Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
The primary responsibility of the independent Data Safety Monitoring Board is to protect the safety and welfare of people who participate in clinical trials approved by the Therapeutics Development Network.
The highest priority of every clinical trial is to protect the health and safety of the people who volunteer to participate. The U.S. government has strict guidelines and safeguards in place to keep risks as low as possible and ensure that any risks are worth the potential benefits. All trials must also be approved by the research institution's own Institutional Review Board.
The Cystic Fibrosis Foundation provides an additional layer of protection for every CF clinical trial by funding a Data Safety Monitoring Board (DSMB), an independent committee of experts in CF clinical care, clinical and basic science research, bioethics, and biostatistics.
The DSMB is charged with protecting the safety and welfare of people participating in clinical trials sanctioned by the Therapeutics Development Network (TDN) and ensuring the scientific integrity of those trials.
Many of the risks for people with CF who participate in clinical trials are inherent to the disease, and thus are shared across different clinical trials. Maintaining a core of expertise specifically related to CF makes the DSMB very effective at monitoring CF clinical trials and protecting CF research participants as new drugs are developed.
Although both the TDN and DSMB are sponsored by the CF Foundation, the two entities are organizationally separate from each other and the Foundation.
The CF Foundation is the only voluntary health organization to organize a DSMB whose members are experts in CF and are not involved in any way with the trial, the sponsor, or its participants.
A Data Monitoring Committee (DMC) is a subcommittee of the DSMB that is formed for a particular clinical trial. The specific role of the DMC is delineated in a charter developed with the sponsor and principal investigator.
The DMC for a clinical trial reviews the protocol and proposed safety monitoring plan before the trial starts. During the trial, the DMC is responsible for real-time monitoring for serious adverse events and reviewing interim analyses of safety and outcomes data to ensure the continued safety, scientific validity, and merit of the clinical trial.
DMCs vary in size and nature depending upon the study, but usually include three to five CF clinicians, a biostatistician, plus ad hoc members with specific expertise as required. All DMC members sign a confidentiality agreement and are aware of the critical importance of maintaining any and all information about the given study in the strictest confidence.
A DMC charter is developed by the study sponsor to clearly define the function, composition, and operating expectations of the study's DMC. To request guidelines and a standard template for DMC charter development, contact the DSMB Program Coordinator, Miriam Hunt, at email@example.com. Statistical consultation on the charter is available from the TDN Coordinating Center.
To assess whether the study is designed to adequately protect the safety of participants and data quality, the DMC reviews the protocol, draft DMC charter, investigator brochure, and any other relevant information on the study drug prior to study initiation. To help avoid redundancy or any discrepancies, the critique and ratings from the TDN protocol review process are also provided to the DMC.
During the study, the DMC is responsible for evaluating significant adverse events in real time and conducting interim reviews of outcome and safety data to make recommendations on continuing, terminating, or modifying the trial. According to the DMC charter, major study design modifications and/or protocol amendments should be reviewed and approved by the DMC prior to implementation.
For more detailed information about working with the DSMB, including a sponsor checklist, see the TDN Protocol Review and Data Safety Monitoring Processes brochure and flow chart.
Study sponsors should contact the DSMB at least six weeks in advance of submitting a protocol to the TDN.
Any questions or comments about the DSMB should be directed to Miriam Hunt, senior DSMB program coordinator, at 520-626-8800 or firstname.lastname@example.org.
Follow Us On
With more than 70 chapters and offices across the country, it’s easy to find and join a local Cystic Fibrosis Foundation chapter near you.
Cystic Fibrosis Foundation
4550 Montgomery Ave.
Suite 1100 N
Bethesda, MD 20814
800-344-4823 (toll free)
Sign up for our emails