Pulmonary Fibrosis Advocates Cloud Based Mult-Disease Registry PCORI Grant

Pulmonary Fibrosis Advocates - Cloud Based, Patient Accessible, Cross-Disease Adaptable Registry

12401 Minnetonka Boulevard, Suite 200
Minnetonka MN 55305-3994
952.933.9990 | www.pfadvocates.org

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Creating an Affordable, Easy to Use, Cloud-Based Multi-Disease
National Patient Registry Platform for deployment in 2013

Executive Summary
- Why create a multi-disease national patient registry platform for fibrotic diseases?
Progressive scarring of the heart, blood vessels, lung, liver, kidney and brain leads to millions of
deaths world-wide each year, but to date, there has been no systematic widely available registry
collecting data addressing key issues involved in fibrotic diseases generally: • the course of
disease; • understanding variations in outcomes; • identifying factors that influence prognosis and
quality of life; • defining care patterns and disparities in the care delivery of care; and • measuring
quality of care and treatment effectiveness.

In its most basic form, a disease registry is an information collection system designed to support
organized care management.1 Patient registries are fundamental to basic, applied and translational
research in disease causation, prevention and treatment, yet most so-called ‘rare diseases’ (those
with patient populations of less than 200,000) lack research-enhancing registries. An estimated 60
million people in Europe and North America are living with such rare diseases.

One example is idiopathic pulmonary fibrosis (IPF), which kills an estimated 40,000 Americans
each year, comparable to the toll for breast cancer. Although IPF is showing alarming increases
in incidence and prevalence, there is no known cause or FDA-approved treatment nor is there a
national registry for IPF.

We intend to create an intuitive, web-based registry platform designed to address issues that would
be applicable for all rare, low and intermediate frequency fibrotic diseases. Such a registry, using
Idiopathic Pulmonary Fibrosis as the pilot disease, but readily and affordably adaptable to other
diseases, including non-fibrotic illnesses, will fill a critical void in our public health infrastructure and
will benefit patients, families, treatment providers, researchers, pharmaceutical and device
industries and policy makers2.

Until now, patient registries have been based on custom software and data management programs
created for specific diseases with the software and data stored on local networks. These locally
managed networks and registries are typically expensive to build and are not easily accessible by
patients or by researchers beyond local or regional research or clinical groups.

With the introduction of cloud computing and a new generation of software development tools, it is
now possible to build a first class disease registry that can be securely accessible to patients and
to the entire health care community. This new registry platform, carefully designed with easy, web-
based entry-level access to patients, family members and caregivers, will exponentially increase
patient participation – especially participation by currently under-served and under-represented
patient communities.

The new registry platform will be created by merging an existing and widely used disease
monitoring program with a commercially available, HIPAA compliant cloud-based software
development platform. The system will be designed to be adaptable to the unique needs of different

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illnesses and research protocols and will address HL7 standards for cross platform compatibility
with diverse EHR/EMR systems. We estimate that the system will be operational for initial
application to pulmonary fibrosis nationwide by the end of 2013.

This new multi-disease national patient registry platform effort is consistent with the JOINT
DECLARATION OF KEY PRINCIPLES FOR RARE DISEASE PATIENT REGISTRIES that was announced in
November of 2012 by the European Organization for Rare Diseases (EURORDIS), the National
Organization for Rare Disorders (NORD) and the Canadian Organization for Rare Disorders
(CORD) including:
 “Patient Registries should be recognized as a global priority in the field of Rare
Diseases
 “Interoperability and harmonization between Rare Disease Patient Registries
should be consistently pursued
 “A minimum set of Common Data Elements should be consistently used in all Rare
Disease Regsitries
 “Rare Disease Registries should include data directly reported by patients along
with data reported by healthcare professionals
 “Public-Private Partnerships should be encouraged to ensure sustainability of Rare
Disease Patient Registries, and
 “Rare Disease Patient Registries should serve as key instruments for building and
empowering patient communities”
http://rarediseases.org/docs/registries-2012

Sponsoring Organization
Consistent with the intent of the PCORI grant, this initiative originated with and is being sponsored
by the PULMONARY FIBROSIS ADVOCATES, a national not-for-profit [501(c)(4)] advocacy group
consisting of patients, families, caregivers, clinicians and researchers. Our Board of Directors3
includes former six-term Congressman Brian Baird, Ph.D., whose father died of PF and who
sponsored legislation in Congress to create a PF registry. Our Director, Paul Fogelberg, is a PF
patient and patient support group leader. Our clinical team is led by a uniquely experienced and
highly-regarded 7-member MEDICAL ADVISORY BOARD4 and we have pledged to incorporate other
vital stakeholders from the patient, public and private sectors deemed essential to refining the most
useful and widely effective format for the new registry platform.

Softw are and System Design Elements and Team
Software design & development is led by DataWeb of Puyallup WA
(dataweb.com), an early entrant in cloud computing and electronic health
records. DataWeb’s fully hosted software development technologies form the foundation of full
featured electronic health records application currently in use at a number of colleges as the
application for teaching EHR concepts and usage. The DataWeb Platform is scheduled to be fully
HIPPA compliant by May 15, 2013. Another major benefit of working with DataWeb is its unique
ability to provide web-based language-translation services making the new patient registry platform
especially user-friendly to the non-English speaking population. By using DataWeb’s hosted
language services application, translation services can be quickly provided at the time the patient
is registered. We will also partner with nationally respected public and private medical research
centers that currently use HIPAA-compliant servers to host and facilitate the collection and
distribution of registry data. Instructions and links to the DataWeb EHR Website are provided on
the accompanying final submission form and at the end of this document after the references.

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In addition to the unique expertise and market-proven resources of DataWeb, we will
incorporate field-tested designs and experience from the Chronic Disease Electronic
Management System. CDEMS is a software application developed by the Washington
State Diabetes Prevention and Control Program in 2002. It is a Microsoft Access
database application designed to assist medical providers and management in tracking the care
and outcomes of patients with chronic health conditions.

CDEMS is pre-coded to track diabetes, asthma and adult preventive health but is customizable to
change those tracking measures or define measures for monitoring other chronic conditions.
Printed progress notes, patient
intervention lists, and summary
reports generated from the
registry database can proactively
and positively alter the way
services are delivered while
measuring quality improvement
efforts. Since its inception,
community health centers, rural
clinics, primary practices,
hospitals, IPA’s and quality
improvement projects have used
CDEMS across the US and in
Canada, India, Haiti, the Marshall
Islands and South Africa. The
map shows many of the sites
using CDEMS in the USA and
lists others.

There are many advantages to using CDEMS as a basis for this initiative. As noted, CDEMS was
designed to be customizable in order to accommodate the requirements of other chronic diseases.
The sustained and ongoing experience with the system, it’s proven adaptability in applications
across diseases and its broad existing user base across the nation (and worldwide) means it has
been tested and proven in multiple applications and has a strong existing community of users who
are familiar with the system. At this point, the main need is to make CDEMS accessible on the
cloud and allow for wide user-friendly direct patient, clinician and researcher interface.

Primary support for CDEMS today comes from the Office of Health
Services Research (OHSR) at West Virginia University, which has
made extensive use of, and substantial updates and upgrades to the system. The Leader of the
OHSR team at WVU has agreed to work with us on this project. The direct link to the CDEMS site
now maintained by OHSR at WVU (including a download of the software and accompanying
information) is http://publichealth.hsc.wvu.edu/ohsr/Dowloads. Login instructions for the
CDEMS Website, and a link to an instructional video for the download, are included at the end of
this submission following the references.

Based on guidance from our patient, clinical and research advisory team, our initiative will update
and adapt CDEMS to make the system cloud-based for easier accessibility to patients, physicians
and researchers. In addition, we will allow for new data sets to be included, ensure full HIPAA
compliance regarding permissions, informed consent, data storage and access and other matters.
Our planning will also accommodate future enhancements including incorporation of HL7
Standards [Health Level Seven International – www.hl7.org/about/index.cfm?ref=nav] for
compatibility and eventual interface with multiple EHR/EMR systems.

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Patient Engagement
A key element of this project, enabled by its cloud-based adaptation, is to establish simple browser-
based mechanisms by which patients, families, caregivers, clinicians and researchers can more
directly access and interact with registries. Presently, nearly all patient registries depend on
providers or researchers soliciting patients for entrance. Our new registry system will allow patients
to directly access a user friendly, browser-based interface through which they can sign up for the
registry and enter basic information about their condition and treatment. This unique entry-level
access for patients will be part of a tiered (and appropriately fire-walled) access structure designed
to promote the maximum level of patient sign-ups while also providing necessary restricted access
to more technical information provided by the patient’s health care team or researchers.

The rationale for this new approach is that many patients are willing and eager to participate in
research and would be glad to voluntarily sign up for a registry if given the opportunity and a user-
friendly mechanism for doing so. Currently, unless a willing patient lives near a specialized
research center or has a health care provider involved in a defined research protocol, this is not
easily or practically accomplished. Indeed, from the patient’s perspective, in most instances it is
impossible. As noted previously, this new registry platform will exponentially increase patient
participation – especially participation by currently under-served and under-represented patient
communities.

Because the system proposed here will be cloud-based, patients will be able to access it directly
from standard web browsers or apps at home or in any provider’s office. Protocols will be
established through which researchers can access the database to identify and enlist suitable
patients for participation in research studies and/or clinical trials. With help from patients, clinicians,
research and technology team members, a standard process will be established with appropriate
security and participation protocols to determine how access to the data for research use is granted,
and for what purposes.

Idiopathic Pulmonary Fibrosis (IPF) as the Demonstration Disease
Idiopathic Pulmonary Fibrosis is an ideal disease to demonstrate the functionality of this multi-
disease registry platform. Progressive scarring of the heart, blood vessels, lung, liver, kidney and
brain leads to millions of deaths world-wide each year. Idiopathic Pulmonary Fibrosis (IPF) is a
prototype for this class of disorders.

IPF is a progressive scarring of the lungs estimated to afflict 200,000 Americans and up to 1 million
persons world-wide. In IPF, there is unrelenting spread of scar-forming cells from scarred airspaces
into contiguous anatomically intact airspaces. This creates an expanding web-like network of scar
tissue that causes death by asphyxiation in an average of 3 to 5 years. Although the cellular and
molecular events characterizing IPF have been extensively investigated for more than three
decades, there are still no FDA-approved drugs for IPF. The only proven therapeutic option is lung
transplantation.5

Presently, IPF claims as many lives each year as breast cancer (40,000), yet it has no national
patient registry, there are relatively few ongoing clinical trials, and there is presently no known
cause or effective treatment. Establishment of a national patient surveillance registry has been
identified as a top priority by patients, patient advocacy groups, both treating and research
physicians and pharmaceutical companies. The NHLBI Workshop, Strategic Planning for IPF
Research (Nov. 27-28, 2012) Executive Summary notes the need to:

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“• Develop additional clinical tools and collaborations among different
stakeholders to facilitate future clinical trials/studies of IPF;” and
“• Identify new, creative means for conducting efficient and cost-effective clinical
trials and for partnering with industry to sponsor phase II/III clinical trials.”
http://www.nhlbi.nih.gov/meetings/workshops/fibrosis.htm

PF patients have shown themselves to be desirous of more research and are highly amenable to
participation. A strong network of PF patient support groups exists nationwide – at least 76 groups
in 34 states - and could quickly publicize and disseminate information about the registry, explain
why patients should sign up and help them do so. These efforts will be further aided by national
organizations such as the PULMONARY FIBROSIS ADVOCATES (PFA), the Pulmonary Fibrosis
Foundation (PFF) and through effective use of social media (including Facebook, Twitter and
various patient websites and blogs). A broad and willing pilot patient population – as exists in the
PF community – is the ideal prototype to test and refine the pilot-project.

Projected Cost
By using the CDEMS design and architecture, plus the rapid development and integrated security
features of the DataWeb Platform, we will deliver a first class disease registry application at a much
reduced cost. Estimated costs for developing and refining the DataWeb and CDEMS software and
applying it to a single disease, beginning with pulmonary fibrosis (but readily adaptable to virtually
all other fibrotic diseases), is approximately $80,000 to $100,000. For comparison purposes,
current disease-specific registries often have development costs that exceed millions of dollars and
are, therefore, prohibitive to implement for most so-called ‘rare’ illnesses.

Once established, the new registry platform will be made available to other diseases and patient
populations, with relatively small costs of adaptation and refinements of sustaining specific
applications to be generated by combined efforts of additional patient groups, clinicians, research
users, and private sector interests such as insurance companies, pharmaceutical or medical device
companies as appropriate to the specific illness and treatment approaches.

How This Proposal Meets Evaluation Criteria
1. The actual or described technical capacity to efficiently and effectively connect patients
and researchers, across multiple platforms and levels of complexity.
The integration of an existing data registry (CDEMS) in combination with a commercially successful
cloud-based EMR system (DataWeb) and the involvement of the programming expertise from those
entities demonstrate that the technical capacity is present to achieve project goals. In addition, the
patient, medical and research advisory team includes some of the nation’s most prominent and
respected treatment and scientific professionals in fibrotic diseases.

On the patient side, PULMONARY FIBROSIS ADVOCATES includes patients from across the country
and has direct tie-in support from multiple PF Patient Support Groups. The PFA is also in close
working contact with other, related organizations and we have pledged to work cooperatively with
them to design, build and disseminate information about the registry. As described above, the
registry will be designed to have entry level, self-initiated patient enrollment and basic demographic
and disease data entry. There will then be a second level data access element for more technical
clinical or other research information to be entered as dictated by treating physicians and/or
research personnel. The registry will be easily customizable by expert researchers and treatment
professionals for each specific disease entity, treatment, or research protocol.

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2. Usability, scalability and sustainability across diverse populations with differing levels
of access to, understanding of and experience with technology and health/health care
information.
This criterion is really at the essence of our proposal. The CDEMS platform has already shown
practical adaptability across multiple diseases and settings and is currently in use in many different
locations around the world; however, it is not cloud-based. Therefore, the system we are designing
will enhance access to any stakeholder who has internet connectivity. By placing the system on
the cloud, and allowing for direct web-based interface, patients across the country, whether in rural
or other underserved settings, will be readily able to participate. Many patients, such as those with
PF, are often so ill it is hard to come in to clinics or other settings where fixed location registries are
housed. By accessing the system on the web, patients, or close family members, can sign on and
enter data without having to visit a clinic or research site. This feature will also make “real time”
data entry much more practical for patients, which has significant research and treatment
advantages. We will also provide the ability for patients (or family members/caregivers) to easily
request translation and interpretation services at the time of registration. This will eliminate another
barrier that often makes it difficult for non-English speaking patients to take full-advantage of these
important registry resources. Our registry platform is designed to be user-friendly in both content
and technical interface so an average individual with basic linguistic and computer literacy can
easily sign up for the registry and enter core data. If additional, more technical data is needed, that
can be accomplished with assistance of medical or research staff as needed for a given purpose.

3. Degree of development of the model or prototype
A significant advantage of this proposal is that the core platforms, the CDEMS registry and the
Data Web Platform, are already operational and proven. The programming team at DataWeb
specializes in translating database systems into cloud-based structures. That Team has already
reviewed the underlying CDEMS design and code. Their market-tested technology team is
confident it can be adapted to meet the requirements described in the proposal within six-to-eight
months (i.e., in 2013).

4. Differences in the ways that patients, caregivers and researchers understand, describe
and seek answers to the problems they face or the issues they’re trying to address
(e.g., different terms for similar concepts, general levels of health literacy, etc.).
Unlike most existing (institution-specific) registries, this new registry platform directly empowers
patients and many more caregivers by allowing and encouraging patients to sign themselves up
for patient registries. In addition, uniform protocols for access and use of these registries will
facilitate both public and private researcher to access exponentially expanded patient populations
for research and clinical trial purposes. The advantage of a single, national (or international)
cloud-based multi-disease national registry platform will minimize conflicts in definitions and
protocols that currently inhibit wide adoption or use of research-friendly patient information.

5. Maximizing “patient-centeredness” – ability to account for and effectively focus on and
address patients’ needs – while also emphasizing and facilitating researchers’ need to
emphasize the scientific rigor of any resulting collaboration.
Prior items have detailed how patient access to and participation in registries will be transformed
by the system the PFA is developing. We will use a tiered access system which allows patients to
sign up with basic personal and demographic information but will limit access to more technical
information to ensure accuracy and validity of that data. The precise nature of the technical data
to be gathered will be guided by the medical and research advisory panel for each specific disease

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to which the registry is ultimately applied. This tiered approach provides the maximum patient
involvement while still preserving the rigor of the research data.

6. The particular challenges of serving "hard-to-reach" audiences, including, but not
limited to, ethnic and racial minorities, rural populations, the elderly, the disabled/
physically challenged (i.e., Section 508 compliance) and those for whom English is a
second language.
As described in item 5 above, direct web access will significantly enhance accessibility issues to
rural and other underserved populations. So too, user-friendly web access will enhance access by
persons whose illness or disability makes it difficult to come in person to research or clinical
settings. For ESL issues, as noted above, our unique registry platform design features the ability
for patients (or family members/caregivers) to easily request translation and interpretation services
at the time of registration. This will eliminate another barrier that often makes it difficult for non-
English speaking patients to take full-advantage of these important registry resources.
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Footnotes

1
US Department of Health and Human Services, Health Information Technology and Quality
Improvement;
HHS web site, March 2013
See also: Registries for Evaluating Patient Outcomes: A User’s Guide, 2nd Ed., Gliklich, RE,
Dreyer, RA, editors, Agency for Healthcare Research and Quality, September 2010, Rockville MD

2
Registry of Patient Registries (RoPR)
http://effectivehealthcare.ahrq.gov/index.cfm/search-for-guides-reviews-and-
reports/?pageaction=displayproduct&productid=690

3
PFA Board of Directors (7 members)
Brian N. Baird, Ph.D. Edmonds WA
Former United States Representative (D, WA-3 / 6 terms)

Paul A. Fogelberg Minnetonka MN
Patient-Advocate & Director, PFA; President, The Professional Education Group

Dr. Jerome (Jerry) & Froma Sandler Bethesda MD
Lisa Sandler Spaeth Memorial Pulmonary Fibrosis Research Fund, Johns Hopkins University

C. Thomas Vangsness, Jr., MD Los Angeles CA
University of Southern California

Mark A. Shreve Titusville NJ
Founder & CEO, Coalition for Pulmonary Fibrosis (‘01-‘09); Senior Advisor, Pulmonary Fibrosis Foundation (2010-present)

Timothy P.M. Whelan, MD Charleston SC
Medical Director of Lung Transplantation, Medical University of South Carolina

4
PFA Medical Advisory Board (7 members)
Peter Bitterman, MD Professor of Medicine, Vice Chairman of Research, Department of
Medicine, University of Minnesota, Minneapolis
Timothy P.M. Whelan, MD Medical Director of Lung Transplantation, Medical University of South
Carolina, Associate Professor of Medicine
Lawrence C. Mohr, MD Professor of Medicine, Biometry and Epidemiology and Director of the
Environmental Biosciences Program at the Medical University of SC
Joao de Andrade, MD Associate Professor; Associate Director, Pulmonary Fellowship Program;
Director, UAB Interstitial Lung Disease Program – UAB Health System

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Richard Castriotta, MD Prof. of Internal Medicine, Pulmonary Division Dir. and Chair-Elect
of the Interfaculty Council at the Univ. of Texas Health Science Center
at Houston; Chief of Pulmonary Medicine, Chairman of the Institutional
Ethics Committee and Medical Director of the Sleep Disorders Center at
Memorial Hermann Hospital in the Texas Medical Center
Jesse Roman, MD Professor and Chairman of the Department of Medicine, Distinguished
University Scholar, University of Louisville, KY
Kevin M. Chan, MD Medical Director, Lung Transplantation, University of Michigan Health
System

5
Registries for Evaluating Patient Outcomes: A User’s Guide: 2nd Edition
http://www.effectivehealthcare.ahrq.gov/index.cfm/search-for-guides-reviews-and-
reports?/pageaction=displayproduct&productid=401
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Login instructions for CDEMS at West Virginia University

http://publichealth.hsc.wvu.edu/ohsr/Downloads

From that link the CDEMS software can then be downloaded and explored along with documentation,
reports, training videos etc.. CDEMS requires Microsoft Access to run. For an instructional video to assist
in the installation process of the CDEMS software, see http://www.youtube.com/watch?v=fdKq21Z31jo

Login Instructions for DataWeb EHR application as used in teaching settings at the
University of Washington - NB (This works best if accessed through Internet Explorer):
1. http://combsjbl.varipoint.com
2. Username: teststudentehrlab
3. Password: aloha123
Once on the site, click the ‘Select’ button to enter
From there, since this link is to a teaching demo of the EHR program, one can select the
profession level for which lessons are tailored, e.g. receptionist, nurse etc. Once this is
selected, samples of the database fields in the EHR system that would be accessed by those
professionals are provided. To see the kind of information available in the system, click the
drop down menu bars in the dark background across the top of the page.

______________________________________________________________________
Copyright © 2013, PULMONARY FIBROSIS ADVOCATES, Minnetonka, MN – All Rights Reserved

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Pulmonary Fibrosis Advocates Cloud Based Mult-Disease Registry PCORI Grant

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