There is a vast population across all of the APEC economies that is being overlooked by the healthcare system due to the inability to either diagnose or manage rare diseases. Accordingly, no APEC Economy will be able to claim that is has successfully implemented the “Healthy Asia-Pacific 2020” initiative if it has not adequately and equitably met the needs of those with rare diseases. 

This project seeks to help APEC developing economies adopt an inclusive approach to “Healthy Asia Pacific 2020” implementation by addressing barriers to healthcare services for populations with rare diseases.  With an estimated 200 million people in the APEC region living with one of the 7,000 known rare diseases, APEC Economies cannot afford not to improve access to screening, diagnosis, appropriate medical management and support to people with rare diseases. This project will establish a tripartite task force and convene a 2-day policy dialogue to facilitate the sharing of best practices, policies, standards, and guidelines for addressing rare diseases, drawing on the experiences of developed APEC economies.  Economies will then work together to develop an APEC Rare Disease Action Plan to facilitate greater alignment of domestic policies and implementation of best practices and to provide a framework for short-term and long-term multi-sectoral collaborations to advance progress in key areas.

By addressing the barriers to healthcare services for rare diseases, APEC Economies will improve the economic and social inclusion of those affected by rare diseases, including caregivers, and ensure a more inclusive “Healthy Asia Pacific 2020”.

Background

In November 2016, APEC Leaders recognized that “health is the foundation of economic prosperity and human development” and highlighted the importance of “promoting health systems towards the achievement of Universal Health Coverage (UHC), which are resilient, sustainable, accessible and responsive to current and future needs.”  The Healthy Asia Pacific 2020 Initiative, welcomed by APEC Leaders in 2014, also calls on APEC Economies to strengthen health systems to support UHC, and in doing so, to “collaborate with stakeholders and share experiences to provide the whole population, including the poor, vulnerable and marginalized groups, with access to safe, effective, high quality and affordable primary health care.” No APEC economy will be able to claim that is has achieved APEC Leaders’ vision for health, or successfully implemented “Healthy Asia-Pacific 2020”, if it has not adequately and equitably met the needs of those with rare diseases.

There is a vast population across all of the APEC economies that is being overlooked by the healthcare system due to the inability to either diagnose or manage rare diseases. Persons with rare diseases are often trapped in a vicious cycle of vulnerability and poverty due to exclusion from health care and education systems, as well as job markets.  APEC economies cannot afford not to improve access to screening, diagnosis, appropriate medical management and support to people with rare diseases.  Rare diseases are one of the most scientifically complex health challenges of our time. There are currently 7,000 known rare diseases. According to the World Health Organization (WHO), rare diseases affect roughly one in fifteen people globally with a majority of these diseases affecting children.  An estimated 200 million people in the APEC region, and 400 million people worldwide, are living with a rare disease.  Rare diseases are often difficult to diagnose, and in many cases, few or no treatment options are available. Approximately 80% of rare diseases are caused by abnormalities in a person’s genes. Rare disease management remains immensely challenging across the region, due to high costs, limited resources, lack of scientific knowledge, and financial incentives.  The APEC economies are at vastly different stages of progress in addressing rare diseases and there is no alignment of priorities. However, APEC developed economies maintain best practices and established approaches in rare disease management that can be shared and effectively implemented in APEC developing economies. Economies typically take a similar journey in addressing rare diseases and thus there is no need to reinvent the wheel. The proposed project seeks to help APEC developing economies adopt an inclusive approach to “Healthy Asia Pacific 2020” implementation by addressing barriers to healthcare services for populations with rare diseases.

This project will first establish a tripartite (academic, government, industry) APEC LSIF Rare Disease Network to identify and collect information/data on those barriers in APEC economies.  The collected information will be synthesized and used to develop and design a 2-day policy dialogue in mid-2018.  The policy dialogue will facilitate the sharing of best practices, policies, standards, and guidelines for addressing rare diseases, drawing on the experiences of APEC developed economies.  Participating economies will then work together to develop an APEC Rare Disease Action Plan to facilitate greater alignment of domestic policies and best practices and to provide a framework for short-term and long-term regional, multi-sectoral collaborations to advance progress in key areas. By addressing the barriers to healthcare services for rare diseases, APEC economies will improve the economic and social inclusion of those affected by rare diseases including caregivers, and ensure a more inclusive “Healthy Asia Pacific 2020”. Participating APEC economies will discuss and determine an appropriate timeline for the Action Plan to be implemented.  The Project Overseer currently proposes the end of 2025 as the target date for implementation of the Action Plan.

Health Ministers and health officials recognized the importance of addressing rare diseases at the 7th APEC High-Level Meeting on Health & the Economy (HLM7) in August 2017 in Ho Chi Minh City, Viet Nam.  HLM7 “welcomed the launch of a new APEC initiative to address barriers to the diagnosis and treatment of rare diseases in the region” and “noted that such efforts will improve the economic and social inclusion of those affected by rare diseases, including caregivers, and ensure a more inclusive Healthy Asia Pacific 2020”. HLM7 also “welcomed the development of an action plan to facilitate greater alignment of domestic policies and best practices and to provide a framework for regional collaboration”.

The APEC Life Sciences Innovation Forum (LSIF) established the tripartite APEC LSIF Rare Disease Network (RDN) with participation from government, academia, and industry. The Rare Disease Network’s first task was to identify and compile information on the various barriers that economies face in addressing rare diseases. Over time, the RDN plans to continue to scale in size, diversity, and depth of engagement as the entity shifts from its role of informing the development of an APEC strategy on rare diseases to supporting economies in implementing the strategy. In 2018, the RDN organized a series of Stakeholder Consultations in Australia; People’s Republic of China; Republic of Korea; Chinese Taipei; Thailand; and Viet Nam to learn more about the local, frontline experiences with rare disease. Hearing a diversity of perspectives and patient experiences were the priority objectives; the RDN leadership connected with government officials, academics, industry representatives, and other members of the rare disease community including patients in these six (6) economies.

The information assembled through the stakeholder consultations informed the development of a 2-day policy dialogue: the inaugural APEC Policy Dialogue on Rare Diseases in Beijing, China in June 2018. The event facilitated candid discussion between senior leadership from government agencies overseeing health and social services, academic experts from universities and teaching hospitals, industry executives, and leaders from civil society, including patient groups. At the widely-attended dialogue, APEC economies shared best practices and policies for addressing rare diseases and began to collaborate on the development of this APEC Action Plan on Rare Diseases.