TEXT:
Responding to Ebola: The View From the FDA
Laurie Scudder, DNP, NP, Luciana Borio, MD
Disclosures August 20, 2014
WHO Backs Use of Experimental Treatments for Ebola
U.S. Emergency Labs Ready to Work on Ebola Drugs If Asked
Keeping Ebola ∈Check: Advice From the CDC
DRUG & REFERENCE INFORMATION
Editor's Note: The issue of compassionate use of unapproved therapies has been brought to the attention of all healthcare providers recently after the decision to treat 2 Americans infected with the Ebola virus with an experimental serum never before used ∈ humans. With the Ebola epidemic deemed to be out of control by the World Health Organization (WHO), the need for effective treatments has researchers and regulators scrambling to find, test, and potentially approve previously untested therapies.
In a press release ∈August, WHO discussed the ethics of using Ebola drugs that have never been cleared for human use, wary of a long history of medicines being tested on people who were never properly informed of the risks. In the countries hardest hit by Ebola, suspicion of foreign medical workers is already widespread.
Medscape spoke with Luciana Borio, MD, at the US Food and Drug Administration (FDA) about the issue of compassionate use and their efforts to respond to this crisis. Dr. Borio serves as the Acting Deputy Chief Scientist and Assistant Commissioner for Counterterrorism Policy ∈ the Office of Counterterrorism and Emerging Threats at the FDA. In this capacity, Dr. Borio is responsible for providing leadership, coordination, and oversight for FDA's national and global health security, counterterrorism, and emerging threat portfolios.
She serves as FDA's point of entry on policy and planning matters concerning counterterrorism and emerging threats, and collaborates across the US government and internationally on actions to advance global health security and US national security.
Medscape: Can you define "compassionate use" as a regulatory and legal concept?
Luciana Borio, MD: I'd like to begin by taking a step back. Ultimately, the goal for the FDA is to have products that are approved, licensed, or cleared to prevent or treat diseases. However, during a public health emergency, such as the one we are currently facing with Ebola, we don't always have these approved products available, and the best way for us to meet patients' needs might be the use of an unapproved product.
FDA has mechanisms that we can use to facilitate patient access to investigational products; one of these is the Emergency Investigational New Drug (IND) application, often referred to as "compassionate use." It is one of a number of mechanisms under our expanded access program.
Through this emergency IND mechanism, we can allow the use of an investigational product outside of a clinical trial being conducted to develop the product toward approval, ∈ order to treat a patient with a serious or immediately life-threatening disease when the patient has no comparable or satisfactory alternative treatment option.
To evaluate a request for an emergency IND, we have to assess whether the benefits outweigh the risks on the basis of the best available scientific evidence that we have because, clearly, the data package at this point is often quite incomplete. The product may be effective or may not, and we also may see sometimes unexpected serious side effects. Sometimes we don't know exactly what is the most appropriate dose, for example, to give to a sick patient. We have only incomplete information about the use of these products, but we do the best we can to conduct a proper benefit/risk assessment with the available scientific data.
Under this mechanism, it is usually the patient's clinician who contacts the FDA requesting the emergency IND or compassionate use exemption. If, after an evaluation of the application, FDA believes the use is appropriate, the doctor will then make arrangements directly with the product developer to obtain the product. The manufacturer may or may not be willing to make the product available outside of the clinical trial, and FDA cannot compel the company to do so. So when the company provides expanded access to its product, it is doing so voluntarily.
http://www.medscape.com/viewarticle/829611 (Accessed on August 25th, 2014, edited)
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