ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)
ISSN 2383-9457 (ONLINE)
| Active ingredient | NCT Number | FDA-approved use on approval date | Type of Primary endpoint | Measured PRO concept as secondary endpoint | PROM |
|---|---|---|---|---|---|
| Diseases of the Skin and Subcutaneous Tissue (n=5) | |||||
| Abrocitinib |
NCT03349060 NCT03575871 NCT03720470 |
To treat refractory, moderate-to-severe atopic dermatitis | ClinRO | Worst itch intensity in adults with moderate-tosevere AD | PP-NRS PSAAD |
| Risankizumab-rzaa |
NCT02684370 NCT02684357 |
To treat moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy | ClinRO | Quality of life of dermatology patients | DLQI |
| Daily symptom experience of adults and adolescents with moderate-to-severe atopic dermatitis | PSS | ||||
| Deucravacitinib |
NCT03624127 NCT03611751 |
To treat moderate-to-severe plaque psoriasis | ClinRO | Symptoms and signs in psoriasis | PSSD |
| Guselkumab |
NCT02207231 NCT02207244 |
For the treatment of adult patients with moderate-to-severe plaque psoriasis | ClinRO | Symptoms and signs in psoriasis | PSSD |
| Quality of life of dermatology patients | DLQI | ||||
| Tralokinumab-ldrm |
NCT03131648 NCT03160885 NCT03363854 |
To treat moderate-to-severe atopic dermatitis | ClinRO | Worst itch intensity in children from 6 to 11 with moderate-to-severe AD | Worst Daily Pruritus NRS |
| AD severity; Assessment of Therapeutic Education | SCORAD | ||||
| Quality of life of dermatology patients | DLQI | ||||
| Endocrine, Nutritional, and Metabolic Diseases (n=6) | |||||
| Tafamidis meglumine |
NCT01994889 |
To treat heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis in adults | Biomarker | Quality of life in patients with congestive heart failure | KCCQ-OS |
| Setmelanotide |
NCT02896192 NCT03287960 |
To treat obesity and the control of hunger associated with pro-opiomelanocortin deficiency, a rare disorder that causes severe obesity that begins at an early age | Biomarker | Intensity of hunger | (Diary) Global Hunger questionnaire (question #1 and #2) |
| Vutrisiran |
NCT03759379 |
To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis | ClinRO, Biomarker | Patients’ perceptions of the effects of neuropathy | Norfolk QoL-DN |
| Patisiran |
NCT01960348 |
To treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adult patients | ClinRO | Patients’ perceptions of the effects of neuropathy | Norfolk QoL-DN |
| Disability in cancer patients due to chemotherapy-induced peripheral neuropathy, by capturing their activity limitations and participation restrictions | R-ODS | ||||
| Autonomic function and symptom severity in patients with autonomic disorders | COMPASS 31 | ||||
| Tezacaftor; Ivacaftor |
NCT02347657 NCT02392234 |
To treat cystic fibrosis in patients age 12 years and older | PerfO | Quality of life assessment of children, adolescents, and adults with cystic fibrosis | CFQ-R |
| Elexacaftor/Ivacaftor/Tezacaftor |
NCT03525444 NCT03525548 |
To treat patients 12 years of age and older with the most common gene mutation that causes cystic fibrosis | PerfO | Quality of life assessment of children, adolescents, and adults with cystic fibrosis | CFQ-R RD |
| Diseases of the Respiratory System (n=3) | |||||
| Benralizumab |
NCT01928771 NCT01914757 NCT02075255 NCT02322775 NCT01238861 |
For add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype | Biomarker | Frequency and inconvenience of daytime asthma symptoms and their effects on activities, and capture awakenings with asthma symptoms | ASD |
| Adequacy of clinical asthma control | ACQ-6 | ||||
| To measure the functional impairments that are most troublesome to patients aged 12 years and older with asthma | AQLQ(s) +12 | ||||
| Tezepelumab-ekko |
NCT02054130 NCT03347279 |
To treat severe asthma as an add-on maintenance therapy | Biomarker | Frequency and inconvenience of daytime asthma symptoms and their effects on activities, and capture awakenings with asthma symptoms | ASD |
| Adequacy of clinical asthma control | ACQ-6 | ||||
| To measure the functional impairments that are most troublesome to patients aged 12 years and older with asthma | AQLQ(s) +12 | ||||
| Health outcome from intervention | EQ-5D-5L | ||||
| Revefenacin |
NCT02459080 NCT02512510 |
To treat patients with chronic obstructive pulmonary disease | PerfO | HRQoL in chronic airways diseases | SGRQ |
| Disease of the Blood and Blood-Forming Organs, Certain Immune Disorders (n=3) | |||||
| Mitapivat |
NCT03548220 |
To treat hemolytic anemia in pyruvate kinase deficiency | Biomarker | Symptoms and impacts of interest of the patients with pyruvate kinase deficiency | PKDD |
| Impacts of PK deficiency on patients’ HRQoL | PKDIA | ||||
| Fedratinib |
NCT01437787 |
To treat adult patients with intermediate- 2 or high-risk primary or secondary myelofibrosis | Biomarker | Burden of myelofibrosis-related symptoms | MFSAF v2.0 |
| Ravulizumab |
NCT02946463 NCT03056040 |
To treat paroxysmal nocturnal hemoglobinuria | Biomarker | Specific quality of life concerns related to fatigue | FACIT-Fatigue |
| Diseases of the Nervous System (n=1) | |||||
| Daridorexant |
NCT03545191 NCT03575104 |
To treat insomnia | Biomarker | Daytime symptoms in people with insomnia disorder | IDSIQ |
| Subjective total sleep time | SDQ | ||||
| Diseases of the Circulatory system (n=1) | |||||
| Mavacamten |
NCT03470545 |
To treat certain classes of obstructive hypertrophic cardiomyopathy | PerfO | Health related quality of life in patients with congestive heart failure | KCCQ-23 CSS |
| Hypertrophic cardiomyopathy | HCMSQ SoB domain | ||||
| Injury, Poisoning and Certain Other Consequences of External Causes (n=1) | |||||
| Belumosudil |
NCT03640481 |
To treat chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy | Biomarker | Effect of this common complication of allogeneic transplantation on patients’ functioning and well-being | Lee Symptom Scale |
| Neoplasms Darolutamide |
NCT02200614 |
To treat adult patients with non-metastatic castration resistant prostate cancer | Biomarker | Severity of pain and the impact of pain on daily functions | BPI-SF |
ACQ-6, Asthma Control Questionnaire-6; AD, Atopic Dermatitis; AQLQ[S]+12, Asthma Quality of Life Questionnaire for 12 years and old; ASD, Asthma Symptom Diary, BPI-SF, Brief Pain Inventory-Short Form; CFQ-R, Cystic Fibrosis Questionnaire-Revised; CFQ-R RD, Cystic Fibrosis Questionnaire-Revised Respiratory Domain; CHF, Congestive Heart Failure; COMPASS 31, Composite Autonomic Symptom Score 31; DLQI, Dermatology Life Quality Index; DUS, Drug Utilisation Study; EQ-5D-5L, European Quality of Life-5 Dimensions 5 Level Version; FACIT, Functional Assessment of Chronic Illness Therapy; FAS, Full Analysis Set; HCM, Hypertrophic Cardiomyopathy; HCMSQ SoB, Hypertrophic Cardiomyopathy Symptom Questionnaire-Shortness of Breath; HRQoL, Health-related Quality of Life; IDSIQ, Insomnia Daytime Symptoms and Impacts Questionnaire; KCCQ-23 CSS, Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score; KCCQ-OS, Kansas City Cardiomyopathy Questionnaire Overall Summary; MFSAF, Myelofibrosis Symptom Assessment Form; Norfolk QoL-DN, Norfolk Quality of Life-Diabetic Neuropathy; NRS, Numeric Rating Scale; OAB, Overactive Bladder; PKDD, Pyruvate Kinase Deficiency Diary; PKDIA, Pyruvate Kinase Deficiency Impact Assessment; PO-SCORAD, Patient-Oriented SCORing Atopic Dermatitis; PP-NRS, Peak Pruritus Numerical Rating Scale; PSAAD, Pruritus and Symptoms Assessment for Atopic Dermatitis; PSS, Psoriasis Symptom Scale; PSSD, Psoriasis Symptoms and Signs Diary; R-ODS, Rasch-built Overall Disability Scale; SCORAD, Patient-Oriented SCORing Atopic Dermatitis; SDQ, Sleep Diary Questionnaire; SGRQ, St. George Respiratory Questionnaire; sSE, subjective Sleep Efficiency; sSOL, subjective Sleep Onset Latency; sTST, subjective Total Sleep Time; sWASO, subjective Wake After Sleep Onset; TSMQ, Treatment Satisfaction Questionnaire for Medication