ProQR Expanded Access Policy

January 2020

Our Promise

At ProQR we are dedicated to improving the lives of patients, families and communities affected by rare and debilitating conditions, through our unique research and development in innovative RNA therapies.

Expanded Access Policy

Consistent with ProQR’s mission to bring innovative medicines to patients, we are focused on enrolling and conducting the clinical trials necessary to understand whether investigational RNA therapies are safe and efficacious. This will allow ProQR to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible.

Currently, ProQR does not make our investigational RNA therapies available outside of our clinical trials. ProQR will continue to regularly evaluate this policy.

Clinical Trials

Clinical trials are undertaken to test the safety and efficacy of investigational therapies so that regulatory authorities – such as the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) in Europe – can decide whether these therapies can be approved for wider use.

A clinical trial involves research using human volunteers (also called participants) that is intended to add to medical knowledge. Clinical trials can be sponsored, or funded, by pharmaceutical companies, academic medical centers, voluntary groups, governmental bodies and other organizations. Guidelines for these trials include criteria for volunteer participants.

To be eligible to participate in a clinical trial, a participant must meet the inclusion/exclusion criteria. These criteria are important because they help investigators identify patients who can take part in the trial. The criteria also help investigators identify a group of participants with similar characteristics of a condition. For instance, the study participants’ genetic profiles could be analyzed to see if they correspond to the study population. Clinical trials are generally multi-phased and can be spread over several years.

For the volunteer participants who complete a ProQR clinical trial, ProQR plans to continue to provide the investigational drug in open label, long-term extension trials, if the safety and efficacy
of the drug continues to support its use.

For further information

ProQR recommends any interested participants discuss with their treating physician to understand whether a clinical trial or research study would be the correct option for them. The treating physician can then contact the clinical trial site closest to you.

Please be aware that in the case of a clinical trial/study site not featuring in your country, this does not limit your participation.

If you have any questions about any of our investigational products, please submit them to ProQR will acknowledge receipt and respond to inquiries within 7 business days of receipt

More information related to ProQR clinical trials can be found at and