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💊Rare Disease Day 2026: Spotlight on Hope, Innovation & Action
Today — Rare Disease Day 2026 — millions of people, families and allies across the globe are coming together to raise awareness of the challenges and opportunities facing people living with rare diseases. Rare Disease Day was first launched in 2008 by EURORDIS–Rare Diseases Europe, and its central purpose is simple but powerful: to increase visibility for rare diseases and promote equitable access to diagnosis, care and therapies for all who need them.
This year’s campaign emphasises equity and representation, recognising that many people living with rare conditions still face barriers to care, research participation and treatment access because of where they live, what condition they have, or how health systems prioritise resources.
🧬 How Orphan Drug Designations Support Rare Therapy Development
For medicines targeting rare diseases — where patient populations are small and financial incentives for development are weak under normal market conditions — orphan drug designations are a crucial part of the pathway to new therapies.
🎯 What Is an Orphan Drug Designation?
In the European Union, a medicine can receive an orphan designation when it is intended to treat a disease that is:
- life-threatening or chronically debilitating, and
- affects no more than 5 in 10,000 people, or is unlikely to generate sufficient returns to justify development in the absence of incentives.
The EMA’s Committee for Orphan Medicinal Products (COMP) evaluates applications, and if the criteria are met, the European Commission formalises the designation.
In the United Kingdom, the MHRA evaluates orphan designations alongside marketing authorisation applications, and criteria are set out under the Human Medicines Regulations. Since the implementation of the Windsor Framework, UK orphan marketing authorisations can be considered UK-wide, regardless of whether an EU orphan designation exists.
💡 Why Orphan Drug Designation Matters
Orphan drug designation isn’t just a label — it unlocks real incentives that make development feasible:
🏆 Market Exclusivity
Once authorised, a designated orphan medicine typically enjoys 10 years of market exclusivity in the EU, meaning similar medicines cannot be approved for the same condition — a powerful incentive for investment. In many cases, this can be extended by two additional years if paediatric studies are completed.
📉 Fee Reductions & Scientific Support
Designated products can benefit from reduced regulatory fees, protocol assistance (regulatory advice tailored to rare disease methodology), and additional scientific guidance — all of which reduce financial and technical barriers for sponsors.
📊 Flexible Pathways
Regulators increasingly accommodate innovative evidence approaches, such as real-world evidence, adaptive trial designs, and rare disease-specific endpoint strategies — recognising that traditional large trials may not be feasible. This evolution was highlighted in recent Rare Disease Day discussions emphasising methodological innovation.
🤝 Patient Advocacy: Driving Progress from the Ground Up
🗣️ Patients at the Centre of Research
Patient advocacy groups — from grassroots organisations to global alliances — are critical partners in rare disease research and therapy development. Organisations such as EURORDIS–Rare Diseases Europe represent hundreds of patient groups and thousands of rare diseases, creating platforms for patient voices to influence research priorities, policy and regulatory practices.
This year’s Rare Disease Day campaign emphasises equity — not just equality — meaning that support must be tailored to individual needs to ensure fair outcomes for people living with rare conditions.
🧪 From Awareness to Action
Patient advocates work in many ways to accelerate therapy development:
- Shaping Trial Design: Patients help define what outcomes matter most — ensuring clinical trials are meaningful to real lived experience.
- Driving Funding & Policy: Advocacy networks influence research funding agendas, health policy decisions and national rare disease frameworks (such as in the UK and Europe).
- Connecting Stakeholders: By bringing together clinicians, researchers, industry and regulators, advocates help ensure rare disease communities are partners in each step of development rather than passive subjects.
These collaborative efforts not only raise the profile of rare diseases but transform awareness into action that ultimately leads to new and better therapies.
🏛️ Where Regulators Are Heading: EMA & MHRA
🌍 European Medicines Agency (EMA)
The EMA continues to evolve its approach to orphan medicines and rare disease therapies. On Rare Disease Day, key regulatory stakeholders highlighted progress on accelerating assessments, using real-world evidence, and advancing innovative methodologies to support rare drug development.
The agency also works closely with patient organisations and supports joint initiatives to improve repurposing of existing medicines for rare conditions, expanding the toolbox for regulatory recognition of unmet needs.
UK Medicines and Healthcare products Regulatory Agency (MHRA)
In the UK, the MHRA is developing a comprehensive regulatory framework to better support rare disease therapy development, licensing and delivery — part of broader work to ensure that the UK remains a supportive environment for rare disease research and access.
Combined with national rare disease action plans — such as England’s Rare Diseases Action Plan 2025, which emphasises early diagnosis, clinical research support and access to specialist care — regulators and policymakers in the UK are aligning strategies to improve outcomes for people living with rare conditions.
💜 Rare Disease Day: A Call to Keep Up the Momentum
Rare Disease Day is more than a date on the calendar — it’s a renewed call to action. It reminds us that while scientific innovation and regulatory incentives have opened new possibilities, millions of people still live without effective treatments for their conditions. Continued progress depends on collaboration between patients, advocates, researchers, policymakers and regulators.
Today — and every day — we celebrate the resilience of the rare disease community, the advances enabling new therapies, and the collective work that continues to turn awareness into equitable access, scientific progress into real results, and unmet needs into hope.
At Regvista, we remain committed to support biopharmaceutical leaders navigating their transformative journey—bringing cutting-edge therapies to market faster and safer. Please feel free to contact us by submitting your enquiry to deployment@regvista.co.uk