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Testimony to FDA October 25, 2012

IFFGD President and Co-Founder, Nancy J. Norton submitted the following written statement on October 25, 2012 to the Food and Drug Administration Division of Dockets Management in response to docket number: FDA-2012-N-0967, the Prescription Drug User Fee Act Patient-Focused Drug Development Meeting:

Thank you for the opportunity to present the views of the International Foundation for Functional Gastrointestinal Disorders (IFFGD) regarding the importance of functional gastrointestinal and motility disorders (FGIMD) research.

Established in 1991, IFFGD is a patient-driven nonprofit organization dedicated to assisting individuals affected by FGMIDs, and providing education and support for patients, healthcare providers, and the public. In order to provide patients with better treatment options and to eventually find cures, IFFGD also works to advance critical research on FGIMDs. IFFGD has worked closely with NIH on many priorities, including the NIH State-of-the-Science Conference on the Prevention of Fecal and Urinary Incontinence in Adults through the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Institute of Child Health and Human Development (NICHD), and the Office of Medical Applications of Research (OMAR). I have served on the National Commission on Digestive Diseases (NCDD), which released a long-range road map for digestive disease research in 2009, entitled Opportunities and Challenges in Digestive Diseases Research: Recommendations of the National Commission on Digestive Diseases.

Irritable Bowel Syndrome (IBS)
IBS, one of the most common functional gastrointestinal disorders, strikes all demographic groups. It affects 30 to 45 million Americans; conservatively, at least 1 out of every 10 people. About 2 in 3 IBS patients are female. About 1 in 3 IBS patients are male. IBS affects people of all ages, even children. Between 9 to 23% of the worldwide population suffers from IBS, resulting in significant human suffering and disability. IBS as a chronic disease is characterized by a group of symptoms that may vary from person to person, but typically include abdominal pain and discomfort associated with a change in bowel pattern, such as diarrhea and/or constipation. As a “functional disorder,” IBS affects the way the muscles and nerves work, but the bowel does not appear to be damaged on medical tests. Without a definitive diagnostic test, many cases of IBS go undiagnosed for years. During this time, patients risk misdiagnosis, exposure to multiple physician visits, multiple medications, and unnecessary diagnostic tests and procedure including surgeries. Even after IBS is identified, treatment options are sorely lacking and vary widely from patient to patient. What is known is that IBS requires a multidisciplinary approach to research and treatment.

IBS can be emotionally and physically debilitating. Due to persistent pain and bowel unpredictability, individuals who suffer from this disorder may distance themselves from social interactions and work, and may even fear leaving their home. Stigma surrounding bowel habits may act as barrier to treatment, as patients are not comfortable discussing their symptoms with doctors.

The Patient Perspective
IFFGD has focused on incorporating the IBS patient perspective in research for over a decade. In 2002 we conducted a survey and published the “IBS in the Real World Survey.” In 2009 we published “IBS Patients: Their Illness Experience and Unmet Needs,” an international survey (72% U.S.) we conducted in collaboration with the UNC Center for Functional GI and Motility Disorders. The 2009 study was a web-based survey used to characterize the following factors among patients with IBS: 1) What are the demographic, clinical, and health status profiles of these patients?, 2) How severe do they perceive their IBS compared to standardized measures?, 3) What factors contribute to severity and can that information help identify endpoints in clinical trials?, 4) What types of medications and other treatments are being taken and what are their side effects? 5) How much risk would patients take to achieve clinical improvement?, and 6) How satisfied are patients with their care? This lead to a peer-reviewed article in the “Journal of Clinical Gastroenterology” entitled International Survey of Patients with IBS: Symptom features and their severity, health status, treatments, and risk taking to achieve clinical benefit (Article attached). The study of 1,966 patients confirmed the severity and quality of life issues faced by IBS patients. Additionally, the survey painted a picture of bleak expectations on symptom improvement, satisfaction with treatment, and evaluating risk associated with medication.

IFFGD strongly urges FDA to carefully consider recognizing “irritable bowel syndrome” as one of the twenty disease areas selected for specific review through this new initiative for the following reasons:

– IBS dramatically affects functioning and the activities of everyday life. In the International Survey of Patients with IBS, the 1,966 respondents reported impaired health status restricting on average 73 days of activity in a year. The impact of IBS can range from mild inconvenience to severe debilitation. It can control many aspects of a person’s emotional, social and professional life. Persons with moderate to severe IBS must struggle with symptoms that often impair their physical, emotional, economic, educational, and social well-being.

– The disease reflects a wide range of symptoms with varying severity. Severity is typically influenced by multiple factors. In theInternational Survey of Patients with IBS, pain, bowel difficulties, bloating and eating/dietary restrictions were reported in some combination by over 90% of respondents, and 35% endorsed all four.Other factors endorsed by over 25% of respondents included social limitations, inability to leave home, work or school limitations, limitations in thinking, trouble sleeping, nausea, limitations in home activities, poor quality of life, and incontinence.

– Data from the International Survey of Patients with IBS indicate that IBS is a complex syndrome defined by a combination of symptoms that contribute to severity. Clinical endpoints for IBS would reasonably encompass a reduction in the number and intensity of these symptoms, primarily pain, bowel difficulties, bloating, and eating or dietary limitations.

– IBS has no cure and there is no specific treatment to encompass the breadth of the symptom experience. There are 3 drugs currently FDA approved for treatment of IBS subgroups; 1 for IBS with diarrhea and 2 for IBS with constipation. Each is effective in only a portion of patients for whom they are indicated. Multiple other drugs and therapies are tried to relieve single symptoms; none are universally effective. In the International Survey of Patients with IBS, participants trying to control their IBS symptoms reported most often taking non-narcotic pain medications, antidepressants for pain, acid reducers, antidiarrheals, and narcotic medications. In addition, complementary and alternative medicine is being used by 37% of these respondents. Only 8% are very or extremely satisfied with all types of IBS treatment.

In recent years, the Food and Drug Administration (FDA) has tried to develop an acceptable endpoint to determine efficacy for investigational drugs in IBS, and sought new treatments for IBS that have very low risk-benefit ratios primarily for safety concerns. Though treatments are partly based on severity, it is necessary to consider the patient’s self-perceptions of severity, and their judgments relating to choosing medications based on the risks they would be willing to take to achieve symptom benefit.

In closing, IFFGD once again asks FDA to please select “IBS” for the new patient-focused drug development initiative. Furthermore, we believe current risk-benefit profiles for those living with IBS are inadequate. Startling research shows patients are willing to incur a much higher risk to achieve benefit than has ever been previously assumed by FDA. In fact, when respondents in the International Survey of Patients with IBS were asked how much risk they would assume to take a medication providing total relief from IBS symptoms but with serious adverse effects, 8% said they would accept a 1 in 100 chance of death to be symptom-free. Six percent (6%) would accept a 1 in 100 chance of serious and disabling side effects to be symptom-free. IFFGD welcomes the opportunity to work in conjunction with the FDA to obtain input from patients on the drug development process.

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