Understanding Early Access, Compassionate Use, and Exempt Medicinal Products (EMPs) in Ireland

The Health Products Regulatory Authority (HPRA) plays a central role in enabling patient access to unauthorised medicines in Ireland. This access is tightly controlled and occurs only under specific pathways designed to support patients with serious or unmet medical needs. This article outlines the key mechanisms – Early Access Programs, Compassionate Use Programs, and Exempt Medicinal Products (EMPs) – and clarifies what Qualified Persons (QPs) need to know when involved in EMP supply and oversight.

1. Early Access and Compassionate Use Pathways

Early Access Programs allow patients with unmet medical needs to receive unauthorised medicines when no suitable authorised alternatives exist. These medicines must be undergoing clinical trials or in the process of a marketing authorisation application.

Compassionate Use Programs support patients with life-threatening, chronic, or severely debilitating conditions. They provide access to unauthorised medicines outside clinical trials and are coordinated at EU Member State level.

2. Exempt Medicinal Products (EMPs): What Are They?

EMPs are medicines not authorised in Ireland but supplied to meet the specific clinical needs of an individual patient. They may be authorised elsewhere, under clinical trials, or under review for marketing authorisation. EMPs are supplied only in response to a genuine, unsolicited request from a prescriber for a named patient when no authorised alternative exists.

Although these products have not been approved by the HPRA for quality, safety, or efficacy, in many cases EMPs are a licensed product from another market. As such, these medicines will have undergone an evaluation of their safety, quality, and efficacy as part of their marketing authorisation approval in that jurisdiction, carried out by the respective competent authority for medicines in that market.

3. EMPs vs Investigational Medicinal Products (IMPs)

It is critical to distinguish between Investigational Medicinal Products (IMPs) and Exempt Medicinal Products (EMPs). IMPs are intended for patients enrolled in HPRA-approved clinical trials. To supply these, a company must obtain a dedicated Manufacturer's/Importer's Authorisation (MIA(IMP)) or a variation to an existing MIA.

Conversely, EMPs are medicines lacking a national marketing authorisation in Ireland, though they may be licensed in other jurisdictions (e.g., the USA) or be currently undergoing clinical trials or regulatory review. Unlike IMPs, EMPs are intended for patients who are not currently part of a trial but require treatment for life-threatening or severely debilitating conditions. EMPs are supplied through Member State-coordinated Managed Access Programs, which operate under specific national rules and are frequently provided to patients free of charge.

EMPs are generally approved and QP Released in other jurisdictions as commercial products, whereas IMPs are QP released for specific clinical trials under an IMP MIA.

4. Legal and Regulatory Framework for EMPs

The supply of EMPs in Ireland is governed by the Medicinal Products (Control of Placing on the Market) Regulations 2007 and the Medicinal Products (Control of Wholesale Distribution) Regulations 2007.

Prescribers assume personal responsibility for prescribing EMPs. When a valid prescription for an EMP is issued in accordance with these Regulations, an authorised wholesaler may source the product from another market and supply it to the patient through their pharmacy, in line with that prescription.

5. HPRA Notification Requirements for EMPs

Wholesalers and manufacturers sourcing EMPs must notify the HPRA of each importation and report adverse reactions. Notification is completed via the Exempt Products Notification System (accessed via the HPRA website) within two working days of receiving the product.

6. What QPs in Ireland Need to Know About EMP Release

In Ireland, an MIA is required for onward supply of unauthorised products. Importation of unauthorised products (specifically EMPs) is only permitted under a WDA if it is to be supplied to Ireland only.

Regarding the level of QP review, QP(s) listed on the MIA are expected to maintain oversight and responsibility of the activities being performed under the scope of the MIA, but specific requirements regarding review, release, and certification of product are dependent on the national requirements for the countries to which the unauthorised products are being supplied. EMP-related activities are typically recorded under sections 1.4 or 2.3 of the (commercial) MIA application.

Summary

Ireland provides several controlled pathways for accessing unauthorised medicines. EMPs play a vital role in enabling access to treatments not yet authorised in Ireland. Understanding the regulatory framework, notification requirements, and QP responsibilities is essential for compliant supply.

If your organisation requires support with WDA or MIA applications, EU QP oversight for EMP supply, or any aspect of GMP or GDP compliance, our team is here to help. We work closely with partners across the sector to provide practical, reliable quality solutions tailored to their needs.

If you'd like to explore how we can support your operations, we'd be delighted to discuss your requirements and see how we can assist.