Orphan Drugs Clinical Trials - Live Online Training

    Orphan Drugs Clinical Trials

    27 – 28 November 2023, live online training


    This training course presents a unique blend of Orphan Drugs regulatory guidance and clinical trials strategies. Delegates will explore the legal basis for approvals, learn how to design a study and which methodology to use. Evidence-Based Medicine data analysis, conclusions, and reporting will be thoroughly discussed.

    Who should attend?

    Rare Disease Specialists, Clinical Research Associates, Clinical Operations Analysts, Clinical Diagnostic Specialists, Pharmacovigilance Scientists, Regulatory Managers and Specialists, Process Science Research & Development, Drug Product Development Scientists, Orphan Drug Products Formulators, Quality & Compliance Associates

    Learning Objectives

    • EMA and FDA expectations for Orphan Drugs clinical studies
    • The Importance of avoiding Bias and Regression to the Mean
    • Working with Evidence Based Medicine and Clinical trials
    • How to design a study and which methodology to use?
    • Practical implementation of regulatory pathways
    • Where do we need randomised trials?

    Key Topics

    • Orphan Drugs EU and US regulatory guidance
    • Legal basis for approvals (Regulatory pathways) & Working Groups
    • Trial concept, design and conclusions
    • The Importance of Precision and Confidence Intervals
    • Study designs and methodology considerations
    • What constitutes convincing evidence?

    Our online training experience includes

    • Our client zone – a single source for all training materials as well as pre and post-training communication
    • Live interactive format via the Zoom platform
    • Direct interaction with the trainer
    • Q&As, case studies, breakout rooms
    • Revisit recorded sessions for 30 days
    • Digital and LinkedIn certificates


    Simon has spent 30 years working in clinical trials, mostly in the pharmaceutical industry but also including five years at the UK and European regulatory agencies.
    Orphan Drugs Clinical Trials online training agenda

    Orphan Drugs Clinical Trials

    Download the full training agenda to reveal complete session details, training takeaways, case studies, daily schedule, special features and full trainer bio.

    Online Participation
    1 650  Price incl. VAT: 1 650 
    Equivalent of approx. 1 849 USD.
    The price calculation in USD is informative and might slightly differ from your bank exchange rate

    Price per delegate includes:

    Online participation

    Online workbook & materials


    Revisit recorded sessions for 7 days

    PC, microphone, camera, stable internet connection and the Zoom application.

    Yes, we will make every effort to ensure best possible interaction. You will see the slides, interact with the trainer and fellow participants. Questions and group exercises will be facilitated through the online meeting platform.

    Yes, you can transfer the registration to a colleague at any time.

    Yes, you may transfer your registration to a different event up to one month before the scheduled dates, provided the training fee has been paid in full.

    pharma learning leader

    Lydia Makori

    +421 222 200 543

    Training Details
    • Start Date
      27. November 2023
      01:00 PM Vienna time
      12:00 PM London time
      07:00 AM New York time
      04:00 AM Los Angeles time
    • End Date
      28. November 2023
    What participants said about this course

    "This course exceeded my expectations. The content is highly relevant and valuable for anyone who is developing drugs for orphan diseases."
    Co-Founder & Chief Scientific Officer ,Healx Ltd.

    "I gained a lot of input from trainer and his experience. This was very useful."
    Clinical Development Professional, Dompe

    "I improved my knowledge about Orphan Drug designation especially regarding statistical aspects."
    Clinical Scientist, Italfarmaco

    "The training improved my knowledge about the topic, as well as offered the possibilities to establish a working platform."
    Clinical R&D Manager, Italfarmaco

    "As a CRA, and after this training, I really would like to have the challenge of monitoring an Orphan Drugs clinical trial."
    Clinical Research Associate, Vall d’Hebron Institute of Research

    "A very detailed & dilligent training on complex clinical development and particularities of it in rare diseases! The training has really helped me to start thinking of how to improve the way I work."
    Medical Director EMEA, Lupin Pharmaceuticals

    "Highly recommend this course for anyone working in Medical affairs and new to the concept of Clinical trials."
    Medical Science Liaison Rare Diseases, Alnylam Pharmaceuticals