Specific guidance for companies aiming to more quickly develop drugs for chronic fatigue syndrome (CFS) and myalgic encephalomyelitis (ME) will be available next year from the FDA, said Janet Maynard, from the agency’s office of pulmonary, allergy and rheumatology products.

During last week’s public teleconference to discuss CFS/ME treatments, Maynard said the FDA wants to “catalyze” drug development in CFS and ME, and the new draft guidance may help, though she cautioned that such documents “are not roadmaps.”

Ever since Philadelphia-based Hemispherx Bioscience Inc., which had the only drug in development for CFS, was hit with a complete response letter for its Toll-like receptor 3 modulator Ampligen (rintatolimod), the pressure’s been on. (See BioWorld Today, Feb. 6, 2013.)

Patients rallied, trying to make the agency establish better guidelines and maybe even attack CFS and the related condition of ME in the full-court way that once was applied to HIV. Their pleas at the Arthritis Advisory Committee meeting in December 2012 seemed to leave the gatekeepers unfazed, but the work continued, including a hunger fast by one CFS patient, Robert Miller, given Ampligen through a special program. (See BioWorld Today, Dec. 21, 2012.)

Miller testified during the April workshop, saying he was about to “crash” with another episode of CFS – a disease that clinicians once believed did not exist.

“Because of Ampligen, I will bounce back,” Miller said. “I’ve already started to feel better just from sitting and resting, but had I not been on Ampligen for this trip, I would not have made it here. Before Ampligen, I was literally bedbound. When people talk about being bedbound, I mean, we’re like bricks, we can’t be moved. My wife would come in and check on me to see if I was breathing because I would sleep for 18 days at a time. I didn’t get up to eat. I didn’t get up to go to the rest room.”

The FDA last week followed up April’s workshop on CFS and ME with the stakeholder teleconference last week to talk about next steps. It’s all part of an effort mandated to the FDA by the Patient-Focused Drug Development initiative (part of the fifth authorization of the Prescription Drug User Fee Act), intended to more systematically gather patient perspectives on their condition and available therapies.

Over the next five years, the FDA is holding at least 20 public meetings, each focused on a specific disease area. CFS and ME were the first among the 20 diseases to be talked about.

Ahead of the teleconference, the agency released its summary of the workshop, “The Voice of the Patient,” which concluded that the FDA recognizes “patients have a very unique ability to contribute to our understanding of this broader context of the disease, which is important to our role, and that of others, in the drug development process.”

Regulators, the report said, “share the patient community’s commitment to facilitate the development of safe and effective drug therapies for this disease,” but no specific action was promised.

Moderated by Sandra Kweder, deputy director of the FDA’s office of new drugs, the teleconference included remarks from Sara Eggers, from the drug-safety operations department, who also handled the first day of the two-day workshop in April and provided an overview of that event first.

The substance of the teleconference came later, when the phone lines opened to patients and their advocates. One pointed out that the FDA “has heard testimony [regarding CFS] for more than a decade,” yet had taken no steps. “What I’m hearing today, particularly from Janet, is that you are acting upon it. This is very encouraging.”

The usual, time-consuming route of ushering a drug through the channels for approval may not be the smartest way to go in the short term, the caller suggested.

“What we need now is exactly what you’re describing – plus possibly another stakeholders’ meeting – is to bring in the pharmaceutical companies, who have already got drugs that are being used off-label by leading doctors, leading clinicians, to treat the patients,” she said. “Patients are going to these doctors in very minuscule numbers compared to the number suffering, because they can’t get to the doctors,” since there is no organized, nationwide network of access.

The caller mentioned “antivirals, Ampligen, rituximab [Rituxan, Biogen Idec Inc. and Roche AG] and others. These companies need to be brought to the table. You need to seek them out, and the patient community will be more than happy to help you with that.”