How does perspective and setting influence how you view RWE: Safety assessments of novel treatments
31 Jul 2018

Real-world evidence (RWE) has often been discussed in the pharmaceutical and medical technology fields as an emerging tool for assessing safety of new treatments. It was recently discussed in the context of oncology and haematology by The Lancet Haematology Commission paper from 2018, and by Smedby and Eloranta, researchers from Karolinska University Hospital. In the past, the authors state, safety assessments in clinical trials have mostly aimed to capture short-term acute and potentially life-threatening adverse events during intensive chemotherapy, with limited follow-up. Potentially leaving gaps in knowledge concerning chronic, cumulative and late effects with long-term exposure to such treatments. With this is mind, combined with the growing financial and time pressure against doing prolonged patient trials, RWE is increasing in relevance.

There can be a ‘tension of perspectives’ when it comes to the use of RWE. This is especially true in the field of pharmaceuticals, where the stated ‘gold-standards’ are randomised controlled trials (RCTs). When moving forward with research and trials, often the research question and setting of the researcher plays a part in determining the design of studies. Your view on the usefulness of RWE in your study may depend on what your question or setting is, for example if you have a question that has a 10-year vs. 1-year perspective on adverse effects. If you require a 10-year perspective, an RCT will never be able to accurately depict usable results and outcomes, which is where RWE shines.

Examples of RWE used for safety assessments in the field of haematology and oncology, include the 2016 Hodgkin lymphoma study carried out by van Leeuwen. This study found that there was an increased risk of secondary malignancy and heart disease after patients underwent radiotherapy for Hodgkin lymphoma. A similar study from 2007 looking into the short and long-term adverse events of haematopoietic cell transplants, found several potentially severe after-effects, which may not have been picked up on in trials. These findings and others from similar studies, highlight the need for large, representative patient populations in carefully designed clinical, observational studies.

Step in real-world data (RWD) sources. For example, high-quality patient registers, such as those found in the Nordic countries, especially Sweden. Detailed population records have been compiled since the early 18th Century, so the tradition of data collection remains strong within the fields of healthcare and research. Patient registers can be found on any number of disease and intervention areas, with variables and indicators providing a detailed account of each patient’s experience throughout their care. One example includes the Swedish Haematological malignancy quality-of-care registers which include nearly 50 000 patients, with various conditions and at different stages of treatment. The provision of such data, allows long-term assessments of adverse effects from treatments and through linkage to local care and drug registers, can allow for specific monitoring of treatments. For some patient registers, patient reported outcomes have also been added to the disease and treatment data routinely collected, allowing for more holistic outcomes to also be monitored. The Lancet Haematology Commission paper from 2018, also discusses other areas of improvement for the assessment and reporting of adverse events in haematological assessments. Other disease and intervention areas will likely support these discussions too.

The need for improved safety monitoring in premarketing trials in these areas, and potentially other disease areas is crucial and relevant if we are to improve our standard-of-care treatments offered to patients.


Articles of Interest:

Smedby KE, Eloranta S. Real-world evidence in safety assessment of new treatments. The Lancet Haematology; Comment. 2018 Jun.

Thanarajasingam G, Minasian LM, Baron F, et al. Beyond maximum grade: modernising the assessment and reporting of adverse events in haematological malignancies. Lancet Haematol 2018; published online June 12.

van Leeuwen FE, Ng AK. Long­term risk of second malignancy and cardiovascular disease after Hodgkin lymphoma treatment. Hematology Am Soc Hematol Educ Program 2016; 2016: 323–30

Majhail NS, Ness KK, Burns LJ, et al. Late effects in survivors of Hodgkin and non­Hodgkin lymphoma treated with autologous hematopoietic cell transplantation: a report from the bone marrow transplant survivor study. Biol Blood Marrow Transplant 2007; 13: 1153–59.

Blimark CH, Turesson I, Genell A, et al. Outcome and survival of myeloma patients diagnosed 2008–2015. Real­world data on 4904 patients from the Swedish Myeloma Registry. Haematologica 2018; 103: 506–13.

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