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Expanded access

Helping eligible patients gain access to GBT investigational therapies

GBT is committed to developing innovative therapies for patients with sickle cell disease (SCD). Central to that commitment is conducting clinical studies of our investigational therapies. However, in some cases, patients with serious or immediately life-threatening diseases may not be eligible for ongoing clinical studies and may have exhausted available treatment options. In these circumstances, GBT may consider providing our investigational medicines outside the clinical study process through expanded or early access.

How to request access to a GBT investigational medicine

GBT is able to consider providing patients access to an investigational medicine in the U.S. through a treatment protocol as part of an expanded access program. Requests for expanded access must be submitted from sites that meet criteria for participation in a treatment protocol. If you are interested in obtaining access to an investigational medicine, you should discuss available options with your treating physician.

In Europe and regions outside the U.S., GBT is able to consider providing patients access to an investigational medicine through an early access program. Early access programs make medicines available prior to commercial availability upon request by a physician for appropriate patients with no alternative treatment option.

If your treating physician believes that expanded or early access may be an option for you and no appropriate ongoing clinical study is available to you, please ask your treating physician to contact GBT on your behalf at 1-833-GBT-4YOU (1-833-428-4968). GBT will review and follow up with the treating physician for further information as needed in order to determine whether a formal request for expanded or early access is appropriate.

How expanded and early access decisions are made

GBT is committed to a fair, impartial, and consistent approach to evaluating each request for access to our investigational medicines. All decisions are based on clinical evidence and guided by the principles outlined below:

  • No ongoing or pending clinical studies are available to the patient.
  • The request for expanded or early access has been made by a qualified and licensed physician with expertise and facilities appropriate for administration of the therapy, monitoring, and managing and reporting side effects, as well as patient experience.
  • Substantial evidence exists supporting both the safety and the efficacy of our investigational medicine for an indication, typically after positive Phase 3 data on efficacy and safety become available.
  • The potential benefits to the patient seeking access to the investigational medicine outweigh the collective potential risks to the patient. The patient’s underlying medical conditions do not pose safety risks that have not been sufficiently studied. GBT evaluates these benefits and risks based on a case-by-case basis in consultation with the patient’s qualified treating physician.
  • Expanded or early access will not compromise or delay the clinical studies needed for potential drug approval and broad availability to patients.
  • An adequate supply of the investigational medicine exists.
  • Making the investigational medicine available outside of a clinical study setting is logistically feasible.
  • All necessary regulatory/institutional approvals have been obtained to allow drug administration.

Existing expanded and early access programs

Additional information about existing expanded and early access programs for GBT’s investigational medicines can be found at the following links: