{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T03:52:10Z","timestamp":1789617130423,"version":"build-2803163510"},"reference-count":45,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Ludwig Boltzmann Institute for Health Technology Assessment","award":["n\/a"],"award-info":[{"award-number":["n\/a"]}]}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Journal of Market Access &amp; Health Policy"],"published-print":{"date-parts":[[2019,1,1]]},"DOI":"10.1080\/20016689.2019.1600939","type":"journal-article","created":{"date-parts":[[2019,4,19]],"date-time":"2019-04-19T09:07:30Z","timestamp":1555664850000},"page":"1600939","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":32,"title":["Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption"],"prefix":"10.3390","volume":"7","author":[{"given":"Claudia","family":"Eder","sequence":"first","affiliation":[{"name":"Ludwig Boltzmann Institute for Health Technology Assessment, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Wild","sequence":"additional","affiliation":[{"name":"Ludwig Boltzmann Institute for Health Technology Assessment, Vienna, Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2019,4,19]]},"reference":[{"key":"CIT0001","author":"European Commission","journal-title":"Off J Eur Union"},{"issue":"7","key":"CIT0002","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1007\/s00103-011-1310-5","volume":"54","author":"Buljovcic Z.","year":"2011","journal-title":"Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz"},{"key":"CIT0003","unstructured":"European Medicines Agency. Orphan designation: overview. [cited 2018 Nov 21]: Available from: https:\/\/www.ema.europa.eu\/en\/human-regulatory\/overview\/orphan-designation-overview."},{"key":"CIT0004","unstructured":"gov.uk, GUIDANCE ON THE UK\u2019S ARRANGEMENTS UNDER THE HOSPITAL EXEMPTION SCHEME. [cited 2018 21.Nov]. Available from: https:\/\/assets.publishing.service.gov.uk\/government\/uploads\/system\/uploads\/attachment_data\/file\/397738\/Guidance_on_the_UK_s_arrangements_under_the_hospital_exemption_scheme.pdf"},{"key":"CIT0005","doi-asserted-by":"publisher","DOI":"10.1002\/stem.1517"},{"key":"CIT0006","unstructured":"European Medicines Agency. First two CAR-T cell medicines recommended for approval in the European Union. [cited 2018 21.Nov]. Available from: https:\/\/www.ema.europa.eu\/en\/news\/first-two-car-t-cell-medicines-recommended-approval-european-union"},{"key":"CIT0007","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-10-3126"},{"key":"CIT0008","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2015.09.1294"},{"key":"CIT0009","doi-asserted-by":"publisher","DOI":"10.1016\/j.stem.2016.08.012"},{"key":"CIT0010","unstructured":"European Medicines Agency., MACI. 2014. [cited 2018 Nov 21]. Available from: http:\/\/www.ema.europa.eu\/ema\/index.jsp?curl=pages\/medicines\/human\/referrals\/Maci\/human_referral_000380.jsp&mid=WC0b01ac05805c516f"},{"key":"CIT0011","unstructured":"European Medicines Agency. Closure of EU manufacturing site for MACI. 2014. [cited 2018 Nov 21]. Available from: http:\/\/www.ema.europa.eu\/docs\/en_GB\/document_library\/Referrals_document\/Maci_20\/WC500173680.pdf"},{"key":"CIT0012","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.14"},{"key":"CIT0013","unstructured":"Glybera (alipogene tiparvovec) for Treatment of Lipoprotein Lipase Deficiency (LPLD). [cited 2018 18.Aug]. Available from: https:\/\/www.drugdevelopment-technology.com\/projects\/glybera-alipogene-tiparvovec-treatment-lipoprotein-lipase-deficiency-lpld\/."},{"key":"CIT0014","unstructured":"World\u00b4s fist gene therapy to be withdrawn in (sic!) from market in Europe. 2017 [cited 2018 Aug 16]. Available from: https:\/\/sciencebusiness.net\/news\/80248\/World\u2019s-first-gene-therapy-to-be-withdrawn-in-from-market-in-Europe."},{"key":"CIT0015","doi-asserted-by":"publisher","DOI":"10.1016\/j.reth.2014.10.001"},{"key":"CIT0016","unstructured":"European Medicines Agency, CHMP summary of positive opinion for Luxturna in Opinion. 2018. [cited 2018 Aug 18]. Available from: http:\/\/www.ema.europa.eu\/docs\/en_GB\/document_library\/Summary_of_opinion_-_Initial_authorisation\/human\/004451\/WC500255715.pdf"},{"key":"CIT0017","volume-title":"57th Annual Meeting of the Anerican Society of Hematology","year":"2015"},{"key":"CIT0018","unstructured":"bluebirdbio, bluebird bio presents updated data from phase 2\/3 starbeam study of investigational lenti-D\u2122 gene therapy for CALD and initial data from observational study ALD-103 of allogeneic hematopoietic stem cell transplant in CALD at 2018 SSIEM. 2018. [cited 2018 Aug 18]. Available from: https:\/\/www.businesswire.com\/news\/home\/20180905005434\/en\/bluebird-bio-Presents-Updated-Data-Phase-23"},{"key":"CIT0019","unstructured":"Cytori, Enrollment completed in randomized clinical trial of Habeo\u2122 cell therapy for scleroderma and impaired hand function.[cited 2018 18.Aug]. Available from: http:\/\/ir.cytori.com\/investor-relations\/news\/news-details\/2018\/Enrollment-Completed-in-Randomized-Clinical-Trial-of-Habeo-Cell-Therapy-for-Scleroderma-and-Impaired-Hand-Function\/default.aspx"},{"key":"CIT0020","unstructured":"Cytori, Cytori announces top-line 24- and 48-Week Results from the STAR Trial of Habeo\u2122 cell therapy in patients with scleroderma. [cited 2018 Aug 18]. Available from: http:\/\/ir.cytori.com\/investor-relations\/news\/news-details\/2017\/Cytori-Announces-Top-Line-24\u2013and-48-Week-Results-from-the-STAR-Trial-of-Habeo-Cell-Therapy-in-Patients-with-Scleroderma\/default.aspx"},{"key":"CIT0021","unstructured":"Cytori launches managed access programme for ECCS-50 for scleroderma. 2016 [cited 2018 Sept 27]. Available from: https:\/\/www.europeanpharmaceuticalreview.com\/news\/37778\/cytori-launches-managed-access-programme-eccs-50-scleroderma\/."},{"key":"CIT0022","unstructured":"Histogenics, Neocart phase III clinical trial results call. 2018. Presented 2018 Sept 5"},{"key":"CIT0023","unstructured":"BusinessWire. Juno Therapeutics Presents Updated TRANSCEND NHL 001 trial data demonstrating high durable response rates in patients with relapsed or refractory CD19+ Aggressive non-hodgkin lymphoma. 2017 [cited 2018 Oct 1]. Available from: https:\/\/www.businesswire.com\/news\/home\/20170605005425\/en\/Juno-Therapeutics-Presents-Updated-TRANSCEND-NHL-001."},{"key":"CIT0024","unstructured":"bb2121: is CAR T-cell therapy moving to Myeloma? 2018 [cited 2018 Oct 2]. Available from: https:\/\/www.ashclinicalnews.org\/on-location\/bb2121-car-t-cell-therapy-moving-myeloma\/."},{"key":"CIT0025","unstructured":"bio, b. Making hope a reality \u2013 bluebird style. 2018 [cited 2018 Oct 2]. Available from: http:\/\/investor.bluebirdbio.com\/encrypt\/files?file=nasdaq_kms\/assets\/2018\/03\/07\/11-53-56\/BLUECompanyOverviewJanuary2018.pdf&file_alias=10561."},{"key":"CIT0026","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2017.12.595"},{"key":"CIT0027","unstructured":"Biotherapeutics A, Expanded access protocol for tabelecleucel in subjects with EBV-associated viremia or malignancies. 2016. [cited 2018 Oct 2]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02822495?term=ATA129&rank=3"},{"key":"CIT0028","unstructured":"Sharon W High objective response rate, OS seen with ATA129 in PTLD. in Hematology News. 2018. [cited 2018 Oct 2]. Available from: https:\/\/www.mdedge.com\/hematologynews\/article\/159375\/aggressive-lymphomas\/high-objective-response-rate-os-seen-ata129-ptld"},{"key":"CIT0029","first-page":"1165","volume":"9","author":"Meyerson C","year":"2015","journal-title":"Clin Ophthalmol"},{"key":"CIT0030","unstructured":"Rexgenero, REX-001. [cited 2.Oct.2018] Available from: http:\/\/www.rexgenero.com\/rex-001\/"},{"key":"CIT0031","unstructured":"Rexgenero Ltd., The efficacy and safety of REX-001 to treat ischemic ulcers in subjects with CLI rutherford category 5 and DM. 2017. [cited 2018 Oct 2]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03174522"},{"key":"CIT0032","unstructured":"Athersys inc, Athersys announces enrollment of first patient in masters-2 phase 3 study of multistem\u00ae treatment for ischemic stroke. 2018, [cited 2018 Oct 2]. Available from: http:\/\/www.athersys.com\/news-releases\/news-release-details\/athersys-announces-enrollment-first-patient-masters-2-phase-3"},{"key":"CIT0033","unstructured":"Athersys inc., Athersys announces results from phase 2 study of multistem(R) cell therapy for ulcerative colitis. 2014. [cited 2018 Oct 2]. Available from: http:\/\/www.athersys.com\/news-releases\/news-release-details\/athersys-announces-results-phase-2-study-multistemr-cell-therapy"},{"key":"CIT0034","unstructured":"Athersys inc., Athersys announces positive results from phase I study of multistem(R) in Heart Attack Patients. 2010. [cited 2018 Oct 2]. Available from: http:\/\/www.athersys.com\/news-releases\/news-release-details\/athersys-announces-positive-results-phase-i-study-multistemr"},{"key":"CIT0035","unstructured":"Athersys inc., A., athersys announces positive results of multistem(r) clinical trial for hematopoietic stem cell transplant support and prevention of graft-versus-host disease. 2012. [cited 2018 Oct 2]. Available from: http:\/\/www.athersys.com\/news-releases\/news-release-details\/athersys-announces-positive-results-multistemr-clinical-trial"},{"key":"CIT0036","unstructured":"Pluristem. PLX products. [cited 2.Oct.2018]; Available from: http:\/\/www.pluristem.com\/placental-expanded-plx-products\/."},{"key":"CIT0037","unstructured":"European Commission., Hospital exemption for ATMPs (implementation of Art 28 (2)of Regulation 1394\/2007): update on feedback received by the Commission, in PHARM 608. European Commission."},{"key":"CIT0038","unstructured":"European Medicines Agency. Cerepro: withdrawal of the marketing authorisation application. 2010 [cited 2018 Nov 21]. Available from: https:\/\/www.ema.europa.eu\/documents\/other\/withdrawal-letter-cerepro_en-0.pdf."},{"issue":"12","key":"CIT0039","first-page":"A61","volume":"28","author":"Seimetz D","year":"2017","journal-title":"Hum Gene Ther"},{"key":"CIT0040","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2017.08.1825"},{"key":"CIT0041","volume-title":"26th annual EuroMeeting","author":"Salmikangas P","year":"2014"},{"key":"CIT0042","unstructured":"Novartis, Novartis announces positive CHMP opinion for one-time gene therapy Luxturna\u00ae to treat children and adults with rare inherited retinal disease. 2018. [cited 2018 Mar 10]. Available from: https:\/\/www.novartis.com\/news\/media-releases\/novartis-announces-positive-chmp-opinion-one-time-gene-therapy-luxturna-treat-children-and-adults-rare-inherited-retinal-disease"},{"key":"CIT0043","unstructured":"bluebirdbio, bluebird bio Announces European medicines agency\u2019s acceptance of marketing authorization application for lentiglobin\u2122 gene therapy for the treatment of transfusion-dependent \u03b2-thalassemia. 2018. [cited 2019 Mar 10]. Available from: http:\/\/investor.bluebirdbio.com\/news-releases\/news-release-details\/bluebird-bio-announces-european-medicines-agencys-acceptance"},{"key":"CIT0044","unstructured":"Pharma K, Kiadis Pharma on track with European regulatory review for ATIR101. 2018. [cited 2019 Mar 10]. Available from: https:\/\/www.kiadis.com\/kiadis-pharma-on-track-with-european-regulatory-review-for-atir101\/"},{"key":"CIT0045","unstructured":"European Medicines Agency, European Medicines Agency decision P\/0290\/2018. [cited 10.Mar.2019]Available from: https:\/\/www.ema.europa.eu\/en\/documents\/pip-decision\/p\/0290\/2018-ema-decision-12-september-2018-acceptance-modification-agreed-paediatric-investigation-plan_en.pdf"}],"container-title":["Journal of Market Access &amp; Health Policy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/tandfonline.com\/doi\/pdf\/10.1080\/20016689.2019.1600939","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/20016689.2019.1600939","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,18]],"date-time":"2024-01-18T09:02:43Z","timestamp":1705568563000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/search?q=10.1080\/20016689.2019.1600939"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,1,1]]},"references-count":45,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2019,1,1]]}},"alternative-id":["10.1080\/20016689.2019.1600939"],"URL":"https:\/\/doi.org\/10.1080\/20016689.2019.1600939","relation":{},"ISSN":["2001-6689"],"issn-type":[{"value":"2001-6689","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,1,1]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=zjma20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=zjma20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2018-12-03","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-03-10","order":1,"name":"revised","label":"Revised","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-03-18","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-04-19","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}