Working toward Global Equity in Rare Diseases
Universal Health Coverage Day, promoted by the World Health Organization, is celebrated annually on December 12. December 16, 2021, marked a significant step toward achieving the UHC vision to leave no one behind as the United Nations adopted a resolution by consensus on Persons Living with a Rare Disease and their Families.
Interview with Flaminia Macchia and Matt Bolz-Johnson, Rare Diseases International
What is Universal Health Coverage?
In September 2019, the United Nations reached consensus on a Political Declaration calling for all nations to provide Universal Health Coverage (UHC) by 2030. UHC builds on health as a fundamental human right and asserts that all people, everywhere, should have access to the quality health services they need, without experiencing financial hardship. The three dimensions of UHC are:
More equitable (“all people”)
More accessible / more services (“have access to quality services they need”)
More affordable / less direct costs (“without financial hardship”)
A key ask by the rare disease community when progressing toward UHC is prioritizing equity in health systems to “leave no one behind,” including the most marginalized and hard-to-reach populations.
What is the new UN Resolution on Persons Living with a Rare Disease and their Families?
The UN Resolution on “Addressing the challenges of persons living with a rare disease and their families” supports the aims of UHC as it calls for increased care and attention to a community that is often marginalized and underserved in health systems across the world. This Resolution goes beyond UHC as it recognizes that Persons Living with a Rare Disease (PLWRD) and their families face inequities and challenges not only in healthcare but across all life domains. The Resolution calls for the UN Member States to consider the global needs of this community, which include good health and wellbeing, but also reducing inequalities, accessing quality education, having equal opportunity for decent work and economic growth, a chance at no poverty, and gender equality. The UN General Assembly formally adopted the Resolution on December 16, 2021.
The asks of the Resolution are:
Participation and inclusion – collect data to identify patterns of discrimination and address causes that prevent full involvement of PLWRD and their families in society;
Appropriate care – strengthen efforts to address challenges of PLWRD to attain UHC; promote global networking of experts and centers of care; enhance collaboration and coordination of international research efforts;
National action – encourage UN member states to adopt national RD plans and measures to address critical challenges of PLWRD and their families;
Inclusion in the UN system – include the challenges experienced by PLWRD in other UN Agencies and Programmes, as well as in the monitoring and evaluation of the SDGs;
Monitor progress and implementation – decide to submit the Resolution at its 78th session, under the item “Social development”.
Working at the intersection of both Resolutions to attain global equity in rare diseases
For health coverage to be truly universal, it needs to focus on equity; particularly, reducing inequalities and facilitating equal opportunities to attain the greatest level of health and well-being for all persons.
The World Health Organization (WHO) supports Member States’ efforts to attain UHC. The UN Resolution on PLWRD and their families promotes ways for Member States to address challenges of this community for achieving UHC, including promotion of global care networks and coordination of international research efforts. Therefore, the WHO is partnering with Rare Diseases International (RDI), the global alliance of Persons Living with a Rare Disease across all countries and all rare diseases, to coordinate global action to help address the healthcare needs of this vulnerable population.
More specially, RDI is exploring the best way to pool and coordinate rare disease expertise, services, and research under a global network for rare diseases. This initiative concretely contributes to the realization of UHC irrespective of capacity at the national level; pooling resources globally will result in improved healthcare systems’ ability to treat rare diseases locally.
Clinical networks have already developed within disease areas, e.g., Hemophilia, Duchenne Muscular Dystrophy and Cystic Fibrosis in the US; at the national level, e.g., French Filières de Santé Maladies Rares (FSMR) and China’s National Network of Rare Diseases (NNRD); and at the regional level, e.g., European Reference Networks (ERNs).
RDI, along with the rare disease community globally, are exploring ways to connect these existing networks, engage countries and regions not currently connected, and build collaboration at the international level. Clinical networks represent a new dawn for how healthcare will be delivered to the global community.
Only by working together will we make this opportunity meaningful for each of the local communities to leave no one behind.
Resources and references:
Health for All Advocacy Toolkit: https://csemonline.net/uhc-advocacy-toolkit/
RDI UHC Toolkit: https://www.rarediseasesinternational.org/uhc4rarediseases/
RDI’s statement of Equity vs Equality: https://www.rarediseasesinternational.org/wp-content/uploads/2021/12/FINAL-EQUITY-STATEMENT-FOR-UHC-DAY.pdf?utm_source=Health+Equity+Statement
About the Interviewees
Flaminia Macchia was the Executive Director of Rare Diseases International (RDI) at the time of this interview. RDI is a network of over 80 member organizations representing rare disease patient groups in over 100 countries worldwide. RDI has been a key driving force of international rare disease efforts. The group advocates to make rare diseases a global policy priority, represent members on international platforms, and help members build their capacity to act locally, regionally, and globally.
Matt Bolz-Johnson was a Programme Director at Rare Diseases International (RDI), focusing on building a collaborative global network for rare diseases at the time of this interview. Matt’s other responsibilities include being the ERN & Healthcare Advisor at EURORDIS, where he advocates for the rare disease community by shaping the development and delivery of European Reference Networks (ERNs). Specifically, Matt has ensured a patient-centered approach, including developing European Patient Advocacy Groups that are formally linked to each ERN.
