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FDA Compassionate Use

Rare genetic disorders present significant challenges to access appropriate treatment.  Where experimental medications are available and demonstrate a likely benefit we can work with medical doctors to obtain medications through the FDA Compassionate use process.  An overview of the process is provided below:


The FDA's compassionate use process, also known as expanded access, is a pathway for patients who have serious or life-threatening illnesses to gain access to investigational drugs or medical devices that have not yet been approved by the FDA for general use. 


It's important to note that the compassionate use process is not a guarantee that a patient will receive an investigational product, and that the FDA must balance the needs of individual patients with the need to ensure the safety and effectiveness of new treatments



To request access to an investigational product through the compassionate use process, a patient or their physician must first contact the drug or device manufacturer to request permission to use the product outside of a clinical trial. The manufacturer will then submit a request to the FDA on behalf of the patient, which must include detailed information about the patient's medical condition, the investigational product, and the proposed treatment plan.



The FDA will review the request to determine whether the potential benefits of the treatment outweigh the potential risks for the patient. The FDA will also consider whether there are any alternative treatments available, and whether the patient is eligible for any ongoing clinical trials.


If the FDA approves the request, the patient may receive the investigational product outside of a clinical trial. However, the patient must give informed consent, and the manufacturer must provide the product for free or at cost.  

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