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Related Concept Videos

Drug Regulation01:25

Drug Regulation

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...
Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

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...
Drug Control Governance: Regulatory Bodies and Their Impact01:03

Drug Control Governance: Regulatory Bodies and Their Impact

Drug control governance involves the oversight and regulation of pharmaceuticals to ensure their safety and efficacy while preventing illegal drug use and trafficking. Regulatory bodies, including the US Food and Drug Administration (FDA) and the European Union's European Medicines Agency (EMA), play a central role in this process. These agencies evaluate the safety and efficacy of drugs before they can be marketed. They fund clinical trials and assess the benefits and risks associated with a...
Drug Products: Biologics, Biosimilars and Interchangeables01:28

Drug Products: Biologics, Biosimilars and Interchangeables

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...
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions01:15

Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure (CHF).
FDA Approved Drugs: Changes to Approved Drugs01:26

FDA Approved Drugs: Changes to Approved Drugs

Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...

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Radiosynthesis, Quality Control, and Small Animal Positron Emission Tomography Imaging of 68Ga-Labelled Nano Molecules
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Radiosynthesis, Quality Control, and Small Animal Positron Emission Tomography Imaging of 68Ga-Labelled Nano Molecules

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Orphan drugs: the regulatory environment.

Pedro Franco1

  • 1European Medicines Agency, Canary Wharf, London, UK. pedro.franco@ema.europa.eu

Drug Discovery Today
|September 18, 2012
PubMed
Summary

Orphan drug legislation varies globally, impacting rare disease treatments. Understanding these diverse regulatory frameworks is key to improving international collaboration for rare diseases.

Area of Science:

  • Health Law
  • Regulatory Science
  • Pharmaceutical Policy

Background:

  • Rare disease definitions and orphan drug policies differ significantly across regions.
  • Global regulatory landscapes for orphan drugs are complex and evolving.
  • International collaboration is hindered by a lack of harmonized legislation.

Purpose of the Study:

  • To provide a comprehensive overview of the global legal and regulatory environment for orphan drugs.
  • To compare and contrast orphan drug legislation in key regions including the US, EU, Japan, Australia, Singapore, Taiwan, and Canada.
  • To discuss the necessity and evolution of specific orphan drug policies worldwide.

Main Methods:

  • Comparative legal analysis of orphan drug legislation.
  • Review of historical development of orphan drug policies in selected countries/regions.

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Nutrient Regulation by Continuous Feeding for Large-scale Expansion of Mammalian Cells in Spheroids

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  • Examination of incentives, designation criteria, and approval processes for orphan drugs.

    • Main Results:

    • Significant variations exist in orphan drug legislation worldwide, despite some similarities.
    • Key differences identified in incentives, orphan status criteria, and drug authorization pathways.
    • The historical evolution of these policies shows a trend towards recognizing the need for specific orphan drug frameworks.

    Conclusions:

    • Harmonizing global orphan drug legislation is crucial for advancing rare disease research and treatment.
    • Understanding the nuances of different regulatory systems can foster better international cooperation.
    • Effective policies are essential to incentivize the development of treatments for rare diseases.