Dr Lorna Pender
Global Patient Engagement Lead, Clinigen
Often, the routes to access support for people in the rare disease community are patient advocacy groups, charities and patient organisations. However, these organisations may lack knowledge of healthcare, industry and medicines access.
Having delivered frontline healthcare for patients in the NHS, I’ve experienced, firsthand, the difficulties faced by the rare disease community as people try to navigate access to diagnosis, medicines, early access programmes and clinical trials.
Patients missing out on treatment and information
To effectively navigate potential access to treatment routes, patient organisations require up-to-date knowledge on various areas of medicine access. Traditionally, this navigation and signposting of information would be led by the doctors and nurses when consulting with a patient.
Without a national health service medical speciality that exists to provide expertise in rare diseases, people can embark on a medical speciality ping pong for years, resulting in an uncertain diagnostic odyssey and potentially missing out on accessing medicines through early access programmes and clinical trials.
To effectively navigate potential access to treatment
routes, patient organisations require up-to-date
knowledge on various areas of medicine access.
Training programme for patient groups
Clinigen is dedicated to serving patient communities through early access programmes, clinical trial medicine supply and sourcing of hard-to-reach medicines. However, it became apparent that in order to gain a comprehensive understanding of the challenges faced by the rare community and to deliver effective programmes to our pharmaceutical and biotech clients, we would need to establish a commitment to partnership with the rare community. This partnership would provide a training programme to upskill patient groups in areas where educational needs remain unmet.
That is why Clinigen is launching Navigate, a patient organisation education programme, which will start with a research questionnaire to better understand what people in the rare community and patient groups need to know to be able to navigate towards early access, medicines development and government authorities assessing the cost-effectiveness of medicines. The training programme will be co-designed with RARE Revolution, Flutters and Strutters and CRD Consulting Ltd, which represent the voice of the rare community. With this education programme, the rare disease community can better access the solutions they need.
