Supporting Access for All Patients

Access to Approved Medicines

Our UltraCare® patient programs help facilitate access to our treatments

At Ultragenyx, we are passionate about educating and supporting patients, families, and caregivers affected by rare and ultra-rare diseases. We created UltraCare to help patients and caregivers understand their insurance coverage; find financial support including helping to determine their eligibility for financial assistance and patient assistance programs, no matter what type of insurance a patient may have; and access the various resources provided in the patient support programs. All patients in the United States with a confirmed diagnosis will have access to our approved therapies, regardless of their ability to pay. Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657 to assist patients and their families.

For more information, visit UltraCare.

Access to Investigational Therapies

Helping patients and families navigate and explore investigational treatment options

Receiving a diagnosis of a rare disease is often life altering for the patient and their family, and we have a responsibility to support patients and their families as they explore available treatment options. In developing new medicines for children and adults with rare and ultra-rare diseases, we want to ensure that 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 – are available to patients through approved mechanisms. The preferred way to gain access to our investigational therapies is by participating in a clinical trial. However, some patients with a serious or immediately life-threatening disease may not be eligible to participate in clinical studies and may not have other treatment options. In those cases, we make our investigational therapies available to qualified patients under certain circumstances of critical and compelling need through our early access program. We appreciate that our patients’ lives may be in crisis and the critical obligation we share to engage and identify potential options.

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.

Learn more about our ongoing clinical trials

We recognize that some patients with serious or immediately life-threatening diseases may not be able or eligible to participate in a clinical trial and may not have other treatment options available to them. We evaluate requests for individual patients to receive investigational therapies outside of a clinical study on a case-by-case basis. We are committed to providing any help and support that we can for patients in crisis.

Our decisions for early access will be made on an equitable basis for each investigational therapy. Each disease we are studying may have different specific criteria due to the differences in urgency and known data on safety and efficacy. We consider the following when reviewing a request:

  • Serious manifestation of disease
    • Whether you/your child has a serious manifestation of a condition that is potentially irreversibly disabling or immediately life-threatening
  • Ability to join a clinical study
    • Eligibility requirements or geographic limitations to participate in an ongoing clinical study
    • Limited or no availability of other, appropriate treatment options. For example, if you/your child has tried standard treatments without success, exhausted all reasonable available treatment options, or can no longer tolerate existing treatment, you/your child may be an appropriate candidate.
  • Benefit of treatment outweighs risks
    • Disease or symptoms that suggest the benefits of treatment outweigh any known or potential risks so the potential to help the patient is plausible, based on available information
  • Adequate supply of therapy
    • Supply levels of the investigational therapy available to meet the request
  • Appropriate local regulatory pathway
    • Whether regulatory authorities where you/your child live allow early access to investigational therapies through a locally appropriate regulation
  • Other possible considerations
    • Other criteria, fully reviewed with the requesting physician, based on the complexity and diversity of situations for each patient and disease situation

Country-specific laws and regulations will apply and may affect availability of the investigational therapy in that country or region. Ultragenyx may restrict availability in some countries.

For Patients/Caregivers/Patient Advocates
Requests for early access to investigational therapies must be made by a qualified physician, who can make the request on your behalf. You and your family should discuss your situation with your treating physician, including current status, available treatment options, and reason for considering treatment with an investigational therapy.

For more information on early access, visit the U.S. Food and Drug Administration (FDA) website.

For Physicians/Healthcare Professionals
All requests for early access for a single patient should be submitted by the patient’s treating physician, who must provide adequate information to demonstrate that the patient meets the outlined considerations for early access. The patient’s treating physician 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 EarlyAccess@ultragenyx.com.

For more information on early access, visit the U.S. Food and Drug Administration (FDA) website.

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. We will then evaluate the request and supporting documents to determine the urgency and need of the request. We will first determine if an appropriate Ultragenyx-sponsored clinical study or an investigator-initiated clinical study is regionally available. If no study is available, we will consider early access outside of a clinical study.

Listening and connecting with patients is central to what we do. We take a hands-on approach to supporting patients and their families, and we are there for them at one of the most important times in their life – when they are experiencing a medical crisis – and beyond.

Visit our patient advocacy site