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Related Concept Videos

Preclinical Development: Overview01:28

Preclinical Development: Overview

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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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Clinical Trials: Overview01:11

Clinical Trials: Overview

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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...
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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 Discovery: Overview01:26

Drug Discovery: Overview

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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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Clinically Relevant Drug Product Specifications: Methods of Establishment01:29

Clinically Relevant Drug Product Specifications: Methods of Establishment

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Product specifications define the acceptable quality of a pharmaceutical product by ensuring identity, purity, potency, and strength. These specifications serve as benchmarks during development, manufacturing, and post-approval quality control. Clinically relevant specifications are particularly important because they directly relate to a drug's safety and efficacy in clinical use.Dissolution studies are critical biopharmaceutic tools that link in vitro behavior to in vivo performance. They...
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Clinical Trials01:16

Clinical Trials

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Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
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Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
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Orphan drug clinical development.

Olivier Blin1, Marie-Noelle Lefebvre1, Olivier Rascol2

  • 1Service de pharmacologie clinique et pharmacovigilance, Aix Marseille Univ, AP-HM, INSERM, Inst Neurosci Syst, 13005 Marseille, France; Orphandev-FCRIN, INSERM, 13005 Marseille, France.

Therapie
|April 6, 2020
PubMed
Summary

Developing orphan drugs presents unique challenges in clinical trials. Successful development requires strong collaboration among stakeholders to bring effective treatments to patients with unmet medical needs.

Keywords:
Clinical trialMarket authorizationOrphan drugsPatientRare diseases

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Area of Science:

  • Pharmacology and Drug Development
  • Clinical Trial Design
  • Regulatory Affairs

Background:

  • Orphan drug development faces significant hurdles including design, recruitment, and cost.
  • A collaborative approach is essential to navigate these complexities.

Purpose of the Study:

  • To review and discuss the key pillars for successful orphan drug clinical development.
  • To identify strategies for overcoming common challenges in orphan drug trials.

Main Methods:

  • Review of existing literature and case studies on orphan drug development.
  • Analysis of collaborative models between academic centers, pharmaceutical companies, patient representatives, and health authorities.

Main Results:

  • Successful orphan drug development hinges on integrated strategies addressing trial design, patient recruitment, and regulatory pathways.
  • Effective collaboration is crucial for innovative approaches and favorable benefit-risk assessments.

Conclusions:

  • Key pillars for successful orphan drug development include robust collaboration, innovative trial designs, and patient-centric approaches.
  • Addressing unmet medical needs requires a concerted effort to streamline the path to market for orphan drugs.