儿童超适应证用药，医生为何顾虑重重？

Core Viewpoint - The article discusses the challenges and considerations surrounding off-label drug use, particularly in pediatric patients with rare diseases, emphasizing the need to alleviate physicians' concerns regarding prescriptions [1][3][7]. Group 1: Physician Concerns - Physicians are hesitant to prescribe off-label medications due to concerns about ethical and legal implications, especially when treating vulnerable populations like children [2][4]. - A significant percentage of doctors in Turkey and the U.S. avoid high-risk treatments, with estimates suggesting around 50% in China, particularly for pediatric cases [2][3]. - The lack of evidence-based data for pediatric medications exacerbates physicians' reluctance to prescribe off-label treatments [3][4]. Group 2: Regulatory and Policy Framework - The National Health Commission supports reasonable off-label drug use when no better treatment options exist, provided that informed consent is obtained from patients [3][7]. - Recent guidelines and expert consensus have been published to standardize off-label drug use for pediatric rare diseases, addressing the absence of established protocols [5][7]. - Despite supportive policies, off-label drug use remains outside the scope of basic medical insurance coverage, placing financial burdens on patients [8]. Group 3: Drug Development and Research - The development of pediatric medications is hindered by ethical constraints in clinical trials, leading to a reliance on adult data for pediatric applications [8][9]. - The process of expanding drug indications for children is complex, often requiring extensive real-world data to demonstrate safety and efficacy [9][10]. - The pharmaceutical industry shows limited motivation to pursue new indications for pediatric use, making investigator-initiated trials (IIT) crucial for supporting new indications [9][10].