Demystify Your Disease’s R&D Readiness

Keeping Up with Advances

Keeping up with new therapeutic approaches and technologies can be time consuming. However, there are resources that provide summaries and announcements via newsletters or email alerts. Others have webpages that can be checked periodically. 

  • Federal resources: The National Institutes of Health (NIH) and the FDA offer multiple ways to learn about new and emerging developments, including:
    • Free NIH Email Updates, which allow you to choose the news topic areas you would like sent to your email.  Topics under Science include:
      • Basic Research Information.
      • Biomedical Technology Information.
      • Clinical Research Information.
      • Data Science Information.
      • Genetics/Genomics Information.
      • Translational Research Information.
    • News & Events for Human Drugs is an FDA webpage that provides information about new developments.
    • Preclinical Innovation and Core Technologies are NCATS webpages highlighting novel approaches to improve the process of joining basic science discoveries with initial testing of therapies in humans.
      • You can sign up for the monthly NCATS e-newsletter highlighting the latest translational science advances, research news, and upcoming events.
      • NCATS provides a way for subscribers to receive announcements about NCATS’ news releases, funding opportunities, events, program information, and more.
    • Accomplishments in Genomic Medicine is a website with a list of interesting advances in the realm of genomic medicine since 2011 compiled by the National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG).
      • GMWG published a recent paper in the American Journal of Human Genetics based on the notable accomplishments gathered from their list. It can be found at 2019 Genomic Medicine Year in Review .
    • News and Events from National Cancer Institute (NCI)-funded Research and Programs is an NCI webpage highlighting emerging and new approaches and technologies. 
    • Genomics and Precision Health Weekly Update is a Centers for Disease Control and Prevention (CDC) webpage highlighting CDC publications in genomics and precision health.
      • Features the latest information and publications on the public health impact of human genomics and family history, pathogen genomics and advanced molecular detection, machine learning and precision health technologies. 
      • Provides links to specific topics including gene therapy, proteomics, and rare diseases.
      • Sends  email updates (sign up is located in the box on the left side of the page, below the menu).
    • Check the website of the NIH Institutes and Centers (ICs) that includes research areas that may be applicable to your disease(s). Each IC offers options for learning about new developments, whether a webpage, news announcements, or newsletters. 
  • Technology resources:
    • Pharmaceutical Research and Manufacturers of America (PhRMA) has several webpages that highlight new developments in the pharmaceutical industry. Although not directed to patient groups, this is one of the public websites of the industry written in plain language.
  • Other resources: Large umbrella disease and health organizations as well as innovative public-private partnerships offer webpages with updates, newsletters, webinars, and even in-person conferences that will enable you to learn about new and emerging techniques and developments. Such resources include: 
    • Global Genes is  a nonprofit rare disease group dedicated to connecting, empowering, and inspiring the rare disease community. Many of Global Genes resources and events provide updates on genomics, innovation, and drug development for rare disease research progress, including:
      • Latest News and Rare Daily , which provide links to short articles, interviews, and RareCasts (informative podcasts).
      • Event Hub lists upcoming webinars and conferences.
    • The National Organization for Rare Disorders (NORD) is a nonprofit patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them.  
      • You can Sign Up for Email Updates to learn about NORD events, news, and funding opportunities, including:
        • Medical Updates & Research News.
        • Monthly NORD E-Newsletter.
        • NORD Events.
        • NORD Webinar Series.
      • RareEDU Webinar Archive (2017 to present) is home to the NORD’s live, interactive webinars. Many of the topics of the webinars relate to therapy development including understanding the Orphan Drug Act, several about different aspects of gene therapy, and ways to engage with the FDA
    • EveryLife Foundation for Rare Diseases is a nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation holds yearly workshops on scientific advances.
      • Rare Disease Scientific Workshop brings together leading stakeholders from industry, academia, patient advocacy organizations and government.
        • Agendas and audiovisual recordings of previous workshops are available.
    • OrphaNews is a freely available, twice-monthly electronic newsletter presenting an overview of scientific and political news about rare diseases and orphan drugs

Resources

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)
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 is CRISPR-Cas9? Wellcome Genome Campus YourGenome (link)
Somatic Cell Genome Editing National Center for Advancing Translational Sciences (NCATS) (link)
Finding Disease and Technology Info
PubMed National Library of Medicine (NLM) (link)
ClinicalTrials.gov National Library of Medicine (NLM) (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)
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)
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)
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)