{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T09:03:10Z","timestamp":1790758990165,"version":"4.1.0"},"reference-count":49,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T00:00:00Z","timestamp":1790726400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T00:00:00Z","timestamp":1790726400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2026,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Nutritional therapy is critical in managing inherited metabolic diseases (IMDs), and includes specialized diets and single nutritional therapy products (sNTPs) such as vitamins, cofactors, and amino acids. Many sNTPs function as medicines, but are regulated as food (e.g., food supplements), which can limit access, reimbursement, and consistent supply. Essential medicines, as defined by World Health Organization (WHO), meet key healthcare needs and must be available, affordable, and quality\u2010assured. Some sNTPs might meet this threshold, supporting their potential inclusion in the WHO essential medicines list. A modified Delphi process was conducted with a panel of 36 experts from 18 European countries to identify which sNTPs are essential for IMD treatment. Ninety\u2010six sNTPs and their target IMDs were assessed using a literature\u2010informed five\u2010criteria framework, that included evaluation of the level of scientific evidence for usefulness and effectiveness. Overall, 31 sNTPs were deemed essential in the management of 55 IMDs, consisting of vitamins (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200912), minerals (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20094), amino acids (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20096), sugars (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20092), and other nutrients (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u20097). Evidence supporting their use ranged from levels 3 to 4 of the Oxford Centre for Evidence\u2010Based Medicine (OCEBM), reflecting the limited clinical trial data inherent to rare diseases. Seven of these are already included in the WHO essential medicines list. Recognition of the remaining 24 essential sNTPs could improve their availability and reimbursement, and in some cases, may warrant formal authorization as medicines. Subsequent research should focus on access inequities, cost barriers, and incorporate patient perspectives to optimize the use and impact of sNTPs in IMD management.\n                  <\/jats:p>","DOI":"10.1002\/jimd.70252","type":"journal-article","created":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T08:32:58Z","timestamp":1790757178000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Essential Oral Single Nutritional Therapy Products for Inherited Metabolic Diseases: Evidence and Consensus Assessment Using a Modified Delphi Method"],"prefix":"10.1002","volume":"49","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3498-5747","authenticated-orcid":false,"given":"Nina N.","family":"Stolwijk","sequence":"first","affiliation":[{"name":"Medicines for Society (Medicijn voor de Maatschappij) Platform at Amsterdam UMC\u2014University of Amsterdam  Amsterdam the Netherlands"},{"name":"Department of Endocrinology and Metabolism. Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, MetabERN University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-1617-2789","authenticated-orcid":false,"given":"Bart M. F.","family":"Penninx","sequence":"additional","affiliation":[{"name":"Medicines for Society (Medicijn voor de Maatschappij) Platform at Amsterdam UMC\u2014University of Amsterdam  Amsterdam the Netherlands"},{"name":"Department of Endocrinology and Metabolism. Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, MetabERN University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5027-5916","authenticated-orcid":false,"given":"Annet M.","family":"Bosch","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Amsterdam UMC Location University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9934-6831","authenticated-orcid":false,"given":"Mirjam","family":"Langeveld","sequence":"additional","affiliation":[{"name":"Department of Endocrinology and Metabolism. Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, MetabERN University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gajja S.","family":"Salomons","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Amsterdam UMC Location University of Amsterdam  Amsterdam the Netherlands"},{"name":"Department of Laboratory Medicine, Laboratory Genetic Metabolic Diseases Amsterdam UMC Location University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0464-1078","authenticated-orcid":false,"given":"Carla E. M.","family":"Hollak","sequence":"additional","affiliation":[{"name":"Medicines for Society (Medicijn voor de Maatschappij) Platform at Amsterdam UMC\u2014University of Amsterdam  Amsterdam the Netherlands"},{"name":"Department of Endocrinology and Metabolism. Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, MetabERN University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"Essential Nutritional Therapy Products for IMDs Consensus Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,9,30]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2012.07.005"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12677"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12100"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2014.10.009"},{"key":"e_1_2_13_6_1","unstructured":"Organization WH Essential Medicines https:\/\/www.who.int\/westernpacific\/health\u2010topics\/essential\u2010medicines#tab=tab_1."},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2013.12.002"},{"issue":"4","key":"e_1_2_13_8_1","doi-asserted-by":"crossref","first-page":"1008","DOI":"10.1046\/j.1365-2648.2000.t01-1-01567.x","article-title":"Research Guidelines for the Delphi Survey Technique","volume":"32","author":"Hasson F.","year":"2000","journal-title":"Journal of Advanced Nursing"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010019\u20101091\u20108"},{"key":"e_1_2_13_10_1","unstructured":"Regulation (EC) No 178\/2002 of the European Parliament and of the Council of 28 January 2002 Laying Down the General Principles and Requirements of Food Law Establishing the European Food Safety Authority and Laying Down Procedures in Matters of Food Safety OJ L 31 1.2.2002 pp. 1\u201324. Consolidated Version 26\/07\/2019."},{"key":"e_1_2_13_11_1","unstructured":"Directive 2001\/83\/EC of the European Parliament and of the Council of 6 November 2001 on the Community Code Relating to Medicinal Products for Human Use OJ L 311 28.11.2001 pp. 67\u2013128 Consolidated Version 26\/07\/2019."},{"key":"e_1_2_13_12_1","doi-asserted-by":"crossref","DOI":"10.1007\/978-3-662-63123-2","volume-title":"Inborn Metabolic Diseases: Diagnosis and Treatment","author":"Saudubray J. M.","year":"2022"},{"key":"e_1_2_13_13_1","volume-title":"The Oxford Levels of Evidence 2","author":"OCEBM Levels of Evidence Working Group","year":"2011"},{"key":"e_1_2_13_14_1","unstructured":"15 November 2007. Case C\u2010319\/05. 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