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Related Concept Videos

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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Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption01:23

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

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Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
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Pharmacokinetics in Pediatric Patients: Drug Metabolism01:24

Pharmacokinetics in Pediatric Patients: Drug Metabolism

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In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
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Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

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Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
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Bioequivalence of Drugs: Drugs with Multiple Indications01:09

Bioequivalence of Drugs: Drugs with Multiple Indications

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The concept of therapeutic equivalence (TE) in drugs with multiple indications is complex. A generic drug may be therapeutically equivalent to a brand-name product for one specific indication, but this doesn't necessarily mean it's equivalent for all other indications. Evidence of TE in one patient group and bioequivalence shown in healthy volunteers can support—but not confirm—TE for other indications. However, definitive proof requires individual clinical studies for each...
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Pharmacokinetics in Pediatric Patients: Drug Distribution01:17

Pharmacokinetics in Pediatric Patients: Drug Distribution

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Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight,...
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Orphan Drugs and Diseases: A Systematic Review.

Heena Dixit Tiwari1, Manoj Kumar Sahoo2, M R Murali3

  • 1Department of Hospital Administration, Malwanchal University, Indore, Madhya Pradesh, India.

Journal of Pharmacy & Bioallied Sciences
|April 20, 2026
PubMed
Summary
This summary is machine-generated.

Rare diseases affect millions globally, but orphan drug development faces hurdles. Harmonizing definitions and ethical pricing is crucial for equitable access to treatments.

Keywords:
Healthcare policyorphan drugspharmacoeconomicsrare diseasessystematic review

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Area of Science:

  • Medical Science
  • Health Economics
  • Pharmaceutical Policy

Background:

  • Rare diseases collectively impact over 300 million people worldwide.
  • Orphan drug development is hindered by regulatory, economic, and clinical trial challenges.
  • Existing literature lacks a systematic overview of rare diseases and orphan drugs.

Purpose of the Study:

  • To systematically review the current landscape of orphan drugs and rare diseases.
  • To analyze definitions, therapeutic strategies, economic evaluations, and stakeholder perspectives.
  • To identify key factors influencing equitable access to rare disease treatments.

Main Methods:

  • Systematic review of 70 screened articles, selecting 10 relevant studies.
  • Included study types: systematic reviews, scoping reviews, observational studies, health economic evaluations.
  • Data extraction focused on definitions, healthcare roles, cost analyses, and clinical outcomes.

Main Results:

  • Global definitions of rare diseases are inconsistent, affecting drug approvals.
  • Economic evaluations highlight disparities in orphan drug funding.
  • Pharmacist and stakeholder roles are increasingly vital for therapeutic access.
  • High costs, ethical issues, and diagnostic advances are prominent themes.
  • Pediatric and syndromic rare conditions (e.g., Duchenne muscular dystrophy, Turner syndrome) are frequently discussed.

Conclusions:

  • Harmonized rare disease definitions are essential for streamlined drug approval.
  • Ethical pricing models and interdisciplinary healthcare roles are needed for sustainability.
  • Evidence-based policies are critical to ensure equitable access to rare disease therapies.