Description of the goods or services required
19/81 Neoadjuvant endocrine therapy for postmenopausal women with breast cancer
The aim of the HTA Programme is to ensure that high quality research information on the effectiveness, costs and broader impact of health technology is produced in the most efficient way for those who use, manage, provide care in or develop policy for the NHS. Topics for research are identified and prioritised to meet the needs of the NHS. Health technology assessment forms a substantial portfolio of work within the National Institute for Health Research and each year about fifty new studies are commissioned to help answer questions of direct importance to the NHS. The studies include both primary research and evidence synthesis.
Research Question: What is the effectiveness of neoadjuvant endocrine therapy for postmenopausal women with breast cancer?
1.Intervention: Neoadjuvant endocrine therapy (aromatase inhibitor). Duration and regimen to be defined and justified by applicants.
2.Patient group: Postmenopausal women with oestrogen-receptor (ER) positive/HER2-negative breast cancer. To be defined and justified by applicants.
Applications are encouraged which include recruitment from geographic populations with high disease burden which have been historically underserved by research activity in this field.
3.Setting: Secondary care.
4.Comparator: Applicants to define and justify the most appropriate comparator to neoadjuvant endocrine therapy e.g. neoadjuvant chemotherapy.
5.Study design: A randomised controlled trial with an internal pilot phase to test the ability to recruit and randomise. Clear stop/go criteria should be provided to inform progression from pilot to full trial.
6.Important outcomes: Response rates (e.g. reduction in tumour size, breast conservation rates).
Other outcomes: Adverse events (e.g. disease progression), patient reported outcome measures/ quality of life (including menopausal side-effects), cost-effectiveness.
7.Minimum duration of follow-up: At least 1 year.
Longer-term follow up: If appropriate, researchers should consider obtaining consent from participants to allow potential future follow up through efficient means (such as routine data) as part of a separately funded study.
A diagnosis of breast cancer can be overwhelming and frightening. At a difficult time, women are faced with making important decisions about their treatment, supported and informed by clinicians and current evidence.
One such decision concerns neoadjuvant therapies, which have the potential to reduce the size of a tumour and result in breast conserving surgery rather than a mastectomy. Breast conserving surgery is generally more acceptable to women with improved patient reported outcomes. While neoadjuvant endocrine therapy is currently being used in the NHS, there is a lack of evidence about its effectiveness and whether it should be recommended for some postmenopausal women with ER-positive/HER2-negative cancer. Consequently, this important decision is being made on inadequate information.
To address this uncertainty, the recent NICE guideline committee recommended research to examine whether neoadjuvant endocrine therapy is effective. The results should be suitable to inform practice and updates to future guidelines. Evidence from the proposed research will help to address the uncertainty about whether neoadjuvant endocrine therapy is effective for the target group and enable clinicians to guide patients more effectively. It may give a further treatment option for women faced with a cancer diagnosis and more certainty about the decisions they have to make.