New Cardiovascular Drug Bucks Competitive Oncology-Only Trend in UK Early Access to Medicines Scheme

One year on since the launch of the UK's Early Access to Medicines Scheme (EAMS), Novartis’ investigational heart failure drug LCZ696 (sacubitril/valsartan) has become the fifth therapy to be awarded a 'promising innovative medicine' (PIM) designation. Significantly for health technology manufacturers, LCZ696 is the first non-oncology therapy to receive a PIM, which, as Dr Steven Bradshaw and Dr David Carr of Market Access Solutions (MKTXS) discuss, is an important step for broadening early access to a wider range of medicines.

The EAMS is a venture from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) aimed to speed up access to as yet unlicensed medicines where there is a high unmet medical need. The first in a two-step process, PIM designation recognizes a product as a good candidate for the EAMS based on phase I/II data. Following this, the candidatemust go through an 'EAMS scientific opinion' where the risk-benefit profile is evaluated based on further evidence generation. Only then, with a positive assessment, will the MHRA recommend its use before the full licence is approved.

The scheme offers several advantages to manufacturers of innovative medicines. The EAMS provides a way to enter the market before competitors and enables real-world data collection earlier to aid HTA evaluations. Manufactures also have the reassurance that the process is confidential, so failed applications will not be publicised.

"The EAMS offers a way to enter the market before competitors and enables real-world data collection earlier to aid HTA evaluations."

Prior to this latest designation, only oncology therapies had gained entry into the scheme, with the first awarded in September 2014 to DCVax®-L, a brain cancer vaccine developed by Northwest Biotherapeutics. More recently, Novartis' Zykadia® (ceritinib) for anaplastic lymphoma kinase positive advanced non-small cell lung cancer (ALK+ NSCLC) gained a PIM. However, it is in the anti-PD-1 space that sees the most competition, with both Bristol-Myers Squibb's Opdivo® (nivolumab) and Merck's Keytruda® (pembrolizumab) in the mix for advanced melanoma. Here, Keytruda® has  outdone its rival by receiving a positive scientific opinion in the second step of the process and has become the first drug available to UK patients as part of the EAMS.

"While the EAMS is in place, the manufacturer must provide the therapy free of charge."

The commissioning process runs in parallel to the EAMS via NHS England, with Clinical Reference Groups (CRGs) steering how and where the medicines are used. Importantly though, while the EAMS is in place, the manufacturer must provide the therapy free of charge. So, while manufacturers would hope to recoup this cost through expedited access, on the other hand, this needs to be balanced by the possible expectation of commissioners that price reductions would be offered through the avoidance of expensive late-phase clinical trials. Manufacturers will then have to consider the impact that UK access decisions have on other markets, bearing in mind that the medicine may already be priced and available in some regions, as is the case for Opdivo® and Keytruda® in the US.

As for the early access landscape outside of oncology, the PIM designation for LZ696 is a signal to manufacturers that the EAMS is not just for high-cost cancer medicines. Plus, the fact that Novartis has followed up its February FDA priority review of LZ696 quickly with PIM designation in the UK, is a sign that access strategies are aligning across the Atlantic, which is encouraging for patients in the UK, who often face a longer wait for innovative treatment than their overseas counterparts.

Altogether, the EAMS offers health technology manufacturers an attractive opportunity to speed up market access in the UK; one that needs to be navigated with ever greater rapidity and clarity.

Dr Steven Bradshaw is the European Director of Market Access Solutions (MKTXS)

Dr David Carr, PhD is a Market Access Analyst at MKTXS

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