“Good Morning members of the IOM Committee on the Epilepsies and to my friends and colleagues here in the battle against epilepsy. Thank you all for the good work you are doing and for the opportunity to share my perspective with you this morning.
For those of you who do not know me, I am the father of Sylvie, a young girl with epilepsy, whose seizures have for 13 years been unresponsive to all available therapies.
Sylvie has been the inspiration for my work over the years on the Board of EF and as a Co-Founder with Dr. Orrin Devinsky of the Epilepsy Therapy Project.
The Epilepsy Therapy Project is a non-profit organization that works to accelerate the development of new therapies in a time frame that matters to people living with epilepsy today. Our website, epilepsy.com, offers in-depth information on available and emerging therapies and community to over 400k monthly unique users.
More than a decade ago now I was traveling on business in California when I received a call that my until then healthy 9-month-old daughter had just had a seizure in our back yard and was on her way to Denver Children’s Hospital. I raced to the airport and took the next flight home, arriving to find Sylvie in status with the hospital staff frantic, trying to find an IV medicine to break the seizure.
I am happy to say that a little more than 13 years later, this last Wednesday morning, Sylvie graduated from middle school in Lincoln with wonderful support from her teachers, aids and classmates. But I am not happy to say that she graduated alone in a special ceremony attached to her Class Awards presentations because the daily waves of myoclonic and absence seizures that fill her evenings prevented her from joining her class at the graduation ceremony scheduled for that evening.
Sylvie has had and continues to have the good fortune to have access to compassionate and thoughtful care from dedicated epilepsy specialists. She has tried every epilepsy medication plausibly relevant to her condition, has an implanted medical device (a VNS) and now takes three medications, four times a day. She still lives with uncontrolled and often wrenching seizures.
Sylvie is not alone.
Wolfgang Loscher’s recent article in Epilepsia suggests that the introduction of Bromides as a therapy in epilepsy in the 1880’s brought seizure control, albeit with side-effects, to some 2/3 of those with epilepsy. 1/3 continued to live with active or uncontrolled epilepsy and seizures. Unfortunately and unacceptably, it is still true today that approximately 1/3 continue to live with active epilepsy over a century later. Many others (perhaps another one third of those with epilepsy) face unacceptable side-effects including severe fatigue, osteoporosis, risks of liver failure, special risks in pregnancy and drug, drug interactions among others.
For patients like Sylvie who take multiple medications and have ongoing seizures, the seizures are not only debilitating, but too often fatal. One aspect of epilepsy that is still too little discussed is Sudden Unexplained Death in Epilepsy (SUDEP). We need better data but SUDEP is estimated to account for 18% of all deaths among all those with epilepsy. And for those with refractory epilepsy, the risk is notably higher. A study of patients evaluated as candidates for epilepsy surgery found an almost 10% rate of mortality over a decade (Dasheiff 1991). So I just want to highlight that while we focus today on critically important issues around systems and pathways of care, we have a fundamental unsolved problem in the need for effective and safe therapies in epilepsy.
We have seen so much progress in cancer, in aids, in heart disease, in so many areas of medicine.
And, yes, we have made progress in epilepsy with new medications with improved side-effect profiles that have brought seizure freedom to some and fewer seizures to others. New medical devices are beginning to offer better seizure control to individuals with severe epilepsy. We have seen advances in epilepsy surgery and in the application of dietary therapy to epilepsy.
But in epilepsy, stepping back to look at the big picture, approximately 1/3 live as does my daughter Sylvie today with active seizures despite all available therapies. And alarmingly, you could tell the same story 13 years ago when Sylvie had her first seizure, 30 years ago, 50 years ago and maybe since the last decades of the 19th century.
That is not acceptable and certainly not for a disease affecting as many as 3 million people in the U.S. and 50 million worldwide, a disease that especially impacts the lives of children and the elderly, and which presents special problems for newborns, for women and today for too many veterans returning home from undeclared overseas wars with Traumatic Brain Injury and a life that will include seizures and epilepsy.
Epilepsy is for many a devastating and too often fatal disorder. This message is not out there.
In an understandable effort to overcome stigma, too often patients, professionals, even epilepsy advocacy groups have failed to give voice to the terrible and unsolved nature of epilepsy. And in an effort to protect patients and families from a painful truth without easy answers, too often caregivers have avoided discussions of SUDEP. But not being told means patients and families are also denied the opportunity to step up and become advocates for change, for answers and for new therapies.
Epilepsy and SUDEP too often remain well-kept secrets. And this means that the broader public and the communities we live in are to a great degree unaware of the extraordinary challenges and pain, of the enormous unmet need faced daily by individuals and families with epilepsy.
Patients and families must be our own best advocates. Only awareness and positive action can eliminate stigma, build resources and spur progress.
We need not only to be empowered to seek out the best available therapies today but also to provide and mobilize support for the development of new therapies to control seizures and epilepsy, to solve side-effects and ultimately to end SUDEP. And this in short is the mission of the Epilepsy Therapy Project and epilepsy.com.
As I conclude, I want today to call out one issue that looms large as an obstacle to the development of new therapies.
It has become increasingly clear that for epilepsy, a primary impediment to new therapies and therefore new investments lies at the end of the development process and not the beginning.
Congressional and public attention has focused overwhelmingly on the side-effects of new therapies. For FDA, pitfalls and poor outcomes from approvals become scandals while little or no reward is given for approvals that go right and save or improve lives. With little attention to costs or timelines, the FDA regulatory process has steadily become longer, more expensive, and unbalanced in the weighting of risk and benefit in many disease areas including epilepsy.
Safety matters and we all want new therapies with fewer or no side effects. But epilepsy is a devastating disease. Its contours include unemployment and underemployment, progressive memory and cognitive decline, high rates of depression, anxiety, and psychosis, accidents and accidental death, suicide, status and SUDEP. And side-effects, risk and time to approval all have to be viewed against this canvas.
Regulatory overreach and paralysis takes nuanced decision making and judgment about risk and benefit (as well as autonomy) away from patients, families and their nurses and physicians; it keeps valuable new therapies out of the hands of patients and discourages new investment in innovation. It takes lives from those we love.
In epilepsy, we are fortunate to have thoughtful individuals at FDA who have engaged with the community to improve on our now rigid and often unreasonable regulatory trial pathway. At the same time, promising and efficacious new medical devices and medicines are already available to patients and families in Europe that are still today being held hostage at our FDA. Other new therapies seem frozen in a byzantine process in which time has no consequence to patient lives. That is not right and it is not acceptable.
I want to thank the Institute of Medicine, the panel and this audience for putting a spotlight on this unsolved problem of epilepsy and seizures.
For Sylvie, for the millions of people living with uncontrolled epilepsy or unacceptable side-effects in this country and around the world, for families who have had dear ones suffer with epilepsy or lost them to epilepsy and SUDEP, the time to take positive steps to make new therapies a reality is NOW!”
Chairman and Co-Founder
Epilepsy Therapy Project
Welcome to the Wiki. This space is created for epilepsy.com members to share their own experiences and expertise to help refine and expand the discussion around important topics.
No members have yet contributed to this topic. If you are not yet an epilepsy.com member, register today to get started on this Wiki topic and the many other advantages of being a member. If you are a member and wish to be the first to edit this Wiki topic, please make sure to login, then click on the orange "Start Wiki" button at the top of this page. Or, learn more about Wikis.