Provision of unlicensed medicines

This webpage outlines Norgine’s stance on the provision of unlicensed medicines for unmet medical needs.

Norgine’s primary focus is to enrol patients in clinical trials to acquire the necessary data for marketing authorisation approval and reimbursement. Norgine prioritises clinical trial enrolment and acknowledges that regulatory authorities’ approval is the sole pathway to make medicines widely accessible to patients under qualified healthcare professional supervision.

Norgine also recognises the importance of providing access to unlicensed medicines that are not yet approved and/or reimbursed for patients with unmet medical needs. Our policy and programmes may provide an option for providers and patients faced with these challenges.

This includes:

• Norgine Expanded Access – the provision of a medicine in development following an unsolicited request from a healthcare provider prior to the time that the drug is licensed and available in the local market for the requested indication.
• Requests for commercial availability for treatment access to a medicine that is licensed in some countries but not licensed where the treatment would occur.

 

Norgine Expanded Access

Provision of investigational medicines for unmet medical needs may be referred to as single patient use, named patient use, compassionate use, expanded access, special access, managed access, importation of unlicensed medicine, or other names. At Norgine, this provision is referred to as ‘Expanded Access’.

All requests for therapeutic use of a Norgine investigational medicine must be made by a healthcare professional (HCP), or a health authority or healthcare provider.

Decisions to supply medicines under Norgine Expanded Access programmes are made by Norgine senior medical staff only, based on medical and ethical criteria.

For an expanded access request to be considered, they must meet all the following criteria:

• Present a serious or life-threatening medical condition.
• No comparable or satisfactory alternative treatment options are available for that specific patient.
• Have exhausted all available treatment options and lack access to ongoing clinical trials for the investigational medicine (e.g. be unable to participate in a clinical trial due to specific eligibility criteria or geographic constraints).
• There is sufficient evidence to believe the potential benefit to the patient justifies the potential risk.
• Fulfil the eligibility criteria established by the company medical team. 

Other criteria considered in requests for investigational medicines:

• The expanded access request should not compromise the ongoing clinical development and/or marketing authorisation application.
• Request for use in larger access programmes will only be considered in countries where Norgine plans and/or has ongoing efforts in place for applying for marketing authorisation and/or reimbursement for the investigational medicine (not necessarily applied for named (or individual) patients)
• Ethical considerations relating to fairness to similar patients
• The proposed therapeutic use complies with local laws and regulations.
• Norgine is legally able to supply the investigational medicine in the requested territories.

These criteria help Norgine ensure a consistent approach for our decisions on therapeutic use of our unlicensed medicines. However, the decision to fulfil the request lies ultimately at the sole discretion of Norgine.

At the point that an expanded access request is received, there may be limited information about the benefits and risks related to its use. Early studies relating to how the body handles the medicine, and its toxic effects will usually have been completed, however, other studies which provide further information on the medicine may not yet be complete. This means that there could be limited information on how to use the medicine, the exact dose to use, and full information on the efficacy and safety (side effect) profile. As such, careful and detailed assessments must be undertaken to determine whether it is appropriate to make the medicine available to a specific patient at this stage.

 

Requests for commercial availability for treatment access to a medicine that is licensed in some countries but not licensed where the treatment would occur

Norgine can also consider requests for treatment access to a medicine that is licensed in some countries but not licensed where treatment would occur.

Requests for commercial availability of unlicensed medicines must be from a HCP, Healthcare organisation or government health department and be received via an official email address or on headed paper. Decisions on supply are based on availability of supply, distribution considerations and appropriate assessment of country of request.

 

Job code: UK-COR-NP-2400008 Date of Prep: October 2024