A Dolon Institute campaign
Rare diseases affect millions of people across Europe, yet their low prevalence, complex biology and limited patient populations create significant barriers to developing and accessing effective treatments. This Dolon Institute infographic, produced for Rare Disease Day 2023, explores the case for treating rare diseases differently within healthcare and innovation frameworks.
The infographic sets out four key reasons why rare diseases warrant special treatment: it’s fair, it’s needed, rare diseases are fundamentally different, and we can afford it. Together, these arguments challenge the idea that rarity alone should determine how healthcare resources and investment are allocated.
It’s fair. Traditional approaches to resource allocation can disadvantage people with rare diseases because they prioritise maximising health benefits across the largest possible population. The infographic argues that rarity, alongside disease severity, should be considered when determining where investment and healthcare resources are directed.
It’s needed. The economics of rare disease development are inherently challenging. Small patient populations mean more complex and risky development programmes and smaller potential returns. Without appropriate incentives, investment is likely to flow towards more prevalent diseases. Evidence cited by Dolon suggests that dedicated orphan medicine incentives in Europe have played a significant role in stimulating innovation.
Rare diseases are fundamentally different. Conventional clinical evidence requirements can be difficult to apply when trials involve very small populations, single-arm designs, surrogate endpoints or significant disease heterogeneity. The infographic highlights the importance of incorporating patient perspectives and adapting value assessment frameworks to reflect the realities of rare disease evidence generation.
We can afford it. While orphan medicines can carry high individual prices, the infographic argues that focusing solely on headline prices or aggregate spending misses the wider dynamics of pharmaceutical expenditure. Genericisation, biosimilar competition and savings from improved outcomes can help offset increased spending on rare disease medicines.
Ultimately, the infographic argues that continuing to address unmet need in rare diseases is both a policy choice and an achievable priority. Ensuring appropriate incentives, evidence frameworks and resource allocation can help sustain innovation and improve outcomes for patients who have historically been underserved.