This campaign is initiated and funded by Takeda.

Elevate Care For Rare

Despite 1 in 17 people being affected by a rare disease,1 there is low awareness amongst the public and healthcare professionals,2 meaning people with rare diseases are often misdiagnosed and experience care inequity.3,4,5,6

And yet, it affects more of us than some people realise. Be it a loved one, a friend, a colleague, or even yourself. In the UK, this amounts to approximately 3.5 million people.1,3

So, in 2024, we are calling on Members of Parliament (MPs) to represent all their constituents and Elevate Care for Rare.

We Need Your Help

Multiple voices are stronger than one, so to Elevate Care for Rare we need your help!

We are hosting a Parliamentary event in the House of Commons with members of the rare disease community on Wednesday 24th April.

We’re calling on you to write to your local MP today and urge them to attend the event, to support the rare disease communities they represent, hear about their experiences and learn more about how they can help overcome some of the challenges people living with a rare disease face.


Ask your MP to attend
- It takes 2 minutes!

1

Pop in your postcode and find your MP and their email address here.

2

Draft a message asking your MP to attend. See below for an example.

3

Feel free to personalise the suggested template we’ve created and provide personal examples if you feel comfortable.

4

Hit send!

Dear XXX,

I am writing as your constituent to ask you to attend Takeda’s I am number 17: Elevate Care for Rare drop-in event, after Prime Minister’s Questions on Wednesday 24th April between 13:30-15:30, in Terrace Dining Room A.

Despite 1 in 17 people being affected by a rare disease, there is low awareness amongst the public and healthcare professionals, meaning people with rare diseases are often misdiagnosed and experience care inequity. Therefore, the theme of the campaign is to ‘Elevate Care for Rare’, calling for everyone living with a rare disease to have access to high quality care, regardless of the prevalence of their condition…

Find out your local MP and contact details


Together, we can make a difference and ensure that the 1 in 17 affected by a rare disease get the care and support they need.

Let's not wait any longer.

Let's act NOW to ELEVATE CARE FOR RARE!

What will the Parliamentary event look like and what are we calling for?


During the Parliamentary event, we will be calling for equitable access to care for people affected by a rare disease. This means people living with a rare disease should be able to access the care they need, whenever and however they need it, irrespective of the prevalence of their condition.

To make this happen, we are calling for the inclusion of a statement within the NHS Constitution recognising the need for equity in access to high quality care for all patients in England, regardless of their condition.

Beyond this, we are calling on Parliamentarians to advocate for our policy missions that will directly support with the core challenges people living with a rare disease face in the healthcare system.



>90%

In a survey of rare disease patients and carers, the majority reported having felt anxious, stressed, and/or depressed due to the condition.7

Survey of 1,795 rare disease patients and carers – for this question eligible responses came from 1231 patients and 564 carers, with over 1683 respondents to this question

19%

In a survey, 19% of people living with a rare disease said a challenge they have faced was awareness amongst healthcare professionals.3

Survey of 6,293 people (68% were individuals living with a rare disease)

12%

In a survey of patients affected by rare disease, only 12% of people living with rare disease have a care coordinator.4

Survey of 760 adult patients affected by a rare disease

Our Policy Missions


1. Reducing variation in services

Many people living with a rare disease do not have a diagnosis, while others have to undergo years of tests, hospital appointments and misdiagnoses before receiving theirs.8 The diagnostic odyssey often represents a challenging and taxing journey, leading to substantial financial, emotional and societal impacts.8,9

Beyond diagnosis, there is variation and inequity in the organisation of services received by the rare disease community. This includes excessive appointment scheduling, lack of communication between providers, and a shortage of resources and capacity within services.10

Did you know?

  • From a survey of patients and caregivers*, it takes an average of 5.6 years, 8 clinicians and 2-3 misdiagnoses before the correct rare disease is identified.8
  • During the 10 years prior to diagnosis, rare disease patients cost NHS England in excess of £3.4 billion.11**
  • Whilst undiagnosed, the cost per patient is on average, more than twice that of other patients – an average difference of £7,000 more per year.9,11**

* Survey of 887 patients and caregivers based in the US and the UK
** Report published in November 2018

Our Policy asks

  • NHS service specifications for rare conditions must include outcomes aligned with what is important to patients
  • England’s 2025 Rare Diseases Action Plan should commit to the inclusion of rare diseases in the Core20PLUS5 framework

Our Policy asks

  • From a survey of patients and caregivers*, it takes an average of 5.6 years, 8 clinicians and 2-3 misdiagnoses before the correct rare disease is identified.8
  • During the 10 years prior to diagnosis, rare disease patients cost NHS England in excess of £3.4 billion.11**
  • Whilst undiagnosed, the cost per patient is on average, more than twice that of other patients – an average difference of £7,000 more per year.9,11**

* Survey of 887 patients and caregivers based in the US and the UK
** Report published in November 2018

  • NHS service specifications for rare conditions must include outcomes aligned with what is important to patients
  • England’s 2025 Rare Diseases Action Plan should commit to the inclusion of rare diseases in the Core20PLUS5 framework

2. Improving coordination of care

The healthcare landscape as a rare disease patient is a complex journey. It is often marked by unique challenges that can significantly impact mental health.7

Research indicates that people with a rare disease report having a worse experience of health care compared with those living with more chronic diseases,12 often having to manage their own care with no access to a care coordinator or care plan.4,5,6

The burden of care coordination can affect many facets of a patient’s life; often rippling out to affect relationships, employment and families and carers.13

Did you know?

In a survey of patients affected by a rare disease*

  • Only 10% of people living with a rare disease have a care plan.4
  • Only 12% of people living with a rare disease have a care coordinator.4

* Survey of 760 adult patients affected by a rare disease

Our Policy asks

  • We are calling for all patients to have access to care coordinators in rare disease services
  • NHS England should introduce digital care records or patient passports for individuals living with a rare disease

Our Policy asks

In a survey of patients affected by a rare disease*

  • Only 10% of people living with a rare disease have a care plan.4
  • Only 12% of people living with a rare disease have a care coordinator.4

* Survey of 760 adult patients affected by a rare disease

  • We are calling for all patients to have access to care coordinators in rare disease services
  • NHS England should introduce digital care records or patient passports for individuals living with a rare disease

3. Strengthening awareness of a rare disease

Despite the substantial impact rare disease can have on individuals and their families, they often remain under-discussed and obscure, both amongst the public and in health services.2

This double blow increases the burden on patients to equip themselves with knowledge of their condition and become their own advocates and activists.8

Did you know?

  • In a survey, 19% of people living with a rare disease said a challenge they have faced was awareness amongst healthcare professionals.3*
  • Another challenge that is particular to rare conditions and which negatively affects mental health is not being believed or taken seriously by healthcare professionals.7**

* Survey of 6,293 people (68% were individuals living with a rare disease)
** Survey of 1,795 rare disease patients and carers

Our Policy asks

  • We are calling for the establishment of a ‘rare aware’ designation that can be awarded to hospitals/GP practices that have trained their staff on rare disease care
  • We are calling for the mandatory inclusion of rare disease education modules in professional qualifications overseen by the GMC and NMC

Our Policy asks

  • In a survey, 19% of people living with a rare disease said a challenge they have faced was awareness amongst healthcare professionals.3*
  • Another challenge that is particular to rare conditions and which negatively affects mental health is not being believed or taken seriously by healthcare professionals.7**

* Survey of 6,293 people (68% were individuals living with a rare disease)
** Survey of 1,795 rare disease patients and carers

  • We are calling for the establishment of a ‘rare aware’ designation that can be awarded to hospitals/GP practices that have trained their staff on rare disease care
  • We are calling for the mandatory inclusion of rare disease education modules in professional qualifications overseen by the GMC and NMC

References

1 Rare Disease UK. Facts and figures https://geneticalliance.org.uk/campaigns-and-research/facts-and-figures/ [Last accessed April 2024].

2 Genomics Education Programme, Rare disease education hub. https://www.genomicseducation.hee.nhs.uk/rare-disease-education-hub/ [Last accessed April 2024].

3 Department of Health and Social Care 2021, UK Rare Diseases Framework. https://www.gov.uk/government/publications/uk-rare-diseases-framework [Last accessed April 2024].

4 Morris S, et al. Co-ordinated care for people affected by rare diseases: The CONCORD mixed-methods study. Health and Social Care Delivery Research. 2022 Mar 1;10(5):1-220.

5 NHS, National Cancer Patient Experience Survey 2022. https://www.ncpes.co.uk/latest-national-results/ [Last accessed April 2024].

6 2022 UK Parkinson’s Audit, Summary Report. https://www.parkinsons.org.uk/sites/default/files/2023-06/2022%20Summary%20Report%20-%20FINAL.pdf [Last accessed April 2024].

7 Spencer-Tansley R, et al. Mental health care for rare disease in the UK–recommendations from a quantitative survey and multi-stakeholder workshop. BMC Health Services Research. 2022 May 14;22(1):648.

8 Global Genes, Rare Disease Impact Report: Insights from patients and the medical community. https://globalgenes.org/wp-content/uploads/2013/04/ShireReport-1.pdf [Last accessed April 2024].

9 Health Data Research UK, Cloud-based integration of phenotype and genotype data for rare disease research. https://www.hdruk.ac.uk/projects/cloud-based-integration-of-phenotype-and-genotype-data-for-rare-disease-research/ [Last accessed April 2024].

10 Genetic Alliance UK, The Hidden Costs of Rare Diseases: A feasibility study. https://geneticalliance.org.uk/wp-content/uploads/2024/02/hidden-costs-2016.pdf [Last accessed April 2024].

11 Mendelian, Health Economics Report. https://www.mendelian.co/blog/the-cost-of-undiagnosed-rare-diseases [Last accessed April 2024].

12 Eurordis, Improve our experience of health care! https://download2.eurordis.org/rbv/HCARE/HCARE_FS_long.pdf [Last accessed April 2024].

13 Rare Disease UK, Illuminating the Rare Reality. https://geneticalliance.org.uk/wp-content/uploads/2024/02/Illuminating-the-rare-reality-2019.pdf [Last accessed April 2024].

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