An update on access to medicines policy for rare conditions
A flurry of policy activity is underway that concerns access to innovative new medicines in the UK. We know this is atopic of importance for many of our member organisations, including the MND Association, which recently authored an article on challenges it had experienced with Early Access to Medicines Schemes (EAMS).
A flurry of policy activity is underway that concerns access to innovative new medicines in the UK. We know this is atopic of importance for many of our member organisations, including the MND Association, which recently authored an article on challenges it had experienced with Early Access to Medicines Schemes (EAMS).
Genetic Alliance UK has gathered a number of case studies like this from across our community to share with MPs via the Westminster All Party Parliamentary Group (APPG) on Genetic, Rare and Undiagnosed Conditions, and held two meetings focused on these access challenges in more depth (July 2025 and February 2026).
In light of this, we felt it would be helpful to outline some of the key announcements from the last few weeks, and how they may impact people living with genetic, rare and undiagnosed conditions. In short, while the direction of travel is positive, many details are yet to emerge and Genetic Alliance UK is continuing to monitor these changes closely.
NICE updates: methods and an increased willingness-to-pay
To coincide with Rare Disease Day 2026 in February, the National Institute for Health and Care Excellence (NICE) launched the first Quality Standard for Rare Diseases. Since then, it has outlined two changes concerning the methods that NICE uses for health technology assessments (HTA) of new medicines.
- 25 March: A change to how health-related quality of life is measured was shared. Moving from a three- to a five-level structure (EQ-5D-5L), the change aims to improve the sensitivity in how outcomes are measured. Register to attend a webinar hosted by NICE on the change on 29 April (10:30-11:30).
- 1 April: A change to the cost-effectiveness threshold came into effect, increasing the ‘willingness-to-pay’ cut-off used by NICE in standard technology appraisals (STA) of new medicines from £20,000-30,000 to £25,000-35,000. This is the first change to the threshold since being introduced in 2004, and NICE announced that it will lead to an estimated additional 3-5 new medicines being approved per year.
For rare conditions, the impact of these changes are anticipated to be at most, modest. The threshold increase does not apply to the Highly Specialised Technology (HST) route for HTA, for example. The Office of Health Economics (OHE) also shared a helpful analysis of some of the challenges that it anticipates with the switch to using the EQ 5D-5L.
We therefore want to highlight two open NICE consultations to provide patient voice on these changes: a consultation on the updated quality-of-life measure (ends 27 May).
A second consultation is open to prioritise topics for a 2026 review of the NICE manual. This will include changes to reflect the current landscape and aims to increase transparency about how NICE prioritises changes to national policy (ends 19 May).
Alignment with the MHRA and other initiatives
The MHRA provided updates on a number of initiatives reflecting a broader shift towards a more agile way of reviewing the evidence for new medicines. This includes work to expand collaboration with other countries via the Access Consortium (Australia, Canada, Singapore, Switzerland and the UK), for which the MHRA provides the Secretariat.
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- 1 April: the new MHRA-NICE Alignment Pathway officially opened on 1 April. Like the Innovative Licensing and Access Pathway (ILAP), this aims to improve coordination and streamline decision-making between the two bodies.
- 10 April: The Access Consortium published more detail on its ‘Promise Pilot Pathway’, a priority review of treatments for serious or life-threatening conditions where no other treatment currently exists. In short, this pathway aims to allow regulators across the five countries to review a single application simultaneously.
- 28 April: The new clinical trial regulations will come into effect. These reforms, touted as a solution to address a number of challenges with how clinical trials currently run in the UK, aim to grant researchers more flexibility in how evidence is generated for new treatments and diagnostics.
Together, these developments may support faster and more coordinated access to medicines for people affected by rare conditions. However, like a number of complementary initiatives, such as the scope for a potential ‘third’ type of license for individualised therapies, and the Rare Therapies Launchpad, these remain at an early stage and we will share more when updates become available.
The UK-US partnership deal in context
On 2 April, the UK and US announced a trade deal had been reached, built around the principle that all countries should pay a ‘fair share’ for new medicines. The headline is that medicines exported to the US will face 0% tariffs, though many details in the full text of the agreement remain unclear, including how it will work beyond the initial six months’ term.
One consideration is what this could mean for UK medicine pricing. Historically, high US prices helped fund research and development (R&D), effectively subsidising lower prices elsewhere. If US prices fall, industry may look to recover income from other markets, including the UK. The key question is whether that pressure would be greater or smaller than the NHS’s increased willingness-to-pay threshold.
Alongside the announcement, two new cancer medicines were approved by NICE. However, it is too early to say whether this signals a more meaningful shift to also benefit medicines for rare conditions. As members of the Charities Medicines Access Coalition (CMAC), chaired by our member organisation Myeloma UK, we share the view that:
‘Continued investment in research and innovation is essential to delivering the breakthrough treatments patients urgently need. Today’s announcement is a welcome step, but just one piece of a much bigger picture.’
Scott Purdon, Chair of CMAC and Head of Patient Advocacy at Myeloma UK
The Government’s Medicines Investment and Commercial Environment Review
As part of the US trade deal, the UK Government has committed to increase spend on innovative medicines to 0.35% of GDP by 2028 and 0.6% by 2036, rising from around 10% to 12% of the NHS budget over the next ten years.
In response, the Association of the British Pharmaceutical Industry (ABPI) and His Majesty’s Government (HMG) have convened a joint taskforce to explore how to meet this target via a rapid ‘sprint’ based policy development process.
This aims to inform the future operation of the UK’s commercial environment, covering the pricing of individual medicines or vaccines, overall spending and market size, and the breadth and speed of uptake of new medicines. So far on:
- 2 April: the ABPI published a report outlining some of the current challenges.
- 8 April: Genetic Alliance UK participated in a workshop at the Department of Health and Social Care (DHSC) via our membership with CMAC.
- 23 April: We responded to a survey that the ABPI circulated outlining the specific challenges our community has raised via the Westminster APPG, broader enablers to the commercial environment (e.g. data, newborn screening) and aligning with the recommendations in the Bioindustry Association (BIA)’s recent report.
The taskforce is now deliberating ideas for potential ‘pilots’ that will inform Ministerial direction in June 2026. We are working with fellow CMAC members to call for greater opportunities for patient voice in this process and will share updates as these develop.
In parallel, we submitted a response to the House of Lords’ call for evidence on how to regulate the use of artificial intelligence (AI) and personalised medicines. AI is of increasing importance to access policy, given the significant potential and specific challenges that it presents for people living with rare conditions.
To explore these issues further, we recently published a new report on behalf of the LifeArc Translational Centres for Rare Disease Hub (March 2026). Through the Hub’s Policy Working Group, which Genetic Alliance UK jointly steers with LifeArc, we will continue to monitor how recent changes will shape the UK’s life sciences sector, including shifts in the research funding landscape.
Read our ‘More trials, better data, faster access’ report here. You may also find it helpful to register for an ‘Ask me anything’ (AMA) online event that the MHRA is hosting with the National Commission on AI on 20 May (09:30-10:30am).
If you are interested in discussing any of the above consultations with us, please feel welcome to get in touch via [email protected]