TY - JOUR
T1 - Food or medicine? A European regulatory perspective on nutritional therapy products to treat inborn errors of metabolism
AU - Stolwijk, N. N.
AU - Bosch, A. M.
AU - Bouwhuis, N.
AU - Häberle, J.
AU - van Karnebeek, C.
AU - van Spronsen, F. J.
AU - Langeveld, M.
AU - Hollak, C. E. M.
N1 - Funding Information: We wish to extend our sincere appreciation to our metabolic dieticians Jorien Haverkamp and Corrie Timmer for their valuable insights regarding dietary interventions for IEMs in clinical practice and to Dr. Arnold Versteeg, LLM PhD, for his invaluable assistance in interpreting the legal and regulatory aspects. Publisher Copyright: © 2023 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.
PY - 2023/11
Y1 - 2023/11
N2 - 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.
AB - 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.
KW - European Union
KW - food supplements
KW - foods for special medical purposes
KW - inborn errors of metabolism
KW - medical food
KW - nutritional therapy
UR - http://www.scopus.com/inward/record.url?scp=85170712073&partnerID=8YFLogxK
U2 - https://doi.org/10.1002/jimd.12677
DO - https://doi.org/10.1002/jimd.12677
M3 - Article
C2 - 37650776
SN - 0141-8955
VL - 46
SP - 1017
EP - 1028
JO - Journal of Inherited Metabolic Disease
JF - Journal of Inherited Metabolic Disease
IS - 6
ER -