NIHRDataInsights/HRCSData huggingface dataset:
Title: Pertuzumab for the adjuvant treatment of HER2-positive breast cancer [ID1192]
Funder: Department of Health and Social Care

Abstract: To appraise the clinical and cost effectiveness of pertuzumab within its marketing authorisation for the adjuvant treatment of human epidermal growth factor receptor 2 (HER2) positive breast cancer.

Lay abstract: Breast cancer is described as ‘early’ if it is restricted to the breast, or the breast and nearby lymph nodes, and has not spread to other parts of the body (that is at clinical stages 1 and 2). It is described as ‘locally advanced’ if the cancer is in a large part of the breast (more than 5 cm) but has not spread to other parts of the body (clinical stage 3), and described as ‘advanced’ if it has spread to other parts of the body and cannot be completely removed by surgery (clinical stage 4). Human epidermal growth factor receptor 2 (HER2) is a receptor for a growth factor which occurs naturally in the body. When human epidermal growth factor attaches itself to HER2 receptors on breast cancer cells, it can stimulate the cells to divide and grow. Some breast cancer cells have more HER2 receptors than others. In this case, the tumour is described as being HER2-positive.In 2014, there were approximately 46,500 new diagnoses of breast cancer in England (1). It is estimated that approximately 15-25% of women with breast cancer will have HER2-positive tumours. Men are less likely to have HER-2 positive breast cancers (2). NICE clinical guideline 80 (CG80) recommends that early breast cancer be treated with surgery (to remove the tumour) followed by adjuvant treatment to reduce the risk of the cancer coming back (recurrence). CG 80 recommends adjuvant chemotherapy with docetaxel containing regimen in patients with lymph node-positive breast cancer. It also recommends trastuzumab (a monoclonal antibody) as an adjuvant treatment for women with early-stage HER2-positive breast cancer following surgery. References 1. Cancer Research UK (2016) Breast cancer incidence (invasive) statistics. Accessed November 2017. 2. Macmillan (2015) HER-2 positive breast cancer. Accessed November 2017.

Research activity codes: 6.1 PHARMACEUTICALS
Health categories: CANCER AND NEOPLASMS
Title: Axicabtagene ciloleucel for treating relapsed or refractory diffuse large B-cell non-Hodgkin lymphoma [ID1115]
Funder: Department of Health and Social Care

Abstract: To appraise the clinical and cost effectiveness of axicabtagene ciloleucel within its marketing authorisation for treating relapsed or refractory diffuse large B-cell lymphoma, primary mediastinal large B-cell lymphoma or transformed follicular lymphoma.

Lay abstract: Lymphomas are cancers of the lymphatic system, which is a part of the immune system. Lymphomas are divided into Hodgkin lymphoma and non- Hodgkin lymphoma. Non-Hodgkin lymphomas (NHL) are a diverse group of conditions which are categorised according to the cell type affected (B-cell or T-cell), as well as the clinical features and rate of progression of the disease. The most common B-cell lymphomas are follicular lymphoma (FL) which is a slow growing, low grade form of NHL and diffuse large B-cell lymphomas (DLBCL), a fast growing (‘aggressive’), high grade form of NHL. Some FL will transform into high grade DLBCL (transformed high grade FL). Primary mediastinal large B-cell lymphoma (PMBCL) is a rare type of NHL which develops in the mediastinum. The symptoms differ depending on what organ or tissues the lymphoma is affecting. NHL often presents as painless lumps (enlarged lymph nodes) in the neck, armpit or groin but sometimes may start in other parts of the body such as the stomach or bowel (extranodal disease). People may also have loss of appetite, tiredness or night sweats. There were around 11,690 new cases of non-Hodgkin lymphoma (NHL) in England in 2015 with 6,322 of these DLBCL (1). Approximately 3% of lymphomas in the UK are PMBCL and 10-70% of low grade lymphomas transform into a high grade form (2),(3). Most people diagnosed with DLBCL are 65 or over (4). 5-year survival rates for DLBCL are around 65-70% for stage I and II and around 50% at stages III and IV(5). The most widely used first-line treatment for DLBCL (including transformed follicular lymphoma and primary mediastinal (thymic) large B-cell lymphoma), is R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone). Sometimes etoposide is added to this regimen. NICE guideline CG52 recommends salvage therapy with rituximab in combination with chemotherapy for relapsed or refractory disease followed by stem cell transplantation. If stem cell transplantation is not suitable chemotherapy or immunotherapy may be used alone. NICE TA306 recommends pixantrone monotherapy for people who have multiply relapsed, been treated previously with rituximab and are on the third or fourth line of treatment. References 1. Office for National Statistics. Cancer Registration Statistics, England, 2015. Office of National Statistics. Accessed November 2017. 2. High grade NHL. Cancer research UK. Accessed November 2017. 3. Low grade NHL. Cancer research UK. Accessed November 2017. 4. Diffuse B-cell lymphoma. Lymphoma association. Accessed November 2017. 5. Survival for high grade lymphomas. Cancer Research UK. Accessed November 2017.

Research activity codes: 6.1 PHARMACEUTICALS
Health categories: CANCER AND NEOPLASMS
Title: E014 - Communication skills training for healthcare professionals working with people who have cancer
Funder: Department of Health and Social Care

Abstract: By 2030 3.4 million people will be living with cancer in the UK. Effective communication benefits the well-being of cancer patients and health care professionals (HCP). Although the average rating of the overall cancer patient experience in the NHS is 8.74/10 (1), there is significant variation in how this is delivered across the country and internationally as well as between different population groups. The National Cancer Transformation Board recognizes patients’ concerns particularly about poor communication (2) and in their five year forward view proposes communication skills training (CST) courses can be effective in improving HCPs communication skills. Considerable effort and expense is being dedicated to review communications skills for NHS staff, and to CST within the NHS. However in our 2013 review update there was not enough evidence to conclude which types of CST are more effective, what the optimal length is for CST and whether the effects of CST are sustained over time. In our literature search this year we retrieved 103 articles that potentially fulfill our inclusion criteria. With new evidence available, our review update should help to clarify what kind of CST is most beneficial to patients and HCPs as well as demonstrating how cost-effective the approaches are.


Research activity codes: 8.1 ORGANISATION AND DELIVERY OF SERVICES
Health categories: CANCER AND NEOPLASMS
Title: Enzalutamide for treating non-metastatic hormone-relapsed prostate cancer [ID1359]
Funder: Department of Health and Social Care

Abstract: To appraise the clinical and cost effectiveness of enzalutamide within its marketing authorisation for treating non-metastatic hormone-relapsed prostate cancer.

Lay abstract: Prostate cancer is a condition in which tumours develop in the prostate, a gland in the male reproductive system. The exact cause is unknown but environmental and genetic factors are associated with an increased risk of developing prostate cancer.1,2 The incidence of prostate cancer increases with age and is higher in people of black African-Caribbean family origin and people with a family history of the condition.1 In England in 2016, about 40,500 people were diagnosed with prostate cancer3 and about 9,900 people died from the condition.4 Between 2015 to 2016, 84% of people diagnosed in England with prostate cancer had non-metastatic disease, that is, disease that has not spread to other parts of the body (for example, the bones).5 Non-metastatic disease includes localised prostate cancer, where the cancer is confined to the prostate, and locally advanced prostate cancer, where the cancer has spread to the area just outside the prostate. NICE clinical guideline 175 classifies localised prostate cancer to be at low, intermediate or high risk of progression based on prostate-specific antigenconcentration, Gleason score (based on a biopsy) and clinical stage. People with intermediate or high risk non-metastatic prostate cancer may be offered hormone therapy. Prostate cancer may initially respond to hormone therapybut eventually become resistant to it. This clinical condition is described as ‘hormone-relapsed’ prostate cancer, but the terms ‘castration-resistantprostate cancer’, ‘hormone-refractory prostate cancer’ and ‘androgenindependent prostate cancer’ are also used.a Hormone-relapsed prostatecancer is diagnosed by rising prostate-specific antigen levels. There are currently no licensed treatments for non-metastatic, hormonerelapsedprostate cancer. The main treatment is androgen deprivation therapywhich may include anti-androgens, such as, bicalutamide. Everyone ismonitored for evidence of disease metastasis, at which point, other treatmentsare considered. References 1. Cancer Research UK (2015) Prostate cancer risks and causes. Accessed July 2018. 2. Macmillan Cancer Support (2015) Potential causes of prostate cancer. Accessed July 2018. 3. Office for National Statistics (2018) Cancer registration statistics, England, 2016. Accessed July 2018. 4. Cancer Research UK (2018) Prostate cancer mortality statistics. Accessed July 2018. 5. National Prostate Cancer Audit (2017) Annual report 2017. Accessed July 2018.

Research activity codes: 6.1 PHARMACEUTICALS
Health categories: CANCER AND NEOPLASMS
Title: Cemiplimab for treating cutaneous squamous cell carcinoma [ID1367]
Funder: Department of Health and Social Care

Abstract: To appraise the clinical and cost effectiveness of cemiplimab within its marketing authorisation for treating cutaneous squamous cell carcinoma.

Lay abstract: Cutaneous squamous cell carcinoma (SCC) is a non-melanoma form of skin cancer that starts in the cells lining the top of the epidermis (the outer layer of the skin), their role is to produce keratin which protects the outer layer of the skin. The skin constantly sheds these cells.1 Cutaneous SCC presents itself on the surface of the skin as a firm pink lump with a rough surface and are tender to touch.1 There is a small risk (up to 5%) for cutaneous SCC lesions to spread but if they do, then they spread deeper into layers of the skin as well as metastasise across the body.2 Cutaneous SCC accounts for about 20% of skin cancers2 and 23% of non-melanoma skin cancers3. Around 122,000 cases of non-melanoma skin cancer were registered in 2015 in England.2 Deaths from cutaneous SCC are rare, however the prognosis for metastatic cutaneous SCC is poor, with a median overall survival of less than 2 years.4 Surgery is the main treatment for non-melanoma skin cancer. For locally advanced and metastatic disease, the European consensus-based interdisciplinary guideline4 suggests alternative treatment options if surgery is not feasible such as radiotherapy and chemotherapy (e.g. cisplatin, carboplatin, 5-fluorouracil and bleomycin). In addition, electrochemotherapy can be used for locally advanced lesions and EGFR inhibitors (e.g. cetuximab) can be considered as second line treatments after chemotherapy.4 The treatment used will depend on the type, size and location of the non-melanoma skin cancer.1 References 1. NHS Choices (2017) Non-melanoma skin cancer [accessed at 05/02/2018] https://www.nhs.uk/conditions/non-melanoma-skin-cancer/ 2. Cancer Research UK (2017) Skin cancer 3. National cancer Intelligence Network (2013) Non-melanoma skin cancer in England, Scotland, Northern Ireland, and Ireland 4. Stratigos A, Garbe C, Lebbe C et al. ‘Diagnosis and treatment of invasive squamous cell carcinoma of the skin: European consensus-based interdisciplinary guideline’. Eur J Cancer. (2015) Volume 51, Issue 14, Pages 1989–2007. http://www.eado.org/medias/Content/Files/2015-Stratigos-EurGuidelineSCC-EJC.pdf [Accessed at 24/08/2018]

Research activity codes: 6.1 PHARMACEUTICALS
Health categories: CANCER AND NEOPLASMS
Title: The ProtecT trial - evaluating the effectiveness of treatments for clinically localised prostate cancer
Funder: Department of Health and Social Care

Abstract: The overall aim is to evaluate the effectiveness, cost-effectiveness and acceptability of treatments for men with localised prostate cancer within the context of a pragmatic randomised controlled trial. This will compare 3 treatments (active monitoring, radical prostatectomy and radical radiotherapy). Specific objectives are as follows: 1) To assess survival at 5, 10 years and 15 years following treatment 2) To investigate a number of short and medium-term outcomes, including: disease progression (biochemical and clinical), treatment complications, lower urinary tract symptoms, psychosocial impact of case-finding and treatment, including generic health status, quality of life and sexual function 3) To estimate the resource use and costs of case-finding, treatment and follow-up, and to compare costs and outcomes of treatment in terms of survival and health related quality of life. 4) To collect material suitable for basic science research, and develop a network for collaborative research.


Research activity codes: 6.4 SURGERY;6.5 RADIOTHERAPY AND OTHER NON-INVASIVE THERAPIES
Health categories: CANCER AND NEOPLASMS
Title: Evaluating the feasibility and acceptability of early decongestive treatment for newly presenting breast cancer-related lymphoedema (BCRL) of the upper limb.
Funder: Health Education England

Abstract: The international recommendation is early decongestion for all breast cancer-related lymphoedema (BCRL) cases, chronic arm swelling, to reduce lymphoedema when it is most responsive to treatment. Current UK treatment for BCRL is self-management using compression hosiery. Decongestive lymphoedema treatment (DLT) is generally reserved for severe cases. The Fellowship will develop DLT for implementation at first presentation for women with BCRL. Stage 1&2 develops the intervention; stage 3 tests feasibility and acceptability of DLT via an exploratory trial. If feasible and acceptable, implementation of DLT for women newly-presenting with BCRL may improve health outcomes and well-being, and potentially reduce healthcare costs.

Lay abstract: The international recommendation is early decongestion for all breast cancer-related lymphoedema (BCRL) cases, chronic arm swelling, to reduce lymphoedema when it is most responsive to treatment. Current UK treatment for BCRL is self-management using compression hosiery. Decongestive lymphoedema treatment (DLT) is generally reserved for severe cases. The Fellowship will develop DLT for implementation at first presentation for women with BCRL. Stage 1&2 develops the intervention; stage 3 tests feasibility and acceptability of DLT via an exploratory trial. If feasible and acceptable, implementation of DLT for women newly-presenting with BCRL may improve health outcomes and well-being, and potentially reduce healthcare costs.

Research activity codes: 6.7 PHYSICAL
Health categories: CANCER AND NEOPLASMS
Title: Exploring the influence of social networks and their potential to enhance supported self management for cancer survivors
Funder: Health Education England

Abstract: People surviving cancer can have long term health needs after treatment. Self-management support is a way to help those with long term disabilities to look after themselves. This research aims to describe how social contacts can influence the way cancer survivors look after themselves at home. This will be achieved by sending a questionnaire to 200 people who have completed treatment in the last year. A sample of these individuals will also be interviewed. This information could be used by healthcare staff and charities to develop new ways to support cancer survivors by using resources in their communities.

Lay abstract: People surviving cancer can have long term health needs after treatment. Self-management support is a way to help those with long term disabilities to look after themselves. This research aims to describe how social contacts can influence the way cancer survivors look after themselves at home. This will be achieved by sending a questionnaire to 200 people who have completed treatment in the last year. A sample of these individuals will also be interviewed. This information could be used by healthcare staff and charities to develop new ways to support cancer survivors by using resources in their communities.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: CANCER AND NEOPLASMS
Title: Re-purposing of 13-cis-retinoic acid for children with neuroblastoma
Funder: Department of Health and Social Care

Abstract: Neuroblastoma is a rare and aggressive cancer that predominantly affects children aged 5 years or less. Children with neuroblastoma are commonly treated with a combination of therapies including a drug called 13-cis-retinoic acid (13-CRA). Treatment with 13-CRA improves survival chances of children with neuroblastoma and is standard therapy for this disease worldwide. Presently, 13-CRA is only available as a capsule. As few young children will take capsules, carers often have to administer 13-CRA by first extracting liquid out of the capsule. Studies show that this method of administration commonly results in children being given the wrong dose of drug which, in turn, increases risks of treatment failure and drug toxicity. Furthermore, since 13-CRA can cause birth defects, pregnant women (e.g. parent of a child with NBL) risk accidently exposing their unborn babies when drawing up 13-CRA from capsules. Despite requests by governmental agencies, no manufacturer has yet developed a child-friendly preparation of 13-CRA. This project aims to develop a fully tested and licensed oral liquid preparation of 13-CRA for use in children with neuroblastoma. This product will be available worldwide and addresses an unmet need for children with a disease that still has poor outcomes

Lay abstract: Neuroblastoma is a rare and aggressive cancer that predominantly affects children aged 5 years or less. Children with neuroblastoma are commonly treated with a combination of therapies including a drug called 13-cis-retinoic acid (13-CRA). Treatment with 13-CRA improves survival chances of children with neuroblastoma and is standard therapy for this disease worldwide. Presently, 13-CRA is only available as a capsule. As few young children will take capsules, carers often have to administer 13-CRA by first extracting liquid out of the capsule. Studies show that this method of administration commonly results in children being given the wrong dose of drug which, in turn, increases risks of treatment failure and drug toxicity. Furthermore, since 13-CRA can cause birth defects, pregnant women (e.g. parent of a child with NBL) risk accidently exposing their unborn babies when drawing up 13-CRA from capsules. Despite requests by governmental agencies, no manufacturer has yet developed a child-friendly preparation of 13-CRA. This project aims to develop a fully tested and licensed oral liquid preparation of 13-CRA for use in children with neuroblastoma. This product will be available worldwide and addresses an unmet need for children with a disease that still has poor outcomes

Research activity codes: 5.1 PHARMACEUTICALS;6.1 PHARMACEUTICALS
Health categories: CANCER AND NEOPLASMS
Title: The Application of Next generation Sequencing (NGS) to the Analysis of Minimal Residual Disease (MRD) in Childhood Acute Lymphoblastic Leukaemia (ALL)
Funder: Department of Health and Social Care

Abstract: There are approximately 400 new cases and 30 relapses of childhood ALL per year in the UK. Critical to the improved outcome of these children has been risk-stratification using a biomarker known as Minimal Residual Disease (MRD). Whilst extremely predictive of outcome, the current method for measuring MRD, (real time quantitative polymerase chain reaction based measurement of patient specific immunoglobulin and T-cell receptor gene rearrangements (Ig/TCR RQ-PCR)) is time-consuming, expensive, requires significant technical expertise, requires analysis of bone marrow samples and fails to give a result in almost 10% of cases. We are planning to develop the current technique for use on an Illumina MiSeq next generation sequencing (NGS) platform. The key goals of our project are to adapt the current PCR methodology so that reproducible and quantitative results are obtained on the Illumina platform, and to develop a novel bioinformatic pipeline that will enable interpretation of the 25 million sequencing reads that this technique generates but that can be easily used by NHS laboratory staff. In achieving these aims we will deliver a cheaper, faster, more sensitive test, with wider patient applicability, whilst also enabling us to further understand the mechanisms of disease relapse.

Lay abstract: There are approximately 400 new cases and 30 relapses of childhood ALL per year in the UK. Critical to the improved outcome of these children has been risk-stratification using a biomarker known as Minimal Residual Disease (MRD). Whilst extremely predictive of outcome, the current method for measuring MRD, (real time quantitative polymerase chain reaction based measurement of patient specific immunoglobulin and T-cell receptor gene rearrangements (Ig/TCR RQ-PCR)) is time-consuming, expensive, requires significant technical expertise, requires analysis of bone marrow samples and fails to give a result in almost 10% of cases. We are planning to develop the current technique for use on an Illumina MiSeq next generation sequencing (NGS) platform. The key goals of our project are to adapt the current PCR methodology so that reproducible and quantitative results are obtained on the Illumina platform, and to develop a novel bioinformatic pipeline that will enable interpretation of the 25 million sequencing reads that this technique generates but that can be easily used by NHS laboratory staff. In achieving these aims we will deliver a cheaper, faster, more sensitive test, with wider patient applicability, whilst also enabling us to further understand the mechanisms of disease relapse.

Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES
Health categories: CANCER AND NEOPLASMS