Aspire4Rare launched for better rare disease health policies

Ensuring faster diagnoses, better care, access to new treatments is goal of effort

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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A Rubik's cube is used to illustrate rare disease.

UCB has launched Aspire4Rare, a framework to “future-proof” health policies for rare diseases like aromatic l-amino-acid decarboxylase (AADC) deficiency, with the goal being to ensure faster diagnoses, better care, and access to new treatments.

Developed in collaboration with a panel of experts in rare diseases from the U.S. and Europe, the Aspire4Rare report offers a practical guide that includes tried and tested actions to steer policymakers in reforming healthcare systems.

“Rare diseases affect millions worldwide, yet their fragmentation makes it challenging to address them comprehensively within health policies. The report recognizes the current challenges in accessing appropriate care,” a news release from UCB states.

UCB’s Aspire4Rare also backs a call for a new European action plan on rare diseases. The last action plan was published more than 14 years ago and policy makers say updated coordination among European Union member states is needed.

The World Health Organization’s Health Systems Framework was used as a starting point to build on leadership and governance, access to essential medicines, health information, health service delivery, financing, and health workforce.

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How to evaluate a health policy

The panel met last year to discuss the best approach for evaluating health policies. They agreed that rare diseases should be integrated into already existing structures and health policies, but still treated as unique.

“We need to find ways to work rare disease considerations into existing policies and structures. People living with rare diseases have lots of unique challenges, but they may also get diabetes and other noncommunicable diseases,” said Sheela Upadhyaya, one of the experts.

Health policies should include patient input and be able to change, especially where resistance to change worsens geographic inequalities for those with rare diseases. Public awareness of rare diseases also needs to increase.

The panel agreed that health policies for rare diseases should focus on quality of life and ongoing research. Smooth transitions from child to adult health services are crucial, along with better data collection with a focus on patients.

Here, “patient advocacy groups have a critical role to play in collecting the data needed to make effective policy for people living with rare diseases,” said Rachel Sher, a panel member.

Along with a list of good practice examples from different countries and projects, the Aspire4Rare report features a defined set of outcomes and progress measures for each of the framework’s building blocks that can be used to assess how well local health policies perform.