Food or medicine? A European regulatory perspective on nutritional therapy products to treat inborn errors of metabolism

Abstract

BackgroundDietary 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.MethodsSearches 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.ResultsIn 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.ConclusionsEvaluating 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.This article is protected by copyright. All rights reserved.

3D Printing of Dietary Products for the Management of Inborn Errors of Intermediary Metabolism in Pediatric Populations

“…Insufficient products trigger physiological perturbations, necessitating dietary supplementation for homeostasis control in most diseases ( Figure 1 g) [ 23 ]. Assessing nutritional therapy products may reveal a justified need for a pharmaceutical or dietary product [ 24 ]. As a result of dietary treatment, patients often require supplementation for normal cellular function.…”