The case for integrated service delivery in the UK for patients with rare diseases

Rare Disease UK is a national alliance of key stakeholders including patient organisations, industry, clinicians, academics and individuals bought together to call for a response to the unmet care needs of those with rare diseases who currently struggle to get access to integrated care and support from the NHS. There are over 6000 rare diseases affecting over 3.5 million people (one in 17) in the UK. Collectively, rare diseases are not rare.

On 9 June 2009 EU Health Ministers unanimously adopted the European Council's Recommendation on an action in the field of rare diseases. The Recommendation calls on Member States to elaborate and adopt plans or strategies for rare diseases to ensure that patients with rare diseases have access to high quality care, including diagnostics, treatments, habilitation for those living with rare disease and, if possible, effective orphan drugs. It is imperative that the UK Government embraces the Recommendation as an opportunity to effect real improvements for people with rare diseases. Rare Disease UK will continue to push for a strategic plan for integrated service delivery for individuals and families affected by rare disease across the UK.

Why we need a national strategy for integrated service delivery

Although much progress has been made in the past decade with innovative commissioning strategies and the establishment of centres of excellence delivering multidisciplinary care for some conditions, there is currently no coherent strategy for care and support for people affected by rare disease. Many patients with rare conditions have difficulties accessing high quality care and services, in many cases due to the lack of communication and co-ordination of services already in existence. Due to the rarity of many conditions, health professionals often have little or no experience in supporting patients in order to find optimum care pathways which can often be difficult or impossible. This leaves patients feeling frustrated and as if they're rattling around the system.

A national strategy for integrated service delivery is needed to improve the current fragmentation of services and enable patients and health professionals to provide and use best practice care. This will ensure that all patients with rare diseases can not only be diagnosed quickly, but also have timely access to the care and support they need.

Wider benefits

As well as benefiting patients and families affected by rare disease, a strategy for integrated service delivery would help the NHS in the UK make the most efficient use of resources and scarce expertise by targeting healthcare resources for maximum benefit. At a time where funding for public services is likely to be under pressure, more effective use of the available resources is particularly important.

A clear strategy on rare disease research, embracing both the academic and private sectors, would help to ensure the UK's position as a world leader in biomedical research - an objective which the Office for Life Sciences recently emphasised was vitally important to the UK in its Blueprint. Coordinating and fostering rare disease research would mean more possibilities for diagnosis and treatment, make the UK a more attractive research location and lead to knock-on benefits for medical research in general, particularly for conditions with a genetic component such as asthma or diabetes.

The UK is in danger of slipping behind other EU Member States that are taking action. France has implemented a comprehensive plan, the Netherlands has implemented a national strategy and Italy has adopted regional strategies. Bulgaria and Portugal have also adopted national plans. The UK needs to consider the Recommendation as a positive opportunity for patients with rare disease in this country.

Next steps

Rare Disease UK is aiming to work in partnership with government, both national and devolved, and colleagues in the NHS across all four home nations to develop and implement an effective strategy for integrated service delivery. We have set up five Working Groups of expert stakeholders to look into the issues that a national strategy will need to address and to develop a set of measures to ensure the effectiveness of a strategy in the UK. We aim to ensure that the commitment expressed by adopting the Recommendation is now acted upon and that a real difference is made to the lives of the millions of patients living with rare disease.

For more information on Rare Disease UK or to join the alliance visit The Rare Disease UK Alliance Website.