Vitaccess Real MG Registry: A Prospective International Observational Patient Registry in Myasthenia Gravis Linking Clinical and Patient-reported Data.

Vitaccess Real MG (VRMG) is a patient registry designed to capture longitudinal observational data on myasthenia gravis (MG), its treatment, and impact on symptoms, daily activities, and quality of life (QoL). The duration of the registry is 10 years from launch, and approximately 600 patients will be recruited in the US and Europe with no defined upper limit. The registry will link relevant patient- and healthcare professional (HCP)-reported data with clinical data from medical records.
Patients will be recruited at clinical sites in all participating countries. In the US only, patients can additionally be recruited via community neurologists or direct-to-patient recruitment.

开始日期2024-07-31

申办/合作机构

Vitaccess Ltd.初创企业

NCT05843669 / Recruiting临床4期

An Open-label, Multicenter, Single-group Study to Evaluate the Effects of Long-term Use of Mucinex® in Patients With Stable Chronic Bronchitis.

This is an open-label, multicenter, single-group study designed to determine the effectiveness of Mucinex® when used by patients to treat SCB over a 12-week period, following a 2-week run-in period of no treatment (to establish a baseline).

开始日期2023-07-06

申办/合作机构

American Health Research, LLC

[+1]

NCT06060197 / CompletedN/A

Caregiver-reported Economic Burden and Quality of Life Impact of Monocarboxylate Transporter 8 (MCT8) Deficiency

Caregivers face many responsibilities outside of their role as a friend or parent, which can lead to emotional, financial, social, and professional challenges. To better understand the impact of MCT8 deficiency on caregivers, Egetis Therapeutics are conducting an online survey for adult caregivers of persons living with the MCT8 deficiency.

开始日期2022-08-23

申办/合作机构

Rare Thyroid Therapeutics International AB

[+1]

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项与 Vitaccess Ltd. 相关的文献（医药）

2023-09-01·Heliyon

Paving the way for patient centricity in real-world evidence (RWE): Qualitative interviews to identify considerations for wider implementation of patient-reported outcomes in RWE generation

Article

作者: Aiyegbusi, Olalekan Lee ; Calvert, Melanie J ; Wilson, Roger ; Maruszczyk, Konrad ; Bottomley, Catherine ; Collis, Philip ; Keeley, Thomas ; McMullan, Christel

ObjectivesReal-world evidence (RWE) generation can be enhanced by including patient-reported outcomes (PROs). Methods for collecting and using PRO data in the real-world setting are currently underdeveloped and there is no international guidance specific to its use in this context. This study explored stakeholders' perspectives and needs for using PROs in RWE generation. Barriers, facilitators, and opportunities for wider use of PROs in real-world studies were also investigated.MethodsOnline semi-structured interviews were conducted with international stakeholders: patients, patient advocates, regulators, payers, clinicians, academic researchers, and industry experts. Interviews were recorded, transcribed verbatim and analysed using NVivo 20. Thematic analysis was conducted based on the updated Consolidated Framework for Implementation Research (CFIR).ResultsTwenty-three interviews were conducted. Participants confirmed that the use of PROs in RWE generation is not yet well established. Participants expressed a mixed level of confidence in the value of PROs collected in a real-world setting. Operational challenges associated with collecting routine PRO data to inform care delivery at the individual level (e.g., setting up infrastructure) need to be addressed. Methodological and other challenges (e.g., financing research) associated with collecting prospective de novo data in a real-world setting should be considered to facilitate PRO utilisation in real-world studies.ConclusionsSeveral opportunities and challenges were identified regarding the broader use of PROs in RWE research. Joint efforts from different stakeholders are needed to maximise PRO implementation, with consideration given to each stakeholders' specific needs (e.g., by developing good practices).

2023-05-11·BMJ open

Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG).

Article

作者: Beauchamp, Jon ; Paci, Sandra ; Larkin, Mark ; Palace, Jacqueline ; Meisel, Andreas ; Ramirez, Joyce ; Berrih-Aknin, Sonia ; Saccà, Francesco ; De Ruyck, Femke ; Amini, Fatemeh ; Muppidi, Srikanth ; Claeys, Kristl G ; Philips, Glenn ; Quinn, Casey

OBJECTIVESThis study aims to explore the impact of myasthenia gravis (MG) - in terms of treatments, side effects, comorbidities, psychological health and work or study- in the real world from a patient perspective.DESIGN AND PARTICIPANTSThis is a prospective, observational, digital, longitudinal study. Adults diagnosed with MG residing in the USA, Japan, Germany, the UK, Italy, Spain or Canada were eligible to participate in the study. There were no other exclusion criteria. Participants used a bespoke smartphone application to confirm eligibility, provide consent and enter data about their MG into a profile, a tracker to record MG-related events and a series of patient-reported outcome instruments. 1693 participants completed at least 1 survey and were included in this analysis.RESULTSResults are presented as a percentage of respondents to each survey question. The study population was largely female (69% of 1586 respondents), with an average age of 49.9 years (SD 14.8). In the previous 12 months, 83.7% of 1412 respondents confirmed that they had received one or more routine treatments for MG, and 67.1% of 255 respondents confirmed that they had experienced a side effect in the previous month. Commonly experienced comorbidities reported by 966 respondents were thyroid problems, hypertension and anxiety, experienced by 37.5%, 31.4% and 28.0% of respondents, respectively.According to 889 respondents to the Hospital Anxiety and Depression Scale survey, 52.7% and 43.2% had a score indicative of at least mild anxiety and mild depression, respectively. Of 257 respondents, 33.0% reported experiencing a work or study impact in the past month.CONCLUSIONSThis analysis of baseline characteristics of the MyRealWorld MG study population indicates that, despite current treatments, patients experience notable burden. Further scheduled analyses will develop a longitudinal picture of MG burden.TRIAL REGISTRATION NUMBERNCT04176211.

2022-12-31·Future Rare Diseases

Sickle Cell Disease: A Global Patient Registry Review

作者: Llewellyn, Samuel ; Amini, Fatemeh ; Borecka, Oktawia ; Ofori, Akosua

Sickle cell disease (SCD) is one of the most common inherited diseases. This review identified and assessed key characteristics of the increasing number of SCD registries reporting patients data. Registries were identified through targeted searches. Registry publications/websites were assessed for availability of information on patient outcomes. Of 33 identified registries, 21 were still active. Data were available for demographics (97%), diagnosis/disease classification (91%), treatments (82%), hospitalization (52%), mortality (36%), comorbidities (36%) and quality of life (18%). Only three registries collected data on patient-reported outcomes (PROs), and only two on productivity loss. SCD registries are a valuable source of real-world information. There is a paucity of quality of life, patient-reported outcomes and productivity loss data.

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