Cystic Fibrosis Foundation Patient Registry (CFFPR)
What is it? The CFFPR is a database established to track the health and treatments of people with cystic fibrosis
(CF). It collects data for appropriately 28,000 patients annually.
How long has it been in existence? Created in 1966, the CFFPR has evolved over the years to continuously
meet the changing evidence needs of patients, providers, and other decision makers. Using a standard set of data
elements, the database contains detailed demographic and diagnostic data dating back to 1986.
Where is its central home? The database is based in the United States.
Who oversees the Registry? It is operated and managed by the Cystic Fibrosis Foundation.
How does it work? Data are entered by staff at accredited CF Care Centers through a secure Web-based portal.
Data quality checks are administered periodically.
What are its accomplishments? The CFFPR is widely recognized for being the nation’s only comprehensive
source of validated data for CF. Used by patients, care providers, and researchers, the Registry helps: identify areas
for care improvement; conduct research studies on all or a subset of CF patients; and customize treatment plans
for families and patients.
CFFPR
The CFFPR tracks the health of people with CF in the United
States. The Registry 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.
What is Cystic Fibrosis?
Cystic fibrosis is a lifelong genetic disease that causes mucus to
build up and clog some of the body’s organs. The malfunctioning
of the CF transmembrane conductance regulator, or CFTR gene,
which is responsible for regulating salt and water movement
between cells, causes the disease. A gene defect causes mucus
produced by the body to become thicker and stickier than that of
a non-CF patient. This buildup of mucus causes long-term lung
and digestive track problems.
The CFFPR is used to improve health outcomes for CF patients in
the United States by:
• Generating knowledge through research using Registry data;
• Identifying areas for quality improvement;
• Improving the development of measures, guidelines, and implementation strategies to reduce disparities; and
• Determining the safety and effectiveness of treatments applied
in practice.
History
The Cystic Fibrosis Foundation (CF Foundation) launched in
1955 with a mission to support the development of new drugs to
improve the quality of life for CF patients, and ultimately find a
cure for the disease. Recognizing the critical role of data collection and measurement in understanding the disease, the Foundation created a Registry in 1966. Around this time, there was
controversy over optimal treatments for the disease, and researchers and providers noted a need for thorough information on treatments and patient outcomes. Since its inception, the Registry has
evolved from its original goal of better characterizing CF patients,
to helping support quality improvement and research activities.1
Care Centers
Data included in the CFFPR are populated through Care Centers – medical centers that are certified by the CF Foundation as
administering and providing care aligned with national standards.
2 | Cystic Fibrosis Foundation Patient Registry (CFFPR)
Currently, there are more than 110 centers in the United States,
and each undergoes an annual review by the Foundation before it
receives continued accreditation, and thus, access to the CFFPR.
A requirement for accreditation is that the Centers enter data into
the Registry.2
Notable Attribute
The Foundation provides monetary payments to Care Centers to
incentivize timely and accurate data entry. Payments are proportional to the number and completeness of data.3
The investigation and study of new and improved therapies for CF
patients is ongoing in the United States. One noted feature of the
Registry is its use to study novel therapies. Pharmaceutical companies often rely on the CFFPR to conduct post-market approval
studies; and use of the Registry for this purpose is often less
expensive than traditional clinical trials.9 The Registry also allows
for long-term patient follow up, which is often difficult to achieve
in traditional trials.10
Management & Operation
Uses and Users
The CFFPR is governed by the CF Foundation, which hosts an
advisory committee composed of providers, researchers, and
database experts who provide strategic oversight. Significant resources are provided by the Foundation to maintain the Registry,
including user support, maintenance of proper documentation,
conduct of quality control measures, and oversight of technical
upgrades and data warehouse management.4 In addition to funding from the Foundation, pharmaceutical companies can enter
into licensing agreements to use the Registry for post-market
approval studies, as required by the United States Food and Drug
Administration.5
Data Collection – What & How
Data are collected through a secure Web-based portal.6 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. The CF Foundation provides data entry personnel with user manuals and data entry guidelines.7
Patient-level information collected includes:
1. State of residence
2.Height
3.Weight
4.Sex
5. Results of diagnostic tests (e.g., sweat tests and genotyping)
6. Results of regular lung function tests
7. Medication use
8.Hospitalizations
9. Results of microbial cultures (i.e., samples)
10.Complications related to CF
Highlight: CFFPR Viewed as a Leader
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.8
Multiple stakeholders reference, use and/or rely on data and associated outputs from the CFFPR to guide their work and improve
health outcomes.
Products include:
• Web-based Data Portal: An electronic platform that enables entry of and secure access to CFFPR data. The portal also serves
as a repository for clinical tools, care guidelines, and reports of
all quality improvement activities conducted by CF Centers.11
• Annual Report: The Foundation releases annual reports that
summarize all data collected for the calendar year.
• Center Director Report: Care Centers receive a detailed version
of the annual report that includes case report forms of data
collection. This report is intended to inform clinicians providing care for CF patients only, and therefore is not in the public
domain.
• Online Resources: The CFFPR offers online resources, such as
guidance documents and clinical tools with information on
evidence-based practices, which are available through the electronic platform.
• Scientific Papers and Presentations: Research findings based on
Registry data are shared via peer-reviewed journal publications
and presentations. These products are intended primarily for
the scientific and policy communities.
• Independent Patient Reports: Care Center staff can download
patient reports from the Web portal; these provide data on past
clinical visits and hospitalizations, and track individual patient
characteristics (e.g., weight) over time. These reports are used
by clinicians to prepare for patient visits and may also be shared
with patients to help initiate discussions to promote disease
self-management.12
• Center Level Reports: Center reports compare Centers with each
other, which allows for benchmarking performance against
peers.
• Risk-Adjusted Outcomes Reports: Risk-adjusted outcomes reports are published for public use on the Foundation’s website.
Users include:
• Care Centers: Centers use data to inform internal quality
improvement efforts, such as improving care practices. Centers
can create customized queries to pull data from the Registry to
inform specific activities.
3 | Cystic Fibrosis Foundation Patient Registry (CFFPR)
• Clinicians: Clinicians access data through the Web portal to
monitor patients in real time. Clinicians can also access educational materials, including best practices for treatment, to be
used in clinical decision making. Lastly, clinicians use individualized data, located in the portal, to monitor and assess their
own performance.
• Researchers: Extramural researchers can request access to
an analytic data set with CFFPR that has been cleaned and
de-identified. All research requests go through a formal review
process and are put forth to a review committee led by the CF
Foundation. This committee evaluates the scientific merit of
data requests for all external research studies and then searches
and subsequently pulls data from the data warehouse.
• Patients and Caregivers: The CFFPR Annual Report and
educational materials informed by CFFPR data are publically
available on the CF Foundation website. As of now, patients do
not have direct access to Registry data in other forms.13
• Pharmaceutical companies: Pharmaceutical companies can
partner with the CFFPR to conduct post-market-approval
studies for new therapies; these studies can be performed in less
time relative to traditional studies. When research results are
generated more quickly, and with a comparable level of rigor,
both providers and patients benefit.
Contributions
The CFFPR is often credited with having a profound effect on CF
Care Centers and the administration of patient care. Significant
highlights include:
1. Serving as historical archive for rare, pediatric disease: 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.14
2. Proving that early diagnosis improves health outcomes: a study
looking to determine 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 was 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.15
3. 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.16
4. Benchmarking across Care Centers: a recent study investigated
clinical best practices resulting in best patient outcomes across
Care Centers. The Foundation organized “benchmarking
teams” composed of providers, researchers, and representatives from other disciplines to investigate adult and pediatric
outcomes in 10 Care Centers. The teams visited each Center
and performed a series of data analyses, informant interviews,
and surveys.17 Findings illuminated that improving patient
outcomes is not only limited to the development of new therapies and treatments; it must also include adapting key attitudes
and practices within Care Centers. For example, high performing Centers had well-functioning and diverse care teams
in terms of their job functions (e.g., providers, technologists).18
Care Centers performing at varying levels in terms of patient
outcomes (e.g., those performing worse than similar Centers)
are able to learn from their peers through the dissemination
of this information. Many Centers cite that the ability to learn
from others is a main attribute of the Registry.
Looking Forward
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.
Using Registry Data for CER to Identify Optimal
Medication Regimens
With the ongoing expansion of new therapies for CF, there is an
increasing desire among clinicians and researchers to use the Registry to document ‘real-world’ treatment effects, such as environmental effects on patient outcomes, and for comparative effectiveness research. Currently, it is difficult to compare diseases with
different severities (e.g., mild to severe cases) using registries. The
Foundation is working toward making the Registry an important
tool for conducting this research, specifically determining the best
treatment alternatives and combinations for patients.19
Comparing CF in Other Countries
The success of the Registry has led other countries around the
world, such as New Zealand, to develop their own CF Registries.
Efforts are underway to create linkages between these registries,
allowing for international comparisons and quality improvement
initiatives.20,21
4 | Cystic Fibrosis Foundation Patient Registry (CFFPR)
Cited Example: Comparing Toronto and Boston Care Centers
Two cystic fibrosis clinic populations of similar size and age distribution were compared with respect to growth, pulmonary function, and survival. CFFPR data was pulled for 499 patients in Boston area Care Centers. Similarly, data for 534 CF patients in Toronto, Canada were collected.
Patients compared were of similar pulmonary function. Comparing data from these two cities uncovered that the differences in patient growth
and survival rates may be influenced by nutritional guidance as a treatment therapy, specifically the restriction of dietary fat. In Toronto, patients
replaced traditional diets with an alternative approach of high-fat, high calorie diets. Further evidence resulted in confirmation that nutritional
factors are directly related to CF outcomes.24
The ability to compare study populations across countries is a benefit to researchers, providers and patients, all of whom rely on accurate
information to influence their decision-making process and work toward improving health outcomes for CF patients.
Minimizing Data Entry Burden
Currently, when a Center submits an incorrect or incomplete data
element to the Web portal, the entire patient file is flagged. Then,
the Registry notifies the Center of the error. The Center must
investigate the patient file to see which piece of information is
problematic and then resubmit the entire file. The Foundation is
looking to integrate technology that would allow Centers to only
submit missing or incorrect data elements, as opposed to full files.
Doing so will minimize administrative burden on Care Center
sites.22
Linking and Leveraging Additional Sources of Data
The CFFPR is investigating how to collaborate with state health
exchanges that are currently collecting massive amounts of patient
data. One way to collaborate includes pulling data from exchanges
directly into the Registry, thus avoiding data duplication for Care
Centers. Though this activity is on the horizon, researchers noted
that full implementation of state data exchanges are needed before
extraction is possible.23
Efforts for Possible Consideration
Stakeholders have noted the benefit of linking Registry data with
claims data, which will allow for studies that illuminate the value
(e.g., cost effectiveness) of CF treatment.25 Efforts to find data
sources with sufficient CF representation are underway.
Additionally, patients and caregivers have noted that the Registry
patient report is a useful resource and often is used to inform
their therapy options. Looking forward, patients remarked that
having full access to their raw data via the Registry Web portal, in
addition to patient reports, would be beneficial.26
Citations
1
Schechter, M., et al. “The Cystic Fibrosis Foundation Patient
Registry as a tool for use in quality improvement,” BMJ Quality
and Safety, Vol. 23. I9 – i14, 2014.
2
Key informant interview, Summer 2014.
3
Ibid.
4
Schecter, M., et al. “The Cystic Fibrosis Foundation Patient
Registry as a tool for use in quality improvement,” BMJ Quality
and Safety, Vol. 23. I9 – i14, 2014.
Key informant interview, Summer 2014.
5
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.
6
Key Informant Interview, Summer 2014.
7
Ibid.
8
Ibid.
9
Ibid.
10
Ibid.
11
Ibid.
12
Ibid.
13
Ibid.
14
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.
15
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.
16
Boyle, M. et al., “Key Findings of the US Cystic Fibrosis Foundation’s Clinical Practice Benchmarking Project,” BMJ Quality
and Safety, Vol. 23. I5-i22, 2014.
17
Ibid.
18
Schechter, M., et al., “The Cystic Fibrosis Foundation Patient
Registry as a tool for use in quality improvement,” BMJ Quality
and Safety, Vol. 23. I9 – i14, 2014.
19
Key informant interview, Summer 2014.
20
Goss, C.H. et al, “Children and young adults with CF in the
USA have better lung function compared with the UK.” BMJ
Thorax, September 2014.
21
Key informant interview, Summer 2014.
22
Ibid.
23
5 | Cystic Fibrosis Foundation Patient Registry (CFFPR)
Corey, M. et al, “A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and
Toronto.” Journal of Clinical Epidemiology, Vol. 41 (6): 583 –
591. 1988.
24
Ibid.
25
Ibid.
26
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.