Receiving a diagnosis of a rare disease can be life-altering for patients and families. We know that therapies that are being studied in clinical trials, or which are approved in geographies outside a patient’s home country, can be a powerful source of hope as families work to identify potential treatment options.
Participating in a clinical study is the best way for patients to access our investigational therapies — those that have not yet been approved by regulatory authorities such as the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) in Europe. We recognize that some patients with serious or immediately life-threatening disease may not be eligible to participate in clinical studies and may have no other treatment options. In certain circumstances, our early access program may provide a potential pathway for qualified patients.
Requesting access
For patients and caregivers
Patients and families should discuss their specific situation with their treating physician, including current medical status, available treatment options, and the reason for considering treatment with an investigational therapy.
All requests for early access for a single patient must be submitted by the patient’s treating physician and should include adequate information to demonstrate that the patient meets the outlined considerations for early access.
For healthcare providers
Physicians requesting early access must be appropriately licensed, fully qualified to administer the investigational therapy, and have the expertise and facilities for monitoring, managing, and reporting any adverse events during the course of treatment. The patient’s treating physician must be willing to meet and follow all applicable legal and regulatory requirements for early access while caring for the patient receiving the investigational therapy and must comply with all applicable reporting requirements.
Qualified physicians can submit a request by contacting Ultragenyx at
Evaluation and response
Once a request for early access is received, our team will evaluate the request and supporting documentation based on our criteria. We work to acknowledge receipt of a request from the patient’s treating physician within 5 business days and make every effort to respond within 24 hours. The timeline for a decision will depend on the unique circumstances of each case.
Clinical study information
Participating in a clinical study
To participate in a clinical study, a patient must meet certain eligibility criteria, which include certain demographic and disease characteristics that can allow researchers to appropriately evaluate the investigational therapy. If you are considering participating in a clinical study, you should discuss it with your physician.
Discover more about Ultragenyx:
Pipeline
See the progress we have made with our diverse portfolio of investigational therapies.
Explore our pipeline