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Budgetary Impact of Medicinal Therapies for Rare Diseases in Bulgaria

Iskrov, Georgi G ; Jakovljevic, Mihajlo Michael and Stefanov, Rumen S (2018) In Folia medica 60(1). p.79-91
Abstract

BACKGROUND: Rare diseases have been continually outlined as one of the causes for the National Health Insurance Fund's (NHIF) deficit spending in Bulgaria.

AIM: To estimate the budgetary impact of rare disease medicinal therapies from NHIF perspective for 2014 and 2016.

MATERIALS AND METHODS: Budgetary impact of rare diseases is calculated as a percentage of NHIF total pharmaceutical spending. Total expenditure per ICD-10 code, mean annual number of patients reimbursed and mean annual cost per patient are analysed.

RESULTS: Budgetary impact of rare diseases reached a plateau of about 9% of NHIF total pharmaceutical spending for 2014-2016. Mean number of patients reimbursed and mean annual cost per patient increased by... (More)

BACKGROUND: Rare diseases have been continually outlined as one of the causes for the National Health Insurance Fund's (NHIF) deficit spending in Bulgaria.

AIM: To estimate the budgetary impact of rare disease medicinal therapies from NHIF perspective for 2014 and 2016.

MATERIALS AND METHODS: Budgetary impact of rare diseases is calculated as a percentage of NHIF total pharmaceutical spending. Total expenditure per ICD-10 code, mean annual number of patients reimbursed and mean annual cost per patient are analysed.

RESULTS: Budgetary impact of rare diseases reached a plateau of about 9% of NHIF total pharmaceutical spending for 2014-2016. Mean number of patients reimbursed and mean annual cost per patient increased by median rates of 4.27% and 2.54%, respectively. Glycogen storage disease, neuropathic heredofamilial amyloidosis and C1 esterase inhibitor deficiency stood out, as they had the second, fourth and fifth most expensive medicinal treatment cost. While accounting for only 92 patients in 2016, these three conditions contributed for 22.89% of NHIF total expenditure on rare disease medicinal therapies. For comparison, coagulation defects, with the biggest total cost per indication, had a similar budgetary impact - 24.88%, but for 277 patients reimbursed.

CONCLUSIONS: Our study does not support the concerns about uncontrolled growth of expenditures for rare disease medicinal therapies. Nevertheless, there is a need for enhanced post-marketing surveillance and performance-based payment of these treatments. Development, collection and analysis of local real-world data have been increasingly applied as a tool to advance these health policy goals.

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author
; and
publishing date
type
Contribution to journal
publication status
published
subject
in
Folia medica
volume
60
issue
1
pages
79 - 91
publisher
University of Medicine
external identifiers
  • pmid:29668449
  • scopus:85065797418
ISSN
0204-8043
DOI
10.2478/folmed-2018-0015
language
English
LU publication?
no
id
20398c7c-5c78-426a-a486-f1ffb565b517
date added to LUP
2018-09-01 22:28:33
date last changed
2024-04-15 10:42:40
@article{20398c7c-5c78-426a-a486-f1ffb565b517,
  abstract     = {{<p>BACKGROUND: Rare diseases have been continually outlined as one of the causes for the National Health Insurance Fund's (NHIF) deficit spending in Bulgaria.</p><p>AIM: To estimate the budgetary impact of rare disease medicinal therapies from NHIF perspective for 2014 and 2016.</p><p>MATERIALS AND METHODS: Budgetary impact of rare diseases is calculated as a percentage of NHIF total pharmaceutical spending. Total expenditure per ICD-10 code, mean annual number of patients reimbursed and mean annual cost per patient are analysed.</p><p>RESULTS: Budgetary impact of rare diseases reached a plateau of about 9% of NHIF total pharmaceutical spending for 2014-2016. Mean number of patients reimbursed and mean annual cost per patient increased by median rates of 4.27% and 2.54%, respectively. Glycogen storage disease, neuropathic heredofamilial amyloidosis and C1 esterase inhibitor deficiency stood out, as they had the second, fourth and fifth most expensive medicinal treatment cost. While accounting for only 92 patients in 2016, these three conditions contributed for 22.89% of NHIF total expenditure on rare disease medicinal therapies. For comparison, coagulation defects, with the biggest total cost per indication, had a similar budgetary impact - 24.88%, but for 277 patients reimbursed.</p><p>CONCLUSIONS: Our study does not support the concerns about uncontrolled growth of expenditures for rare disease medicinal therapies. Nevertheless, there is a need for enhanced post-marketing surveillance and performance-based payment of these treatments. Development, collection and analysis of local real-world data have been increasingly applied as a tool to advance these health policy goals.</p>}},
  author       = {{Iskrov, Georgi G and Jakovljevic, Mihajlo Michael and Stefanov, Rumen S}},
  issn         = {{0204-8043}},
  language     = {{eng}},
  month        = {{03}},
  number       = {{1}},
  pages        = {{79--91}},
  publisher    = {{University of Medicine}},
  series       = {{Folia medica}},
  title        = {{Budgetary Impact of Medicinal Therapies for Rare Diseases in Bulgaria}},
  url          = {{http://dx.doi.org/10.2478/folmed-2018-0015}},
  doi          = {{10.2478/folmed-2018-0015}},
  volume       = {{60}},
  year         = {{2018}},
}