Corporate Policy Statement on Expanded Access to Millendo Investigational Medications
Millendo Therapeutics, Inc. (Millendo) is focused on the development of new treatments for orphan endocrine diseases where current therapies do not exist or are insufficient. Although the optimal way of accessing such investigational drugs is through participation in clinical trials, we acknowledge that there are patients with these types of conditions who do not qualify for enrollment in a currently active clinical trial. Therefore, it is the policy of Millendo to evaluate on a case-by-case basis all requests for access to our investigational medications. This policy may be updated from time to time as new data and information become available.
Seeking use of an unapproved, investigational medication is permitted by many regulatory agencies, including the U.S. Food and Drug Administration (FDA) in the United States. The procedures to be used are specified by regulation and are commonly referred to as "Expanded Access" or "Compassionate Use" The purpose of this policy is to provide a way for patients and their physicians to learn whether it is possible to be treated with an investigational medication that is being developed by Millendo.
Expanded Access: use of an unapproved, investigational medication outside of the context of a clinical trial, where no other therapeutic alternatives exist; Expanded Access is regulated by FDA in the United States.
Compassionate Use: has the same meaning as “Expanded Access” and is a term more commonly used outside the United States.
Criteria Used by Millendo to Evaluate Requests for Expanded Access:
A number of important factors must be considered when evaluating a request for Expanded Access. A patient will be considered for Expanded Access when the following criteria are met:
The request for Expanded Access is submitted by the treating physician, who is appropriately licensed;
The patient’s condition is serious or life-threatening;
Participation in a clinical trial is not an option, either because the patient is not eligible to enroll or because there are no appropriate ongoing clinical trials;
There is agreement between the patient’s treating physician and the medical lead at Millendo that use of the investigational medication has reasonable potential to provide a benefit to the patient;
The proposed dose of the investigational medication is within the existing dose range for which human safety data are available; and
The drug product required is available for treatment use without compromising supplies that have been designated for other uses.
Response to Requests for Expanded Access:
To the extent possible, Millendo will provide written responses to requests for Expanded Access within 15 business days of receipt of each request.