There is a substantial unmet need for treatments in the field of pediatric rare diseases, and investigator initiated trial(IIT) provide a critical pathway for testing and developing new drugs or treatment strategies. However, healthcare institutions, when conducting such research, must address compliance risks related to project approval, contract management, data protection, and conflict of interest management. This study aims to analyze the particularities and challenges of IIT in pediatric rare diseases, review relevant regulations and regulatory requirements, and provide healthcare institutions with a reference framework for compliance risk management to maximize the benefits of IIT. Based on literature review, analysis of laws and regulations, practical work experience, and frameworks from other institutions, we summarize the unique aspects of pediatric rare disease IIT in terms of participant characteristics, innovative technologies, and organizational structures.On this basis, targeted compliance management recommendations are proposed, which include establishing a risk rating and full-cycle risk monitoring mechanism, a consent and ethical review mechanism tailored to pediatric participants, a robust contract management mechanism, a comprehensive data security management mechanism, and a multidisciplinary team and multi-channel compensation mechanism. The study concludes that healthcare institutions, funders, and other collaborating entities should implement compliance management in line with the characteristics of IIT to ensure the safety and effectiveness of research and facilitate innovation and development in the treatment of pediatric rare diseases.

Objective To explore the role of serum N-terminal peptide of type Ⅲ procollagen(PⅢNP),insulin-like growth factor(IGF-1),and N-terminal pro-B-type natriuretic peptide(NTpro-BNP)in the disease evaluation and prognostic prediction in elderly patients with chronic left heart failure(CLHF).Methods A total of 92 elderly patients with CLHF who were admitted to Liyang People's Hospital from March 2020 to March 2023 were selected as research objects.There were 34 cases of Class Ⅱ,29 cases of Class Ⅲ,and 29 cases of Class Ⅳ according to the New York Heart Association(NYHA)functional classification.The patients were assignedto good prognosis group(n=62)and poor prognosis group(n=30).The general information and the levels of PⅢNP,IGF-1,and NTpro-BNP were compared between the two groups.Logistic regression analysis was used to identify the factors influencing the prognosis of elderly patients with CLHF,and a nomogram was constructed.Spearman correlation analysis was employed to examine the relationships between these indicators.Receiver operating characteristic(ROC)curves were constructed to analyze the predictive efficiency for the prognosis of elderly patients with CLHF.Results The diastolic pressure,systolic pressure,LVEDD,LVSDD,PⅢNP,and NTpro-BNP increased by the increase of NYHA classification,while the LVEF and IGF-1 decreased by the increase of NYHA classification(P<0.05).The levels of PⅢNP and NTpro-BNP in the good prognosis group were lower than those in the poor prognosis group,while IGF-1 in the good prognosis group was higher than that in the poor prognosis group(P<0.05).Logistic analysis indicated that PⅢNP,IGF-1,and NTpro-BNP were the factors affecting the prognosis of elderly patients with CLHF(P<0.05).Spearman correlation analysis revealed that PⅢNP and NTpro-BNP levels were positively correlated with NYHA functional classification,while IGF-1 was negatively correlated with NYHA functional classification(P<0.05).ROC curve results showed that the combined predictive value of PⅢNP,IGF-1,and NTpro-BNP was significantly superior to each parameter.Conclusion Serum levels of PⅢNP,IGF-1,and NTpro-BNP provide valuable assessment for the condition and prediction for prognosis of elderly CLHF patients.