Access to unlicensed or investigational medicines outside of clinical trials prior to marketing authorization plays a critical role in addressing urgent, unmet clinical needs in patient populations. Within the European Union (EU), such access is governed by defined regulatory mechanisms designed to ensure patient safety while providing flexibility in exceptional circumstances.
Two commonly used pathways for early access are Named Patient Programs (NPPs) and Compassionate Use Programs (CUPs). While both enable access to unapproved therapies, they differ in scope, regulatory requirements, and implementation. This article outlines the key distinctions between NPPs and CUPs, with focus on the EU context.
What is a Named Patient Program?
A Named Patient Program (NPP) is a regulatory pathway, regulated as an ‘exception’ to the premise of Directive 2001/83/EC, referenced under Article 5, that permits the supply of a medicinal product to a healthcare professional for the treatment of an individual patient, prior to the product’s authorization in that country, i.e. unlicensed.
This program is used in cases of serious or life-threatening conditions where no suitable alternative treatments are available. Access is granted on a named patient basis under the responsibility of the prescribing physician and is subject to national regulatory frameworks and specific conditions set by the competent authority.
What is a Compassionate Use Program?
A Compassionate Use Program (CUP) provides access to an unlicensed medical product intended for conditions that are life-threatening, chronic, or severely debilitating, to a group of patients.
CUPs are designed to support patients who are ineligible for on-going clinical trials and cannot be treated with any approved alternatives or when clinical trials have ended and to allow a patient to still receive the investigation product. The medicinal product offered in a CUP must not be advertised, and the treatment decision must remain unsolicited.
Although regulated under a common EU Regulation – Article 83 of Regulation (EC) No 726/2004 – the regulatory implementation of CUPs is down to each national competence authority (CA), thus the application process, submission requirements, approval timelines, and other implementation aspects of CUPs can vary significantly across European countries.
Pre-requisites of a CUP
As stipulated by the European Medicines Agency (EMA) a CUP can only be established if the medicine meets the following pre-requisites:
- The medicinal product in question must either be undergoing a clinical trial on the same indication in an ICH country or be the subject of a marketing authorization application for the same indication.
- A reasonable safety profile must be established (usually through early-stage clinical trial, e.g. Phase II).
Deciding Which Pathway to Choose
Deciding which pathway to choose requires consideration of multitude of factors, including regulatory requirements, Sponsor expectations, and patient needs. WEP’s Regulatory team has put together a helpful resource outlining a general decision tree and some key differences in these 2 pathways.
Click here to access the decision tree.
Conclusion
The regulatory landscape in Europe for unlicensed supply, while regulated more so than other regions, is still heterogenous at a national level. EMA recommendations premised on the EC Directives and Regulation serve as a general guideline, and each Member State leverages its own national regulations when implementing these programs.
At WEP, our Regulatory team has the expertise and legislative intelligence on all unlicensed supply globally, including the EU Member States, and help our clients navigate these varied regulatory landscapes, developing tailored regulatory strategies that meet client needs as well as maintain regulatory compliance, with the ultimate goal of serving patients who are in need of these life-saving treatments.
