{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:45:13Z","timestamp":1787013913869,"version":"build-2736575974"},"reference-count":0,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2017,9,5]],"date-time":"2017-09-05T00:00:00Z","timestamp":1504569600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Bioethics"],"published-print":{"date-parts":[[2018,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>A balance needs to be struck between facilitating compassionate access to innovative treatments for those in desperate need, and the duty to protect such vulnerable individuals from the harms of untested\/unlicensed treatments. We introduced a principle\u2010based framework (2009) to evaluate such requests and describe its application in the context of recently evolved UK, US and European regulatory processes.<\/jats:p>\n                  <jats:p>24 referrals (20 individual; four group) were received by our quaternary children's hospital Clinical Ethics Committee (CEC) over the 5\u2010year period (2011\u201016). The CEC\u2010rapid response group evaluated individual cases within 48\u2010hours; the main referrers being haematology\/oncology, immunology or transplant services (14). Most requests were for drug\/vaccine\/pre\u2010trial access (13) or biological\/cellular therapies (8). The majority of individual requests were approved (19\/20); neutral or negative opinions were given in 5, including 3 group requests.<\/jats:p>\n                  <jats:p>Recently evolved regulatory processes share common criteria and conditions to our framework including: demonstration of clinical need; sound scientific basis with lack of viable alternative; risks\u2010benefit\/best interests evaluation; arrangements for fully informed consent; no compromise of arrangements to test treatment for licensing purposes; consideration of resource implications. There are differences between individual processes and with our framework, with respect to procedures, scope, application format, costs and obligation to make available all outcome data.<\/jats:p>\n                  <jats:p>Our experience has emphasized the need for an independent, principled, consistent, fair and transparent response to the increasing demand for innovative treatment on a compassionate basis. We believe that there is a need for harmonization of the recent proliferation of regulation and legislation in this area.<\/jats:p>","DOI":"10.1111\/bioe.12381","type":"journal-article","created":{"date-parts":[[2017,9,5]],"date-time":"2017-09-05T09:06:44Z","timestamp":1504602404000},"page":"36-42","source":"Crossref","is-referenced-by-count":18,"title":["Medical Innovation in a Children's Hospital: \u2018\n                    <i>Diseases desperate grown by desperate appliance are relieved, or not at all\u2019<\/i>"],"prefix":"10.1111","volume":"32","author":[{"given":"Vic","family":"Larcher","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helen","family":"Turnham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0919-6882","authenticated-orcid":false,"given":"Joe","family":"Brierley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2017,9,5]]},"container-title":["Bioethics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fbioe.12381","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/bioe.12381","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,29]],"date-time":"2023-09-29T21:43:46Z","timestamp":1696023826000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/bioe.12381"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,9,5]]},"references-count":0,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2018,1]]}},"alternative-id":["10.1111\/bioe.12381"],"URL":"https:\/\/doi.org\/10.1111\/bioe.12381","archive":["Portico"],"relation":{},"ISSN":["0269-9702","1467-8519"],"issn-type":[{"value":"0269-9702","type":"print"},{"value":"1467-8519","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017,9,5]]}}}