Mary Dimmock presented a shortened version of these comments at the FDA meeting today on the Patient Focused Drug Development initiative. She has kindly given me permission to post her comments in full here.
My name is Mary Dimmock and I am a member of a national alliance of patient organizations and patient advocates representing patients with Chronic Fatigue Syndrome, also known as myalgic encephalomyelitis or ME/CFS.
The popular misconception is that ME/CFS is chronic tiredness due to deconditioning, depression or poor diet. That is what I thought until my energetic, smart, adventurous 23 year old son was struck down by ME/CFS after contracting Giardia while backpacking across Asia. Overnight, he went from academic excellence and scaling mountains to being unable to work, seldom able to leave the house and too often unable to do more than lay on his side in a dark room, in constant pain.
He is not alone. One million Americans of all ages, races and socioeconomic groups and 17 million people worldwide have been struck down overnight by this complex, multi-system disease that causes significant immune and neuroendocrine abnormalities; brain dysfunction and neurocognitive defects; cardiovascular and autonomic disturbances and abnormalities in energy production including mitochondrial dysfunction. As a result, patients suffer devastating functional impairment that results from the profound exhaustion, unrefreshing sleep, joint and muscle pain and cognitive problems that include difficulty thinking, slower processing speed and impairment of memory. These symptoms are exacerbated after even minimal mental or physical activity and can result in a relapse called post-exertional neuroimmune exhaustion that can last hours, days, or weeks. For some, even minimal activities like talking to a friend on the phone for a few minutes, taking a bath or making a meal for themselves can cause post-exertional neuroimmune exhaustion.
The CDC has said that ME/CFS can be as disabling as multiple sclerosis, lupus, rheumatoid arthritis, heart disease, end-stage renal disease… and similar chronic conditions.” Patients can be sick for decades and many have been sick since outbreaks in the mid-1980s. Twenty-five percent of patients are bed-bound, house-bound or wheel-chair bound. Ten percent of patients are pediatric, some as young as 5. The inability to attend school, play with friends or even participate in family activities during their developmental years has a particularly harsh and lifelong effect on children. Overall recovery is rare and one study found that patients are more likely to die prematurely from cancer, heart disease or suicide.
And yet, today, there are no treatments for ME/CFS that are capable of even minimally improving patient functionality of changing the long-term outcome of the disease. There is only one ‘disease modifying’ drug being progressed through the FDA review process. The lack of agreement on definition, endpoints and how to measure treatment outcomes has impeded drug development. The drugs that are used provide only very limited relief for specific symptoms like pain but have little impact on the overall level of functioning. There are a small handful of experts nationwide who will prescribe antivirals or immune modulators to try to change the course of the disease but most doctors are unwilling to prescribe such drugs. Some of this reluctance is because they do not recognize that for patients, the disease severity warrants the risk.
The resultant reality for many patients is that they will spend every one of the rest of their days in pain, disability and isolation, functionally so limited that they are unable to work, care for their families or even sometimes take care of themselves.
It is time that we peeled back the misunderstanding and looked closely at the severity of this disease, the dramatic loss of functionality that these patients experience and the total lack of approved treatments. It is time we looked at how these patients would view the benefit-risk of a drug that would give them back even a small portion of their lives.
Please include ME/CFS for the one million Americans who suffer from this terrible disease. Please do this for all those sons and daughters who are not yet sick so that they never have to experience the nightmare that my son lives every single day.