Pandora Org, a national nonprofit ME/CFS advocacy organization, and other patient organizations say a recent report from the FDA shows it must take further action to open up opportunities for ME/CFS drug development. Titled The Voice of the Patient, this report is a detailed summary of an April 25 FDA patient-focused drug development meeting, the first of its kind, at which patients explained the reality of Myalgic Encephalomyelitis (ME)/Chronic Fatigue Syndrome (CFS).

"The report reveals the personal and professional devastation caused by the cognitive and energy dysfunctions of ME/CFS," said Lori Chapo-Kroger, RN, president of Pandora Org.

The FDA's patient-focused drug development initiative is a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) that aims to more systematically gather patient perspectives and available therapies to treat their conditions. As part of this commitment, FDA is holding at least 20 public meetings over the next five years, each focused on a specific disease area. ME/CFS was the first to be addressed.

The meeting was the result of a year-long patient campaign targeted at the FDA and asking for a stakeholders' meeting. Letters were sent to the FDA and key congressional members. The effort secured letters from senators Robert P. Casey (D-PA), Kay Hagan (D-NC) and Richard Blumenthal (D-CT).

"We appreciate the response of [the FDA]," said Chapo-Kroger. "However, there is still no FDA drug development guidance or clarified endpoints and outcome measures for this disease after two decades, and the April meeting failed to focus on that topic. Therefore, more action is needed to remove these roadblocks and further facilitate drug development for this disease that is causing tremendous suffering and even death."

No FDA-approved drug is labeled for ME/CS. In the last 20 years, at least eight drugs have been in the pipeline with one remaining stuck. The patient organizations know once one treatment is approved, additional research and treatments will follow; 13 drugs were approved in the nine years following the approval of AZT for HIV/AIDS.

"The FDA has considerable latitude in determining what burden of proof a drug must meet, including the legal authority to waive well-controlled and adequate studies, particularly for disorders such as ME/CFS that have no FDA-approved drugs," said Chapo-Kroger. "We expect the FDA to provide considerable creativity and flexibility."

Approximately 17 million people worldwide, at least one million in the U.S., have ME/CFS. A quarter of all patients are entirely house, bed or wheelchair bound. One in 10 dies prematurely due to major organ failure, cancer, heart disease or suicide. The Centers for Disease Control and Prevention estimates the ME/CFS economic impact to be more than $14 billion per year in healthcare costs; an 84% increase over the average American’s per year medical costs.