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1823. Voretigene neparvovec for biallelic RPE65-mediated retinal disease: effectiveness and value : final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, February 14, 2018
Subject(s):
Genetic Therapy
Leber Congenital Amaurosis -- genetics
Leber Congenital Amaurosis -- therapy
Retinal Diseases -- genetics
Retinal Diseases -- therapy
Retinal Pigment Epithelium -- abnormalities
Mutation
Randomized Controlled Trials as Topic
Treatment Outcome
Visual Acuity
Humans
United States

1824. Chimeric antigen receptor T-cell therapy for B-cell cancers: effectiveness and value : final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, March 23, 2018
Subject(s):
Immunotherapy, Adoptive
Leukemia, B-Cell -- drug therapy
Lymphoma, B-Cell -- drug therapy
Lymphoma, Non-Hodgkin -- drug therapy
Precursor Cell Lymphoblastic Leukemia-Lymphoma -- drug therapy
Adult
Child
Clinical Trials as Topic
Clofarabine -- therapeutic use
Cost-Benefit Analysis
Immunotherapy, Adoptive -- adverse effects
Immunotherapy, Adoptive -- economics
Receptors, Chimeric Antigen -- therapeutic use
Treatment Outcome
Humans
United States

1825. Emicizumab for hemophilia A with inhibitors: effectiveness and value : final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, April 16, 2018
Subject(s):
Antibodies, Bispecific -- therapeutic use
Antibodies, Monoclonal -- therapeutic use
Hemophilia A -- drug therapy
Antibodies, Bispecific -- adverse effects
Antibodies, Monoclonal -- adverse effects
Cost-Benefit Analysis
Treatment Outcome
Humans
United States

1826. Modulator treatments for cystic fibrosis: effectiveness and value : final evidence report and meeting summary

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, June 7, 2018
Subject(s):
Cystic Fibrosis -- drug therapy
Cystic Fibrosis Transmembrane Conductance Regulator -- therapeutic use
Clinical Trials as Topic
Cost of Illness
Cost-Benefit Analysis
Cystic Fibrosis Transmembrane Conductance Regulator -- adverse effects
Treatment Outcome
Humans
United States

1827. Calcitonin gene-related peptide (CGRP) inhibitors as preventive treatments for patients with episodic or chronic migraine: effectiveness and value : final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, July 3, 2018
Subject(s):
Calcitonin Gene-Related Peptide Receptor Antagonists -- therapeutic use
Migraine Disorders -- drug therapy
Migraine Disorders -- prevention & control
Calcitonin Gene-Related Peptide Receptor Antagonists -- adverse effects
Chronic Disease
Clinical Trials as Topic
Treatment Outcome
Humans
United States

1828. Elagolix for treating endometriosis: final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, August 3, 2018
Subject(s):
Endometriosis -- drug therapy
Gonadotropin-Releasing Hormone -- antagonists & inhibitors
Analgesics -- adverse effects
Analgesics -- therapeutic use
Cost-Benefit Analysis
Pain -- drug therapy
Randomized Controlled Trials as Topic
Treatment Outcome
Humans
United States

1829. Targeted immunomodulators for the treatment of moderate-to-severe plaque psoriasis: effectiveness and value : condition update : final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, August 3, 2018
Subject(s):
Immunologic Factors -- therapeutic use
Immunomodulation
Psoriasis -- drug therapy
Certolizumab Pegol -- therapeutic use
Clinical Trials as Topic
Cost-Benefit Analysis
Immunologic Factors -- adverse effects
Immunologic Factors -- economics
Quality of Life
Treatment Outcome
Humans
United States

1830. Inotersen and patisiran for hereditary transthyretin amyloidosis: effectiveness and value : final evidence report

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, October 4, 2018
Subject(s):
Amyloidosis, Familial -- drug therapy
RNA, Small Interfering -- therapeutic use
Amyloid Neuropathies, Familial -- drug therapy
Amyloidosis, Familial -- complications
Clinical Trials as Topic
Cost-Benefit Analysis
RNA, Small Interfering -- adverse effects
RNA, Small Interfering -- economics
Treatment Outcome
Humans
United States