Hematological malignancies are a diverse group of cancers that affect the blood, bone marrow, lymph nodes, and spleen. The complexity and variability of these diseases demand innovative and personalized approaches to treatment that traditional clinical trial methodologies often lack. Designing patient-centric hematology clinical trials requires a comprehensive understanding of the patient experience, coupled with a commitment to inclusivity, transparency, and real-world relevance. In this blog, we explore the evolution of patient centricity in hematological malignancy trials and offer five key considerations for implementation.
From Clinician to Patient Voice
The clinical research industry has been undergoing a paradigm shift towards study approaches that engage patients as informed collaborators throughout the development process. Traditionally, when establishing a clinical trial protocol, sponsors have approached clinicians for guidance on what is best for patients, their treatment, and their disease. However, while clinical input remains critical for things like the inclusion/exclusion criteria and other elements of the protocol, it’s important to bear in mind that physicians are still one step removed from the real patient disease experience. And for us to truly understand the burden of disease, sponsors and CROs need to view clinical trials through the lens of the patient.
How to Shift the Focus to Patients and Caregivers: Five Tips
When executing a patient-centric hematological malignancy trial, there are multiple dimensions to consider, from enrollment to convenience, to technical support and data management. Here are five strategies to consider.
- Focus on Early and Continuous Patient Involvement
From the outset, involve patients in the design process of clinical trials. This ensures that the research questions, trial objectives, and endpoints align with the experiences and priorities of those living with hematological malignancies. Establish patient advisory boards or focus groups to facilitate ongoing collaboration throughout the trial, allowing for the incorporation of real-world insights and feedback. Advocacy groups can also provide valuable feedback on the appropriateness of a study protocol and the optimal methods and channels for reaching the targeted patient population.
- Patient-Reported Outcomes (PROs)
Incorporate patient-reported outcomes as primary or secondary endpoints in the trial design. These outcomes provide a direct assessment of the impact of the disease and the treatment on the patients’ quality of life, symptoms, and overall well-being. Including PROs ensures that the trial’s success is measured not only by traditional clinical metrics but also by improvements that matter most to patients.
- Offer Patient-Friendly Trial Logistics
Streamline trial logistics to minimize burden on patients and their caregivers. Consider factors such as the frequency of clinic visits, travel requirements, and the overall impact on daily life. Hematological malignancy patients tend to be either very young or very elderly, making them unable to tolerate long drives and long visit days, especially those that require intense PK draws, for instance. Implement telemedicine options where feasible and provide support services to address non-medical needs, recognizing that patient and caregiver convenience and comfort contribute to overall trial success.
- Incorporate Technology for Remote Monitoring
In the era of digital health, leveraging technology for remote monitoring can greatly enhance the patient centricity of clinical trials. Utilize wearable devices, mobile applications, or telehealth services to collect real-time data, monitor patient well-being, and enable remote consultations. This not only provides a more comprehensive dataset but also enhances the overall trial experience by reducing the need for frequent onsite visits.
- Provide Post-Trial Engagement
Engage patients beyond the trial endpoint. Share results with participants in an understandable format and acknowledge their contribution to advancing medical knowledge. Encourage ongoing involvement in long-term follow-up studies, and foster a sense of community among trial participants, recognizing the lasting impact of their participation.
More Than a Trendy Phrase
Patient centricity is no longer just a buzzword, but a fundamental reimagining of how clinical trials are conceived and executed. By recognizing the unique needs and perspectives of patients, we can design more effective, inclusive, and impactful hematological malignancy trials, ultimately improving the lives of those affected by these complex diseases.
With more than 60 hematology studies conducted in the last five years, Premier offers significant expertise in the design and execution of hematological malignancy trials. To learn more about how we can support your study, contact us.