학술논문
Food or medicine? A European regulatory perspective on nutritional therapy products to treat inborn errors of metabolism.
Document Type
Academic Journal
Author
Stolwijk NN; Medicine for Society, Platform at Amsterdam University Medical Center, University of Amsterdam, Amsterdam, The Netherlands.; 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.; Bosch AM; Department of Pediatrics, Division of Metabolic Diseases, Emma Children's Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.; Amsterdam Reproduction and Development Research Institute, Amsterdam, The Netherlands.; Amsterdam Gastroenterology, Endocrinology and Metabolism, Amsterdam, The Netherlands.; Bouwhuis N; Medicine for Society, Platform at Amsterdam University Medical Center, University of Amsterdam, Amsterdam, The Netherlands.; Department of Pharmacy, Amsterdam UMC-University of Amsterdam, Amsterdam, The Netherlands.; Häberle J; Department of Pediatrics, Division of Metabolism, University Children's Hospital Zürich, Zurich, Switzerland.; van Karnebeek C; Department of Pediatrics and Human Genetics, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands.; van Spronsen FJ; Department of Metabolic Diseases, University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Groningen, The Netherlands.; Langeveld M; 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.; Hollak CEM; Medicine for Society, Platform at Amsterdam University Medical Center, University of Amsterdam, Amsterdam, The Netherlands.; 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.
Source
Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE
Subject
Language
English
Abstract
Dietary or nutritional management strategies are the cornerstone of treatment for many inborn errors of metabolism (IEMs). Though a vital part of standard of care, the products prescribed for this are often not formally registered as medication. Instead, they are regulated as food or as food supplements, impacting the level of oversight as well as reimbursed policies. This scoping literature review explores the European regulatory framework relevant to these products and its implications for current clinical practice. Searches of electronic databases (PubMed, InfoCuria) were carried out, supplemented by articles identified by experts, from reference lists, relevant guidelines and case-law by the European Court of Justice. In the European Union (EU), nutritional therapy products are regulated as food supplements, food for special medical purposes (FSMPs) or medication. The requirements and level of oversight increase for each of these categories. Relying on lesser-regulated food products to treat IEMs raises concerns regarding product quality, safety, reimbursement and patient access. In order to ascertain whether a nutritional therapy product functions as medication and thus could be classified as such, we developed a flowchart to assess treatment characteristics (benefit, pharmacological attributes, and safety) with a case-based approach. Evaluating nutritional therapy products might reveal a justifiable need for a pharmaceutical product. A flowchart can facilitate systematically distinguishing products that function medication-like in the management of IEMs. Subsequently, finding and implementing appropriate solutions for these products might help improve the quality, safety and accessibility including reimbursement of treatment for IEMs.
(© 2023 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.)
(© 2023 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.)