2004N-0181 - Critical Path Initiative; Establishment of Docket
FDA Comment Number : EC10
Submitter : Mrs. Nancy Norton Date & Time: 08/02/2004 06:08:17
Organization : IFFGD
International Organization
Category :
Issue Areas/Comments
Comment Issues
8. What factors should guide FDA in setting priorities among the hurdles and solutions identified?
Over the past several years, we have seen a rapid growth in scientific understanding of Irritable Bowel Syndrome. Neurotransmitter research, brain imaging studies, consistent diagnostic criteria, and quality of life studies are all contributing to the legitimization of the disorder as a diagnostic entity. We want to see continued growth of support for education and research, and clinical application of new understandings. We are interested in working with other interested parties to help assure access to safe and effective treatment of IBS and other functional gastrointestinal disorders. Industry, physicians, and patients all have a role in drug treatment. The pharmaceutical industry needs to provide the data necessary for accurate analysis of the risks and benefits of the medication they develop; they need to work closely with physicians, pharmacists, and patients to help ensure clear and useful prescribing information. Physicians need to be certain of the diagnosis before prescribing appropriate medication. Patients need to understand the risks and benefits associated with any medication. Without the responsible participation of all three parties, treatments will not be effective and the risk of side effects will increase. Finally, regulatory agencies, like the FDA, have an obligation to ensure that safe and effective medications are made available. We urge the FDA to consider the impact IBS and other functional gastrointestinal and motility disorders have on patients when looking at the risks and benefits of any medication approved to treat them. As adverse events are reported, we urge that mechanisms be in place to ensure that any possible causal relationship between the drug and the event is clearly supported. We urge consideration of how side effects can be managed before they get to a crisis point for patients. Critical review by indepenedent clinicians and investigators experienced in this area of medicine would be essential to rationally sort out questions about safety and efficacy in the context of the risk/benefit ratio for these disease models. We urge that decisions be made for or against the use of any drug in the face of adequate documentation, and after careful scientific and epidemiological investigation that involves experienced consultants. We also urge that any risk management plan not be perceived as a barrier to access but rather as education to access so a patient can make an informed decision.
Irritable Bowel Syndrome or IBS is a common disorder that affects between 10 and 20 percent of the population, disrupting the lives of millions of people. Similar to other common chronic conditions, recurring symptoms have the power to alter people's lives and need to be taken very seriously. IBS is only one of many functional gastrointestinal and motility disorders for which there are no cures, and limited treatment options. The International Foundation for Functional Gastrointestinal Disorders or IFFGD has been working for the past thirteen years to encourage better access to clinical care, better diagnostic and treatment options for patients with these disorders, and better understanding of what it is like for people to live with the symptoms. IFFGD is enthused to see the FDA establishing this Critical Path Initiative to reduce hurdles in product development. We ask you to consider two issues that are key components of determining benefit and risk in disorders such as IBS: (1) What are the consequences of alternative therapies or no treatment for chronic, multiple symptoms; and (2) What is the level of disability, morbidity, and mortality associated with functional gastrointestinal disorders. The following presents some of our views about the impact IBS can have on a person, some of the problems and issues surrounding the disorder, and implications for treatment.
IBS is a chronic disorder characterized by pain, which can be severe, and a combination of chronic or recurrent gastrointestinal symptoms. Chronic illness may involve repeated episodes of deterioration in the person's quality of life, during which the patient confronts and adjusts to the losses imposed. Hope often shifts from the desire for a cure to finding ways to cope with the disease. The impact of IBS symptoms is comparable to other chronic diseases, and ranges from inconvenient to disabling. For the millions of people with moderate to severe IBS who suffer the multiple symptoms of this disease, there has been little that actually provides sustained relief; from pain, from a constant sense of urgency to have a bowel movement, from diarrhea, from constipation - relief that enables one to attempt to live a normal life. The intense bowel urgency can lead to a socially and emotionally devastating aspect of IBS that is rarely talked about-fecal incontinence. The unpredictable onset of pain adn urgency make it difficult for some to leave home for fear of having an attack. IBS presents unique challenges and does not fit the traditional biomedical model. To patients, the symptoms are often unmentionable and embarrasing, and to physicians, in the absence of structural abnormalities, symptoms may be relegated to being "psychiatric," or not truly existing as clinical entities. However, our understanding of IBS pathophysiology and treatment has changed dramatically over the past decade. At IFFGD, we are concerned about the patient with IBS. It is not a disorder that is easily talked about, even with one's own physician. We believe, therefore, that the true impact of the disorder may not be fully recognized by medical professionals and investigators. It is time for change among clinicians, investigators, health agencies, and public information providers. Too often, the only treatments offered are aimed at supressing the predominent symptom presented by a patient with IBS, rather than attempting to better understand and treat the underlying disorder. IBS is characterized by multiple symptoms. It often requires an integrated approach to treatment of the pain and symptoms of diarrhea, constipation, bloating, gas, urgency, and perhaps fecal soiling. All of this has implications for treatment of IBS. The medical community should increasingly look for strategies to help manage the disease, as well as safe, effective medications to treat IBS.