Access to Investigational Therapies

Access to Investigational Therapies

At Ultragenyx we recognize the difficulty of receiving a rare disease diagnosis for you or someone in your family. Our goal is to support patients and families explore what options may be available to help. We appreciate that our patients lives may be in crisis and something is needed, and we appreciate the critical need for us to engage and help figure out if something can be done as promptly as possible.

We demonstrate our commitment to patients and families by developing potential new therapies for the treatment of rare and ultra-rare diseases and making them available to patients around the world through a variety of mechanisms allowed by regulatory authorities.  We do work hard to get these therapies through the approval process to make them available generally, but even then, crises can occur between the time we start work on an investigational treatment and the time it becomes generally available.    

As an important part of our commitment to patients and their families, Ultragenyx makes available some of its investigational therapies to qualified patients worldwide under certain circumstances of critical and compelling need when the data and other factors support this use through our early access program or alternative clinical study setting.  Early access, sometimes called “compassionate use” or in the United States, Expanded Access, is a process that may help patients obtain investigational therapies that are not yet approved by the country’s regulatory agencies (e.g., the Food and Drug Administration (FDA)) outside of a clinical study.

Though Ultragenyx can make its investigational therapies available through early access, the primary preferred mechanism for access to investigational therapies is by participation in a clinical study.  An investigational therapy is a potential new therapy that has not yet been approved by regulatory authorities such as the FDA in the United States or the European Medicines Agency (EMA) in Europe. Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory authority review and potential approval.  Ultimately, we believe that approval by regulatory authorities gives the opportunity for the broadest access because regulatory approval allows physicians to prescribe the therapy to any patients who might benefit.

Clinical Study Information

To participate in a clinical study, you/your child must meet certain eligibility criteria.  These criteria identify similar disease characteristics which can allow researchers to better evaluate the investigational therapy.  You should discuss participation in a clinical study with your physician who knows your medical history and current status and can discuss if you may be able to join a clinical study.

For information on ongoing Ultragenyx clinical studies:

  • Visit and search registered clinical studies for the disease. To search by company, Ultragenyx, click here.
    • If you locate a clinical study with a participating site that you are interested in, you or your physician may contact that site directly for more information.
  • For a list of ongoing Ultragenyx clinical studies please click here OR contact the Ultragenyx Patient Advocacy group at

 Early Access

At Ultragenyx, we recognize that some patients with serious or immediately life-threatening diseases may not be able or eligible to participate in clinical studies and may not have other treatment options for their condition.  Ultragenyx evaluates requests for individual patients to receive investigational therapies outside of a clinical study on a case-by-case basis. 

As a company, Ultragenyx is committed to engaging in the crises that occur in peoples’ lives and attempting to help and support where possible.  Unfortunately, not every situation will be one where we can help.  We greatly appreciate the challenges patients and families face and hope we can support them whenever possible.


Ultragenyx’s decisions for early access will be made on a fair and 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 which is potentially irreversibly disabling or immediately life-threatening.
  • Not able to join a clinical study
    • Eligibility requirements or geographical limitations to participate in an ongoing clinical study.   
  • No appropriate treatment options
    • 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.
  • Evidence of possible benefit
    • Clinical and/or scientific evidence of possible benefit from the investigational therapy for your/your child’s condition.
  • Benefit of treatment outweighs risks
    • Disease or symptoms that suggests the benefits of treatment outweighs any known or potential risks so that 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.

Requesting Access

For Patients/ Caregivers/ Patient Advocates

Requests for early access to investigational therapies must be made by a qualified physician.  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.  Your physician can make the request for early access on your behalf.  It is important that early access requests are made by medically trained personnel in order to avoid delays.

For more information on Early Access you may visit the United States Food and Drug Administration website.

For Physicians / Healthcare Professionals

All requests for early access for a single patient should be submitted by the patient’s treating physician.

The patient’s treating physician 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 investigational therapy and must comply with all applicable reporting requirements.

Qualified physicians can submit a request by contacting Ultragenyx at

For more information on Early Access for Patients and Physicians you may visit the United States Food and Drug Administration website.

Evaluation & Response

Ultragenyx will review the request and supporting documents from a treating physician to determine the urgency and need of the request. We are committed to acknowledging receipt of a request within 5 business days. We aim to respond to the request including our rationale as promptly as possible so that you and your family can explore all possibilities.   

When evaluating the request, we will consider the best option:

  • First determine if an appropriate Ultragenyx sponsored clinical study or an Investigator-initiated clinical study is regionally available.
  • If no study is available then we will consider early access outside of a clinical study:
    • If the disease has been treated with the investigational therapy and shown to be safe and effective, we will respond promptly and provide guidance to the physician to ensure all applicable regulatory and legal requirements are met.
    • If the disease or condition is one we have not treated or are not studying, our ability to help may be limited. In general, prior treatment of the disease with the investigational therapy is needed for Ultragenyx to consider early access.

As a company, Ultragenyx is committed to engaging in the crises that occur in peoples’ lives and attempting to help and support where possible.