{"id":7866,"date":"2023-06-15T11:26:08","date_gmt":"2023-06-15T09:26:08","guid":{"rendered":"https:\/\/www.icta.fr\/cas-clients\/long-term-safety-evaluation-of-a-medicinal-product-in-inherited-haematological-disorder-in-europe\/"},"modified":"2026-01-14T10:17:17","modified_gmt":"2026-01-14T09:17:17","slug":"long-term-safety-evaluation-of-a-medicinal-product-in-inherited-haematological-disorder-in-europe","status":"publish","type":"casclients","link":"https:\/\/www.icta.fr\/fr\/cas-clients\/long-term-safety-evaluation-of-a-medicinal-product-in-inherited-haematological-disorder-in-europe\/","title":{"rendered":"Long-Term Safety Evaluation of a Medicinal Product in Inherited Haematological Disorder in Europe"},"content":{"rendered":"\n
<\/div>
\n

OBJECTIVES<\/h3>\n\n\n\n
\n
\n

PRIMARY OBJECTIVES<\/h4>\n\n\n\n

<\/p>\n\n\n\n

Long-term safety profile of the test product: poorly documented or unknown risks (not listed in the product information).<\/p>\n\n\n\n

    \n
  • <\/li>\n<\/ul>\n<\/div>\n\n\n\n
    \n

    SECONDARY OBJECTIVES<\/h4>\n\n\n\n

    <\/p>\n\n\n\n

      \n
    • Changes in fertility status in males and females<\/li>\n\n\n\n
    • Pregnancy outcomes<\/li>\n\n\n\n
    • Potential barriers to the compliance to the prescription of the test product<\/li>\n\n\n\n
    • Switch or combination with new treatments<\/li>\n\n\n\n
    • Complications linked to the disease<\/li>\n\n\n\n
    • Quality of life of adult patients<\/li>\n<\/ul>\n<\/div>\n<\/div>\n\n\n\n
      \n
      \n

      SCIENTIFIC and DATA VALORISATION<\/h3>\n\n\n\n
        \n
      • 2 Interim Clinical Study Reports (study ongoing)<\/li>\n\n\n\n
      • 2 DSMB<\/li>\n\n\n\n
      • Overall excellent client satisfaction<\/li>\n<\/ul>\n<\/div>\n\n\n\n
        \n

        Key factors <\/strong>
        of success<\/strong><\/h3>\n\n\n\n
          \n
        • ICTA\u2019s expertise in site selection and management<\/li>\n\n\n\n
        • ICTA\u2019s responsiveness<\/li>\n\n\n\n
        • ICTA\u2019s expertise in Data Management<\/li>\n<\/ul>\n<\/div>\n<\/div>\n\n\n\n
          \n
          \n

          LESSONS LEARNED
          to speed up the start-up phase of the next study<\/h3>\n\n\n\n
            \n
          • Importance of close collaboration between ICTA and Sponsor and active involvement of Sponsor during the course of the study, in keeping the sites engaged<\/li>\n\n\n\n
          • Due to the new GDPR regulation and the new French EC submission process, the site selection should be anticipated in order to collect CV in parallel of the protocol finalization, and the data flow should be described in a separate document for the EC submission<\/li>\n\n\n\n
          • Implement an electronic signature system of the site financial contract to optimize its signature\/collection<\/li>\n\n\n\n
          • Adapt the study objectives according to the reality on the site (content of medical records and routine care visits)<\/li>\n\n\n\n
          • Define the key data and PV requirements well beforehand.<\/li>\n\n\n\n
          • Importance of setting up a steering committee to provide expert clinical guidance and a high standard of scientific quality.<\/li>\n<\/ul>\n<\/div>\n<\/div>\n\n\n\n

            CHALLENGES<\/h3>\n\n\n\n
            KEY CHALLENGES<\/th>ICTA’s SOLUTIONS<\/th>ICTA’s ACHIEVEMENTS<\/th><\/tr><\/thead>
            Sites selection<\/strong>

            Multicenter study carried out in several European countries<\/td>            		ICTA\u2019s longstanding partners in European countries mandated for:

            \u25aa Regulatory submissions
            \u25aa Sites management in 4 European countries: France, Italy, Germany, Greece<\/strong><\/td>            		\u25aa 120 contacted sites
            \u25aa 88 selected sites
            \u25aa 71 active sites
            \u25aa 12 ready-to-include sites
            \u25ba 69% of sites were willing to participate in the study.<\/td><\/tr>
            Site contract management<\/strong>

            \u25aa Several types of contract to be managed likely to lead to errors
            \u25aa Follow-up of sites who agreed to finalize the financial contract<\/td>
            \u25aa Sound explanations to sites
            \u25aa Close follow-up of site contacts and site reminders to obtain required documents and signature
            . Management of physician data privacy<\/td>            		A total of 83 contracts were sent
            \u25aa 83 financial contracts were in conformity with requirements and were signed (\u21d4 100% of active sites<\/strong>)<\/td><\/tr>
            Patients recruitment<\/strong>

            N = 2,500 patients to be enrolled over a planned 5-year period<\/td>            		\u25aa Close follow-up of site (data collection visits, quality control visits, remote monitoring and close-out)
            \u25aaClose collaboration with the Sponsor who is actively involved with site coordination.<\/td>            		A total of 71 active sites <\/strong>recruited at least one patient
            To date:<\/strong>
            \u25aa  1,738 <\/strong>patients recruited after 2.5 years of recruitment (\u21d4 70% of the target sample size<\/strong>)
            \u25aa Discussion to reduce recruitment duration<\/strong><\/td><\/tr>
            Data collection and monitoring<\/strong>

            \u25aa Study burden
            \u25aa Lack of time and\/or resource
            <\/td>            		\u25aa Keep the study simple and easy
            \u25aa Focused on must-have data in the eCRF
            \u25aa Data in accordance with routine care practice data
            \u25aa ICTA resources provided to sites for data entry<\/td>            		Data quality ensured<\/strong>

            \u25aa Monthly report of edit checks and posted queries (including status)
            \u25aa Regular remote monitoring<\/td><\/tr><\/tbody><\/table><\/figure>\n<\/div><\/section>\n\n\n\n
              \n
            • <\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":15,"featured_media":10781,"template":"","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","inline_featured_image":false,"_uag_custom_page_level_css":"","kk_blocks_editor_width":"","_kiokenblocks_attr":"","_kiokenblocks_dimensions":"","footnotes":""},"categories":[],"class_list":["post-7866","casclients","type-casclients","status-publish","has-post-thumbnail","hentry"],"acf":[],"uagb_featured_image_src":{"full":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false],"thumbnail":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder-128x88.jpg",128,88,true],"medium":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder-300x150.jpg",300,150,true],"medium_large":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false],"large":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false],"1536x1536":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false],"2048x2048":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false],"post-thumbnail":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false],"wpm_taille_1":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder-364x250.jpg",364,250,true],"wpm_taille_slider":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder-240x145.jpg",240,145,true],"gform-image-choice-sm":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",300,150,false],"gform-image-choice-md":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",400,200,false],"gform-image-choice-lg":["https:\/\/www.icta.fr\/wp-content\/uploads\/haematological-disorder.jpg",500,250,false]},"uagb_author_info":{"display_name":"Manon ROBERT","author_link":"https:\/\/www.icta.fr\/fr\/author\/mrobert-icta21\/"},"uagb_comment_info":0,"uagb_excerpt":null,"_links":{"self":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients\/7866","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients"}],"about":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/types\/casclients"}],"author":[{"embeddable":true,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/users\/15"}],"version-history":[{"count":9,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients\/7866\/revisions"}],"predecessor-version":[{"id":10793,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients\/7866\/revisions\/10793"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/media\/10781"}],"wp:attachment":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/media?parent=7866"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/categories?post=7866"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}