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Table 2 Nonprimary endpoints used in medical labeling (2000-2012)

From: Potential of patient-reported outcomes as nonprimary endpoints in clinical trials

Product Date of approval Indication PRO measures included
Protonix February 2, 2000 Short-term treatment in the healing and symptomatic relief of erosive esophagitis Daily diary: frequency and severity of acid regurgitation, dysphagia, and daytime and nighttime symptoms
Frova January 18, 2001 Acute treatment of migraine with or without aura in adults Headache diary with associated symptoms
Foradil February 16, 2001 Maintenance treatment of asthma and prevention of bronchospasm in adults and children aged 5 years and older Daily diary: nighttime asthma symptom score, daytime asthma symptom score, number of inhalations of rescue medication
Acute prevention of exercise-induced bronchospasm
Nexium February 20, 2001 Short-term treatment in the healing and symptomatic resolution of diagnostically confirmed erosive esophagitis Daily symptom diary
Maintenance of symptom resolution and healing of erosive esophagitis
Treatment of heartburn and other symptoms related to GERD
Treatment of patients with H. pylori infection and duodenal ulcer disease
Elidel December 13, 2001 Short-term and intermittent long-term therapy in the treatment of mild to moderate atopic dermatitis Unnamed purities assessment
Emend March 27, 2003 Prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy FLIE
Nevanac August 19, 2005 Treatment of pain and inflammation associated with cataract surgery Ocular pain scale
Chantix May 10, 2006 Aid to smoking cessation treatment QSU-Brief
MNWS
SEI
Vyvanse February 23, 2007 Treatment of ADHD CPRS
Soliris March 16, 2007 Treatment of patients with paroxysmal nocturnal hemoglobinuria to reduce hemolysis FACIT-Fatigue
EORTC QLQ-C30
Letairis June 15, 2007 Treatment of pulmonary arterial hypertension (WHO Group I) in patients with WHO class II or III symptoms to improve exercise capacity and delay clinical worsening SF-36 Health Survey
Durezol June 23, 2008 Treatment of inflammation and pain associated with ocular surgery VAS – eye pain/discomfort
VAS – photophobia
Ampyra January 22, 2010 Indicated to improve walking in patients with multiple sclerosis MSWS-12
Egrifta November 10, 2010 Reduction of excess abdominal fat in HIV-infected patients with lipodystrophy Distress associated with belly appearance
Arcapta July 1, 2011 Long-term, once-daily maintenance bronchodilator treatment of airflow obstruction in patients with COPD, including chronic bronchitis and/or emphysema Rescue medication use
Symptom diary
SGRQ
Firazyr August 25, 2011 Treatment of acute attacks of hereditary angioedema Symptom VAS
Rescue medication
Jakafi November 16, 2011 Treatment of patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, postpolycythemia vera myelofibrosis and postessential thrombocythemia myelofibrosis MFSAF diary
Kalydeco January 31, 2012 Treatment of cystic fibrosis in patients aged 6 years and older who have a G551D mutation in the CFTR gene CFQ-R (symptoms domain)
Asclera March 30, 2010 Treatment of uncomplicated spider veins and uncomplicated reticular veins in the lower extremity Patient satisfaction using a verbal rating scale
  1. ADHD, attention deficit hyperactivity disorder; CFQ-R, Cystic fibrosis questionnaire-revised; CFTR, cystic fibrosis transmembrane conductance regulator; COPD, chronic obstructive pulmonary disease; CPRS, Conners’ parent rating scale; EORTC QLQ-C30, European organisation for research and treatment of cancer quality of life questionnaire - core questionnaire; FACIT, Functional assessment of chronic illness therapy; FLIE, Functional living index-emesis; GERD, gastroesophageal reflux disease; MFSAF, Myelofibrosis Symptom assessment form; MNWS, Minnesota nicotine withdrawal scale; MSWS-12, 12-item multiple sclerosis walking scale; PRO, patient-reported outcome; QSU-Brief, Brief questionnaire of smoking urges; SEI, Smoking effects inventory; SGRQ, St. George’s respiratory questionnaire; VAS, visual analogue scale; WHO, World health organization.