Development and validation of a Patient Reported Experience Measure for Experimental Cancer Medicines (PREM-ECM) and their carers (PREM-ECM-Carer):Development & validation of PREM-ECMs

Background: Our aim was to develop a validated Patient Reported Experience Measure (PREM) to capture patient and carer experience during participation in experimental cancer medicine trials (ECM): called PREM-ECM. Methods: Mixed method design, consisting of four stages. Questionnaire items were produced for both patients and carers using interviews, focus groups, and cognitive interviews with patients and carers separately. For both PREMs, a cross-sectional questionnaire study was conducted to identify final items for inclusion using hierarchical item reduction and Rasch analysis. Questionnaire validity and reliability were assessed, including administration feasibility.Results: Initial interview participants suggested the need for three PREMs, two specific to patients: i) a ‘prior’ questionnaire that captured experiences of trial introduction, screening, consenting, and early trial experience (&lt;6 weeks post consent); and ii) ‘on-trial’ that captured experiences of ongoing consent and trial participation; and iii) a PREM specific for carers. The draft 25-item ‘prior’ questionnaire was completed by 162 patients and 162 patients completed the draft 35-item ‘on-trial’ questionnaire. Hierarchical and Rasch analysis produced a 14-item ‘prior’ list and a 15-item list for ‘on-trial’. Both patient PREM’s demonstrated good fit to the Rasch model (X2 p&lt;0.001). The carer 34-draft item questionnaire was completed by 102 participants. Hierarchical and Rasch analysis produced a 13-item list for PREM-ECM-Carer, with good fit to the Rasch model (p=0.62). The pilot testing demonstrated the feasibility of all the PREMs in capturing patient and caregiver experiences in routine clinical settings.Conclusions: The three PREM-ECM questionnaires will be the first validated experience measures for ECM trial patients and their carers. These questionnaires may be used to assess patients’ and their carers’ experiences of ECM and enable robust comparisons across cancer trial units highlighting areas for service improvement. <br/

Patient-reported outcome (PRO) instruments used in patients undergoing adoptive cell therapy (ACT) for the treatment of cancer: a systematic review

Abstract Introduction Adoptive cell therapy (ACT) is a rapidly evolving field. Patient-reported outcomes (PROs) allow patients to report the impact of treatment on their quality of life during and after treatment. The systematic review aims to characterise the breadth of PROs utilised in ACT cancer care and provide guidance for the use of PROs in this patient population in the future. Methods A systematic search was conducted (MEDLINE, PsycINFO, Embase and CINAHL) in August 2021 by two reviewers. Search terms covered the following: “adoptive cell therapy”, “patient-reported outcomes” and “cancer”. Studies were included if they used a PRO measure to report the impact of ACT. The methodological quality of PROs was assessed. Forward and backward reference searching was conducted of any relevant papers. A quality grading scale was applied based on Cochrane and Revenson criteria for classification of high-quality studies. Key data from the studies and the included PROs was extracted by two researchers and tabulated. Results One-hundred nine papers were identified; 11 papers were included. The majority of studies were single-arm trials or observational studies. Twenty-two different PROs were identified; none was ACT specific. The PROMIS-29 and EQ-5D were most commonly used. Few studies collected PRO data in the first 1–2 weeks. Four studies followed patients up for over a year, and a further four studies followed patients for approximately 3 months. Discussion None of the PROs identified have been designed specifically for ACT. Appropriateness of existing instruments should be considered. It should be considered whether it is appropriate to collect data more frequently in the acute stage and then less frequently during follow-up. It should be considered if one tool is suitable at all time points or if the tool should be adapted depending on time since treatment. More research is needed to identify the exact timings of PRO assessments, and qualitative work with patients is needed to determine the most important issues for them throughout the treatment and follow-up

Oncology patients' experiences in experimental medicine cancer trials:a qualitative study

OBJECTIVES: The study aimed to explore patients’ experiences of experimental cancer medicine (ECM) clinical trials. DESIGN: The study’s design was qualitative. Two focus groups with patients were undertaken followed by semistructured interviews, to explore patients’ experiences of ECM clinical trials. Interviews and focus groups were audiorecorded and transcribed verbatim. Data were analysed using thematic analysis. SETTING: A regional cancer centre (tertiary care) in North-West England. PARTICIPANTS: Twelve patients (aged 52–79) participated in one of the two focus groups and 22 patients (aged 42–83) participated in interviews. PRIMARY OUTCOME MEASURE: Patients’ experiences of an ECM trial. RESULTS: Four main themes were identified from the analysis: decision making, information needs, the experience of trial participation and impact of trial participation. Subthemes are presented in the manuscript. CONCLUSION: To make fully informed decisions about trial participation, patients required the simplification of trial information and wanted more information about side effects, their response to trial treatment and the overall trial progress throughout the trial. Patients highlighted the need for improvement for the support provided to their family and friends