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- Clinical trial units and clinical research coordinators: a system facing
crisis'
Authors: Stefano Stabile, Rosita Cenna, Valentina Sinna, Franchina Veronica, Francesca Mannozzi, Irene Federici, Sara Testoni, Celeste Cagnazzo
Pages: 1 - 3
Abstract: We are currently witnessing an increase in procedural and managerial complexities within the field of clinical research, which require greater human and infrastructural resources as well as imply the need for a greater skill set and expertise on the part of professionals. Within this frame of reference clinical trial units and clinical research coordinators play a vital role in the design and conduct of clinical trials in Italy. There is a current recruitment and retention crisis for this specialist role due to a complex set of factors, most likely to have come to a head due to the lack of recognition at the Italian institutional level, that lead to precarious work contracts, lack of identity, and excessive turnover at experimental sites. This article, led by the Italian Group of Clinical Research Coordinator (GIDMcrc), presents some of the issues and ways in which national stakeholders may be able to address this.
PubDate: 2023-01-16
DOI: 10.33393/ao.2023.2508
Issue No: Vol. 10 (2023)
- Manuscript formatting, delayed peer-reviews, and overemphasizing the
impact factor: can something be done'
Authors: Vijay Kothari
Pages: 4 - 5
PubDate: 2023-01-16
DOI: 10.33393/ao.2023.2530
Issue No: Vol. 10 (2023)
- Brentuximab vedotin in combination with doxorubicin, vinblastine and
dacarbazine for first-line treatment of stage IV HL: cost impact on
subsequent lines in Italy
Authors: Francesca Fiorentino, Beatrice Canali, Paolo Morelli, Federica Demma
Pages: 6 - 12
Abstract: Introduction: This study estimates the change in the costs of second-line or later (2L+) treatments compared to the current scenario, associated with the introduction of brentuximab vedotin (Adcetris®) (BV) in combination with doxorubicin, vinblastine and dacarbazine (A+AVD) for the treatment of previously untreated (1L) patients with stage IV classical Hodgkin’s lymphoma (cHL). Methods: An economic model has been developed that estimates the variation in treatment costs of 2L+ associated with the introduction of BV in 1L from the point of view of the Italian National Health System over a time horizon of 3 years. The population eligible to receive a treatment of 2L+ has been estimated from the literature, considering an increasing consumption in the three years of A+AVD in 1L. Two main scenarios and several alternative scenarios were considered to address the uncertainty that characterizes the distribution of market shares of 2L+ treatments. Results: In the baseline scenario, over three years, the introduction of BV in 1L is associated with a cumulative reduction in treatment costs of 2L+ of € 1.74 M. In all scenarios, a reduction in treatment costs of 2L+ is confirmed, with a total saving that varies between € 5.6 M and € 1.3 M compared to the main scenarios. Conclusions: The present analysis shows that the introduction of A+AVD in 1L for the treatment of stage IV CD30+ cHL patients is associated with a reduction in treatment costs of 2L+, even if there are some limitations related to the uncertainty of real cost and population estimates.
PubDate: 2023-01-16
DOI: 10.33393/ao.2023.2486
Issue No: Vol. 10 (2023)
- Cost per responder for vedolizumab and ustekinumab in Crohn’s disease
patients after failure of TNF-α inhibitors in Italy
Authors: Roberto Ravasio, Raffaella Viti, Antonio Saverio Roscini
Pages: 13 - 21
Abstract: Background: The aim of this economic evaluation was to compare the cost per responder between vedolizumab and ustekinumab in patients with Crohn’s disease (CD) after failure of tumor necrosis factor-α inhibitors in Italy. Methods: Clinical efficacy was assessed using the results of an Italian large multicentre observational retrospective cohort study. The aim of the study was to compare the effectiveness of ustekinumab and vedolizumab as second line therapy in Crohn’s disease patients in which tumour necrosis factor-α inhibitors failed. Clinical efficacy of vedolizumab and ustekinumab was measured by clinical response and clinical remission. Treatment costs were based on the number of administrations at 26 or 52 weeks. Cost per responder, based on clinical efficacy and clinical response, was used as a cost-effectiveness indicator. Results: Regardless of the clinical efficacy measure used and the treatment duration considered, the cost per responder was consistently lower for vedolizumab compared with ustekinumab on all clinical measures. Considering the clinical response, the cost per responder at 26 weeks was € 15,640 for vedolizumab and € 23,667 for ustekinumab and at 52 weeks was € 23,927 for vedolizumab and € 30,820 for ustekinumab. Considering the clinical remission, the cost per responder at 26 weeks was € 22,832 for vedolizumab and € 33,786 for ustekinumab and at 52 weeks was € 29,488 for vedolizumab and € 46,847 for ustekinumab. Sensitivity analysis confirmed the validity of results. Conclusion: These results suggest that vedolizumab is a cost-effective option compared with ustekinumab from the perspective of the Italian health service in patients with CD after failure of TNF-α inhibitors.
PubDate: 2023-01-16
DOI: 10.33393/ao.2023.2515
Issue No: Vol. 10 (2023)
- Decentralized Clinical Trials in Italy: state of the art and future
perspectives
Authors: Elisa Zagarrì, Stefania Frasson, Antonella Valerio, Gualberto Gussoni
Pages: 22 - 26
Abstract: Clinical trials are an essential source of high-quality evidence for the assessment of efficacy and safety of healthcare interventions. Nowadays the main criticality of the traditional clinical trial model is perhaps the need to improve patient selection and management, in terms of initial identification, recruitment and retention. Digital technology offers operational solutions that can facilitate many of the activities involved in clinical investigation. Decentralized Clinical Trials (DCTs) could be a new option that provides for the use of remote instruments/methods/activities in the different stages of a clinical trial, so that a range of procedures (such as informed consent, medical visits, administration of a drug or use of a medical device, measurement of clinical parameters, diagnostic testing etc.) can be moved from the research hospital to the patient’s home. Also in Italy the interest in DCTs is progressively growing, and thanks to their potential benefits DCTs can lead to significant advantages not only for patients, but also for the National Health Service and for the country as a whole. It is important that this interest should act as a stimulus, prompting timely initiatives in order to promote and regulate this new methodology for conduct of clinical trials to avoid the risk that, while other countries will be actively involved in the promotion and leading of DCTs, Italy will be selected only as “control arm”.
PubDate: 2023-02-10
DOI: 10.33393/ao.2023.2546
Issue No: Vol. 10 (2023)
- FooDia-Net. A technology platform for nutrition education and active
people involvement in the prevention of diabetes mellitus: results from
the first field trial
Authors: Mariarosaria Savarese, Caterina Bosio, Elisabetta Anna Graps, Guendalina Graffigna
Pages: 27 - 33
Abstract: Introduction: Type 2 diabetes mellitus (DM) is one of the most widespread chronic diseases in the world and, by its nature, it affects several disciplines. Patients often appear unaware and lacking an adequate lifestyle and appropriate nutritional education to prevent its aggravation. Consequently, it appears essential to promote educational initiatives aimed at helping patients diagnosed with type 2 DM to contain the disease progress. FooDia-Net is a project financed by the Ministry of Health which took place between 2017 and 2020 in 5 Italian regions, aimed at experimenting an innovative operating model of multiprofessional and cross-disciplinary management in the prevention of type 2 DM. Method: The FooDia-Net protocol was tested through a pilot project which enabled the evaluation food literacy levels and patient engagement through the administration of pre- and post-performance self-assessment questionnaires to evaluate the educational exercises proposed to the panel. Results: Out of 166 eligible patients, 38 completed the protocol (23%); results show a significant improvement in the levels of food literacy and, from the engagement point of view, despite not being significant statistically, good improvements were registered. Discussion and conclusion: Results were encouraging with regards to the multidisciplinary management of patients with type 2 diabetes. The FooDia-Net platform ensured continuity of care and emotional support for patients remotely.
PubDate: 2023-02-15
DOI: 10.33393/ao.2023.2338
Issue No: Vol. 10 (2023)
- The modern diabetes management: a jump into the future for better care
Authors: Basilio Pintaudi
Pages: 34 - 34
PubDate: 2023-02-28
DOI: 10.33393/ao.2023.2577
Issue No: Vol. 10 (2023)
- X-linked hypophosphatemic rickets: cases series and literature review with
a focus on neurosurgical management
Authors: Federico Baronio, Ferdinando Aliberti, Filomena Carfagnini, Giovanni Luigi Di Gennaro, Daniela Pasquali, Rossella Santoro, Giuseppe Toro, Mino Zucchelli, Anna Grandone
Pages: 35 - 42
Abstract: X-linked hypophosphatemic rickets (XLH) is an X-linked dominant disease caused by mutations in the Phosphate-Regulating Endopeptidase X-Linked (PHEX) gene. Due to its rarity and the wide range of clinical manifestations, management of the disease can be challenging due to several associated clinical implications that may arise during follow-up. The neurological complications associated with XLH are the most severe and often less known, which lead to important comorbidities. With the aim of summarizing the available knowledge on neurosurgical intervention in patients with XLH, we present four emblematic cases with disorders requiring neurosurgical evaluation. Relevant diagnostic delay was seen in two of these cases, with another case demonstrating that complications requiring neurosurgery may be present even in patients with few symptoms. The last case stresses the need for care of adult patients who can present with undiagnosed comorbidities. We also carried out a narrative review on neurosurgical interventions in patients with XLH. Through case reports and a review of the literature, focus is placed on the role of the neurosurgeon in the management of patients with XLH. In fact, neurosurgical intervention can often provide stable outcomes for craniosynostosis and clinical improvement for symptoms related to spinal stenosis. Thus, the neurosurgeon can aid in optimizing management of patients with XLH and should be a member of both adult and pediatric multidisciplinary teams. Lastly, additional studies are needed to determine if the early use of burosumab in infants can help to avoid complications in the long term.
PubDate: 2023-03-07
DOI: 10.33393/ao.2023.2513
Issue No: Vol. 10 (2023)
- La gestione del paziente con rinosinusite cronica e poliposi nasale in
Italia: expert opinion sul percorso diagnostico-terapeutico
Authors: Francesco Menzella, Diego Bagnasco, Maria Beatrice Bilò, Marco Caminati, Carlo Cavaliere, Marco Contoli, Alessio De Massimi, Stefania Gallo, Andrea Matucci, Giancarlo Ottaviano, Oliviero Rossi, Veronica Seccia, Martino Emmi, Frank Rikki Canevari
Pages: 43 - 49
Abstract: Chronic rhinosinusitis (CRS) is an inflammatory disease that affects the nasal mucosa and the paranasal sinuses; it can be characterized by nasal polyposis (CRSwNP) in up to 30% of cases. CRSwNP is frequently associated with bronchial asthma and patients affected show a greater severity of clinical disease with a significantly worse quality of life. CRSwNP shows predominantly an underlying activation of type 2 inflammatory pathways with the increase of eosinophils, IgE, interleukin (IL)-4, IL-5 and IL-13. Biological drugs that target inflammatory molecules are currently a therapeutic option recognized by guidelines for the treatment of uncontrolled form of the disease. The correct definition of the target patient, the type of biological drug to be used and the timing of intervention are crucial to guarantee a personalized therapy and optimize the cost/effectiveness of the treatment. A panel of Italian pneumologists, allergologists, immunologists and ear, nose and throat specialists discussed in a series of virtual expert meetings the main criteria for patient characterization and therapeutic decision, highlighting multidisciplinarity, the constant dialogue between doctor and patient, the organization in networks and the use of registries as strategies to implement the management of CRSwNP patients, to reach the personalization of the treatment and the best use of the biological drug(s).
PubDate: 2023-03-13
DOI: 10.33393/ao.2023.2555
Issue No: Vol. 10 (2023)
- New technologies in the surgical management of endometriosis
Authors: Francesca Massimello, Attilio Di Spiezio Sardo, Giuseppe Bifulco, Stefano Angioni, Vito Cela
Pages: 50 - 54
Abstract: Introduction: Endometriosis is a very common disease that affects up to 10% of the female population. Although medical therapy represents the first-line treatment for endometriosis, it does not always manage to control symptoms. Laparoscopy represents the standard surgical treatment in endometriosis. Robotic-assisted laparoscopy is an innovative mini-invasive surgical technique. Its application in gynecological surgery and in endometriosis has increased in the last decade. Our purpose is to offer an overview of the role of robotic-assisted laparoscopy in the surgical treatment of endometriosis. Methods: We evaluated studies dealing with the new technique in surgery for endometriosis with a focus on robotic surgery. We performed a compressive literature research on PubMed and the Cochrane Library in December 2022. Expert opinion: Robotic-assisted surgery is a feasible and safe approach to endometriosis surgery and is superimposable to laparoscopy in terms of complication rate, blood loss, hospitalization, and long-term improvement of symptoms. The effect of robotic-assisted surgery on operative time is still contradictory and needs to be further investigated. Robotic-assisted laparoscopic surgery can provide particular benefit in the management of women with severe endometriosis secondary to its advantage in surgical precision and ergonomics. Indocyanine green fluorescence angiography could be useful to assist in the vascularization of ureters and bowel anastomosis, to prevent postoperative complication and leakage.
PubDate: 2023-03-17
DOI: 10.33393/ao.2023.2547
Issue No: Vol. 10 (2023)
- Percorso diagnostico-terapeutico e modello organizzativo per
l’erogazione della terapia genica nelle distrofie retiniche ereditarie
in real-life
Authors: Francesco Bandello, Maurizio Battaglia Parodi, Leonardo Colombo, Fabrizio Giansanti, Marcello Pani, Francesco Parmeggiani, Ugo Procoli, Stanislao Rizzo, Annalisa Scopinaro, Andrea Sodi, Annamaria Staiano, Giovanni Staurenghi, Francesca Simonelli
Pages: 55 - 59
Abstract: Background: Leber congenital amaurosis is the first form of inherited retinal dystrophy (IRD) treated with a gene therapy approach using voretigene neparvovec (VN). To date, 42 patients have been treated, 25 of whom, treated at Clinica Oculistica Vanvitelli (Naples), have shown – after 45 days of treatment – a significant increase in dim-light visual function, a widening of the visual field, and an increase in central visual acuity. Objective: A workshop has been organized to foster rapid access, investigate the current organizational scenario, and identify a value framework suitable for making gene therapy delivery efficient in terms of efficacy, safety, and sustainability. Methods: The workshop involved 14 experts in clinical genetics, ophthalmology, and vitreoretinal surgery. All experts were subjected to questions related to three topics: diagnostic-therapeutic pathway of IRD patients, essential requirements for gene therapy delivery centres, and standardized model for therapy delivery. All contributions were judged to be of equal value. Results: The panel identified: the steps of the diagnostic pathway to achieve early diagnosis; the essential criteria that delivery centres must possess in terms of experience, multidisciplinary team, and technical equipment; a standardized model for VN administration. A network of few centres was identified as the best organizational model for VN delivery since it would ensure the gaining of experience and clinical excellence. Conclusion: This paper provides a perspective that can be used as a starting point to standardize the diagnostic-therapeutic pathway of IRD patients and define the essential requirements that centres must meet for VN administration.
PubDate: 2023-03-23
DOI: 10.33393/ao.2023.2565
Issue No: Vol. 10 (2023)
- Patient Reported Outcomes from research to clinical practice.
Opportunities and future challenges
Authors: Elisabetta Iannelli
Pages: 60 - 63
Abstract: PROs, highly considered tools for the personalization of therapies and for research, are still scarcely used in clinical practice. Patient organizations played an important role in the validation process of these tools and are engaged in their dissemination and development.
PubDate: 2023-04-18
DOI: 10.33393/ao.2023.2575
Issue No: Vol. 10 (2023)
- Clinical research: the great absentee of the Italian University training
programs'
Authors: Stefano Stabile, Veronica Franchina, Rosita Cenna, Valentina Sinno, Francesca Mannozzi, Irene Federici, Sara Testoni, Celeste Cagnazzo
Pages: 64 - 68
Abstract: Background: In an era where clinical trials have become more and more complex and regulatory authorities impose very high quality standards, the education of clinical research professionals becomes crucial. As one of ICH-GCP guiding principles, adequate training should be ensured and included in educational programs. Methods: In 2021, the Italian Group of Data Managers and Clinical Research Coordinators shared among professionals involved in clinical research an online survey aimed at investigating quality and characteristics of clinical research training provided during undergraduate and postgraduate Italian programs. Results: The survey was completed by 280 professionals: 178 study coordinators, 29 clinical research associates, 20 project managers, 7 study nurses, and 44 others. The majority were 25-45 years old (n = 242, 86.4%), worked at experimental sites (n = 211, 75.4%), and almost all (n = 252, 90.0%) had at least a master’s degree, mainly in biology/biotechnology (n = 162, 57.9%) and pharmacy (n = 64, 22.9%). Clinical research education during the degree courses was considered poor by 73.6% (n = 206). The knowledge on clinical research professional world at the time of graduation was considered poor by 71.1% of participants (n = 199), like the knowledge of related career opportunities (71.1%, n = 199, poor). According to 85.0% of professionals (n = 238) additional postgraduate trainings were needed, mainly university master courses (47.50%, n = 133) and private institution courses (47.86%, n = 134). Postgraduate trainings were considered very useful by 71.4% (n = 200) of responders. Conclusion: Our data suggest undergraduate programs on clinical research education failing at providing even the basic information on clinical research. Therefore, most professionals resort to specific additional postgraduate courses.
PubDate: 2023-04-28
DOI: 10.33393/ao.2023.2566
Issue No: Vol. 10 (2023)
- Cost-minimization analysis of HYQVIA® in the treatment of primary
immunodeficiency disease (PID) and secondary immunodeficiency disease
(SID) in Italy
Authors: Roberto Ravasio, Silvia Ripoli
Pages: 69 - 77
Abstract: Background: Primary (PID) or secondary (SID) immunodeficiencies are diseases caused by quantitative and/or functional alterations of the different mechanisms involved in the innate and adaptive immune response. This economic evaluation was conducted to compare the cost of treatment of HYQVIA® (hyaluronidase-facilitated subcutaneous infusion of immunoglobulin, fSCIG) compared to intravenous immunoglobulin (IVIG) or subcutaneous immunoglobulin (SCIG), currently reimbursed in Italy, in the treatment of PIDs or SIDs. Methods: A cost-minimisation analysis was carried out, considering the hospital’s perspective. The direct medical costs (cost of immunoglobulins and cost of administration) were assessed. The analysis was conducted considering one year (52 weeks) time horizon. The reference population included adult patients with PID with impaired antibody production or adult patients with SID with severe or recurrent infections, ineffective antimicrobial treatment and documented specific antibody defect (PSAF) or serum IgG level < 4 g/L. Results: In the maintenance treatment of PID, HYQVIA® (€ 20,020.00) was the therapeutic alternative with the lowest mean annual cost compared to HIZENTRA® (€ 22,165.19) and VENITAL® (€ 24,967.68). Moreover, in the maintenance treatment of SIDs, HYQVIA® (€ 17,160.00) was the cost-saving therapeutic alternative compared to VENITAL® (€ 22,107.68). A sensitivity analysis confirmed the base case results. Conclusion: Due to lower costs of administration and different scheme of administration, HYQVIA® was a cost-saving alternative to SCIG e IVIG in the treatment of PID and to IVIG in the treatment of SID
PubDate: 2023-05-02
DOI: 10.33393/ao.2023.2584
Issue No: Vol. 10 (2023)
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