CAR-T Cell Therapies Hematological Cancers White Paper

Using Patient Experience Data to Demonstrate the Value of CAR-T Cell Therapies in Clinical Trials of Hematological Cancers

The development of cell and gene therapies, specifically chimeric antigen receptor T (CAR-T) cell therapy, is transforming cancer treatment. This is especially true for relapsed and refractory hematological malignancies, where CAR-T therapy significantly enhances patients’ health-related quality of life (HRQL). However, high costs, severe adverse events (AEs) and potential long-term impacts necessitate incorporating patient input into the drug development process to inform risk-benefit assessments. 

Various methods, including patient-reported outcomes (PRO) assessments, interviews, social media research and patient preference studies, capture valuable patient experience data (PED) on symptoms, functioning, HRQL and treatment preferences. This PED is essential for health care decision-makers in making approval and access decisions.  

This paper examines the importance of: 

• Patient input and experience data (PED) in understanding the impact of CAR-T cell therapy 
• Opportunities and barriers in generating PED, emphasizing its importance in long-term follow-up 
• Challenges in capturing patient perspectives for CAR-T treatments and potential solutions