Expanded Access at Global Blood Therapeutics

Global Blood Therapeutics (GBT) is passionate and committed to revolutionizing treatment options by developing therapies for patients with severe unmet medical needs. As part of that commitment, we are focused on conducting clinical studies of our experimental therapies, since these clinical studies are the best and most efficient way to make much needed new medical advances broadly available to patients. Clinical studies are research studies designed to determine if a medicine is safe and effective for patients. People who choose to participate in clinical studies play a critical role in developing new medicines. Whenever possible, GBT will refer an individual requesting access to our investigational medicines to an appropriate clinical studies.

For additional information about GBT’s ongoing clinical studies, visit and search the name of the investigational medication, e.g., “GBT440”.

GBT understands that in some cases, patients with serious or immediately life-threatening diseases may not be eligible for any clinical studies and may not have other treatment options. In these very specific circumstances, GBT may consider providing our investigational medicines outside the clinical study process through Expanded access, also referred to as “compassionate use”.

Expanded access regulations allow drug manufacturers to make investigational new drugs (medicines not yet approved by governmental regulatory authorities) available, under specific circumstances, to treat one or more patients with a serious or life-threatening disease or condition who cannot participate in a clinical study.

GBT will evaluate requests received by a treating physician on behalf of a patient on a case-by-case basis, based on the criteria and process outlined below. GBT will grant expanded access to its investigational medicines only in these rare and very specific circumstances.

How to request access to a GBT investigational medicine

If you are interested in obtaining access to an investigational medicine, you should discuss available options with your treating physician. If a GBT clinical study may be a good treatment option for you, we recommend that your doctor contact us on your behalf at

If a clinical study is not available to you, or if no appropriate clinical studies exist, and if your treating physician believes that expanded access may be an option for you, please ask your treating physician to contact GBT on your behalf at GBT will acknowledge receipt of Expanded access requests within 72 hours. GBT will review, and in appropriate cases, follow-up with the treating physician for requisite information and a formal request for consideration.

How GBT makes decisions about expanded access

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:

  • There are no ongoing or pending clinical studies that 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.
  • There is substantial evidence to support 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 relevant 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.
  • There is adequate supply of the investigational medicine.
  • It is logistically feasible to make the investigational medicine available outside of a clinical study setting.
  • All necessary regulatory/institutional approvals have been obtained to allow the administration.