At Ascendis Pharma, patients and their families are at the heart of everything we do. We believe clinical trials are the primary route for patients with serious or life-threatening diseases who are seeking to access our investigational medicines.
However, there may be situations where a patient with a serious or life-threatening condition may not be eligible for an Ascendis Pharma-sponsored clinical trial and may seek access to an investigational medicine outside of a clinical trial setting.
At this time, Ascendis Pharma has no expanded access programs for our investigational medicines. Nonetheless, we will evaluate and respond to expanded access requests on a case-by-case basis, according to Ascendis Pharma policy set forth below.
Although Ascendis Pharma retains sole discretion in deciding whether to grant an expanded access request, the following criteria will be taken into consideration when evaluating such requests:
- The patient suffers from a serious, debilitating or life-threatening illness or condition within the disease areas Ascendis Pharma is investigating, and the efficacy and safety data available at the time are sufficient to determine whether the potential clinical benefit to the patient would likely outweigh the possible risks, including the outcome of the disease.
- The patient is not eligible for an ongoing or planned Ascendis Pharma-sponsored clinical trial.
- Providing access to the investigational medicine must not interfere with the completion of a clinical trial supportive of a Health Authority approval or compromise the potential development of the medicine. Sufficient efficacy and safety data are available to indicate that the potential benefits of access to the investigational medicine in the disease condition may outweigh any potential risks.
- The patient has exhausted all available medicines typically used to treat the disease and is no longer responsive to, or able to tolerate, these treatments. The patient does not qualify for ongoing clinical trials sponsored by Ascendis Pharma due to eligibility criteria or geographic location.
- There must be sufficient clinical data to identify an appropriate dose for the proposed use.
- There must be sufficient supply of the investigational medicine to meet the needs of the expanded access program without impacting any Ascendis Pharma clinical trials.
- The patient meets any other relevant medical criteria for expanded access to the investigational medicine, as determined by Ascendis Pharma.
An expanded access request must provide sufficient supporting information to enable Ascendis Pharma to evaluate your request. Please also include your contact information so that we may follow up with you directly.
If you have any questions about the Ascendis Pharma Expanded Access Policy or wish to make an expanded access request, please contact email@example.com.
We anticipate confirming receipt of any expanded access questions or requests within 10 business days of receipt.
As authorized by the 21st Century Cures Act, Ascendis Pharma reserves the right to revise this policy at any time. Additionally, the posting of this policy by Ascendis Pharma shall not serve as a guarantee of access to any specific investigational medicine by any individual patient.
Information on our clinical trials programs can be found here: https://ascendispharma.com/patients/clinical-trials/
Please continue to check our website for updates to this policy, which will be reevaluated on an ongoing basis.
We make our business decisions based on patient needs and we do our best every day to realize our products’ benefits for patients.