In 2023, the Bio Industry Association (BIA) worked with PwC to conduct a thorough assessment of the progress that has been made in improving access to treatments for rare disease in the UK, and the challenges that remain.

The report, which was developed by the BIA’s Rare Disease Industry Group (RDIG), is based on extensive research and conversations with key stakeholders in the UK rare disease space, including NHS England, the National Institute for Health and Care Excellence (NICE), patient advocacy groups, clinicians and industry.

The BIA report described several positive developments for rare disease in the UK.

UK Rare Diseases Framework

A strategic approach to improve rare disease treatment access, with a focus on faster diagnosis, has increased healthcare professional awareness, resulted in better care coordination, and improved access to specialist care and drugs.

NICE Methods Review

In 2022, NICE created a number of changes to their health technology assessment methods. The objective was to create fairer, faster and more consistent evaluations, which may benefit orphan drugs. These included greater acceptance of uncertain evidence and utilisation of real-world evidence to mitigate against uncertainties in the evidence; the consideration of a broader range of quality-of-life measures; increasing capacity within NICE for complex topics; the addition of the severity modifier to replace the end-of-life criteria, to take account of the severity of a condition, among other changes.

Since the methods review in 2022, NICE has introduced a lighter-touch, proportionate approach to technology appraisals. This approach will enable NICE to apply a light-touch faster evaluation to low-risk treatment evaluations. NICE has also introduced a real-world evidence framework. This aims to provide guidance and improve the quality of real-world evidence that informs guidance.

Innovative Licensing and Access Pathway (ILAP)

The Innovative Licensing and Access Pathway (ILAP) was introduced by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in January 2021 to accelerate access to innovative therapies, including ones for rare disease. ILAP operates alongside the MHRA’s Innovation Passport programme, collaborating with various health agencies including NICE, Scottish Medicines Consortium (SMC), All Wales Medicines Strategy Group (AWMSG) and NHS England and Improvement (NHSE&I).

In this pathway, manufacturers have the opportunity to secure advice throughout the product/therapy development journey, in the form of unique, product-specific roadmaps towards patient access in the UK healthcare system. The road maps should enable developers to access tools from a toolkit to design an efficient and “regulation and access ready” development programme.

The Innovative Medicines Fund

2021 also saw the introduction of a £340 million Innovative Medicines Fund (IMF) in the UK, which is providing early access to life-saving drugs. The fund mirrors the Cancer Drugs Fund (CDF) and was designed to support NHS England in offering patients potentially transformative new drugs while further real-world evidence is collected. The evidence collected informs a final decision by NICE on whether the treatment is clinically effective and a good use of taxpayer money in the long term. The aim was to reduce access delays and boost patient outcomes in the interim.

The IMF: key points

• Purpose: IMF offers faster access to non-cancer medicines at a fair price, ensuring prompt treatment with cutting-edge health technology.
• Funding: A total of £680 million ringfenced NHS funds, including the £340 in the Cancer Drugs Fund, support this initiative.
• Managed access: IMF accelerates access to potentially life-saving drugs, including those for rare diseases.
• NICE Recommendations: It follows NICE recommendations for non-cancer medicines, starting funding upon NICE guidance publication.
• Collaboration: NHS clinicians contribute to building evidence for new treatments, utilising NHS expertise and infrastructure.

Is all as rosy as it sounds?

The BIA report concludes that the “UK has made positive progress in improving access to rare disease treatments in recent years, and a period of transition is underway”. It states that “recent changes to marketing authorisation and pricing and reimbursement processes and new market access mechanisms are steps in the right direction”.

However, the report also found that while significant progress had been made, several challenges remain which are limiting both the speed of access to new treatments, and the number of patients that are able to receive them. The BIA report highlights areas for enhancement across all these initiatives.

The UK Rare Disease Framework

The UK Rare Disease Framework has provided an excellent backdrop and foundation for an action-based approach to addressing the key challenges for rare disease. However, the actions outlined and committed to by the delivery partners need to be delivered, along with positive change to making diagnosis faster, raise awareness with healthcare professionals, improve care coordination and access to specialist care, treatment and drugs.

The 2024 action plan is focusing on delivering what has already been committed to as well as a small number of new actions. Given the complexity of the system, we should all remember that these priorities do not just belong to the key delivery partners such as Department of Health, NHS England, NICE, MHRA et cetera. Collectively, we should own and undertake activities to support and augment these efforts.

NICE Methods Review

The updates to NICE’s methods and processes were hailed as a way forward for rare disease therapies, with the end-of-life modifier being replaced by the severity modifier, and increased flexibility in accepting different types of evidence, alongside other updates. However, it clear from the outcomes from recent approval processes for rare disease therapies that the severity modifier is not being used/applied for in standard technology appraisals in the way anticipated. There is also little evidence to show that the other modifiers and flexibilities are being applied. These updates are clearly not delivering what was first envisaged and begs the question: what will help? What should be done by NICE and others?

 I suggest that data needs to be captured to show how many rare disease technologies have either not launched in the UK or not been treated in a flexible way. I also suggest that evidence should be presented to NICE from recent appraisals showing where modifiers could or should have been considered. I am open to collaborating on a project of this nature with the BIA or Association of the British Pharmaceutical Industry (ABPI).

NICE should undertake its own reflective exercise to explore what it should do to encourage the use of these flexibilities, by giving committees the mandate to apply them, ensuring the guidance produced explains the circumstances in which they have and have not applied.

It is fair to say these flexibilities should not be applied in all cases, but explanation is needed so that it is clear when these flexibilities can be applied.

Innovative Licensing and Access Pathway (ILAP)

Leaving the EU enabled the MHRA to become more forward-thinking, and this is demonstrated by the introduction of ILAP. This new pathway enables timely discussions regarding evidentiary needs across regulatory and Health Technology Assessment (HTA) spheres, fosters synchronised evaluation procedures and optimises access routes within the UK healthcare framework. While ILAP has effectively advanced numerous topics, including therapies, through its system, its efficacy is currently hindered by resource limitations. It remains to be seen if these can be resolved to provide the guidance and support needed to expedite the development and access pathways, especially when the technology is addressing an unmet need in a rare disease.

The ILAP concept is great and much needed: stakeholders, particularly in rare disease, have been calling out for more alignment among regulators and healthcare system payers for a long time. However, the reality is that the demand from the industry to enter the ILAP process has resulted in capacity constraints at ILAP partners (such as the MHRA, NICE and NHS), which has limited the effectiveness of the scheme. There has been a commitment to review the process and the outcome of this is awaited.

The Innovative Medicines Fund (IMF)

It seems it has been difficult to find suitable candidates to enter the Innovative Medicines Fund. On 9 February 2024, the government revealed that NICE had not yet made a recommendation for managed access under the IMF. Thus, the fund had been used exclusively for interim funding to accelerate patient access to medicines recommended by NICE for routine commissioning.

To date, four medicines have received interim funding through the IMF. Three drugs—for the rare genetic disorder Wolman disease, for hidradenitis suppurativa, and for chronic hepatitis D—were in the IMF in November 2023.

NICE and NHS England are looking into options for rapid entry to managed access. It remains to be seen what approaches will be developed. And if more people living with rare diseases will benefit.

What are the next steps for innovation and access in the UK?

The UK has always had and always will have a culture of developing and implementing innovative new ideas, especially in areas of life sciences. The BIA report put this into context by explaining how access to treatments for rare diseases in the UK compares to access in countries that are members of the Organisation for Economic Co-operation and Development (OECD). While England and Scotland have a quicker turnaround time from marketing authorisation (MA) to inclusion on public reimbursement lists compared to Spain and France (according to recent data from the European Federation of Pharmaceutical Industries and Associations (EFPIA), there’s still room for improvement compared to countries like Germany, where the process is much faster.

The report also highlights examples from other OECD countries, showcasing pathways and funding dedicated to improving access to orphan drugs, including specialised managed access or reimbursement pathways and dedicated funding mechanisms.

The activities discussed here, and examined in the BIA report, are just some of the activities in the UK that support innovation and access. There are many more. This has enabled the UK to stay relevant, attractive for investors and a place that sets the pace for other countries around the world. However, the policy environment, against a challenging fiscal background, is making it difficult to bring all these activities together to create a better access pathway for new innovations.

The innovative approaches that have been put into place are only effective if they work in practice—this means effective, efficient processes, and policies that enable access to therapies and innovation that also deliver value in a publicly funded system.

To read the BIA report, clickhere.

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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.

Sheela excels in developing partnership and collaboration-enabling change and progress. 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 is chair in a project at ISPOR (The Professional Society for Health Economics and Outcomes Research) in rare disease.

Sheela can be contacted atsheelaupadhyaya@gmail.com,or connect with her onLinkedIn.

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