Expert questions benefits of new UK drug approval pathway

A new UK drug approval pathway, designed to speed up the availability of new medicines, benefits industry over patients and the NHS, argues an expert in The BMJ today.

The pathway aligns regulatory review by the Medicines and Healthcare products Regulatory Agency (MHRA) with the National Institute for Health and Care Excellence (NICE) health technology appraisal process, so that decisions are reached simultaneously. 

But Huseyin Naci at the London School of Economics and Political Science notes that these two bodies require different forms of evidence. The MHRA focuses on a product's clinical effectiveness and safety, while NICE assesses its comparative effectiveness and long term value for money against current NHS treatments.

Rather than aligning evidentiary standards, the pathway runs two independent processes in parallel with the aim of synchronising their conclusions, he explains, offering companies earlier revenue and a longer rebate-free window, while benefits for patients and the NHS are less clear. 

He acknowledges that earlier access to effective new drugs can matter for patients with significant unmet needs, but says, unlike the patient-focused Innovative Licensing and Access Pathway (ILAP), the new pathway expedites all new medicines regardless of added therapeutic benefit. 

The pathway will also impose timelines to ensure that MHRA and NICE decisions are reached simultaneously. Yet Naci points out that fixed drug evaluation deadlines have historically been linked to higher rates of adverse events, and fast-tracked medicines are more prone to post-marketing safety issues than those approved through standard routes. 

A further problem is that NICE committees may be asked to assess products on evidence not yet fully vetted by the MHRA, compounding the uncertainty under which they already operate, he writes.

Earlier adoption of expensive, potentially low-value medicines will also extend the period over which they divert NHS resources from more cost-effective interventions, a problem exacerbated by the 2025 US-UK trade agreement committing NICE to a 25% higher cost-effectiveness threshold, he adds. 

Naci urges the government to ground medicines access policy in patient need and population health, calling for clarification on the assumptions underpinning the pathway and assessment of its anticipated benefits and harms for all stakeholders, not just industry. 

NICE's focus on synchronization of decisions risks overlooking whether the NHS will be adequately protected from the earlier adoption of medicines that prove harmful, ineffective, or poor value, he concludes.