Open Access

Access equity: key questions and challenges - A report from the 1st workshop of the European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity



The European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity aims to identify and address the key challenges to ensuring access equity to healthcare services for people with rare diseases. In this context, access refers to the ability to benefit from any given aspect of treatment and care; equity refers to being ‘fair and impartial’ in providing access.

Identifying key challenges

At the first virtual workshop of the Access Equity Workstream, participants representing a wide range of stakeholders, including healthcare providers, patient groups, research, and industry, shared their perspectives to identify the key challenges to achieving access equity. It was agreed to prioritise three challenges: 1. Patient journey and pathways; 2. Behavioural change, mindsets and incentives; 3. Budget and resources. The Iceberg Model was used to identify the factors (events, patterns, structures, and mental models) which should be prioritised for future discussions about potential interventions.


From an access equity viewpoint, the key events in the patient journey and pathways that need to be addressed are access to diagnosis, screening and treatment, taking account of the considerable national, regional and local variation in the availability of specialist expertise, genetic testing and counselling. There will be debate about sharing limited budgets to treat small numbers of people with rare disorders with gene therapy, compared to treating much larger numbers of patients with common diseases with cheaper drugs. In terms of behavioural change, mindset and incentives, there is a misalignment of personal, collective, organisational and national needs. Among providers, the ‘savings mindset’ is counterproductive for access equity, and short-termism may result in a restricted allocation of funding for innovative and preventive therapies. Industry mindset is largely transactional instead of outcomes-based and the patient community tends to focus on specific diseases instead of the wider implications for the provision of healthcare services and for society. Budgets and resources are key to access equity; reduced/delayed access to expensive new drugs for rare diseases such as haemophilia is a major issue. Cost-effectiveness prioritisation tools used for health technology assessments (HTAs) do not take into account data from small populations, leading to cost-effectiveness ratios that exceed accepted thresholds, and treatments restricted to subgroups of patients within an approved indication. Benchmarking international pricing, service centralisation and cross-border cooperation for delivery of gene therapy need to be addressed if access equity is to be achieved.

Publication timeframe:
Volume Open
Journal Subjects:
Medicine, Basic Medical Science, other, Clinical Medicine, Pharmacy, Pharmacology