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Demystify Your Disease’s R&D Readiness

Finding Disease and Technology Info

Searching for published medical articles, research projects, and clinical trials can help you locate information about the cause of your disease, the biological processes, available animal and cell disease models, and new technologies or therapeutic approaches. Try searching a few key resources, such as: 

  • PubMed is a searchable database of published medical articles maintained by  NIH National Library of Medicine (NLM). Research studies about disease cause and pathology as well as promising novel and innovative approaches and technologies are usually published in medical journals, so you may be able to learn what is known about your disease or a bit more about a new technology by searching PubMed. Although not all of the articles are available for free online, most articles listed in PubMed have a summary (abstract) available.
    • Tips to help you locate articles focused on scientific research: 
      • Use “your disease” (or associated gene or protein if known) as your search term. 
        • Try using the disease term with and without quotation marks.
      • Replace the disease term with any synonyms if you are only finding a limited number of articles. 
      • Add terms such as “gene”, “etiology” (disease cause), “pathology” (disease process) or “treatment” to the main search term or use the advanced search feature to narrow your search results. 
        • You can join any added terms to the disease term or each other using “AND” between the terms. This will limit your search results to articles that contain all the listed terms.
    • Tips to help you understand the impact of a new technology on your disease:
      • Use the technology name or any synonyms as the search term.
        • If you know a disease it is currently being applied to, include the disease name in your search.
        • Many times press releases and even articles in the popular press will note one or more of the publishing researchers names or the actual title of the article, either of which can help in your search. 
      • Scan the published medical article summaries to determine whether the development is currently being:
        • Tested in cell and animal models or humans.
        • Investigated by more than 1 research team.
        • Applied to your disease(s) or a disease that has similar pathological pathways, signs, or symptoms.
        • Reported in a single case report, small study, or in larger studies.
    • If the article is not freely available, the NLM can help you find libraries in your area where you may be able to get the full text. You can search for libraries at National Network of Libraries of Medicine Members Directory . Or you can contact the NLM toll-free at 1-888-346-3656.
    • Alternatively, you can reach out to the contact author of the article and ask if they can provide a full copy to you. To find the contact information on articles found through PubMed, just click on the title in the search list to take you to the summary (abstract) view of the article. The contact information can be found by clicking the “+ expand” next to “Affiliations” just below the list of authors.
    • You can set up email alerts to be notified if new articles are published in a specific search area by simply clicking “Create alert” under the search bar above the listed search results. If you don’t have an account with NLM, the system will walk you through the process. Setting up an account is easy and free.
      • The NIH Library provides a bit more information, including a short video, if you need some help to set up or manage your PubMed alerts: Creating Alerts: PubMed
      • You can choose the frequency you will receive alerts and modify the search in the future.
    • PubMed Help provides answers to frequently asked questions and links to other tips that can help improve your search strategies and locate full text articles if one is not freely available. This resource is frequently updated by NLM.
  • ClinicalTrials.gov was developed by NLM to provide the public with current information on clinical research studies. This resource will only list studies being performed in humans. You can search your disease to determine whether there are any current or past clinical trials or search a new technology to see whether it is or has been studied in clinical trials. Scan the listed clinical trials to determine:
    • Type of clinical trial (treatment or observational).
    • Phase of clinical trial.
    • Number of different centers involved. 
    • Sponsor.
    • Current activity status.
    • Type and number of diseases included in different trials involving the new technique or approach, if you are researching information about a new technology.
  • CenterWatch is a Clinical Trial Listing Service, which provides a list of Institutional Review Board (IRB) -approved clinical trials being conducted internationally. Finding a new technique or approach being used in a clinical trial implies it has advanced to being studied in humans. Likewise finding any study including your disease can help you understand what is known about your disease as well as interested researchers or industry.
  • Research Portfolio Online Reporting Tool (RePORTER) provides access to reports, data, and analyses of research activities at NIH, including the results of NIH-supported research. Although these projects do not usually involve humans, you may want to contact the investigators to learn more about their research, whether it is about your disease or a new technology. This can be a very helpful resource to learn if any cell or animal models are available or being developed for your disease and what aspects of your disease process or cause are being currently researched. To search for studies, enter the name of your disease (or associated gene or protein) or specific technique or approach  in the “Text Search” box and click “Submit Query”.
  • In addition you may wish to reach out to the NIH ICs that focuses on research that either might apply to your disease(s) or a disease that is currently using the technique or approach. 

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)