Alzheimer’s Research UK – Written evidence (INQ0092)


Since 2017, dementia has been the leading cause of death in the UK[1]. Age is the biggest risk factor, and with an ageing population, current projections anticipate that by 2021 there will be over one million people living with dementia in the UK, rising to two million by 2050[2]. Alzheimer’s Research UK is the UK’s leading dementia research charity. Our mission is to bring about the first life-changing dementia treatment by 2025.


Key challenges in dementia drug development pipeline

There are several key challenges in the dementia drug development pipeline:


Current barriers to patient access for new treatments for dementia

Dementia is the only major cause of death we can’t slow down, prevent or cure and this comes at incredible cost. But with several treatments in late stage development, there is growing hope, so now is the time to look ahead and prepare the system for quick and equitable patient access. Alzheimer’s Research UK believes the current market access pathway in England poses significant and specific challenges for new disease modifying treatments for dementia.


Affordability: The newest medicines, driven by innovative research and scientific discovery, are usually expensive. Pharmaceutical companies will want their considerable research investment factored into future pricing arrangements with NHS England. In the current system, some new therapies would not be considered cost effective even if they were given away for free. The pioneering nature of new medicines mean that it can be harder to measure effectiveness in the way that the NHS has traditionally required before it will pay for a medicine. This creates a challenging environment to ensure patients have timely access to new medicines. Our Thinking Differently[3] report outlines the impact new disease modifying treatments (DMTs) for dementia will have on the health system. NHS England budgets will face significant upfront costs for the necessary changes to patient pathways and service delivery, this will be coupled with the cost of the treatment itself.


Uncertainty: NICE Technology Appraisals will struggle to overcome clinical uncertainty that new DMTs will bring, this struggle could see patients being denied access. New, innovative medicines always come with layers of uncertainty, these new DMT’s will be first of kind medicines with little natural history data. The nature of the diseases that cause dementia mean it will be hard to capture disease progression. Consequently, there is a lack of natural history data, captured in a consistent manner through clinical endpoints. This level of uncertainty will be challenging for NICE methods in their current form. However, the new Innovative Medicines Fund could provide a means to collect data over a number of years, although there are outstanding concerns if this approach would not fully overcome all uncertainty and risk issues.


Value Recognition: NICE will struggle to capture the full value of these new treatments upfront, at the point of assessment. This will be especially true of DMTs for early or non-symptomatic stages of dementia. The full benefits to people living with dementia, their carers, and wider society may be evident over several years, keeping people out of supported care and in better health for longer. Current NICE Technology Assessments predominantly consider direct healthcare costs and benefits however, and only some social care costs but not informal care[4]. This could result in an inaccurate assessment of the true value of treatments. Economic modelling with the London School of Economics in our Thinking Differently report showed that the full value of a disease modifying treatment “is only demonstrated when a broader perspective of the savings across sectors is considered”.


Affordability Challenges

Alzheimer’s Research UK recommendations:

  1. The upfront cost of future DMTs will be significant, these treatments will be innovative in nature and therefore expensive. Early discussions between pharmaceutical companies and NHS England commercial medicines team are essential.
  2. The proposed new NHS England commercial framework should lay the foundations for these earlier engagements and discussion between NHS England and the pharmaceutical industry.
  3. Better, earlier horizon scanning and engagement with academics, pharmaceutical companies and patient groups would help the NHS with long-term financial preparation and preparation for the impact on patient pathways and service provision.
  4. New DMTs could be accessed by large patient populations and used over long treatment periods, having significant impacts on the NHS in England. We would want to see responsible pricing from pharmaceutical companies bringing their medicines to the NHS.
  5. We believe the affordability challenges of DMTs will not be overcome with simple discounting or managed access approaches. Novel and flexible approaches to pricing/reimbursement plans with greater risk sharing between pharmaceutical companies and the NHS is required.



Alzheimer’s Research UK recommendations:

  1. Greater flexibility in NICE’s approach to dealing with uncertainty, especially when burden of illness is substantial as is the case for new DMTs for dementia.
  2. NICE, NHS England and pharmaceutical companies should agree on risk sharing approaches to overcome aspects of uncertainty.
  3. Real world evidence collection, similar to the revised Cancer Drugs Fund should be explored. The Innovative Medicines Fund may be one route to achieve this. However, challenges on the length of time needed to overcome uncertainty could remain. In addition, the danger of a ring-frenched fund which could dis-incentivise future pipeline investment would need to be assessed.
  4. Work to explore how to gain agreement on clinical endpoints by pharmaceutical companies across clinical trials for DMTs is needed.


Value Recognition

Alzheimer’s Research UK recommendations:

  1. In Technology Appraisals the scope of costings should be widened to include informal care and wider economic benefits when assessments of new DMTs are undertaken by NICE.
  2. Wider consideration should be given by NICE in their appraisal process to assess the full societal value of new DMTs, including burden of illness and carer quality of life.
  3. Concerns have been expressed that the measuring tool EQ-5D focuses too heavily on physical health at the expense of mental health[5][6]. Identifying reliable and meaningful measures is needed to best to measure quality of life in the context of people living with dementia and their carers.


3 March 2020



[1] England and Wales; Mortality Statistics: Deaths Registered in England and Wales (Series DR) Scotland: National Records of Scotland Vital Events Reference Tables. Northern Ireland; Northern Ireland Statistic & Research Agency Registrar General Annual Report

[2] Prince, M et al (2014) Dementia UK: Update Second Edition report produced by King’s College London and the London School of Economics for the Alzheimer’s Society.

[3] Alzheimer’s Research UK (2018) Thinking Differently Report, available:

[4] Galen Atlantica, Alzheimer’s Research UK (2018), ‘Addressing value assessment and funding challenges facing a disease- modifying treatment for Alzheimer’s disease: A multi-stakeholder perspective’, PIAdT

[5] Connell J et al. (2014), ‘Measuring quality of life in mental health: Are we asking the right questions?’, Soc Sci Med. 2014 Nov; 120: 12–20