Formalizing Healthcare Accessibility for People with Rare Diseases in Romania

The Social Innovation

Magnitude of the Problem, and its Root Causes

In Romania, more than 90% of the patients with rare diseases (RDs) are undiagnosed and struggle to access the services and the support they need, about half of them are children.

During their participation in the Accelerating Healthcare Access Globalizer, co-created by Ashoka and the Philips Foundation in 2020, the RPWA team identified key factors contributing to the improper diagnosis and lack of access to treatment for patients with rare diseases in Romania:
-    Patients with RDs are not found in statistics at national level- there is no national registry for rare diseases.
-    Inadequate codification of RDs (ICD 10 used in the health and care systems covers only about 500 different RDs among almost 8000 known RDs).
-    95 % of RDs do not have approved treatments or therapies anywhere in the world and in Romania we have not yet approved all the existing treatments. 
-    waiting time for getting treatment is at least 4.8 years from the first symptoms which means that many children die before the age of five.
-    the disconnected nature of health services results in an unstructured and uncertain care pathway, with 65% of patients needing to visit more than seven physicians before receiving a diagnosis.
-    40% of patients are misdiagnosed at least once.
-    shrinking capacity of medical staff.

Based on their systems change analysis, RPWA made it their mission to address the disconnected nature of health services by improving formal processes in the Romanian public healthcare system. 

Strategy to Catalyze a Network of Changemakers towards the Targeted Mission

The strategy to increase the connectedness of the health services includes working with the government to coordinate existing social and healthcare services, establish new ones, make them more physically accessible for patients across the country and empower the healthcare providers to build and exercise their agency to adopt new practices to support patients with rare diseases. 

Some of the tactics they deployed towards the mission as a part of the strategy include:

1) Co-creating an Advocacy Campaign to push for Policies to be Inclusive of Needs of Patients with Rare Diseases

RPWA in partnership with Romanian National Alliance for Rare Diseases, Romanian Association for Rare Cancers and other stakeholders recently successfully advocated for rare cancers to be part of the national cancer plan and to include needs of patients with rare diseases in the National Strategy for Health.

By collaborating with the Ministry of Health, National Drug Agency and the National Health Insurance, they are making sure that the care and treatment for patients with rare diseases are accounted for in the policy plans.

2) Involving Community Nurses to Adopt the Role of Case Managers for RDs by Co-creating Training Curriculum with key Stakeholders

RPWA, in collaboration with partners like the Ministry of Healthcare, National Health Insurance, local authorities in Zalău and Sălaj, and medical universities, has designed a curriculum and trained over 1200 community nurses to serve as case managers for patients with rare diseases. They plan to train an additional 2,000 nurses in the coming months, continuing their capacity-building efforts.  

3) Collaborating with Partners to Test and Advocate for a National Case Management Platform

To enable a more structured caregiving pathway for patients with rare diseases, RPWA has established a partnership agreement with Ministry of Health in 2016 and worked with other stakeholders like Medical University in Timișoara, Children Hospital Louis Țurcanu Timișoara and Frambu Center for Rare Disorders Norway on testing a case management module for RDs in the national platform for community nurses and demonstrated feasibility.

In 2024, RPWA has created the RDs module in the case management platform for community nurses, so that they don’t need to report in a different platform or duplicate the work. It makes the experience smoother and less stressful for the patients as they navigate the disconnected caregiving pathway, ensuring better health outcomes.

4) Knowledge Sharing and Consulting with Peers to bring International Best Practices to the National Healthcare System

RPWA is part of a global network of nurses supporting patients with rare diseases and different working groups for rare diseases at EU level. This enables them to bring best practices and learnings to Romania, leveraging expertise from the European Organization for Rare Diseases – Eurordis and Rare Disease International for complex cases.

RPWA and RONARD (another collaborator) participate in the JARDIN (Joint Action for Rare Diseases on Integration of ERNs into National Healthcare Systems), and through that work towards reducing the waiting time for patients and making the patient pathway smoother.

This Joint Action, co-funded by the European Union, is part of a commitment to improve the lives of people, to make the so-called ‘patient journey’ a little easier and more accessible. Therefore, JARDIN’s mission is to improve accessibility to European Reference Networks by integrating them into National Healthcare Systems.