This is the first and most important step. The drug manufacturer must agree to provide the investigational drug. Without its agreement, you will not be able to proceed and your patient will not receive the drug.
Who to contact?
You will need to find a point of contact at the drug company to whom you will send a request to provide the drug, along with your patient’s medical history. In December 2016 the 21st Century Cures Act required companies to post their compassionate use policies, contact information, and the time it takes to acknowledge the receipt of a request. However, not all companies comply.
Your point of contact may be a regulatory affairs official, the chief medical officer, CEO, VP for clinical development, or for smaller companies it may be a general inquiries line.
Some companies post a form on their website for you to complete, others provide a phone number or an email address to send an inquiry. There is no standard industry application form. Most drug manufacturers will consider your request on a case-by-case basis.
There is no legal limit on the time a company can take to respond to a request, and there is no requirement for companies to provide reasons for denials. If the company declines to provide the drug, FDA advises that your options are to use approved treatments, enroll the patient in a clinical trial or an ongoing expanded access program, or to consider designing a new protocol or a new trial.
Obtain LOA from a drug company
If the company agrees to provide the drug, ask for a letter of authorization (LOA). You will need the LOA to apply for FDA’s authorization.
In most cases, manufacturers will have submitted documentation to FDA, and received permission to study the drug you are requesting. The LOA should reference this documentation, including the number of the investigational new drug (IND), or the New Drug Application (NDA) or Biologics License Application (BLA) number. These numbers are issued by FDA to the company.
If a manufacturer does not have an IND, FDA requires the following information in the letter: chemistry, manufacturing, and controls information, and pharmacology and toxicology information, including a description of the manufacturing facility.
The drug manufacturer may prefer that you submit to FDA a separate individual patient IND application [What is it?]. The company will be supplying the drug, but will not itself be submitting the IND for your patient’s access. In this case, you, not the company, will be the sponsor for that IND.
If FDA authorizes the compassionate use request, it will issue another IND number to you. The IND number serves as FDA’s authorization for the company to provide the drug. Upon receipt of the IND number from the FDA, you must then provide that number to the manufacturer so the company can ship the drug directly to you.
How we can help?
• Please search our database of drug manufacturers’ contacts for compassionate use requests (under construction), or contact us for assistance in locating the point of contact at a company. We will do the search for you.
• We can assist you in understanding the criteria that the company may consider when reviewing the application. We can review the request before it is sent.
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