Cystic Fibrosis Foundation Patient Registry
The Cystic Fibrosis Foundation Patient Registry (CFFPR) tracks the health of people with cystic fibrosis (CF), a lifelong
genetic disease that causes mucus to build up and clog some of the body’s organs, in the United States. It gives
clinicians and researchers access to a large sample of data that can be used to identify and study health trends, learn
about effective treatments, and design clinical trials for potential new therapies. Operated and managed by the Cystic
Fibrosis Foundation (CF Foundation), the Registry collects data for appropriately 28,000 patients annually.1
The CFFPR is widely recognized as being the nation’s only comprehensive source of validated data for CF. Used by
patients, care providers and researchers, the Registry helps to:
Identify areas for care improvement;
Conduct research studies on all or a subset of CF patients; and
Customize treatment plans for families and patients.
Data Collection
The CFFPR collects patient-level data, including vital statistics
(e.g., height, weight), diagnostic tests (e.g., lung function, results
of microbial cultures), and history of medication and hospitalization use.
Data are collected through a secure Web-based portal.2 The CF
Foundation partners with a third party vendor to maintain the
electronic data capture platform and perform data auditing and
quality checks. Data are entered into the portal by trained staff at
each Care Center – medical centers that are certified by the CF
Foundation as administering and providing care aligned with national standards. The CF Foundation provides data entry personnel with user manuals and data entry guidelines.3
Key Features
• The CFFPR is recognized as a rich resource of historical
information for nearly all CF patients in the United States.
This archive enables the conduct of longitudinal studies,
which are difficult to undertake with other diseases not as
well documented.4
• The infrastructure (e.g., Web portal) and data capture process of the CFFPR is viewed as an exemplar model; other rare
disease organizations look to the CF Foundation for guidance in developing their individual registries. Specifically, the
Pulmonary Fibrosis Foundation, the Pulmonary Hypertension
Association and the Crohn’s and Colitis Foundation of America
have sought advice from the CF Foundation for the development of their own registries.5
Important Studies
The CFFPR is often credited with having a profound effect on the
administration of patient care. Significant highlights include:
1. Proving that early diagnosis improves health outcomes: a study
aimed at determining the impact of age and condition at the
time of CF diagnosis pulled Registry data on over 27,000 patients between 1986 and 2000. Until this study, better survival
rates as a result of early, presymptomatic identification for
patients were only suggested, not proven by medical providers. The results of the study showed that early screening (e.g.,
prenatally, one-month) advanced survival rates. Furthermore,
early screening in patients with genetic abnormalities (e.g., the
CF gene mutation), but without symptoms, proved beneficial.6
2 | Cystic Fibrosis Foundation Patient Registry
2. Proving the positive relationship between nutritional status and
pulmonary function in children: a study using data from 1991
to 1995 showed that nutritional interventions may slow the
decline of pulmonary function in children with CF. This study
used a large sample from multiple Care Centers across the
United States to document the relationship between growth,
nutritional status, and pulmonary function in children in
the early stages of CF. Findings confirmed the importance of
nutrition for disease treatment – children with high nutritional standards (i.e., those whose diets were highly nutritious)
required less aggressive CF treatment relative to those without
the same nutritional standard. Before this study, all studies
analyzing nutrition in CF children had small sample sizes. The
Registry provided researchers with a large sample of nearly
1,000 patients, thus providing sufficient power to achieve
statistical significance in the results.7
On The Horizon
Despite high rates of adherence to national standards of care by
Care Centers, variations in care quality across Centers persist.
Systematic, near-term improvements and new opportunities for
Registry expansion are under consideration by the CF Foundation
to help reduce unwanted variation among Centers, and achieve
better patient outcomes overall. They are:
– Using Registry data for comparative effectiveness research
(CER) to identify optimal medication regimens;
– Comparing CF in other countries;
– Minimizing the burden of data entry; and
– Linking and leveraging additional sources of data.8
Citations
1
Key informant interview, Summer 2014.
2
American Heart Association, Frequently Asked Questions for Get
With the Guidelines-Stroke and ASTP, available at: http://www.
strokeassociation.org/STROKEORG/Professionals/FrequentlyAsked-Questions-for-Get-With-The-Guidelines-Stroke-andASTP_UCM_315318_Article.jsp. Accessed August 1, 2014.
Key Informant Interview, Summer 2014.
3
Ibid.
4
Ibid.
5
Lai, H. et al., “The Survival Advantage of Patients with Cystic
Fibrosis Diagnosed Through Neonatal Screening: Evidence from
the United States Cystic Fibrosis Foundation Registry Data,”
Journal Pediatrics, Vol. 147: 3 Supplement: S57-63, September
2005.
6
Zemel, B.S., et al., “Longitudinal relationship among growth,
nutritional status, and pulmonary function in children with cystic fibrosis: analysis of the Cystic Fibrosis Foundation National
CF Patient Registry,” Journal of Pediatrics, Vol. 137 (3): 374 – 80,
September 2000.
7
Ibid.
8
About the Authors
Jessica Winkler, M.P.H. is a senior associate at AcademyHealth.
She can be reached at Jessica.Winkler@academyhealth.org.
Acknowledgements
Sponsorship for this project was provided by the Kaiser Permanente Institute for Health Policy. A special thank you to our partners at The Pew Charitable Trusts and all those interviewed for
their contributions to this product. A special thank you to Bruce
Marshall, Erin Moore, Aliza Fink, Craig Lapin, Michael Schechter,
Kathryn Sabadosa and other registry staff for their review and
assistance.
This document represents a synthesis of information generated by a series of key informant interviews. Any views expressed are those of the interviewees.