Intermediate-Size Population Compassionate Use Clinical Trial

An Intermediate-Size Population Compassionate Use Clinical Trial is a Compassionate Use Clinical Trial that involves a more than one patient but fewer than a widespread and large portion of a population.

• Example(s):
  • NCT02094222: Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease,
  • NCT04646031: Expanded Access to T89 for Treatment Use in Intermediate-size Patients Population With COVID-19,
  • NCT05031546: Intermediate-Size Patient Population Expanded Access Program for Intravenous Difelikefalin,
  • …
• Counter-Example(s):
  • Single-Patient Compassionate Use Clinical Trial.
  • Treatment IND/Protocol Compassionate Use Clinical Trial.
• See: Investigational New Drug (IND) Program, Investigational Device Exemption (IDE) Program, Approved Drug, Medical Wearable Device, U.S. Drug Enforcement Administration, U.S. National Institute on Drug Abuse, Decentralized Clinical Trial.

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References

2022

• (ClinicalTrials.gov) ⇒ https://clinicaltrials.gov/ct2/about-studies/glossary Retrieved 2022-01-21.
  • QUOTE: This glossary will help you understand words and phrases frequently used on ClinicalTrials.gov. Many of these words are also used by clinical researchers and others in the same or a similar manner. But the definitions below are provided to explain content on ClinicalTrials.gov only.

    For help with medical terms, see the MedlinePlus® Medical Encyclopedia (...)

    • Expanded access: A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
    • (...)
    • Expanded access type: Describes the category of expanded access under U.S. Food and Drug Administration (FDA) regulations. There are three types of expanded access.
      • Individual Patients: Allows a single patient, with a serious disease or condition who cannot participate in a clinical trial, access to a drug or biological product that has not been approved by the FDA. This category also includes access in an emergency situation.
      • Intermediate-size Population: Allows more than one patient (but generally fewer patients than through a Treatment IND/Protocol) access to a drug or biological product that has not been approved by the FDA. This type of expanded access is used when multiple patients with the same disease or condition seek access to a specific drug or biological product that has not been approved by the FDA.
      • Treatment IND/Protocol: Allows a large, widespread population access to a drug or biological product that has not been approved by the FDA. This type of expanded access can only be provided if the product is already being developed for marketing for the same use as the expanded access use.

2021a

• (Wikipedia, 2021) ⇒ https://en.wikipedia.org/wiki/Expanded_access Retrieved:2021-12-3.
  • Expanded access or compassionate use is the use of an unapproved drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress.

    These programs go under various names, including early access, special access, or managed access program, compassionate use, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access.

    In general the person and their doctor must apply for access to the investigational product, the company has to choose to cooperate, and the medicines regulatory agency needs to agree that the risks and possible benefits of the drug or device are understood well enough to determine if putting the person at risk has sufficient potential benefit. In some countries the government will pay for the drug or device, but in many countries the person must pay for the drug or device, as well as medical services necessary to receive it.

    In the US, compassionate use started with the provision of investigational medicine to certain patients in the late 1970s, and a formal program was established in 1987 in response to HIV/AIDS patients requesting access to drugs in development. An important legal case was Abigail Alliance v. von Eschenbach, in which the Abigail Alliance, a group that advocates for access to investigational drugs for people who are terminally ill, tried to establish such access as a legal right. The Supreme Court declined to hear the case, effectively upholding previous cases that have maintained that there is not a constitutional right to unapproved medical products.

2021b

• (Wikipedia, 2021) ⇒ https://en.wikipedia.org/wiki/Glossary_of_clinical_research Retrieved:2021-12-3.
  • Compassionate use
    • A method of providing experimental therapeutics prior to final FDA approval for use in humans. This procedure is used with very sick individuals who have no other treatment options. Often, case-by-case approval must be obtained from the FDA for "compassionate use" of a drug or therapy. (NLM)
  • Compassionate use trial
    • A way to provide an investigational therapy to a patient who is not eligible to receive that therapy in a clinical trial, but who has a serious or life-threatening illness for which other treatments are not available. Compassionate use trials allow patients to receive promising but not yet fully studied or approved therapies when no other treatment option exists. Also called expanded access trial. (NCI)
  • (...)
  • Expanded access
    • Refers to any of the FDA procedures, such as compassionate use, parallel track, and treatment IND that distribute experimental drugs to participants who are failing on currently available treatments for their condition and also are unable to participate in ongoing clinical trials. (NLM)
  • Expanded access trial
    • A way to provide an investigational therapy to a patient who is not eligible to receive that therapy in a clinical trial, but who has a serious or life-threatening illness for which other treatments are not available. Expanded access allows a patient to receive promising but not yet fully studied or approved therapies when no other treatment option exists. Also called compassionate use trial. (NCI)

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