Application of Health Economics and Outcomes Research (HEOR) Perspectives in a Clinical Case Study of Niemann-Pick Disease, a Rare Disease

OBJECTIVES: This case study aims to present a clinical scenario of a 1-year-old male patient with Niemann-Pick Disease (NPD) and explore the potential application of Health Economics and Outcomes Research (HEOR) perspectives to inform healthcare decisions. The objectives include assessing the economic impact, treatment outcomes, and overall value of interventions for Niemann-Pick Disease and highlighting the importance of considering the economic and clinical implications of healthcare decisions in the context of a rare disease.

METHODS: The methods involve the collection and analysis of clinical data, including the patient's medical history, symptoms, and diagnostic findings. The study also describes the interventions utilized, such as clobazam, Levetiracetam, platelet transfusion, and intravenous fluids, to address the patient's symptoms. While no original research or formal study design was implemented, the case study draws upon clinical expertise, medical records, and relevant literature to inform the presented findings and observations.

RESULTS: As no research was conducted in this case study, there are no specific findings or quantitative results to report. However, the study describes the successful management of seizure activity and symptomatic relief observed in the patient through the use of clobazam, Levetiracetam, and supportive care measures such as platelet transfusion and intravenous fluids.

CONCLUSIONS: This case study highlights the potential benefits of integrating Health Economics and Outcomes Research (HEOR) perspectives into clinical decision-making for rare diseases like Niemann-Pick Disease. It underscores the importance of considering both economic and clinical implications in healthcare decisions and emphasizes the need for early diagnosis, prompt access to appropriate treatments, and optimized resource allocation. While no research was performed in this particular case, the study encourages further exploration of HEOR approaches by stakeholders, including academic entities, government-affiliated organizations, payers, providers, and industry, to improve patient outcomes and enhance resource efficiency in the management of rare diseases.