MHRA launches EAMS consultation

The UK MHRA has launched the Early Access to Medicines Scheme (EAMS) consultation.

What is the EAMS scheme?
  • The Early Access to Medicines Scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.
  • The scheme was launched in April 2014 following a public consultation and government response. Under the scheme, the MHRA gives a scientific opinion on the benefit/risk balance of using a medicine.
How does the scheme work?

EAMS is essentially a two-step process:

Step 1 – Promising Innovative Medicine (PIM) designation
  • PIM designation is the first step.
  • It gives an indication that a product may be eligible for EAMS patient access based on early clinical data.
  • It is issued by MHRA after an MHRA scientific meeting.
Step 2 – the EAMS scientific opinion
  • When a company has sufficient data to support patient access, they apply to MHRA for the second step – the EAMS scientific opinion.
  • This considers the risks and benefits of new chemical and biological entities (unlicensed medicines) or new indications of already marketed drugs (off-label use setting), taking into account the assessment of available quality, non-clinical and clinical data.
  • The scientific opinion supports the prescriber and patient in deciding on whether to use a medicine before its licence is approved.
  • Since launch there have been over 100 PIM designation and 50 SO applications.
  • A positive scientific opinion is only issued if the criteria for EAMS are fulfilled.
  • All EAMS scientific opinion applications are reviewed by the independent Commission on Human Medicines (CHM), an advisory non-departmental public body, sponsored by the Department of Health and Social Care (DHSC).
  • Positive scientific opinions are published on the MHRA website alongside a public assessment report and the EAMS Treatment Protocols, describing for healthcare professionals and patients how to use the medicine.
Why EAMS has to change

EAMS provides an important regulatory flexibility for early patient access. Significant numbers of patients have benefited in a variety of conditions since 2014. However, changes are now needed to enhance the flexibility of the scheme and maximise benefits to patients and participating pharmaceutical companies, ensuring that EAMS remains an attractive option for companies to provide medicines to patients prior to licensing.

What is the consultation about?

The aim of the consultation proposal is to ensure that EAMS remains an attractive option for patients, healthcare professionals and companies, so that cutting-edge therapies are available for patients where there is an unmet clinical need.

Currently, there is no UK legislation that specifically covers EAMS. The scheme is entirely non-statutory. Obtaining an EAMS approval from the MHRA is effectively delivering a scientific opinion. The opinion provides independent reassurance from the medicines regulator that the benefits outweigh the risks.

Since there is no legislative provision that covers or specifically mentions EAMS, there are some aspects that would benefit from clarification in law, providing a legal basis for the delivery of EAMS. The MHRA would like to introduce new provisions into the Human Medicines Regulations 2012 (HMRs) in order to:

  • Provide a specific statutory basis for EAMS, therefore improving regulatory certainty regarding this important patient access flexibility and reducing regulatory burden
  • Address a key recommendation from an independent review on EAMS on data collection.

To achieve the above, the MHRA are proposing to introduce a specific EAMS legal provision that will describe the scheme and its goals. This aims to:

  • provide legal clarity for those companies who may be considering using EAMS
  • to provide a framework on the requirements for safety monitoring and collection of data.

MHRA’s objective is to reduce regulatory procedural burden where possible whilst supporting earlier patient access in areas of unmet medical need where the benefits have been shown to outweigh the risks, and the risks are monitored.

Doing the above would minimise the burden on those supplying EAMS medicines and for those companies wishing to collect real-world data during the scheme. This will be delivered whilst continuing to ensure the safety of EAMS products through pharmacovigilance (safety monitoring), maximising patient access and benefit.

MHRA considers that the provision will include the following:

  • EAMS key principles of operation -To describe the objectives of EAMS and clearly detail its principles of operation
  • Simplifying the supply of EAMS medicines -A new provision for EAMS medicines is proposed to simplify and harmonise expectations for their manufacture, assembly and importation. A flexible approach is proposed, providing a statutory basis for EAMS supplies that avoids the need to classify supply aspects under existing legislation for unlicensed medicines or off-label provision
  • Provide a supportive framework the collection of real-world data (RWD) – In order to support the collection of real world data, legislation is proposed to allow a company which holds an EAMS scientific opinion to be able to collect data in the context of EAMS regardless of whether the product is considered an investigational medicinal product or not
  • Clarifying the liability for prescribers and patients -The MHRA would like a provision in legislation to provide clarity around the use of EAMS medicines, in line with the General Medical Council (GMC) recommendations on prescribing unlicensed or off label medicines:
  • Pharmacovigilance (safety monitoring) for EAMS medicines – In order to best support safe use and to reassure the individual patient and the healthcare professional when prescribing an EAMS medicine, several pharmacovigilance requirements are being considered for inclusion in legislation. They are, the requirements to:
    • to operate a risk management system describing appropriate pharmacovigilance and risk minimisation activities
    • collate and maintain records of suspected adverse drug reactions and report to the MHRA
    • submit periodic reports summarising the suspected adverse drug reactions for the EAMS medicine
    • notify MHRA of new information that may impact the benefit/risk balance.

You can read more about the consultation proposals (and questions) here.

Responding to the consultation

You can respond to the consultation questions here.

The consultation will close on 17th September 2021.

Source: MHRA website