Preparing for the Potential Rollout of New Alzheimer's Drugs: Towards a Policy, Research, and Innovation Agenda

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Jan 30, 2024

Senior White man sitting in a chair in his living room, looking out the window, photo by SolStock/Getty Images

Photo by SolStock/Getty Images

Dementia is one of the leading causes of death in the United Kingdom, affecting not only patients, but also placing a significant burden on families, caregivers, and the health care system. In 2020 dementia was estimated to cost the United Kingdom nearly £35 billion per year. Alzheimer's disease is the most common type of dementia, but lacks effective treatments.

In recent years, there has been growing excitement about a suite of promising new drugs that appear to slow cognitive decline in individuals with early-stage Alzheimer's disease. In 2023, 141 unique drugs were being tested in clinical trials, as registered on ClinicalTrials.gov, over three quarters of them disease-modifying drugs. Based on data from clinical trials, two of these drugs, Lecanemab and Donanemab, could slow the cognitive decline in early-stage Alzheimer's disease patients by approximately 2535 percent. If approved, they would be the first new Alzheimer's disease drugs in the United Kingdom in nearly 20 years and the first disease-modifying treatments available.

That will create a new challenge, though; how to put these treatments into practice, and how to pay for them. Integrating these drugs into the existing dementia care system will require a shift in the way the health system operates dementia care pathways—posing both challenges and offering opportunities for innovation. In response, NHS England has recently established a national programme team to consider how new early Alzheimer's disease treatments, once approved by regulatory agencies, can be embedded into NHS care pathways.

RAND Europe's work helps health systems prepare for innovation, so that scientific advances can be met with societal readiness to absorb the fruits of innovation in sustainable and equitable ways. In this perspective, we discuss what this means in the context of preparedness for the potential roll-out of promising new Alzheimer's disease treatments.

Integrating new drugs into the existing dementia care system will require a shift in the way the health system operates dementia care pathways.

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What makes the rollout of new Alzheimer's disease drugs uniquely challenging?

While the new drugs offer hope in reducing the burden of the disease, their rollout requires careful consideration: actually administering the drugs to patients would be just one step in a complex care pathway.

A number of challenges need to be addressed to ensure the successful rollout of new Alzheimer's disease drugs, but there are also several opportunities in this field for health innovation.

A large population of immediately eligible early-stage patients can overwhelm health system capacity. Alzheimer's disease drugs have a potential to be indicated to a large number of patients as they target early stages of the disease. In 2023, there were about 460,000 recorded diagnoses of dementia (of various types) in England, however, close to 40 percent of patients with dementia are not diagnosed, suggesting that the eligible population is much larger. Two challenges arise here, firstly, identifying eligible patients and secondly, such a large immediately eligible population is likely to create a significant bottleneck at the start of the care pathway. Large inequalities already exist in diagnosis of dementia with the rates of dementia diagnosis varying greatly (PDF) by location. The screening and distribution of new treatments carry a risk to exacerbate such inequalities, unless carefully considered and managed by health systems.

Health care workforce capacity constraints, accentuated by a shift of who provides care and how. The issue is two-fold. Firstly, the new drugs on the horizon would require specialised care to manage diagnosis, infusions, and monitoring of potential adverse effects, which can put a strain on already limited staff capacity. Second, different skills and specialties might be required than those that are currently needed to manage dementia care in both primary and secondary care settings: not only do we need more staff, but we also will face a shift in demand for the specialties providing the needed care. Currently, many memory clinics that provide early support for patients with Alzheimer's disease primarily focus on psychiatry and gerontology services, and will likely lack neurologist and neuroradiologist skills critical to the new pathway. While newly required skills and capacity can be built up eventually, this takes time. There may also be additional demand placed on primary care as a first point of contact for referrals to specialist care settings, if availability of novel treatments increases demand for referrals.

Infrastructure constraints such as low availability of neuroimaging equipment would also create challenges to diagnosing and treating eligible patients with early Alzheimer's disease with the new drugs. For example, positron emission tomography (PET) scans and lumbar puncture are needed to confirm diagnosis before the drugs can be prescribed, as well as magnetic resonance imaging (MRI) and PET scans for monitoring of adverse side effects during infusion and for the management of disease. The Royal College of Radiologists reported in 2017 that the United Kingdom had some of the lowest number of MRI scanners in Europe and significantly fewer than in the United States. In the United Kingdom, many clinics are already struggling to meet demand for dementia diagnosis, with long wait times and lack of scanners. New Alzheimer's drugs also require regular (biweekly or monthly) infusions, which should be provided in supervised settings (e.g., clinics). Infusion capacity is already stretched at many centres and is set to increase with the number of infused drugs in Alzheimer's disease neurology and cancer. Limited specialist and infusion capacity in the United Kingdom has been projected to result in potential waiting times for treatment of up to a year.

Reflecting on the Challenges—Issues for a Policy, Research, and Innovation Agenda

The potential new Alzheimer's drugs offer numerous opportunities for health systems to improve patient outcomes and ensure wider societal benefit. Reflecting on the challenges outlined above, the priority areas for decisionmakers to consider include:

Ensuring Fair and Equitable Access to Diagnosis and Treatment

The large number of initially eligible patients combined with the diagnostic, treatment, and monitoring capacity constraints will mean that not all patients can start using the drugs immediately when they become available. A transparent framework to prioritize patients for screening, diagnosis, and treatment will be needed to make sure not to exacerbate existing inequalities, considering the clinical and ethical implications of any potential decisions to prioritise some patients over others.

Securing the Needed Health Care Workforce

Support for workforce capacity building and training is necessary to ensure that health care professionals are equipped with the skills and knowledge to provide effective care within new treatment pathways. Not only will the health system need to recruit, retain, and train a sufficient number of health care professionals to deal with likely demand—it will also likely require a different skill and specialty mix within dementia care pathways. This calls for understanding both the nature of newly required specialist skills—for example an increased need for more neurologists, including those with neurobehavioural expertise, and neuroradiologists—and how different professions need to work together to provide optimal care. There are important policy questions as to how regional bodies and national level decisionmakers can work together to ensure an appropriately trained and coordinated workforce that can work within the new referral and treatment pathways.

Supporting Informed Decisionmaking

The burden on patients undergoing treatment with the new drugs, such as the need to attend regular infusions, and the potential risks of these drugs, will require ongoing engagement of health care providers with patients and caregivers to support informed decisionmaking. Awareness-raising on the potential opportunities and challenges associated with the new treatments is needed amongst patients, the public, and the health care professionals (including referring physicians in primary care as well as specialists in secondary care) in order to help with effective communications. While the new drugs offer the potential to slow disease progression, they do not cure the disease or reverse the symptoms. Therefore, in addition to treatment-specific services, patients will continue to need existing disease management support. To be effective, these drugs must be given as early as possible after diagnosis and require regular infusions and constant monitoring of potential adverse effects, creating an additional potential burden (and out of pocket expenses if travel is considered for example) on patients and carers.

Understanding the Economic Implications of the Novel Treatment Prospects

How to pay for these treatments and the health systems changes to make use of them will be a central challenge. Reducing the disease burden of Alzheimer's will have wider benefits in also reducing the burden on families and social care, but under current funding arrangements this will not directly mean more money for the NHS. It is important to consider the overall and long-term economics of the new package of care to ensure that it is sustainable and cost-effective, and to establish appropriate commissioning arrangements—envisaged by NHS England to be through Integrated Care Boards as the primary commissioning route for the new care pathways

Investing in Diagnostic Innovation

Innovation in the diagnostics space holds promise (but also uncertainty) in terms of reducing the strain on NHS capacity to diagnose early cases of Alzheimer's disease, by making diagnosis a simpler and/or more-automated process. Although the National Screening Committee in the United Kingdom currently does not recommend population screening for Alzheimer's disease due to a lack of effective screening and treatment options, continued research and development may change this in the future. The range of promising new technologies includes genetic (PDF) or biochemical markers of Alzheimer's disease that could potentially detect the disease more than a decade before the onset of symptoms; computer-aided artificial intelligence (AI) based approaches to support accurate and timely interpretation of MRI images, potentially helping to deal with demands on diagnostic capacity; and AI solutions to detecting passive biomarkers of Alzheimer's disease. Technology-enabled remote monitoring and care models also have potential to aid in the management of Alzheimer's disease. However, many of these innovations are still in development and their real-world utility remains to be seen.

Reinforced Emphasis on Prevention

Innovation…will create opportunities and needs for sharing learning about what works, where, how and why.

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As the new drugs do not offer a cure yet, prevention of Alzheimer's disease remains an unsolved challenge, requiring continued attention. Current research suggests that up to 40 percent of dementia cases worldwide could be prevented or delayed. In some populations, such as those with hearing loss, simple solutions such as hearing aids have been associated with reduced risks of further cognitive decline. Understanding further opportunities to prevent dementia, including Alzheimer's disease, through lifestyle and clinical interventions, has a significant potential to decrease its burden on patients, carers, and society. Therefore, despite the new early Alzheimer's disease treatment prospects, research into prevention should be key to a long-term strategy to manage and minimise Alzheimer's disease burden on patients, health systems, and society.

Creating and Nurturing a Learning Health Care System

Innovation in the Alzheimer's disease treatment space will create opportunities and needs for sharing learning about what works, where, how, and why in real world implementation settings. Clinical trials provide evidence on patient outcomes in specific settings in the short term. However longer-term real world data from diverse health systems will be needed to optimise patient treatment pathways once new drugs enter the UK market. This includes gathering evidence to inform practices related to the selection of patients for treatment, long-term management of treatment, and the management of adverse events. As for any new patient pathway, it will be important to monitor and evaluate its success and continued improvement, and to learn from the practices and experiences with introducing new treatments within different localities within a health care system and across diverse health care systems globally.

Figure 1: Key Challenges (brown) and Opportunities (teal) in Patient Pathways with New Drugs for Alzheimer's Disease

Screening of population at risk for cognitive impairment
Challenge: Inequalities in access to screening
Challenge: Continued need for prevention research and measures
Opportunity: Novel instruments and biomarkers for screening

Referral of patients with mild cognitive impairment for specialised diagnostics
Challenge: A change in needed specialists and skills
Challenge: Low availability of imaging equipment and specialist capacity
Opportunity: AI tools to facilitate early diagnostics

Treatment of patients with early Alzheimer’s disease
Challenge: Lack of infusion capacity
Challenge: Regular infusions at specialised centres creating extra burden for patients and carers
Opportunity: Long-term benefits of longer independent living

Monitoring of treated patients for disease progression and management of adverse reactions
Challenge: Low availability of imaging equipment for monitoring
Challenge: Unclear algorithms for long-term term administration and monitoring as yet
Opportunity: AI based tools and novel biomarkers could facilitate routine monitoring
Opportunity: Data captured in a learning health care system could be used to optimise treatment efficacy

Care for patients with late-stage Alzheimer’s disease
Challenge: Support for families and patients will still be needed

Agne Ulyte is a senior analyst in the Health and Wellbeing team at RAND Europe. Saoirse Moriarty is a research assistant in the Health and Wellbeing team. Sonja Marjanovic leads RAND Europe's portfolio of research in health care innovation.