UK Joins the Early Access Space with its Early Access to Medicines Scheme

A recent report by the British government showed that the rate of uptake of new medicines within the United Kingdom (UK) lags significantly behind a group of sixteen developed countries, including France, Germany, Japan, and the United States. At one year post-launch, the uptake of new medicines averages just 11% that of comparator nations, and only reaches 90% uptake as compared to other nations even after 5 years post-launch. One key driver of this slow uptake is the requirement that drugs be approved by the National Institute for Health and Care Excellence (NICE), the UK’s health technology assessment (HTA) body, which requires proof of a product’s efficacy, safety, and cost-effectiveness prior to reimbursement through the National Health Service (NHS). This approval process can delay patient access to care, depriving patients with potentially life-threatening diseases of access to innovative therapies that have already been approved elsewhere.

Other countries have used early access schemes for years to give patients access to new therapies prior to regulatory and / or HTA approval, such as Law 648 / 96 in Italy, or the Nominative and Cohort Temporary Authorisation for Use (ATU) programmes in France. In contrast, in the UK, limited programmes such as the ‘Specials’ programme (under the Human Medicines Regulations) and the Cancer Drugs Fund were the country’s only options for many years. However, in March 2014 the UK unveiled the Early Access to Medicines Scheme (EAMS) to give patients with life-threatening or seriously debilitating conditions with high unmet need access to innovative medicines that have not yet received marketing authorisation in the UK. In order to obtain access through EAMS, manufacturers must first obtain a Promising Innovative Medicine (PIM) designation by the Medicines and Healthcare Products Regulatory Agency (MHRA). To acquire a PIM designation, a product must (1) be for a condition that is life-threatening or seriously debilitating with high unmet need, (2) be likely to offer major advantage of current treatment methods in the UK, and (3) its potential adverse effects must be likely to be outweighed by its benefits. After achieving this designation, the manufacturer must then complete its clinical development programme for the product within a reasonable time period, at which point it will advance to the scientific opinion assessment step. So far, only 7 therapies have received a PIM designation, including ZYKADIA (ceritinib), OPDIVO (nivolumab), and KEYTRUDA (pembrolizumab); so far, only KEYTRUDA has received a positive scientific assessment by EAMS.

EAMS will enable patients in the UK to more quickly obtain access to treatments that substantially improve survival and quality of life; additionally, there are several potential benefits that manufacturers should consider from having their drugs evaluated through EAMS. First, the process is relatively inexpensive (£4,027 for PIM and £29,000 for ‘scientific opinion’, renewal after 1 year is £14,500); small and large manufacturers alike can leverage a positive PIM designation as a signal to investors that a therapy holds promise from a regulatory perspective. In addition, the process enables manufacturers to receive early feedback and advice regarding a product’s efficacy and safety profile, as well as collect valuable real-world evidence quickly prior to a formal appraisal by NICE. However, these benefits must be taken into consideration that manufacturers will be required to provide the product and any necessary non-standard diagnostic technology to patients free-of-charge. Ultimately, time will tell regarding how early access through EAMS translates to improving success for reimbursement via appraisals by NICE.