희귀난치성 질환자의 의약품 접근성 제고 방안 = Policy measures to enhance access to drugs for rare diseases

희귀난치성 질환자의 의약품 접근성 제고 방안 = Policy measures to enhance access to drugs for rare diseases


희귀난치성질환 ; 의약품
□ 희귀난치성 질환자의 의약품 접근성 현황과 문제점
□ 희귀난치성질환 치료제의 접근성 향상을 위한 외국의 제도 현황
□ 희귀난치성질환 치료제의 공급 안정과 접근성 향상 방안

This study explored the concepts and issues surrounding rare disease and orphan medicines from the perspective of resource allocation in health care, and suggests policy measures to rationally ensure access to and availability of orphan medicines.
Advances in health technology have led to enhanced diagnosis of rare diseases and increasing orphan drug development. Most orphan drugs are expensive on a per patient basis and so increase economic burden to third-party payers as well as individual patients. Given the limited health care resources and budget, there have been increasing concerns with regard to reimbursement and pricing of orphan drugs in health care system.
Orphan drugs have several distinct characteristics that may distinguish them from other new drugs, including life-threatening condition, high cost and lack of evidence. However, most of those issues are not unique to orphan drugs but have been reviewed and discussed in health care priority setting. Therefore, when addressing reimbursement issues in National Health Insurance, which is intended for the whole population, orphan drugs should be reviewed and evaluated under the same principle that are applied to other new drugs. When it comes to whether a drug should be funded, the severity of the disease the drug aims to treat and its expected benefit should come before the 'rarity of the disease'.
The small number of patients with rare diseases makes it difficult for pharmaceutical companies to conduct large randomized controlled trials. Lack of evidence on the clinical effectiveness of orphan drugs and their very high price make decisions on pricing even more difficult in health insurance system. To address the financial risk in pricing orphan drugs with limited evidence, third-party payers need various strategies for price negotiation and risk-sharing agreement with drug companies. Evidence development in real world as a condition of coverage could be considered to promote evidence-based reimbursement of orphan drugs which is expected to grow rapidly in pharmaceutical market.
For patients who need expensive and life saving orphan drugs rejected for reimbursement in treating their serious rare conditions, a separate funding mechanism outside the National Health Insurance will be helpful.
제1장 서 론
제1절 연구의 배경과 필요성
제2절 연구 목적
제3절 연구 내용 및 방법

제2장 희귀질환 및 희귀의약품에 관한 이론적 고찰
제1절 희귀질환 및 희귀의약품의 일반적 고찰
제2절 희귀질환의 의료보장과 자원배분에 관한 이론적 고찰

제3장 국내 건강보장체계에서 희귀질환 치료제 관련 현황
제1절 건강보장체계에서 희귀질환자 건강보장의 원칙
제2절 희귀질환 치료제의 급여와 약가 결정 체계 및 운영
제3절 허가 또는 급여되지 않은 희귀질환 치료제의 접근성
제4절 희귀질환 치료제의 공급 관리 현황

제4장 희귀질환 치료제 관련 외국의 건강보장제도 현황
제1절 주요 쟁점별 제도 현황
제2절 프랑스의 희귀질환 치료제 보장 현황
제3절 네덜란드의 희귀질환 치료제 보장 현황

제4절 이태리의 희귀질환 치료제 보장 현황
제5절 영국의 희귀질환 치료제 보장 현황
제6절 미국의 희귀질환 치료제 보장 현황
제7절 캐나다의 희귀질환 치료제 보장 현황
제8절 호주의 희귀질환 치료제 보장 현황

제5장 희귀질환자의 합리적 의약품 접근성 제고를 위한 정책방안
제1절 희귀질환 치료제에 관한 보장 원칙
제2절 희귀질환 치료제에 관한 건강보장 정책방안

제6장 결론

보고서 번호
연구보고서 2010-02
KIHASA 주제 분류
보건의료 > 식품/의약품
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