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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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Drug Regulation01:25

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Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
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Drug Products: Biologics, Biosimilars and Interchangeables01:28

Drug Products: Biologics, Biosimilars and Interchangeables

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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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Drug Discovery: Overview01:26

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Preclinical Development: Overview01:28

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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
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Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption01:23

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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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Related Experiment Video

Updated: Feb 27, 2026

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
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Orphan Drug Debate: A Cheat Sheet.

Krishna R Patel

    Managed Care (Langhorne, Pa.)
    |June 30, 2017
    PubMed
    Summary
    This summary is machine-generated.

    The Orphan Drug Act of 1983 has achieved successes but faces controversy due to high drug prices and alleged exploitation by pharmaceutical companies.

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    Area of Science:

    • Health Policy
    • Pharmaceutical Economics
    • Regulatory Law

    Background:

    • The Orphan Drug Act (ODA) of 1983 was enacted to incentivize the development of drugs for rare diseases.
    • Despite its successes, the ODA has become a subject of debate regarding drug pricing and industry practices.

    Purpose of the Study:

    • To examine the successes and controversies surrounding the 1983 Orphan Drug Act.
    • To outline the key arguments in the ongoing debate about the ODA's impact.

    Main Methods:

    • This analysis synthesizes existing literature and policy discussions.
    • It reviews key provisions of the Orphan Drug Act and its legislative history.

    Main Results:

    • The ODA has facilitated the approval of numerous drugs for rare conditions, representing a significant public health achievement.
    • However, concerns persist regarding the high cost of orphan drugs and allegations of companies leveraging the act for non-orphan drugs or excessive profits.

    Conclusions:

    • The Orphan Drug Act presents a complex case of regulatory success intertwined with economic and ethical challenges.
    • Further policy evaluation is needed to balance incentives for rare disease drug development with affordable access and prevent potential misuse.