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Study Encourages Mixed Methods Research for Rare Disease Clinical Trials

Alison Rodriguez
Mixed methods research incorporating interviews found a high concordance between patient-reported qualitative data and assessed tumor response in patients with Merkel cell carcinoma.
Rare diseases often present challenging paths to find effective treatment options. Mixed methods research help overcome such challenges by combining quantitative and qualitative data, and therefore providing a better understanding of research questions. The recent JAVELIN Merkel 200 trial used a convergent mixed methods design to study Merkel cell carcinoma (MCC).

The study incorporated interviews with 9 patients receiving avelumab in the JAVELIN Merkel 200 trial. The interviews took place at baseline prior to receiving study treatment, at 13 weeks, and 25 weeks after the first avelumab administration. The interviews intended to gain insight into the physical functioning, fatigue/energy, and pain that the patient may be experiencing. They also recorded patient perceptions of the overall change in their cancer-related health status.

“While mixed methods research is recognised and well established in the social and behavioural sciences, it has only recently emerged in clinical research,” the study stated. “Mixed method research provides the advantages of qualitative research, which includes a large and rich amount of explorative data allowing expression of patients’ voices and exploration of the disease and its management.”

Following an analysis of the data collected through the interviews, the researchers found a high concordance between patient-reported qualitative data and assessed tumor response. There were 8 patients who clinically improved and had perceived a subjective improvement in their disease since the beginning of the study. The one patient whose disease worsened had a perceived deterioration, according to the study.

In addition, patient-perceived benefit in physical functioning, fatigue/energy, and pain was subsequent to the measured change in clinical status as evaluated by the tumor response—suggesting that patient-reported assessment should be assessed over the long term in order to fully understand meaningful treatment effects.

“Embedding qualitative research in clinical trials to complement the quantitative data is an innovative approach to characterise meaningful treatment effect,” the study noted.. “This application of mixed methods research has the potential to overcome the hurdles associated with clinical outcomes assessment in rare diseases.”

The researchers emphasized their hope this study will promote the use of the mixed methods approach within a clinical trial setting in order to help overcome the challenges associated with rare diseases.

“We acknowledge that the sample size is very limited, however in this context of a very rare and aggressive disease on which very little is known, we believe that our data, descriptive and exploratory, are worth communicating and hopefully will encourage others to do so in this field,” concluded the researchers.


Bharmal M, Guillemin I, Marrel A, et al. How to address the challenge of evaluating treatment benefits-risks in rare diseases? A convergent mixed methods approach applied within a Merkel cell carcinoma phase 2 clinical trial. [published online June 18, 2018]. Orphanet J Rare Dis. doi: 10.1186/s13023-018-0835-1.

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