Imagine being a patient – or the family member of a patient – with a debilitating rare disease that does not have an effective treatment. Finally, a new, first-of-its-kind medication is approved by the European Medicines Agency (EMA). Patients in other countries such as France and Germany receive it almost straight away. But, in the UK, you must wait for at least a year or more.
That’s the reality facing many people. In England, the average waiting time from EMA approval until a new medicine is made available to patients is 329 days. For medicines developed for certain life-threatening, rare conditions, the average wait is 362 days. It’s too long, especially for conditions with no – or limited – treatment options.
Around a third of new medicines never make it into the hands of patients at all. Between 2018 and 2021, only 66 per cent of new medicines that received marketing authorisation from the EMA were made available to patients in England via a positive recommendation from NICE (the National Institute for Health and Care Excellence). In 2022, a new Innovative Medicines Fund (IMF) was established, with an annual budget of £340m, to enable faster access to non-cancer medicines, including those for rare diseases. More than a year after its launch, the fund
stands untouched, with no medicines being approved and the promise of faster access for patients unfulfilled. Even once innovative medicines have been approved for use in the UK, uptake – ie their usage by the NHS – remains lower than in many other European countries.
As a result, patients are missing out on potentially life-changing treatment. One analysis of just four disease areas – stroke prevention, kidney disease, asthma, and type 2 diabetes – revealed that approximately 1.2 million eligible patients are currently unable to access innovative treatments, significantly impacting health outcomes and the well-being of the nation.
It’s only two years since the UK government pledged in its Life Sciences Vision to transform the UK into a “life sciences superpower,” utilising “the full breadth of our regulatory freedoms from Brexit” to make the UK the best place in Europe to invest. Not only is this not happening, but we appear to be heading in the opposite direction. How can we arrest this decline and pivot towards where we want to be – a true life science superpower, where citizens have access to the best possible healthcare, including innovative medicines?
In theory, in a post-Brexit world, greater autonomy provides potential for earlier regulatory approval and faster access to innovative medicines. But such improvements remain only theoretical.
In England, both the regulator that licenses the products (the Medicines and Healthcare products Regulatory Agency, or MHRA) and NICE, the health technology assessment (HTA) body that reimburses them, are under tremendous pressure. There is a relative tsunami of innovative medicines for assessment on the horizon, and the novelty and complexity of many of these, particularly for rare diseases, bring challenges. Generating evidence is difficult.
There’s often no standard of care or meaningful comparator. Knowledge and understanding of rare conditions among regulatory and HTA bodies is often understandably limited.
One of the big issues is that the rate of scientific advancement has often outpaced the means of assessment. Innovative treatments are often unsuited for review by rigid and outdated methodologies which have not fundamentally changed for decades. For example, NICE makes recommendations on the reimbursement of new treatments utilising an incremental cost-effectiveness ratio (ICER) threshold range that has been in use since 2004 and has remained unchanged. Even if the NICE threshold had increased with inflation, there would be 12 per cent more reimbursed medicines available in the UK. These medicines offer patients the promise of hope. By not making them available, we are doing those who might benefit from them a huge disservice.
There is some progress in the right direction. On the regulatory side, the new international recognition procedure (IRP) in place from January 2024 will allow the MHRA to take into account the decisions of other trusted regulatory partners around the world to expedite decision-making in the UK. This new procedure has the potential to offer faster routes to market for new medicines.
On the HTA side, in September 2023, NICE agreed new targets: to speed up appraisals by 15 per cent and to publish final guidance 90 days after a UK licence
is granted, which is essentially half of its previous stated target of six months.
Such trends and targets are promising, but are they achievable? Without increasing capacity and resources at both agencies, it could be argued that they’re being set up to fail.
One way to improve patient access to medicines is to give them a voice in the decision-making process. In Scotland, the HTA body (the Scottish Medicines Consortium, or SMC) offers the opportunity to request a Patient and Clinician Engagement (PACE) meeting for the assessment of medicines for rare conditions, which gives patient groups and clinicians a stronger voice. It’s a good example of putting patients at the centre – something that is also key to achieving the life sciences vision.
If the polls are any indication, the current government has little time left in office in which to make an impact. What a great legacy it would be to make a real difference to patients’ lives, improve access to medicines and make progress towards achieving the government’s stated ambition of making the UK a life science superpower.
This article has been commissioned by Ipsen ALLSC-UK-001007 | November 2023