RWD 기반 의약품 성과 평가
- Type
- Research report
- Issued Date
- 2024-01
- Publisher
- 건강보험심사평가원
- Table Of Contents
- 제1장 서론 ···································································· 1
1. 사업 배경 ······························································································ 3
가. 실제 임상자료(Real World Data, RWD)의 의의 ·········································· 3
나. 전향적 RWD 수집의 필요성 ········································································ 4
2. 사업 목적 ······························································································ 7
제2장 연구개요 및 계획 ···································································· 9
1. 대상 약제 및 참여 연구자 선정 ····························································· 11
가. 대상 약제 선정 ························································································ 11
나. 참여 연구진의 구성 ·················································································· 14
2. 사업의 전체적인 개요 및 계획 ······························································· 17
가. 개요 ········································································································ 17
나. 연구대상자 ······························································································· 18
다. 전향적모집군의 환자증례기록지(Case Report Form, CRF) 개발 및 보완 ······ 20
라. 후향적대조군의 환자증례기록지(Case Report Form, CRF) 개발 및 보완 ······ 23
마. 환자보고성과(PRO) 및 경제성평가를 위한 수집항목, 지표 ····························· 24
바. 자료의 분석과 해석 방법 ··········································································· 26
사. 자료분석 결과에 대한 재현성 확인 ····························································· 31
아. 경제성평가 ······························································································· 31
자. 대상자의 연구 탈락 및 제외 기준 ······························································ 34
차. 부작용을 포함한 안전성의 평가기준, 평가방법 ············································· 35
3. 전산시스템의 사용 ················································································ 39
가. 임상정보 입력 시스템(Electronic Data Capture, EDC) 구축 ······················ 39
나. 환자보고성과(PRO)의 조사 및 자료 수집 ···················································· 43
4. 기타 사항 ···························································································· 44
가. 환자증례기록지(Case Report Form, CRF) ················································ 44
나. 자료의 질 보장 및 자료보안 ······································································ 44
다. 연구윤리 ·································································································· 48
라. 보안관리 계획 ·························································································· 50
제3장 연구 수행 내용 ···································································· 53
1. 참여 기관 및 IRB 승인 ········································································ 55
가. 참여 기관 ································································································ 55
나. 연구 심의 및 등록 ···················································································· 56
2. 대상자 등록 현황 ················································································· 68
3. 자료의 질 관리 방법 및 결과 ································································ 70
가. 참여기관 조사자간 일치도 및 신뢰도 점검 ·················································· 70
나. 수집된 데이터 관리 ·················································································· 74
4. e-CRF 구축 및 조사지침서 구축 및 보완 ·············································· 77
5. RWD 기반 외부대조군을 이용한 단일군 연구 수행 가이드라인 ················· 81
가. RWD 기반 외부대조군을 이용한 단일군 연구 ············································· 82
나. RWD 기반 외부대조군을 이용한 단일군 연구의 고려사항 ····························· 94
다. RWD 기반 외부대조군을 이용한 단일군 연구 프로토콜 사례 ······················ 100
6. RWD-PRO 설문지 사이트 개발 및 유지 ············································· 110
가. 개발 개요 ······························································································ 110
나. 설문시스템 화면설계 / 구현 ···································································· 112
7. 암환자 재정영향 설문조사 고찰 현황 ···················································· 154
가. 암환자 재정영향 설문연구 문헌고찰 실시 ·················································· 154
나. 암환자 재정부담 설문지 검토 ··································································· 155
다. 설문조사 개발시 고려사항 ········································································ 164
8. 경제성평가 모형 구축 및 분석 계획 ····················································· 164
가. 경제성평가 모형 구축을 위한 문헌고찰 ····················································· 164
제4장 연구 결과 ···································································· 171
1. 자료의 질 관리 결과 ··········································································· 173
가. 참여기관 조사자간 일치도 평가 ································································ 173
나. 수집된 기록의 신뢰도 점검 ······································································ 177
다. 참여기관 조사자 교육 ·············································································· 179
2. 전향적모집군과 후향적대조군 분석 ························································ 180
가. 현황 ······································································································ 180
나. 전향적모집군과 후향적대조군의 기본특성 ··················································· 181
라. 전향적모집군과 후향적대조군의 최초 암 진단시 특성 ·································· 184
마. 전향적모집군과 후향적대조군의 과거 암 치료 이력 ····································· 188
바. 전향적모집군의 기저치(baseline) 검사 소견 ·············································· 189
사. 전향적모집군의 ramucirumab과 paclitaxel 병용요법 현황 ························· 191
아. 전향적모집군에서 ramucirumab과 paclitaxel 병용요법의 안전성 ················ 199
자. 전향적모집군에서 ramucirumab과 paclitaxel 병용요법의 반응률 ················ 203
차. 전향적모집군 및 후향적대조군에서 치료 지속 여부 및 중단 사유 ················· 205
카. 전향적모집군과 후향적대조군 성향점수 매칭 전후 기본 특성 ······················· 207
타. 전향적모집군과 후향적대조군 성향점수 매칭 전후 생존 분석 ······················· 212
파. 전향적모집군과 후향적대조군에서 OS, PFS 하위그룹 분석 ························· 226
3. 후향적모집군(K-RWE)과 후향적 대조군 분석 ········································ 239
가. 개요 및 현황 ·························································································· 239
나. 후향적모집군과 후향적대조군의 기본특성 ··················································· 240
다. 후향적모집군과 후향적대조군의 최초 암 진단시 특성 ·································· 242
라. 후향적모집군과 후향적대조군의 과거 암 치료 이력 ····································· 245
마. 후향적모집군의 기저치(baseline) 검사 소견 ·············································· 246
바. 후향적모집군에서 ramucirumab과 paclitaxel 병용요법의 안전성 ················ 248
사. 후향적모집군에서 ramucirumab과 paclitaxel 병용요법의 반응률 ················ 250
아. 후향적모집군 및 후향적대조군에서 치료 지속 여부 및 중단 사유 ················· 251
자. 후향적모집군과 후향적대조군 성향점수 매칭 전후 기본 특성 ······················· 251
차. 후향적모집군과 후향적대조군 간 생존분석 ················································· 256
카. 후향적모집군 및 후향적대조군에서 OS, PFS 하위그룹 분석 ······················· 262
타. 후향적모집군과 후향적대조군의 자료원 비교를 위한 성향점수 매칭 ·············· 268
파. 후향적모집군과 후향적대조군의 자료원에 따른 생존 분석 ···························· 272
거. 후향적모집군 및 후향적대조군에서 자료원에 따른 OS, PFS 하위그룹 분석 ·· 279
4. 전향적모집군과 RAINBOW시험군 분석 ················································ 283
가. 전향적모집군과 RAINBOW시험군의 설계 ················································· 283
나. 전향적모집군과 RAINBOW시험군의 공변량 특성 ······································· 285
다. 전향적모집군과 RAINBOW시험군의 MAIC 매칭 ······································· 288
라. 전향적모집군과 RAINBOW시험군의 MAIC 매칭 전후 특성 ························ 289
마. 전향적모집군과 RAINBOW시험군의 MAIC 매칭 후 생존 분석 ··················· 290
5. 전향적 RWD에서 핵심적 수집항목 ······················································· 295
6. 환자보고성과(PRO) 조사 결과: 삶의 질, 재정독성 ································· 297
가. 건강관련 효용조사 ·················································································· 297
나. 재정독성 및 비급여 조사 ········································································· 305
7. 경제성평가 모형 구축 및 분석 관련 선행연구 고찰 결과 ························ 317
가. 경제성평가 모형 구축 ·············································································· 317
나. Partitioned Survival Analysis를 위한 생존 곡선들 (OS와 PFS)과 모수추정 ·· 318
다. 경제성평가 결과 ····················································································· 322
제5장 고찰 및 제언 ···································································· 325
1. 전향적 조사의 장단점 ········································································· 327
2. 후향적 조사의 장단점 ········································································· 328
3. 참여 기관의 IRB 진행과 동의 취득 ······················································ 328
4. 환자보고성과(PRO) 조사 결과 고찰 ······················································ 328
5. 경제성평가 결과 고찰 ········································································· 330
6. 결과 분석 과정에서의 고찰 ·································································· 331
7. 사망일자 출처에 따른 분석 결과 고찰 ·················································· 332
8. 결론 ·································································································· 333
◾ 참고 문헌 ····················································································· 337
◾ 부록 ····························································································· 345
- Publisher
- 건강보험심사평가원
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- KOR
- Citation
- 장대영. (2024-01). RWD 기반 의약품 성과 평가.
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