Are cancer patients better off if they participate in clinical trials? A mixed methods study

• Published in: BMC cancer Vol. 20; no. 1; p. 401
• Main Authors: Engelbak Nielsen, Zandra, Eriksson, Stefan, Schram Harsløf, Laurine Bente, Petri, Suzanne, Helgesson, Gert, Mangset, Margrete, Godskesen, Tove E
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 08-05-2020; BioMed Central; BMC
• Subjects: Adult; Attitude of Health Personnel; Cancer; Cancer och onkologi; Cancer patients; Cancer research; Clinical trials; Clinical Trials as Topic - statistics & numerical data; Codes; Comparative analysis; Drugs; Ethics; Female; Health Personnel - psychology; Hematology; Humans; Hälso- och sjukvårdsorganisation, hälsopolitik och hälsoekonomi; Hälsovetenskap; Immunotherapy; Interviews; Klinisk medicin; Literature review; Literature reviews; Male; Medical care quality; Medical research; Medicin och hälsovetenskap; Middle Aged; Mixed methods; Mixed methods research; Models, Theoretical; Neoplasms; Neoplasms - therapy; Nurses; Oncology; Outcome Assessment, Health Care; Outcomes; Participation; Patient Participation; Patient Selection; Patients; Pharmacological clinical trials; Physicians; Qualitative; Research Design; Researchers; Studies; Survival; Treatment Outcome; Sweden; Denmark; Pharmacological clinical trials; Mixed methods; Physicians; Nurses; Literature review; Qualitative; Survival; Neoplasms; Outcomes
• ISSN: 1471-2407; 1471-2407
• DOI: 10.1186/s12885-020-06916-z

Research and cancer care are closely intertwined; however, it is not clear whether physicians and nurses believe that clinical trials offer the best treatment for patients and, if so, whether this belief is justified. The aim of this study was therefore: (i) to explore how physicians and nurses perceive the benefits of clinical trial participation compared with standard care and (ii) whether it is justified to claim that clinical trial participation improves outcomes for cancer patients.
A mixed methods approach was used employing semi-structured interviews with 57 physicians and nurses in oncology and haematology and a literature review of the evidence for trial superiority, i.e. the idea that receiving treatment in a clinical trial leads to a better outcome compared with standard care. Inductive thematic analysis was used to examine the interview data. A literature review comprising nine articles was conducted according to a conceptual framework developed by Peppercorn et al. and evaluated recent evidence on trial superiority.
Our findings show that many physicians and nurses make claims supporting trial superiority, however very little evidence is available in the literature comparing outcomes for trial participants and non-participants that supports their assertions.
Despite the recent rapid development and use of targeted therapy and immunotherapy, we find no support for trial participation to provide better outcomes for cancer patients than standard care. Hence, our present results are in line with previous results from Peppercorn et al. A weaker version of the superiority claim is that even if a trial does not bring about a direct positive effect, it brings about indirect positive effects. However, as the value of such indirect effects is dependent on the individual's specific circumstances and preferences, their existence cannot establish the general claim that treatment in trials is superior. Belief in trial superiority is therefore unfounded. Hence, if such beliefs are communicated to patients in a trial recruitment context, it would provide misleading information. Instead emphasis should be on patients volunteering to give an altruistic contribution to the furthering of knowledge and to the potential benefit of future patients.