Pembrolizumab, a treatment for advanced melanoma marketed by Merck Sharp & Dohme, has become the first drug to be approved by the MHRA under the Early Access to Medicines Scheme (EAMS). The EAMS, on which we reported in our April 2014 newsletter, is designed to provide earlier availability of promising new medicines that do not yet have a marketing authorisation, to UK patients who have a pressing unmet clinical need. The MHRA's positive opinion on prembrolizumab is given for use in melanoma patients after other treatments have failed.

The approval process for the EAMS involves two stages of evaluation:

  1. the applicant seeks a "Promising innovative medicine" (PIM) designation, which indicates that the MHRA believes the product may be eligible for the EAMS based on early clinical data (normally data from Phase III, and exceptionally Phase II, clinical trials); and
  2. once the PIM designation is obtained, the MHRA, if it deems appropriate, issues a scientific opinion describing the risks and benefits of the medicine based on the data gathered. Prior to the scientific opinion, a pre-submission meeting takes place between the applicant and the MHRA to ensure that the product is suitable for a scientific opinion application, and to discuss the format of the data the applicant will need to submit to support the opinion.

The MHRA will then issue a public assessment report (PAR), which includes the following details:

  • how the product is used and how it works
  • summary of the key clinical studies
  • the risks and benefits of the product
  • the reason for the positive EAMS scientific opinion
  • any uncertainties
  • information about ongoing clinical studies
  • measures in place to monitor and manage risk

It is a condition for approval under the EAMS that the applicant provides the treatment to the NHS free of charge for as long as it is in the scheme.

The PIM was awarded in respect of pembrolizumab on 10 October 2014 and the MHRA issued a positive scientific opinion on 9 March 2015. The EAMS itself was launched in April 2014. The MHRA has so far issued five PIMs, so there are likely to be further approvals for the EAMS pending. We will continue to monitor developments on this front.