{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T03:16:06Z","timestamp":1740107766565,"version":"3.37.3"},"reference-count":33,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2022,9,30]],"date-time":"2022-09-30T00:00:00Z","timestamp":1664496000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2022,9,30]],"date-time":"2022-09-30T00:00:00Z","timestamp":1664496000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100003385","name":"Georg-August-Universit\u00e4t G\u00f6ttingen","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100003385","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ethik Med"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:title>Zusammenfassung<\/jats:title><jats:p>Compassionate Use beschreibt die Anwendung zulassungs\u00fcberschreitender Arzneimittel f\u00fcr Patient*innengruppen, die an einer lebensbedrohlichen oder zu einer schweren Behinderung f\u00fchrenden Erkrankung leiden, ohne dass eine alternative Therapieoption besteht. An \u00c4rzt*innen vorbei werden solche Programme ausschlie\u00dflich von Pharmaunternehmen initiiert, was viele ethische Konflikte mit sich bringt. Eine neue Dimension erreichte das 2020 gestartete Programm f\u00fcr <jats:italic>Onasemnogenum abeparvovecum<\/jats:italic> zur Therapie von Spinaler Muskelatrophie bei Kindern, welches die Krankheit nach nur einmaliger Gabe stoppen sollte. Die globale Allokation von nur 100 zur Verf\u00fcgung gestellten Dosierungen per Losverfahren stellte ein Novum bei der Allokation in Compassionate Use-Programmen dar und wurde vielfach kritisiert. Diese Arbeit untersucht m\u00f6gliche alternative Allokationsprinzipien auf das Beispiel von <jats:italic>Onasemnogenum abeparvovecum<\/jats:italic>. F\u00fcr jedes in Frage kommende Prinzip der Allokation medizinischer G\u00fcter bestehen Aspekte, die im Hinblick auf die dr\u00e4ngende Zeit und die globale Verteilung bedacht werden m\u00fcssen. Dies l\u00e4sst einige Prinzipien wie First-Come-First-Served vernachl\u00e4ssigbar erscheinen. Verbliebene Prinzipien werden hierarchisch geordnet, um einen Algorithmus abzuleiten, der eine Alternative zum Losverfahren darstellen kann. Eine Kombination von Teilnahmebereitschaft bei Forschung, Dringlichkeit und Erfolgsaussicht (bezogen auf die Existenz supportiver Therapieoptionen) kann in \u00e4hnlichen F\u00e4llen bei zuk\u00fcnftigen globalen Compassionate Use-Programmen bei Kindern erwogen werden. Da universelle Algorithmen nur schwer definierbar sind, sollten Allokationskriterien in jedem Fall durch ein unabh\u00e4ngiges Expert*innengremium diskutiert werden. Sowohl die Konstitution eines solchen Gremiums sowie deren verpflichtende Konsultierung sind gefordert, um f\u00fcr Entlastung aller Beteiligten zu sorgen und Willk\u00fcr vorzubeugen.<\/jats:p>","DOI":"10.1007\/s00481-022-00722-w","type":"journal-article","created":{"date-parts":[[2022,9,30]],"date-time":"2022-09-30T13:09:33Z","timestamp":1664543373000},"page":"497-514","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Allokation von einmalig zu applizierenden Arzneimitteln bei Kindern in globalen Compassionate Use-Programmen","Allocation of single-use drugs in children in global compassionate use programs"],"prefix":"10.1007","volume":"34","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4302-9167","authenticated-orcid":false,"given":"Clemens","family":"Miller","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2022,9,30]]},"reference":[{"key":"722_CR1","unstructured":"AveXis (2019) Letter to the SMA community. https:\/\/www.novartis.com\/sites\/www.novartis.com\/files\/global-map-community-statement-on-avxs-101.pdf. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR2","unstructured":"BeNeLuxA Initiative (2020) No \u201clottery for life\u201d \u2013 Statement by Beneluxa Health Ministers addressing the global managed access program designed by Novartis and Avexis. https:\/\/beneluxa.org\/news1. Zugegriffen: 22. M\u00e4rz 2020"},{"key":"722_CR3","first-page":"82","volume":"38","author":"H Blasius","year":"2014","unstructured":"Blasius\u00a0H (2014) Arzneimittelzulassung: Teil 2: Europ\u00e4isches Zulassungssystem, \u00c4nderungen an Arzneimitteln nach der Zulassung. Dtsch Apoth\u00a0Z 38:82","journal-title":"Dtsch Apoth Z"},{"key":"722_CR4","doi-asserted-by":"publisher","first-page":"1293","DOI":"10.1007\/s00112-015-0004-8","volume":"163","author":"S Borell","year":"2015","unstructured":"Borell\u00a0S, Pechmann\u00a0A, Kirschner\u00a0J (2015) Spinale Muskelatrophie. Monatsschr Kinderheilkd 163:1293\u20131304. https:\/\/doi.org\/10.1007\/s00112-015-0004-8","journal-title":"Monatsschr Kinderheilkd"},{"key":"722_CR5","doi-asserted-by":"publisher","first-page":"136","DOI":"10.1186\/s12916-017-0910-9","volume":"15","author":"J Borysowski","year":"2017","unstructured":"Borysowski\u00a0J, Ehni\u00a0HJ, G\u00f3rski\u00a0A (2017) Ethics review in compassionate use. BMC Med 15:136. https:\/\/doi.org\/10.1186\/s12916-017-0910-9","journal-title":"BMC Med"},{"key":"722_CR6","doi-asserted-by":"publisher","first-page":"979","DOI":"10.1001\/jama.2016.0416","volume":"315","author":"AL Caplan","year":"2016","unstructured":"Caplan\u00a0AL, Ray\u00a0A (2016) The ethical challenges of compassionate use. JAMA 315:979\u2013980. https:\/\/doi.org\/10.1001\/jama.2016.0416","journal-title":"JAMA"},{"key":"722_CR7","doi-asserted-by":"publisher","first-page":"761","DOI":"10.1136\/medethics-2016-103917","volume":"44","author":"AL Caplan","year":"2018","unstructured":"Caplan\u00a0AL, Teagarden\u00a0JR, Kearns\u00a0L, Bateman-House\u00a0AS, Mitchell\u00a0E, Arawi\u00a0T, Upshur\u00a0R, Singh\u00a0I, Rozynska\u00a0J, Cwik\u00a0V, Gardner\u00a0SL (2018) Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials. J\u00a0Med Ethics 44:761\u2013767. https:\/\/doi.org\/10.1136\/medethics-2016-103917","journal-title":"J Med Ethics"},{"key":"722_CR8","unstructured":"Chapman M (2019) Up to 100 free Zolgensma treatments going out worldwide in 2020 under access program. https:\/\/smanewstoday.com\/news-posts\/2019\/12\/28\/novartis-global-access-program-for-zolgensma\/. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR9","unstructured":"Deutsches \u00c4rzteblatt (2020) Medizinethiker ruft Politik auf, Medikamentenpreise zu deckeln. https:\/\/www.aerzteblatt.de\/nachrichten\/109188\/Medizinethiker-ruft-Politik-auf-Medikamentenpreise-zu-deckeln. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR10","unstructured":"DIVI \u2013 Deutsche Interdisziplin\u00e4re Vereinigung f\u00fcr Intensiv- und Notfallmedizin (2020) Schwerkranke Kinder: Intensiv- und Notfallmediziner kritisieren Verlosung von Medikamenten gegen t\u00f6dliche Muskelkrankheit. https:\/\/www.divi.de\/joomlatools-files\/docman-files\/pressemeldungen-nach-themen\/kinder-jugendliche-und-familie\/200217-divi-pressemeldung-divi-kritisiert-Medikamenten-Verlosung-Zolgensma.pdf. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR11","doi-asserted-by":"publisher","first-page":"1386","DOI":"10.1056\/NEJMc1700103","volume":"376","author":"NS Downing","year":"2017","unstructured":"Downing\u00a0NS, Zhang\u00a0AD, Ross\u00a0JS (2017) Regulatory review of new therapeutic agents \u2013 FDA versus EMA, 2011\u20132015. N\u00a0Engl J Med 376:1386\u20131387. https:\/\/doi.org\/10.1056\/NEJMc1700103","journal-title":"N Engl J Med"},{"key":"722_CR12","doi-asserted-by":"publisher","first-page":"m580","DOI":"10.1136\/bmj.m580","volume":"368","author":"O Dyer","year":"2020","unstructured":"Dyer\u00a0O (2020) Health ministers condemn Novartis lottery for Zolgensma, the world\u2019s most expensive drug. BMJ 368:m580. https:\/\/doi.org\/10.1136\/bmj.m580","journal-title":"BMJ"},{"key":"722_CR13","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMsb2005114","author":"EJ Emanuel","year":"2020","unstructured":"Emanuel\u00a0EJ, Persad\u00a0G, Upshur\u00a0R, Thome\u00a0B, Parker\u00a0M, Glickman\u00a0A, Zhang\u00a0C, Boyle\u00a0C, Smith\u00a0M, Phillips\u00a0JP (2020) Fair allocation of scarce medical resources in the time of Covid-19. N\u00a0Engl J Med. https:\/\/doi.org\/10.1056\/NEJMsb2005114","journal-title":"N Engl J Med"},{"key":"722_CR14","unstructured":"Ettel A (2020) Die Zolgensma-Lotterie offenbart das neue Medikamenten-Dilemma. https:\/\/www.welt.de\/wirtschaft\/article205731629\/Novartis-stoesst-mit-Medikamenten-Losverfahren-Debatte-an.html. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR15","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","volume":"377","author":"RS Finkel","year":"2017","unstructured":"Finkel\u00a0RS, Mercuri\u00a0E, Darras\u00a0BT, Connolly\u00a0AM, Kuntz\u00a0NL, Kirschner\u00a0J, Chiriboga\u00a0CA, Saito\u00a0K, Servais\u00a0L, Tizzano\u00a0E, Topaloglu\u00a0H, Tulinius\u00a0M, Montes\u00a0J, Glanzman\u00a0AM, Bishop\u00a0K, Zhong\u00a0ZJ, Gheuens\u00a0S, Bennett\u00a0CF, Schneider\u00a0E, Farwell\u00a0W, de Vivo\u00a0DC (2017) Nusinersen versus sham control in Infantile-Onset Spinal Muscular Atrophy. N\u00a0Engl J Med 377:1723\u20131732. https:\/\/doi.org\/10.1056\/NEJMoa1702752","journal-title":"N Engl J Med"},{"key":"722_CR16","doi-asserted-by":"publisher","first-page":"1453","DOI":"10.1377\/hlthaff.2014.0122","volume":"33","author":"C Frank","year":"2014","unstructured":"Frank\u00a0C, Himmelstein\u00a0DU, Woolhandler\u00a0S, Bor\u00a0DH, Wolfe\u00a0SM, Heymann\u00a0O, Zallman\u00a0L, Lasser\u00a0KE (2014) Era of faster FDA drug approval has also seen increased black-box warnings and market withdrawals. Health Aff 33:1453\u20131459. https:\/\/doi.org\/10.1377\/hlthaff.2014.0122","journal-title":"Health Aff"},{"key":"722_CR17","doi-asserted-by":"publisher","first-page":"155","DOI":"10.1016\/j.nmd.2009.11.014","volume":"20","author":"AM Glanzman","year":"2010","unstructured":"Glanzman\u00a0AM, Mazzone\u00a0E, Main\u00a0M, Pelliccioni\u00a0M, Wood\u00a0J, Swoboda\u00a0KJ, Scott\u00a0C, Pane\u00a0M, Messina\u00a0S, Bertini\u00a0E, Mercuri\u00a0E, Finkel\u00a0RS (2010) The Children\u2019s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromuscul Disord 20:155\u2013161. https:\/\/doi.org\/10.1016\/j.nmd.2009.11.014","journal-title":"Neuromuscul Disord"},{"key":"722_CR18","series-title":"Bochumer Schriften zum Sozial- und Gesundheitsrecht","doi-asserted-by":"publisher","DOI":"10.5771\/9783845279916","volume-title":"Der Zugang zu noch nicht zugelassenen Arzneimitteln: Rechtsfragen und Regulierungsprobleme","author":"S Huster","year":"2016","unstructured":"Huster\u00a0S, Stadelhoff\u00a0S, Streng-Baunemann\u00a0AF (2016) Der Zugang zu noch nicht zugelassenen Arzneimitteln: Rechtsfragen und Regulierungsprobleme, 1.\u00a0Aufl. Bochumer Schriften zum Sozial- und Gesundheitsrecht, Bd. 21. Nomos, Baden-Baden","edition":"1"},{"key":"722_CR19","doi-asserted-by":"publisher","DOI":"10.3390\/jpm11010045","author":"A Kantor","year":"2021","unstructured":"Kantor\u00a0A, Haga\u00a0SB (2021) The potential benefit of expedited development and approval programs in precision medicine. J\u00a0Pers Med. https:\/\/doi.org\/10.3390\/jpm11010045","journal-title":"J Pers Med"},{"key":"722_CR20","unstructured":"Kaulen H (2020) Per Los zur lebensrettenden Spritze. https:\/\/www.faz.net\/aktuell\/wissen\/medizin-ernaehrung\/umstrittene-zolgensma-therapie-per-los-zur-lebensrettenden-spritze-16627779.html. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR21","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1186\/s12916-016-0568-8","volume":"14","author":"TK Mackey","year":"2016","unstructured":"Mackey\u00a0TK, Schoenfeld\u00a0VJ (2016) Going \u201csocial\u201d to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access. BMC Med 14:17. https:\/\/doi.org\/10.1186\/s12916-016-0568-8","journal-title":"BMC Med"},{"key":"722_CR22","doi-asserted-by":"publisher","first-page":"352","DOI":"10.1016\/j.clinthera.2022.01.001","volume":"44","author":"R Marinus","year":"2022","unstructured":"Marinus\u00a0R, Mofid\u00a0S, Mpandzou\u00a0M, K\u00fchler\u00a0TC (2022) Rolling reviews during COVID-19: The European Union experience in a\u00a0global context. Clin Ther 44:352\u2013363. https:\/\/doi.org\/10.1016\/j.clinthera.2022.01.001","journal-title":"Clin Ther"},{"key":"722_CR23","first-page":"A-362 \/ B-318 \/","volume":"117","author":"T Maybaum","year":"2020","unstructured":"Maybaum\u00a0T (2020) Zolgensma-H\u00e4rtefall-programm: Begrenzung auf dringliche F\u00e4lle vorgeschlagen. Dtsch Arztebl 117:A-362 \/ B\u2011318 \/ C\u2011306","journal-title":"Dtsch Arztebl"},{"key":"722_CR24","unstructured":"Meier N (2016) Wer rettet Klara? http:\/\/www.zeit.de\/2016\/39\/medikamenten-zulassung-heilung-ethikfrage. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR25","doi-asserted-by":"publisher","first-page":"986","DOI":"10.1016\/S0140-6736(19)33178-2","volume":"395","author":"H Naci","year":"2020","unstructured":"Naci\u00a0H, Salcher-Konrad\u00a0M, Kesselheim\u00a0AS, Wieseler\u00a0B, Rochaix\u00a0L, Redberg\u00a0RF, Salanti\u00a0G, Jackson\u00a0E, Garner\u00a0S, Stroup\u00a0TS, Cipriani\u00a0A (2020) Generating comparative evidence on new drugs and devices before approval. Lancet 395:986\u2013997. https:\/\/doi.org\/10.1016\/S0140-6736(19)33178-2","journal-title":"Lancet"},{"key":"722_CR26","doi-asserted-by":"publisher","first-page":"423","DOI":"10.1016\/S0140-6736(09)60137-9","volume":"373","author":"G Persad","year":"2009","unstructured":"Persad\u00a0G, Wertheimer\u00a0A, Emanuel\u00a0EJ (2009) Principles for allocation of scarce medical interventions. Lancet 373:423\u2013431. https:\/\/doi.org\/10.1016\/S0140-6736(09)60137-9","journal-title":"Lancet"},{"key":"722_CR31","doi-asserted-by":"publisher","first-page":"1786","DOI":"10.1056\/NEJMe1712106","volume":"377","author":"AT van der Ploeg","year":"2017","unstructured":"van der Ploeg\u00a0AT (2017) The dilemma of two innovative therapies for spinal muscular atrophy. N\u00a0Engl J Med 377:1786\u20131787. https:\/\/doi.org\/10.1056\/NEJMe1712106","journal-title":"N Engl J Med"},{"key":"722_CR27","doi-asserted-by":"publisher","first-page":"809","DOI":"10.1007\/s00115-019-0761-z","volume":"90","author":"A Saffari","year":"2019","unstructured":"Saffari\u00a0A, Weiler\u00a0M, Hoffmann\u00a0GF, Ziegler\u00a0A (2019) Gentherapien f\u00fcr neuromuskul\u00e4re Erkrankungen (Gene therapies for neuromuscular diseases). Nervenarzt 90:809\u2013816. https:\/\/doi.org\/10.1007\/s00115-019-0761-z","journal-title":"Nervenarzt"},{"key":"722_CR28","doi-asserted-by":"publisher","DOI":"10.1007\/978-3-642-28907-1","volume-title":"Basiswissen Humangenetik","author":"CP Schaaf","year":"2013","unstructured":"Schaaf\u00a0CP, Zschocke\u00a0J (2013) Basiswissen Humangenetik, 2.\u00a0Aufl. Springer, Berlin, Heidelberg","edition":"2"},{"key":"722_CR29","unstructured":"Sucker-Sket K (2019) Zolgensma: Novartis will globales H\u00e4rtefallprogramm starten: 2\u2011Millionen-Dollar-Gentherapeutikum. https:\/\/www.deutsche-apotheker-zeitung.de\/news\/artikel\/2019\/12\/20\/zolgensma-novartis-will-globales-haertefallprogramm-starten\/chapter. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR30","unstructured":"TreatSMA (2019) Zolgensma to be made available globally under a managed-access programme. https:\/\/www.treatsma.uk\/2019\/12\/zolgensma-to-be-made-available-globally-under-a-managed-access-programme\/. Zugegriffen: 30. Apr. 2022"},{"key":"722_CR32","doi-asserted-by":"publisher","first-page":"1358","DOI":"10.1007\/s00115-017-0447-3","volume":"88","author":"K Vill","year":"2017","unstructured":"Vill\u00a0K, Blaschek\u00a0A, Schara\u00a0U, K\u00f6lbel\u00a0H, Hohenfellner\u00a0K, Harms\u00a0E, Olgem\u00f6ller\u00a0B, Walter\u00a0MC, M\u00fcller-Felber\u00a0W (2017) Spinale Muskelatrophie : Zeit f\u00fcr das Neugeborenenscreening? (Spinal muscular atrophy : time for newborn screening?). Nervenarzt 88:1358\u20131366. https:\/\/doi.org\/10.1007\/s00115-017-0447-3","journal-title":"Nervenarzt"},{"key":"722_CR33","unstructured":"Voigt B, Freuler R (2020) \u201eDie Medikamenten-Lotterie ist nicht fair\u201c, sagt Medizinethikerin Tanja Krones. https:\/\/nzzas.nzz.ch\/wissen\/medizin-ethikerin-ueber-zolgesma-medikamenten-lotterie-ist-unfair-ld.1539341. Zugegriffen: 30. Apr. 2022"}],"container-title":["Ethik in der Medizin"],"original-title":[],"language":"de","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00481-022-00722-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00481-022-00722-w\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00481-022-00722-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,11,25]],"date-time":"2022-11-25T22:38:54Z","timestamp":1669415934000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00481-022-00722-w"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,9,30]]},"references-count":33,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2022,12]]}},"alternative-id":["722"],"URL":"https:\/\/doi.org\/10.1007\/s00481-022-00722-w","relation":{},"ISSN":["0935-7335","1437-1618"],"issn-type":[{"type":"print","value":"0935-7335"},{"type":"electronic","value":"1437-1618"}],"subject":[],"published":{"date-parts":[[2022,9,30]]},"assertion":[{"value":"31 May 2022","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 August 2022","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"30 September 2022","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Einhaltung ethischer Richtlinien"}},{"value":"C.\u00a0Miller gibt an, dass kein Interessenkonflikt besteht.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Interessenkonflikt"}},{"value":"F\u00fcr diesen Beitrag wurden vom Autor keine Studien an Menschen oder Tieren durchgef\u00fchrt. F\u00fcr die aufgef\u00fchrten Studien gelten die jeweils dort angegebenen ethischen Richtlinien.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethische Standards"}}]}}