Firestormm
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Regulatory Focus News:
FDA Says Sponsors of Chronic Fatigue Syndrome Drugs can Rely on Patient-Reported Outcomes
10 March 2014
Draft guidance: HERE
Federal Register: HERE
By Alexander Gaffney, RAC
The US Food and Drug Administration (FDA) has released a new draft guidance document intended to expedite the development of drugs to treat chronic fatigue syndrome (also known as myalgic encephalomyelitis)—a poorly understood condition that has recently been subject to significant interest by FDA regulators.
Background
Chronic fatigue syndrome (CFS) affects patients by causing severe, persistent and often debilitating fatigue. Little is known about the underlying causes of the condition, or even if it is a single condition or a group of related conditions. Despite numerous theories—including some that have since been discredited—there are no tests to determine its cause.
That leaves patients in something of a bind: The effects of CFS are real, but without an understanding of its underlying causes, researchers and manufacturers are unable to target potential or existing therapies to treat it.
That bind was put on display and amplified in February 2013, when FDA—following the advice of one of its advisory committee panels—did not recommend Hemispherx's Ampligen (rintalolimod), saying it needed additional clinical evidence and data before it could consider approving the drug.
But CFS patients are also the first to benefit from a special provision of the Food and Drug Administration Safety and Innovation Act (FDASIA), which calls for FDA to set up a "Patient-Focused Drug Development Initiative" to better take into account the views of patients during the regulatory review process.
"The human drug and biologic review process could benefit from a more systematic and expansive approach to obtaining input from patients who experience a particular disease or condition," FDA explained in a Federal Register posting announcing the program in September 2012.
In October 2012, FDA sat down with CFS patients in order to hear their views on the regulatory process, most notably including their tolerance for risk...
Read more: HERE