<abstract xmlns="http://www.w3.org/1999/xhtml">

<sec>
<h3>Introduction</h3>
<p>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.</p>
</sec>
<sec>
<h3>Identifying key challenges</h3>
<p>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.</p>
</sec>
<sec>
<h3>Summary</h3>
<p>From an access equity viewpoint, the key events in <bold>the patient journey and pathways</bold> 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 <bold>behavioural change, mindset and incentives</bold>, 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. <bold>Budgets and resources</bold> 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.</p>
</sec>
</abstract>

Introduction
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.


Summary
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.

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 Journal of Haemophilia Practice

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.

Introduction
The European Haemophilia Consortium (EHC) Think Tank Workstream on Access Equity aims to identify and address the key challenges to ensuring...

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

Article Category: Systems Change

Published Online: Jul 17, 2023

Page range: 56 - 61

DOI: https://doi.org/10.2478/jhp-2023-0011

Keywords
Access equity, Patient journey, Behaviour change, Mindset, Budgets, Resources

© 2023 Skouw-Rasmussen Naja et al., published by Sciendo

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.

Figure 1.

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

Article Category: Systems Change

Published Online: Jul 17, 2023

Page range: 56 - 61

DOI: https://doi.org/10.2478/jhp-2023-0011

KeywordsAccess equity, Patient journey, Behaviour change, Mindset, Budgets, Resources

© 2023 Skouw-Rasmussen Naja et al., published by Sciendo

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.

Figure 1.

Keywords
Access equity, Patient journey, Behaviour change, Mindset, Budgets, Resources