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Treating Family Members

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101. Nivolumab for previously treated non-squamous non-small-cell lung cancer

Recommendations 4 2 The technology 5 3 Evidence 6 4 Committee discussion 7 Nature of the condition 7 Clinical management of the condition 7 Comparators 8 Clinical effectiveness 8 Cost effectiveness 11 End-of-life considerations 20 Innovation 21 Cancer Drugs Fund 21 Overall conclusions 24 Summary of appraisal committee's key conclusions 25 5 Implementation 32 6 Appraisal committee members and NICE project team 33 Appraisal committee members 33 NICE project team 33 Nivolumab for previously treated non-squamous (...) Nivolumab for previously treated non-squamous non-small-cell lung cancer Niv Nivolumab for pre olumab for previously treated non- viously treated non- squamous non-small-cell lung cancer squamous non-small-cell lung cancer T echnology appraisal guidance Published: 1 November 2017 nice.org.uk/guidance/ta484 © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

102. Regorafenib for previously treated unresectable or metastatic gastrointestinal stromal tumours

Implementation 16 5 Appraisal committee members and NICE project team 17 Appraisal committee members 17 NICE project team 17 Regorafenib for previously treated unresectable or metastatic gastrointestinal stromal tumours (TA488) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 181 1 Recommendations Recommendations 1.1 Regorafenib is recommended as an option for treating unresectable or metastatic gastrointestinal stromal (...) and that these are managed on an individual basis to achieve a balance between the minimal effective dose and side effects that can be tolerated. The patient experts explained that regorafenib allows people to Regorafenib for previously treated unresectable or metastatic gastrointestinal stromal tumours (TA488) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 7 of 18remain actively engaged in work and family life, while reducing anxiety

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

103. Cladribine tablets for treating relapsing?remitting multiple sclerosis

-effectiveness results and conclusion 18 4 Implementation 20 5 Appraisal committee members and NICE project team 21 Appraisal committee members 21 NICE project team 21 Cladribine tablets for treating relapsing–remitting multiple sclerosis (TA493) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 221 1 Recommendations Recommendations 1.1 Cladribine tablets are recommended as an option for treating highly active multiple (...) Cladribine tablets for treating relapsing?remitting multiple sclerosis Cladribine tablets for treating Cladribine tablets for treating relapsing–remitting multiple sclerosis relapsing–remitting multiple sclerosis T echnology appraisal guidance Published: 6 December 2017 nice.org.uk/guidance/ta493 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

104. Ibrutinib for treating Waldenstrom?s macroglobulinaemia

discussion 7 Clinical effectiveness 7 Cost effectiveness 11 Potential equality issues 15 Pharmaceutical Price Regulation Scheme (PPRS) 2014 15 Summary of appraisal committee's key conclusions 16 5 Implementation 22 6 Recommendation for data collection 23 7 Appraisal committee members and NICE project team 24 Appraisal committee members 24 NICE project team 24 Ibrutinib for treating Waldenstrom’s macroglobulinaemia (TA491) © NICE 2018. All rights reserved. Subject to Notice of rights (https (...) Ibrutinib for treating Waldenstrom?s macroglobulinaemia Ibrutinib for treating W Ibrutinib for treating Waldenstrom aldenstrom’s ’s macroglobulinaemia macroglobulinaemia T echnology appraisal guidance Published: 22 November 2017 nice.org.uk/guidance/ta491 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE, arrived

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

105. Asfotase alfa for treating paediatric-onset hypophosphatasia

are likely to spend many days in hospital with their child, which reduces time with other family members and results in time away from work (or stopping work entirely). The daily lives of carers are affected because of the child's seizures and the need to regularly monitor oxygen levels. Patient experts highlighted that because of the limited numbers of centres treating hypophosphatasia in England, long journeys for appointments or inpatient stays may be needed regularly. This sometimes leads to families (...) Conclusion 43 Summary of evaluation committee's key conclusions 44 6 Implementation 52 7 Evaluation committee members and NICE project team 53 Evaluation committee members 53 NICE project team 53 Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6) © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 541 1 Recommendations Recommendations 1.1 Asfotase alfa is recommended as an option for treating paediatric

2017 National Institute for Health and Clinical Excellence - Highly specialised technology

106. Certolizumab pegol and secukinumab for treating active psoriatic arthritis after inadequate response to DMARDs

Certolizumab pegol and secukinumab for treating active psoriatic arthritis after inadequate response to DMARDs Certolizumab pegol and secukinumab for Certolizumab pegol and secukinumab for treating activ treating active psoriatic arthritis after e psoriatic arthritis after inadequate response to DMARDs inadequate response to DMARDs T echnology appraisal guidance Published: 24 May 2017 nice.org.uk/guidance/ta445 © NICE 2018. All rights reserved. Subject to Notice of rights (https (...) to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities. Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible. Certolizumab pegol and secukinumab for treating active psoriatic arthritis after inadequate response to DMARDs (TA445) © NICE 2018. All rights

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

107. Olaratumab in combination with doxorubicin for treating advanced soft tissue sarcoma

project team 19 Appraisal committee members 19 NICE project team 19 Olaratumab in combination with doxorubicin for treating advanced soft tissue sarcoma (TA465) © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 201 1 Recommendations Recommendations 1.1 Olaratumab, in combination with doxorubicin, is recommended for use within the Cancer Drugs Fund as an option for advanced soft tissue sarcoma in adults, only (...) in combination with doxorubicin for treating advanced soft tissue sarcoma (TA465) © NICE 2019. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 18 of 206 6 Appr Appraisal committee members and NICE project team aisal committee members and NICE project team Appraisal committee members The technology appraisal committees are standing advisory committees of NICE. This topic was considered by members of the existing standing committees who

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

108. Bisphosphonates for treating osteoporosis

committee members and NICE project team 24 Appraisal committee members 24 NICE project team 24 Bisphosphonates for treating osteoporosis (TA464) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 25This guidance partially replaces TA160 and TA161. 1 1 Recommendations Recommendations The purpose of this technology appraisal was to establish at what level of absolute fracture risk bisphosphonates are cost-effective (...) a decision support aid to help support this discussion. If the person wishes to try a bisphosphonate, they should be able to start treatment with the least expensive formulation, with no other local formulary or funding restrictions. Bisphosphonates for treating osteoporosis (TA464) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 23 of 255 5 Appr Appraisal committee members and NICE project team aisal committee members

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

109. Cabozantinib for previously treated advanced renal cell carcinoma

Evidence 6 4 Committee discussion 7 Treatment pathway 7 Population and comparators 8 Clinical effectiveness 9 Cost effectiveness 11 End-of-life considerations 17 Results of cost-effectiveness analyses 17 Innovation 19 Pharmaceutical Price Regulation Scheme (PPRS) 2014 19 Summary of appraisal committee's key conclusions 19 5 Implementation 25 6 Appraisal committee members and NICE project team 26 Appraisal committee members 26 NICE project team 26 Cabozantinib for previously treated advanced renal cell (...) curves for all the treatments. However, the committee agreed that it was a more flexible family, which improved the curve fits to the Kaplan–Meier Cabozantinib for previously treated advanced renal cell carcinoma (TA463) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 10 of 27data on overall and progression-free survival for all treatments in the network compared with the original parametric modelling using the log

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

110. Adalimumab and dexamethasone for treating non-infectious uveitis

Evidence 7 4 Committee discussion 8 Clinical need and management of non-infectious uveitis 8 Clinical effectiveness 9 Cost effectiveness 12 Summary of appraisal committee's key conclusions 19 5 Implementation 27 6 Appraisal committee members and NICE project team 28 Appraisal committee members 28 NICE project team 28 Adalimumab and dexamethasone for treating non-infectious uveitis (TA460) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice (...) Adalimumab and dexamethasone for treating non-infectious uveitis Adalimumab and de Adalimumab and dexamethasone for xamethasone for treating non-infectious uv treating non-infectious uveitis eitis T echnology appraisal guidance Published: 26 July 2017 nice.org.uk/guidance/ta460 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

111. Trastuzumab emtansine for treating HER2-positive advanced breast cancer after trastuzumab and a taxane

committee members and NICE project team 30 Appraisal committee members 30 NICE project team 30 Update information 32 Trastuzumab emtansine for treating HER2-positive advanced breast cancer after trastuzumab and a taxane (TA458) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 32This guidance replaces TA371. 1 1 Recommendations Recommendations 1.1 Trastuzumab emtansine is recommended, within its marketing (...) Trastuzumab emtansine for treating HER2-positive advanced breast cancer after trastuzumab and a taxane T T r rastuzumab emtansine for treating astuzumab emtansine for treating HER2-positiv HER2-positive advanced breast cancer e advanced breast cancer after tr after trastuzumab and a taxane astuzumab and a taxane T echnology appraisal guidance Published: 19 July 2017 nice.org.uk/guidance/ta458 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

112. Carfilzomib for previously treated multiple myeloma

problem and treatment pathway 8 Clinical effectiveness 11 Cost effectiveness 14 Most plausible incremental cost-effectiveness ratio 17 End-of-life considerations 19 Conclusion 19 Summary of appraisal committee's key conclusions 20 5 Implementation 27 6 Appraisal committee members and NICE project team 28 Appraisal committee members 28 NICE project team 28 Carfilzomib for previously treated multiple myeloma (TA457) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk (...) treated multiple myeloma (TA457) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 27 of 296 6 Appr Appraisal committee members and NICE project team aisal committee members and NICE project team Appraisal committee members The 4 technology appraisal committees are standing advisory committees of NICE. This topic was considered by committee C. Committee members are asked to declare any interests in the technology

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

113. Migalastat for treating Fabry disease

members 31 NICE project team 31 Migalastat for treating Fabry disease (HST4) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 311 1 Recommendations Recommendations 1.1 Migalastat is recommended, within its marketing authorisation, as an option for treating Fabry disease in people over 16 years of age with an amenable mutation, only if migalastat is provided with the discount agreed in the patient access scheme (...) is taken on by family members. Many people with Fabry disease have had psychological difficulties coming to terms with a lifelong progressive disorder, particularly before the introduction of enzyme replacement therapy (ERT) in 2001. ERT has a number of benefits but it also has limitations. The infusion dosage schedule of every 2 weeks means that people with Fabry disease cannot plan trips away from home. ERT must be kept refrigerated and there are risks of developing an infusion- related infection

2017 National Institute for Health and Clinical Excellence - Highly specialised technology

114. Apremilast for treating active psoriatic arthritis

Clinical effectiveness 9 Cost effectiveness 11 Rapid review 18 Summary of appraisal committee's key conclusions 22 5 Implementation 29 6 Appraisal committee members and NICE project team 30 Appraisal committee members 30 NICE project team 30 Apremilast for treating active psoriatic arthritis (TA433) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 31This guidance replaces TA372. 1 1 Recommendations Recommendations (...) Apremilast for treating active psoriatic arthritis Apremilast for treating activ Apremilast for treating active psoriatic e psoriatic arthritis arthritis T echnology appraisal guidance Published: 22 February 2017 nice.org.uk/guidance/ta433 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE, arrived at after careful

2017 National Institute for Health and Clinical Excellence - Technology Appraisals

115. Budesonide (Jorveza) - to treat adults with eosinophilic oesophagitis

Budesonide (Jorveza) - to treat adults with eosinophilic oesophagitis 30 Churchill Place ? Canary Wharf ? London E14 5EU ? United Kingdom An agency of the European Union Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5520 Send a question via our website www.ema.europa.eu/contact © European Medicines Agency, 2018. Reproduction is authorised provided the source is acknowledged. 09 November 2017 EMA/774645/2017 Assessment report Jorveza International non-proprietary name: budesonide (...) analysis set-double blind (phase) FAS-FU Full analysis set-follow-up (phase) FAS-OLI Full analysis set-open-label induction (phase) FU Follow-up GC Gas Chromatography GERD Gastroesophageal reflux disease hpf High-power field HPLC High performance liquid chromatography ICH International Council for Harmonisation IDMC Independent Data Monitoring Committee IgE Immunoglobulin E IL Interleukin IMP Investigational medicinal product IR Infrared ITT Intention-to-treat LOCF Last observation carried forward LC

2018 European Medicines Agency - EPARs

116. Treating Family Members

Treating Family Members Treating Family Members Toggle navigation Brain Head & Neck Chest Endocrine Abdomen Musculoskeletal Skin Infectious Disease Hematology & Oncology Cohorts Diagnostics Emergency Findings Procedures Prevention & Management Pharmacy Resuscitation Trauma Emergency Procedures Ultrasound Cardiovascular Emergencies Lung Emergencies Infectious Disease Pediatrics Neurologic Emergencies Skin Exposure Miscellaneous Abuse Cancer Administration 4 Treating Family Members Treating (...) Family Members Aka: Treating Family Members II. Precautions Do not write controlled substance prescriptions (e.g. s, s) for family or friends Home treatment of minor symptoms is reasonable However, major symptoms are best treated with formal evaluations Rendered care may be sub-standard of the care you would deliver to others Clinician may not ask their family member sensitive questions Clinician may stretch their care beyond their level of expertise Family members may have misconceptions

2016 FP Notebook

117. Preferences of Current and Potential Patients and Family Members Regarding Implementation of Electronic Communication Portals in Intensive Care Units. (Abstract)

members of patients currently admitted to ICUs at an academic medical center in Boston (cohort C).Respondents, especially current ICU family members, supported an electronic communication portal, including access via an electronic tablet. They wanted at least daily updates, one-paragraph summaries of family meetings including a list of key decisions made, and knowledge of the role and experience of treating clinicians. Overall, they preferred detailed rather than "big picture" information. Respondents (...) Preferences of Current and Potential Patients and Family Members Regarding Implementation of Electronic Communication Portals in Intensive Care Units. The quality of communication with patients and family members in intensive care units (ICUs) is a focus of current interest for clinical care improvement. Electronic communication portals are commonly used in other healthcare settings to improve communication. We do not know whether patients and family members desire such portals in ICUs

2015 Annals of the American Thoracic Society

118. Clinical Presentation, Long-Term Follow-Up, and Outcomes of 1001 Arrhythmogenic Right Ventricular Dysplasia/Cardiomyopathy Patients and Family Members. Full Text available with Trip Pro

from a cardiac cause (2% versus 0%) than family members without mutations.Long-term outcome was favorable in diagnosed and treated ARVD/C index-patients and family members. Outcome in index-patients was modulated by implantable cardioverter-defibrillator implantation, but not by mutation status and familial background of disease. One third of family members developed ARVD/C. Outcome in family members was determined by symptoms at first evaluation and mutations.© 2015 American Heart Association, Inc. (...) Clinical Presentation, Long-Term Follow-Up, and Outcomes of 1001 Arrhythmogenic Right Ventricular Dysplasia/Cardiomyopathy Patients and Family Members. Arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) is a progressive cardiomyopathy. We aimed to define long-term outcome in a transatlantic cohort of 1001 individuals.Clinical and genetic characteristics and follow-up data of ARVD/C index-patients (n=439, fulfilling of 2010 criteria in all) and family members (n=562) were

2015 Circulation. Cardiovascular genetics

119. Autoantibodies binding to ubiquitin-fold modifier-conjugating enzyme 1 (Ufc1) and pleckstrin homology domain containing, family G (with RhoGef domain) member 2 (Plekhg2) are associated with mycobacterial infections. Full Text available with Trip Pro

Autoantibodies binding to ubiquitin-fold modifier-conjugating enzyme 1 (Ufc1) and pleckstrin homology domain containing, family G (with RhoGef domain) member 2 (Plekhg2) are associated with mycobacterial infections. The diagnosis of extrapulmonary tuberculous infections and nontuberculous mycobacterial (NTM) infections is difficult because the symptoms are nonspecific and suitable specimens for bacterial culture are often not available. Recent publications reported the existence (...) of autoantibodies in tuberculous infections. We screened for specific autoantibodies in mycobacterial infections.We screened four in 29 patients with active mycobacterial infections and different controls using protein array technology. We could identify autoantibodies against ubiquitin-fold modifier-conjugating enzyme 1 (Ufc1) and pleckstrin homology domain containing, family G (with RhoGef domain) member 2 (Plekhg2) in all four patients. Subsequently, we designed enzyme-linked immunosorbent assays (ELISAs

2015 HIV medicine

120. Psychiatric Neurosurgery (PNS) for Obsessive Compulsive Disorder (OCD): A Qualitative Analysis of Patient and Family Member Experiences

Electrical stimulation of the anterior limb of the internal capsule or bed nucleus of the stria terminalis Deep Brain Stimulation Family Members Family members of patients who have previously been treated with a deep brain stimulation procedure for obsessive compulsive disorder will be included in this group Anterior Capsulotomy Patients Patients who have previously been treated with an anterior capsulotomy procedure for obsessive compulsive disorder will be included in this group Procedure: anterior (...) capsulotomy Anterior Capsulotomy Family Members Family members of patients who have previously been treated with an anterior capsulotomy procedure for obsessive compulsive disorder will be included in this group Outcome Measures Go to Primary Outcome Measures : Semi-structured Interview [ Time Frame: up to 180 months after surgery ] A semi-structured interview of patients and one of their family members will be conducted. Patients and family members will be interviewed separately. The interview

2015 Clinical Trials

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