Dataset Information

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study.

ABSTRACT:

Objectives

Examine whether data from early access to medicines in the USA can be used to inform National Institute for Health and Care Excellence (NICE) health technology assessments (HTA) in oncology.

Design

Retrospective cohort study.

Setting

Oncology-based community and academic treatment centres in the USA.

Participants

Patients present in a nationwide electronic health record (EHR)-derived deidentified database.

Interventions

Cancer drugs that underwent NICE technology appraisal (TA) between 2014 and 2019.

Primary and secondary outcome measures

The count and follow-up time of US patients, available in the EHR, who were exposed to cancer drugs of interest in the period between Food and Drug Administration (FDA) approval and dates relevant to the NICE appraisal process.

Results

In 59 of 60 TAs analysed, the cancer therapy was approved in the USA before the final appraisal by NICE. The median time from FDA approval to the publication of NICE recommendations was 18.5 months, at which time the US EHR-derived database had, on average, 269 patients (SD=356) exposed to the new therapy, with a median of 75.3 person-years (IQR: 13.1-173) in time-at-risk. A case study generated evidence on real-world overall survival and treatment duration.

Conclusions

Across different cancer therapies, there was substantial variability in US real-world data accumulated between FDA approval and NICE decision milestones. The applicability of these data to generate evidence for HTA decision-making should be assessed on a case-by-case basis depending on the intended HTA use case.

SUBMITTER: Mpofu P

PROVIDER: S-EPMC10582952 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study.

Mpofu Philani P Kent Seamus S Jónsson Páll P Pittell Harlan H Groves Brad B Altomare Ivy I Copeland Amanda A Baxi Shrujal S Bargo Danielle D Sujenthiran Arun A Adamson Blythe B

BMJ open 20231017 10

<h4>Objectives</h4>Examine whether data from early access to medicines in the USA can be used to inform National Institute for Health and Care Excellence (NICE) health technology assessments (HTA) in oncology.<h4>Design</h4>Retrospective cohort study.<h4>Setting</h4>Oncology-based community and academic treatment centres in the USA.<h4>Participants</h4>Patients present in a nationwide electronic health record (EHR)-derived deidentified database.<h4>Interventions</h4>Cancer drugs that underwent N ...[more]

PMID: 37848301

Similar Datasets

Project description:ObjectivesDealing with uncertainty is one of the critical topics in health technology assessment. The greater decision uncertainty in appraisals, the less clear the clinical- and cost-effectiveness of the health technology. Although the development of targeted cancer therapies (TCTs) has improved patient health care, additional complexity has been introduced in drug appraisals due to targeting more specific populations. Real-world data (RWD) are expected to provide helpful information to fill the evidence gaps in appraisals. This study compared appraisals of TCTs with those of non-targeted cancer therapies (non-TCTs) regarding sources of uncertainty and reviewed how RWD have been used to supplement the information in these appraisals.MethodsThis study reviews single technology appraisals (STAs) of oncology medicines performed by the National Institute for Health and Care Excellence (NICE) over 11 years up to December 2021. Three key sources of uncertainty were identified for comparison (generalisability of clinical trials, availability of direct treatment comparison, maturity of survival data in clinical trials). To measure the intensity of use of RWD in appraisals, three components were identified (overall survival, volume of treatment, and choice of comparators).ResultsTCTs received more recommendations for provision through the Cancer Drugs Fund (27.7, 23.6% for non-TCT), whereas similar proportions were recommended for routine commissioning. With respect to sources of uncertainty, the external validity of clinical trials was greater in TCT appraisals (p = 0.026), whereas mature survival data were available in fewer TCT appraisals (p = 0.027). Both groups showed similar patterns of use of RWD. There was no clear evidence that RWD have been used more intensively in appraisals of TCT.ConclusionsSome differences in uncertainty were found between TCT and non-TCT appraisals. The appraisal of TCT is generally challenging, but these challenges are neither new nor distinctive. The same sources of uncertainty were often found in the non-TCT appraisals. The uncertainty when appraising TCT stems from insufficient data rather than the characteristics of the drugs. Although RWD might be expected to play a more active role in appraisals of TCT, the use of RWD has generally been limited.

Dataset Information

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study.

Objectives

Design

Setting

Participants

Interventions

Primary and secondary outcome measures

Results

Conclusions

Publications

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study.

Similar Datasets

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