Real-World Evidence Studies in Oncology Therapeutics: Hope or Hype?

Randomized controlled trial (RCT) remains a gold standard in evidence-based medicine for assessing the efficacy and safety of cancer therapies. However, due to some inherent methodological limitations of RCT, such as stringent inclusion criteria, highly specific treatment, ethical and scientific compromise in rare cancer, and inability to adequately assess safety, real-world evidence (RWE) has been adjudged as a suitable option to complement data obtained from RCT. Moreover, in the context of cancer therapeutics, few notable merits pertain to developing a novel product for rare cancer subtypes, establishing new indications for already approved drugs, optimization of treatment regimen and sequence, a better description of long-term safety, and supporting the reimbursement-related decision. However, the implementation of RWE for the aforementioned purposes will be limited by various challenges, especially in the context of developing economies such as India. Special attention should be given to the availability of data, maintaining the quality standard, and establishing stringent regulations for privacy and security along with active regulatory engagement with relevant stakeholders. Such activities will be key to facilitating the use of RWE in cancer therapeutics.