Assessment of the British Society of Blood and Marrow Transplantation and Cellular Therapy (BSBMTCT) registry using the Registry Evaluation and Quality Standards Tool (REQueST)

Background

The National Institute for Health and Care Excellence (NICE) commissioned the Newcastle External Assessment Centre (EAC) to undertake an evaluation of registries to support the Cancer Drugs Fund (CDF) and Highly Specialised Technologies (HST) real world evidence development using EUnetHTA’s Registry Evaluation and Quality Standards Tool (REQueST).

The REQueST tool is a standardised set of questions contained within an Excel file which enables:

• registry owners to assess/demonstrate the quality of their data collection
• HTA/regulatory professionals to assess the quality of evidence and elements of good practice

The tool comprises 3 sections:

1. Methodological Information – to screen for registries whose data and methodology match the requirements of the HTA/regulatory study or research question(s)
2. Essential Standards – an assessment of registry governance to assure general data quality and protection
3. Additional Requirements – any specific requirements for the evidence questions

The BSBMTCT registry is to be used to provide information to support the Managed Access Agreement (MAA) for Tisagenlecleucel (Kymriah, Novartis) for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged up to 25 years (see NICE Technology Assessment TA554 [published Dec 2018 – to be reviewed June 2023]). Tisagenlecleucel is a chimeric antigen receptor (CAR) T‑cell therapy. It contains the patient’s own T cells that have been modified to attach to and kill cancer cells. It is recommended for use under the CDF only if the conditions in the MAA are followed: https://www.nice.org.uk/guidance/ta554/resources/managed-access-agreement-december-2018-pdf-6651288397. 

The primary sources of data collection are ongoing clinical trials: ELIANA, ENSIGN and B2101J, however a secondary source of data collection was identified as the BSBMTCT data registry which is held at Guy’s and St Thomas’ NHS Trust and collects data on all bone marrow transplants in the UK, and more recently cell therapies. This could be used to determine the rate and time to subsequent stem cell transplants.

The REQueST tool was used to assess the suitability of the registry for this purpose.

Assessment of the BSBMTCT registry

The tool was completed and returned to the EAC by Julia Lee – Head of the BSBMTCT Data Registry. The information provided was independently assessed by the EAC and NICE. A meeting was held between EAC/NICE to agree feedback and further specific questions to be asked. After the first submission, the majority of items in the tool were assessed as‘Needs development / Clarification’ (see output below).

1. 1^st submission: Output from tool

After the second submission, the majority of items were assessed as ‘Satisfactory’ however with the information available, 5 items were assessed as ‘Not suitable’ (see output below).

• 2nd submission: Output from tool

The items assessed as ‘Not suitable’ are summarised below with an explanation and recommendation:

Conclusions

The information provided in the REQueST tool enabled 18 out of the 23 items to be assessed as ‘satisfactory’ for the purposes of the MAA. For the 5 remaining items (described above), an ‘unsuitable’ assessment was made as there was no further opportunity within this study to request further information and/or clarification. Recommendations have been made for each of the 5 items and these should be followed up between NICE and the BSBMTCT at the earliest opportunity.