PROVE: retrospective, non‐interventional, Phase IV study of perampanel in real‐world clinical care of patients with epilepsy

PROVE is a retrospective, phase IV study assessing retention, dosing, efficacy, and safety of perampanel when administered to patients during routine clinical care. We report an interim analysis of preadolescent (1 to <12 years) and adolescent (12 to <18 years) patients. Data were obtained from medical records of patients with epilepsy initiating perampanel after January 1, 2014; cut-off date for this analysis was October 10, 2018. Overall, 151 preadolescent and 183 adolescent patients were included. Retention rates following 24 months on perampanel were 42.5% (preadolescent subgroup; n = 31/73) and 55.7% (adolescent subgroup; n = 54/97). Treatment-emergent adverse events occurred in 53 (35.1%) preadolescent (most common: aggression, irritability, and somnolence) and 78 (42.6%) adolescent patients (most common: somnolence, aggression, and dizziness). These data indicate that daily oral doses of perampanel are generally well tolerated during routine clinical care, with favorable retention rates for ≤2 years, in patients aged 1 to <18 years.

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The Value of Real-World Data in Understanding Prostate Cancer Risk and Improving Clinical Care: Examples from Swedish Registries

Cancers ◽

10.3390/cancers13040875 ◽

2021 ◽

Vol 13 (4) ◽

pp. 875

Author(s):

Kerri Beckmann ◽

Hans Garmo ◽

Ingela Franck Lissbrant ◽

Pär Stattin

Keyword(s):

Prostate Cancer ◽

Real World ◽

Clinical Care ◽

Prostate Cancer Risk ◽

Real World Data ◽

Level Of Evidence ◽

World Data ◽

Cancer Data ◽

Using Data ◽

Randomised Controlled

Real-world data (RWD), that is, data from sources other than controlled clinical trials, play an increasingly important role in medical research. The development of quality clinical registers, increasing access to administrative data sources, growing computing power and data linkage capacities have contributed to greater availability of RWD. Evidence derived from RWD increases our understanding of prostate cancer (PCa) aetiology, natural history and effective management. While randomised controlled trials offer the best level of evidence for establishing the efficacy of medical interventions and making causal inferences, studies using RWD offer complementary evidence about the effectiveness, long-term outcomes and safety of interventions in real-world settings. RWD provide the only means of addressing questions about risk factors and exposures that cannot be “controlled”, or when assessing rare outcomes. This review provides examples of the value of RWD for generating evidence about PCa, focusing on studies using data from a quality clinical register, namely the National Prostate Cancer Register (NPCR) Sweden, with longitudinal data on advanced PCa in Patient-overview Prostate Cancer (PPC) and data linkages to other sources in Prostate Cancer data Base Sweden (PCBaSe).

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Clinical care: Real‐world data

Diabetic Medicine ◽

10.1111/dme.5_14244 ◽

2020 ◽

Vol 37 (S1) ◽

pp. 14-16

Keyword(s):

Real World ◽

Clinical Care ◽

Real World Data ◽

World Data

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Real-world application of breast-specific gamma imaging, initial experience at a community breast center and its potential impact on clinical care

The American Journal of Surgery ◽

10.1016/j.amjsurg.2008.01.006 ◽

2008 ◽

Vol 195 (5) ◽

pp. 631-635 ◽

Cited By ~ 13

Author(s):

Minhao Zhou ◽

Nathalie Johnson ◽

Deb Blanchard ◽

Sally Bryn ◽

Joanne Nelson

Keyword(s):

Real World ◽

Clinical Care ◽

Initial Experience ◽

Gamma Imaging ◽

Breast Center ◽

Real World Application ◽

Potential Impact

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UK phase IV, observational study to assess quality of life in patients (pts) with pancreatic neuroendocrine tumours (pNETS) receiving treatment with everolimus: The “real-world” OBLIQUE study

Annals of Oncology ◽

10.1093/annonc/mdw369.22 ◽

2016 ◽

Vol 27 ◽

pp. vi143

Author(s):

J. Ramage ◽

P. Punia ◽

O. Faluyi ◽

A. Frilling ◽

T. Meyer ◽

...

Keyword(s):

Quality Of Life ◽

Observational Study ◽

Real World ◽

Neuroendocrine Tumours ◽

The Real ◽

Pancreatic Neuroendocrine Tumours ◽

Assess Quality ◽

Phase Iv

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P129: A phase IV real world study on the use of low dose methoxyflurane (PENTHROX™) for the treatment of moderate to severe trauma pain in the Canadian emergency department (ADVANCE-ED): an interim report on secondary outcomes

CJEM ◽

10.1017/cem.2020.333 ◽

2020 ◽

Vol 22 (S1) ◽

pp. S111-S111

Author(s):

S. Campbell ◽

E. Simard ◽

A. Arcand ◽

L. Blagrove ◽

P. Piraino ◽

...

Keyword(s):

Pain Relief ◽

Adverse Events ◽

Acute Pain ◽

Real World ◽

Rescue Medication ◽

Low Dose ◽

Open Label ◽

Open Label Study ◽

Label Study ◽

Phase Iv

Introduction: Inhaled low dose methoxyflurane (MEOF) was recently approved in Canada for the short-term relief of moderate to severe acute pain associated with trauma or interventional medical procedures in conscious adult patients. ADVANCE-ED is an ongoing phase IV, prospective open label study undertaken to generate real-world evidence to complement the global clinical development program through evaluation of the effectiveness of low dose MEOF in Canadian emergency departments (EDs). Methods: This multi-centre study is enrolling adult (≥18 yrs) patients with moderate to severe acute pain (NRS0-10 ≥ 4) associated with minor trauma. To address limitations from the pivotal study, this study allows patients who were excluded in the pivotal trials: namely, those with severe (≥7) pain, and those using OTC or stably dosed analgesics for other conditions, including chronic pain. Eligible patients receive a single treatment of up to 2 x 3 mL MEOF (2nd 3 mL to be provided only upon request), self-administered by the patient under medical supervision. Rescue medication is permitted at any time, if required. Results: Here we describe the patient demographics and treatment satisfaction (Global Medication Performance, GMP) at 50% enrolment (n = 49). Mean (SD) patient age is 48.0 (17.1) yrs and 55.1% are female. Mean pain (SD) reported at enrolment is 8.3 (1.5), with 73.4% of patients with NRS0-10 ≥ 8. Injuries are overwhelmingly limb trauma (87.8%). The most common type is sprain/strain (40.8%), followed by fracture (32.7%). At 5 minutes post-start of administration (STA) of MEOF, 80.4% of patients reported pain relief; this increased to 91.3% at 15 minutes, and 100% of patients reported pain relief by 30 minutes post-STA. GMP was assessed as “good”, “very good” or “excellent” by ≥80% of patients both 20 minutes post-start of administration (STA) of MEOF (83.3%) and at discharge (85.8%). When asked to what extent their expectation of pain relief had been met, 32.7% responded good, 26.5% responded “very good” and 22.4% responded “excellent”. Three quarters of enrolled patients (75.5%) did not require rescue medication. The most common (≥5%) treatment-related adverse events were dizziness (n = 14, 28.6%) and euphoric mood (n = 4, 8.2%). No serious adverse events have been reported. Conclusion: Based on 50% of the patients enrolled in this prospective, open label study, responses to inhaled low-dose MEOF are within expectation for both effectiveness and tolerability.

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Real-world continuous physiologic monitoring in paediatric cardiomyopathy patients: a safety and feasibility study

Cardiology in the Young ◽

10.1017/s1047951119001987 ◽

2019 ◽

Vol 29 (11) ◽

pp. 1400-1401

Author(s):

Catherine A. Lebo ◽

Kimberly Y. Lin ◽

Joseph W. Rossano

Keyword(s):

Disease Management ◽

Real World ◽

Chronic Disease Management ◽

Clinical Care ◽

Activity Monitor ◽

Mobile Technologies ◽

Physiologic Monitoring ◽

Activity Data ◽

Complex Patient ◽

Device Use

AbstractChronic disease management may be improved with the integration of mobile technologies capable of real-world physiologic monitoring. We performed a prospective, single-centre, pilot study to assess the feasibility of a consumer-based activity monitor use in outpatient management of adolescent cardiomyopathy patients. During the 3-month study period, the activity monitor was worn consistently and provided detailed physiologic and activity data with no adverse events related to the device use. Survey data revealed that the majority of subjects approve of these technologies becoming integrated into routine clinical care. Ongoing study is in need to define optimal disease management for this complex patient population in the age of consumer-driven healthcare.

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P015: A phase IV protocol for a real world study on the use of low dose methoxyflurane (PENTHROX™) for the treatment of moderate to severe trauma pain in the Canadian emergency department (ADVANCE-ED)

CJEM ◽

10.1017/cem.2019.206 ◽

2019 ◽

Vol 21 (S1) ◽

pp. S68

Author(s):

S. Campbell ◽

L. Belle Blagrove ◽

P. Piraino ◽

S. Dhani

Keyword(s):

Acute Pain ◽

Real World ◽

Emergency Departments ◽

Rescue Medication ◽

Low Dose ◽

Adult Patients ◽

Open Label ◽

Multi Centre Study ◽

Centre Study ◽

Phase Iv

Introduction: Pain is a significant driver of demand in emergency care and 65% of adult patients with trauma also report moderate to severe pain. Inhaled low dose methoxyflurane (MEOF) a rapid-acting patient administered inhalational analgesic was recently approved in Canada for the short-term relief of moderate to severe acute pain associated with trauma or interventional medical procedures in conscious adult patients. This study will generate real-world evidence to complement the global clinical development program through evaluation of the effectiveness of MEOF in Canadian emergency departments. Methods: This is a phase IV, prospective open label, multi-centre study. Approximately 100 adult (≥18 yrs) patients with moderate to severe acute pain (NRS0-10≥4) associated with single system trauma will be enrolled at 5-10 EDs across Canada. Patients will receive a single treatment of up to 2 x 3 mL MEOF (2nd 3 mL to be provided only upon request), self-administered by the patient under medical supervision. Rescue medication will be permitted at any time, if required. Results: Planned Assessments and Outcome Measures: Pain will be assessed using the NRS0-10 at 4 time points: screening/triage, 5 minutes and 20 minutes post-start of administration (STA) of MEOF, and when ready for discharge. Secondary assessments will include the speed of action of analgesia (from STA of MEOF); patient and physician satisfaction with treatment (as assessed through Global Medical Performance (GMP) at 20 minutes post-STA and when ready for discharge); patient and physician fulfilment of pain relief expectations (assessed when ready for discharge); use of rescue medication and treatment-emergent adverse events. Exploratory outcomes will include the time to disposition, time to readiness for discharge and responder analysis. The primary outcome measure will be the change in pain intensity over 20 minutes from the start of administration of MEOF as measured on the NRS0-10. Conclusion: We report on the methodology of a phase IV, prospective open label, multi-centre study, evaluating the use of MEOF for the management of acute traumatic pain in Canadian Emergency Departments.

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