At this time, Ascendis Pharma has one expanded access program available for U.S.-based patients with hypoparathyroidism:
United States Expanded Access Program
The U.S. Food & Drug Administration’s (FDA’s) website states that expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
All requests for access to Ascendis Pharma’s investigational drugs, including Right to Try Act described by the US Federal Food, Drug & Cosmetic Act (FFDCA) section 561B, must be sponsored by a healthcare professional (HCP). Applicable therapies must be in active development with an Investigational New Drug (IND) application. This process will be the responsibility of the Chief Medical Officer of Ascendis Pharma.
Ascendis Pharma will follow all FDA regulations and guidance describing criteria that must be met to qualify for EAP access. A cross functional team within Ascendis will evaluate each application based on the criteria set forth in the document below. Not all EAP requests will be granted.
Ascendis has one Expanded Access Program available for patients with hypoparathyroidism
1. The decision to seek access for treatment utilizing the EAP must be initiated by the patient’s physician. Ascendis Pharma will not incentivize the healthcare provider (HCP) in any way for accessing any Investigational Treatment. The HCP will have sole responsibility and accountability for the clinical management and treatment of the patient.
2. Compliance Considerations:
- Requests for EAP use must comply with local standards of care, along with the laws and regulations that govern the use of any Investigational Treatment.
- An HCP request to access EAP on behalf of a patient may entail disclosure of personal patient medical information for assessment by Ascendis Pharma in order to assess eligibility. All patient information must be redacted so that no personally identifiable information is transmitted.
- Requests require both HCP to initiate the request as well as signed consent by the patient
Criteria for Evaluating Individual Access
HCP Criteria and Responsibilities:
- Must be currently licensed in his/her respective jurisdiction and must not ever have been convicted of a criminal offence relating to any therapy, food, or cosmetic law.
- HCP must not be on the current FDA debarment list or that of any of the relevant local health authorities in the relevant jurisdiction.
- Provide all necessary paperwork to Sponsor for routing to an Investigational Review Board (IRB) or Ethics Committee (EC) as appropriate in their relevant health authority’s jurisdiction.
- The HCP must agree to oversee the patient’s treatment and supervise administration of the Investigational Therapy in accordance with Ascendis Pharma’s defined access criteria and under appropriate regulatory and ethical standards including those set by regulatory authorities and IRBs/ECs.
- The HCP must agree to obtain informed consent and agreement for treatment use of the Investigational Therapy, and collection, analysis, publication, and transfer of any patient data as appropriate from the patient or his/her legal guardians.
- The HCP must agree to maintain and release to regulatory authorities all treatment records and data as required by local regulations.
- The HCP must agree to report adverse events and provide patient level safety data as required by local health authorities and per the agreement with Ascendis Pharma
Patient Eligibility Criteria:
1. A patient may obtain access to an Investigational Therapy for treatment use via EAP only through a licensed HCP.
2. Generally, patient eligibility for consideration of treatment with Investigational Therapy include, but not limited to:
- Patient must be diagnosed with either a serious, debilitating, or life-threatening medical condition.
- A clearly documented biological or clinical rationale should support the potential clinical benefit to the patient.
- Suitable treatment options are not available to treat the ongoing condition OR the patient has a documented attempt at current standard of care but has not responded to treatment.
- The patient does not have an option to participate in a relevant Ascendis sponsored clinical trial because of non-eligibility or the trial is not conducted in the patient's region.
- The patient must have continued access to the HCP for ongoing care.
Requests for EAP must be made by the patient’s treating physician.
If you have any questions about the Ascendis Pharma Expanded Access Policy or wish to make an expanded access request, please contact [email protected].
After receiving an expanded access question or request we aim to have a response back within 10 business days.
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 about our clinical trials programs can be found here: https://ascendispharma.com/patients/clinical-trials/ or on https://clinicaltrials.gov
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.