Earlier this month, Sanofi’s atopic dermatitis drug, dupilumab, became the latest drug to be approved for the Early Access to Medicines Scheme (EAMS) in the UK. But what exactly is the EAMS, and how do drug companies get their drugs included in this scheme?

The EAMS was established in 2014 to help patients with serious or life-threatening conditions, who have no viable treatment options available to them, access promising new medications that are not yet licensed in the UK. The scheme has now been running successfully for almost three years, and demonstrates a commitment from the UK government as well as industry to bringing medication to patients at a faster rate than ever before.

The scheme is voluntary for pharmaceutical companies and sits alongside existing legislative frameworks that allow unlicensed medicines access.

Typically the EAMS process is applied for after medicines have completed PH III trials, although applications can be accepted after PH II trials in exceptional circumstance, provided sufficient safety & efficacy data can be submitted.

The Medicines and Healthcare products Regulatory Agency (MHRA) is charged with evaluating EAMS submissions and granting EAMS status. The MHRA opinion will last for one year, although it can be renewed, and it in no way affects or replaces the current licensing procedure for drugs in the UK.

For companies interested in applying to the EAMS, there is a two-step evaluation process. The first step for a company is to receive a Promising Innovative Medicine (PIM) designation. This designation will act as an indication that a drug is a promising candidate for the EAMS, and will be issued after an MHRA scientific meeting. The MHRA will base its decision on the early non-clinical and clinical data available on a new drug.

In order to receive a PIM designation, three criteria must be met:

  • The condition the drug is intended to treat must be either life-threatening or seriously debilitating
  • The product must be likely to offer a major advantage over the approved medications currently available in the UK
  • There must be a positive benefit risk balance, meaning the potential benefit of taking the product must outweigh the potential risks

To apply, companies must complete the PIM application form, indicating how their product meets the aforementioned criteria. To download this form, and receive further submission guidelines, visit the GOV.UK website.

A company will be notified of its application approval or rejection via a designation letter. If a company receives a positive PIM designation, it will have to complete a clinical development programme before moving on to the second evaluation process – the EAMS scientific opinion assessment.

In addition to having a PIM designation, companies applying for an EAMS scientific opinion must also attend a pre-submission meeting with the MHRA. The purpose of this meeting is to evaluate whether the suitability criteria for the scheme are likely to be met and explain the format of the data required to be submitted to support the MHRA’s benefit/risk opinion. Based on the outcome of this meeting, the MHRA will either designate the product a suitable candidate for the EAMS scientific opinion, or will reject the product and terminate the application process.

To apply for a pre-submission meeting, companies must submit a pre-submission meeting request form, which can also be found on the GOV.UK website.

Following a pre-submission meeting, companies with a suitable candidate must then apply for a scientific meeting by submitting the following documents:

  • A scientific opinion application form
  • A cover letter
  • A summary of the pharmacovigilance system master file
  • A risk management plan based

A company will be notified of a positive or negative scientific opinion within 90 days of the submission date. Positive scientific opinions will be published on the MHRA website. Negative opinions will not be published and there will be no right of appeal.

The opinion lasts one year, although it can be renewed. Companies should request a renewal at least two months prior to the expiry of the existing opinion. The MHRA can withdraw a positive scientific opinion at any time, if following scientific assessments show that the benefit risk balance is no longer favourable.

Any drug provided through the scheme must be provided for free. When a drug included in the scheme receives marketing authorization, it will then be appraised for use by the National Institute for Health and Care Excellence (NICE).

The EAMS process can be supportive to a drug development strategy, as the EAMS endorsement provides an independent and objective evidence base review by a regulator and allows earlier engagement with payers in the UK.

For further information, and to view the number of applications currently pending, refused, and granted for the EAMS, visit the GOV.UK website.