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FDA wants to fast-track drugs for Chronic Fatigue Syndrome

Monday 24 March 2014


From's Adrienne Dellwo:



FDA Wants to Fast-Track Drugs for Chronic Fatigue Syndrome

March 19, 2014

The U.S. FDA is putting out guidance for drug companies on how they can quickly get drugs approved for chronic fatigue syndrome (ME/CFS). For now, it's seeking comment on a new document that's expected to go into effect.

ME/CFS was first recognized several decades ago, but we still don't have any approved drugs for it. Major problems are that researchers are still arguing over definitions and inclusion criteria, we don't yet understand the underlying causes, and what's currently labeled "chronic fatigue syndrome" could include multiple subtypes or even several different, but related, conditions.

That all makes it hard for drug manufacturers to target the problem and explore potential therapies. The problem was made clear with the first drug to seek FDA approval: Ampligen (rintatolimod), made by Hemispherx Biopharma. The company's application ran into problems with providing enough evidence to convince regulators that it should go on the market.

Now, however, the agency says ME/CFS will be first condition to fall under a special provision of its Safety and Innovation Act. That means the FDA will set up something called a "Patient-Focused Drug Development Initiative," which allows researchers to include the views of patients instead of just data.

Citing the unmet medical need of ME/CFS patients, the FDA has issued a draft of a guidance document to help companies develop new drugs and get them through the system quickly. Comments and suggestions will be taken for 60 days, after which the FDA will review them and make final decisions about the document.

Once the final document is out, it would not create any regulations, but would instead offer insight into the FDA's current mode of thinking, which can better help drug companies design trials and navigate the approval process.

The Background section of the document provides an excellent summation:

"CFS/ME is a complex disease that can be debilitating. The exact cause or causes of CFS/ME are unknown. Symptoms affect several body systems and may include severe fatigue or exhaustion, unrefreshing sleep, weakness, muscle and joint pain, impaired memory or mental concentration, tender lymph nodes, sore throat, headaches, and sleep dysfunction. Many patients experience post-exertional malaise, which may occur without warning and upon even minimal physical or cognitive exertion, and is associated with acute exacerbation of these symptoms. The nature and severity of symptoms vary from person to person, and diagnosis is challenging because there are no specific tests for the diseases. Post-exertional malaise and cognitive impairment are symptoms that can be particular severe and disabling.

According to the Centers for Disease Control and Prevention (CDC), between one and four million people in the United States are afflicted with CFS. Although CFS/ME is most common in 40- to 60-year-old women, CFS/ME affects both sexes and all racial, age, and socioeconomic groups. The disease may occur with a sudden onset, such as following and infection, or may occur with a gradual onset. Some patients improve spontaneously; however, many patients experience a prolonged course of illness with either periods of remission and exacerbation or steady decline.

CFS/ME affects patients' ability to function in daily activities of work, school household management, and personal care, Many patients with CFS/ME are bedbound some or all of the time and experience loss of careers, decreased quality of family life, social isolation, and feelings of hopelessness."

- FDA Draft Guidance document on CFS/ME

Under these provisions, drug makers would be able to get fast-track designations, breakthrough therapy designations, accelerated approval, and priority review status. All of those features could help get drugs through the process and to the public much faster.

One part of the document that's sure to be controversial is that the FDA is leaving it open to the researchers to use whatever case definition they want, while making clear that the criteria need to be justified and clearly explained. (See what definitions of ME/CFS are currently in use: Exploring Inflammation & Autoimmunity in ME/CFS.)

Patient-Reported Outcomes

A key element of the document is the inclusion of patient-reported outcomes (PRO.) The FDA is asking for researchers to include reliable measures of PRO in their reports of drug efficacy, alongside of or possibly instead of objective measures.

That allows for easier reporting of results that can't be measured by anything other than asking the patient how they feel. For a condition like ME/CFS, which is notoriously difficult to measure and quantify, this is a huge point.

You can read the entire draft document here: Chronic Fatigue Syndrome/Myalgic Encephalomyelitis: Developing Drug Products for Treatment. Instructions for submitting comments to the FDA are on the title page.

Importance of Drug Research

A lot of people are skeptical of drug research, and rightfully so. Drug companies obviously have a huge financial stake in them, so it only stands to reason that there'd be pressure on researchers to get positive results.

However, ME/CFS has always been drastically under-researched. Any quality research that's done on it can help us understand what's going on in your body is a good thing. Something discovered in a drug trial could spur a whole new line of inquiry that could eventually mean people like you suffer a lot less, and that's on top of possibly getting some drugs approved.

Companies are also more likely to invest in ME/CFS research if drugs are likely to be fast-tracked. Government and private grants may come more easily due to increased attention from the FDA, as well.

FDA approval of ME/CFS drugs would even benefit the people with this condition who don't take the drugs. Before drug advertisements, most people had never heard of fibromyalgia. The ads have raised awareness and public acceptance immensely. Drug companies even educate doctors (albeit, with their own goals in mind) about illnesses, meaning more doctors will start to have a clue about ME/CFS.

Do you think it's high time the FDA made ME/CFS a priority? Do you think the agency should have been more specific about definitions and inclusion criteria? What types of drugs would you like to see come to market? Leave your comments here!


Photo © Martin Poole/Getty Images


The above originally appeared here.



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