Real world data is no longer just for those trained in health economics and outcomes research — it can and will touch everyone in the pharma/healthcare space.
CBI asked industry's foremost RWD thought leaders a variety of questions to better understand how bio/pharmaceutical teams can collaborate and capture data in an aggregated form to continue to improve the value of products in development with real world, real-time data.
Digital Healthcare Trends: Transformation Towards Better Care RelationshipKumaraguru Veerasamy
Digital health encompasses digital care programs, technologies with health, healthcare, living, and society to enhance the efficiency of healthcare delivery and to make medicine more personalized and precise. With the increasing adoption of telemedicine, wearable devices, mobile health apps (especially during the recent COVID-19 pandemic) and VR/AR; digital health is poised to take healthcare forward.
Pharmacovigilance in USA and Europe_Katalyst HLSKatalyst HLS
Introduction to Drug Safety & Pharmacovigilance in USA and Europe for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
This Module provides guidance on planning and conducting the legally required audits, the role, context and management of pharmacovigilance audit activity.
The principles in this module are aligned with internationally accepted auditing standards, issued by relevant international auditing standardization organizations and support a risk-based approach to pharmacovigilance audits.
This is a deck I created to frame out my vision about healthcare and used in my job search. This is the exciting space in healthcare right now - communications, segmentation, data mining.
Signal detection and management activities are at the core of ensuring drug safety. A complex process of signal detection; through their validation and confirmation; analysis and prioritisation; and signal assessment to recommending action.
Find out more at out training: http://bit.ly/1W31NCF
These slides were used for a invited presentation @ Patheon Seminar – Bridgewater, NJ, 31 July 2014.
Some modification have been made to connect the dots for the audience who will review this slide-deck on the internet.
This presentation provides a very brief snap-shot of a day long training program conducted recently at a company in India. In preparing the day long training session I had asked the following questions; (1) How to effectively communicate to an audience of a group of young and bright Indian professionals in any company in India and their supervisors/management about the importance of cGMPs and QbD? (2) How do I understand their challenges, perspectives and biases? (3) How do I connect with them to share the joy of Quality by Design?
The response received has been overwhelming from the audiences in India and yesterday at the Patheon Seminar in Bridgewater, NJ. I hope you will also the see some of the important dots and the connections. How this content connects to regulatory requirements is not covered in this slide deck – it connects via ‘A, B, C, D’ to 21 CFR, Quality Systems Approach to cGMP, ICH 7, 8, 9, 10, and 11.
Digital Healthcare Trends: Transformation Towards Better Care RelationshipKumaraguru Veerasamy
Digital health encompasses digital care programs, technologies with health, healthcare, living, and society to enhance the efficiency of healthcare delivery and to make medicine more personalized and precise. With the increasing adoption of telemedicine, wearable devices, mobile health apps (especially during the recent COVID-19 pandemic) and VR/AR; digital health is poised to take healthcare forward.
Pharmacovigilance in USA and Europe_Katalyst HLSKatalyst HLS
Introduction to Drug Safety & Pharmacovigilance in USA and Europe for Pharmaceuticals, Bio-Pharmaceuticals, Medical Devices, Cosmeceuticals and Foods.
Contact:
"Katalyst Healthcares & Life Sciences"
South Plainfield, NJ, USA
info@KatalystHLS.com
This Module provides guidance on planning and conducting the legally required audits, the role, context and management of pharmacovigilance audit activity.
The principles in this module are aligned with internationally accepted auditing standards, issued by relevant international auditing standardization organizations and support a risk-based approach to pharmacovigilance audits.
This is a deck I created to frame out my vision about healthcare and used in my job search. This is the exciting space in healthcare right now - communications, segmentation, data mining.
Signal detection and management activities are at the core of ensuring drug safety. A complex process of signal detection; through their validation and confirmation; analysis and prioritisation; and signal assessment to recommending action.
Find out more at out training: http://bit.ly/1W31NCF
These slides were used for a invited presentation @ Patheon Seminar – Bridgewater, NJ, 31 July 2014.
Some modification have been made to connect the dots for the audience who will review this slide-deck on the internet.
This presentation provides a very brief snap-shot of a day long training program conducted recently at a company in India. In preparing the day long training session I had asked the following questions; (1) How to effectively communicate to an audience of a group of young and bright Indian professionals in any company in India and their supervisors/management about the importance of cGMPs and QbD? (2) How do I understand their challenges, perspectives and biases? (3) How do I connect with them to share the joy of Quality by Design?
The response received has been overwhelming from the audiences in India and yesterday at the Patheon Seminar in Bridgewater, NJ. I hope you will also the see some of the important dots and the connections. How this content connects to regulatory requirements is not covered in this slide deck – it connects via ‘A, B, C, D’ to 21 CFR, Quality Systems Approach to cGMP, ICH 7, 8, 9, 10, and 11.
To comprehend the regulatory requirements to import medical Medical devices and authorization procedures in regulated markets of the United States and Australia
Practical Steps to Selecting and Implementing an eTMFMontrium
There are many benefits to moving to an electronic Trial Master File (eTMF) and many organizations are either thinking of moving, or are already in the process of doing so.
However, there are a great deal of things to consider before being able to successfully move to an electronic platform which are often overlooked, often resulting result in unsuccessful or sub-optimal eTMF implementations.
This webinar takes you through some of the practical steps that you need to go through to successfully implement and benefit from an eTMF.
Reshaping Global ICSR Reporting to Deliver Real-time Visibility and OversightVeeva Systems
Learn how you can reshape global ICSR reporting to deliver real-time visibility and oversight.
In this presentation we discuss:
- Challenges and approaches to safety data management and ICRS reporting
- How technology is making it easier to comply with global and regional requirements
- Considerations for incorporating affiliates into one pharmacovigilance solution
- Strategies for adopting a single multi-lingual safety solution
View the on-demand webinar here: https://bit.ly/35Pfyik
Digital Health Market has exploded in the last few years. Will that continue? What are the main areas of growth in digital days and what the future will bring us.
“Mobile Health(mHealth) is the use of mobile and wireless devices, the technology, to provide Health outcomes, Healthcare services and Health research.”
A brief description about Pharmacovigilance, aims and scope, need of pharmacovigilance, programs by WHO for international drug safety monitoring, UMC, VIGIBASE, WHO causality assessment scale and specific regulatory bodies of various countries
TMF Fundamentals - An Introduction to Better Trial Master File Management - M...Montrium
In this presentation, we explore the TMF fundamentals and an introduction to better managing your TMF. We'll start by diving into the world of TMF management, how to properly leverage the regulations, which documents constitute as TMF-worthy and what basic metrics you can track to increase the efficiency of your trial.
Finally, we will also discuss some of the features Montrium has developed to facilitate the management of the Trial Master File in a fundamental way.
This presentation covers the following topics:
-Fundamentals of TMF management process
-How to use the regulations to ensure success
-What cross functional groups hold TMF worthy documents
-TMF management challenges and how you can alleviate them
-Base metrics to track and what they mean to your organization
-How an eTMF helps TMF Management
You can follow along with this presentation via our webinar:
https://info.montrium.com/tmf-fundamentals-an-introduction-to-better-trial-master-file-management
Patients recognize the benefits of technology-enhanced care, yet only 1 out of 10 use remote patient monitoring today. Read three keys to adoption. https://accntu.re/3fnEy6r
Accelerating Patient Care with Real World EvidenceCitiusTech
Life sciences and pharma companies are evolving their strategies to utilize Real World Data (RWD) to demonstrate value of pharmaceutical and medical device innovations. Technology advancements at the point of care and improvements in data collection strategies have led to a significant increase in the availability of RWD in healthcare
Real World Evidence (RWE) can provide actionable patient insights and accelerates time to market of new medical products in order to gain competitive advantage
With the emergence of wearable technologies, Internet of Things (IOT), Cognitive Computing, Genomics, Blockchain, etc., future RWE data sources will become more diverse and extensive. This document introduces the concept of Real World Evidence studies in healthcare, describes the various data sources for performing real world analytics and illustrates the role of RWE in better patient care. It then summarizes challenges faced while performing RWE analytics with respect to regulatory compliance, data accessibility and sharing, analysis reporting, costs etc.
Analysis of what patient services proposed by pharma companies should be. This document explains: 1. why patient centricity is essential? - 2. how to craft a patient-centric strategy? - 3. How to implement patient-centric initiatives?
3 Phases of Healthcare Data Governance in AnalyticsHealth Catalyst
Healthcare data governance is a broad topic and covers more than data stewardship, storage, and technical roles and responsibilities. And it’s not easy to implement. It’s necessary, though, for health systems that are entering the world of analytics because the governance structure will enable the organizations to drive higher-quality, low cost care. In order for healthcare data governance to be most effective however, it needs to be adaptive because real healthcare data governance is much more fluid than any plan laid out on paper. Typically there are three phases that characterize successful analytics implementations: the early stage, the mid-term stage, and the steady state. As health systems begin to determine the effectiveness of their data governance strategy, it’s important to look at key metrics from their analytics implementations that will either trend up, remain solid, or trend down.
Extended Real-World Data: The Life Science Industry’s Number One AssetHealth Catalyst
The life science industry has historically relied on sanitized clinical trials and commoditized data sources (largely claims) to inform its drug development process—an under-substantiated approach that didn’t reflect how a new drug would affect broader patient populations. In an effort to gain more accurate insight into the patient experience and bring drugs to market more efficiently and safely, the industry is now expanding into extended real-world data (RWD).
To access the needed breadth and depth of patient-centric data, life science companies must partner with a healthcare transformation company that has three key qualities:
A broad and deep data asset.
Extensive provider partnerships.
An outcomes-improvement engine to support the next generation of drug development.
Bridging the Gap between Clinical Development and Patient Access with Real-Wo...Covance
Real World Evidence (RWE) is defined by the FDA as 'clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD).' RWD is the data collected from sources other than traditional clinical trials. **Disclaimer: This article was previously published. Sciformix is now a Covance company.
To comprehend the regulatory requirements to import medical Medical devices and authorization procedures in regulated markets of the United States and Australia
Practical Steps to Selecting and Implementing an eTMFMontrium
There are many benefits to moving to an electronic Trial Master File (eTMF) and many organizations are either thinking of moving, or are already in the process of doing so.
However, there are a great deal of things to consider before being able to successfully move to an electronic platform which are often overlooked, often resulting result in unsuccessful or sub-optimal eTMF implementations.
This webinar takes you through some of the practical steps that you need to go through to successfully implement and benefit from an eTMF.
Reshaping Global ICSR Reporting to Deliver Real-time Visibility and OversightVeeva Systems
Learn how you can reshape global ICSR reporting to deliver real-time visibility and oversight.
In this presentation we discuss:
- Challenges and approaches to safety data management and ICRS reporting
- How technology is making it easier to comply with global and regional requirements
- Considerations for incorporating affiliates into one pharmacovigilance solution
- Strategies for adopting a single multi-lingual safety solution
View the on-demand webinar here: https://bit.ly/35Pfyik
Digital Health Market has exploded in the last few years. Will that continue? What are the main areas of growth in digital days and what the future will bring us.
“Mobile Health(mHealth) is the use of mobile and wireless devices, the technology, to provide Health outcomes, Healthcare services and Health research.”
A brief description about Pharmacovigilance, aims and scope, need of pharmacovigilance, programs by WHO for international drug safety monitoring, UMC, VIGIBASE, WHO causality assessment scale and specific regulatory bodies of various countries
TMF Fundamentals - An Introduction to Better Trial Master File Management - M...Montrium
In this presentation, we explore the TMF fundamentals and an introduction to better managing your TMF. We'll start by diving into the world of TMF management, how to properly leverage the regulations, which documents constitute as TMF-worthy and what basic metrics you can track to increase the efficiency of your trial.
Finally, we will also discuss some of the features Montrium has developed to facilitate the management of the Trial Master File in a fundamental way.
This presentation covers the following topics:
-Fundamentals of TMF management process
-How to use the regulations to ensure success
-What cross functional groups hold TMF worthy documents
-TMF management challenges and how you can alleviate them
-Base metrics to track and what they mean to your organization
-How an eTMF helps TMF Management
You can follow along with this presentation via our webinar:
https://info.montrium.com/tmf-fundamentals-an-introduction-to-better-trial-master-file-management
Patients recognize the benefits of technology-enhanced care, yet only 1 out of 10 use remote patient monitoring today. Read three keys to adoption. https://accntu.re/3fnEy6r
Accelerating Patient Care with Real World EvidenceCitiusTech
Life sciences and pharma companies are evolving their strategies to utilize Real World Data (RWD) to demonstrate value of pharmaceutical and medical device innovations. Technology advancements at the point of care and improvements in data collection strategies have led to a significant increase in the availability of RWD in healthcare
Real World Evidence (RWE) can provide actionable patient insights and accelerates time to market of new medical products in order to gain competitive advantage
With the emergence of wearable technologies, Internet of Things (IOT), Cognitive Computing, Genomics, Blockchain, etc., future RWE data sources will become more diverse and extensive. This document introduces the concept of Real World Evidence studies in healthcare, describes the various data sources for performing real world analytics and illustrates the role of RWE in better patient care. It then summarizes challenges faced while performing RWE analytics with respect to regulatory compliance, data accessibility and sharing, analysis reporting, costs etc.
Analysis of what patient services proposed by pharma companies should be. This document explains: 1. why patient centricity is essential? - 2. how to craft a patient-centric strategy? - 3. How to implement patient-centric initiatives?
3 Phases of Healthcare Data Governance in AnalyticsHealth Catalyst
Healthcare data governance is a broad topic and covers more than data stewardship, storage, and technical roles and responsibilities. And it’s not easy to implement. It’s necessary, though, for health systems that are entering the world of analytics because the governance structure will enable the organizations to drive higher-quality, low cost care. In order for healthcare data governance to be most effective however, it needs to be adaptive because real healthcare data governance is much more fluid than any plan laid out on paper. Typically there are three phases that characterize successful analytics implementations: the early stage, the mid-term stage, and the steady state. As health systems begin to determine the effectiveness of their data governance strategy, it’s important to look at key metrics from their analytics implementations that will either trend up, remain solid, or trend down.
Extended Real-World Data: The Life Science Industry’s Number One AssetHealth Catalyst
The life science industry has historically relied on sanitized clinical trials and commoditized data sources (largely claims) to inform its drug development process—an under-substantiated approach that didn’t reflect how a new drug would affect broader patient populations. In an effort to gain more accurate insight into the patient experience and bring drugs to market more efficiently and safely, the industry is now expanding into extended real-world data (RWD).
To access the needed breadth and depth of patient-centric data, life science companies must partner with a healthcare transformation company that has three key qualities:
A broad and deep data asset.
Extensive provider partnerships.
An outcomes-improvement engine to support the next generation of drug development.
Bridging the Gap between Clinical Development and Patient Access with Real-Wo...Covance
Real World Evidence (RWE) is defined by the FDA as 'clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD).' RWD is the data collected from sources other than traditional clinical trials. **Disclaimer: This article was previously published. Sciformix is now a Covance company.
Data-driven Healthcare for the Pharmaceutical IndustryLindaWatson19
The tremendous opportunity of a data-driven strategy is apparent to the pharmaceutical industry, as all these informational assets exhibiting volume, variety, and velocity need to be ingested and analyzed for enhanced insight leading to better business decisions to address proactively the needs of patient care, while getting to market cheaper, faster, with better products.
The Role of Real-World Evidence in Supporting a Product's Value StoryCovance
Randomized clinical trials (RCTs) are the gold standard for gaining regulatory approval for marketing authorization for medical products. RCTs typically measure short-term efficacy and safety of a product compared to placebo in a fairly homogeneous population and under ideal, controlled conditions. In contrast, the real world consists of a heterogeneous population in which patient care is much less controlled and thus, more complex. Treatment decisions made in this setting are predicated on a wider array of co-morbid conditions, competing medications, physician preference and risk of adverse events than those observed in RCT populations. Evidence generated from real-world settings reflects this complexity, complementing evidence derived from rigorously controlled RCTs.
Healthcare data and its impact upon the patient care decision process via accurate, real-time, reliable data from disparate sources is creating a digital health revolution. Data-driven healthcare is beginning to have a huge impact addressing the challenges of every provider, through efficient handling of huge volumes of patient care data.
Providers need to move towards real-time analytics that have become critical to demonstrate their quality of care, as reimbursement by government programs can be contingent upon how providers are measured in “Quality of Care”. For example, the Medicare Access and CHIP Reauthorization Act (MACRA) of 2015, also called the Permanent Doc Fix, changes the way Medicare doctors are reimbursed with the implementation of a merit based incentive. The performance-based pressure is huge, which makes it imperative that every provider consider technology solutions. Read more at https://www.solix.com/solutions/data-driven-solutions/healthcare/
4 Essential Lessons for Adopting Predictive Analytics in HealthcareHealth Catalyst
Predictive analytics is quite a popular current topic. Unfortunately, there are many potential side tracks or pit falls for those that do not approach this carefully. Fortunately for healthcare, there are numerous existing models from other industries that are very efficient at risk stratification in the realm of population management. David Crocket, PhD shares 4 key pitfalls to avoid for those beginning predictive analytics. These include
1) confusing data with insight
2) confusing insight with value
3) overestimating the ability to interpret the data
4) underestimating the challenge of implementation.
When developing a business strategy centered on the patient, organizations must adapt and implement programs that foster information sharing and collaboration while providing faster and greater access to life-changing products.
mHealth Israel_GEARING COMMUNICATIONS TO RAISE CAPITAL AND ATTRACT CUSTOMERS_...Levi Shapiro
Presentation by Gil Bashe, Managing Director, Healthcare Practice, Finn Partners: "GEARING COMMUNICATIONS TO RAISE CAPITAL AND ATTRACT CUSTOMERS- FROM PLAN TO PARTNERS TO PATIENTS". Includes tips to avoid failure by embracing complexity, description of the Health Ecosystem Landscape, developing a plan to impact care, cost and outcomes, overview of the US Payer market, and top digital health influencers.
Chatty Kathy - UNC Bootcamp Final Project Presentation - Final Version - 5.23...John Andrews
SlideShare Description for "Chatty Kathy - UNC Bootcamp Final Project Presentation"
Title: Chatty Kathy: Enhancing Physical Activity Among Older Adults
Description:
Discover how Chatty Kathy, an innovative project developed at the UNC Bootcamp, aims to tackle the challenge of low physical activity among older adults. Our AI-driven solution uses peer interaction to boost and sustain exercise levels, significantly improving health outcomes. This presentation covers our problem statement, the rationale behind Chatty Kathy, synthetic data and persona creation, model performance metrics, a visual demonstration of the project, and potential future developments. Join us for an insightful Q&A session to explore the potential of this groundbreaking project.
Project Team: Jay Requarth, Jana Avery, John Andrews, Dr. Dick Davis II, Nee Buntoum, Nam Yeongjin & Mat Nicholas
2. EVIDENCEREQUIRED
Budget impact
Unmet need/
disease burden
Patient
recruitment
Understand
standard
of care
Post marketing
commitments
(safety etc.)
Adherence
Differentiation in
sub-populations Differentiate with
or vs. protected
formulation
Launch Pricing
Review
New
Competition
New
Formulation
Indication
Competitor
Goes
Generic
NOW PAST
Trial design
Utilization/
prescribing
patterns
Long-term
clinical
outcomes
Head to head
comparative
effectiveness
Target
populations
Effects of
switching on
outcomes
Usage
difference
DEVELOPMENT GROWTH PHASE MATURE PHASE
Real World Evidence Lifecycle*
REAL WORLD
DATA
“The New Currency
in Healthcare”
Real World Data (RWD) have become the
bio/pharmaceutical industry’s treasure trove for
information to inspire stakeholder decision-making.
As an industry, professionals have increasingly been
looking to RWD to not only assess the benefits and
risks of new medicines in clinical and real world
settings, but also as a way to advise healthcare
reimbursement decisions worldwide.
In the past, real world data was collected only during
the post-approval phase of clinical trials and within
patient registries. However, the industry is experiencing
a shift toward RWD collection earlier in the clinical
trial process (as early as Phase II). Gone are the days
of beginning to collect RWD after a product has been
approved; early data generation is especially critical
in today’s market, given how saturated the healthcare
marketplace is for many types of medications.
New medicines in development undergo intense
evaluation prior to regulatory approval. Instead of
just being evaluated by the risk-benefit ratio of the
medication, regulators and payers are increasingly
assessing whether a new drug adds significant benefit
over an established standard of care. In order to
eventually achieve favorable placement on a payer’s
formulary, the price of the product must also be
justified by a correlated positive health outcome for
the patient. Evidence collected in real-life settings are
the only available source that can accurately answer
these questions.
As data collection and storage capabilities continue
to advance, RWD are less limited, which helps HEOR
teams to fully capitalize on RWD to prove product
value. In our technologically advanced world, we sit on
the cusp of a golden era of access to digitized RWD,
optimized by the adoption of electronic health records
and the integration of clinical and biological information
with other data, including registries, treatment
databases, mobile platforms, social media and patient
surveys (to name a few). Optimal use of RWD can
provide a greater understanding of medicines and
diseases, the impact on treatment standards and the
long-term impact of medications on patient health.
RWD Intensifying Across Product Lifecycle
* IMS Health, “Real-World Insights. Real-World Impact.” www.imshealth.com
3. Illustrated by the graph, more evidence is required
at every stage of product development growth and
maturity than ever before. Anonymized patient-
level data are becoming more accessible around the
globe, allowing RWD to become the new currency in
healthcare. The accessibility of data at all stages of
development allow healthcare decisions to be more
accurately informed by millions of patient experiences
across the globe, all in real world settings. Using
RWD, patients, healthcare providers and payers can
better assess the value of treatments and services
based on actual patient health outcomes and the total
cost of care.
Who is utilizing RWD today?
• Clinical development teams: to design clinical
trials based on actual treatment practices
• HEOR, epidemiology and drug safety researchers:
to gain faster insights from richer patient
databases
• Market access teams: to inform payers and
HTAs with evidence of their product’s value
• Brand and franchise teams: to understand their
markets, differentiate their products and improve
stakeholder engagement
In addition, the “patient as a partner” is a new
stakeholder that now must be critically engaged
throughout the whole product lifecycle. Increasingly,
payers are expressing that the voice of the patient
will be a factor in decision making to help provide
evidence that proves a superior product. As a
result, bio/pharma teams are learning how patients
can aid with the development of clinical tools and
measurements through patient reported outcomes
and voluntary patient data sharing.
As one can see, the benefits of fully utilizing RWD
are immense for healthcare costs and patient health
across the bio/pharmaceutical industry. However,
RWD are also incredibly challenging. The data sources
vary widely in quality, reliability and consistency,
which requires a certain level of calculated risk when
evaluating medicines. Additionally, the industry lacks
policy solutions and full stakeholder cooperation.
This is what
they have
to say…
We had the opportunity to sit down with industry’s foremost RWD thought leaders. We asked them a variety of
questions on the topic to better understand how bio/pharmaceutical teams can collaborate and capture data in an
aggregated form to continue to improve the value of products in development with real world, real-time data.
4. In your experience, how is it beneficial to
capitalize upon real world evidence to prove
superior product value?
In a value-based healthcare system demonstrating real
world outcomes and comparative value will become
the most important factor in driving market adoption.
Without that, potentially great products will never leave
the shelves. This is already becoming true for some
payers, but will become much more true for providers
who take on population risk.
Can you name two barriers of using real world
data and the strategies to overcome them?
Much real world data is often not being used for
the purposes that it was intended, (e.g. billing data
being used for outcomes); it’s not longitudinal at the
patient level; it’s not representative of the population
you want to understand and it’s not complete. The key
strategies to overcoming these limitations are a greater
focus on upfront planning for what is needed; what data
are available and how can those data be supplemented
with other data sources or primary collection to close
the gaps.
The industry has called real world data “the new
currency in healthcare.” Do you agree with this
statement and if so, why or why not?
What works in the real world is what every stakeholder,
patient, provider, payer and manufacturer needs to
know. In that way, I agree. But much of what we call
real world data is not the right data to tell us what
works. So, I’d qualify the statement with the “right”
real world data.
Specifically in what ways do you anticipate big
data value will increasingly become a driving
force for a more sustainable healthcare system?
The greatest opportunity for big data is that it will help
healthcare systems make better decisions. Using novel
big data capabilities, we are quickly becoming able to
answer questions like which patient is most likely to be
at risk for a fall, infection or readmission; who is most
likely to benefit from a diabetes treatment program;
which patient needs another scan to rule out a cancer
or which patients should have an end of life discussion.
These are important questions in a healthcare system
where resources are inevitably constrained.
ABOUT DR. GLIKLICH...
Dr. Rich Gliklich serves as an Executive in Residence at General Catalyst Partners
where he leverages his operational experience and domain expertise to invest
in and support businesses in the healthcare and life sciences industries. Prior to
joining General Catalyst, Dr. Gliklich was President of the Outcome division of
Quintiles. Prior to Quintiles, he was Founder, CEO and Chairman of Outcome, a
health information and services company that served more than 3000 healthcare
organizations and a majority of the global top 30 lifesciences companies. He led
Outcome from its start as a spin-off from his Harvard affiliated research laboratory
in 1998 through its acquisition by Quintiles in October 2011. In addition to his
experience as an entrepreneur and executive, Dr. Gliklich is well known in the
areas of outcomes and analytics. He is senior editor of the landmark publication
by the U.S. Agency for Healthcare Research and Quality (AHRQ) handbook
“Registries for Evaluating Patient Outcomes: A User’s Guide,” as well as PI for the
national Outcome Measures Framework project and has led several key national
and international efforts focused on evaluating the safety, effectiveness, value
and quality of healthcare. Dr. Gliklich is also co-founder of Cyft, a revolutionary
healthcare prediction platform. He is a graduate of Yale University and Harvard
Medical School. In addition, he is also a surgeon and a chaired Professor at
Harvard Medical School.
Rich Gliklich, M.D.,
Leffenfeld Professor, part time, Harvard Medical School;
XIR, General Catalyst Partner
5. In your opinion, what is most important to
working with payers, in regards to partnering
around value demonstration, shared risk and
pay for performance?
We should work with them to agree to a value definition
that makes sense in the quest to improve patient health,
to make sure we are actually sharing the risk and it
not only falls on the manufacturer and to have a clear
definition of what is meant by performance.
Why do you think it is important to incorporate
patient perceptions and experiences into clinical
research? How does patient satisfaction lead to
better HEOR evidence?
Let me start with a quote from Professor Michael E.
Porter at Harvard: “Today, value tends to be defined
by what can be easily measured in our current
misaligned structure, rather than what actually
matters for patients.” As the patient is the ultimate
end user of healthcare, I think their viewpoint should
be included in the equation. Patient satisfaction
complements other data to better understand the
value based on HEOR evidence.
Can you define value and discuss the impact on
collecting evidence for value generation?
Value to me is defined as outcomes divided by costs.
The challenge lies in the composition of the numerator
“outcomes.” We have a tendency to only define
outcomes as the result of efficacy, safety, tolerability
and maybe add a quality component to include the
patient perspective. The challenge is to determine
the right proportion of each bucket and also question
whether this really represents the patient perspective.
In your opinion, how do early access programs
support or complement data gleaned from
clinical trials?
They may support the notion of effectiveness of an
intervention in the real world, although somewhat
biased towards the patients that are in urgent need of
the treatment as opposed to patients that will receive
the innovation post launch.
Bjorn Bolinder,
Group Director, U.S. Health Services, Oncology,
Bristol-Myers Squibb
ABOUT MR. BOLINDER...
Bjorn Bolinder is an economist with 23 years of experience
in the pharmaceutical industry, of which 18 years he dedicated
to the health outcomes area, with responsibilities at affiliate,
US and global level. Mr. Bolinder has broad therapeutic area
knowledge, with the majority of experience in the diabetes,
cardiovascular, CNS and Oncology areas.
6. Why do you think it is important to incorporate
patient perceptions and experiences into clinical
research? How does patient satisfaction lead to
better HEOR evidence?
Patients are the central focus of our research. It is
critical for us to listen to them beyond the data points
we are working to collect. Patient feedback and insights
are changing the way we develop protocols, recruitment
and retention strategies and develop positive
experiences in our research programs.
The industry has called real world data “the new
currency in healthcare.” Do you agree with this
statement and if so, why or why not?
Real world data is impacting the evidence we generate
in research programs and late phase research
stakeholders, including regulators, payers, providers and
patients, want this data. This trend also appears to be
expanding as we are now seeing real world evidence
generation included in clinical development and patient
support programs.
How does your organization currently capitalize
on real world evidence to prove superior product
value? What are your biggest challenges in
reporting evidence to stakeholders?
In order for Quintiles to improve our customers’
probability of success we are focused on combining
our scientific and patient engagement expertise,
proven global processes and operations and Quintiles
Infosario technology together for real world and late
phase research studies. This approach is enabling our
customers to prove their products’ value and generate
evidence for their stakeholders in novel ways that
enhance patient engagement, retention and compliance.
John Reites,
Senior Director, Health Engagement and Communications,
Quintiles
ABOUT MR. REITES...
John Reites is the Head of Product and Strategy for Quintiles Health
Engagement & Communications. Mr. Reites is responsible for developing
engagement innovations and patient-centered strategies to improve Quintiles’
customer’s probability of success. Mr. Reites is a speaker and author of various
conference presentations, industry articles and peer-reviewed publications,
providing subject matter expertise for direct-to-patient research, patient
recruitment, patient communities, direct-to-patient late phase research and
digital health approaches.
Mr. Reites has over 12 years global research experience conducting more
than 150 programs spanning Phase I – IV studies and Commercial programs.
His experience includes health research programs conducted in 52 countries
reaching over 5,000 providers and 38,000 patients across various therapeutic
areas. He provided oversight and development support for Quintiles patient
communities of >3.4 million members in 7 countries including MediGuard.org
and ClinicalResearch.com. He has also lead the development and execution
of direct-to-patient research programs integrating ePRO, medical chart data,
at-home labs and digital health devices.
Prior to his current role, he developed novel study design approaches
and new business units from start-up to global scale within Quintiles.
He has held multiple leadership positions within Clinical Operations,
Global Project Management and Late Phase Research during his tenure.
7. The industry has called real world data “the new
currency in healthcare.” Do you agree with this
statement and if so, why or why not?
Real world data will be the new currency — but what
will it be worth to each organization? Unfortunately, it
starts off as exhaust. The majority of real world data
is captured as unstructured free text and the available
structured data can be up to 80% inaccurate. The
organizations that will thrive in a value-based economy
will be those that are able to transform this byproduct of
care into healthcare organizations’ most valuable asset.
Specifically, in what ways do you anticipate big
data value will increasingly become a driving
force for a more sustainable healthcare system?
In a value-based world, it becomes critical to answer the
three fundamental questions of improvement: 1) what
are we doing; 2) to whom are we doing it and; 3) is it
working? The ability to begin to understand this is
a prerequisite to progress and yet there’s not a
healthcare delivery organization that can answer
all three. If the term “big data” makes it popular to
begin actually measuring what we’re doing, then I’m
a huge big data advocate.
In your opinion, what are the most eminent ways
patient considerations can impact: 1) trail design;
2) study participation; and 3) payer decisions?
Healthcare is transitioning from a system that values
outputs to a system that values outcomes. Outputs
are finite transactions. Outcomes are product of a
patients experience with each of healthcare’s dozens
of stakeholders. In an outcomes-based model, the
patient “owns” the outcome and therefore becomes the
most valuable of all stakeholders. Organizations that
recognize this and act accordingly will thrive.
Leonard D’Avolio, Ph.D.,
Director of Informatics, Ariadne Labs;
Assistant Professor, Harvard Medical School;
Co-Founder, Cyft
ABOUT DR. D’AVOLIO...
Leonard D’Avolio, Ph.D., is the Director of the Informatics and Measurement Platform
at Ariadne Labs. In this role, Dr. D’Avolio works across all project teams to design and
develop infrastructure and methods that turn raw data into actionable knowledge
and ideas into scalable solutions. This includes building programs and infrastructure,
supporting an international project data capture and analysis, and developing
software capable of detecting novel relationships across heterogeneous data sources.
Prior to his role at Ariadne Labs, he was head of a team he founded within the
Department of Veterans Affairs. There he was responsible for the development of
national programs such as the development of the VA’s genomic science infrastructure
and the first randomized controlled trial embedded entirely within an electronic
medical record system. He also developed several software programs to make data
mining and natural language processing accessible to non-technical end users. He has
also been an investigator on several projects funded by the Agency for Healthcare
Research and Quality (AHRQ), Department of Defense, and the National Institutes
of Health (NIH) focused on using information technology to improve the conduct of
clinical science and care.
Dr. D’Avolio earned his Master’s Degree in Information Technology from the McCallum
School of Business at Bentley University and his Ph.D., which brought him expertise
in data mining and natural language processing, from the University of California,
Los Angeles. Prior to obtaining his graduate degrees he spent several years in the
software industry.
8. In your opinion, how do early access programs
support or complement data gleaned from
clinical trials?
It is important to keep in mind that the primary
purpose for an Expanded Access Program (or early
access program) is to provide access to investigational
products to patients with serious or life threatening
conditions with limited alternative treatment options.
These programs are often purposefully not designed
with objectives to compliment the clinical trial data.
Manufacturers should carefully consider the collection
of efficacy data in EAP design given the potential
unintended consequences of such an approach.
Adverse drug reactions must of course be collected
and if new safety signals present during the EAP,
there could be implications to the marketing
application that is presumably in process.
In the event that a post marketing patient assistance
program/HUB is planned, the EAP could present an
opportunity to collect information that will in turn
inform the optimal design of such a program.
In your opinion, what are the most eminent ways
patient considerations can impact: 1) trail design;
2) study participation; and 3) payer decisions?
A patient-focused approach to trial design is essential
to the success of the initiative to collect real world
evidence. The study must accommodate the patient’s
“usual care” experience, be conducive to unbiased
evidence capture, and allow for optimal patient
participation retention rates.
Aaron Berger,
Project Director,
UBC – An Express Scripts Company
ABOUT MR. BERGER...
Mr. Berger joined the UBC team in 2003 and has since served in a range of
diverse roles leveraging UBC global capabilities supporting the execution of per
and post marketing product development programs, Expanded Access Program
programs, REMS implementation, and Pharmacovigilance service delivery.
Mr. Berger started his career at UBC as a CRA followed by a move to Project
Management and now serves as a Project Director.
As a Project Director, Mr. Berger provides support and direction to UBC project
teams conducting late stage studies, EAPs, registries and observational studies.
He oversees multi-disciplinary project teams and assists them in meeting project
timelines according to UBC quality standards and study contract specifications.
He is also responsible for operations, budget oversight, and regulatory
requirements throughout the duration of the project. Mr. Berger plays an integral
role in the development and implementation of integrated solutions to the
challenges manufacturers face as products travel through the development life
cycle and commercialization. Mr. Berger received his Bachelor’s degree from the
University of Kansas and holds the Project Management Professional credential.
9. In your experience, how is it beneficial to
capitalize upon real world evidence to prove
superior product value?
At the conclusion of Phase III, important questions
remain regarding the value of the product in the
marketplace. There is significant value in collecting
real world evidence to prove superior product value as
treatment and reimbursement decisions may depend
on showing effectiveness and safety in usual care. The
ability to describe the burden of illness and economic,
clinical and humanistic utility of a product in patients in
usual care settings can demonstrate superior product
value. It is important to first understand the payer
perspective to determine what value messages matter
and then determine the types of studies that should be
conducted to generate real world evidence.
In your opinion, how do early access programs
support or complement data gleaned from
clinical trials?
Early access programs (i.e., Compassionate Use,
Expanded Access) which allow patients with no
other appropriate treatment options to receive an
investigational agent prior to market uptake, can provide
valuable insight into future real world use. While patients
in an early access program tend to be considered
“sicker” than patients in clinical trials, evaluation of safety
profiles (e.g., new safety signals) and effectiveness (e.g.,
progression free survival [PFS] and overall survival [OS])
can successfully be undertaken to provide insights into
PFS and OS as well as adverse events that may not have
been identified in the more homogeneous clinical trial
populations. Early access programs can also supplement
data collected in clinical trials since they may include
patient populations that are not be fully represented in
the clinical trials (physicians not included as investigators
and patients excluded from clinical trials).
Can you name two barriers of using real world
data and the strategies to overcome them?
Two barriers that we normally face when dealing with
real world data are data quality and selection bias.
Achieving robust data quality and completeness can be
challenging in real world studies that do not interfere with
usual medical care. In retrospective chart review studies,
patient medical chart documentation may be conflicting,
missing or incomplete. In prospective studies, patient
survey data may be missing or incomplete depending
on the mode, length and frequency of administration.
Selection bias is an inherent risk wherein the patient
population studied may not represent the target
population of interest thereby lowering the value of data
collected. Patients are randomly assigned to a treatment
during a clinical trial in order to minimize bias between
the groups being studied. In “real world” studies such as
observational cohort studies, physicians prescribe the
drug as per usual care, which may be outside of labeling,
i.e., the patient characteristics, dosing or indication may
differ from the approved label and the patient population
that had been studied in the clinical trials.
In order to overcome these barriers, adequate planning
must occur when designing the study. Even in the most
hurried of situations, adequate planning is imperative.
Most suitable data sources/study designs to answer
the research questions need to be determined and the
optimal design selected for ensuring data output will
be of the highest quality possible. Obtaining study data
from electronic medical records (EMRs) rather than
paper records when possible and using an electronic
data capture (EDC) system to facilitate data cleaning at
time of entry are paramount. Ensuring the data collection
tool/PROs used is as simple as possible to increase
compliance and reduce burden on the site and the patient
is also crucial. Having sites identify a sampling frame of
the total potential eligible denominator population of
interest and having an EDC system determine patient
enrollment by random or sequential sampling will ensure
that there is more oversight on patient sampling and a
systematic process implemented similarly across all sites.
While we cannot eliminate selection bias, collecting data
at enrollment to characterize the patient population can
allow for stratification and other analyses post-hoc to
possibly control for confounding.
Dara Stein, MSc,
Senior Research Scientist Value Demonstration Team,
UBC – An Express Scripts Company
ABOUT MS. STEIN...
Dara Stein, MSc, is a Senior Research Scientist for the Value Demonstration team within the Safety,
Epidemiology, Registries and Risk Management (SERRM) Group, at United BioSource Corporation (UBC)
in Montreal, Quebec. Ms. Stein holds a BSc in Human Kinetics from the University of Ottawa, and a MSc
in Human Nutrition from McGill University. Prior to joining UBC, she worked as a Research Associate in
the Division of Clinical Epidemiology of the Research Institute of the McGill University Health Centre.
Ms. Stein has experience designing and managing health services observational research studies
including retrospective chart reviews and prospective and cross-sectional cohort studies. Ms. Stein has
experience with various therapeutic areas including oncology, cardiovascular diseases, intensive care,
peritonitis, hepatitis, and acute bleeding events. The research aims of studies Ms. Stein has worked on
range from drug utilization, safety, burden of illness, and treatment patterns and resource utilization
including direct and indirect cost of healthcare. Ms. Stein has experience with the design of over 15
multi-national chart review studies in various therapeutic areas. Ms. Stein has a keen interest in disease
prevention and management as well as burden of illness evaluations.
10. What are five best practices for implementing
organizational change to effectively generate
value evidence?
1. Ability to develop strategic context for
value evidence;
2. Expertise in the design, execution and interpretation
of studies that provide such evidence;
3. An infrastructure that supports scientifically
rigorous research;
4. A prior intention to disclose results of research;
5. Direct application of such research to
reimbursement dossiers.
In your opinion, what is most important to
working with payers, in regards to partnering
around value demonstration, shared risk and
pay for performance?
Assessment of payer needs to properly value innovation
and willingness to generate evidence of value in the
payer’s environment. All else (e.g. subsequent risk
sharing, etc.) derives from this effort.
The industry has called real world data “the new
currency in healthcare.” Do you agree with this
statement and if so, why or why not?
It’s new in the sense that real world evidence of value
has gained increasing recognition as important to the
proper assessment of an innovation. It’s also new in the
sense that informatics technology has advanced to the
stage where real world data generation is both feasible
and scientifically rigorous.
When hiring quality evidence teams, what are
the biggest hurdles in regards to proper training?
The demand for the competencies in this area is
enormous and still growing. Therefore, competition
for available talent is fierce. Potential employers are
reluctant to provide on the job training since the need
for these competencies is urgent.
How would you define the role of HEOR teams
in generating real world evidence, PPO, clinical
trials and HEOR data within a company’s internal
organizational structure?
HEOR staff are contributors to market access strategy
and they derive the evidence of value, both adjunctive
to clinical trials and embedded within trials
(e.g., costs, resource use, clinical outcomes and
patient reported outcomes).
In your opinion, what are the most eminent ways
patient considerations can impact: 1) trail design;
2) study participation; and 3) payer decisions?
1. Trial design: trials capture measures that are relevant
to patients;
2. Study participation: trials enroll patient with a high
unmet need and the promise of innovative therapies
to address those needs;
3. Payer decisions: payers are incentivized to
consider patient-specific measures in their
reimbursement deliberations.
Gilbert J. L’Italien, Ph.D.,
Senior Director, Health Economics & Outcomes Research, Biogen;
Adjunct Assistant Professor, Yale University School of Medicine
ABOUT DR. L’ITALIEN...
Prior to his current HEOR role at Biogen, Dr. L’Italien has held roles of increasing responsibility within Bristol
Myers Squibb Company (Executive Director) across multiple disease areas. Dr. L’Italien has led BMS initiatives
for early dialogue with HTA, ranging from NICE consultation, EUnetHTA early dialogue and Adaptive Licensing
(EMA-HTA) pilots. In 2003, he won the prestigious BMS Ondetti & Cushman Award for development of a
database organization unique to the industry. Dr. L’Italien played a leading role in defining the metabolic
value proposition for the antipsychotic drug, Abilify, and most recently led an initiative for CHMP qualification
of imaging biomarkers in Alzheimer’s disease. In 2014, he led the development of an economic model which
supported a favorable decision by the U.S. Preventative Task Force (USPTF) on reimbursement for universal
screening of HCV. Prior to his Biogen and BMS/Yale roles, Dr. L’Italien was Director of Vascular Research at
Massachusetts General Hospital and held a faculty appointment at Harvard Medical School.
Dr. L’Italien has further participated in advisory committees for several government institutions such as the
Agency for Health Care Research and Quality (AHRQ), The CDC Foundation Viral Hepatitis Action Coalition
(VHAC), the Multiple Sclerosis Outcome Assessments Consortium (MSOAC), the Alzheimer’s Disease
Technical Working Group, Green Park Collaborative, and the Merit Review Committee for the Patient
Centered Outcomes Research Institute (PCORI).
His major research interests include methodological enhancements to comparative effectiveness research,
the application of HEOR to address regulatory, clinical development and reimbursement challenges. He is
also a committed philanthropist and volunteer. Of note, he recently designed and led a 2-year field public
health intervention that effectively treated 3,000 indigenous adults and children in rural Mexico to
ameliorate the burden of soil transmitted helminthes (STH).
11. Medical Communications and
Information Summit
November 5-6, 2015
San Francisco, CA
REAL WORLD EVIDENCE
Your Next Opportunity to Learn More About
Expanded Access
Programs
July 22-23, 2015
Washington, DC
Benefit-Risk Assessment and
Decision Making Summit
August 6-7, 2015
Alexandria, PA
Evidence for
Value-Based Programs
August 11-12, 2015
Boston, MA
Oncology Endpoints &
QOL Assessments
November 2-3, 2015
Philadelphia, PA
Reimbursement and Access
August 12-13, 2015
Philadelphia, PA
10th Annual Value Based
Oncology Management
October 14-15, 2015
Chicago, IL
12. Life Sciences
CBI, UBM Life Sciences, educates the life sciences industry
through dynamic conferences, interactive online content
and professional networking engagements. In the quest
to be the industry leader in professional development,
CBI is dedicated to persistent market research, analysis
and optimization to supply your team with the education
and tools needed to succeed in the ever-changing
pharmaceutical, biotech and medical device industries.
Our vision is a framework that guides our personal
and professional dedication to market education and
innovation. The pillars of this framework are:
• People: Strive to educate and inspire our
respected delegates and partners
• Portfolio: Develop a robust portfolio of offerings that
provide a myriad of solutions to industry challenges
• Partnership: Build meaningful relationships
with sponsors and partners by providing
unparalleled business development opportunities
• Performance: Always be lean, agile and scrupulous
in the development of our products and every other
facet of our business
Our values guide the way we conduct ourselves
in everything we do.
• Thought Leadership: Create and share valuable
content that provides insight into main topics of
interest for the life sciences community.
• Collaboration: Utilize established relationships
with industry leaders to identify market and
patient needs, and produce the strongest channels
for education and professional development.
• Agility: Respond swiftly to market shifts and
trends to continuously be ahead of the curve.
• Trust: Build sustainable, long-term relationships
with the life sciences community to establish CBI
as their trusted partner.
• Passion: Strive for excellence, remembering
that the connections we foster may ultimately
play a role in helping someone in need.