Compassionate-Use Clinical Study

A Compassionate-Use Clinical Study is a interventional clinical study that allows patients with serious or life-threatening health conditions, who do not meet a clinical trial eligibility criteria, to receive an experimental therapy.

• AKA: Temporary Authorization for Named-Patient Use Program, Expanded Access Program.
• Context:
  • It can be part of a Investigational New Drug (IND) Program or Investigational Device Exemption (IDE) Program.
  • It can be categorized as:
    • Single-Patient Compassionate Use Clinical Trial;
    • Intermediate-Size Population Compassionate Use Clinical Trial;
    • Treatment IND/Protocol Compassionate Use Clinical Trial.
  • It can be categorized as:
    • Available Expanded Access;
    • No-Longer-Available Expanded Access ;
    • Temporarily-Not-Available Expanded Access;
    • Approved-for-Marketing Expanded Access.
  • …
• Example(s):
  • Patients in USA who only allowed to use medical cannabis under the program who had certain conditions such as: glaucoma, sleep disorders, cancer, etc.
  • NCT03620318: Individual Patient Compassionate Use of Crenolanib,
  • NCT03723148: Individual Patient Compassionate Use of Fedratinib,
  • NCT04233047: Fostemsavir Compassionate Use Named Patient Program for the Treatment of Human Immunodeficiency Virus 1 (HIV-1),
  • NCT03723135: Expanded Access for CC-486 (compassionate use clinical study of oral azacitidine as viable treatment of acute myelogenous leukemia),
  • …
• Counter-Example(s):
  • Emergency Use Authorization (EUA);
  • Right-to-Try Law.
• See: Drug, Medical Wearable Device, U.S. Drug Enforcement Administration, U.S. National Institute on Drug Abuse, Decentralized Clinical Trial, Observational Clinical Study, Placebo-Controlled Clinical Trial, Active-Treatment Controlled Clinical Trial.

---

---

References

• https://scholar.google.com/scholar?oi=gsb40&q=%22compassionate%20use%22%20%22such%20as%22&lookup=0&hl=en

2022a

• (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 status:
      • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
      • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
      • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
      • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
    • 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.

2022b

• (Wikipedia, 2022) ⇒ https://en.wikipedia.org/wiki/Investigational_New_Drug#Types Retrieved:2022-1-22.
  • Emergency Use INDs, also called compassionate use or single-patient INDs, are filed for emergency use of an unapproved drug when the clinical situation does not allow sufficient time to submit an IND in accordance with 21 CFR §§ 312.23, 312.24. These are most commonly used for life-threatening conditions for which there is no standard treatment.
  • Treatment INDs are filed to make a drug available for treatment of serious or immediately life-threatening conditions prior to FDA approval. Serious diseases or conditions are stroke, schizophrenia, rheumatoid arthritis, osteoarthritis, chronic depression, seizures, Alzheimer's dementia, amyotrophic lateral sclerosis (ALS), and narcolepsy.

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#C 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)

2021c

• (Wikipedia, 2021) ⇒ https://en.wikipedia.org/wiki/Glossary_of_clinical_research#E Retrieved:2021-12-31.
  • 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)

2020

• (Grein et al., 2020) ⇒ Jonathan Grein, Norio Ohmagari, Daniel Shin, George Diaz, Erika Asperges, Antonella Castagna, Torsten Feldt et al. (2020). “Compassionate Use of Remdesivir for Patients with Severe Covid-19.” New England Journal of Medicine 382, no. 24
  • ABSTRACT: We provided remdesivir on a compassionate-use basis to patients hospitalized with Covid-19, the illness caused by infection with SARS-CoV-2. ...

    ... In this cohort of patients hospitalized for severe Covid-19 who were treated with compassionate-use remdesivir, clinical improvement was observed in 36 of 53 patients (68%). Measurement of efficacy will require ongoing randomized, placebo-controlled trials of remdesivir therapy.

---