Early Access: A Guide to Named Patient Sales, Compassionate Use & EAMS
A Guide to Early Access: Navigating Named Patient Sales, Compassionate Use & EAMS
What happens when a promising medicine is approved in one country (like the US) but not yet in the UK or EU? For patients with high unmet needs, waiting is not always an option. This is where ‘early access’ pathways come in.
However, the landscape is a minefield of confusing terms: Named Patient Sales, Compassionate Use Programmes, EAMS, ‘Specials’. They all mean different things, involve different stakeholders, and carry different legal responsibilities. This guide clarifies the key pathways for providing unlicensed medicines to patients in the UK and Europe.
The 3 Key Pathways: Who, What, and Why?
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1. Named Patient Program (NPP) |
A “one-off” supply for a single, individual patient. |
The Physician |
The Physician (under their direct professional responsibility). |
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2. Compassionate Use Program (CUP) |
A structured program to provide a drug to a group (cohort) of patients. |
The Company |
The Company (Sponsor). |
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3. Formal Access Scheme (EAMS) |
A formal, regulatory-assessed scheme for a cohort of patients (UK-specific). |
The Company |
The Company (Sponsor), with MHRA oversight. |
Early Access in the UK: EAMS vs. “Specials”
In the UK, the ‘Named Patient Program’ is known as the ‘Specials’ scheme. This is a reactive pathway. It happens when a doctor identifies a specific patient who could benefit from an unlicensed medicine (like a drug approved in the US) and takes personal responsibility for prescribing it. The doctor makes an unsolicited request for a “special” supply. This is done on a case-by-case basis and is not a mechanism for broad marketing or promotion.
The “Company-Led” Route: The Early Access to Medicines Scheme (EAMS)
EAMS is a proactive, formal system run by the MHRA. A company can apply for this before a full marketing authorisation. It’s a two-step process:
- PIM Designation: The company first gets a “Promising Innovative Medicine” (PIM) designation.
- Scientific Opinion: The company submits data for an MHRA scientific opinion. If positive, the drug can be made available to a defined cohort of patients.
This is a powerful, structured pathway that can generate data and lead to a smoother full-approval process.
Early Access in the EU: CUPs vs. NPPs
The “Company-Led” Route: Compassionate Use Programs (CUPs)
“The EU framework allows for ‘Compassionate Use Programs’ (CUPs) for cohorts of patients. Unlike the UK’s EAMS, there isn’t one single EU-wide program. The EMA’s CHMP can provide an opinion on a CUP, but the programs themselves are run and approved at the national level by individual Member States (e.g., France’s ATU, Germany’s program). This requires a coordinated, country-by-country strategy.
The “Physician-Led” Route: Named Patient Programs (NPPs)
Similar to the UK “Specials” scheme, all EU member states have a provision (under Article 5 of the directive) that allows a physician to request an unlicensed medicine for an individual, named patient under their direct responsibility. This is the ‘Named Patient Sale’ pathway. The rules, paperwork, and reimbursement vary significantly between countries like Germany, France, Italy, and Spain.
Case Study
Let’s take a real-world example of a monoclonal antibody with US Emergency Use Authorization for pre-exposure prophylaxis (PrEP) of COVID-19 in immunocompromised patients. As this product is not authorised by the MHRA or EMA, it cannot be actively marketed.
However, an expert consultant (like a haematologist or immunologist) in the UK or Germany may identify a specific high-risk patient who could benefit. That doctor, under their own responsibility, could make an unsolicited request for a Named Patient Sale. This is a reactive supply. Managing these one-off requests requires a compliant, agile, and logistically sound system.