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      SARS-CoV2 disrupts clinical research - the role of a rare disease-specific trial network

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          Abstract

          Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) has disrupted clinical trials worldwide [1]. This could delay the approval of new medicines and reduce access to investigational treatments via clinical trials. This particularly impacts patients with rare diseases such as cystic fibrosis (CF).

          Abstract

          Rare disease patients may suffer delayed access to new drugs as SARS-CoV-2 is disrupting clinical trials. Our survey demonstrates that the European cystic fibrosis clinical trials network is ideally placed to track and address such disruption.

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          Preserving Clinical Trial Integrity During the Coronavirus Pandemic

          Mary M. McDermott, Anne Newman (2020)
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            Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease

            Michal Shteinberg, Jennifer L. Taylor-Cousar (2020)
            Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation. While the hope is that these drug compounds will prevent lung damage when started early in life, there is an ongoing need to care for people with advanced lung disease. The focus of this review is the accumulating data from clinical trials and case series regarding the benefits of CFTR modulator therapy in people with advanced pulmonary disease. We address the impact of treatment with ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor on lung function, pulmonary exacerbations, nutrition and quality of life. Adverse events of the different CFTR modulators, as well as the potential for drug–drug interactions, are discussed. CFTR modulators are efficacious in improving lung function and exacerbations, even in advanced CF. However, adverse drug reactions and drug interactions are frequent. http://bit.ly/37aN81E
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              Disease-specific clinical trials networks: the example of cystic fibrosis

              Kris De Boeck, Veerle Bulteel, Isabelle Fajac (2016)
              This article describes the steps of the development and the structure of a disease-specific clinical trials network for cystic fibrosis in Europe. Activities such as reviewing study protocols, feasibility assessments, training and standardizing of procedures, and outcome measurements help to bring high-quality clinical trials to the patients. Cooperation with the pharmaceutical industry, other research networks, patient organizations, and regulatory agencies is very important throughout all activities. Conclusion: The European Cystic Fibrosis Society—Clinical Trials Network facilitates the development of new treatments for a rare disease and could be a prototype for other diseases. What is Known: • Clinical research has led to the first approved treatments targeting the basic Cystic Fibrosis defect.• For a rare disease like Cystic Fibrosis, multicenter international collaboration is needed to obtain solid evidence when testing possible new treatments. What is New: • The Clinical Trials Network established by the European Cystic Fibrosis Society has grown to a fully operational network with well-defined structures, procedures and partnerships.• Standardization of outcome parameters, protocol review, feasibility assessment and other activities help to develop high quality, efficient, relevant and feasible clinical trials, with the aim to bring new treatments to the patients.
              Article 0 comments Cited 6 times – based on 0 reviews     Review now
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                Author and article information

                Journal
                Journal ID (nlm-ta): Eur Respir J
                Journal ID (iso-abbrev): Eur. Respir. J
                Journal ID (publisher-id): ERJ
                Journal ID (hwp): erj
                Title: The European Respiratory Journal
                Publisher: European Respiratory Society
                ISSN (Print): 0903-1936
                ISSN (Electronic): 1399-3003
                Publication date (Electronic): 07 August 2020
                Publication date PMC-release: 07 August 2020
                Electronic Location Identifier: 2002114
                Affiliations
                [1 ]CF Centre Cologne, Children's Hospital, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany
                [2 ]European Cystic Fibrosis Society, Karup, Denmark
                [3 ]Centre for Experimental Medicine, Queen's University Belfast, Belfast, UK
                [4 ]CF Center and Lung Transplant Unit, University Hospitals Leuven, Leuven, Belgium
                Author notes
                Silke van Koningsbruggen-Rietschel. E-mail: Silke.vanKoningsbruggen@ 123456uk-koeln.de
                Article
                Publisher ID: ERJ-02114-2020
                DOI: 10.1183/13993003.02114-2020
                PMC ID: 7411273
                PubMed ID: 32764115
                SO-VID: b5f3d885-0678-46c0-81f8-55aaea1ef2ae
                Copyright © Copyright ©ERS 2020
                License:

                This article is open access and distributed under the terms of the Creative Commons Attribution Non-Commercial Licence 4.0.

                History
                Date received : 2 June 2020
                Date accepted : 20 July 2020
                Categories
                Subject: Correspondence

                ScienceOpen disciplines: Respiratory medicine
                Data availability:
                ScienceOpen disciplines: Respiratory medicine

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