Alpha-1-antitrypsin deficiency
(pulmonary disease or lung disease) Patients with congenital alpha-1 antitrypsin deficiency Plasma alpha-1 proteinase inhibitor Traditional Alpha-1 protease inhibitor augmentation Acid sphingomyelinase deficiency non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) assessment of % predicted Dlco and spleen volume Traditional hydrolytic lysosomal sphingomyelin-specific enzyme Acromegaly Patients with acromegaly who don't respond to or cannot undergo other standard therapies Serum Insulin-like growth factor-I (IGF-1) Traditional Growth hormone receptor antagonist Acromegaly  Patients with acromegaly who don't respond to or cannot undergo other standard therapies lk Traditional Somatostatin analog Activated PI3 kinase delta syndrome activated phosphoinositide 3-kinase delta (PI3K delta) syndrome (APDS) Co Primary: improvement in lymphoproliferation as measured by a change from baseline in lymphadenopathy measured by the log10-transformed sum of product diameters and the normalization of immunophenotype as measured by the percentage of naïve B cells out of total B cells Traditional kinase inhibitor - phosphatidylinositide 3-kinase d (PI3K-d) inhibitor Acute Bronchospasm Patients with acute bronchospasm associated with reversible obstructive airway disease or exercise Forced expiratory volume in 1 second (FEV1) Traditional  Beta-2 adrenergic agonist Alzheimer's disease Patients with mild cognitive impairment or mild dementia stage of Alzheimer's disease  Reduction in amyloid beta plaques Accelerated Monoclonal antibody Amyotrophic lateral sclerosis (ALS) Adults who have ALS with a mutation in the superoxide dismutase 1 (SOD1) gene Change  in plasma neurofilament light chain (NfL) Accelerated  antisense oligonucleotide Anemia Pateints with anemia due to chronic kidney disease (CKD)  mean change in Hb Traditional^† hypoxia-inducible factor prolyl hydroxylase (HIF PH) inhibitor Anthrax vaccine Persons at high risk of exposure to anthrax Anti-protective antigen antibody response Traditional Induction of immunity Anticoagulation reversal (needed due to life-threatening or uncontrolled bleeding) Patients treated with a direct or indirect FXa inhibitor when reversal of anticoagulation is needed Percent change in anti-FXa activity, from baseline to nadir Accelerated Binding and sequestering FXa inhibitors Asthma Patients with asthma Forced expiratory volume in 1 second (FEV1)   Traditional  Corticosteroid; Beta-2 adrenergic agonist The prevention of disease caused by chikungunya virus (CHIKV) in individuals 18 years of age and older who are at increased risk of exposure to CHIKV 18 years of age and older who are at increased risk of exposure to CHIKV CHIKV-specific neutralizing antibody titer ≥150 as determined by micro-plaque reduction neutralization test (μPRNT50) Accelerated IXCHIQ elicits CHIKV-specific immune responses. The exact mechanism of protection has not been determined but protection is thought to be mediated by CHIKV-specific neutralizing antibodies. Chronic kidney disease  Patients with chronic kidney disease secondary to multiple etiologies  Estimated glomerular filtration rate or serum creatinine  Traditional Mechanism agnostic* Chronic obstructive pulmonary disease (COPD) Patients with COPD Forced expiratory volume in 1 second (FEV1) Traditional  Corticosteroid; Long-acting beta2-adrenergic agonist; Anticholinergic; Phosphodiesterase 4 inhibitor Chronic graft versus host disease Patient with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy overall response rate (ORR) Traditional kinase inhibitor - Bruton's tyrosine kinase (BTK) Cushing's disease  Patients with Cushing’s disease for whom pituitary surgery is not an option or has not been curative  Urine free cortisol   Traditional Somatostatin analog  Cushing's syndrome Patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery Urine free cortisol ˟   Traditional   Cortisol synthesis inhibitor  Cystic fibrosis Patients with cystic fibrosis Forced expiratory volume in 1 second (FEV1) Traditional  Cystic fibrosis transmembrane conductance regulator potentiator Cystinuria Patients with cystinuria Urinary cystine Traditional Reducing and complexing thiol Cytomegalovirus (CMV)  CMV seropositive and hematopoietic transplant recipients requiring prophylaxis   Plasma CMV-DNA exceeding threshold for starting treatment 
   Traditional Antiviral Diphtheria vaccine (in combination vaccines) Persons to be immunized against diphtheria  Anti-diphtheria toxoid antibody  Traditional Induction of immunity Duchenne muscular dystrophy (DMD) Patients with DMD who have a confirmed mutation of the DMD gene that is amenable to exon skipping Skeletal muscle dystrophin Accelerated Antisense oligonucleotide Exocrine pancreatic insufficiency Patients with exocrine pancreatic insufficiency due to cystic fibrosis, chronic pancreatitis, pancreatectomy, or other conditions Fecal coefficient of fat absorption  Traditional Pancreatic enzymes that catalyze the hydrolysis of fats, proteins, and starches.   Extravascular hemolysis extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) Change in hemoglobin (Hgb)  Traditional^† Complement factor D inhibitor Fabry disease Patients with confirmed Fabry disease Complete/near complete clearance of GL-3 inclusions in biopsied renal peritubular capillaries (using the Fabrazyme Scoring System) Accelerated Enzyme replacement therapy  Fabry disease Patients with confirmed Fabry disease and amenable GLA gene variants Reduction of GL-3 inclusions in biopsied renal peritubular capillaries (using the BLISS methodology) Accelerated Pharmacological chaperone Fabry disease Patients with Fabry disease Sustained treatment effect on loss of renal function across the various stages of renal disease Traditional Hydrolytic lysosomal neutral glycosphingolipid-specific enzyme Female hypogonadotropic
  hypogonadism Infertile women with hypogonadotropic hypogonadism Follicle size, serum estradiol and progesterone# Traditional Gonadotropin First aid antiseptic; Health care antiseptic; Consumer antiseptic General public, consumers, and health care professionals Bacterial count Traditional and Monograph    Antimicrobial  Geographic atrophy geographic atrophy (GA) secondary to age-related macular degeneration (AMD) mean rate of geographic atrophy (GA) growth  Traditional complement inhibitor Gout Patients with gout Serum uric acid Traditional  Xanthine oxidase inhibitor; URAT1 inhibitor; Uricase Helicobacter pylori infection Helicobacter pylori (H. pylori) infection Successful H. pylori eradication  Traditional potassium-competitive acid blocker (PCAB) / penicillin class antibacterial  Hepatitis A (Hep A) vaccine Persons to be immunized against Hep A  Anti-Hep A antigen antibody   Traditional Induction of immunity Hepatitis B (Hep B) vaccine Persons to be immunized against Hep B Plasma hepatitis B virus (HBV) DNA  Traditional Induction of immunity Hepatitis B Virus (all known subtypes) vaccine Adults > 18 yo  to be immunized against  all known suptypes of hepatitis b virus Neutralizing antibody >/= 10 mIU/mL Traditional Induction of antibodies to HBsAg. Hepatitis B Virus (HBV) Patients with HBV infection with or without cirrhosis  Undetectable plasma HBV-DNA for indefinite treatment or HBsAg loss for finite treatment Traditional  Antiviral Hepatitis C Virus (HCV) Patients with HCV infection with or without cirrhosis Sustained viral response (HCV-RNA)  Traditional  Antiviral  Hepatitis D Virus (HDV) Patients with HDV infection with or without cirrhosis ≥ 2 log reduction in HDV-RNA plus normalization of ALT or HDV below the LLOQ˟ Accelerated Antiviral Hepatorenal syndrome  Patients with hepatorenal syndrome type 1 Serum creatinine˟ Traditional  Mechanism agnostic* Heterotopic ossification patients with fibrodysplasia ossificans progressiva (FOP) annualized change in new heterotopic ossification (HO) volume Traditional Retinoid Histiocytosis patients with histiocytic neoplasms overall response rate (ORR) Traditional Kinase inhibitor - mitogen-activated protein kinase (MEK) inhibitor Homozygous sitosterolemia (phytosterolemia) Patients with homozygous sitosterolemia (phytosterolemia) Plasma sitosterol and campesterol Traditional Dietary cholesterol absorption inhibitor Human Immunodeficiency Virus-1 (HIV-1) Patients with HIV-1 Undetectable plasma HIV RNA Traditional Antiviral  Human Immunodeficiency Virus-1 (HIV-1) Patients at high risk of sexually acquired HIV-1  Serum HIV antibody  Traditional Antiviral  Human Immunodeficiency Virus-1 (HIV-1) Highly treatment-experienced  HIV-1 patients  Greater than 0.5 log reduction in plasma HIV RNA Traditional Antiviral  Human Papillomavirus Persons (18 through 45 years of age) to be immunized against human papillomavirus Cervical intraepithelial neoplasia Traditional Induction of immunity Hypercholesterolemia Patients with heterozygous familial and nonfamilial hypercholesterolemia Serum LDL cholesterol Traditional Lipid-lowering Hypercholesterolemia  Patients with homozygous familial hypercholesterolemia Serum LDL cholesterol Traditional Lipid-lowering  Hyperkalemia  Patients with hyperkalemia Serum potassium Traditional Potassium binder Hyperphosphatemia Dialysis patients with hyperphosphatemia  Serum phosphate  Traditional Phosphate binder Hypertension  Patients with hypertension  Blood pressure Traditional Mechanism agnostic* Hypertriglyceridemia Patients with severe hypertriglyceridemia Serum triglycerides Traditional  Lipid-lowering Hypokalemia  Patients with hypokalemia Serum potassium  Traditional Potassium salts Hyponatremia  Patients with hypervolemic and euvolemic hyponatremia Serum sodium Traditional Vasopressin receptor antagonist Hypophosphatemia X-linked hypophosphatemia (XLH) Serum Phosphorus Concentration Traditional^† fibroblast growth factor 23 (FGF23) blocking antibody  Hypotension Patients with distributive shock Blood pressure Traditional Alpha  and beta adrenergic agonist; Vasopressin analog Hypothyroidism Patients with hypothyroidism Serum thyroid-stimulating hormone (TSH) Traditional Thyroid hormone analog  Influenza A H5N1 vaccine Persons to be immunized against influenza Hemagglutination inhibition antibody Traditional Induction of Immunity Influenza vaccine Persons to be immunized against influenza   Hemagglutination inhibition antibody  Accelerated Induction of immunity Interoperative hemorrhage Patients who require reduction of blood pressure to reduce bleeding during surgery Blood pressure Traditional Vasodilator Invasive pneumococcal disease  Patients with invasive pneumococcal disease  Opsonophagocytosis assay titers Traditional Induction of immunity Japanese encephalitis vaccine Persons to be immunized against Japanese encephalitis  Neutralizing antibody  Traditional Induction of immunity Lipodystrophy Patients with congenital or acquired generalized lipodystrophy Serum hemoglobin A1C, fasting glucose and triglycerides  Traditional  Leptin analog Lupus nephritis Patients with active lupus nephritis Complete renal response (CRR), defined as 1) a response in the urine proteinuria (protein-creatine ratio) and 2) preservation/improvement of renal function (estimated glomerular filtration rate)    Traditional  Immunosuppressant Lysosomal Acid Lipase (LAL) deficiency Patients with LAL deficiency Serum LDL-c levels Traditional Hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme Male hypogonadotropic hypogonadism with inferility Men with selected cases of hypogonadotropic hypogonadism with inferility Sperm parameters Traditional Gonadotropin Meningococcal (serogroups A, C, Y, W) meningitis vaccine Persons to be immunized against meningococcal meningitis Serum bactericidal antibody Traditional Induction of Immunity Meningococcal ACYW-135 vaccine Persons to be immunized against meningococcal meningitis Serum bactericidal antibody  Traditional Induction of immunity Meningococcal Group B vaccine Persons (18 through 25 years of age) to be immunized against meningococcal meningitis Serum bactericidal antibody  Traditional Induction of immunity Methylmalonic acidemia Patients with acute hyperammonemia due to methylmalonic acidemia Plasma ammonia  Traditional Carbamoyl Phosphate Synthetase 1 activator To mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma Subsequent autologous transplantation in patients with multiple myeloma Proportion of patients who achieve a cell collection goal of ≥ 6 × 106 CD34+ cells/kg Traditional Inhibitor of the C-X-C Motif Chemokine Receptor 4 (CXCR4) Monkeypox vaccine Persons to be immunized against monkeypox Vaccinia-neutralizing antibody Traditional Induction of immunity Mycobacterium avium complex (MAC) lung disease  Patients with MAC lung disease  Sputum culture conversion to negative by six months  Accelerated Antimicrobial  Myelodysplastic syndrome Patients with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation  Complete remission (CR) or partial remission (PR) Traditional Isocitrate dehydrogenase-1 (IDH1) inhibitor Myelofibrosis Myelofibrosis (MF) in adults with anemia Splenic volume response Traditional^† Kinase inhibitor - multi-kinase inhibitor (JAK1, JAK2, ACVR1/ALK2) N-acetylglutamate Synthase (NAGS) deficiency Patients with hyperammonemia due to NAGS deficiency Plasma ammonia  Traditional Carbamoyl Phosphate Synthetase 1 activator Nonalcoholic steatohepatitis (NASH) / metabolic dysfunction associated steatohepatitis (MASH) Precirrhotic NASH patients with liver fibrosis Histopathologic findings of either 1) resolution of steatohepatitis with no worsening of fibrosis OR 2) improvement of fibrosis with no worsening of steatohepatitis OR 3) Both# Accelerated Anti-fibrotic; Anti-inflammatory Nonmalignant hematology Patients with Thrombocytopenia due to immune (idiopathic) thrombocytopenia or chronic hepatitis C Platelet count response Traditional  Mechanism agnostic* Nonmalignant hematology Patients with chronic iron overload or non-transfusion-dependent thalassemia syndromes Serum ferritin and liver iron concentration  Traditional Iron chelator Nonmalignant hematology Patients with anemia due to (1) chronic kidney disease, (2) chemotherapy-induced anemia, (3) zidoviduine in patients with HIV-infection Hematologic response and reduction in transfusion Traditional Mechanism agnostic* Nonmalignant hematology Patients with severe aplastic anemia   Hematologic response  Traditional Mechanism agnostic* Nonmalignant hematology Patients with methemoglobinemia Serum methemoglobin Accelerated Oxidation-reduction agent Nonmalignant hematology Patients in need of reversal of anticoagulant effects for emergency surgery/urgent procedures and in life-threatening or uncontrolled bleeding. Change in coagulation parameters Traditional Humanized monoclonal antibody fragment Nonmalignant hematology Patients with sickle cell disease Hemoglobin response rate Accelerated Hemoglobin S polymerization inhibitor Ocular hypertension Ocular hypertension Mean intraocular pressure (IOP) Traditional Relatively selective prostaglandin E2 (EP2) receptor agonist Open angle glaucoma Open-angle glaucoma  Mean intraocular pressure (IOP) Traditional Relatively selective prostaglandin E2 (EP2) receptor agonist Opioid use disorder Patients with opioid use disorder Urine toxicology test for opioids Traditional 1. Partial opioid agonist Osteoporosis Postmenopausal women with osteoporosis New morphometric vertebral fractures Traditional Estrogen agonist/antagonist; Parathyroid hormone analog; Bisphosphonate; RANK ligand (RANKL) inhibitor Osteoporosis Patients with glucocorticoid induced osteoporosis Bone mineral density¤  Traditional Bisphosphonate; Parathyroid hormone analog; RANKL inhibitor Osteoporosis Men with osteoporosis Bone mineral density¤ Traditional Parathyroid hormone analog; Bisphosphonate; RANK ligand (RANKL) inhibitor Overweight Initial body mass index (BMI) of 30 kg/m2 or greater (obese) Percent change in body mass index (BMI) Traditional Sympathomimetic amine anorectic Paget's disease Patients with Paget's disease Serum alkaline phosphatase Traditional Bisphosphonate Peri-implantitis Patients with peri-implantitis Probing pocket depth˟ Traditional Antimicrobial Periodontitis Patients with chronic periodontitis with a mean probing pocket depth of greater than 5mm Probing pocket depth Traditional Antimicrobial Pertussis (in combination vaccines) Persons (18 through 64 years of age) to be immunized against pertussis Serum antibody concentrations Traditional Induction of immunity Phenylketonuria 1. Patients with hyperphenylalaninemia due to tetrahydrobiopterin-responsive phenylketonuria
2. Adults with PKU who have uncontrolled plasma Phe>600 micromol/L on existing management Plasma phenylalanine Traditional 1. Phenylalanine hydroxylase activator
2. Phenylalanine-metabolizing enzyme Pneumococcal conjugate vaccine Persons ( ≥ 50 years of age) to be immunized against pneumonia and invasive disease Opsonophagocytic antibody response Traditional/Accelerated Induction of immunity Polio vaccine Persons to be immunized against polio Neutralizing antibody response Traditional Induction of immunity Polycystic kidney disease Patients with autosomal dominant polycystic kidney disease with or without associated polycystic liver disease Total kidney volume˟ Accelerated Mechanism agnostic* Preterm birth Women with a singleton pregnancy who have a history of singleton spontaneous preterm birth Delivery prior to 37 weeks gestation Accelerated  Progesterone analog Primary biliary cholangitis Patients with primary biliary cholangitis Serum alkaline phosphatase and bilirubin# Accelerated Anti-fibrotic; Anti-inflammatory Primary glomerular diseases associated with significant proteinuria Patients with primary glomerular disease associated with significant proteinuria, primary immunoglobulin A nephropathy (IgAN) Proteinuria (urinary protein/creatinine ratio) Accelerated Mechanism agnostic* Primary hyperoxaluria type 1 (PH1) Patients with primary hyperoxaluria type 1 (PH1) Urinary oxalate Traditional siRNA against hyroxyacid oxidase 1 gene Primary hyperparathyroidism Patients with hypercalcemia due to primary hyperparathyroidism Serum calcium Traditional Calcium-sensing receptor agonist Propionic acidemia Patients with acute hyperammonemia due to propionc acidemia Plasma ammonia  Traditional Carbamoyl Phosphate Synthetase 1 activator Pulmonary fibrosis Patients with pulmonary fibrosis Forced vital capacity (FVC) Traditional Mechanism agnostic* Pulmonary tuberculosis Patients with active pulmonary tuberculosis Sputum culture conversion to negative Accelerated Antimicrobial Pyruvate kinase deficiency anemia Hemolytic anemia in adults with pyruvate kinase (PK) deficiency Hemoglobin (Hgb) response Traditional† pyruvate kinase activator Rabies immune globulin Patients with suspected exposure to a rabid animal Rabies neutralizing activity and antibody response Traditional Passive immunity Rabies Vaccine Persons to be immunized against rabies Neutralizing antibody Traditional Induction of immunity Secondary hyperparathyroidism associated with chronic kidney disease Patients with secondary hyperparathyroidism associated with chronic kidney disease Serum intact parathyroid hormone (iPTH) Traditional Calcium-sensing receptor agonist; Vitamin D3 analog Smallpox vaccine Persons to be immunized against smallpox Vaccinia-neutralizing antibody Traditional Induction of immunity Smallpox vaccine Persons to be immunized against smallpox Vaccination site take reaction (replicating smallpox vaccines only) Traditional Induction of immunity Supportive cancer care Patients with delayed methotrexate clearance due to impaired renal function Plasma methotrexate Traditional  Carboxypeptidase Supportive cancer care Patients with leukemia, lymphoma, and solid tumor malignancies who are receiving anti-cancer therapy expected to result in tumor lysis and subsequent elevation of uric acid Serum uric acid Traditional Uric acid specific enzyme Supportive cancer care Patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs Duration of severe neutropenia Traditional Leukocyte growth factor Systemic sclerosis-interstitial lung disease Patents with systemic sclerosis-interstitial lung disease Forced vital capacity (FVC) Traditional Mechanism agnostic* Testosterone deficiency Men with primary or hypogonadotropic hypogonadism Serum testosterone Traditional Androgen, GnRH analog Tetanus vaccine Persons to be immunized against tetanus Anti-tetanus toxoid antibody Traditional Induction of immunity Tick-borne encephalitis vaccine Persons to be immunized against tick-borne encephalitis Neutralizing antibody Traditional Induction of immunity Tobacco dependence Cigarette smokers Exhaled carbon monoxide Traditional Smoking cessation Tumor-induced osteomalacia FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors Serum Phosphorus Concentration Traditional^† fibroblast growth factor 23 (FGF23) blocking antibody Type 1 diabetes mellitus Patients with type 1 diabetes mellitus Serum hemoglobin A1C Traditional Glucose-lowering Type 1 Gaucher disease Patients with Type 1 Gaucher disease Spleen volume, liver volume, hemoglobin and platelet count# Traditional Glucosylceramide synthase inhibitor; Hydrolytic lysozomal glucocerebroside-specific enzyme Type 2 diabetes mellitus Patients with type 2 diabetes mellitus Serum hemoglobin A1C Traditional Glucose-lowering Yellow fever vaccine Persons to be immunized against yellow fever Neutralizing antibody Traditional Induction of immunity

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