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Rare disease research and trial design

Learn an overview of the purpose and structure of clinical research by devling into what’s included in pre-clinical research and clinical research.

To help you understand the basics of clincial research, we’ll explore key definitions, such as blinded clinical trials and randomisation, before moving into the ethical considerations to be aware of when it comes to clinical trials, such as evaluating the risk-benefit ratio and informed consent.

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Clinical trials for rare diseases do come with unique challenges, so our guide outlines those hurdles and provides clear solutions to help you overcome them.

Enroll now to gain the background knowledge needed to begin getting involved in research!

With thanks to:

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Costello Medical provided pro-bono support in the creation of this course.

Resource type

Course

Skill level

Intermediate

Duration

Est. 1-2 hours

Last updated

May 2022

Resource type

Course

Skill level

Intermediate

Duration

Est. 1-2 hours

Last updated

May 2022

What will you learn?

  • How clinical research is traditionally structured
  • The considerations and adaptations for rare diseases
  • Ethical considerations to be aware of when engaging in research

Who is this course for?

This guide has been developed for patient group leaders, staff or volunteers to:

 

  • Provide information and guidance for patient groups on how scientific research and trials are designed and run
  • Aid further understanding of the challenges and potential solutions when designing clinical trials in rare diseases
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