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Related Concept Videos

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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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.
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Clinical Trials: Overview01:11

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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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Drug Products: Biologics, Biosimilars and Interchangeables01:28

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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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Clinical Trials01:16

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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.
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Drug Administration and Therapy Phases: Overview01:26

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Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
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Related Experiment Video

Updated: Feb 25, 2026

An Organotypic High Throughput System for Characterization of Drug Sensitivity of Primary Multiple Myeloma Cells
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Characterizing expanded access and compassionate use programs for experimental drugs.

Jennifer E Miller1,2, Joseph S Ross3,4,5, Kenneth I Moch6

  • 1Division of Medical Ethics, Department of Population Health, NYU School of Medicine, 227 East 30th Street, Office 723, New York, NY, 10016, USA. Jennifer.miller@nyumc.org.

BMC Research Notes
|July 30, 2017
PubMed
Summary
This summary is machine-generated.

Most expanded access programs provide drugs, with 76% of experimental drugs ultimately receiving FDA approval. These industry-funded programs primarily serve adults with conditions like HIV and leukemia.

Keywords:
21st Century Cures ActAccess to medicinesBioethicsCompassionate useEthicsExpanded accessExperimental drugsPharmaceutical industryPolicyReal-world evidenceRight to Try Laws

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Area of Science:

  • Clinical research
  • Drug development
  • Regulatory affairs

Background:

  • Expanded access and compassionate use programs provide investigational medical products to patients with serious or life-threatening conditions when no comparable or satisfactory alternative therapy options exist.
  • These programs play a crucial role in the drug development pathway, offering patients access to potentially life-saving treatments outside of clinical trials.

Purpose of the Study:

  • To characterize the landscape of expanded access and compassionate use programs registered on ClinicalTrials.gov.
  • To determine the proportion of drugs provided through these programs that subsequently gain FDA marketing approval.

Main Methods:

  • A comprehensive search of ClinicalTrials.gov was conducted to identify all registered expanded access and compassionate use programs.
  • Data on program funding, type of product provided (drug, biologic, device), conditions treated, patient population, and reported outcomes were extracted and analyzed.

Main Results:

  • 398 expanded access programs were identified, with 61% industry-funded. Most programs provided access to drugs (71%), covering conditions including HIV, leukemia, and multiple myeloma.
  • 76% of the 210 unique experimental drugs accessed through these programs ultimately received FDA approval.
  • The majority of programs (82%) enrolled adults and seniors, and only 2% reported results on ClinicalTrials.gov.

Conclusions:

  • Expanded access programs are predominantly industry-funded and provide access to a wide range of investigational drugs for various conditions.
  • A high percentage of drugs made available through these programs achieve subsequent FDA marketing approval, suggesting their role in facilitating drug development and patient access.