Toward Fit-for-Purpose Data for Drug Assessment in Non-small Cell Lung Cancer: A Core Dataset.

Considering the high unmet medical need in people with non-small cell lung cancer (NSCLC), the drug assessment for targeted therapies often rely on small populations and single-arm trials, challenging the evaluation by regulatory authorities, health technology assessment bodies (HTAb), and clinicians. Registry-based real-world data (RWD) can enhance the evaluation and appropriate use of these novel medicines. Therefore, we developed a core dataset that provides a starting point to complement the evaluation of NSCLC drug effects based on registry-based RWD. The core dataset was developed through a literature review of existing core datasets, followed by two multi-stakeholder expert meetings for better understanding of important variables for drug evaluation purposes by different stakeholders. The experts included Dutch clinicians, hospital pharmacists, regulators, HTAb, industry representatives, academics, and a patient representative. Following the literature review, four existing core datasets were identified and used to extract important variables. After two expert meetings, this core dataset was redefined and the final core dataset includes general patient characteristics (n = 6), disease-specific factors (n = 19), treatment details, including systemic therapy, surgery, and radiotherapy (n = 22), outcomes such as death, progression, response (n = 9), and patient-reported outcome measures (n = 17). This core dataset contains relevant variables that provides guidance for the collection of RWD to develop a relevant patient registry for NSCLC and to enhance its fitness-for-purpose. These RWD are intended to address most regulatory, HTA, and healthcare professionals' questions, thereby supporting the generation of real-world evidence for the more appropriate use of NSCLC drugs.