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Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

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Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
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Pharmaceutical Equivalents01:26

Pharmaceutical Equivalents

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As defined by regulatory standards, pharmaceutical equivalents require generic drug products to have identical dosage forms and chemically identical active pharmaceutical ingredients (APIs). They must adhere to compendial or applicable standards for potency, content uniformity, disintegration times, and dissolution rates. In the case of modified-release dosage forms, variations in drug content are permissible as long as the delivered amount remains consistent with the innovator drug product.
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Drug Products: Biologics, Biosimilars and Interchangeables01:28

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Body:Biologics, derived from living sources such as humans, animals, or microorganisms, represent a significant category of pharmaceuticals. These complex molecules, developed through advanced biotechnological methods or purified from natural sources, include essential medical treatments like insulin and growth hormones. The complexity of biologics arises from their large molecular structures and the intricate processes required for their production, making them distinct from conventional...
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Pharmaceutical Alternatives: Excipients and Impurities-Related Therapeutic Nonequivalence01:19

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Pharmaceutical products contain more than just the active drug; they also contain various excipients such as binders, solubilizers, stabilizers, preservatives, and other elements. In some cases, impurities or contaminants might be present. Traditionally, quality control in pharmaceuticals has primarily focused on the analysis of the active drug, often overlooking the impact of these additional components. The recent issue with heparin contamination by over-sulfated chondroitin sulfate, a...
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Drug Nomenclature01:17

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During the development of a new pharmaceutical, the manufacturer initially assigns a code name to the drug. Once approved, the drug receives a United States Adopted Name (USAN)—a generic, nonproprietary designation. Upon being listed in the United States Pharmacopeia, this nonproprietary name becomes the drug's official name. Additionally, the manufacturer assigns a proprietary name or trademark, which serves as the brand name under which the drug is marketed. It is worth noting that...
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Dosage Regimen: Individualization01:24

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Individualization in dosing regimens is the customization of medication doses for individual patients. Its necessity arises from the goal of maximizing therapeutic benefits while minimizing risks. This approach is pivotal because human responses to drugs can vary widely; what is effective for one person may be inadequate or excessive for another. Interpatient (intersubject) variability refers to differences in drug responses between individuals, while intrapatient (intrasubject) variability...
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Updated: Oct 19, 2025

Sample Extraction and Simultaneous Chromatographic Quantitation of Doxorubicin and Mitomycin C Following Drug Combination Delivery in Nanoparticles to Tumor-bearing Mice
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Orphan Drugs, Compounded Medication and Pharmaceutical Commons.

Kim Hendrickx1, Marc Dooms2

  • 1Research Foundation-Flanders (FWO)/Life Sciences and Society Lab, Leuven, Belgium.

Frontiers in Pharmacology
|September 27, 2021
PubMed
Summary

Regulatory incentives for rare diseases are questioned for common food ingredients. These ingredients, used in medicinal compounding, should be considered pharmaceutical commons, not requiring intellectual property protection.

Keywords:
commonscompounded medicationdrug research and developmentintellectual propertyopen scienceorphan drugspoint of care manufacturingrare diseases

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Area of Science:

  • Pharmacology
  • Drug Regulation
  • Medicinal Chemistry

Background:

  • Orphan drug regulations aim to incentivize rare disease treatment development.
  • Intellectual property (IP) rights protect these incentivized drugs.
  • Medicinal compounding is a recognized practice for preparing medications.

Purpose of the Study:

  • To evaluate the justification of orphan drug regulations and IP rights for common food ingredients.
  • To analyze the role of medicinal compounding in rare disease treatment.
  • To propose classifying these ingredients as 'pharmaceutical commons'.

Main Methods:

  • Literature review of food ingredients with clinical evidence for rare diseases.
  • Analysis of marketing authorization and IP status of these ingredients.
  • Examination of the practice of medicinal compounding.

Main Results:

  • Identified well-known food ingredients with clinical evidence for rare diseases.
  • These ingredients have obtained marketing authorization as expensive orphan drugs with IP protection.
  • These ingredients are integral to medicinal compounding, a form of point-of-care manufacturing.

Conclusions:

  • Orphan drug incentives and IP rights may not be justified for common food ingredients used in compounding.
  • These ingredients function as 'pharmaceutical commons' due to their accessibility and established use.
  • Re-evaluation of regulatory frameworks for such substances is suggested.