{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T17:21:10Z","timestamp":1792516870765,"version":"4.5.0"},"reference-count":74,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,11,8]],"date-time":"2021-11-08T00:00:00Z","timestamp":1636329600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2021,12]]},"DOI":"10.1016\/j.omtm.2021.11.003","type":"journal-article","created":{"date-parts":[[2021,11,10]],"date-time":"2021-11-10T21:10:40Z","timestamp":1636578640000},"page":"606-618","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":63,"special_numbering":"C","title":["Current landscape of clinical development and approval of advanced therapies"],"prefix":"10.1016","volume":"23","author":[{"given":"Carolina","family":"Iglesias-Lopez","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonia","family":"Agust\u00ed","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antoni","family":"Vallano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Merce","family":"Obach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2021.11.003_bib121","article-title":"Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States","volume":"10:921","author":"Iglesias-Lopez","year":"2019","journal-title":"Front Pharmacol."},{"key":"10.1016\/j.omtm.2021.11.003_bib1","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1016\/j.jcyt.2020.11.008","article-title":"Comparison of regulatory pathways for the approval of advanced therapies in the European Union and the United States","volume":"23","author":"Iglesias-Lopez","year":"2021","journal-title":"Cytotherapy"},{"key":"10.1016\/j.omtm.2021.11.003_bib3","doi-asserted-by":"crossref","first-page":"1431","DOI":"10.18609\/cgti.2019.147","article-title":"Clinical trials of advanced therapy investigational medicinal products in Spain: preparing for the European clinical trials regulation","volume":"5","author":"Alamo","year":"2019","journal-title":"Cell Gene Ther. Insights"},{"key":"10.1016\/j.omtm.2021.11.003_bib120","article-title":"Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union","volume":"12:773712","author":"Iglesias-Lopez","year":"2021","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtm.2021.11.003_bib5","doi-asserted-by":"crossref","first-page":"31036","DOI":"10.3402\/jmahp.v4.31036","article-title":"Advanced therapy medicinal products: current and future perspectives","volume":"4","author":"Hanna","year":"2016","journal-title":"J.\u00a0Mark. Access Health Policy"},{"key":"10.1016\/j.omtm.2021.11.003_bib8","series-title":"Adaptive Pathways","year":"2021"},{"key":"10.1016\/j.omtm.2021.11.003_bib9","doi-asserted-by":"crossref","first-page":"1505","DOI":"10.18609\/cgti.2019.156","article-title":"Post-marketing safety and efficacy surveillance of cell and gene therapies in the EU: a critical review","volume":"5","author":"Fritsche","year":"2019","journal-title":"Cell Gene Ther. Insights"},{"key":"10.1016\/j.omtm.2021.11.003_bib12","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1056\/NEJMoa1804980","article-title":"Tisagenlecleucel in adult relapsed or refractory diffuse large B-cell lymphoma","volume":"380","author":"Schuster","year":"2018","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib13","doi-asserted-by":"crossref","first-page":"625","DOI":"10.1056\/NEJMoa1710504","article-title":"Nusinersen versus sham control in later-onset spinal muscular atrophy","volume":"378","author":"Mercuri","year":"2018","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib16","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/j.omtm.2020.05.035","article-title":"Mitigating deficiencies in evidence during regulatory assessments of advanced therapies: a comparative study with other biologicals","volume":"18","author":"Elsallab","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.11.003_bib17","doi-asserted-by":"crossref","first-page":"759","DOI":"10.18609\/cgti.2019.088","article-title":"EU market authorisation strategy: lessons from the first 22 ATMP submitted to the EMA","volume":"5","author":"Ball","year":"2019","journal-title":"Cell Gene Ther. Insights"},{"key":"10.1016\/j.omtm.2021.11.003_bib18","doi-asserted-by":"crossref","first-page":"1328","DOI":"10.1016\/j.drudis.2018.03.008","article-title":"EU decision-making for marketing authorization of advanced therapy medicinal products: a case study","volume":"23","author":"de Wilde","year":"2018","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.omtm.2021.11.003_bib19","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1038\/nrd.2018.200","article-title":"European regulatory experience with advanced therapy medicinal products","volume":"18","author":"Barkholt","year":"2018","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtm.2021.11.003_bib20","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1016\/j.drudis.2018.12.007","article-title":"Hurdles in gene therapy regulatory approval: a retrospective analysis of European Marketing Authorization Applications","volume":"24","author":"Carvalho","year":"2019","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.omtm.2021.11.003_bib21","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1186\/s13023-018-0931-2","article-title":"Recommendations for the design of small population clinical trials","volume":"13","author":"Day","year":"2018","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2021.11.003_bib24","series-title":"Recent Advances in Methodology for Clinical Trials in Small Populations: The InSPiRe Project","author":"Friede","year":"2018"},{"key":"10.1016\/j.omtm.2021.11.003_bib25","doi-asserted-by":"crossref","unstructured":"Park, J.J.H., Siden, E., Zoratti, M.J., Dron, L., Harari, O., Singer, J., Lester, R.T., Thorlund, K., and Mills, E.J. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols.Trials 20:572.","DOI":"10.1186\/s13063-019-3664-1"},{"key":"10.1016\/j.omtm.2021.11.003_bib26","doi-asserted-by":"crossref","first-page":"100568","DOI":"10.1016\/j.conctc.2020.100568","article-title":"Master protocols: new directions in drug discovery","volume":"18","author":"Bogin","year":"2020","journal-title":"Contemp. Clin. Trials Commun."},{"key":"10.1016\/j.omtm.2021.11.003_bib27","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1056\/NEJMra1510062","article-title":"Master protocols to study multiple therapies, multiple diseases, or both","volume":"377","author":"Woodcock","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib28","series-title":"Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry","year":"2018"},{"key":"10.1016\/j.omtm.2021.11.003_bib29","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jclinepi.2020.04.025","article-title":"An overview of platform trials with a checklist for clinical readers","volume":"125","author":"Park","year":"2020","journal-title":"J.\u00a0Clin. Epidemiol."},{"key":"10.1016\/j.omtm.2021.11.003_bib32","doi-asserted-by":"crossref","first-page":"3040","DOI":"10.1007\/s12325-020-01397-9","article-title":"A review of two regulatory approved anti-CD19 CAR T-cell therapies in diffuse large B-cell lymphoma: why are indirect treatment comparisons not feasible?","volume":"37","author":"Zhang","year":"2020","journal-title":"Adv. Ther."},{"key":"10.1016\/j.omtm.2021.11.003_bib35","series-title":"Committee for Medicinal Products for human Use (CHMP) Assessment Report","year":"2020"},{"key":"10.1016\/j.omtm.2021.11.003_bib38","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1016\/j.jalz.2016.07.005","article-title":"The DIAN-TU next generation Alzheimer\u2019s prevention trial: adaptive design and disease progression model","volume":"13","author":"Bateman","year":"2017","journal-title":"Alzheimers Dement."},{"key":"10.1016\/j.omtm.2021.11.003_bib40","doi-asserted-by":"crossref","first-page":"3577","DOI":"10.3390\/jcm9113577","article-title":"CAR T-cells in multiple myeloma are ready for prime time","volume":"9","author":"Rodr\u00edguez-Otero","year":"2020","journal-title":"J.\u00a0Clin. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib46","doi-asserted-by":"crossref","first-page":"235","DOI":"10.4155\/cli.12.5","article-title":"Perspective on adaptive designs: 4 years European Medicines Agency reflection paper, 1 year draft US FDA guidance \u2013 where are we now?","volume":"2","author":"Brenda Gaydos","year":"2012","journal-title":"Clin. Invest."},{"key":"10.1016\/j.omtm.2021.11.003_bib49","doi-asserted-by":"crossref","first-page":"3615","DOI":"10.1182\/blood-2011-12-396879","article-title":"Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency","volume":"120","author":"Hassan","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.11.003_bib50","series-title":"Committee for Medicinal Products for Human Use (CHMP) Assessment Report","year":"2021"},{"key":"10.1016\/j.omtm.2021.11.003_bib51","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1056\/NEJMoa1709866","article-title":"Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia","volume":"378","author":"Maude","year":"2018","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib52","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"10.1016\/j.omtm.2021.11.003_bib53","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1002\/acn3.283","article-title":"Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study","volume":"3","author":"Kolb","year":"2016","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"10.1016\/j.omtm.2021.11.003_bib55","doi-asserted-by":"crossref","first-page":"1800","DOI":"10.1182\/blood-2017-03-769620","article-title":"Outcomes in refractory diffuse large B-cell lymphoma: results from the international SCHOLAR-1 study","volume":"130","author":"Crump","year":"2017","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.11.003_bib56","doi-asserted-by":"crossref","first-page":"3574","DOI":"10.1182\/blood-2008-02-140095","article-title":"A survey of fully haploidentical hematopoietic stem cell transplantation in adults with high-risk acute leukemia: a risk factor analysis of outcomes for patients in remission at transplantation","volume":"112","author":"Ciceri","year":"2008","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.11.003_bib58","author":"Walton"},{"key":"10.1016\/j.omtm.2021.11.003_bib60","series-title":"Framework for FDA\u2019s Real-World Evidence Program","year":"2018"},{"key":"10.1016\/j.omtm.2021.11.003_bib61","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1186\/s13023-020-1332-x","article-title":"A roadmap to using historical controls in clinical trials - by drug information association adaptive design scientific working group (DIA-ADSWG)","volume":"15","author":"Ghadessi","year":"2020","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2021.11.003_bib63","first-page":"22821866","article-title":"Protocol NDS-MM-003","author":"Celgene","year":"2018","journal-title":"Celgene Corporation"},{"key":"10.1016\/j.omtm.2021.11.003_bib64","doi-asserted-by":"crossref","first-page":"1172","DOI":"10.1016\/j.ymthe.2018.04.007","article-title":"Registry contributions to strengthen cell and gene therapeutic evidence","volume":"26","author":"Abou-El-Enein","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2021.11.003_bib66","doi-asserted-by":"crossref","first-page":"e104","DOI":"10.1016\/S1470-2045(19)30729-6","article-title":"CAR T-cell product performance in haematological malignancies before and after marketing authorisation","volume":"21","author":"Elsallab","year":"2020","journal-title":"Lancet Oncol."},{"key":"10.1016\/j.omtm.2021.11.003_bib69","series-title":"Background - Progression-free Survival: What Does it Mean for Psychological Well-Being or Quality of Life?","author":"Gutman","year":"2013"},{"key":"10.1016\/j.omtm.2021.11.003_bib70","doi-asserted-by":"crossref","first-page":"e1002873","DOI":"10.1371\/journal.pmed.1002873","article-title":"The use of validated and nonvalidated surrogate endpoints in two European Medicines Agency expedited approval pathways: a cross-sectional study of products authorised 2011\u20132018","volume":"16","author":"Bruce","year":"2019","journal-title":"PLoS Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib71","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1634\/theoncologist.13-S2-19","article-title":"Endpoints for assessing drug activity in clinical trials","volume":"13","author":"Pazdur","year":"2008","journal-title":"Oncologist"},{"key":"10.1016\/j.omtm.2021.11.003_bib73","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/j.omtm.2018.10.003","article-title":"Challenges in advanced therapy medicinal product development: a survey among companies in Europe","volume":"11","author":"ten Ham","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.11.003_bib76","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1136\/archdischild-2020-319063","article-title":"How to use the Bayley scales of infant and toddler development","volume":"106","author":"Del Rosario","year":"2020","journal-title":"Arch. Dis. Child. Educ. Pract. Ed."},{"key":"10.1016\/j.omtm.2021.11.003_bib78","doi-asserted-by":"crossref","first-page":"1055","DOI":"10.1007\/s40273-020-00935-1","article-title":"Surrogate endpoints in health technology assessment: an international review of methodological guidelines","volume":"38","author":"Grigore","year":"2020","journal-title":"Pharmacoeconomics"},{"key":"10.1016\/j.omtm.2021.11.003_bib79","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1016\/j.jval.2016.10.011","article-title":"Time to review the role of surrogate end points in health policy: state of the art and the way forward","volume":"20","author":"Ciani","year":"2017","journal-title":"Value Health"},{"key":"10.1016\/j.omtm.2021.11.003_bib83","author":"M\u00e9ndez-Hermida"},{"key":"10.1016\/j.omtm.2021.11.003_bib84","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/j.ejca.2020.07.006","article-title":"Clinical development of cell therapies for cancer: the regulators\u2019 perspective","volume":"138","author":"Exley","year":"2020","journal-title":"Eur. J. Cancer"},{"key":"10.1016\/j.omtm.2021.11.003_bib85","first-page":"23861","article-title":"Immunotherapy with CAR-modified T\u00a0cells: toxicities and overcoming strategies","volume":"2018","author":"Sun","year":"2018","journal-title":"J.\u00a0Immunol. Res."},{"key":"10.1016\/j.omtm.2021.11.003_bib87","doi-asserted-by":"crossref","first-page":"57","DOI":"10.2131\/jts.46.57","article-title":"Development challenges associated with rAAV-based gene therapies","volume":"46","author":"Bolt","year":"2021","journal-title":"J.\u00a0Toxicol. Sci."},{"key":"10.1016\/j.omtm.2021.11.003_bib88","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1158\/2159-8290.CD-17-1368","article-title":"Chimeric antigen receptor T\u00a0cell-mediated neurotoxicity in nonhuman primates","volume":"8","author":"Taraseviciute","year":"2018","journal-title":"Cancer Discov."},{"key":"10.1016\/j.omtm.2021.11.003_bib91","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1016\/j.yrtph.2013.07.014","article-title":"The risk-based approach to ATMP development - generally accepted by regulators but infrequently used by companies","volume":"67","author":"Kooijman","year":"2013","journal-title":"Regul. Toxicol. Pharmacol."},{"key":"10.1016\/j.omtm.2021.11.003_bib93","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1016\/j.eclinm.2019.01.015","article-title":"Cell and gene therapy trials: are we facing an \u2018evidence crisis\u2019?","volume":"7","author":"Abou-El-Enein","year":"2019","journal-title":"EClinicalMedicine"},{"issue":"7","key":"10.1016\/j.omtm.2021.11.003_bib94","doi-asserted-by":"crossref","first-page":"655","DOI":"10.2217\/rme-2021-0026","article-title":"Comparability: what we can learn from the review of advanced therapy medicinal products","volume":"16","author":"Cockroft","year":"2021","journal-title":"Regen. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib95","doi-asserted-by":"crossref","first-page":"2459","DOI":"10.1111\/bcp.14672","article-title":"Towards a better use of scientific advice for developers of advanced therapies","volume":"87","author":"Tavridou","year":"2021","journal-title":"Br. J. Clin. Pharmacol."},{"key":"10.1016\/j.omtm.2021.11.003_bib96","doi-asserted-by":"crossref","first-page":"886","DOI":"10.1016\/j.jcyt.2021.02.004","article-title":"Standardization, workforce development and advocacy in cell and gene therapies: a summary of the 2020 Regenerative Medicine Interchange","volume":"23","author":"McNiece","year":"2021","journal-title":"Cytotherapy"},{"key":"10.1016\/j.omtm.2021.11.003_bib97","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1038\/s41571-021-00476-2","article-title":"Immunogenicity of CAR T\u00a0cells in cancer therapy","volume":"18","author":"Wagner","year":"2021","journal-title":"Nat. Rev. Clin. Oncol."},{"key":"10.1016\/j.omtm.2021.11.003_bib98","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1182\/blood-2013-01-306647","article-title":"Immune responses to AAV vectors: overcoming barriers to successful gene therapy","volume":"122","author":"Mingozzi","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.11.003_bib100","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1016\/j.omtm.2021.04.001","article-title":"Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs","volume":"21","author":"Drago","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2021.11.003_bib101","first-page":"49","article-title":"Translation of regenerative medicine products into the clinic in the United States: FDA perspective","author":"Lee","year":"2015","journal-title":"Transl. Regen. Med."},{"key":"10.1016\/j.omtm.2021.11.003_bib102","doi-asserted-by":"crossref","first-page":"55","DOI":"10.3389\/fbioe.2017.00055","article-title":"Addressing pressing needs in the development of advanced therapies","volume":"5","author":"Morrow","year":"2017","journal-title":"Front. Bioeng. Biotechnol."},{"issue":"11","key":"10.1016\/j.omtm.2021.11.003_bib104","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1007\/s12325-018-0805-y","article-title":"Interpretation and impact of real-world clinical data for the practicing clinician","volume":"35","author":"Blonde","year":"2018","journal-title":"Adv. Ther."},{"key":"10.1016\/j.omtm.2021.11.003_bib105","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.tibtech.2016.07.007","article-title":"Strategies for derisking translational processes for biomedical technologies","volume":"35","author":"Abou-El-Enein","year":"2017","journal-title":"Trends Biotechnol."},{"key":"10.1016\/j.omtm.2021.11.003_bib106","doi-asserted-by":"crossref","first-page":"3095","DOI":"10.1200\/JCO.19.02103","article-title":"Axicabtagene ciloleucel in the non-trial setting: outcomes and correlates of response, resistance, and toxicity","volume":"38","author":"Jacobson","year":"2020","journal-title":"J.\u00a0Clin. Oncol."},{"key":"10.1016\/j.omtm.2021.11.003_bib107","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1182\/blood-2018-99-117199","article-title":"Axicabtagene ciloleucel in the real world: outcomes and predictors of response, resistance and toxicity","volume":"132","author":"Jacobson","year":"2018","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2021.11.003_bib108","doi-asserted-by":"crossref","first-page":"305","DOI":"10.3389\/fphar.2016.00305","article-title":"Payers\u2019 views of the changes arising through the possible adoption of adaptive pathways","volume":"7","author":"Ermisch","year":"2016","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtm.2021.11.003_bib109","doi-asserted-by":"crossref","first-page":"i2978","DOI":"10.1136\/bmj.i2978","article-title":"Letting post-marketing bridge the evidence gap: the case of orphan drugs","volume":"353","author":"Joppi","year":"2016","journal-title":"BMJ"},{"key":"10.1016\/j.omtm.2021.11.003_bib112","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1007\/s40259-015-0140-7","article-title":"Sipuleucel-T (Provenge\u00ae) - autopsy of an innovative paradigm change in cancer treatment: why a single-product biotech company failed to capitalize on its breakthrough invention","volume":"29","author":"Jaros\u0142awski","year":"2015","journal-title":"BioDrugs"},{"key":"10.1016\/j.omtm.2021.11.003_bib113","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1016\/j.stem.2016.08.012","article-title":"Overcoming challenges facing advanced therapies in the EU market","volume":"19","author":"Abou-El-Enein","year":"2016","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.omtm.2021.11.003_bib114","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1016\/j.jval.2019.12.008","article-title":"Managed entry agreements: policy analysis from the European perspective","volume":"23","author":"Dabbous","year":"2020","journal-title":"Value Health"},{"key":"10.1016\/j.omtm.2021.11.003_bib115","doi-asserted-by":"crossref","first-page":"1034","DOI":"10.1111\/bcp.14279","article-title":"Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines","volume":"86","author":"Moseley","year":"2020","journal-title":"Br. J. Clin. Pharmacol."},{"key":"10.1016\/j.omtm.2021.11.003_bib117","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1089\/humc.2013.087","article-title":"Lessons learned from the clinical development and market authorization of Glybera","volume":"24","author":"Bryant","year":"2013","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"10.1016\/j.omtm.2021.11.003_bib118","doi-asserted-by":"crossref","first-page":"517","DOI":"10.1016\/j.drudis.2016.01.003","article-title":"Translational medicine guide transforms drug development processes: the recent Merck experience","volume":"21","author":"Dolgos","year":"2016","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.omtm.2021.11.003_bib119","doi-asserted-by":"crossref","first-page":"130","DOI":"10.3389\/fbioe.2018.00130","article-title":"Recent progress in European advanced therapy medicinal products and beyond","volume":"6","author":"Yu","year":"2018","journal-title":"Front. Bioeng. Biotechnol."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001753?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050121001753?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:26:20Z","timestamp":1762421180000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050121001753"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,12]]},"references-count":74,"alternative-id":["S2329050121001753"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2021.11.003","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2021,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Current landscape of clinical development and approval of advanced therapies","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2021.11.003","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Authors.","name":"copyright","label":"Copyright"}]}}