중증질환 치료에 있어 효과 불확실성에 대한 선호와 희망의 가치
Risk preference and the value of hope in the treatment of severe illnesses
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2018년 ISPOR 특별 태스크 포스는 미국 의료기술 가치 평가에서 고려할 수 있는 여러 가치 요소를 새롭게 제안한 바 있다. 그중 하나가 ‘희망의 가치’인데, 이는 중증질환 치료에 있어 조기 사망과 장기 생존 가능성을 모두 내포하는 효과 편차가 크고 불확실한 치료제에 대한 환자의 잠재적 선호를 지칭한다. 희망의 가치를 새로운 가치 요소로 주장하는 논자들은 말기 암 선고와 같은 절망적 상황에서 개인은 ‘운 좋은 소수가 되길 희망하며’ 장기 생존 가능성을 위해 생명 단축의 위험을 기꺼이 감수할 의향이 있다고 본다. 본 연구에서는 ‘희망의 가치’에 대한 이론적 타당성과 실증 근거를 비판적으로 검토하고, 이를 통해 이론적·방법론적 보완점을 제시하고자 한다. 체계적 문헌 검색 결과 총 5개의 실증 연구를 확인하였는데, 모두 미국에서 수행되었다. 이들은 전이암 등 중증질환 치료에 있어 효과가 작지만 확실한 치료제와 불확실한 치료제 간에 선호를 측정하였다. 조사 대상자들은 나름 일관되게 효과가 불확실한 치료제를 선호하였으나, 효과 분포나 확률도 치료제 선호에 일부 영향을 미치는 것으로 나타났다. 하지만 이에 대한 이론적 검토나 구체적 평가 방안에 대한 논의는 매우 제한적이었다. 특히, 효과 분포의 오른쪽 기울어짐(positive skewness)에 대한 선호를 위험 추구적 선호로만 설명하기 어렵다는 점, 효과 불확실성에 대한 잠재적 선호는 일관성을 유지하기 어렵다는 점, ‘희망의 가치’가 ‘희망’의 가치를 포괄적으로 반영하지 못한다는 점, 치료 전(ex-ante) 선호는 치료 후(ex-post) 선호와 다를 수 있다는 점, 다른 가치 요소와의 중복 가능성 등에 대한 이슈들이 제대로 다루어지지 않았다. 중증질환 치료에 있어 희망은 환자의 예후와 삶의 질에 큰 영향을 미치는 매우 중요한 요소이다. 하지만 어떠한 새로운 요소를 정책 의사결정 과정에서 고려하려면 강건한 이론적·실증적 근거가 뒷받침되어야 하므로 이에 관한 추가 연구가 먼저 수행되어야 한다.
Abstract
In 2018, the International Society for Pharmacoeconomics and Outcomes Research Special Task Force proposed new value elements, including the ‘value of hope’, to be considered in the United States value framework for Health Technology Assessment. The value of hope refers to potential patient preferences for treatments with a wider outcome distribution, offering the possibility of long-term survival in severe illness treatment (i.e., a long survival tail). The introduction of this element acknowledges that individuals facing dire circumstances, such as terminal cancer diagnoses, may willingly accept the risk of shortened life expectancy in the hope of becoming one of the few fortunate who achieve long-term survival. This study critically evaluates the theoretical validity and empirical evidence of the value of hope. A systematic literature search identified five empirical studies, all conducted in the US, assessing preferences between treatments with certain but limited effects and treatments with uncertain effects, especially for severe illnesses like advanced cancers. The findings revealed that respondents tended to prefer uncertain treatments when the two options offered equal or constant survivals, but preferences were also partly influenced by other factors such as survival skewness and probabilities. However, several theoretical and methodological gaps require further examination. Preferences for positive skewness may not solely reflect risk-seeking preferences, which could thereby lead to inconsistency in their direction, unlike average costs and effects. Furthermore, the ‘value of hope’ concept might not fully capture the essence of ‘hope’, and preferences before experiencing the actual outcomes (ex-ante) may differ from those after experiencing the outcomes (ex-post). There is also potential overlap with other value elements, such as the real option value. While hope plays a critical role in the treatment of severe illnesses, significantly influencing patients' prognosis and overall quality of life, further theoretical and empirical evidence is required before considering the ‘value of hope’ as an additional value element in policy decision-making.
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