w wz 16«2y Kor. J. Clin. Pharm., Vol. 16, No. 2. 2006 ü t xy y w tœ ½ BÁ x BC B y w w w C y w w w Current Status and Expectations of Orphan Drugs in Korea -In point of supplying medicines for the rare diseases- Hee-Eun Kim a and Hye-Sun Gwak a,b a Graduate School of Clinical Health Sciences, Ewha Womans University, Seoul 120-750, Korea b College of Pharmacy, Ewha Womans University, Seoul 120-750, Korea This study was aimed to investigate the current status and expectations of orphan drugs in Korea. The Korea Orphan Drug Center was established to supply many medicines for the patients with rare diseases. Among the medicines supplied by the Center, the number designated as the orphan drugs by the KFDA is quite few. However, a few of medicines are not under circulation even if the items are designated as the orphan drugs. Neoplasm-related medicines, infectious and parasitic disease-related medicines, endocrine, nutritional and metabolic disease-related medicines are the ones circulated most. There are several unapproved drugs among the medicines supplied by the Center. It s because the director of the Center can import the goods without a process to getting an approval from the KFDA. The Korea Orphan Drug Center has contributed to the selection of the medicines for treating the rare disease. On the contrary, some problems remain in the supply process. The safety and effectiveness of the medicines supplied by the Center are not guaranteed. So far, rare diseases have no specific legal definition, and therefore are only referred to in terms of the population of patients, which prevent from establishing the range of medicines. The introduction of Special Access Program or Access to Unapproved Therapeutic Goods will be the solution of these problems. In addition, it is another solution to keep intimate relations with the Rare and Intractable Disease Center and the Medicine Safety Information Center which will be open soon. Key words Orphan Drugs, Korea Orphan Drug Center, Rare Disease t e w w w yw œ w» w. ú Á w w» t y w w t t» w Áœ š. wš y e t Á w n ù, t ƒ., y ù e ƒ š w. Correspondence to : x y w w w/ w w p x 11-1 Tel: +82-2-3277-4376, Fax: +82-2-3277-2851 E-mail: hsgwak@ewha.ac.kr w, ú w» w» ƒ y w, wz w.» w y ƒw, y ƒ š v w t w ƒw 1). t w z» w ù w t wš. ù w t w y ù ƒ e w w. w t yw w w t l w. t l» ƒ, w t œ w ww w ù 2001 9 1 Õ y t 107
108 Kor. J. Clin. Pharm., Vol. 16, No. 2, 2006 t Ö š w ƒe t ww t l t ƒ w. 2002 1 12 w³ e mw t l t y e t w ww x ƒ wš w. w 2002 œ t 72 t, y 748, 2003 œ t 93 t, y 1,619, 2004 œ t 110t, y 2,903 2002 z œ t y ƒ w w ƒwš. w x t l t œ t t š t w t ww, ü ƒ t w w w w. w j ƒ wù ü w t xy w w ü m š t y e ƒ š w w w w. wù ü m t œ w ù w ƒ w t w œ.» y t ƒw t l t œ xy w w v». ù y w ƒ š w. w ƒ ƒ,,, e e w ƒ» 2). ¾ t l mw œ w t yw w». t l t t Õ t w³ Ö Áš t y e t œ wš. y e t, ³ w.» t Õ t w³ Ö» w, t l q w š w. ³ w t ü y ƒ 20,000 w w e t w y t x t w œ y e j w ƒ š t t w t. t l mw t œ ƒw, y š y ù ƒ e w t k y w d» w š. ù œ, y, t k y «, t sƒ z d üswš. t t w ü t xy r š t l mw œ t y t w, ù ƒ t œ ùkú ƒ ƒ d w š, m wz w wš w. t t t t l š w. t t š 2005-45y m ü t xy q w. w t l š m l mw œ t t y y r., t œ ü w t œ q w š w. ù txy t t š 2005-45y, Õ t w³ Ö 3) w 2005 8 3 ¾ t 107 w (»y421, 429, 617)ƒ 26 ƒ w (»y 629) 15,» (»y149) 9. 2005 10 t l mxy w t 109 t 277. 78 220 t x ü m 31 57 t m ùkû. t 28% w w m š w. m t xƒ 4 (4 t ), x ƒ w 1 (1 t ), 3 (6 t ), 2 (2t ), 19 (42 t ), ƒ 2 (2t ) 4). m ƒ xƒ, ƒ (8 ) wz m ƒ w ù 23 wz œ ƒ n w. t š ü m š w. t l tœ xy t l t œ xy 2002 72t,
ü t xy 109 y 748, 2003 93 t, y 1,619, 2004 110t 2,903 w ƒ t œ 2002 14, 2003 27, 2004 47 w w ƒ ùküš 4). t l œ w t w w w» (WHO) (ICD-10) 5)» w w 2002 t 25t œ t 35% wš, ü Á Á y t 13t 18%, Á» y t 12t 17% w. 2003 t 28t 30%, Á» y t 18t 19%, ü Á Á y t 15 t 16% ùkü. 2004 Á» y t 26t 24%, t 26t 24%, ü Á Á y t 15% w. t, Á» y t, ü Á Á y t œ wš, 3 t t w 2002 70%, 2003 65%, 2004 63% wš (Table 1). 2002 l 2004 ¾ t lƒ œ w t r, œ» ƒ œ w t 2002 t(11, 84.28%), ü Á Á y t(9, 6.72%), Á Á y t (5, 4.08%) š, 2003 t(14, 53.08%), Á» y t(9, 33.71%), ü Á Á y t(1, 4.10%), 2004 t(28, 59.8%), Á» y t(11, 24.96%), ü Á Á y t(2, 5.73%). wr y» ƒ œ w t 2002 t(574, 72.94%), Á Á y t(62, 7.88%), ü Á Á y t(44, 5.59%) š, 2003 t(964, 56.14%), Á» y t(350, 20.38%), k w t(135, 5.88%) š, 2004 t(1,530, 44.37%), Á» y t(972, 28.19%),, z t(476, 13.81%). t l œ w t t œ y ƒ ùkû. y y k, w. t lƒ 2002 2004 œ w t t 2002 12t (17%), 2003 11t (12%), 2004 13t (12%) w. t l mw œ t xƒ t xƒ t r 2004 œ t 110 Table 1. Medication classification supplied by Korea Orphan Drug Center in 2002~2004 Disease Classification Medication Number (%) Year 2002 Year 2003 Year 2004 Certain infectious and parasitic diseases 12 (17) 18 (19) 26 (24) Diseases of the blood and blood-forming organs and certain disorders involving the immune mechanism 1 (1) 3 (3) 3 (3) Diseases of the circulatory system 4 (6) 3 (3) 3 (3) Diseases of the digestive system 1 (1) Diseases of the ear and mastoid process 1 (1) 1 (1) Diseases of the eye and adnexa 2 (3) 4 (4) 4 (4) Diseases of the genitourinary system 1 (1) Diseases of the musculoskeletal system and connective tissue 1 (1) 3 (3) Diseases of the nervous system 5 (7) 11 (12) 8 (7) Diseases of the respiratory system 1 (1) 1 (1) 2 (2) Diseases of the skin andgsubcutaneous tissue 1 (1) Endocrine, nutritional and metabolic diseases 13 (18) 15 (16) 17 (15) Factors influencing health status and contact with health services 1 (1) 2 (2) 3 (3) Injury, poisoning and certain other consequences of external causes 6 (8) 5 (5) 7 (6) Mental and behavioural disorders 1 (1) 1 (1) Neoplasms 25 (35) 28 (30) 26 (24) Symptoms, signs and abnormal clinical and laboratory findings, not elsewhere classified 5 (5) Total 72 (100) 93 (100) 110 (100)
110 Kor. J. Clin. Pharm., Vol. 16, No. 2, 2006 t xƒ t ù t t 34t (23%) š, ü, x t m w œ w t 2t (1%), ü ƒ m ù xƒ œ yw w ù y ƒ w w œ w t 36t (25%). ü xƒ t w œ w 38t (35%) 6). xƒ t w» w t w Table 2 Á» y t 11t (29%), t 9t (24%), ü Á Á y t 6t (16%) ùkû. š t l t œ w t y e t š w. t t t š Õ t w³ Ö w t t w» š w. ù y e t» w», t l q w w. ƒ w ƒ. w t l mw t œ w t, Á z w t e w», y e t y w w w. Õ t w³ Ö w t ü y ( )ƒ 20,000 w w e t w y t Table 2. Disease classification of unapproved medications supplied by Korea Orphan Drug Center in 2004 Disease Classification Medication numberg(%) Certain infectious and parasitic diseases 11 (29) Neoplasms 9 (24) Endocrine, nutritional and metabolic diseases 6 (16) Diseases of the nervous system 4 (11) Factors influencing health status and contact with health services 3 (8) Diseases of the eye and adnexa 2 (5) Diseases of the blood and blood-forming organsgand certain disorders involving the immune mechanism 1 (3) Diseases of the digestive system 1 (3) Injury, poisoning and certain other consequencesgof external causes 1 (3) Total 38 (100) x t w œ y e j w ƒ š t t w t ùkù. y e t w q w š w. y w» 20,000 w ew œ m ƒ w. w ù ƒ y w, m ù 1 ƒ 20,000 w, m w y w w š w 1). t lƒ œ w t t w ù t w, y e t w. w ³ ƒe t w³e ùkù t l w t wš», w q w š w. y e t w w ƒ yw, y w» š. t l mw œ t t wš x ƒ. wr t œ y y ƒ, t l mw œ t w y»»ƒ. t lƒ xƒ ù ü ƒ xƒ t, w t l mw œ w t xw œ w., xƒ t w eù y t» yw v ƒ 7-9). ù t ƒ w,, y,,, y sww x k w w v ƒ. y k w w, xƒ e w xƒ t w» e û w y t v w w mw w w w. ü ü xƒƒ» t Á Á z w w w š y w y w t š w w sww w w. w y t Á Á z w w t l( t t ) w t
ü t xy 111 w w š, š l w ü y sww. w mƒ ó ù w m w z m w w w ü l yw w w. š ƒ xƒ t w mw œ w v ƒ, y z œ w xk ù,» l t w w w w ƒw. w v w, t w w w w w. š w t w t lƒ œ w, ü qxƒ ù w t œ ƒ w w mw. œ w w w w w l w y š w., w w œ m w w. w œ w œ w w» y 2z š w w, š ü œ ü š sww w, œ t xƒ w v w. œ w mw w v ƒ. Á z š t lƒ w, w t wš w ƒ w. m w Á z y w w, xƒ t w w, t z z» w. w z» w, w w, t y ƒ» œ w w w, š š w yw ³ w v ƒ. w œ y w t l w w» w v w. w mw t œ w w xƒ ³ ƒ. Áùe y l mw Áùe y œ t t t v w. 1999 t lƒ t l mw t œ ƒwš œ w t t y ƒwš. w ƒƒ y šm š y ƒ, š e w t k s x wr q wš» w. w r» y w ü xy w. ù y w ƒ v w, y w xk w w v ƒ. š t w ü xy w, ù t w w t t š ü m š. t l mw t œ ƒ ùkùš. t l œ t, œ y «, t, t l š ƒ k» z. t l t œ w w w w š, eù y xƒ t w r. eù y w xƒ t w ùkú w w e wš. t l mw t œ w w ƒ w. w Áùe y l t ¼ w x w y w xk t œ ƒ w. w x ƒ» w Á w. š x 1.»Á Á k. y w y z w. w z, 2000 2. š. ù y p w. w y w 2000; 5: 52-67 3. t t yr http://www.kfda.go.kr/ 4. w t l yr http://www.kodc.or.kr/
112 Kor. J. Clin. Pharm., Vol. 16, No. 2, 2006 5. WHO International Classification of Diseases yr http://www3.who.int/icd/ 6. t t t yr http://ezdrug. kfda.go.kr/ 7. Health Canada. Special access program. Therapeutic Products Directorate, 2002 8. Therapeutic Goods Administration. Access to unapproved therapeutic goods in Australia. Therapeutic Goods Administration, 2001 9. Therapeutic Goods Administration. Drugs designated as orphan drugs. Therapeutic Goods Administration, 2002