Real World Evidence in Oncology
Written by Dr Niamh O’Reilly
The burden of cancer is increasing globally, making the search for new oncology therapies a major priority for the research community and a consistent area of investment for pharmaceutical companies. In 2018, over 138,000 people in Australia were diagnosed with cancer and over 48,000 people died from cancer (1). There were approximately 130 cancer treating medicines available on the Pharmaceutical Benefits Scheme (PBS) in 2018, of which 70 have been approved since 2013 (2). This is an indication of the accelerating pace of new drug discovery in oncology bringing new therapeutic options to healthcare professionals (HCPs) and patients. However, this deluge of new therapies can also leave HCPs, patients and payers with many questions around how these therapies perform in real world clinical practice.
The Evidence Gap
Randomised controlled trials (RCTs) are the gold standard for informing regulatory approval of new therapies and expanded indications for already approved therapies. However, they are costly and time intensive and since studies include a limited patient population who meet specific criteria there is often an evidence gap between clinical research data and real world clinical practice outcomes (3). This evidence gap coupled with the increasing need to provide timely access to new therapies, particularly in the oncology space, has led to the need for real world data (RWD). RWD is useful for payers as a supplement to existing RCT data to show the clinical and cost-effectiveness of therapies over an extended time frame.
RWD is data relating to patient health status and/or delivery of health care generated outside of conventional RCTs. This data can be derived from a variety of sources including patient registries, electronic health records (EHRs), insurance data, as well as mobile applications and devices (4). RWE is generated from these data sources and potentially used to inform regulatory and reimbursement decision making. RWE supplements RCT data by providing information on disease progression and overall survival over extended time periods (5). RWE is also useful where large RCTs are not feasible, such as assessment of treatment effectiveness in rare diseases, niche indications and patient subgroups (5).
Closing the Gap with RWE
Many countries have implemented programs for the collection and analysis of RWD via patient registries. Some have also developed strategies for use of RWE to capture information relevant for research and drug discovery, as well as regulatory and reimbursement decisions. For example, in America the FDA recently published a framework for its ‘RWE Program’ to inform the strategic use of RWE to enhance regulatory decision making (6). In England, the National Cancer Registration and Analysis Service (NCRAS) was launched in 2013, which provides comprehensive clinical information on all 350,000 people diagnosed with cancer in England each year, as well as 141 million historical cancer records (5).
RWE in Australia
In Australia, the Therapeutic Good Administration (TGA) has implemented an expedited ‘Provisional Approval pathway’ for the registration of new medicines in certain circumstances such as in the case of innovative cancer therapies (7). Providing timely access for patients to often highly expensive drugs also requires clinical and cost-effectiveness assessment by the Pharmaceutical Benefits Advisory Committee (PBAC) prior to reimbursement listing on the PBS. Although there is no formal framework for the use of RWE in Australia, there have been several instances where the PBAC has requested post-marketing RWD to enhance the clinical and cost-effectiveness evidence for certain cancer drugs to track especially longer term outcomes.
One example of the successful use of RWE involves the immuno-oncology drug ipilimumab for treatment of patients with malignant melanoma, which was approved on the Managed Entry Scheme (MES). Inclusion on the MES involved a risk-sharing ‘pay for performance’ arrangement whereby rebates would be payable should the two-year overall survival rates in real world clinical practice not align with the RCT outcome. To meet this requirement the sponsor collected RWD from consenting patients prescribed ipilimumab over the 2-year follow-up period. In this case, patients’ overall survival, the gold standard endpoint in RCTs, was supported by the RWE.
This is an example of the value of RWE in removing payer doubts around clinical and/or economic effectiveness, thereby benefitting patients through earlier access to subsidised medicines.
CRC’s experienced Medical Affairs team has the expertise to identify and work with key stake holders to understand where evidence gaps exist and develop strategies for generating RWE to inform reimbursement decisions.
- Australian Government Cancer Australia. All cancers in Australia. 2018. Available at: https://bit.ly/2cfR33K
- Australian Government Dept of Health. Cancer Fact Sheet. 2018. Available at: https://bit.ly/2DJEefs
- Chatterjee A., et al. Real-world evidence: Driving a new drug-development paradigm in oncology. 2018. McKinsey&Company. Available at: https://mck.co/2KpCtrs
- Eastman, P. Potential Value of Real-World Evidence in Lung Cancer Care. 2018. Oncology Times. Available at: https://bit.ly/2R5IVmL
- Real-World Evidence in Oncology. 2018. IQVIA. Available at: https://bit.ly/2Dus9ds
- US Food and Drug Administration. Framework for Real World Evidence. 2018. Available at: https://bit.ly/2B0i7ze
- Kim H., et al. A real-world example of coverage with evidence development in Australia – ipilimumab for the treatment of metastatic melanoma. 2018. Journal of Pharmaceutical Policy and Practice