Transcript Document

PI CONNECT:
Melding Two Databases To Guide Research And Outcomes
Measurements In Patients With Primary
Immunodeficiency
Marcia Boyle
President & Founder
Immune Deficiency Foundation
Elyse Murphy
Medical Science Liaison Leader, Immunology
CSL Behring
INGID Programme: Selected Abstracts 03
October 31, 2014
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The Changing Face of Medicine
 Advent of Electronic Medical Records (EMR)
• Systematic collection of comprehensive individual data
• Easily accessible and transferable to improve care coordination
• American Recovery and Reinvestment Act of 2009 passed in US
mandated meaningful use of EMR (Electronic Medical Records) and EHR
(Electronic Health Records)
• Barriers include costs, training, and cross compatibility
 Digital registries and databases
• Accumulate knowledge on disease progression and optimal disease
management
 e Health era
• Healthcare Apps
• Provider recommendations centered around stand-alone wellness, diet,
and fitness.
• Difficult to assess effectiveness, safety and outcomes
Terry. “mHealth Apps Should Be Reviewed and Certified, Experts Argue”,
Medscape Medical News .June 10, 2014
97%
96%
98%
97%
98%
99%
97%
2012
98%
2009
*No 2009 date for Switzerland
http://ehrintelligence.com/2012/11/15/us-canada-gain-on-other-countries-in-emr-adoption accessed 9.3.2014
46%
41%
69%
82%
72%
67%
68%
37%
56%
92%
95%
Adoption of Electronic Medical Record
US Physicians Perception of EMR Benefits
The Changing Face of Medicine
 Patient voice becoming integral part of research
• Data gathered:
o Quality of Life Tools: General, Health Related, Disease
Specific
o Treatment Satisfaction Surveys
o Symptom Assessment Tools
o Other non validated questionnaires
• Impact of data:
o Improve or set standards of care
o Identify patients concerns
o Address treatment related issues that affect quality-of-life
o Develop interventions
http://www.cdc.gov/hrqol
Phillips KM et al. Quality of life outcomes in patients with chronic myeloid leukemia treated
with tyrosine kinase inhibitors: a controlled comparison. Supportive Care in Cancer April
2013, Volume 21, Issue 4, pp 1097-1103
Gap: Exquisite Need for Real Life Data in PI
 Data gathered from patient input
• Paper records
• Electronic personal health record (ePHR), Apps
• Drawbacks – can lack clinical records, missing
data
 Data from HCP input for PI patient registries:
• United Stated Immunodeficiency Network
(USIDNET)
• European Society Immunodeficiency (ESID)
• Drawbacks- lacks patient perspective; missing
data points from other treating physicians (of that
same pt) who have not contributed to registry.
How Can This Be Achieved?
Opportunity to create/capture patient and HCP
data
• IDF granted an award from the Patient
Centered Outcomes Research Institute
(PCORI) to create a patient-powered
research network titled PI CONNECT, to be
part of PCORnet: the national patientcentered clinical research network.
Patient Centered Outcomes Research
Institute (PCORI)
 Independent non-profit created and funded by the
U.S. Government
 Mission: Improves health care delivery and
outcomes by
• Promoting evidence-based information
• Incorporating patients and caregiver input into
research
 Focus: Comparative effectiveness research that
answers questions that matter to patients and
involves patients every step of the way
PCORnet
 PCORnet: The National Patient-Centered Clinical Research
Network
• Goal: Create a large, highly representative, distributed
national network for conducting clinical outcomes
research.
• Improve the U.S.A’s capacity to conduct comparative
effectiveness research (CER).
• CER in this case refers to use of patient-reported
outcomes rather than cost effectiveness
 PCORnet aims to facilitate the shift in health research from
researcher-driven to patient-centered research.
 Overall, this initiative will promote a more comprehensive,
complete, longitudinal data infrastructure.
PI CONNECT
eHealth Evolution for PI
 2011- IDF launched an online electronic personal health record
(ePHR)
• Developed for individuals and families living with primary
immunodeficiency diseases
• Patients track their symptoms, record medications and log
health information
• No cost for patients and family members
 Fall 2014 - IDF transformed ePHR with new features, including
cutting edge tracking and reporting tools:
• Sync with Microsoft HealthVault
• Set health and fitness goals and track progress
• Quality of Life survey instruments
• Connection to PI CONNECT
IDF ePHR Integrates with
• Microsoft HealthVault is a web-based program to
store and maintain health and fitness information.
• Information from various apps and devices can be
added to IDF ePHR through HealthVault.
IDF ePHR Users
 Original IDF eHealthRecord supported almost 1,000
users with 48 different PI types.
 Demographics of IDF ePHR users
• 22% - 17 years or younger
• 35% - 18-44 years
• 32% - 45-64 years
• 10% - 65 years or older
 Sex
• 63% - Female
• 37% - Male
USIDNET
 The United States Immunodeficiency Network (USIDNET)
oversees a Registry of Patients with PI.
• Funded by National Institute of Allergy and Infectious
Diseases (NIAID)
• Governed by a Steering Committee of top immunologists
• Administered as a program of IDF
 Physician-validated clinical data on over 3,800 patients with PI
• Laboratory results
• Genetic and molecular information
• Diagnostic criteria
 Longitudinal Data
• Incumbent upon clinicians updating patient records over time
• Updates do not always happen
PI CONNECT
Allows IDF to create a premier research network for the PI
Community by doing the following:
 IDF ePHR: portal for the PI CONNECT system
 Through the ePHR, patients can:
• Electronically consent into USIDNET Registry
• Update their records with patient generated data
• Download their EHR/EMR compatible files
 Once a patient consents, patient information can be
validated by their physicians
 Data identified by source
 Research Forum: interactive communication between
researchers and patients
Electronic Consent
 USIDNET Patient Consent Process
• IRB-approved
• Traditional consent language on paper
• 11 pages; patient must initial every page and several signatures are
required
• Time burden on patient and clinician
• Large room for error and risk of incomplete/non-compliant consent
forms
 PI CONNECT Electronic Consent Process
• IRB-approved
• One page, simplified and plain-language e-consent
o Patients can read and truly understand what they’re agreeing to
do
o Assured of their data’s safety and security
• One click
• No staff time required
PI CONNECT
 Anticipate 1250 patients will be involved with PI
CONNECT at the end of 18-month PCORI award
period
• Based on 0.5% of the PI patient population,
which was identified by IDF prevalence survey
 Both USIDNET registry and IDF ePHR use the
same servers and platforms, making transfer of
data possible and secure
Boyle JM, Buckley RH (2007). "Population prevalence of diagnosed primary immunodeficiency diseases in the
United States". J. Clin. Immunol. 27 (5): 497–502
Case Study: Value of Blended Data
Autoimmunity and Inflammation in XLA*
 Two sources: USIDNET and Patient Reported IDF Survey
 USIDNET Steering Committee- Hypothesis-Incidence of
Autoimmune/Inflammatory conditions in registry among
patients with XLA is under reported
• Asked IDF to perform a survey of patients with XLA and help
compare that survey data to data on XLA patients in registry.
• Patients reported higher incidence of diagnoses of AI as well
as higher incidence of symptoms consistent with an AI
diagnosis than what was found in the registry
• Use of both a patient survey and USIDNET Registry data
was important in gaining a better understanding of the
association of inflammatory disease in XLA.
Hernandez-Trujillo et al. Autoimmunity and Inflammation in X-linked Agammaglobulinemia. JCI Aug
2014, Volume 34, Issue 6, pp 627-632
USIDNET vs. XLA Survey Reported
Conditions
Condition
USIDNET
Registry
(N=149)
XLA Survey
(N=128)
p value
Aches, malaise or fatigue
22 (14.8)
44 (34.4)
P < .001
Pain, swelling, arthralgia
18 (12.1)
28 (25.0)
P = .035
Abdominal pain
10 (6.7)
22 (17.2)
P = .008
Diarrhea
13 (8.7)
27 (21.1)
P = .005
Chills
1 (.7)
17 (13.3)
P < .001
Shortness of breath
1 (.7)
21 (16.4)
P < .001
3 (2.0)
14 (10.9)
P = .002
Constipation
2011 Immune Deficiency Foundation XLA Survey
Looking Ahead with PI CONNECT
 Provide a larger, merged database of validated
patient reported outcomes from the IDF ePHR and
clinician data from the USIDNET registry
 Combined dataset will help prioritize research efforts
and illuminate patient input on treatment, quality of
life and healthcare resource utilization
 Provide insights on treatment, optimize outcomes
 Give patients a seat at the research table through
better data and the PI CONNECT Research Forum
Types of Queries That Can Be Asked of
Data Networks
 Demographics
 Treatment choices and trends
 Quality of Life – PROMIS 29 from NIH will be
built into ePHR
 PCORnet queries - 11 Clinical Data Research
Networks and 18 Patient-Powered Research
Networks
Thank you!