Orphan Drugs Clinical Trials

    2022 Dates TBC, 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?


    • Training materials will be provided in digital form
    • All delegates will receive digital and LinkedIn certificates


    Clinical Trials Consulting & Training, CEO
    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.
    Online Participation
    1 450 Price incl. VAT: 1 450

    Price per delegate including:

    Online participation

    Online workbook & materials


    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.

    Lydia Makori

    +421 222 200 543