Pre- Approval Access

New medicines undergoing clinical development are often referred to as “investigational medicines” before approval by a regulatory body such as the Food and Drug Administration (FDA) for the United States, the European Medicines Agency (EMA) for the European Union, and the Pharmaceuticals and Medical Devices Agency (PMDA) for Japan. Clinical trials are the most common route for patient access to investigational medicines. Participation in clinical trials is vital to the evaluation of safety and effectiveness of new medicines. Takeda encourages patients to discuss with their licensed healthcare professional whether participating in a clinical trial could be a potential option for them. Information about Takeda’s open clinical trials is available on the Takeda Clinical Trials website or on www.clinicaltrials.gov.

In some cases, Takeda may be able to provide patients access to its investigational medicines outside of a clinical trial if certain conditions are met. The use of investigational medicines outside of a clinical trial is known as Pre-Approval Access or Expanded Access (sometimes also referred to as compassionate use). A patient’s treating physician may submit a request to Takeda for pre-approval access to an investigational medicine, and Takeda will review each such request taking into account a number of criteria that include but are not limited to:

• Patient must have a serious, life-threatening or severely debilitating disease
• Patient must be unable to participate in a clinical trial due to eligibility criteria or other reasons
• No satisfactory or comparable alternative treatment options are available
• Potential benefits of treatment with the investigational medicine outweigh the potential risks and an appropriate dose has been established, both in the determination of Takeda
• Adequate supply is available to support both clinical development activities and pre-approval access
• Such access must be allowed under local country laws and regulations

To request pre-approval access to an investigational medicine, a licensed healthcare professional should contact Takeda by emailing [email protected]. Takeda will make every effort to respond within three business days.

In addition, more information on existing Pre-Approval/Expanded Access Programs in the U.S. may be found by searching for Expanded Access records on www.clinicaltrials.gov.