What is the compassionate use program?
What is the compassionate use program?
Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable …
How do you qualify for compassionate use?
To be eligible for Right to Try, a person must:
- Be diagnosed with a life-threatening disease or condition.
- Have tried all approved treatment options for the disease or condition.
- Have a doctor certify that they are unable to participate in a clinical trial for the investigational drug.
What are early access programs?
Early access programs are a means by which investigational therapies are made available, in certain circumstances, to treat patients with serious diseases who are unable to participate in an ongoing clinical trial or whose treatment options are otherwise limited.
What is extended access program?
An Expanded Access Program (EAP) allows physicians and patients access to pre-approval, investigational drugs outside of the clinical trial setting. An EAP can also be called a Managed Access Program (MAP), Early Access Program, or Compassionate Use Program (CUP).
Is EUA the same as FDA approved?
The FDA also explains that an EUA is a different standard than an FDA approval; however, in the case of an investigational vaccine developed for the prevention of COVID-19, both require submitting extensive data demonstrating the vaccine’s safety and effectiveness.
How does an EUA work?
Under an EUA, FDA may allow the use of unapproved medical products, or unapproved uses of approved medical products in an emergency to diagnose, treat, or prevent serious or life-threatening diseases or conditions when certain statutory criteria have been met, including that there are no adequate, approved, and …
Who pays for compassionate use drugs?
You may pay out of pocket for experimental treatment. The drug company may charge you for the experimental drug. Also, your insurance company is unlikely to pay associated costs of your treatment, such as fees for your doctor to administer the experimental drug and monitor side effects.
What is name patient use?
“Named-patient basis” is another way of obtaining medicine before authorization where a doctor requests supply of an unauthorized medicine directly from the manufacturer to treat an individual patient under the doctor’s direct responsibility.
Who pays expanded access?
The cost of expanded access then is partly borne by the patient’s doctor, who must invest significant extra time to request the drug and comply with the additional responsibilities associated with using it. These programs are for the primary purpose of treatment, not research.
How is a compassionate use program set up?
A compassionate use program is usually set up and managed by the drug companies, or a third-party provider (like WEP Clinical). However, in some cases a research or medical institution can also run a program. The drug manufacturer must agree to provide the drug through compassionate use in order for patients to access it.
How is ICT 107 used to treat glioblastoma?
It is designed to activate a patient’s own immune system to target six different tumor-associated antigens, or proteins, found on glioblastoma cancer cells. ICT-107 was designed an orphan drug by the U.S. Food and Drug Administration (FDA) in 2010 and by the European Medicines Agency (EMA) in 2014.
Is there a Phase 1 trial for ICT 107?
A Phase 1 trial evaluated the safety and immunogenicity (ability to trigger an immune response) of ICT-107 in 21 patients. The vaccine was administered three times at two-week intervals.
What kind of cells are in ICT 107?
ICT-107 consists of a patient’s own dendritic cells (DCs), specialized immune system cells, which are pulsed with six synthetic peptides found on glioblastoma tumor cells.