Celiac disease is one of the most common autoimmune conditions in the U.S. but frequently goes undiagnosed. The cause is currently unknown and it can develop at any point in life. People who have a relative with the disease are more likely to develop it, and women are more likely than men to have it.
When people with celiac disease eat food with gluten, their immune system reacts by damaging areas of the lining of their intestines. Symptoms can vary from patient to patient but can include abdominal pain, bloating, gas, indigestion, nausea, vomiting, decreased appetite and unexplained weight loss. This variance in symptoms is one reason celiac disease can be difficult to diagnose.
There is currently no cure for celiac disease but it can be managed with a gluten-free diet. Wheat, barley, and rye contain gluten but other grains (including quinoa, buckwheat, millet and amaranth) do not.
Dr. Peter H.R. Green, Director of The Celiac Disease Center at Columbia University and Professor of Clinical Medicine at the College of Physicians and Surgeons at Columbia University in New York City, spoke with BIOtechNow to raise awareness of what celiac disease is, how it is diagnosed and the treatment options.
In today’s budget-constrained world, the goal for health care is not only to save lives but also to save money. On Friday, May 11, the Food and Drug Administration (FDA) will hold a one-day public hearing on important medicines, known as biosimilars, that are under FDA consideration to come to the United States (U.S.) and offer the opportunity to help achieve the second prong of the health care goal: to save money. However, similar to any new medicine approval, patient safety must be paramount.
Biosimilars are attempted copies of innovative biotech medicines that have been available in the U.S. for a number of years. As members of the biotech community know, biologics have revolutionized the treatment of many serious and life threatening diseases in the short history of this industry. Biosimilars offer the hope of bringing life saving biologic drugs from the bench to the bedside in a cost effective manner and serve as another tool in the toolbox of healthcare providers. Unlike traditional pharmaceutical drugs that are made from chemicals and have structures that are well characterized, most biologics are made from living cells, with highly complex structures that are not easily understood, characterized or replicated. As a result, biosimilar medicines differ from generic drugs in that they are “similar to” but not exact copies of the innovator drug they attempt to replicate.
In 2010, the Patient Protection and Affordable Care Act (PPACA) granted the FDA the authority to approve biosimilars. Later that year the agency began consulting with patient groups, physicians and industry to exchange information that would eventually lead to the approval of biosimilars in the U.S. A few months ago the FDA took the first concrete step to create a pathway and issued draft guidance. The public hearing on Friday will allow stakeholders to provide testimony in response to the draft guidance and I am honored to be able to speak on behalf of the Alliance for Safe Biologic Medicines (ASBM).
In addition to my day job seeing patients as a practicing endocrinologist, I serve as the Chairman of ASBM, an organization that is working to raise awareness of biologics and biosimilars, as well as the important steps that should be taken to ensure safe use of these important medicines. Our organization is composed of diverse healthcare groups—from patients to physicians, medical innovators, and others who have come together to ensure that patient safety is at the forefront of the biosimilars policy discussion. We were pleased but not surprised to see the FDA lay out a science-based approach in the draft guidance largely building on the foundation laid by the European Medicines Agency (EMA).
The EMA began to establish the first formal regulatory pathway for biosimilars in 2003 and has gathered much data over the past 8 years that can, at a minimum help inform the development of policy in the U.S. Policy makers should take advantage of this opportunity to learn from their experiences, both positive and negative.
We must also acknowledge that are differences between the EMA and FDA approach most notably in the area of “interchangeability” where the FDA has the authority to designate a biosimilar as interchangeable with its reference product and the EMA does not. This difference is not insignificant for patients in the U.S. because in theory it could lead to biosimilars being automatically substituted for the originator product by the pharmacist without consulting the prescribing physician. In the draft guidance the FDA signaled that it is not ready to prescribe a path to interchangeability at this time, and instead indicated that additional guidance will be necessary to better understand the challenges involved.
Differences aside, there is much to learn from the EMA experience and we believe that it provides a solid blueprint for the FDA to follow. At a minimum, ASBM members believe that the FDA must require biosimilar manufacturers to conduct clinical studies and produce analytical data sufficient to reassure patients and doctors that their products are safe. We also believe that unique nonproprietary names must be assigned to all biologic therapies so that physicians and patients know what caused an adverse event if one occurs. Finally, as a practicing physician it is imperative that doctors and patients should be able to carefully choose the best course of treatment rather than have legislators and regulators decide for them.
For nearly 18 months, ASBM has been working to educate the public and policymakers about the great promise that biosimilars offer to patients in the U.S. Our message has been relatively simple, making lower cost medicines available to patients should be a priority but their availability must come with the absolute assurance that they are first and foremost safe. Our organization and its members have written Op-Eds, conducted webinars and hosted policy forums to exchange information about the issues and challenges associated with biosimilars. I am proud of the work we have done but ultimately realize as the late great Karen Carpenter sang so many years ago “we’ve only just begun.”
Ten years ago, Greg and wife Leigh brought their new daughter, Mallie, home from the hospital.
Mallie
When she was 9 days old, Mallie was fussy, leading Greg and Leigh to discover that their baby girl had fractured her right tibia and fibula. The new parents immediately took their daughter to the doctor, who advised them to take Mallie straight to the emergency room. They discovered that Mallie was born with osteogenesis imperfecta, a genetic bone disorder characterized by fragile bones that break easily.
“We were just absolutely frightened,” Leigh remembered. “I am a nurse, and I don’t remember hearing any of this from nursing school.”
Osteogenesis imperfecta (OI) is a rare heterogeneous disease of connective tissue that affects 8/100,000, or about 30,000 persons in the United States. Although the cardinal manifestation of OI is bone fragility, this rare disease encompasses all body systems. A person is born with this disorder and affected throughout his or her lifetime. Thus, an infant may be born with multiple fractures, or a child can break his arm and collarbone while putting on a T-shirt. Each fracture is as painful as the first!
Many children and adults with OI have multiple surgeries to try to correct bone deformities and straighten bones so they can live independently. Mallie had her first surgery when she was just 2 ½ years old! Rodding surgery is considered major surgery, and the Holmans knew that it would require their little girl to deal with an overnight stay at the hospital, a hip-spica cast for six weeks, and then months of physical therapy. This surgery was necessary to relieve the bowing in her legs and thus, to prevent osteogenesis imperfecta from stealing Mallie’s ability to walk. To make matters worse, the Holmans learned that Mallie would likely need to repeat the surgery every two to three years, depending on how fast she grows, to replace the rods with longer ones.
Mallie, who turned ten this year, is an excellent student, and she is active in a variety of activities, including swim team, golf, band and orchestra. Although she tries not to allow OI to slow her down so she can still just be a kid, Mallie recently fractured her forearm and reinjured her tibia in her lower leg. Her arm was so severely injured that she needed surgery to place two rods in her bones. Even though this was her fifth surgery in eight years, it was still an extremely frightening and traumatic experience for her. After a brief overnight stay in the hospital, Mallie was home and showing off her unbreakable spirit.
The Role of the OI Foundation
The Osteogenesis Imperfecta (OI) Foundation provides information and resources to parents, doctors, educators, and adults with OI; organizes the National Conference on OI for families to gain the medical, research, and coping information they need (to be held in July this year); and advocates for increased funding from the National Institutes of Health, foundations and corporations.
The Foundation also funds groundbreaking research, including the multi-site Longitudinal Study of OI, which is tracking the health of 500 children and adults with OI for 5 years. The goal of the study is to develop a more detailed picture of osteogenesis imperfecta across a lifespan and to improve the level of care.
Build Awareness and Improve Lives!
The OI Foundation is hosting its second annual National OI Awareness Week from May 5-12, 2012. Awareness Week is a week-long event designed to educate communities across the country about osteogenesis imperfecta. National OI Awareness Week is held in conjunction with Wishbone Day on May 6, an international community awareness effort.
Hundreds of volunteers have planned fundraising events, secured proclamations from their city and state officials proclaiming May 5-12 ‘OI Awareness Week’, and are distributing educational materials throughout their communities.
Excitement for this year’s Awareness Week is growing—just last month, the City of Rochester, NY proclaimed May 5-12, 2012 ‘Osteogenesis Imperfecta Awareness Week’!
Awareness efforts like National OI Awareness Week help educate the greater community, leading to additional funding towards research programs and vital information and resources for our caregivers, doctors, nurses, teachers, and the OI community.
For more information or to donate, please visit our website.
Together, we are creating a better future for children and adults living with OI!
Routine immunization of one birth cohort (i.e. people born in a particular year) during childhood prevents about 20 million cases of disease and 42,000 deaths in the U.S. These statistics are staggering, especially when we consider how far vaccinology and preventive medicine have come in such a short time period. Today, vaccines help protect children against 14 diseases before the age of two. Many diseases that parents once feared have been long forgotten. In the 1950s, nearly every child in the U.S. contracted measles. In 2011, 222 measles cases were reported in the U.S according to a report published last week by the Centers for Disease Control and Prevention (CDC), and this was the highest number of reported measles cases in the U.S. in 15 years.
New vaccines continue to be developed by private companies, both large and small, and by non-profit product development organizations. In 2006, vaccines against rotavirus, a leading cause of severe diarrhea in infants and young children in the U.S., were introduced. A study published by the CDC in the New England Journal of Medicine last September estimated that rotavirus vaccination prevented approximately 65,000 hospitalizations of children under age 5 from 2007 to 2009 and resulted in $278 million in healthcare cost savings. Innovative vaccines against diseases such as tuberculosis, meningococcal B disease, and respiratory syncytial virus (RSV) are currently in development and could significantly improve the health of children if added to the CDC’s childhood immunization schedule.
With these statistics, it may seem hard to deny the benefits of immunization. Yet, vaccines are sometimes the victims of their own success. They are so effective that parents are unable to remember the illness and associated disability and death that a vaccine prevents. To remind the American public about the positive impact of vaccination on the lives of infants and children, National Infant Immunization Week was launched in 1994. To learn more about this annual initiative led by the CDC, Dan McGirt, Director of Communications at BIO, spoke with Dr. Yabo Beysolow, a Medical Officer at CDC as well as a pediatrician and mother of three children. The podcast is available here.
In recognition of National Infant Immunization Week, Dr. Yabo Beysolow, Medical Officer at the CDC, talks about the benefits of immunization for children under the age of two and the achievements of immunization programs in promoting healthy communities throughout the U.S.
