Off-Label and Non-Licensed
Endocrinology Medicine Use in
Turkey: A Retrospective
Analysis of Computer Records
in the Turkish Ministry of
Health
Kockaya, Guvenc; Tanyeri, Pelin; Vural, İsmail Mert;
Akbulat, Akif; Akar, Halil; Tokaç, Mahmut; Kerman, Saim
guvenckockaya@yahoo.com
General Directorate of Pharmaceuticals and Pharmacy, Turkey
Abstract
• Objectives: Off-label is defined by the Turkish
Ministry of Health (MoHT) as the use of licensed
pharmaceutical products in doses outside of or
exceeding the scope of the registered indication, and
the use of non-licensed, but imported medicinal
products for the purpose of individual treatment.
• Methods: The use of off-label or non-licensed
endocrinology medicines was evaluated to provide an
understanding of Turkey’s perspective within this area
of health care provision. A computer search was
performed of IEGM’s database. A patient base using
off-label endocrinology medicine applications from 19
June 2009 to 19 June 2010 were searched. The key
word “endocrinology” was used in the search.
Outcomes were evaluated in the light of indications for
use.
Abstract
• Results: The computer search showed that 357
applications were submitted for off-label
endocrinology medicine use. It was concluded that the
highest application percentage was established by
“osteoporosis” in all of the applications (43%,
155/357). The highest application was established by
Ankara province (28%, 44/155). University hospitals
had the highest off-label osteoporosis medicine use
applications within the given timeline (65%, 102/155).
Specialized physicians in the fields of endocrinology
and metabolism (adult and paediatric) had the highest
number of off-label osteoporosis applications (71%,
111/155). It was concluded that the highest application
percentage was established by “teriparatide use in
osteoporosis” (87%, 136/155) in all of the osteoporosis
applications; 92 of 136 applications were approved.
There was a significiant difference between the T score
(L1-4) of rejected and approved applications for
patients (3,07 ± 1,85 and 3,23 ± 1.63, respectively; p <
0.001).
Abstract
• Discussion and conclusion: Yet, there was not a
significiant difference between ages of patients for
whom applications were rejected or approved. In
addition, it could be said that off-label use can lead to
reimbursement restrictions in endocrinology, especially
for teriparatide-like oncology medicines. In Turkey,
physicians who want to prescribe an off-label or nonlicensed pharmaceutical or a medicine that has a
different use from reimbursement indications needs to
apply through the off-label medicine use process.
• Funding sources: There was not any funding source
or conflict of interest for the manuscript
Introduction & Objectives
• In Turkey, physicians can prescribe medications offlabel or unlicensed under the control of the Ministry of
Health, General Directorate of Pharmaceuticals and
Pharmacy (IEGM) (http:// www.iegm.gov.tr).
• The IEGM evaluates off-label and unlicensed
medication use for each patient by using application
procedures. The physician who wants to prescribe an
off-label or unlicensed pharmaceutical has to apply to
the IEGM for patient base approval.
• If the IEGM approves the off-label or unlicensed
prescription, it will be reimbursed by the Turkish
Social Security Institution (SSI)
(http://www.sgk.gov.tr).
• Off-label is defined by the Turkish Ministry of Health
(MoHT) as the use of licensed pharmaceutical products
in doses outside of or exceeding the scope of the
registered indication, and the use of non-licensed, but
imported medicinal products for the purpose of
individual treatment.
Methods
• The use of off-label or non-licensed endocrinology
medicines was evaluated to provide an understanding
of Turkey’s perspective within this area of health care
provision. A computer search was performed of
IEGM’s database. A patient base using off-label
endocrinology medicine applications from 19 June
2009 to 19 June 2010 were searched. The key word
“endocrinology” was used in the search. Outcomes
were evaluated in the light of indications for use.
Results
• The computer search showed that 357 applications
were submitted for off-label endocrinology medicine
use. It was concluded that the highest application
percentage was established by “osteoporosis” in all of
the applications (43%, 155/357).
• The highest application was established by Ankara
province (28%, 44/155). University hospitals had the
highest off-label osteoporosis medicine use
applications within the given timeline (65%, 102/155).
Specialized physicians in the fields of endocrinology
and metabolism (adult and paediatric) had the highest
number of off-label osteoporosis applications (71%,
111/155).
• It was concluded that the highest application
percentage was established by “teriparatide use in
osteoporosis” (87%, 136/155) in all of the osteoporosis
applications; 92 of 136 applications were approved.
There was a significiant difference between the T score
(L1-4) of rejected and approved applications for
patients (3,07 ± 1,85 and 3,23 ± 1.63, respectively; p <
0.001).
Table 1: Application Numbers of Off-label Medicine Use in
Different Diseases Area
Disease
Aplication
Number
Osteoporosis
155
Acromegaly
40
Osteogenesis imperfecta
21
Hiperparatiroidism (Primer&Sekonder) 14
Hipoparatiroidism (Primer&Sekonder) 13
Paget Disease
9
Puberty Abnormalities
8
Turner Sendrom
8
Hypophysis adenoma (TSH releasing)
7
Growth Hormone Deficiency
6
Cushing Sendrom
5
Others
71
Total
357
Table 2: Osteoporosis Applications from Cities
City
Ankara
Adana
Afyon
Afyonkarahisar
Antalya
Aydın
Bursa
Denizli
Düzce
Edirne
Eskişehir
Hatay
Isparta
İstanbul
İzmir
Kayseri
Kocaeli
Malatya
Manisa
Mersin
Samsun
Trabzon
Numbers of Application
44
4
2
2
1
3
17
4
1
1
1
3
1
39
12
4
2
3
4
4
3
1
Conclusion
• Yet, there was not a significiant difference between
ages of patients for whom applications were rejected or
approved.
• In addition, it could be said that off-label use can lead
to reimbursement restrictions in endocrinology,
especially for teriparatide-like oncology medicines.
• In Turkey, physicians who want to prescribe an offlabel or non-licensed pharmaceutical or a medicine that
has a different use from reimbursement indications
needs to apply through the off-label medicine use
process.