• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » EU-US collaboration to boost medicine development for rare diseases

EU-US collaboration to boost medicine development for rare diseases

September 29, 2016
CenterWatch Staff

The EMA and the FDA have set up a new cluster on rare diseases to share experiences and best practices on each other’s regulatory approach to the development of medicines for these diseases.

While rare diseases are estimated to affect 30 million people in the European Union and approximately the same number in the U.S., each disease individually concerns a limited number of patients. Therefore, global collaboration in this area is particularly important to ensure that the limited number of studies that can be conducted, due to the small populations, can benefit all patients regardless of where they live.

The agencies will exchange information on various aspects of the development and scientific evaluation of medicines for rare diseases. These include topics such as:

  • the design of clinical trials in small populations and the use of statistical analysis methods;
  • the selection and validation of trial endpoints, i.e. target outcomes of a trial;
  • preclinical evidence to support development programs;
  • the design of post-marketing studies, in particular in the context of early access mechanisms such as EMA’s conditional marketing authorization and FDA’s accelerated approval;
  • risk management strategies for long-term safety issues with medicines for rare diseases.

The cluster will provide a forum for confidential exchange of draft documents, policies under development, and more detailed information supporting the scientific basis for decision making on medicine development.

The existing EMA/FDA cluster on orphan medicinal products will continue to focus on information sharing and collaboration on orphan designation and exclusivity, the agencies’ mechanisms to encourage the development of medicines for rare diseases.

The first meeting of the rare diseases cluster took place by teleconference on September 23, 2016. The cluster will initially meet once a month via teleconference and will be chaired jointly by FDA and EMA.

The creation of this cluster is the latest step in EMA’s and FDA’s wider objective to expand and reinforce international collaboration.

The clusters established by EMA and FDA focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened collaboration. The currently existing EMA/FDA clusters discuss issues related to patient engagement, biosimilars, orphan medicines, medicines to treat cancer, medicines for children and pharmacovigilance, among other topics.

The information exchange is covered by the confidentiality arrangements between the two regulators.

Upcoming Events

  • 16Feb

    Fundamentals of FDA Inspection Management: Reduce Anxiety, Increase Inspection Success

  • 21May

    WCG MAGI Clinical Research Conference – 2023 East

Featured Products

  • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

  • Surviving an FDA GCP Inspection

    Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

Featured Stories

  • SurveywBlueBackground-360x240.png

    Sites Name Tech Acceptance as Essential Factor in Selection of Sponsors, Survey Finds

  • TrendsInsights2023-360x240.png

    WCG Clinical Research Trends and Insights for 2023, Part Two

  • TimeMoneyEffort-360x240.png

    Time is Money and So Is Effort, Budgeting Experts Say

  • TrendsInsights2023A-360x240.png

    WCG Clinical Research Trends and Insights for 2023, Part Three

Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

The information you need to adapt your monitoring plan to changing times.

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy
  • Do Not Sell or Share My Data

Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2023. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing