Patients & Physicians

Expanded Access at Global Blood Therapeutics

Global Blood Therapeutics (GBT) is committed to revolutionizing patient care by developing innovative therapies for patients with severe unmet medical needs. As part of that commitment, we focus on conducting clinical studies of our investigational therapies. However, we understand that 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 Access.

How to request access to a GBT investigational medicine

GBT is able to consider providing patients access to an investigational medicine 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.

If your treating physician believes that expanded 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 medicalinformation@gbt.com. GBT will acknowledge receipt of expanded access requests within 72 hours. 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 access is appropriate.

How expanded 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 access has been made by a qualified and licensed physician with expertise and facilities appropriate for the administration of the therapy, monitoring, 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 III 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 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 access study protocols

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