On 13 July 2021, Alzheimer Europe held an online session of its popular Alzheimer's Association Academy series. These Academy sessions have been running since December 2015, and have been held online since the start of the COVID pandemic. The Academy session on 13 July was focused on European systems for approval, reimbursement and health technology assessment (HTA) of new drugs for Alzheimer’s disease (AD). Participants included representatives of Alzheimer Europe national member associations from 21 different countries and representatives of the European Working Group of People with Dementia (EWGPWD), with presentations delivered by 3 expert speakers.
The first speaker was Natalie Bère of the European Medicines Agency (EMA; Stakeholders and Communication Division), who spoke about the European authorisation process for new medicines and the way in which the EMA involves patients and patient representatives. Natalie explained how the EMA is structured around seven scientific committees, with a Management Board that hosts representatives from Member States, Civil Society and EU Institutions. The EMA Office for Therapies for Neurological and Psychiatric Diseases is responsible for evaluating medicines for AD, and is currently analysing the submission of Aducanumab by Biogen. She highlighted the importance of public-private partnership projects such as EPAD and AMYPAD in bringing stakeholders together in support of clinical trials and developments for AD and dementia.
The second presentation was delivered by Diana O’Rourke, a Senior Advisor at the UK National Institute for Health and Care Excellence (NICE), and was entitled “National approaches to pricing, reimbursement and health technology assessment (HTA)”. Diana explained that systems for pricing and reimbursement of medicines are operated at national level, illustrating differences between national systems using examples from the UK and Germany. Generally, HTA agencies make a scientific assessment of the evidence for the drug (e.g. relative clinical efficacy) and assess its economic impact (budget impact evaluation), also considering wider factors such as the target population, the burden of illness or unmet need, the availability of alternatives and the impact on quality of life.
The final presentation was delivered by Jacoline Bouvy (Technical Director at NICE Scientific Advice), who provided an overview of HTA assessments of current and future AD medicines. She started by outlining the two key questions posed by HTA bodies like NICE: how well does the health technology work compared to standard practice in the health system, and how much does this health technology cost compared to what is standard practice in the health system. She went on to discuss the AD drugs that are currently available to patients, describing the HTA assessment processes for donepezil, galantamine, rivastigmine and memantine. In the second half of her presentation, Jacoline reflected on some of the lessons learned from the HTA assessment of the acetylcholinesterase inhibitor drugs and memantine, and what this might mean for future disease-modifying therapies for AD. Noting that new drugs in clinical trials for AD are more frequently targeted at the underlying causes of disease (termed ‘disease-modifying therapies’) and also tend to be targeted at earlier or preclinical stages of AD, she highlighted challenges that may arise in assessing the cost-benefit equation, and in identifying appropriate, meaningful endpoints and outcomes for these drugs.
