Approved-for-Marketing Expanded Access

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An Approved-for-Marketing Expanded Access is a Compassionate Use Clinical Trial in which an investigational treatment or drug being studied has been clinically approved for use by the public.



References

2022

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.[1][2][3]

      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.

  1. Patil, S (2016). "Early access programs: Benefits, challenges, and key considerations for successful implementation". Perspectives in Clinical Research. 7 (1): 4–8. doi:10.4103/2229-3485.173779. PMC 4763516. PMID 26955570.
  2. Balasubramanian, G.; Morampudi, S.; Chhabra, P.; Gowda, A.; Zomorodi, B. (2016), "An overview of Compassionate Use Programs in the European Union member states", Intractable & Rare Diseases Research, 5 (4): 244–254, doi:10.5582/irdr.2016.01054, PMC 5116859, PMID 27904819
  3. Hyry, HI; Manuel, J; Cox, TM; Roos, JC (21 August 2015). "Compassionate use of orphan drugs". Orphanet Journal of Rare Diseases. 10: 100. doi:10.1186/s13023-015-0306-x. PMC 4546220. PMID 26292942.

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)