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.
AimThis core dataset contains relevant variables that provides guidance for the collection of real-world data (RWD) to develop a relevant non-small cell lung cancer (NSCLC) patient registry and to enhance its fitness-for-purpose data. These RWD are intended to address most regulatory, health technology assessment (HTA), and healthcare questions, thereby supporting the generation of real-world evidence for the more appropriate use of NSCLC drugs.
Potentially relevant COS
Existing COS intended to assess the value of lung cancer drugs to support the structured collection of these data in clinical practice were searched in the COMET database. No COS were found that fully met our scope, nevertheless, two relevant lung cancer COS were included in our study to serve as the foundation of our core dataset. The aim of our core dataset differs from these two COS, since they focus on value-based healthcare across the entire treatment pathway (e.g. diagnostics, time to first treatment) for lung cancer patients, while we focus on drug assessment in NSCLC using registry based RWD.
1) K.S. Mak, et al. Defining a standard set of patient-centred outcomes for lung cancer, European Respiratory Journal 48 (2016) 852–860.
2) B.H. de Rooij, et al. Development of an updated, standardized, patient-centered outcome set for lung cancer, Lung Cancer 173 (2022) 5–13.
Geeske F Grit, Maaike van Dartel, Laura Rodwell, Hanneke van der Woude, Cornelis Boersma, Peter GM Mol, Doranne Hilarius
Disease Category: Cancer
Disease Name: Non-small-cell lung cancer (NSCLC)
Age Range: 18 - 120
Sex: Either
Nature of Intervention: Drug
- Clinical experts
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Policy makers
- Regulatory agency representatives
- Researchers
- COS for registry
- Literature review
- Semi structured discussion
The core dataset was developed through a literature review of existing core datasets, followed by two multi-stakeholder expert meetings to provide input for better understanding of important variables.