Navigating the UK Early Access to Medicines Scheme for Applicants

1. Overview of EAMS

The Early Access to Medicines Scheme (EAMS) is a UK regulatory framework designed to give patients with life-threatening or seriously debilitating conditions access to promising new medicines before they have received full marketing authorisation when there is a clear unmet medical need. Under the scheme, the Medicines and Healthcare products Regulatory Agency (MHRA) issues a Scientific Opinion (SO) on whether the available data supports a positive risk-benefit profile for early patient use.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

The EAMS process is voluntary and does not replace the standard marketing authorisation route. A positive Scientific Opinion supports healthcare professionals to prescribe the product during the EAMS period, helping patients access potentially transformative treatments earlier.
Source: Early Access to Medicines Scheme: Overview

2. Two-Step Evaluation and Application Process

The EAMS follows two key evaluation steps:

Promising Innovative Medicine (PIM) designation

The applicant must first seek a PIM designation, which signals that early clinical evidence suggests the medicine may address an unmet need in a serious condition. A successful PIM application typically leads to continued engagement with MHRA on evidence requirements.
Source: Early Access to Medicines Scheme: Overview

Scientific Opinion (SO) Application

Once PIM designation is obtained, the applicant may proceed to submit a Scientific Opinion dossier. Before submission, the applicant typically requests a Pre-Submission Meeting (PSM) with MHRA to discuss the data package, format, and timing. There is no fee for the PSM. The Scientific Opinion includes clinical and non-clinical data, with dossiers ideally in electronic Common Technical Document (eCTD) format or agreed alternative.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

3. Key Criteria for EAMS Applications

Applicants need to demonstrate that the candidate medicine meets EAMS criteria, including:

• The medicine is intended for a life-threatening or seriously debilitating condition.
• There is a clear unmet medical need with no suitable alternative therapies.
• The medicine shows potential major advantage over existing methods for prevention, diagnosis, or treatment in the UK.
• The potential benefits outweigh known or potential risks, supporting a reasonable expectation of a favourable risk-benefit balance.
• The applicant can manufacture and supply the medicine to consistent quality standards.
  Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

4. Scientific Opinion Validity and Outcomes

A Scientific Opinion is usually valid for one year from issuance. It may expire earlier if:

• A marketing authorisation or relevant extension is granted.
• The MHRA determines the benefit/risk profile is no longer favourable.
  Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

The Scientific Opinion is publicly communicated along with a Public Assessment Report (PAR) and associated treatment protocol, which outlines conditions for safe use and prescribing guidance during the EAMS period.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

5. Real-World Data and Pharmacovigilance

The EAMS guidance encourages the collection of real-world data (RWD) during the EAMS period, which may supplement clinical evidence, particularly for rare diseases or unmet medical needs. While RWD collection is not mandatory, it must focus on clinical management within the scheme and adhere to consent requirements.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

In addition to RWD, applicants must comply with standard pharmacovigilance reporting and periodic reporting obligations for suspected adverse drug reactions and new safety or efficacy information.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

6. End of EAMS Period and Winding Down

When a medicine gains a marketing authorisation (or relevant indication extension), the Scientific Opinion typically ends. However, a winding-down period may be agreed with MHRA to ensure continuity of access for patients already enrolled, up to a maximum of one year. During this period, supply may continue under conditions agreed with MHRA and aligned with updated authorised product information.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

7. Scope and Geographical Coverage

The EAMS applies across the whole of the United Kingdom, including England, Scotland, Wales, and Northern Ireland. Following the Windsor Framework implementation (from 1 January 2025), UK marketing authorisations will be applicable across the entire UK, and EU authorisations will no longer impact EAMS Scientific Opinions within Northern Ireland.
Source: Apply for the Early Access to Medicines Scheme (EAMS) - GOV.UK

8. Conclusion

The Early Access to Medicines Scheme is a structured, transparent process aimed at responsibly enabling early patient access to innovative medicines where there is a significant unmet medical need and a positive benefit/risk expectation. For applicants, early engagement with MHRA, careful planning of evidence submissions, and adherence to reporting requirements are key to successful participation in the scheme.
Source: Early Access to Medicines Scheme: Overview