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Related Concept Videos

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...
Preclinical Development: Overview01:28

Preclinical Development: Overview

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...
Clinical Trials: Overview01:11

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
Drug Discovery: Overview01:26

Drug Discovery: Overview

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...
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
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).

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An Organotypic High Throughput System for Characterization of Drug Sensitivity of Primary Multiple Myeloma Cells
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Published on: July 15, 2015

Accelerating orphan drug development.

Timothy R Coté1, Kui Xu, Anne R Pariser

  • 1Office of the Commissioner, Office of Orphan Products Development, Silver Spring, Maryland 20993, USA. timothy.cote@fda.hhs.gov

Nature Reviews. Drug Discovery
|December 2, 2010
PubMed
Summary
This summary is machine-generated.

Drug development for rare diseases is growing. Regulators, like the US Food and Drug Administration (FDA), are key to accelerating progress in creating new therapies for these conditions.

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Area of Science:

  • Pharmacology
  • Regulatory Science
  • Rare Diseases

Background:

  • Recent surge in interest in developing drugs for rare diseases.
  • The US Food and Drug Administration (FDA) plays a crucial role in this area.
  • Focus on regulatory strategies to advance rare disease drug development.

Discussion:

  • The importance of regulatory pathways in incentivizing rare disease drug research.
  • How regulatory agencies can facilitate clinical trials and drug approvals for rare conditions.
  • The impact of regulatory support on the pharmaceutical industry's engagement with rare diseases.

Key Insights:

  • Increased focus on rare disease drug development.
  • Regulatory bodies are essential catalysts for progress.
  • FDA's role in accelerating therapeutic advancements.

Outlook:

  • Continued growth in rare disease drug development is anticipated.
  • Further evolution of regulatory frameworks to support innovation.
  • Potential for new treatments to reach patients with unmet needs.