Aparito has dedicated the last seven years to creating change in the clinical trial experience for patients living with a rare disease. Our contribution to the rare disease space was acknowledged in 2022 with the illustrious EURORDIS Black Pearl Award for Health Technology.
Rare disease clinical trials
A rare condition is defined by its prevalence in the population. In the US, it means a disease affecting fewer than 200,000 people at any given time. In Europe, it means affecting fewer than 5 people in 10,000.
According to a 2021 rare diseases conference, it is established that among 7,000 rare diseases that we know, 95% have not yet effective treatment. Due to the low number of patients and their difficulty to travel, recruitment may be extremely complicated. The design itself of the trials imposes additional barriers since the diseases are not well characterised. Patient engagement is of utmost importance in such cases since patients are often experts when it comes to understanding the natural progression of their condition.
Nevertheless, those last two years have opened new ways of thinking: clinical trials were delayed, interrupted or not even started especially in the area of rare diseases. Decentralised and hybrid clinical trials became viable options and could bring more opportunities to conduct rare disease trials during periods of travel restriction and increased risk to already vulnerable patients.
This academic-led trial for patients affected by Duchenne Muscular Dystrophy (DMD) emphasises the barriers encountered in organising an international, multi-centre trial for a rare condition (Checklist and future).
Rare disease clinical trials bring challenges in both enrolment and study design.
In addition to those, developing medicines for rare diseases is often cut short by lack of funding due to perceptions that it does not deliver a favourable return on investment.
Aparito has a commitment to rare disease patients and we see opportunities rather than obstacles that leverage our Atom5TM technology to make a difference in patients’ lives and create more meaningful outcomes.