“I just wanted to speak to someone- and there was no one…”: using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients
How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis
17 April 2023
Developments in corneal transplants
20 April 2023

Orphanet J Rare Dis. 2023 Apr 17;18(1):86. doi: 10.1186/s13023-023-02680-y. PMID: 37069697; PMCID: PMC10111696.

“I just wanted to speak to someone- and there was no one…”: using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients

Authors: Ian Litchfield 1, Melanie J Calvert 2 3 4 5 6 7, Francesca Kinsella 4 5 8 9, Nisha Sungum 10 11, Olalekan L Aiyegbusi 2 3 4 5 6 8 7

Affiliation:

  • 1Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK. I.litchfield@bham.ac.uk.
  • 2Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, B15 2TT, UK.
  • 3Centre for Patient Reported Outcomes Research, University of Birmingham, Birmingham, UK.
  • 4NIHR Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK.
  • 5NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK.
  • 6Applied Research Collaboration (ARC) – West Midlands, Birmingham, UK.
  • 7Birmingham Health Partners (BHP) Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
  • 8Institute of Immunology and Immunotherapy, University of Birmingham, Birmingham, UK.
  • 9Centre for Clinical Haematology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
  • 10Midlands and Wales Advanced Therapy Treatment Centre, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
  • 11Research Development and Innovation, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.

Abstract:

Background: Advanced therapy medicinal products such as Chimeric antigen receptor T-cell therapy offer ground-breaking opportunities for the treatment of various cancers, inherited diseases, and chronic conditions. With development of these novel therapies continuing to increase it’s important to learn from the experiences of patients who were among the first recipients of ATMPs. In this way we can improve the clinical and psychosocial support offered to early patient recipients in the future to support the successful completion of treatments and trials.

Study design: We conducted a qualitative investigation informed by the principles of the key informant technique to capture the experience of some of the first patients to experience CAR-T therapy in the UK. A directed content analysis was used to populate a theoretical framework informed by Burden of Treatment Theory to determine the lessons that can be learnt in supporting their care, support, and ongoing self-management.

Results: A total of five key informants were interviewed. Their experiences were described within the three domains of the burden of treatment framework; (1) The health care tasks delegated to patients, Participants described the frequency of follow-up and the resources involved, the esoteric nature of the information provided by clinicians; (2) Exacerbating factors of the treatment, which notably included the lack of understanding of the clinical impacts of the treatment in the broader health service, and the lack of a peer network to support patient understanding; (3) Consequences of the treatment, in which they described the anxiety induced by the process surrounding their selection for treatment, and the feeling of loneliness and isolation at being amongst the very first recipients.

Conclusions: If ATMPs are to be successfully introduced at the rates forecast, then it is important that the burden placed on early recipients is minimised. We have discovered how they can feel emotionally isolated, clinically vulnerable, and structurally unsupported by a disparate and pressured health service. We recommend that where possible, structured peer support be put in place alongside signposting to additional information that includes the planned pattern of follow-up, and the management of discharged patients would ideally accommodate individual circumstances and preferences to minimize the burden of treatment.

Keywords: Burden of treatment; Chimeric antigen receptor (CAR); Personalised-care; T-cell therapy

Download: https://ojrd.biomedcentral.com/articles/10.1186/s13023-023-02680-yhttps://ojrd.biomedcentral.com/articles/10.1186/s13023-023-02680-y