scholarly journals An overview of clinical activities in Endo-ERN: the need for alignment of future network criteria

2020 ◽  
Vol 183 (2) ◽  
pp. 141-148
Author(s):  
Friso de Vries ◽  
Mees Bruin ◽  
Angelica Cersosimo ◽  
Charlotte N van Beuzekom ◽  
S Faisal Ahmed ◽  
...  
Keyword(s):  
Continuous Monitoring ◽  
Monitoring Program ◽  
Reference Network ◽  
Clinical Activity ◽  
Reference Centre ◽  
European Reference Network ◽  
It Systems ◽  
Patient Reported ◽  
Expert Input ◽  
Original Application

Objective Given that volumes of patients and interventions are important criteria to qualify as a reference centre (RC) for the European Reference Network on Rare Endocrine Conditions (Endo-ERN), the present study aimed to evaluate the data that were reported in the original application against subsequent assessments of activity and review the criteria that may define RCs using two main thematic groups (MTGs): Pituitary and Thyroid, as examples. Methods Review of content in application forms and continuous monitoring data and of a survey distributed to RCs. A list of ‘key procedures’ for the assessment of performance of RCs was composed with the help of the Pituitary and Thyroid MTG chairs. Results In the original application, the number of undefined procedures ranged from 20 to 5500/year (Pituitary) and from 10 to 2700/year (phyroid) between applicants. In the survey, the number of key procedures per centre ranged from 18 to 150/year (Pituitary) and from 20 to 1376/year (Thyroid). The median numbers of new patients reported in the continuous monitoring program were comparable with the application and survey; however, some centres reported large variations. Conclusions Monitoring of clinical activity in an ERN requires clear definitions that are optimally aligned with clinical practice, diagnosis registration, and hospital IT systems. This is a particular challenge in the rare disease field where the centre may also provide expert input in collaboration with local hospitals. Application of uniform definitions, in addition to condition-specific clinical benchmarks, which can include patient-reported- as well as clinician-reported outcome measures, is urgently needed to allow benchmarking of care across Endo-ERN.

scholarly journals SUN-070 European Registries for Rare Endocrine Conditions (EuRRECa): Results from the Platform for E-reporting of Rare Endocrine Conditions (e-REC)

2020 ◽  
Vol 4 (Supplement_1) ◽  
Author(s):  
Salma R Ali ◽  
Jillian Bryce ◽  
Martine Cools ◽  
Thomas Danne ◽  
Mehul T Dattani ◽  
...  
Keyword(s):  
Median Number ◽  
Reference Network ◽  
Clinical Activity ◽  
Monthly Basis ◽  
Web Based ◽  
Electronic Reporting ◽  
Sex Development ◽  
European Reference Network ◽  
Reporting Programme ◽  
Network Methods

Abstract Background EuRRECa (European Registries for Rare Endocrine Conditions) is a group of web-based projects that work closely with the European Reference Network (ERN) for Rare Endocrine Conditions (endo-ern.eu) and helps the ERN with inventorying its clinical activity. To understand the number of new encounters of rare conditions within this network, it has launched an e-reporting programme for rare endocrine conditions (e-REC) that are covered within this network. Methods 46 endocrine centres within 18 countries volunteered to participate in e-REC from July 2018 to June 2019. An electronic reporting ‘card’ developed through REDcap was issued monthly to enquire whether clinicians had encountered a new case of any condition within the 8 Endo-ERN main thematic groups (MTGs). Results On a monthly basis over 1 year, a median of 14 (range 11, 21) paediatric centres and 13 (11, 25) adult centres actively reported cases. A median of 53 (22, 80) paediatric cases and 96 (42, 250) adult cases were reported monthly. Amongst paediatric cases, conditions within the Sex Development and Maturation (SDM) theme were most commonly reported comprising 38% of all reported conditions, with XY, DSD being the most commonly reported condition (24% of cases). Amongst adults, Pituitary and Thyroid conditions were most commonly reported, 34% and 26% of all conditions, respectively. Amongst conditions within the Pituitary group, pituitary adenoma was the most commonly reported condition (74% of cases) and non-metastatic thyroid carcinoma was the most commonly reported condition (95% of cases) amongst the Thyroid group. In children, the median number of cases reported per centre was 21 (9, 32) for conditions affecting SDM. In adults, a median of 37 (6, 75) Pituitary and 22 (5, 80) Thyroid cases were reported per centre. Conclusion e-REC is a simple, yet effective, platform that can be used to capture information on new encounters with patients with several rare conditions and can be adapted to serve the needs of other discrete networks that are interested in understanding the occurrence of rare conditions.

scholarly journals Patients with rare endocrine conditions have corresponding views on unmet needs in clinical research

Endocrine ◽  
2021 ◽  
Author(s):  
Johan P. de Graaf ◽  
◽  
Friso de Vries ◽  
Anne Dirkson ◽  
Olaf Hiort ◽  
...  
Keyword(s):  
Clinical Research ◽  
Social Life ◽  
Unmet Needs ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Reference Network ◽  
Ability To Work ◽  
European Languages ◽  
European Reference Network ◽  
Patient Reported

Abstract Purpose European Reference Network on Rare Endocrine Conditions' (Endo-ERN) mission is to reduce and ultimately abolish inequalities in care for patients with rare endocrine conditions in Europe. This study assesses which themes related to rare endocrine conditions are prioritized by patients for clinical research. Methods A survey was developed, translated into 22 different European languages, and distributed to patients with rare endocrine conditions. Patients were asked to give priority scores to listed prespecified topics: fertility, heritability, tiredness, daily medicine intake, sleep quality, physical discomfort, and ability to work, partake in social life, and sports. They were also asked to suggest further important areas for research in open fields. Results After data cleaning, 1378 survey responses were analyzed. Most responses were received from Northern (47%) and Western Europeans (39%), while Southern (11%) and Eastern Europe (2%) were underrepresented. Respondents were most interested in research concerning ability to participate in social life and work. Patients suggested key areas to work: long-term side effects of medical treatments and quality of life. Some priorities differed between disease groups, both for prespecified and open topics and reflected aspects of patients’ individual conditions. Conclusions With this large survey, Endo-ERN gained insight into patients’ unmet needs in scientific research. Patients prioritized research on ability to work and participation in social activities, though needs differ between the disease groups. Clinical experts should incorporate the results of this survey into the design of future studies on rare endocrine conditions. We aim to utilize these results in designing patient-reported outcome measures for the disease areas covered by Endo-ERN.

Collecting patient reported outcomes to facilitate prescription opioid management: Development and testing of the Opioid Management App (OM-App) (Preprint)

2020 ◽  
Author(s):  
Jessica Robinson-Papp ◽  
Gabriela Cedillo ◽  
Richa Deshpande ◽  
Mary Catherine George ◽  
Qiuchen Yang ◽  
...  
Keyword(s):  
Chronic Pain ◽  
Pain Intensity ◽  
Risk Behaviors ◽  
Digital Health ◽  
Monitoring Program ◽  
Prescription Opioid ◽  
Research Database ◽  
Cross Sectional ◽  
Study Duration ◽  
Patient Reported

BACKGROUND Collecting patient-reported data needed by clinicians to adhere to opioid prescribing guidelines represents a significant time burden. OBJECTIVE We developed and tested an opioid management app (OM-App) to collect these data directly from patients. METHODS OM-App used a pre-existing digital health platform to deliver daily questions to patients via text-message and organize responses into a dashboard. We pilot tested OM-App over 9 months in 40 diverse participants with HIV who were prescribed opioids for chronic pain. Feasibility outcomes included: ability to export/integrate OM-App data with other research data; patient-reported barriers and adherence to OM-App use; capture of opioid-related harms, risk behaviors and pain intensity/interference; comparison of OM-App data to urine drug testing, prescription drug monitoring program data, and validated questionnaires. RESULTS OM-App data was exported/integrated into the research database after minor modifications. Thirty-nine of 40 participants were able to use OM-App, and over the study duration 70% of all OM-App questions were answered. Although the cross-sectional prevalence of opioid-related harms and risk behaviors reported via OM-App was low, some of these were not obtained via the other measures, and over the study duration all queried harms/risks were reported at least once via OM-App. Clinically meaningful changes in pain intensity/interference were captured. CONCLUSIONS OM-App was used by our diverse patient population to produce clinically relevant opioid- and pain-related data, which was successfully exported and integrated into a research database. These findings suggest that OM-App may be a useful tool for remote monitoring of patients prescribed opioids for chronic pain. CLINICALTRIAL NCT03669939 INTERNATIONAL REGISTERED REPORT RR2-doi:10.1016/j.conctc.2019.100468

scholarly journals Educational aspects of rare and orphan lung diseases

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Tiago M. Alfaro ◽  
Marlies S. Wijsenbeek ◽  
Pippa Powell ◽  
Daiana Stolz ◽  
John R. Hurst ◽  
...  
Keyword(s):  
Lung Diseases ◽  
Healthcare Professionals ◽  
Treatment Options ◽  
Delayed Diagnosis ◽  
Reference Network ◽  
Delay In Diagnosis ◽  
Positive Effects ◽  
European Reference Network ◽  
Patient Representatives ◽  
Rare Lung Diseases

AbstractPeople with rare lung diseases often suffer the burden of delayed diagnosis, limited treatment options, and difficulties in finding expert physicians. One of the reasons for the delay in diagnosis is the limited training for healthcare practitioners on rare diseases. This review explores the main concerns and needs for education on rare lung diseases from the perspectives of both patients and professionals. Despite the increasing interest in rare lung disorders and some recent breakthrough developments on the management of several diseases, healthcare professionals, including general practitioners and hospital workers, receive little education on this topic. Nonetheless, many healthcare professionals show much interest in receiving further training, especially on diagnosis. Patients and families want easier access to high-quality education materials to help them manage their own disease. Well-educated patients are better equipped to deal with chronic diseases, but patient education can be challenging as patients’ individual health issues, and diverse backgrounds can create significant barriers. Raising more awareness for rare lung diseases and further development of patient-centred international expert networks like the European Reference Network on Rare Lung Diseases (ERN-LUNG), which includes both experts and patient representatives, are essential for improving care and education on rare lung diseases. Initiatives such as the Rare Disease Day, have been successful in increasing awareness for rare conditions. The development of online tools for accessing information has had positive effects and should be further supported and extended in the future.

scholarly journals A survey of the European Reference Network EpiCARE on clinical practice for selected rare epilepsies

2020 ◽  
Author(s):  
Tobias Baumgartner ◽  
Mar Carreño ◽  
Rodrigo Rocamora ◽  
Francesca Bisulli ◽  
Antonella Boni ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Reference Network ◽  
European Reference Network

scholarly journals eHealth for patients with rare diseases: the eHealth Working Group of the European Reference Network on Rare Multisystemic Vascular Diseases (VASCERN)

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Alessia Paglialonga ◽  
Raffaella Gaetano ◽  
Leema Robert ◽  
Marine Hurard ◽  
Luisa Maria Botella ◽  
...  
Keyword(s):  
Rare Diseases ◽  
Working Group ◽  
Vascular Diseases ◽  
Mobile App ◽  
Reference Network ◽  
Training And Education ◽  
Digital Platforms ◽  
Patient Organizations ◽  
European Reference Network ◽  
Strategic Assets

Abstract Background The European Reference Network on Rare Multisystemic Vascular Diseases (VASCERN) was launched in 2017 and involves, to date, 35 highly specialised multidisciplinary expert centres (from the 30 full Healthcare Provider members) coming from 11 countries and more than 70 patient organizations from 16 countries. The eHealth Working Group (WG) of VASCERN was set up to develop practical, patient-centred solutions and strategies for effective use of eHealth tools to answer the needs of patients with multisystemic vascular rare diseases. The eHealth WG Following the identified patients’ needs and following the guiding principles of collaboration and patient-centredness, the eHealth WG was created with the following aims: to develop a mobile app to help patients find expert centres and patient organizations, and to develop resources (Pills of Knowledge, PoK) for training and education via digital platforms (eLearning). The mobile app includes, to date, functionalities that allow users to find expert centres and patient organizations across Europe in the area of rare multisystemic vascular diseases. Discussed app developments include personalized digital patient passports, educational material, emergency management guidelines and remote consultations. Regarding training and education, a variety of PoK have been developed. The PoK cover several topics, target several user groups, and are delivered in various formats so that they are easy-to-use, easy-to-understand, informative, and viable for delivery and sharing through digital platforms (eLearning) including, e.g., the VASCERN YouTube™ channel. Conclusion Overall, the work carried out by the eHealth WG of VASCERN can be seen as a pilot experience that may serve as a basis to for collaborative development of patient-centred eHealth tools that answer the needs of patients with various rare diseases, not limited to rare multisystemic vascular diseases. By expanding the multidisciplinary approach here described, clinical and research networks can take advantage of eHealth services and use them as strategic assets in achieving the ultimate goal of ensuring equity of access to prevention programs, timely and accurate diagnosis and specialized care for patients with rare diseases throughout Europe.

scholarly journals Boosting care and knowledge about hereditary cancer: European Reference Network on Genetic Tumour Risk Syndromes

2018 ◽  
Vol 18 (2) ◽  
pp. 281-284 ◽  
Author(s):  
Janet R. Vos ◽  
◽  
Lisette Giepmans ◽  
Claas Röhl ◽  
Nicoline Geverink ◽  
...  
Keyword(s):  
Hereditary Cancer ◽  
Reference Network ◽  
European Reference Network ◽  
Genetic Tumour

scholarly journals Dystonia Management: What to Expect From the Future? The Perspectives of Patients and Clinicians Within DystoniaNet Europe

2021 ◽  
Vol 12 ◽  
Author(s):  
Marenka Smit ◽  
Alberto Albanese ◽  
Monika Benson ◽  
Mark J. Edwards ◽  
Holm Graessner ◽  
...  
Keyword(s):  
Information Exchange ◽  
Wide Spectrum ◽  
Neurological Diseases ◽  
Evidence Base ◽  
Diagnostic Process ◽  
Reference Network ◽  
Novel Treatments ◽  
European Reference Network ◽  
Specialized Care ◽  
The Central Nervous System

Improved care for people with dystonia presents a number of challenges. Major gaps in knowledge exist with regard to how to optimize the diagnostic process, how to leverage discoveries in pathophysiology into biomarkers, and how to develop an evidence base for current and novel treatments. These challenges are made greater by the realization of the wide spectrum of symptoms and difficulties faced by people with dystonia, which go well-beyond motor symptoms. A network of clinicians, scientists, and patients could provide resources to facilitate information exchange at different levels, share mutual experiences, and support each other's innovative projects. In the past, collaborative initiatives have been launched, including the American Dystonia Coalition, the European Cooperation in Science and Technology (COST—which however only existed for a limited time), and the Dutch DystonieNet project. The European Reference Network on Rare Neurological Diseases includes dystonia among other rare conditions affecting the central nervous system in a dedicated stream. Currently, we aim to broaden the scope of these initiatives to a comprehensive European level by further expanding the DystoniaNet network, in close collaboration with the ERN-RND. In line with the ERN-RND, the mission of DystoniaNet Europe is to improve care and quality of life for people with dystonia by, among other endeavors, facilitating access to specialized care, overcoming the disparity in education of medical professionals, and serving as a solid platform to foster international clinical and research collaborations. In this review, both professionals within the dystonia field and patients and caregivers representing Dystonia Europe highlight important unsolved issues and promising new strategies and the role that a European network can play in activating them.

CONTACT-01: A phase III, randomized study of atezolizumab plus cabozantinib versus docetaxel in patients with metastatic non-small cell lung cancer (mNSCLC) previously treated with PD-L1/PD-1 inhibitors and platinum-containing chemotherapy.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. TPS9134-TPS9134
Author(s):  
Joel W. Neal ◽  
Palak Kundu ◽  
Tomohiro Tanaka ◽  
Ida Enquist ◽  
Sid Patel ◽  
...  
Keyword(s):  
Kinase Inhibitor ◽  
Randomized Study ◽  
Phase Iii ◽  
Clinical Activity ◽  
Open Label ◽  
Eligibility Criteria ◽  
Recist 1.1 ◽  
Platinum Based Chemotherapy ◽  
Patient Reported ◽  
Previously Treated

TPS9134 Background: Patients with mNSCLC who progress on anti–PD-L1/PD-1 therapy administered in combination with or after platinum-based chemotherapy (PBC) are mainly treated with docetaxel or pemetrexed monotherapy. These therapies only have modest clinical activity, leaving a high unmet medical need. Cabozantinib, a tyrosine kinase inhibitor (TKI), promotes an immune-permissive environment and may enhance the efficacy of PD-L1/PD-1 inhibitors, offering a promising second/third-line therapeutic opportunity for patients with mNSCLC. In a Phase Ib multi-cohort study (COSMIC-021; NCT03170960), cabozantinib plus atezolizumab (anti–PD-L1) showed an acceptable safety profile and promising efficacy (ORR: 27%; mDOR: 5.7 mo [range: 2.6-6.9]; disease control rate [CR + PR + SD]: 83%) in 30 patients with mNSCLC who had progressed after prior anti–PD-L1/PD-1 therapy plus chemotherapy (Neal et al. J Clin Oncol 2020). The Phase III CONTACT-01 study will further evaluate the efficacy and safety of atezolizumab plus cabozantinib versus docetaxel monotherapy in patients with mNSCLC who have progressed during or after prior treatment with anti–PD-L1/PD-1 therapy and PBC. Methods: CONTACT-01 (NCT04471428) is a Phase III, multi-center, randomized, open-label study that will enroll ≈350 patients from 150 to 200 sites internationally. Key eligibility criteria include histologically or cytologically confirmed mNSCLC, disease progression with concurrent or sequential anti–PD-L1/PD-1 treatment and PBC, measurable disease (RECIST 1.1), ECOG PS of 0-1 and the availability of tissue specimens for centralized PD-L1 testing or known PD-L1 status using a health authority–approved PD-L1 assay. Patients with NSCLC previously treated with cabozantinib, docetaxel or anti–PD-L1/PD-1 + VEGFR TKIs are excluded. Patients with known sensitizing EGFR/ALK mutations and active or untreated CNS metastases are also excluded. Patients will be randomized 1:1 to receive either atezolizumab (1200 mg IV every 3 weeks) + cabozantinib (40 mg orally once daily) or docetaxel (75 mg/m2 IV every 3 weeks). The primary endpoint is OS. Secondary endpoints include investigator-assessed PFS, ORR and DOR per RECIST 1.1; TTD in patient-reported physical function and global health status (EORTC QLQ-C30); investigator-assessed PFS rates at 6 months and 1 year; OS rates at 1 and 2 years; safety and PK. Clinical trial information: NCT04471428.

Sign in / Sign up

Export Citation Format

Share Document