National patient perspectives on US federally-funded hemophilia treatment center care, services and gaps

Background: The US federally –funded comprehensive care hemophilia treatment centers (HTCs) provide multidisciplinary, integrated care to individuals with rare inherited bleeding disorders.  The 135 US HTCS have served as a safety network for over 35,000 individuals with these complex and costly disorders since 1976.  However, despite the longevity of the network, a systematic national patient perspective on HTC care has not been conducted.   In 2012, HRSA funded the first National Hemophilia Program Coordinating Center (NHPCC) to evaluate national service needs and establish priorities for quality improvement.  

Goal:  To conduct the first nationwide patient assessment of HTC care, services and unmet needs.

Methodology: In In fall of 2013, 133 HTCs mailed questionnaires to households of active patients who received HTC care in 2012, as defined by a standardized hemophilia dataset (HDS).   The questionnaire was anonymous, in English and Spanish, and included four pages of closed-ended questions.   The questions assessed patients’ perception of HTC services and information needed and received patient, centered care, and barriers.  

Results: Of the 24,808 questionnaires mailed, 4004 (16.1%) were returned.   Comparison to the HDS showed gender was representative (73% survey vs 69% HDS).  Under- represented were African Americans (5% survey vs 9% HDS),  Hispanic/Latino (9% survey vs 13% HDS) and younger respondents (50% vs 63%%).   Assessed barriers to care included transportation, time, distance, travel costs, lack of insurance and burden of insurance costs.  Most respondents reported no or slight barriers.   Moderate or severe barriers included time off from work or school for appointments (11% moderate, 4% severe), distance to HTC  (14% moderate, 4% severe) and high out of pocket costs (9% moderate, 7% severe).    51 items assessed problems receiving services and information.  Overall, there was a positive response to care and services received with very few gaps in services, information, and patient centered care. However, adjusted logistic regression tests revealed that the odds of having  five or more problems were twice as high for minority groups relative to Whites categories, with African Americans reporting odds almost three  times as high.

Conclusion:  This first standardized US patient needs assessment demonstrates that services and information met patients’ needs to a high degree – often with 70-80% giving the highest response.      However, minority groups had significantly more problems accessing and receiving needed care.  The NHPCC and regional HTCs need to further evaluate the challenges facing the minority populations and develop strategies to improve care.