Expanded Access

At BioMarin, we are committed to developing safe and effective therapies for rare conditions and advancing those therapies as quickly as possible.

As part of that commitment, we support expanded access programs when we have substantial scientific evidence to support both the safety and the efficacy of an investigational product for an indication, and when it is logistically practicable.

BioMarin has a two-step process for determining whether the company will provide an experimental therapy through expanded access.

Expanded Access Process

  1. 1

    First, the company will evaluate whether:

    • There is substantial scientific evidence to support both the safety and the efficacy of a product for an indication, typically after positive Phase 3 data are available.
    • It has been established that providing the medicine on an expanded access basis will not compromise clinical trials or the regulatory pathway.
    • The product is intended to treat a serious or life-threatening disease, and existing approved therapies are not appropriate or effective, such that consideration of using an unapproved therapy is justified.
    • There is adequate supply of the experimental therapy.
    • The compound can be administered – and it is logistically feasible to make it available – outside of the clinical trial setting.

    If the company decides that, as a first step, expanded use is appropriate and should be available as a general matter, then we will evaluate the request.

  2. 2

    Second, the company will assess the following criteria:

    • The patient’s underlying medical conditions do not pose safety risks that have not been sufficiently studied.
    • The request has been made by a qualified and licensed physician with expertise and facilities appropriate for the administration of the therapy and the capabilities to monitor, manage and report side effects, as well as to assess patient experience.
    • The patient’s condition is of a type and stage commensurate with the indication for which the investigational therapy is likely to be approved.
    • The patient does not qualify for participation in ongoing or pending clinical trials.
    • All necessary regulatory/institutional approvals have been obtained to allow the administration.

Contact Global Medical Information to Make a Request

Requests for access to experimental therapies must be made by a qualified and licensed physician through the BioMarin Medical Information team and will be referred to a team for evaluation. Qualified and licensed physicians may make such requests by submitting contact information and a description of the request through our Medical Information Request Form. We anticipate being able to acknowledge receipt of such requests within five business days.

For information on each of our current clinical trials, visit our Clinical Trials page. This page provides information linking to clinicaltrials.gov, the U.S. government website that provides information about all clinical trials.