The goals of drug development process involves supporting all stakeholders in improving patient care, addressing challenges associated with financial sustainability and facilitating implementation of innovative medicines.
Oncology Drug Development
The pharmaceutical industry continues to invest towards new advances in cancer therapy such as increasing availability of immunotherapy, enhancing focus on personalised treatment and emerging technologies of gene editing. The standards for assessing safety and efficacy in randomised controlled trials (RCT) are important in development of cancer drugs. Cost, patient recruitment difficulties and generalisability are recognised limitations of randomised controlled trials (RCTs). The potential of developing successful anticancer drugs is hampered by the elevated expenditure and time taken in traditional regulatory approval pathway. Pragmatic trials are designed to assess comparative effectiveness and evaluate the interventions in real life, whereas confirmatory trials test interventions under optimal conditions. Prospectively designed randomised pragmatic trial with regulatory compliance can provide sufficient evidence for verifying clinical benefit and ensuring approval.
The need for rethinking traditional approaches for drug development approval includes use of real world data. There has been a dramatic increase in usage of observational data for rapid, informed and widespread accessibility or availability of important anticancer medicines. Real-world evidence (RWE) can be useful during all stages of drug development lifecycle from discovery to management of a licensed product. Thoughtful and balanced fashion of accounting real-world evidence in regulatory and health technology assessment can justify and enrich sound decision-making.
Real World Evidence
Real world or Observational data do not arise from experimental or interventional studies or result of clinical trials. This data is collected retrospectively or prospectively from routine clinical practice. Real world evidence can be collected from different sources like patient and population surveys or databases, patient chart reviews, cohort studies and health registries. RWE uses real world data (RWD) to generate foresight, insight and explorative findings on diseases, patients and products. The interest to include real world data is overwhelming as routinely collected data increases external validity of randomised clinical trials on anticancer drugs and generates evidence on factors determining treatment effects in the real world pertaining to pharmaceutical policies, health systems, patient preferences or doctor patient relationship. This data complements information derived from randomised clinical trials; supplement RCT data beyond the duration of follow-up; helps in decision-making and determining the value of treatment to reflect a variety of outcomes in improving quality of life, overall survival or adverse effects of treatment; captures rare events, long-term outcomes and supports biomarker validation; provides valuable clinical insights; improves information supplied for health technology assessments while maintaining patient confidentiality.
The advantages of data collection are numerous in cancer drug development which includes reduction of timelines, costs and number of patients in randomised trials along with confirming results of such trials. The number of patients available for proper assessment of benefit-risk in clinical trials may be too small. A potential solution to complement results from randomised trials with real-world data factored can help weigh the ultimate benefit-risk of anticancer products. It can provide useful information on defining standard of care, burden of illness, disease progression and resource utilisation. RWE can guide conditional reimbursement depending on feasibility, clinical uncertainty and transaction overheads. Observational studies can fill critical gaps with regard to health technology assessment. In spite of advanced strategies to adjust various biases and confounding factors, translation into real-world evidence is a critical challenge. The translation process depends on the kind of products, the treatment effects of clinical development and available alternative treatment approaches. Handling challenges of RWE is necessary before integrating into drug development process.
Scope and Advances
New advances in digital technology can facilitate collection and analysis of real-world data benefiting patients and healthcare systems. The increasing access to electronic health records (EHRs) and administrative databases provides opportunities to conduct observational studies in collecting data, serve to help regulators define the benefit-risk balance of new drugs and support assessment of added value outside an experimental situation by health technology assessment (HTA) bodies. The difficulty in obtaining good quality data can be sorted by linking registries protected by law that contain data of cancer patients for cross-border analysis and other research purposes. Data collection and analysing systems must function with a decentralised infrastructure.
New pathways and several initiatives are developed for better cancer therapy like scientific concept of adaptive pathway approach for drug development and data generation. This implies iterative development with restricted patient population followed by evidence gathering and progressively expanding to a wider population. The early involvement of patients along with real-life gathered evidence supplements clinical trial data and HTA bodies in product development. RWE is anticipated to be more important than licensing and market authorisation (MA). Developing a framework for harmonized RWD can assist healthcare providers and improve clinical, economic and humanistic outcome. Increased awareness and education of RWE can help overcome various challenges and barriers.