Real-World Evidence is Needed to Complement Clinical Trial Data for the Development of a Product’s Proposition: A Case Study on Real-World Evidence on Pembrolizumab Plus Pemetrexed and Platinum for Advanced NSCLC

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Randomized clinical trials (RCTs) remain the gold standard of scientific data for demonstrating a treatment’s safety and efficacy, yet there is a need for real-world evidence (RWE) to address healthcare policy and economic concerns regarding a treatment’s effectiveness. A limitation of oncology clinical trials is their concentration on treatments within a particular line of therapy. According to the results of the KN-189 RCT, pembrolizumab in combination with pemetrexed and platinum chemotherapy (pembro+pem+plat) was approved by the FDA for the front-line treatment of patients with metastatic non-squamous non-small cell lung cancer (NSCLC) and is currently recommended and preferred as a first-line treatment for patients with advanced NSCLC. The objective of this capstone is a) to demonstrate the importance of RWE data and its ability to compliment RCT data via a case study on pembro+pem+plat about treatment patterns, maintenance utilization, and survival outcomes, and b) to identify RWE gaps to support the value-proposition strategy for pembrolizumab in first-line lung cancer. A literature search identified current pattern of care and patient survival in first-line lung cancer patients that received maintenance treatment (MT) versus no MT. RWE data demonstrate that the median survival in patients who received pembro+pem+plat maintenance was comparable to the median observed in the KN-189 RCT. Also, the rate of MT was lower than the MT rate observed in RCT (44% vs 76-79%). MT was associated with significant reduced risk of death versus no MT. The authors identified four additional RWE gaps to support the use of pembro+pem+plat in first-line NSCLC patients: quality of life data, cost and healthcare resource utilization, cost of adverse events, and an indirect-treatment-comparison. Results of this analysis give context to how regulatory approvals and treatment guidelines for advanced NSCLC are applied in clinical practice. They could also be utilized to inform decision-making amongst physicians and payers.



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