Sciensus and Rare Patient Voice flag rare disease access barriers in Europe

By AI, Created 12:30 PM UTC, May 20, 2026, /AGP/ – A new poster presented at ISPOR 2026 finds rare disease patients and caregivers across five European countries still face uneven access to home delivery, long collection times and added financial burden. The results point to service gaps that could shape orphan drug launches, early access planning and patient support design.

Why it matters: - Rare disease treatment access is not just a clinical issue. The survey links logistics, awareness and support services to patient and caregiver wellbeing. - The findings suggest service design choices can affect access burden, emotional strain and out-of-pocket pressure across Europe. - The poster points to practical steps for orphan drug launch planning, early access programme design and patient support services.

What happened: - Sciensus and Rare Patient Voice, now part of Konovo, presented new data at ISPOR 2026. - The poster, Surveying treatment access and support for rare diseases in Europe: A Quantitative Cross-Country and Patient–Caregiver Comparative Analysis, examines rare disease access across the UK, France, Germany, Italy and Spain. - The analysis used a cross-sectional survey of 217 eligible respondents. - The study statistically compared outcomes between patients and caregivers and identified differences in key access indicators. - Sciensus said the work highlights treatment access burden faced by people living with rare diseases and their caregivers across Europe.

The details: - Half of respondents reported no access to home delivery or said they were unaware of it. - 28% spent more than one hour collecting treatment. - Respondents with home delivery reported significantly better wellbeing than those without it. - Home delivery access varied by country, with the highest levels of unmet need in Italy and Spain. - Caregivers were more likely than patients to lack access or awareness of home delivery. - Caregivers also spent longer collecting medicines. - Financial burden increased with collection time. - The poster says the pattern reinforces the potential value of patient-centred service design and adaptive support models.

Between the lines: - The results suggest access barriers are showing up in daily life, not just in prescribing or reimbursement. - The caregiver gap matters because rare disease care often depends on family support, so a logistics problem can spread beyond the patient. - Pam Cusick of Rare Patient Voice said clinician unfamiliarity may contribute to delays in diagnosis and treatment, while supply chain disruptions continue to affect reliable access to care. - Cusick also said the results point to psychological burden, caregiver strain and geographic disparities in access to specialists. - Sciensus Clinical Director Dr Sherif Raouf said the findings quantify how logistics, awareness and support services shape the lived treatment experience. - Sciensus Managing Director for Rare & Specialty Julie Gosper said the project shows Sciensus can amplify patient voice and turn evidence into service improvements.

What’s next: - The poster concludes that expanding home delivery should be a priority. - It also calls for better clinician education, stronger psychological support and reduced supply disruption. - Those changes would matter most for stakeholders working on orphan drug launches, early access programmes and patient support services. - Sciensus says it supports patients with complex and chronic conditions through homecare services, digital tools, distribution and real-world evidence capabilities. - Rare Patient Voice connects patients and family caregivers with research opportunities so their experiences can inform medical products and services.

The bottom line: - The study says rare disease access in Europe is still uneven, and the burden rises when treatment depends on travel, awareness gaps and weak support systems. - More flexible delivery and support models could ease that strain.