Orphan Drugs, Compounded Medication and Pharmaceutical Commons

Hendrickx, Kim; Dooms, Marc

doi:10.3389/fphar.2021.738458

Cited by 13 publications

(6 citation statements)

References 13 publications

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“… Hendrickx and collaborators (2021) propose the use of pharmaceutical compounding not as an alternative, but as a parallel way of developing and providing access to drugs and substances that are known to have activity for certain rare diseases. The authors view pharmaceutical compounding as a practice of self-governing (within the health system) a pharmaceutical commons, as the knowledge and goods to produce these drugs are widely accessible compared to the more privatized industrial system that results in the “enclosure of common therapies” ( Hendrickx et al ., 2021 ).…”

Section: Resultsmentioning

confidence: 99%

Pharmaceutical policy and innovation for rare diseases: A narrative review

Alonso Ruiz,

Large,

Moon

et al. 2023

F1000Res

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This article aims to synthesize the existing literature on the implementation of public policies to incentivize the development of treatments for rare diseases, (diseases with very low prevalence and therefore with low commercial interest) otherwise known as orphan drugs. The implementation of these incentives in the United States (US), Japan, and in the European Union (EU) seems to be related to a substantial increase in treatments for these diseases, and has influenced the way the pharmaceutical research & development (R&D) system operates beyond this policy area. Despite the success of the Orphan Drug model, the academic literature also highlights the negative implications that these public policies have on affordability and access to orphan drugs, as well as on the prioritization of certain disease rare areas over others. The synthesis focuses mostly on the United States’ Orphan Drug Act (ODA) as a model for subsequent policies in other regions and countries. It starts with a historical overview of the creation of the term “rare diseases”, continues with a summary of the evidence available on the US ODA’s positive and negative impacts, and provides a summary of the different proposals to reform these incentives in light of the negative outcomes described. Finally, it describes some key aspects of the Japanese and European policies, as well as some of the challenges captured in the literature related to their impact in Low- and Middle-Income Countries (LMICs).

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Section: Resultsmentioning

confidence: 99%

Pharmaceutical policy and innovation for rare diseases: A narrative review

Alonso Ruiz,

Large,

Moon

et al. 2023

F1000Res

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“…This route bypasses the intensive authorization process while still yielding pharmaceutical preparations, although they are regulated less stringently than authorized medicinal products. Perhaps specific (national) policies could even protect common and often compounded food‐related substances with a well‐established use in rare diseases like IEMs as so‐called “pharmaceutical commons”, safeguarding them from pharmaceutical enclosing 59 …”

Section: Discussionmentioning

confidence: 99%

“…The absence of medicinal nutritional therapy products can result in a process that has been called the "enclosing" of these substances by private pharmaceutical companies. 59 Common nutritional therapy products can be repurposed as orphan medicinal products (OMPs). OMPs are medication for rare diseases, defined as a prevalence less than 5:10 000, a cut-off most IEMs meet.…”

Section: Pharmaceutical Enclosingmentioning

confidence: 99%

“…Perhaps specific (national) policies could even protect common and often compounded food-related substances with a well-established use in rare diseases like IEMs as socalled "pharmaceutical commons", safeguarding them from pharmaceutical enclosing. 59 It is also important to acknowledge that while some nutritional therapy products fail to meet the threshold for functioning as medication, the current regulatory framework might still be insufficient to safeguard necessary product aspects. Amino-acid mixtures, for instance, are, in our opinion, usually rightly classified as food (for special medical purposes); as an integral part of management, however, their quality might still warrant more stringent monitoring and fairer reimbursement policies than those provided for FSMPs.…”

Section: Distinguishing Food From Medicinal Products: An Evaluation F...mentioning

confidence: 99%

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

Stolwijk,

Bosch,

Bouwhuis

et al. 2023

J of Inher Metab Disea

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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.

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“…Topical treatment with a personalized medication is the key factor to satisfy both doctor and patient. As long as the applied product is more specific to the skin type, the greater the chances of effectiveness [1][2][3]. Patient credibility in custom products is much higher than in industrial products [4,5].…”

Section: Introductionmentioning

confidence: 99%

Development and Evaluation of Hydrophilic Cream Bases Suitable for Custom Cosmetic Skincare or Compounding Pharmaceutical Dermal Products

BÎRSAN¹

2022

FARMACIA

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This study aimed to formulate, prepare and characterize cream bases used to make individualized preparations for patients: compounding pharmaceutical or cosmeceutical preparations. In terms of consistency, FIII, which is formulated with 3% stearic acid, 3.5% polyglyceryl-3 methylglucose distearate, and 65.5% Centaurea cyanus flower water, can be recommended as a first choice when an easy spreading capacity on the skin is required. Comparative rheological analysis showed a plastic behaviour for all the proposed formulas, while when evaluating the restructuring behaviour during shearing with successive decreasing speeds, a hysteresis loop was observed. The skin tolerance and the degree of hydration obtained after applying the cream were evaluated. Under the experimental conditions adopted in the screening test, the single application of the proposed products showed good compatibility with the skin. The tests on the 6 human subjects for FI, which is formulated with 3% stearic acid, 3% polyglyceryl-3 methylglucose distearate and 66% Centaurea cyanus flower water, confirmed an increased level of hydration level, on average by 12.8% compared to the zero moment (when the cream was not applied) and for FIII an increase over 6.5%. From the obtained results, two cream bases for compounding pharmaceutical or cosmeceutical preparations formulas with a lower percentage of incorporated stearic acid can be proposed, respectively FI and FIII. RezumatScopul acestui studiu a constat în formularea, prepararea și caracterizarea unor baze de cremă utilizate în vederea realizării preparatelor individualizate pentru pacienți: preparate farmaceutice magistrale sau cosmeceutice. Din punctul de vedere al consistenței, baza de cremă FIII, formulată cu acid stearic 3%, distearat de poligliceril-3 metil glucoză 3,5% și apă de albăstrele 65,5%, poate fi recomandată ca primă alegere atunci când se dorește o capacitate de etalare facilă pe piele. Analiza reologică comparativă a demonstrat pentru toate formulele propuse un comportament plastic, iar la evaluarea comportamentului privind restructurarea din timpul forfecării cu viteze succesive descrescătoare s-a evidențiat bucla de histereză. Au fost evaluate toleranța cutanată și gradul de hidratare obținut după aplicarea cremei. În condițiile experimentale adoptate în testul screening, aplicarea unică a produselor propuse a arătat o bună compatibilitate cu pielea. Evaluarea gradului de hidratare al stratului cornos a indicat că toate formulele propuse au efect hidratant la patru ore după aplicare, observânduse o compatibilitate optimă. În cazul formulării FI, ce conține acid stearic 3%, distearat de poligliceril-3 metil glucoză 3% și apă de albăstrele 66%, testările pe cei 6 subiecți umani au confirmat creșterea nivelului de hidratare în medie cu 12,8% față de momentul zero, iar pentru FIII o creștere de peste 6,5%. FII a crescut gradul de hidratare doar cu 0,7%, iar pentru FIV valorile au crescut cu 5,5%.Din rezultatele obținute se pot propune ca baze de cremă pentru preparate farmaceutice magi...

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Orphan Drugs, Compounded Medication and Pharmaceutical Commons

Cited by 13 publications

References 13 publications

Pharmaceutical policy and innovation for rare diseases: A narrative review

Pharmaceutical policy and innovation for rare diseases: A narrative review

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

Development and Evaluation of Hydrophilic Cream Bases Suitable for Custom Cosmetic Skincare or Compounding Pharmaceutical Dermal Products

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