Clinigen has published new data from its NaviGate programme revealing knowledge gaps within the rare disease patient community to spread awareness for Rare Disease Month (February 2024).
The data aims to raise awareness of over 7,000 rare diseases, which impact more than 300 million people globally.
First launched in September last year, the NaviGate programme is a UK-based pilot educational programme for rare disease patient advocates and organisations that aims to equip the rare disease community to facilitate greater participation in research and development, as well as enable increased access to medicines for more patients.
Involving 50 rare disease patients, parents or carers, patient advocates, organisations and charities, the questionnaire aims to understand the potential knowledge gaps within the rare disease community.
Results revealed that 42% of participants represented patients who were undiagnosed, showing a significant gap in research and knowledge.
Furthermore, the data revealed a knowledge gap surrounding unlicensed medicines – medicines that have not been taken to market in a region due to the manufacturer deciding there are not enough patients for a clinical trial or the trial is too expensive.
Despite 68% of the rare disease patient and carer community being comfortable receiving unlicensed medicines, 32% were not aware that patients could access medicines before they are licensed.
Additionally, only 30% of respondents had heard of early access programmes (EAPs) that provide people with life-threatening or debilitating conditions early access to medicines without commercial authorisation – while only four respondents used EAPs confidently.
In total, 74% of respondents were interested in knowing about available EAPs and 69% wanted to know where they could access quality information about them.
The company aims to build an educational training programme for patients, families, community groups and organisations to use as a resource.
Dr Lorna Pender, global patient engagement lead, Clinigen, said: “Patients need to be backed by knowledge and resources and knowing where they can get access to medicines – even before they are on the market – is vital for people with undiagnosed or rare diseases.
“In collaboration with patients and patient advocacy groups, we aim to share our knowledge with the wide and deserving rare disease community through this initiative.”