全民健康保險罕見疾病用藥探討

Orphan Pharmaceuticals for Rare Diseases under National Health Insurance

繼美國、日本、澳洲、及歐盟之後，台灣是世界上第五個制訂罕見疾病及藥品相關法案的國家。行政院衛生署自91年八月底起，更明訂所公告之罕病，全數獲全民健康保險重大傷病保障，堪稱世界創舉。本研究藉由分析全民健康保險研究資料庫，探討國內罕病在93年之健保使用情形及處方狀態。經由整理研究期間衛生署所核可之罕病國際疾病分類號共96碼，及健保局公告且適用罕見疾病防治及藥物法之65項藥品代碼做為資料擷取條件，向國家衛生研究院提出特殊需求檔案申請，再以SAS程式統計分析門診及住院實務狀況。研究結果顯示，使用孤兒藥品且符合罕病診斷條件者，計門診763件、463人，住院811件、179人，平均年齡約22歲，而10歲以下患者約佔四分之一（門25.9%、住26.8%）。依就醫資料推估，僅極少數具罕病診斷患者有孤兒藥品治療（門3.6%、住0.9%）。最多的就診科別為小兒科（佔門53.6%、住61.8%），其次神經科（佔門23.5%、住19.4%）；最常見診斷是thalassemia major（佔門23.9%、住24.0%）；最常被開立藥品是deferiprone（門176件、住173件）。罕病門、住處方紀錄中之藥品品項數共有31項，其中同時於門診及住院治療中被使用者有20項。使用孤兒藥之罕病患者的就醫地理分佈，傾向集中於都區之醫學中心（佔門88.2%、住83.4%）；有98.5%門診罕病處方於醫療院所藥局自行調劑，過半(54.0%)之給藥日份為28日，而慢性病連續處方佔門診全部處方之12.3%。透過本罕病醫療利用研究，益發彰顯對新藥研發、小兒特殊製劑、醫療服務鼓勵政策等之迫切性及依賴性，亦期待未來能推展社區罕見疾病照護體系，及更多相關改進措施或研究，以達到提供罕病患者更周全醫療服務的目標。

Taiwan, along with the US, Japan, Australia, and European Union countries, lead the legislations on rare diseases and orphan drugs in the world. By Aug. 2002, all of the rare diseases declared by the Taiwan Department of Health have further been included in the Catastrophic Illness Registration whose medical expenses are shielded by the National Health Insurance (NHI). The aim of the study was to investigate the actual NHI utilization by victims of rare diseases and to examine orphan drug prescriptions by utilizing the 2004 National Health Insurance Research Database. The officially enlisted rare diseases (96 ICD-9-CMs) and orphan drugs (65 pharmaceuticals) during the study period were key identifiers for data extraction. A statistical SAS program was used to analyze both ambulatory and inpatient records. Our data revealed that there were 763 claims/463 patients and 811 claims/179 patients, with a mean age of 22, for ambulatory and inpatient cares, respectively. A quarter of the patients were younger than 10 year-old (ambulatory, 25.9%; inpatient, 26.8%). Extremely few rare disease sufferers were prescribed with orphan drugs (ambulatory, 3.6%; inpatient, 0.9%). The most frequently seen specialty was pediatrics (ambulatory, 53.6%; inpatient, 61.8%), followed by neurology (ambulatory, 23.5%; inpatient, 19.4%). The largely encountered diagnosis was thalassemia major (ambulatory, 23.9%; inpatient, 24.0%); mostly prescribed drug was deferiprone (ambulatory, 176 claims; inpatient, 173 claims). There were a total of 31 drug items identified in the ambulatory or inpatient prescription records, with 20 items seen in both settings. A scrutiny on the geographic distributions revealed that medical care providers for rare diseases were virtually medical centers (ambulatory, 88.2%; inpatient, 83.4%) located at metropolitan areas. Ambulatory orphan drug prescriptions dispensed at the in-house pharmacies of medical institutions constituted 98.5%; more than half were 28-day supply for each dispensing. Only 12.3% belonged to chronic-illness refill prescriptions. The study strengthens the urgency and need for orphan drug discovery and development, special formulations for pediatric population, and incentives for orphan drug industries and medical services. The implementation of long-term care systems and related improvements or researches will facilitate the goal of providing rare disease sufferers with sound medical cares in the future.