From Lab to Clinic: Translational Research for Rare Diseases

Discover how research is translated into drugs and treatments for RD patients

On this five-week course from the European Joint Programme on Rare Diseases, you’ll explore the drug development process from the unique perspective of rare diseases.

You will follow the journey from discovery to approved products, learn how therapeutic targets and drug candidates are identified and validated, and examine the special regulatory frameworks.

Describe translational medicine phases like drug discovery, clinical trials and regulatory approval

Guided by experts in the field of rare diseases, you’ll examine current research projects, and learn the definitions and contexts, as well as hearing from patients directly involved in clinical research.

You will explore some of the specific challenges of the drug development process, gaining contextual insights into this vital area of medical research.

Understand the do’s and don’ts when moving from bench to bedside

From first-in-man to post-marketing studies, you’ll trace each step of a clinical trial, learning to plan and design your trial and address any regulatory issues.

You’ll examine how to end a clinical trial and how to approach marketing approval and authorisation. You’ll also investigate data sharing and reuse post-trial.

By the end of this course, you’ll understand how, during the process of translational research, you build evidence that a treatment is safe and effective.

This course is designed for researchers and students in medicine and health-related research fields, as well as healthcare professionals wanting to further their knowledge of translational research in rare diseases.

It is also suitable for biotech and start-up developers and Patients Advocacy Organisation representatives who want to increase their understanding of current practices in therapeutic developments for rare diseases.

This course has been developed through funding from the EJP RD project.

The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N825575.

This course is designed for researchers and students in medicine and health-related research fields, as well as healthcare professionals wanting to further their knowledge of translational research in rare diseases.

It is also suitable for biotech and start-up developers and Patients Advocacy Organisation representatives who want to increase their understanding of current practices in therapeutic developments for rare diseases.

This course has been developed through funding from the EJP RD project.

The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N825575.

• Report how new therapeutic targets are identified
• Explain what is meant by hit identification and leads optimization
• Summarise the concept of drug repurposing and why it is particularly relevant in the field of rare diseases
• Describe what preclinical model systems are and why they are used to study rare diseases and to develop therapeutic approaches
• Summarise the concept of “3Rs” and the ethical aspects of using animals for therapy development
• Report how clinical trials are conducted and sponsored
• Debate the specific challenges of clinical trials for rare diseases
• Describe the role of regulatory agencies and the regulatory framework to develop a treatment for rare diseases
• Discuss why the implication of patients is key in each phase of the therapeutic development, especially in the field of rare diseases
• Explain the concepts of data use and data sharing