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Clinical Trials and Compassionate Use in Multiple Myeloma
Posted: May 26, 2018
Clinical Trials and Compassionate Use in Multiple Myeloma image

Every myeloma treatment available, even our first line therapies, began as a clinical trial. There are so many promising treatments currently in clinical trials, but the process can be long - often taking over a decade before a drug becomes approved and widely available.

This process for new drugs is necessary to ensure our safety - but can feel frustrating for patients, especially those with aggressive disease or limited remaining treatment options that may not have years to wait. With situations like this in mind, the Expanded Access program (also known as 'Compassionate Use') was created by the FDA in 1987. It allows for patients who meet certain requirements to receive drugs that are not yet approved for treatment, and still in the clinical trial process.

There are three main ways that the Expanded Access program is currently utilized: single patient, a small group of patients, or via a protocol by the drug sponsor for a widespread, large group of patients.

The current requirements for all Expanded Access Uses are:

  • The patient and a licensed physician are both willing to participate.
  • The patient's physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient’s disease or condition.
  • That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition.
  • FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;
  • FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval;
  • The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator (or the patient’s physician in the case of a single patient expanded access request) submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with FDA’s statute and applicable regulations for investigational new drugs (IND) or investigational device exemption applications (IDEs), describing the use of the investigational product; and
  • The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial.

So how does the Expanded Access program work? The process starts with your doctor, who will usually contact the drug manufacturer to make sure they are willing to provide the drug for expanded access. If they are, the physician will initiate a investigational new drug (IND) exemption request, which is then submitted to the FDA. If the request is approved by the FDA (99% of requests are), approval must then be obtained from the institutional review board (IRB) where the patient will receive the treatment.  

It can be a challenging process that is not without it’s share of roadblocks. One of the biggest challenges that patients face are managing the cost of receiving the drug. Most insurances will not cover the cost of receiving experimental treatments. Some pharmaceutical companies have programs in place that will cover some of the costs associated with compassionate use, but some do not. When a drug sponsor receives a large number of IND requests, they have the option to create a protocol for a large number of patients to receive their drug via Expanded Access. 

Karyopharm has an expanded access protocol for Selinexor, a selective inhibitor of nuclear export (SINE) which works by disrupting survival mechanisms in myeloma cells. You can find more information about their program at

Considering expanded access? Not sure how to start a conversation with your doctor? You can always call us at (888) 828-2206, and we can help you get started. The resources below are also a great place to continue your research:

The author Jennifer Ahlstrom

about the author
Jennifer Ahlstrom

Myeloma survivor, patient advocate, wife, mom of 6. Believer that patients can help accelerate a cure by weighing in and participating in clinical research. Founder of Myeloma Crowd by HealthTree and the HealthTree Foundation.

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