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Case Study
The Qualitative Patient Story Behind a Myositis Clinical Trial
This case study, conducted by The Henne Group (THG), focused on a clinical-stage biotechnology company conducting a clinical trial for two rare, debilitating autoimmune disorders: Dermatomyositis (DM) and Polymyositis (PM).
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Dermatomyositis (DM): A rare inflammatory disease characterized by muscle weakness and a distinctive skin rash.
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Polymyositis (PM): A rare inflammatory disease that causes muscle weakness, usually in the muscles closest to the trunk of the body.
The Challenge: The Data and the Disconnect
While the trial's primary quantitative endpoint, a composite score of improvement, showed no statistically significant difference between the drug and the placebo, the client's team observed a critical divergence between the numbers and patient behavior:
Rare Low Dropout Rate
An unusually low dropout rate among patients in the long-term, open-label extension (OLE) phase of the trial
Positive, Yet Anecdotal Evidence
Reports from trial investigators indicated "substantial functional improvements" in some patients, a positive signal not captured by the main metric.
The client faced a defining challenge: they needed to understand the story behind the data. Why were patients choosing to remain in a demanding trial when the primary, objective metrics failed to show a clear benefit?
The company engaged The Henne Group (THG) to uncover the nuanced, real-world experiences of trial participants.
The Intervention: Uncovering the "Secret Sauce" of Qualitative Insight
THG and our research partners at MNW Consulting Group designed and executed a strategic qualitative research study. This was not a "cookie-cutter" approach; our expertise lies in developing the precise questions, the Interviewer Guide, that unlock true insight.
Expert Interview Guide Development
The construction of the Interviewer Guide played an integral role in the project's success, moving beyond surface-level queries to explore the lived experience of the disease and the treatment burden.
Strategic Recruitment & Enrollment
THG initiated contact with investigators directly, successfully enrolling key sites into this follow-up study. We coordinated with site staff to invite a diverse pool of trial patients to participate, including those who completed the trial, those who withdrew early, and Spanish-speaking individuals, ensuring all voices were heard.
The Power of Synthesis
Our joint research team conducted sensitive, 40- to 60-minute in-depth, semi-structured interviews via a secure video platform. The skill of the moderator to bring learnings from previous groups to build on themes is the "secret sauce" that ensures we are constantly refining our focus to maximize insight.
Data Point Saturation
A targeted cohort of patients and principal investigators. A core principle of our qualitative methodology is determining when we have reached data point saturation, which is the point in data collection where conducting additional interviews is unlikely to yield new information or themes. This ensures both rigor and efficiency.
Participant Management
THG managed all participant communications and provided stipends to thank them for their valuable time and insights
Assessment and Key Findings
Our team employed a rigorous thematic analysis methodology, utilizing advanced coding software like NVivo, to systematically code the interview transcripts and identify core themes. This moved the conversation beyond isolated anecdotes to deliver a structured, evidence-based understanding of the patient and investigator experience.
Redefining "Success"
For patients, improvement wasn't an abstract score. It was the concrete, life-changing ability to perform daily tasks that had been lost to their disease. Patients described regaining the strength to dry their own hair, to cook a meal, to walk upstairs without assistance, or to hike with their families. These functional gains, while not fully captured by the trial's primary endpoint, were immensely meaningful and motivated them to continue treatment.
The Psychosocial Impact
The study illuminated the deep emotional toll of these diseases, including anxiety and depression linked to functional loss. As their physical symptoms improved, patients reported feeling happier, more motivated, and less anxious, regaining the ability to live a "more normal life".
Weighing the Burden
We captured the significant challenges of the treatment. Patients reported that the weekly treatment could be extremely painful, one described it as "fire going in", and side effects were common. For many, the functional benefits were worth the discomfort; for others, the side effects were the primary reason for discontinuing the trial.
Actionable Clinical Insights
The qualitative data uncovered a critical, actionable direction: one investigator observed that the medication did not appear to be effective for patients with a more intense presentation of their illnesses, suggesting an inefficacy in the necrotizing myopathy subgroup.
Key Results:
Turning Human Experience into Business Strategy
By providing a platform for the patient voice, THG helped our biotechnology client achieve several key outcomes, turning complexity into clarity:
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Explained Patient Behavior: We delivered a clear, evidence-based explanation for the high retention rate in the OLE, linking it directly to patients' perceived functional and quality-of-life improvements.
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Identified Patient-Valued Endpoints: Our research highlighted that improvements in daily activities and endurance are the outcomes most highly valued by this patient population, providing crucial information for designing future clinical trials that capture meaningful change.
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Provided a 360-Degree View of Treatment: The findings gave the client a comprehensive understanding of the entire treatment experience, including the significant burden of painful injections and side effects, vital data for informing potential improvements to the drug's formulation or administration.
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Informed Future R&D Strategy: We delivered a specific, data-driven insight about the drug's apparent lack of efficacy in a key patient subgroup, enabling the client to refine its research and development focus and avoid costly pursuits in non-responsive patient groups.
Conclusion and
Replication
This case study proves the immense value of integrating rigorous qualitative research into a clinical development program. When quantitative data tells only part of the story, in-depth interviews uncover the rich, human context that explains patient behavior, highlight what truly matters to them, and provide actionable insights to guide business strategy.
The Henne Group’s approach is highly effective and replicable for any organization needing to understand the nuanced experiences of patients, caregivers, or healthcare providers. By bringing the voice of the stakeholder to the forefront, we turn data points into a compelling narrative and ambiguity into clarity.
