Expanded Access (Compassionate Use) Statement
Clinical development is a vital part of our company mission to provide innovative medicines, particularly those that treat rare diseases, to patients with unmet treatment needs. We are committed to bringing safe and effective medicines to patients as quickly as possible and view that participating in clinical trials is the best way for patients to access medicines that are not yet approved by regulatory authorities such as the U.S. Food and Drug Administration. In circumstances when participating in a clinical trial is not possible, patients may seek special access to investigational medicines outside of a clinical trial setting and prior to regulatory approval. These situations are typically referred to as expanded access, also known as compassionate use.
We are and will be evaluating medicines in clinical trials for a range of potential conditions. Additional information on a majority of these trials is posted at www.clinicaltrials.gov.
At this time, we are unable to provide unapproved medicines via compassionate use programs. We have considered many factors, including our ability to maintain supply for the clinical trials, enroll and complete the clinical trials, and make reasonable assessments of potential risk versus benefit for patients outside the clinical trial setting.
As the trials progress and we learn more about these medicines and how they perform in patients, we will periodically re- evaluate the potential for expanded programs and update the physician, patient and advocacy communities.
In the event we decide to consider expanded access use in the future, we will evaluate and respond to each expanded access request on a case-by-case basis. We will consider the nature of the request, the patient’s health, the available medical and scientific information about the investigational product, the balance of risk and potential benefit to the patient, the availability of investigational product and the potential regulatory impact.
For additional information about this policy or if you are a treating physician requesting expanded access, please contact us directly at firstname.lastname@example.org with the basis for the request. We anticipate we will acknowledge receipt within twelve (12) business days or less.