HIRA

의약품 급여관리를 위한 실제임상근거(RWE) 플랫폼 마련 후향적 연구(청구자료 분석)

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Type
Research report
Author(s)
김동숙변지혜김지혜김수연이은지김소희김설희이혜영조도연정숙지이정은신주영안형진이대호
Issued Date
2020-10
Keyword
real-world data(RWD)real-world evidence(RWE)HTAHIRA
Abstract
Background
It is more difficult to conduct randomized controlled trials (RCT) in R&D of pharmaceuticals for treating cancer or rare disease than other diseases, therefore leading to increasing the uncertainty in pricing and reimbursement. To resolve these uncertainties of decision-making, health technology assessment (HTA) agencies in many countries including Korea currently explore possibilities for using the real-world evidence (RWE).

Objectives
This study aims to generate the Korea real-world evidence (K-RWE) drawn from the K-RWD (Korea real-world data). Therefore based on the results, this study aims to suggest model for constructing the infrastructure of collecting K-RWD and the possibility using K-RWE for decision-making in pharmaceutical policy (i.e. HTA reassessment).

Methods
We implemented this study to compare effectiveness at the time of drug approval time and after the appproval for two selected anti-cancer medicines (ramucirumab for gastric cancer and trastuzumab emtansine for breast cancer). First, we reviewed the
quality level on K-RWD available such as HIRA claims data, electronic medical records(EMR). Second, we decided the necessary information for pharmaceutical health technology assessment (HTA). Second, we decided the necessary information for pharmaceutical health technology assessement (HTA). Third, we made case report
form (CRF) for two selected medicines each. Fourth, we collected K-RWD through retrospective studies on the effectiveness and safety of two selected medicines. Fifth, we analyzed the effectiveness and safety of two selected anti-cancer medicines using K-RWD (HIRA claims data and EMR in 68 hospitals). Lastly, we explored further steps for robust RWE generation and constructing infrastructure of collecting K-RWD.

Results
HIRA claims data covered almost all Korean people because Korea achieved universal health coverage (UHC), but there is still lack of information such as disease stage, progress of disease, and so on.We surveyed on the quality of EMR data, and 55 of 68
hospitals participated. It displayed the coding rate of RECIST (44.4% of tertiary hospitals vs 73.7% of secondary hospitals 73.7%) and the NCI-CTCAE (58.3% of tertiary hospitals vs 73.7% of secondary hospitals). It needs coding on the ECOG, diagnosis date, the results of RECIST, disease progression, and death date. The difference for review period on IRB among participated hospitals were shown. We confirmed the consistence (gastric cancer team: 73.5±21.3%, breast cancer team: 71.9±16.9%) and confidence (0.89±0.15, 0.86±0.17 respectively) on major contents in CRF among researchers. The missing rate highly showed difference among hospitals. This study compared the results between RCT and RWE. Overall survival (OS) period on ramucirumab showed 9.6 months (95% CI 8.5-10.8) from RCT (n=330), and 10.0 months (95% CI 9.3-10.7) from EMR (n=1,063), and 7.9 months (95% CI 7.6-8.6)
from HIRA claims data (n=1,419). Progression free survival (PFS) period on trastuzumab emtansine respectively displayed 9.6 months from EMILIA study (n=495), and 6.7 months (95% CI 6.2-7.4) from total patients in K-RWE (n=824). PFS in severe patients were 8.0 months (95% CI 6.8-9.4) from 2nd line use in K-RWE (n=329), 6.2 months from TH3RESA study (n=404, 95% CI 5.6-6.9). Shortly, the results from HIRA data were the lowest in gastric cancer and in breast cancer. However, these results required careful interpretation. HIRA data has not clinical information of patients and EMR data did not know whether patients die in others hospitals or nursing homes. Finally, this study found that K-RWD could be better planning to collect data in advance, developing common code on the major contents in individual hospital to integrate each information, and processing common IRB committee on government research.

Discussion
This study confirmed the potential power on K-RWD even though some limitation. K-RWD will be powerful data when these limitations should be resolved and other data could be linked. HIRA also tried to collect the patient reported outcomes (utility, financial burden and improving the subjective symptoms), clinical and safety information. Ultimately, we try to the integrated RWD dataset per individual patient through the linkage between the claims data and EMR. Therefore, it is necessary for prospective collecting K-RWD to know the baseline information of patients for assessment on intervention.
Publisher
건강보험심사평가원
URI
https://repository.hira.or.kr/handle/2019.oak/3035
Alternative Title
A Retrospective Case Study through Real-World Evidence (RWE) for Health Technology Assessment (HTA) in Pharmaceuticals
Table Of Contents
요약
제1장 서론 ----------------------------- 37
1. 연구 배경 ----------------------------- 37
2. 연구 목적 ----------------------------- 41
3. 연구 내용 ----------------------------- 42

제2장 의약품 급여관리 관련 제도 고찰 ----------------------------- 45
1. 국내 의약품 급여관리 제도 개요 ----------------------------- 45
2. 외국의 의약품 급여관리 제도 현황 ----------------------------- 51
3. 국내·외 실제임상자료(RWD) 활용 사례 ----------------------------- 107
4. 국내 보건의료 데이터 현황 ----------------------------- 155

제3장 RWE 활용 의약품 재평가 대상 우선순위 선정 ----------------------------- 183
1. 근거가 불확실한 급여 결정 제도 ----------------------------- 183
2. 급여 당시 근거가 불확실한 의약품 ----------------------------- 191
3. 실제임상근거(RWE) 기반 평가 우선 순위 의약품의 선정 ----------------------------- 212

제4장 대상 약제별 문헌기반 평가 ----------------------------- 215
1. 위암 ----------------------------- 215
2. 유방암 ----------------------------- 226
3. 대상 약제별 체계적 문헌고찰 결과 ----------------------------- 241

제5장 국내 위암 및 유방암의 발생 현황 ----------------------------- 319
1. 연구내용 및 방법 ----------------------------- 319
2. 암환자 의료이용 현황 ----------------------------- 324
3. 암환자 의약품 사용 현황 ----------------------------- 340
4. 위암/유방암 발생률 및 유병률 ----------------------------- 353

제6장 건강보험 청구자료를 활용한 성과연구 ----------------------------- 363
1. 연구내용 및 방법 ----------------------------- 363
2. 대상환자의 일반적 특성 ----------------------------- 371
3. 대상환자 진료비 ----------------------------- 439
4. 성과분석 ----------------------------- 449

제7장 고찰 및 결론 ----------------------------- 475
1. 연구고찰 ----------------------------- 475
2. 의약품의 실제임상자료(RWD) 수집 개선 방안 ----------------------------- 484
3. 의약품의 실제임상자료(RWD)/실제임상근거(RWE) 활용 가치 ----------------------------- 488

참고문헌 ----------------------------- 490

ABSTRACT ----------------------------- 511
부 록 ----------------------------- 515
Publisher
건강보험심사평가원
Location
KOR
Citation
김동숙 et al. (202010). 의약품 급여관리를 위한 실제임상근거(RWE) 플랫폼 마련 후향적 연구(청구자료 분석).
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