The UK Government has committed to increasing spending on new medicines over the next few years. This is an exciting opportunity, but for NHS patients to reap the benefits of faster access to new potentially life-changing treatments, urgent improvements to medicines policy are needed.
The new Secretary of State for Health and Social Care will be tasked with continuing the department’s work exploring how the government will deliver on its commitment.
We believe that improving the rate of access to “multi-indication medicines” should be an urgent priority for medicines access policy reform.
Without changes to how each indication of these medicines is valued by the NHS, patients – including those with rare and less common conditions who have historically had fewer treatment options – could miss out on this opportunity.
The scientific breakthrough of multi-indication medicines offers real promise for patients
Scientific developments in recent years have found that some diseases have the same underlying biological drivers – meaning that a medicine developed to treat one disease, or “indication”, can have clinical benefit in others too.
Our six companies (Alexion, AstraZeneca Rare Disease; Daiichi Sankyo; Jazz Pharmaceuticals; Johnson & Johnson Innovative Medicine; Novartis; and Sanofi), and the wider life sciences sector, have all invested in this science. Collectively, our group is considering launching approximately 178 indications in England by 2030, across 51 multi-indication medicines.
Of these 178 indications, approximately 52 per cent are to treat cancers, and 31 per cent are to treat rare and ultra-rare diseases and cancers. The remaining indications include long-term diseases, autoimmune conditions and cardiovascular diseases.
This is only a subset of a much larger pipeline of multi-indication medicines across the sector, demonstrating their significance to the future of patient care. This also means these medicines will be significant for the government’s ability to achieve its commitment to increase spending on innovative medicines to 0.6 per cent of GDP by 2036.
But this opportunity is at serious risk of being missed. Identifying, developing and trialing these medicines is just the first part of their journey to NHS patients. The final step is the medicine’s assessment by the National Institute for Health and Care Excellence (NICE) for NHS reimbursement in England – and this is where the system isn’t working for patients.
England recently ranked tenth in full patient access to new medicines (33 per cent of EU-approved medicines being available to NHS patients), falling behind countries including Germany (93 per cent), Italy (73 per cent) and the Netherlands (52 per cent).
We are seeing a concerning trend of a high rate of ‘terminations’ of assessments for multi-indication medicines which means patients are missing out on access – as recently acknowledged by NICE.
Research has found that approximately 829,000 patients in the UK may have lost out on a treatment option between 2014 and 2023 as a result. This amounts to over 16,000 years lived in good health – referred to as “quality-adjusted life years” (QALY) – being lost each year.
Without urgent changes to the way these medicines are currently assessed for NHS use, there is a high risk that this trend will continue and that patients will keep missing out. This is particularly problematic for those who have no other treatment options available on the NHS, like those with rare diseases, of which only 5 per cent have treatments available.
NHS England’s approach to pricing multi-indication medicines isn’t working for patients
A solution does exist: “indication-specific pricing” (ISP). This non-standard pricing option allows eligible multi-indication medicines to have a different price for each indication, to reflect the variation in the value a medicine provides to different patient populations. Otherwise, medicines must have one set price across all indications, which can cause them to be determined as not cost-effective, meaning they are not made available on the NHS.
However, to be eligible for ISP, NHS England currently requires a multi-indication medicine to provide an additional price discount to below the standard cost-effectiveness threshold. This is set out in the “Commercial Framework for New Medicines” document, which is due to be reviewed and updated later this year.
This additional discount is required on top of an already robust evaluation by NICE to determine the cost-effective price of the medicine, undermining this independent process and disconnecting the final price from its value to patients.
More concerning is that this requirement risks creating inequity in treatment access for smaller or rarer patient populations. Higher levels of uncertainty, smaller trial numbers, and limited data for smaller or rarer patient populations often result in cost-effectiveness estimates towards the higher end of the range – which prevents their recommendation for NHS use.
Fundamentally, requiring a price cut to below the NICE standard threshold is a significant barrier to NHS access to a new indication – ultimately to the detriment of patients.
Consistent access to multi-indication medicines must be secured
A new multi-indication medicine could be potentially life-changing for a patient. A technical policy barrier should not get in the way of their access to it.
Addressing this challenge by removing the requirement for an additional price discount to below the standard cost-effectiveness threshold will overcome a significant hurdle to access for patients relying on these medicines in future, and would be a central mechanism for the Secretary of State to support in delivering on the government’s commitments to improve access to new medicines. This will support in bringing about improvements in health, getting people back into work and supporting the NHS.
The Task and Finish Group on Indication Specific Pricing consists of Alexion, AstraZeneca Rare Disease; Daiichi Sankyo; Jazz Pharmaceuticals; Johnson & Johnson Innovative Medicine; Novartis; and Sanofi.
MAX-XU-2602336(v1.0)
Date of Preparation: July 2026
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