Characteristics of Patients Enrolled in a Pharmacist Managed Rare Disease Program: Highlighting National Diversity and Representation
Author(s)
Kazumi Patel, MS1, Joaquim Fernandes, MS1, Dingwei Dai, PhD, MD1, Ashley Czonstkowsky, PharmD2, Lucille Accetta, RPh, MPH, MBA2, Jennifer McMahon, PharmD2, Gregg Carmen, RPh2.
1CVS Healthspire, Woonsocket, RI, USA, 2CVS Specialty Pharmacy Therapy Operations, Woonsocket, RI, USA.
1CVS Healthspire, Woonsocket, RI, USA, 2CVS Specialty Pharmacy Therapy Operations, Woonsocket, RI, USA.
Presentation Documents
OBJECTIVES: Rare diseases are defined by unique patient needs and limited treatment options, making it essential to have programs that promote equitable access and diverse representation nationwide. This study aims to characterize patients enrolled in CVS Health Pharmacist-Led Rare Disease Program (Ph-RDP), highlighting the diversity of the patients and its national reach.
METHODS: Characteristics assessed included age, gender, rare disease type, geographic region, rural-urban residency, Social Vulnerability Index (SVI) quartile and drug treatment. Program engagement metrics included duration of enrollment and number of completed assessments.
RESULTS: Among the 7,633 patients enrolled in the Ph-RDP, 55% were diagnosed with sleep disorders, primarily treated with pitolisant, while 34% had sickle cell disease, with voxelotor being the most common medication. The patient population was predominantly female (65%) with mean age of 34 years, and a geographically diverse national representation: South (46%), Midwest (22%), Northeast (18%), and West (13%). Notably, 44% of patients were in rural areas and 30% in urban areas. SVI quartile distribution showed 16% of the patients in the most vulnerable quartile, average SVI was 0.48. The program demonstrated high engagement, with 49% of patients completing at least five assessments and 48% continuing in the program for over 200 days.
CONCLUSIONS: The findings highlight the program’s broad reach, including substantial patient enrollment from rural and urban areas and high vulnerability areas as a proxy for providing accessible care to underserved populations. Patient participation and high engagement levels, evidenced by the number of assessments and duration of enrollment, emphasize the program’s potential impact in improving care and health outcomes.
METHODS: Characteristics assessed included age, gender, rare disease type, geographic region, rural-urban residency, Social Vulnerability Index (SVI) quartile and drug treatment. Program engagement metrics included duration of enrollment and number of completed assessments.
RESULTS: Among the 7,633 patients enrolled in the Ph-RDP, 55% were diagnosed with sleep disorders, primarily treated with pitolisant, while 34% had sickle cell disease, with voxelotor being the most common medication. The patient population was predominantly female (65%) with mean age of 34 years, and a geographically diverse national representation: South (46%), Midwest (22%), Northeast (18%), and West (13%). Notably, 44% of patients were in rural areas and 30% in urban areas. SVI quartile distribution showed 16% of the patients in the most vulnerable quartile, average SVI was 0.48. The program demonstrated high engagement, with 49% of patients completing at least five assessments and 48% continuing in the program for over 200 days.
CONCLUSIONS: The findings highlight the program’s broad reach, including substantial patient enrollment from rural and urban areas and high vulnerability areas as a proxy for providing accessible care to underserved populations. Patient participation and high engagement levels, evidenced by the number of assessments and duration of enrollment, emphasize the program’s potential impact in improving care and health outcomes.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HSD37
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases