• Title/Summary/Keyword: Orphan

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Current View of Orphan Drug Usage in Tertiary Hospital and Rare Incurable Disease Hospital (상급종합병원과 희귀난치성질환 전문병원의 희귀의약품 사용현황)

  • Choi, Kyung Suk;Jeong, Young mi;Kim, Yu Jeong;Kim, Yoon Hee;Gu, Hyunmin;Lee, Byung Koo;Lee, Eunsook;Rhie, Sandy Jeong
    • Korean Journal of Clinical Pharmacy
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    • v.26 no.2
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    • pp.121-127
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    • 2016
  • Objective: Until now, there is minimal number of research for overall domestic status of orphan drug use in Korea. The purpose of this study is to identify the list of orphan drugs available in Korea and to understand the status of orphan drug usage in tertiary Hospitals and rare incurable disease Hospital. Methods: We made domestic orphan drug lists based on available orphan drugs in Korea. Based on this lists, we conducted e-mail survey from August, 2014 to September, 2014 to identify domestic status of orphan drug usage including the availability and management of orphan drugs. Results: There are three hundred and eighteen orphan drugs (184 ingredients) registered in Ministry of Food and Drug Safety. Among the three hundred and eighteen orphan drugs, Two hundred and twenty-eight drugs (102 ingredients) were selected. Information on each item was collected and documented with generic and brand names, manufacturers, wholesalers, indications, FDA approval status and insurance coverage. Forty-three tertiary hospitals and thirty-two rare incurable hospitals responded to the survey questionnaire (57.3%). According to the survey result, the antineoplastics and immunomodulating agents group has the highest percentage (40%) usage in the hospital. Of fortythree tertiary hospitals, thirteen hospitals manage orphan drugs separately (30.2%). Based on the reply, most of the healthcare professionals commented the drug information related to efficacy and safety including medication counseling of orphan drugs is insufficient. Conclusion: Through this study we anticipate providing an understanding of orphan drug usage status in Korea. We found the limited resources to the information on orphan drugs and this information requires updating on a regular basis. This can be the basis for further studies about preparing drug information, educational resources for rare disease patients.

Rare Disease Research and Orphan Drug Development (희귀질환 연구와 치료제 개발 동향)

  • Lee, Shinhaeng;Lee, Jangik I.
    • Korean Journal of Clinical Pharmacy
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    • v.23 no.1
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    • pp.1-13
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    • 2013
  • 희귀질환이란 일반적으로 그 유병율이 인구 일만 명당 5명 이하인 질환을 말한다. 세계에는 약 7,000여종의 희귀질환이 알려져 있고 학술지에 매 주 대략 5종의 새로운 희귀질환이 보고되고 있다. 희귀 의약품(orphan drug)이란 희귀질환치료제 또는 수익성이 없어 개발을 기피하는 일반질환 치료제로서 정부가 지정한 의약품을 말한다. Orphan 의약품의 개발에는 많은 장점이 있다. Orphan 의약품으로 정부의 지정을 받으면 세금감면을 통한 연구비 지원, 임상시험 비용 지원, 신약허가 심사비 면제, 시장독점권 부여 등의 특혜가 주어진다. 희귀질환의 대부분은 단순한 유전적 결함에 의하는 경우가 많아 치료제의 표적발견이 비교적 쉬우므로 개발 성공률이 높고, 임상시험기간이 짧으며, 시판허가를 받을 확률이 높아 연구개발비용이 적게 든다. 그 결과 전세계 orphan 의약품 시장은 최근 매년 6%씩 성장하여 2014년에는 약 1,120억달러의 시장을 형성할 것으로 추정된다. Orphan 의약품 시장은 현재 매년 8.9%씩 성장하고 글로벌 시장의 51%를 점유하는 미국을 중심으로 확장되어 가고 있으며, 총매출액의 64.3%가 유전자재조합의약품에 의한 것으로 알려져 있다. 이러한 의약품시장의 변화와 사회적 요구에 부응하여 한국 또한 희귀질환 치료제 개발의 활성화를 위해 재정적 지원체계를 구축하고, 허가관리를 개선하며, 법률적 제도를 완비하는 과정에 있다. 현재 희귀질환의 치료적 타겟을 찾아 신물질이나 기존의 약물을 발굴하는 과정이 주로 대학이나 연구 중심 병원에서 이루어지고 있다. 제도가 잘 정립되어 있는 미국 시장을 겨낭하여 orphan 의약품 개발을 전략적으로 수행한다면 큰 성공을 거둘 수 있을 것으로 기대된다.

A Study on the Transition from the Library's Copyright Compensation Regime to the Orphan Works Regime (도서관 보상금 체제에서 고아저작물 체제로의 전환 모색)

  • Joung, Kyounghee
    • Journal of the Korean Society for Library and Information Science
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    • v.48 no.4
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    • pp.193-214
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    • 2014
  • Thise study focuses on the direction to the revisions of copyright law in Korea, so that orphan works in libraries can be used more widely on the Internet. In particular, this study analyzes the problems of the provisions 31 and 50 in the copyright law related to the mass digitations of orphan works in libraries. This study also reviews the Directive 2012/28/EU, 3 orphan works bills in USA, and provisions related to the orphan works in The Enterprise and Regulatory Reform(ERR) Act 2013 in UK. Finally this study suggests the 3 directions to support mass digitations of orphan works in libraries. First, digitized orphan works in libraries are to be made a transmission to the public on the Internet; Second, compensation and approval fees are not required or minimized; and third, libraries have to stop the use of the orphan works if copyright owners require. If not, libraries have to negotiate with the copyright owners about the fees necessary.

A Study on the Nutritional Status of Children Living in Rural Area and City Orphan Asylum -I. Dietary Status and Physical Growth- (농어촌(農漁村)과 시설거주(施設居住) 아동(兒童)의 영양상태(營養狀態)에 관(關)한 연구(硏究) -제1보(第1報) : 식이실태(食餌實態)와 신체발육(身體發育)-)

  • Park, Bock-Hee
    • Journal of the Korean Society of Food Science and Nutrition
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    • v.14 no.2
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    • pp.99-107
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    • 1985
  • This study was carried out to investigate the nutritional and physical status of children, aged 11 and 12 years, living in Haenam Koon (living at home) and in Mokpo City (living in an orphan asylum) located in Chonnam during the period of August 2 nd to 9 th in 1984. The mean values of height, chestgirth, arm circumference and head circumference between both groups of living in the rural area and in the city orphan asylum were not statistically different and similar to Korean Growth Standard. Exceptionally, the values of height and weight of male living at home in the rural area were lower than Korean Growth Standard (p<0.01). The weight of male living in the city orphan asylum was higher han that of male living at home in the rural area (p<0.01), and the skin fold thickness of female living at home was higher than that of male living at home (p <0.01). The physical indices of children living in the city orphan asylum were more or less higher than those of living at home in the rural area, but the relative weight of male was only significant (p<0.05). The animal protein intake was $8.4{\sim}8.6%$ of total food intake of living at home in the rural area and 6.2% in living in the city orphan asylum. The intake of legumes of children living in the city orphan asylum was more two times than that of living at home in the rural area, but overall fruit intake of living at home was more about two times than that of living in the city orphan asylum. The average intake of all nutrients except thiamine and niacin was lower than RDA. Especially the calcium intake of children in both groups was the lowest of all nutrients ($28.9{\sim}40.6%$ of RDA). Children living at home in the rural area had significantly higher intake of fat and ascorbic acid than children living in the city orphan asylum (p<0.01).

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Current Status and Expectations of Orphan Drugs in Korea -In point of supplying medicines for the rare diseases- (국내 희귀의약품의 현황 및 과제 -희귀질환에 대한 의약품 공급을 중심으로)

  • Kim, Hee-Eun;Gwak, Hye-Sun
    • Korean Journal of Clinical Pharmacy
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    • v.16 no.2
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    • pp.107-112
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    • 2006
  • 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.

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Recent Progress in Orphan Nuclear Hormone Receptors

  • Lee, Yoon-Kwang;Tzameli, Iphigeoia;Zavacki, Ann Marie;Moore, David D.
    • BMB Reports
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    • v.31 no.5
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    • pp.419-426
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    • 1998
  • The nuclear hormone receptor superfamily currently includes approximately equal numbers of conventional receptors and orphan receptors, which do not have known ligands. Here, we review recent progress from this laboratory on three orphans, two of which are moving from orphan to conventional receptor status. Perhaps the most unusual is CAR, which is a constitutive transactivator in the absence of ligands but becomes transcriptionally inactive in the presence of its ligands, which are androgen metabolites. The response of CAR to its ligands is thus opposite to that of the conventional receptor paradigm. RIP14 (also known as FXR) is activated by both all-trans retinoic acid and a synthetic retinoid previously thought to specifically target the retinoic acid receptors (RARs), and thus appears to be a novel retinoid receptor. Finally, SHP is a novel orphan that lacks a DNA binding domain and interacts with a number of other receptor superfamily members. While it generally inhibits its targets, including CAR, the retinoid X receptor (RXR), and the estrogen receptor (ER), it stimulates transactivation by the orphan SF-1.

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Analysis of reports on orphan lung diseases in Korean children

  • Jang, Sun-Jung;Seo, Hyun-Kyung;Yi, Sung-Jae;Kim, Kyong-Min;Jee, Hye-Mi;Han, Man-Yong
    • Clinical and Experimental Pediatrics
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    • v.53 no.6
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    • pp.711-717
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    • 2010
  • Purpose: Orphan lung diseases are defined as lung diseases with a prevalence of 1 or less in 2,000 individuals. Despite an increase in the numbers of patients with such diseases, few studies on Korean children have appeared. To obtain epidemiologic and demographic data on these diseases, we systematically reviewed reports on pediatric orphan lung diseases in Korea over the last 50 years. Methods: We reviewed 223 articles that have appeared since 1958 on orphan lung diseases in Korean children. These articles described a total of 519 patients aged between 0 and 18 years. We classified patients by year of publication, diagnosis, geographic region, and journal. Results: Of 519 patients, 401 had congenital cystic lung diseases and 66 had bronchiolitis obliterans. About 80% of patients were described in reports published in three journals, Pediatric Allergy and Respiratory Disease (Korea), the Korean Journal of Pediatrics, and the Korean Journal of Thoracic and Cardiovascular Surgery, in which papers on 157 (30.2%), 138 (26.6%), and 111 (21.4%) patients appeared, respectively. The frequency of publication of case reports has increased since 1990. Of the 519 patients, 401 (77.3%) were from Seoul/Gyeonggi-do and 72 (13.9%) from Busan/Gyeongsangnam-do. Conclusion: The prevalence of pediatric orphan lung disease has increased since 1990, and some provinces of Korea have a higher incidence of these diseases than do others. Studies exploring the incidence of pediatric orphan lung diseases in Korea are needed for effective disease management.

A Study on the Nutritional Status of Children Living in Rural Area and City Orphan Asylum -II. Hematological view- (농어촌(農漁村)과 시설거주(施設居住) 아동(兒童)의 영양상태(營養狀態)에 관(關)한 연구(硏究) -제2보(第2報) : 혈액성장(血液性狀)을 중심(中心)으로-)

  • Park, Bock-Hee
    • Journal of the Korean Society of Food Science and Nutrition
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    • v.14 no.2
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    • pp.108-116
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    • 1985
  • This study was designed to investigate the nutritional anemic state and hematological findings of children, aged 11 and 12 years, living in Haenam Koon (living at home) and in Mokpo City (living in an orphan asylum) located in Chonnam. The mean red blood cell number of male was higher than female in both groups of living in the rural area and in the city orphan asylum (p<0.01), but the white blood cell count was not significantly different. The levels of average hemoglobin, hematocrit and mean corpuscular hemoglobin concentration of the children in both groups were similar, and 19.8% of children living at home in the rural area and 32.1% of children living in the city orphan asylum were anemic. The mean levels of serum total protein, albumin and A/G ratio in children of both group were not statistically different, and 18.4% of children living at home in the rural area and 13.2% of children living in the city orphan asylum were insufficient in the serum total protein value. The average serum cholesterol level of children living at home in the rural area was higher than that of children living in the city orphan asylum(p<0.01), and that of female living at home was higher than that of male (p<0.05). The mean levels of serum iron, total iron binding capacity and transferrin saturation of children living at home in the rural area were significantly higher than those of children living in the city orphan asylum (p<0.01).

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THE ORPHAN STRUCTURE OF BCH(3, m) CODE

  • HWANG, GEUM-SUG
    • Journal of the Korean Society for Industrial and Applied Mathematics
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    • v.6 no.1
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    • pp.109-119
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    • 2002
  • If C is a code, an orphan is a coset without any parent. We investigate the structure of orphans of the code BCH(3, m). All weight 5 cosets and all weight 3 reduced cosets are orphans, and all weight 1,2 and 4 are not orphans. We conjecture that all weight 3 unreduced cosets are not orphans. We prove this conjecture for m = 4, 5.

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A Study on the Depression of Adolescence - A Comparative Study of Normal, Orphan, Physically Handicapped and Deaf - Mute Children - (사춘기 청소년의 우울병에 관한 연구 - 정상아.고아.지체부자유아 및 농아아의 비교연구-)

  • 오경옥
    • Journal of Korean Academy of Nursing
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    • v.7 no.2
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    • pp.117-127
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    • 1977
  • Depressive status of the adolescence in 437 normal, 416 orphan, 133 physically handicapped and 109 deaf-mute children were compared with the Self- Rating Depression Scale developed by Zung, from April 26 to July 4, 1976. The subjects were divided into two groups, early adolescence for age of 10 to 14 and ate adolescence of age of 15 to 18. The results were as follows : 1. The depression score was higher in late adolesce co than in early adolescence except deaf - mutes, 2. The depression score was not different significantly between the sexes in all groups. 3. The depression score was significantly higher in the group of orphan, physically handicapped and deaf - mute children then in the normal. 4. The depression score was significantly higher in the orphans than in the physically handicapped children. It showed tendency to be higher in the deaf - mutes than in the physically handicapped children. 5. In comparison with the rank order of the Self - Rating Depression Scale items, normal group was inclined to have psychological symptoms in higher rank order, but orphan and deaf - mute group was inclined to have somatic symptoms in higher rank order.

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