Author: Sam Bryant, Regulatory Affairs Manager
On 24 May 2025, the World Health Organisation (WHO) adopted a landmark resolution that officially recognises rare diseases as a global health priority. This historic move, endorsed by all Member States at the 78th World Health Assembly, marks a pivotal shift in how rare diseases are addressed within global health systems.
For the more than 300 million people worldwide living with one of over 7,000 rare diseases, this resolution is a long-overdue acknowledgment of their unmet needs. But for biotech companies operating in this space, it also signals new opportunities—and responsibilities.
What the resolution says
The resolution urges countries to:
– Integrate rare diseases into national health planning
– Improve diagnosis and care through universal health coverage (UHC)
– Promote inclusive health policies
– Accelerate innovation, research, and access to affordable treatments
– Support the development of a 10-year global action plan with measurable targets for equity and access
This is more than symbolic. It lays the groundwork for policy harmonisation, funding prioritisation, and regulatory alignment—all of which are critical for biotech developers navigating fragmented global markets.
What this means for biotech companies
For companies developing orphan drugs, the resolution could catalyse:
– Faster regulatory pathways as countries align with WHO’s global action plan
– Improved reimbursement frameworks through UHC integration
– Greater visibility and support for early-stage innovation
– Stronger incentives for cross-border collaboration and data sharing
However, the path forward will still require navigating complex national systems, particularly in Europe, where market access remains highly decentralised for orphan drugs until the application of the Joint Clinical Assessment from 2028.
How to utilise this strategic inflection point
At Kintiga, we see this resolution as a strategic inflection point. With our deep expertise in rare diseases and pan-European market access, we help biotech innovators translate global policy shifts into tangible access outcomes. Whether you are preparing for HTA submissions, navigating pricing negotiations, or planning multi-country launches, our team is here to guide you through the evolving landscape.
At Kintiga, we simplify the complex journey to achieve successful patient access across Europe. By uniting deep local market access expertise, a global perspective, and innovative strategies, we partner with health technology developers to deliver impactful, tailored solutions, maximising both market potential and patient outcomes for orphan drug therapies and beyond.
For further support with understanding how these regulations and framework could impact your market access strategy, please reach out to Kintiga via enquiries@kintiga.com.
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