October 25, 2005
Presentation of Suzanne Vogel Scibilia, M.D.
President, National Alliance on Mental Illness (NAMI)
FDA Psychopharmacologic Drug Advisory Committee
My name is Dr. Suzanne Vogel Scibilia and I am President of the National Alliance on Mental Illness (NAMI). I speak to you today from a number of perspectives – as NAMI’s President, as a practicing Psychiatrist, as a person living with bipolar disorder and as the parent of a child and a daughter of a father who suffers from a severe mental illness. Throughout its 25 year history, NAMI has been a staunch advocate for increased and improved research because our members understand that research is the best hope we have for finding treatments to alleviate the devastating and debilitating symptoms of brain disorders such as schizophrenia, bipolar disorder and major depression. When NAMI was founded, there was little thought that a cure for schizophrenia might one day be discovered. Today, many of our members view this as a distinct possibility, encouraged by the progress that has been made in discovering new treatments and the emergence of evidence based services helping many people achieve levels of recovery and independence that 25 years ago did not seem possible.
At the same time, we know that progress has been very slow in discovering effective treatments – and the road ahead is still very long. The landmark CATIE study recently released by NIMH provides an illustration of how far we still have to go. This study, above all else, shows that none of the existing medications for the treatment of schizophrenia – first generation or second generation – are a panacea . While people derive therapeutic benefits from medications, they appear to be limited, and the side effects of these medications are, for some people quite profound. Clearly, the discovery of new and more effective pharmacologic treatments for schizophrenia is desperately needed. This is true as well for bipolar disorder, major depression and other serious mental illnesses.
NAMI therefore strongly believes that a research environment that encourages discovery and innovation must be fostered and maintained. Recently, legitimate concerns have been raised that consumers do not have full access to information about the medications they are taking, particularly negative information about risks associated with these medications. NAMI firmly supports the need for greater transparency in research. Consumers and their families must have complete access to all information, positive and negative, about research on psychiatric medications. Without this information, informed decisions cannot be made about what medications to take and the concept of informed consent becomes meaningless.
At the same time, in my experience, and that of our members, far more people have been helped than hurt by the psychiatric medications that are available today. Lack of treatment frequently leads to tragic circumstances, including homelessness, involvement with the criminal justice system, and suicides. We must maintain an environment that is conducive to innovation and discovery, one that will allow potential breakthrough medications to enter the marketplace in a timely manner. Psychiatry is one area of medicine that has not had a large number of new, novel medication classes developed in recent years. Innovative research has vastly improved treatment of heart disease compared to psychiatric illnesses.
With this as a backdrop, I will briefly make three points about the specific topic under consideration at this meeting – determining specific standards for studying the long-term efficacy of psychiatric medications. Although I realize that the first eight questions in Dr. Laughren’s memorandum specifically address major depressive disorder and only the final two questions address psychiatric disorders generally, my comments are focused on psychiatric medications broadly.
First, NAMI does not at this time support requiring the accumulation of data on long-term efficacy trials prior to FDA approval of new psychiatric medications. The World Health Organization (WHO) has documented that five out of the leading ten causes of disability world wide are caused by mental illnesses. And, access to evidence based treatments, coupled with intensive community supports, remains extremely low. For example, a number of studies have documented that fewer than half of all people with schizophrenia have access to even minimally adequate treatment. Access limitations are equally profound for people with bipolar disorder, major depression, obsessive-compulsive disorder, anxiety disorders and other major mental illnesses.
The FDA’s process for approving new medications is already quite slow. Many years may pass from the time research on a potential breakthrough medication commences to the time it is approved. NAMI is concerned that requiring long-term efficacy studies prior to approval would have the effect of further slowing an already overly cumbersome process. This could prove most harmful to the people intended to benefit from these treatments. Ten percent of all people who have schizophrenia commit suicide and only one antipsychotic medication (clozapine) has been shown to reduce that risk to date. The rapid development and approval of new medications for the treatment of schizophrenia is therefore of life and death importance.
Second, NAMI does support requiring long-term efficacy studies after psychiatric medications are approved. Serious mental illnesses are chronic in nature – symptoms may be stabilized but subsequently recur. Thus, many people, after finding medications that work in reducing their most debilitating symptoms, remain on long-term maintenance doses of these medications. Understanding the long-term effects of these medications (both positive and negative) is critically important, both to maximize recovery and to minimize risks. Often, risks are not discernible until after individuals have been treated with specific medications for extended periods of time. For example, the development of tardive dyskinesia (TD) occurs over time and varies from individual to individual – for some individuals, TD may develop within months of commencing treatment – for others, it may not occur for years.
Additionally, NAMI believes that long-term efficacy studies should be conducted whenever possible with the financial support of the National Institute of Mental Health (NIMH). Although the results in Phase I of the CATIE study were not as broadly useful as we had hoped, we regard this study as the most important research that has ever been conducted on medications used in the treatment of schizophrenia. And, we eagerly anticipate the findings that will be derived from similar NIMH supported long-term efficacy and safety studies conducted on medications for bipolar disorder (Step-BP) and major depression (Star-D). We believe that the NIMH should continue to target resources for these important purposes.
Finally, NAMI asserts that the use of double-blind, drug withdrawal designs, to conduct long-term studies of psychiatric medications puts vulnerable individuals at significant risk and should not be used.
Simply stated, if a person is responding well to a specific psychiatric medication and wishes to remain on that medication, he or she should not be taken off that medication. This particularly should be the case in studies evaluating the long-term efficacy of antipsychotic medications. In fact, we have serious questions whether an IRB will or ought to approve studies on antipsychotic medications with designs that involve drug withdrawals or the use of placebo.
An appropriate and ethical way to conduct long-term studies on antipsychotic medications is to evaluate these medications against others that have already been demonstrated to be therapeutic. The CATIE study referenced above is an example of such a study. CATIE was designed as a naturalistic study, in which groups of individuals prescribed a number of different antipsychotic medications were followed over an 18 month period to evaluate both the efficacy and safety of these medications.
It is also important to note that drug withdrawal or placebo arm designs are not used to study the long-term effects of medications for other life-threatening diseases, such as cancer. Discontinuing a therapy that is working is viewed as unethical in the field of oncology. Instead, long-term drug studies in oncology are conducted using an active treatment comparator. We believe that the same standards should be applied to research on medications for the treatment of serious mental illnesses.
Clearly, as a primary consumer and family organization, NAMI’s major concern is the safety and well-being of those living with mental illnesses. The FDA is faced with decisions on many difficult issues. In considering these issues, we ask the FDA to responsibly and carefully weight risks and benefits, including public health consequences, and not to succumb to political pressures imposed by those who oppose psychiatric medications. An action by the FDA that is designed to address concerns voiced by a small but vocal segment of the consumer and family member population may have the unintended effect of stifling innovation or delivery of care to the majority of those who suffer. The National Alliance on Mental Illness is the nation's largest consumer and family organization with over 200,000 members. We urge you to give this testimony the weight it deserves.
Thank you for this opportunity to speak to you today. I look forward to any questions you may have.