Key challenges for achieving equity in Rare Diseases

Issue #1: Clear communication of patient impact:

Since healthcare systems are primarily designed to address more common diseases, rare disease patients often face limitations in receiving accurate diagnoses, tailored care and specific treatments. Effective stakeholder communication is important early on in asset development to understand the disease’s impact on patients, patient specific needs and value drivers.

Solution: Primary research with stakeholders such as patient groups, key opinion leaders and payers is crucial to understanding the impact of rare diseases on individuals compared to more common diseases. This type of research can highlight health inequalities.

Engaging with both clinicians and payers to discuss these inequalities emphasises a product’s value proposition and raises awareness to rare disease indications where equity may be lacking.

Issue #2: Addressing disparity between countries:

Differences in healthcare systems across countries cause varying perceptions of rare diseases. Awareness about country-specific access and supply channels such as the existence of the Highly Specialised Technology assessment route in the UK and its interaction with NHS commissioning, is essential in the pursuit of equity, ensuring equal access to healthcare for rare disease patients, regardless of country of origin.

Solution: Market access landscaping and payer research can help bridge the gap by allowing manufacturers to understand country-specific access channels and ultimately support improved diagnosis, treatment and care for rare disease patients.

Issue #3: Understanding the importance of patient advocacy:

Individuals with rare diseases often face disproportionate effects of stigma, discrimination and social marginalisation within society. Patient advocacy plays a vital role in addressing equality however it is often underestimated. Rare Disease Day provides individuals living with a rare disease the opportunity to advocate for change at both local and national levels.

Solution: Engaging with stakeholders, in particular patient groups, through primary research can further support advocacy efforts by providing valuable insights from a patient perspective; identifying specific challenges faced by patients and facilitating the communication of unmet need to payers to address gaps in healthcare delivery.

Relevant Cogentia services:

Achieving equity in rare diseases is key to successful treatment delivery and Cogentia offer support in the following areas to help clients address the issues surrounding equity:

·???????Market access landscapes

·???????Key stakeholder engagement (KOLs, payers, patient groups)

·???????HTA assessment

Even though #RareDiseaseDay2023 has now come to a close, we will continue to raise awareness of the significant and critical impacts of rare diseases, which, despite their name, are more common than one may think.

Together we can make a meaningful difference to the lives of those affected by rare diseases.

For further information on Rare Disease Day, please visit their website at https://www.rarediseaseday.org/

#RareDiseaseDay #CareAboutRARE