How clinical research works

Clinical trials are studies in people that look at new ways to prevent, detect, or treat disease. The aim of clinical trials is to determine if a new test or treatment works and is safe before it is approved by regulatory authorities and made available for use by doctors. Clinical trials vary in their primary goal or endpoint (i.e. the most important outcome of the trial), the number of patients involved, and the specifics of the study design. However, all clinical trials conform to Good Clinical Practice (GCP) and Guidelines set forth by the International Conference on Harmonisation and to a strict set of criteria outlined in a trial protocol. A protocol is a document that describes how a clinical trial will be conducted and ensures the safety of the trial participants and integrity of the data collected.

A bit about Orphan Drugs

ProQR aims to develop medicines for rare genetic diseases. Medicines for rare diseases are usually referred to as 'Orphan Drugs'. An orphan drug is intended for a disease affecting less than 200,000 persons (US) and around less than 250,000 (EU). An Orphan drug designation is granted by regulatory authorities (like FDA or EMA), at any stage of drug development, provided certain criteria are met.

Often, patients diagnosed with rare diseases do not have many- if any- treatment options available. With the aim to stimulate development, there are measures are available drugs designated as orphan drugs.

QR-010 for CF and QR-110 for LCA-10 have been granted orphan drug status in the US and EU. Stay tuned to see how the path forward for these molecules proceeds.

Educational resources

If you are interested in learning more about research and development, you might want to check out the following resources:

The European Patients’ Academy (EUPATI) Toolbox on Medicines R&D
EUPATI focus on education and training to increase the capacity and capability of patients to understand and contribute to medicines research and development and also improve the availability of objective, reliable, patient-friendly information for the public.

European Communication on Research Awareness Needs (ECRAN) Project
The ECRAN Project aims to make understanding clinical trials - a relevant element of medical research - easy, and tells you all about taking part in them.

National Institutes of Health, USA
NIH is the nation’s medical research agency, supporting scientific studies that turn discovery into health. is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.

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