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 in pediatric patients, particularly for rare diseases, emphasizing the need to alleviate physicians' concerns regarding prescribing practices [1][3][6]. Group 1: Physician Concerns - Physicians are often conservative in their prescribing practices, especially for vulnerable populations like children, due to ethical and legal risks associated with off-label drug use [2][3]. - A significant percentage of doctors in Turkey and the U.S. avoid high-risk treatments, with estimates suggesting that around 50% of Chinese doctors may do the same, particularly in pediatric cases [1][2]. Group 2: Off-Label Drug Use - Off-label drug use is defined as using medications beyond the indications approved by regulatory authorities, which can include different dosages, routes of administration, or patient populations [3][5]. - There are two main categories of off-label drug use in pediatric rare diseases: high-cost innovative drugs and older, less expensive medications that may still provide benefits for rare conditions [4][5]. Group 3: Regulatory and Policy Framework - Recent guidelines and expert consensus have been developed to standardize off-label drug use in pediatric rare diseases, although there are still gaps in the regulatory framework [5][6]. - The National Health Commission has reiterated support for necessary off-label drug use, but reimbursement for such uses remains a challenge, as they are not typically covered by basic medical insurance [6][7]. Group 4: Research and Evidence - The expansion of drug indications for pediatric use often relies on real-world evidence, which is difficult to accumulate if physicians are hesitant to prescribe [3][8]. - Clinical trials for pediatric drugs face ethical challenges, leading to a reliance on adult data for pediatric applications, which complicates the approval process for new indications [7][9]. Group 5: Future Directions - There is a need for more patient-centered clinical trials to enhance data collection for pediatric rare diseases, as current methods often yield insufficient participant numbers [9][10]. - The industry is exploring innovative approaches to gather real-world evidence to support the expansion of drug indications for children, particularly in the context of rare diseases [9].