An Agreement with the WHO

Memorandum of Understanding with the WHO

Rare Diseases International has signed a Memorandum of Understanding with the World Health Organization (WHO). This collaboration aims to shape international rare disease policy and strengthen the capacity of health systems to address rare diseases.

In the first two years, activities will focus on harmonising the way rare diseases are defined internationally as well as laying the groundwork for a global network of centres of excellence for rare diseases.

Operational Description of Rare Diseases

RDI is facilitating efforts to develop an operational description of rare diseases based on key prevalence and incidence figures. This should improve the recognition of rare diseases and increase their visibility in health systems. An operational description would also help inform the WHO and Member States when considering national, regional and international policy.

WHO Collaborative Global Network for Rare Diseases (CGN4RD)

The second set of activities in the MoU focuses on the establishment of the WHO Collaborative Global Network for Rare Diseases (CGN4RD). The Network will operate through a “networked care” approach that groups rare diseases by therapeutic areas. The project aims to identify, assess and support centres of expertise connected internationally in one global network

RDI is currently consulting all stakeholders and rare disease patient organisations to design a Needs Assessment Study for the CGN4RD. The assessment will outline the rare disease community’s needs in each of the six WHO regions as well as resources and existing networks on the ground in each region.

With the WHO and all relevant stakeholders, RDI works towards the following long term goals:

1. Increase awareness of rare diseases and their visibility in healthcare system.
2. Strengthen healthcare systems, including improving access to accurate diagnosis, prevention of comorbidity, and promotion of multidisciplinary holistic care.
3. Encourage Member States to adopt strategies to address rare diseases at national level within an international policy framework.
4. Improve access to affordable and quality treatments and care

The plan is timed around WHO’s 13th General Programme of Work 2019-2023 (GPW13) which seeks to increase the number of people benefiting from Universal Health Coverage by one billion.

The actions in the MoU are intended to strengthen healthcare systems by fostering collaboration and aggregating expertise. The agreement should boost initiatives to deliver patient-centred care by promoting a model of multi-disciplinary care that is centred on patients’ needs.

Watch and Learn

Yann Le Cam, EURORDIS CEO and RDI Council member, outlines the agreement with the WHO. Matt Bolz Johnson, CGN4RD Advisor, describes the journey towards a Global Network and Ana Rath, Orphanet Director, discusses the importance of an operational description of rare diseases.

Solidarity with Iranian patients

EURORDIS Partnership with RADOIR

EURORDIS-Rare Diseases Europe has recently signed an agreement with the Rare Disease Foundation of Iran, a non-profit and non-governmental institution that provides services for rare disease patients and their community in Iran.

Created in 2008, RADOIR was established by an entrepreneur, M. Ali Davoudian, and medical professionals who care for patients with rare disorders. The foundation aims to improve the quality of life for patients living with a rare disease and to increase public awareness about the burden of rare diseases on patients, their families and the community.

  • RADOIR encourages the creation of national disease specific associations by providing financial support, mentoring and facilitating their interaction with competent authorities.
  • RADOIR has also opened several care centres in Iran which provide medical imaging services. They also help the patients to cope with their administrative situations and offer a permanent international helpline in Farsi, with twelve members of staff working 24/7. All services are offered free of charge for all patients suffering from rare diseases and cancers. RADOIR also covers basic health insurance costs for patients who are unable to afford it alone.
  • RADOIR has been organising events in Tehran for Rare Disease Day since 2010, gathering patients, health professionals and authorities, as well as holding conferences with more than 1000 participants.

EURORDIS and RADOIR share the common vision that bringing together rare disease patient communities from around the world catalyses international collaboration of all stakeholders. They recently signed a Memorandum of Understanding, which is part of the continued effort to stress the international dimension of the rare disease movement and the global benefits to be gained by international collaboration in this field. Read more about EURORDIS international partnerships.

RDI and RADOIR

RADOIR also became a member of RDI at the end of October, right in time for the launch of the NGO Committee for Rare Diseases, where they intend to play a role due to having consultative status with the United Nations Economic and Social Council.

Solidarity with the Middle East regarding the interests of rare disease patients is required to leverage the efforts of those fighting to promote the cause of rare diseases in the region.

Rare Diseases Conference 2016 featuring the 11th ICORD Annual Meeting

Rare Diseases Conference 2016 featuring the 11th ICORD Annual Meeting

The purpose of the Rare Diseases Conference that took place in South Africa last month was to provide a global forum for all stakeholders within the rare diseases field to participate in open discussion, enhance models of best practice and share specialist knowledge around rare diseases and orphan drugs. Incorporating the annual International Conference on Rare Diseases and Orphan Drugs (ICORD) for 2016, RareX had an extended programme, to incorporate patients, their families and caregivers as well as support groups and minority groups within the rare disease community.

The conference included presentations from members of rare disease organisations all around the world, including the Canadian Organisation for Rare Diseases (CORD), the New Zealand Organisation for Rare Disorders (NZORD) and the International Rare Diseases Research Consortium (IRDiRC). Presentations were given on global rare disease policy, diagnostics, congenital malformation, access to treatment, research and patient organisations.

Also during the conference, the African Alliance for Rare Diseases was launched, with its future patient charter being developed on-site. The African Alliance aims to be a common voice to advocate and address the lack of prevention, delayed diagnosis and lack of reliable diagnostics resulting in diminished access to treatment and supportive care for rare disease patients in Africa. The conference proved to be very useful in forwarding the rare disease agenda in South Africa and across the African continent and will hopefully be just the start of rare disease dialogue in Africa.

Statement from UNDP Administrator Helen Clark to the ICORD Meeting

ICORD is very pleased to announce this statement on rare diseases and the development agenda, presented by Helen Clark, Administrator of the United Nations Development Programme, to the ICORD conference in Cape Town, South Africa, on 20 October 2016. It is very significant as the first official statement from such a high level in global health and development affairs, about the needs of rare diseases.

 

Good morning, I send warm greetings to everyone gathered in Cape Town for the 11th ICORD meeting, the International Conference on Rare Diseases and Orphan Drugs. You are gathered over the next three days to discuss a matter of great importance to both health and international development communities. More than 300 million people around the world live with at least one rare disease. In most cases the nearly 7000 diseases which are classified as rare do not constitute a large enough market to incentivize much needed medical innovation. Where treatments do exist, they are often expensive, and place economic strain on individual patients and their families, as well as on health systems. Numerous studies from around the world, including studies by UNDP, show that ill health and the costs associated with it are major factors which push people into poverty. Thus the topic of this ICORD conference is as relevant to development practitioners as it is to health professionals.

There are many opportunities to address the specific issues faced by people living with rare diseases. Allow me to highlight three of these:

  • The first is the 2030 Agenda for Sustainable Development which was adopted by UN Member States last year. At the heart of this new agenda and the seventeen Sustainable Development Goals which accompany it is a fundamental principle: “to leave no one behind”. The hard work has begun to transform this bold development agenda from words into actions at all levels – national, regional, and local – which will improve the lives and well-being of all. The 2030 Agenda is complex, and there is much to do, including on health. One of the key commitments under Goal 3, “ensuring healthy lives 2 for all”, is to achieve universal health coverage and provide “access to safe, effective, quality, and affordable essential medicines and vaccines for all”. The research on and development of health technologies is an important element of universal health coverage. No country can claim to have achieved universal healthcare if it has not adequately and equitably met the needs of those with rare diseases.
  • Second, the Report of the Secretary-General’s High-Level Panel on Access to Medicines was released last month. The report concluded that greater investments are required from governments to address the absence of adequate market incentives for unmet health needs such as rare diseases. UNDP is working closely with several governments to strengthen their legal and regulatory environments on health technology innovation and access. We welcome the opportunity to work more closely with ICORD in this regard.
  • Third, sustainable development requires whole of government and society responses. Often the key obstacles to achieving an important goal will be outside the immediate sector targeted for attention. The several health-related targets outlined across the SDGs should serve as a reminder of how governments, civil society and patient groups, and industry and development partners can work together to increase access to health technologies within a relatively short time-frame. ICORD, as a multi-stakeholder society of patients, health professionals, researchers, regulators, health officials, and pharmaceutical industry representatives, offers a model of the collaboration which is needed to help achieve these important targets.

On behalf of UNDP, I wish you a productive and successful event at ICORD 2016, and thank you in advance for the work you will do in the coming days and years. Let us continue working toward inclusive and sustainable development for all, including for those with rare diseases.