for Rare Disorders (NORD) (link)
Life After Gene Therapy National Organization for Rare Disorders (NORD) (link)
What is genome editing? National Human Genome Research Institute (NHGRI) (link)
What are genome editing and CRISPR-Cas9? MedlinePlus (link)
What is CRISPR-Cas9? Wellcome Genome Campus YourGenome (link)
Somatic Cell Genome Editing National Center for Advancing Translational Sciences (NCATS) (link)
CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers National Cancer Institute (NCI) (link)
Finding Disease and Technology Info
PubMed National Library of Medicine (NLM) (link)
Creating Alerts: PubMed NIH Library (link)
ClinicalTrials.gov National Library of Medicine (NLM) (link)
CenterWatch Clinical Trial Listings CenterWatch (link)
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)
List of Registries National Institutes of Health Clinical Center (link)
Keeping Up with Advances
Free NIH Email Updates National Institutes of Health (NIH) (link)
News & Events for Human Drugs U. S. Food and Drug Administration (FDA) (link)
What's New Related to Drugs (CDER New) and other drug-related topics U. S. Food and Drug Administration (FDA) (link)
Preclinical Innovation National Center for Advancing Translational Sciences (NCATS) (link)
Core Technologies National Center for Advancing Translational Sciences (NCATS) (link)
Monthly NCATS e-newsletter National Center for Advancing Translational Sciences (NCATS) (link)
Receive NCATS Announcements National Center for Advancing Translational Sciences (NCATS) (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)
Genomics and Precision Health Weekly Update Centers for Disease Control and Prevention (CDC) (link)
PhRMA: Scientific Discoveries Pharmaceutical Research and Manufacturers of America (PhRMA) (link)
PhRMA: Research and Development Pharmaceutical Research and Manufacturers of America (PhRMA) (link)
Global Genes Latest News Global Genes (link)
Global Genes Rare Daily Global Genes (link)
Global Genes Event Hub Global Genes (link)
Sign Up for NORD Email Updates National Organization for Rare Disorders (NORD) (link)
Rare Disease Scientific Workshop EveryLife Foundation for Rare Diseases (link)
OrphaNews Orphanet (link)