How to request expanded access
If a treating physician believes expanded access may be the only option for a patient, the physician should contact Amryt Pharma to make a formal request on behalf of the patient. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, if applicable, and to comply with all other safety, monitoring, and patient consent requirements.
Requests for expanded access may only be made by licensed physicians, in the country where the patient resides.
If you have any questions about expanded access please contact [email protected]
For any other medical information requests, please contact [email protected]
Expanded access programmes
Amryt Pharma is focused on identifying, developing and commercialising innovative medicines that address unmet medical needs, so people with serious diseases can lead improved lives.
To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, and the data generated from these trials will help us obtain approvals from regulatory authorities around the world. These approvals are generally required before medicines can be made widely available to patients.
There are instances where a patient has a serious disease or condition, for which all currently available treatment options have been exhausted and enrolment into a clinical trial is not possible.
In these instances the patient’s physician may seek expanded access to an Amryt Pharma investigational medicine outside of a clinical trial. Expanded access is referred to in various ways, including but not limited to expanded access, early access, managed access, compassionate use and named patient use.
How expanded access works
Amryt Pharma has engaged a group of clinicians, expert in managing the conditions for which our medicinal products are being studied, to participate in a Governance Review Board.
As appropriate, the Board will review requests for a supply of an Amryt drug through expanded access and give medical advice to Amryt on the benefits and potential risks to the patient. Amryt will then review the request based on this expert guidance and in accordance with the local laws and regulations. Amryt Pharma will endeavour to review and respond to each request as quickly as possible once all of the necessary information has been received.
The following criteria will be taken into account during review:
- The patient must have a serious, life-threatening or severely debilitating disease.
- The patient is ineligible for, or otherwise unable to, participate in a clinical trial.
- No satisfactory or comparable treatment alternatives are available.
- Providing the investigational medicine in an expanded access framework will not interfere with the initiation, conduct, or completion of clinical trials or regulatory approval of the investigational medicine.
- Adequate supply is available to support both clinical development activities and expanded access.
- Expanded access is allowed according to the local laws and regulations in the country where the patient resides.
AMYT/ALL/006