Sheela, Michelle and I had the privilege to meet twice in February at rare disease events and to plan our 2024 columns. Consequently, we’ve decided to join forces this month to reflect on the UK chancellor’s Budget announcements in March and look at them through the lens of the rare disease community.

Michelle discusses the impact of the Budget on those living daily with a rare condition and the patient organisations supporting them; Sheela focuses on the effect on NHS providers and the wider services that support the community, and I conclude with a discussion of the consequences for the biotech and pharmaceutical industry.

We have all listened intensely to news stories of families who can’t afford to continue to provide care for their children to a standard they feel acceptable and necessary, to their challenges charging electric wheelchairs or keeping life support equipment running as energy bills increase. Equally worryingly, we have listened as pharmaceutical companies announce they won’t launch potentially life-changing innovations in a particular country or region due to affordability and pricing concerns. Money, sadly, has an impact on all aspects of life, and especially care—the arena close to our and all RARE Revolution readers’ hearts.

Sadly, there is nothing in the Budget that immediately resonates for the rare disease community specifically, and we all struggle in balancing our personal views on what helps attract business to the UK and what measures in the Budget may be perceived as positive steps forwards, versus the reality we see for many readers who are living daily with rare conditions and are not seeing things improve fast enough.

So here goes with our attempts to make sense of what the Budget may mean for the rare disease community. We’d love to hear everyone’s feedback and thoughts as the situation evolves.

Michelle: The impact on those living daily with a rare condition and the patient organisations supporting them

For those living daily with a rare condition and the patient organisations supporting them, the impact the Budget has on the NHS and the bio/pharma industry undoubtedly has a knock-on effect on the rare disease community. More investment in pharma and the NHS could theoretically improve the delivery of services and innovations for the community.

However, as Sheela and Nicki have outlined, the measures put forward in the Budget are long-term investments that are not specific to rare diseases. So how do those advocating for the rare disease community use the measures in a smart way—as levers to help campaign for improved outcomes for their communities?

In my view, as advocates, we must ensure that we link the priorities of the UK Rare Diseases Framework to the policy intent of these Budget investments, particularly those relating to our NHS.

For example, to achieve the goals around improving performance and reducing waiting times in the NHS—challenging goals, as Sheela states—we could highlight the importance of spending on rare disease services that improve diagnosis, understanding and knowledge of rare disease within our NHS workforce and care coordination, which undoubtedly creates efficiencies, helps the NHS reduce waiting times and, more importantly, improves the day-to-day healthcare experiences of the rare disease community.

The investment in improved digital transformation also has the opportunity to really drive forward these efficiencies and improvements in care, but the challenge for the rare community is ensuring small populations are not deprioritised when implementing these changes because of perceptions of rarity.

Outside the NHS and industry, some items of note include a cut in National Insurance contributions from 10% to 8% from April 2024 and for those who are self-employed this cut goes from 8% to 6%. These measures have the potential to have an immediate impact on families living with a rare disease here in the UK; however, some debate exists as to whether families will truly be better off due to the freeze in personal tax thresholds—it has been suggested that some households will be remain worse off.

Other areas our community will be watching with interest include the increase in the income threshold for child benefit from £60,000 to £80,000 from April 2024. This has the potential to support families who rely on one breadwinner due to the care needs of their family members— families who are by no means wealthy and are struggling to manage on a single salary. For those relying on disability benefits, the promise to increase the capacity for processing these benefits is likely a welcome announcement.

The majority of these announcements have the potential to ease pressure a little for the rare community; however, the reality is that when timelines extend to 2025 and beyond we are likely to see little change in this election year, and these policies could be completely changed and revamped if a new government is elected.

Our community are looking for changes that can help them “live well today” not next year, and policymakers need to ensure funding is allocated towards the services that can make that difference now.

Sheela: The impact on NHS providers and the wider services that support the community

The 2024 budget brings two major changes to healthcare spending in the UK. Firstly, the Budget documents state that an additional £2.45 billion in day-to-day funding aims to help the NHS “improve its performance and reduce waiting times”. However, achieving these goals is, as ever, quite challenging.

This money is for things like staff salaries and medicines and will give some confidence to the NHS; however, it might not be enough to cover all the costs the NHS faces, including rising demands, capacity challenges and potential staff strikes. So, for people waiting for diagnosis, support or treatment, and for healthcare professionals trying to deliver more efficient care, the additional money may have no real effect.

Disappointingly, given the current difficulties for people living with a rare condition, this increase in spending is unlikely to make any real difference in their access to healthcare support.

Secondly, there’s £3.4 billion set aside for updating NHS technology and infrastructure. This includes plans to double investment in digital transformation, such as using the NHS app more, going paperless, using artificial intelligence to help automate clinical coding of records and upgrading equipment like MRI scanners. This funding will be spread over three years, starting from 2025, and aims to bring about £35 billion in productivity savings by 2030. Very ambitious.

Investing in better digital technology for the NHS is a positive move. If updating old NHS computer systems does result in the £35 billion of savings for the NHS that the government says, this will support the NHS to work more efficiently from 2025 to 2030. The aim is to make the NHS 1.9% more productive each year during this time, reaching 2% in the last two years, which is a big improvement compared to before. Could this have an impact on service provision, improve care coordination and support the introduction of innovation into an old system? It remains to be seen.

We would do well to remember that while this upgrade is happening people living with rare diseases still need support, healthcare professionals still need to run clinics, see patients and deliver good care. Money can help but time to implement and adopt these things is a rare commodity. Therefore, the returns on this investment will take time to be realised. I for one am going to keep a watchful eye on how this progresses.

Nicola: The impact on the biotech and pharmaceutical industry

Many aspects of relevance for the industry have actually already been defined at the end of December through the voluntary scheme for branded medicines pricing, access and growth negotiations.

There were aspirations going into the Budget for positive changes associated with jobs growth, investment et cetera, and a hope that we may see enhanced tax incentives to attract more business to the UK and a regulatory system that supports innovation and research—all of which might have indirectly helped the rare disease space.

In reality, little has changed in most people’s views and there’s nothing rare-disease specific, but there were key relevant commitments different groups have pulled out, which might have a positive knock-on impact:

• a pledge of £650 million to AstraZeneca, which will fund a biomedical campus in Cambridge and a vaccine manufacturing hub in Liverpool in order to expand the UK’s life science footprint
• over £240 million in funding into building new homes, which could help transform Canary Wharf into a hub for life science companies
• a new expert advisory panel for the HMRC, which should help maximise the efficiency of R&D tax relief support for life science innovators
• announcements around a scheme that enables investment of capital from pension funds into innovative British companies might help deliver financial returns savers need while also delivering the new medicines and technologies society needs

Also in the Budget was £45 million for charitable medical research. Let’s hope some of it finds its way into the orphan disease arena!

One thing we can be sure of is that with a general election looming in 2024 and potential changes to the US presidency, we can certainly expect more announcements that could be relevant before 2025 and can’t rely on any of this for any length of time.

Another thing we are all sure of is that we will all need to come together and drive for further investment and change—whatever the result of the UK general election.

Editor’s note: Next month, Sheela Upadhyaya will discuss the Evaluating patient access to rare disease treatments report from the UK BioIndustry Association.

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About Michelle – a freelance health policy consultant and rare disease specialist at CRD Consulting Ltd

Michelle Conway trained as a nurse, before moving from the NHS to the pharmaceutical industry, where she held multiple roles from nurse specialist to regional market access manager. Michelle discovered a passion for rare disease when working with a biotech organisation to launch a product for an ultra-rare disease. She then supported the company with the launch of several ultra-orphan medicines before setting up as a freelance consultant.

With experience across multiple roles in the biotech and pharmaceutical industries, Michelle has expertise in market access and policy work. Michelle supports projects requiring strategic guidance, expertise and knowledge relating to the policy surrounding improved outcomes in rare diseases and market access for medicines for rare cancers and orphan medicine launch-planning.

She can be contacted atmichelleconway@crdconsulting.co.ukor connect with her onLinkedIn.

About Nicola – NJ Redfern Ltd

Nicola set up NJ Redfern Ltd in 2022 to enable her to consult, coach and collaborate with other organisations, following her experience at bluebird bio. She has a long history in rare disease and oncology within the pharmaceutical and biotech industry and has worked closely with the Cell and Gene Therapy Catapult and the ATTC network in the UK. She is a member of the International Society of Cell and Gene Therapy (ISCT) ethics subgroup, contributes to the UK ATMP Engage community and projects, and speaks at various congresses and webinars about the challenges of bringing cell and gene therapies to market.

Nicola is committed to and motivated by ensuring people living with significant health challenges have a choice and are able to access transformative treatment options quickly once science and innovation reach a point to positively impact their lives. She also believes the UK will be stronger if these new treatments are supported and embraced holistically across our ecosystem.

She can be contacted atnickiredfern@gmail.comor connect with her onLinkedIn.

About Sheela – a life sciences consultant specialising in rare disease

Sheela Upadhyaya is a consultant to the life sciences industry. She led the NICE Highly Specialised Technology Programme, the programme to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS. In her consulting practice she supports a range of strategic activities to advance rare diseases from research all the way to access and adoption. She excels in developing partnership and collaboration-enabling change and progress, specifically in rare diseases. In her consultancy she works with a range of stakeholders on policy and market access issues in the rare disease space. She possesses an excellent ability to challenge the status quo with stakeholders to secure positive access and adoption for orphan medicinal products.

She is co-chair for the Health Technology Assessment International Rare Disease Interest Group and leads a project at ISPOR in rare disease.

Sheela can be contacted atsheelaupadhyaya@gmail.comor connect with her onLinkedIn.

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