COVID-19: A GLOBAL CRISIS - ISPOR
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MARCH/APRIL 2020 VOL. 6, NO. 2
A magazine for the global HEOR community.
COVID-19: CRISIS A GLOBAL
How Can the HEOR Community Contribute?
5 A CALL TO ACTION FOR THE
HEOR COMMUNITY
14 IFOR
NCENTIVIZING FLU SHOTS
THE ELDERLY
18 AFROM
PERSONAL ACCOUNT
WUHAN, CHINA
A GLOBAL PANDEMIC—
39 TWO GLOBAL PERSPECTIVES
< ADVERTISEMENT >
MARCH/APRIL 2020
VOL. 6, NO. 2 TABLE OF CONTENTS
EDITOR-IN-CHIEF FROM THE EDITOR
David Thompson, PhD
4 ISPOR in the Time of Corona
ASSOCIATE EDITORS
Murtuza Bharmal, PhD, MS ISPOR CENTRAL
Benjamin Craig, PhD 5 From the President
7 ISPOR Speaks
EDITORIAL ADVISORY BOARD
8 HEOR News
Soraya Azmi, MBBS, MPH
(United States) 10 Research Roundup
Agnes Benedict, MSc, MA 12 Conferences
(United Kingdom)
14 From the Journals
Karin Groothuis-Oudshoorn, PhD
(The Netherlands) 15 From the Region
Yvonne Lee YL, PhD, MPH
(Singapore)
SPECIAL FEATURE
Martin Marciniak, PhD
(United States) 18 The COVID-19 Virus in Wuhan, China: A Personal Story
George Papadopoulos
(Australia) FEATURE
Louise Parmenter, PhD, MSc 20 The State of Health Technology Assessment
(United Kingdom)
Marisa Santos, PhD, MD
21 By the Numbers
(Brazil)
Stephen W. Schondelmeyer, PhD HEOR ARTICLES
(United States)
27 Are Missing Data Properly Accounted for in Health Economics and
Mondher Toumi, MD, PhD, MSc Outcomes Research?
(France)
31 stimating Comparative Effectiveness When Patients Are Switching
E
Treatments: A Real-World Challenge
34 Call in the PROs: Giving Credence to the Patient’s Perspective in
The mission of Value & Outcomes
Spotlight is to foster dialogue Healthcare Decision Making
within the global health
economics and outcomes Q&A
research (HEOR) community
by reviewing the impact of 39 COVID-19: A Global Pandemic—Two Global Perspectives
HEOR methodologies on health
policy and healthcare delivery
to ultimately improve decision
making for health globally.
Value & Outcomes Spotlight March/April 2020 | 3
EDITORIAL STAFF FROM THE EDITOR
Lyn Beamesderfer
Director, Publications
O
lbeamesderfer@ispor.org
ur whole world has been turned upside down by the novel coronavirus, SARS-CoV-2,
Margaret M. Rafferty
Manager, Publications and the exponential increases in cases and fatalities attributable to COVID-19, the
mrafferty@ispor.org disease to which it gives rise. No person, family, community, or country is immune,
Jennifer A. Brandt and at the time of writing, it is clear that things will get far worse before they will even begin
Editorial Assistant to get better—for most countries, there is currently no light at the end of the tunnel.
jbrandt@ispor.org
Although the economic impact of the COVID-19 pandemic promises to be immense, many
ISPOR CORPORATE OFFICERS of us in the ISPOR community are fortunate enough to be able to maintain our current
Nancy S. Berg employment and carry out our business relationships virtually, interacting with one
Chief Executive Officer/ another via videoconferencing technologies and exchanging work products through email.
Executive Director In this regard, we should consider ourselves lucky. But it’s a decidedly unlucky situation
Sue Capon for those of us who were looking forward to convening this May in Orlando, Florida for
Chief Operating Officer ISPOR 2020, which of course has been cancelled. All of us will have to do our best to make
Richard J. Willke, PhD “Virtual ISPOR 2020” a productive and fruitful online event.
Chief Science Officer
Lucinda Orsini, DPM, MPH Value & Outcomes Spotlight prides itself on being agile in the face of rapidly changing
Associate Chief Science Officer circumstances and we have put that into practice in the current issue. Our planned theme
Betsy Lane is the state of health technology assessment (HTA), and we have the usual feature article
Director and Chief Marketing providing a global overview of HTA practices, with an accompanying By the Numbers
& Communications Officer piece highlighting some interesting comparative statistics across countries. We also have
a variety of HEOR articles of interest, encompassing such topics as statistical handling
ISPOR HEADQUARTERS
of missing data in outcomes research, methods for addressing treatment switching in
505 Lawrence Square Blvd, S
comparative effectiveness research, and the use of patient-reported outcomes to give
Lawrenceville, NJ 08648
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voice to the patient perspective in HTA.
Fax: 609-586-4982
info@ispor.org
In addition to all that, we have scrambled to assemble a wide range of content specific
www.ispor.org to COVID-19. This begins with an insightful essay by ISPOR President Nancy Devlin, PhD
culminating in a call to action for those of us in the HEOR community to respond to this
VALUE & OUTCOMES SPOTLIGHT global crisis. We also have a first-person perspective from Siying Zou, PhD, who lives and
PUBLISHING, SUBSCRIPTION, AND works in the United States but grew up in Wuhan, China, where the virus originated. She
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shares the anguish of hearing the news of her mother becoming ill with COVID-19 and
Print: ISSN 2375-866X details the difficult patient journey that has affected her entire family. Finally, our Q&A
Online: ISSN 2375-8678
section includes 2 interviews of interest, one with Christian Lindmeier, a spokesperson for
USPS: 019121
Published bi-monthly by:
the World Health Organization (WHO), and the other with Mirjam Kretzschmar, PhD, an
ISPOR infectious disease modeler with the University Medical Centre Utrecht in The Netherlands
505 Lawrence Square Blvd, S
and a member of the ISPOR Modeling Task Force.
Lawrenceville, NJ 08648 USA
Periodicals Postage paid at All of us at Value & Outcomes Spotlight
Annapolis, MD 21401 encourage you to stay safe, continue to practice
and at additional mailing offices.
POSTMASTER: Send address changes to:
social distancing, and look to provide help
Value & Outcomes Spotlight where help is needed as we “pull together by
505 Lawrence Square Blvd, S
staying apart” during these difficult times.
Lawrenceville, NJ 08648 USA
Direct photocopy permission and reprint
requests to Director, Publications.
Sincerely,
© 2020 ISPOR—The professional society for health
economics and outcomes research.
While Value & Outcomes Spotlight is designed
to provide accurate information regarding the David Thompson, PhD
subject matters covered, the views, opinions, and Editor-in-Chief,
recommendations expressed, are those of the
contributors and not of the International Society for
Value & Outcomes Spotlight
Pharmacoeconomics and Outcomes Research (ISPOR).
ISPOR CENTRAL
FROM THE PRESIDENT
COVID-19—A Call to Action for Health Economics and
Outcomes Researchers
Nancy J. Devlin, PhD, ISPOR President (2019-2020), Centre for Health Policy,
University of Melbourne, Melbourne, Australia
I am writing this from New Zealand,
where a state of emergency has
been declared and I am experiencing
the COVID-19 pandemic will (and
should) lead us to radically rethink
our world. It has revealed failures in
my second week of lockdown. You political leadership. It has highlighted
will no doubt also be adapting to major weaknesses in public health and
the new personal, family, social, and healthcare systems. It has brought to the
work circumstances in which we find fore fundamental questions about the
ourselves. Having succeeded in the first trade-offs society is prepared to make
challenge—figuring out good ways to between population health and economic
work from home—the new challenge is activity, while also reminding us about the
how to cope with uncertainty about how interconnectedness between health and
long the pandemic will remain at crisis wealth. There are also questions about by government, healthcare systems,
levels, and what the implications will be behavioral responses to the crisis and businesses, and individuals generate
for our work and our families. how to strike the right balance between important questions that HEOR can help
to address.
Economic damage from lockdown measures will not be equally The governments of the world have
taken very different strategies in reacting
distributed—it is likely the misery from this will fall disproportionately to and managing the pandemic, creating
on the poor and those in secondary labor markets, including “gig a kind of wholesale natural experiment
about restrictions on travel, gatherings,
economy” workers, who are pushed into poverty. continuation of businesses and schools,
and self-isolation or quarantine.
I am amazed by the spirit and resilience encouraging and compelling individuals
of my colleagues in Australia and around to behave in a manner consistent with Governments have had to make rapid-
the world. And I am full of admiration collective interests. fire judgments about the willingness
for colleagues with young children; to sustain economic harm to contain
with childcare and schools closed, they ISPOR is the leading international body the health of people, in the presence
somehow continue to work, appearing for health economics and outcomes of considerable uncertainty as to the
on videoconferences with partners and research (HEOR). As a community of effectiveness of those strategies, the
children in the background, all vying for 20,000+ HEOR professionals worldwide, period of time for which they will be
use of laptops and work spaces. we have a responsibility to consider required to be in place in order to
the implications of the pandemic for sustain that effectiveness, and their real
I did not imagine when I began my term our HEOR scientific priorities. We need societal cost. Evaluation to understand
as ISPOR President that one of the to ensure HEOR evidence informs the differences in effects and costs
decisions the Board of Directors would healthcare delivery and policy in and of these measures will be difficult but
have to make this year was cancelling the following the pandemic—the lessons that essential to inform economic and health
ISPOR 2020 conference in Orlando (the we can learn from this will be invaluable policy in the post–COVID-19 world. Yet
first time in ISPOR’s 25-year existence in planning for future, potentially much these evaluations pose methodological
that it has cancelled a major event). This more fatal, crises. challenges: the options are huge in scale
was not a decision taken lightly, and and far from “marginal” changes; the
considerable effort went into assessing Identifying HEOR scientific priorities perspective from which to evaluate them
the implications. But ultimately there was for COVID-19 research necessarily extends beyond the narrow
no option: ISPOR is committed to global Which ethics committee signed off approval “healthcare perspective”; and what
health and we take seriously our duty of for this worldwide study into comparative decision rule do we use to judge value
care for the health and well-being of our public healthcare systems? for money in such a context?
members and stakeholders. —Professor Jo Wolff, Oxford University,
United Kingdom The availability of data is a limiting factor
Beyond changing our immediate in undertaking research at present.
personal and working circumstances, COVID-19 and the responses to it Differences in the availability and use
Value & Outcomes Spotlight March/April 2020 | 5
ISPOR CENTRAL
of testing confounds the interpretation (eg, mental health problems, cancers), program to take the place of the in-
of and comparisons between rates of and future outcomes will be worse, person event in Orlando. In addition to
infection in the populations of different due to delayed diagnosis and missed the plenaries and panel sessions which
countries, and the rates of mortality treatment opportunities. had already been planned, we will add
among those infected. In some countries, an online preconference session where
testing and tracing was maximized; in What are the implications for the health HEOR aspects of the COVID-19 situation
other countries, access to testing was and quality of life of those for whom and their implications for future research
severely restricted, further complicating this period of enforced isolation has priorities will be discussed. I encourage
an understanding of transmission, disrupted the delivery of essential all ISPOR members to log in, tune in,
prevalence of illness, and the relative services, such as those with mental and engage. We welcome your ideas
effectiveness of strategies to limit these. health problems and those with about how ISPOR can help to mobilize
What was the optimal strategy? And how disabilities? What are the effects of HEOR efforts now and over the
much avoidable harm did the world’s isolation and lockdown on the quality coming year to produce better data,
nations’ deviations from that cause? of life of those without pre-existing better evidence, and real solutions.
mental health problems, but who are It is important we learn as much as we
now struggling with anxiety, depression, can from this crisis—and the enormous
COVID-19 and the and uncertainty? How has the lockdown
affected vulnerable populations, including
human and economic cost that has and
is still to be incurred—to better prepare
responses to it by elderly people in residential care? us for the future.
government, healthcare How are prioritization decisions being At the start of the year, ISPOR embarked
systems, businesses, made when demand exceeds capacity on an important new project to establish
and individuals generate in healthcare systems during the peaks
of the COVID-19 crisis—particularly
HEOR research priorities. That project
has made excellent progress and is well
important questions that in systems where “rationing” has not positioned to incorporate new research
HEOR can help to address. previously been accepted? Who is
making these decisions—are they being
topics relating to COVID-19. ISPOR
leaders will meet in the coming months
made consistently—and on what basis? to establish ISPOR’s HEOR science
Economic damage from lockdown Who is benefiting and what is being priorities for the next 5 years, informed
measures will not be equally sacrificed? by results of that work.
distributed—it is likely the misery
from this will fall disproportionately The pandemic has also revealed ISPOR and its member groups
on the poor and those in secondary different levels of preparedness among will organize special webinars and
labor markets, including “gig economy” healthcare systems: lack of personal discussions around COVID-19 and
workers, who are pushed into poverty. protective equipment (PPE) for frontline will continue to support its members
Given what we know about the staff may itself have led to a considerable with the latest information on this
socioeconomic determinants of health, avoidable burden of ill health. What rapidly changing situation. ISPOR is
the health consequences of these are the cost and benefits of improved committed to its mission and to serving
economic measures will therefore also PPE? What are the supply or other its members and the broader healthcare
be unequally distributed—and will have constraints that have led to PPE not audience during the extraordinary global
implications for health well beyond the being available for frontline clinical staff healthcare crisis that COVID-19 presents.
immediate crisis. We need evidence on during COVID-19? More broadly, what The field of HEOR has never been more
that, so effective policy measures can be is the appropriate balance between important.
targeted. spare capacity and technical efficiency in
healthcare provision? Wherever you are, I hope you, your
Meanwhile, what is happening to supply, family, and your community stay safe and
demand, and access to other health
services during the pandemic in the
This is far from an exhaustive account
of the kinds of questions HEOR could
•
stay well. Be kind to each other.
world’s healthcare systems? What health usefully address. ISPOR now has a
needs and healthcare utilization have new and important role to facilitate us
been deferred, and what will be the working together collectively as an HEOR
consequences of that in the post-COVID community to establish the research
world? How have morbidity and mortality priorities. And each of us, as ISPOR
from non-COVID infectious diseases members, should consider how to pivot
(and from noninfectious causes) been our research agendas, to use our skills to
affected by isolation and lockdowns? address the new and emerging research
Presumably some will have been avoided needs.
altogether (eg, car crash fatalities), and
some may worsen (eg, domestic abuse ISPOR’s role
fatalities and injuries, alcoholism), while As I write, ISPOR is working hard to
other health problems will be “stored up” develop its first online conference
6 | March/April 2020 Value & Outcomes Spotlight
ISPOR CENTRAL
ISPOR SPEAKS
Dialogue With Decision Makers: Collaborating With Payers
to Advance Health Economics and Outcomes Research
Nadia Naaman, Senior Director Scientific & Health Policy Initiatives, ISPOR
A few decades ago when ISPOR was
founded, our members represented
mostly healthcare researchers and
health systems throughout the world,
the types of payers vary across countries
and within countries. Payers can be
academicians. But over the years, slowly government bodies at the national and/
but surely, our membership experienced or regional/local levels, private/statutory
a steady expansion of different insurers (both for-profit and nonprofit),
stakeholder groups, such as regulators and self-funding employers.
and assessors, payers and decision
makers, the life sciences industry, As a multistakeholder organization
healthcare providers, and of course, dedicated to improving healthcare
patient engagement organizations. decisions, ISPOR recognizes that payers
are a critical stakeholder group who
ISPOR believes that every healthcare can help apply and advance the science This year, ISPOR is excited to introduce
decision should be informed by the best of health economics and outcomes new opportunities to promote the
scientific research derived from rigorous, research (HEOR). In a strategic effort to dialogue with decision makers that
proven methodologies. We also believe start a dialogue and collaborations with focus on the payer perspective. Our
that the research should be used and these decision makers, ISPOR established goal is to create a series of events and
applied by all healthcare stakeholders. As a payer engagement initiative to increase programs that bring together ISPOR’s
a way to address these needs, ISPOR has ISPOR’s interaction with the payer multistakeholder audience and provide
established several councils to represent community, to drive awareness about the unique opportunities to interact and
these different stakeholders and has benefits of HEOR in healthcare decisions, collaborate with this influential payer
provided a platform where each of these and to ultimately establish ISPOR as a group. With the continued development
stakeholders can interact and engage in key resource and trusted partner for of high-cost therapies, payers and
discussions on key issues. healthcare payers around the world. manufacturers are increasingly engaging
in performance-based managed entry
One of the stakeholder groups Since 2007, ISPOR has hosted an agreements. The collaborative nature
that ISPOR has dedicated a lot of annual Health Technology Assessment of these payment schemes require all
effort and resources to cultivate (HTA) Roundtable, which has grown parties to work in alignment. To ensure
and collaborate with are healthcare to cover the 5 major regions of the the success of these arrangements there
decision makers, especially those world. Roundtable attendees include is a need to agree on the information
responsible for reimbursement representatives from public HTA bodies, provided by manufacturers at the time
policies for pharmaceuticals and other public and private payers, decision of launch, and the proper processes
health technologies, as these are makers, and government-contracted to collect and analyze real-world data
the core people who play an integral academic centers (if no HTA body exists post-launch. As a multistakeholder
role in the healthcare system. These in the country). In North America, the organization devoted to improving
decision makers are responsible for active and candid participation of payers healthcare decisions, ISPOR is well-suited
determining which health technologies has been the key to the high level of to further this discussion and come
are reimbursed, and this key group may interest and sustained overall success of closer to finding solutions.
or may not have the ability to influence these roundtable events.
the final price of the product or service. Healthcare decision making is
In short, this group is collectively referred ISPOR has many resources that are increasingly complex, and the best
to as “payers” and represents the public relevant to payers; however, few payer way to find solutions is to work across
and private organizations who ultimately organizations are using these resources stakeholder types toward a shared
decide whether a health technology is in a systematic way. Recognizing the outcome. ISPOR is dedicated to providing
reimbursed and at what price. importance of this key stakeholder an unbiased, collaborative environment
group, many ISPOR members have asked for interactive dialogue that includes
The ISPOR Book of Terms defines to have more payers participate at ISPOR all perspectives across the healthcare
a “healthcare payer” as the party conferences, as much of the research continuum. We look forward to
responsible for the financing and payment that has been conducted in the HEOR continuing to lead the way in improving
of healthcare for a population of eligible field must be accepted and applied by decision making in health for today,
persons. Due to the heterogeneity of them. tomorrow, and well into the future. •
Value & Outcomes Spotlight March/April 2020 | 7
ISPOR CENTRAL
HEOR NEWS
1 Employer Coverage 5
Potential Costs of Coronavirus Treatment for People With Does Pharma’s Future Lie in China? (pharmaphorum .com)
(Petersen-KFF Health System Tracker) Nooman Haque, managing director, Life Sciences, Silicon
As the new coronavirus spreads within the United States, Valley Bank UK, says if the pharmaceutical industry wants to
questions have arisen over the potential costs people may face continue capitalizing on the power of collaboration, it needs
if they become severely ill and need treatment. To address to open doors to cross-border investment. “Primarily, we
concerns over costs associated with the COVID-19 virus, Vice believe the future lies in a partnership with China,” Haque says.
President Mike Pence met with a group of large private insurers, “The East and West have their own very different healthcare
who agreed to waive copayments and deductibles for COVID-19 challenges, but it is precisely these differences that present us
tests. However, America’s health insurance plans clarified that with a unique opportunity for cross-border collaboration.”
the out-of-pocket costs for treatment (such as hospitalizations https://pharmaphorum.com/r-d/views-analysis-r-d/does-pharmas-
for more serious cases) would not be waived, meaning people future-lie-in-china/?utm_campaign=Industry+news+&utm_
with private insurance who face deductibles could be on the content=twitter&utm_medium=social&utm_source=twitter
hook for large costs.
6 Delivery in India: Addressing Information Gaps for Price
https://www.healthsystemtracker.org/brief/potential-costs-of- Estimating the Unit Costs of Healthcare Service
coronavirus-treatment-for-people-with-employer-coverage/
Setting and Health Technology Assessment (Applied Health
2
4 Ways Government Can Use AI to Track Coronavirus Economics and Health Policy)
(GCN)
India’s flagship National Health insurance program (AB-PMJAY)
Government health agencies can leverage artificial intelligence requires accurate cost information for evidence-based decision
(AI) technology to limit the spread of the new COVID-19 virus making, strategic purchasing of health services, and setting
and other diseases in 4 ways: prediction, detection, response, reimbursement rates. To address the challenge of limited health
and recovery. service cost data, this study used econometric methods to
https://gcn.com/articles/2020/03/10/ai-coronavirus-tracking.aspx identify determinants of cost and estimate unit costs for each
Indian state.
3
ICER Indefinitely Postpones Public Meetings for https://link.springer.com/article/10.1007%2Fs40258-020-00566-9
Sickle Cell Disease and Cystic Fibrosis, Expands Other
7
Assessment Timelines Up to 3 Months (ICER) Shared Decision Making: From Decision Science to Data
Like many other national and international healthcare Science (Medical Decision Making)
organizations holding regular meetings, the Institute for According to this study’s authors, while accurate diagnosis of
Clinical and Economic Review (ICER) has postponed some of patients’ preferences is central to shared decision making,
its meetings and expanded the timeline on other assessments often missing from clinical practice is an approach that links
because of the COVID-19 pandemic. “We are hopeful these pretreatment preferences and patient-reported outcomes.
expanded timeframes will enable all stakeholders to instead The authors (Azza Shaoib, Brian Neelon, and Leslie A. Lenert)
focus on meeting the needs of their patient communities during propose a Bayesian collaborative filtering algorithm that
this national emergency.” combines pretreatment preferences and patient-reported
https://icer-review.org/announcements/covid19_hiatus/ outcomes to provide treatment recommendations.
https://journals.sagepub.com/doi/10.1177/0272989X20903267
4
Medicare For All: If Not Now, When? (Health Affairs Blog)
8 for Generating Large-Scale Cohorts From the EHR for
While some pundits say the strong push by Democrats Model-Assisted Cohort Selection With Bias Analysis
for Medicare for All may succumb to political realities, Adam
Gaffney argues that while the hurdles are formidable, “steep Oncology Research (Flatiron Health)
political odds hardly compel us to abandon Medicare for All.”
To efficiently build research cohorts of greater scale without
He says history suggests that movements organized around
sacrificing quality, a team of data scientists, software engineers,
ambitious demands can over time create the conditions for
and clinicians at Flatiron Health have developed a technique
their passage, and that demands for radical change often
that combines machine learning and natural language
advance, rather than undermine, the prospects for more
processing with human review called Model-Assisted Cohort
incremental progress in the interim.
Selection with Bias Analysis to analyze the data found in
https://www.healthaffairs.org/do/10.1377/hblog20200309.156440/
unstructured documents, such as clinician notes and pathology
full/?utm_campaign=Industry+news+&utm_content=twitter&utm_
medium=social&utm_source=twitter reports, in electronic health records (EHRs).
https://rwe.flatiron.com/machine-learning-bias-analysis-real-world-data
8 | March/April 2020 Value & Outcomes Spotlight
ISPOR CENTRAL
9 11
The Interaction Between Price Negotiations and US Government Aims at High Insulin Prices With Plan
Heterogeneity: Implications for Economic Evaluations for $35 Copay in Medicare (Reuters)
(Medical Decision Making) The Trump administration in March turned back to its pledge to
Although economic evaluation is an important element of the fight high US drug prices with a plan to limit the out-of-pocket
decision-making process for the reimbursement of drugs, cost for insulin, a life-saving medicine, to $35 per month for
and heterogeneity can be considered an explained variation many people with diabetes who are enrolled in Medicare.
in clinical or economic outcomes based on the clinical and https://www.reuters.com/article/us-usa-healthcare-insulin/u-s-
sociodemographic characteristics of patients, this study’s government-aims-at-high-insulin-prices-with-plan-for-35-copay-in-
authors say to their knowledge, the relationship between medicare-idUSKBN20Y1WG
price negotiations and population heterogeneity has not been
12
considered in the literature to date. Hub Providers Can Be the Source of the Best Real-
https://journals.sagepub.com/doi/10.1177/0272989X19900179 World Evidence (Pharmaceutical Commerce)
10
Rational value-based contracts must be based on the ability to
Competitive Physician Prices in Fee-for-Service track the outcomes or continued health of patients. While most
Medicare (Health Affairs Blog) of these data comes from payers’ claims records and electronic
Experts have criticized the administrative approach to setting health records, another source of real-world evidence—and
traditional Medicare prices since its inception. But trying to set one that could play into a much wider range of value-based
up a more competitive pricing system faces several challenges, contracts—is the pharma industry’s hub and patient-support
as outlined by Bryan Dowd, Roger Feldman, and Robert providers, which routinely gather data from patients. “In some
Coulam: (1) the degree to which services are “shoppable” by cases, they are in touch with them daily, not just for medical
beneficiaries; (2) provider consolidation, which reduces the data, but scads of data on patients’ moods, emotions, and
number of bidders and imparts pricing power to the remaining concerns,” says Nicholas Basta.
providers; and (3) the inherent reluctance of consumers to https://pharmaceuticalcommerce.com/opinion/hub-providers-can-be-
change providers, which can make the market less price the-source-of-the-best-real-world-evidence/
competitive.
https://www.healthaffairs.org/do/10.1377/hblog20200312.579807/full/
Value & Outcomes Spotlight March/April 2020 | 9
7
ISPOR CENTRAL
RESEARCH ROUNDUP
Section Editor: George Papadopoulos, Emerald Corporate Group Pty Ltd, Sydney, Australia
Healthcare decision makers (whether they are payers, regulators, clinicians, or health economists) have to
grapple with a variety of evidence presented to them. Interpretation of Kaplan-Meir plots or response rates are
but 2 presentations of that evidence, and we have selected 2 recent articles that discuss the presentation and
interpretation of these data. Finally, qualitative health-preference research also can be utilized, and we present a
paper that discusses a set of guidelines to improve the frequency and quality of reporting.
Proposals on Kaplan–Meier plots in medical research and Reporting formative qualitative research to support the
a survey of stakeholder views: KMunicate development of quantitative preference study protocols
Morris T, Jarvis C, Cragg W, Phillips P, Choodari-Oskooei and corresponding survey instruments: guidelines for
B, Sydes M. BMJ Open. 2019;9(e030215): doi:10.1136/ authors and reviewers
bmjopen-2019-030215 Hollin I, Craig B, Coast J, Beusterien K, Vass C, DiSantostefano R,
Peay H. Patient. Published online: December 2019.
Summary Summary
We all use Kaplan-Meier curves or plots, but how is the Hollin, et al have developed a set of guidelines for authors and
information best communicated to both decision makers and reviewers to improve the frequency and quality of reporting of
non-decision makers? What is the level of uncertainty in the quantitative health preference research. The guidelines focus
difference estimates in survival time between the treatment on formative qualitative research used to develop robust and
groups? In this BMJ Open article, Morris, et al present research acceptable quantitative study protocols and corresponding
on improvements that can be made to the presentation survey instruments in health preference research.
of Kaplan-Meier curves to show the status of patients over
time, and to illustrate the uncertainty of the estimates. The The guidelines have 5 components with subcomponents:
authors then survey stakeholders in order to understand 1. Introductory material (4 domains)
which improvements are preferred. The authors created 6 2. Methods (12)
improvements of the “standard” Kaplan-Meier plot from 3 3. Results/findings (2)
published phase III randomized trials, and surveyed 1174 4. Discussion (2)
participants over a 6-week period. Most proposals were more 5. Other (2)
popular than the “standard” Kaplan-Meier plot. The most
popular proposals were in 2 categories: Relevance
Qualitative research is not often published, but the publication
1. An extended table beneath the plot depicting the numbers of formative qualitative research is a necessary step toward
at risk, censored and having experienced an event at periodic strengthening the foundation of any quantitative study. These
timepoints. guidelines should aid researchers, reviewers, and regulatory
agencies, and at the same time, promote the transparency
2. Confidence intervals around each Kaplan-Meier curve, the within health preference research.
latter one a favorite of mine.
Response rates and durations of response for biomarker-
Relevance based cancer drugs in nonrandomized versus randomized
The presentation of an extended table beneath the plot trials
depicting the numbers at risk (Plot A in Figure 2 of the paper), Gyawali B, D’Andrea E, Franklin J, Kesselheim A. J Natl Compr Canc
together with confidence intervals around the estimates (Plot E Netw. 2020;18(1):36-43. doi:10.6004/jnccn.2019.7345
in Figure 2), would greatly increase the ability of both expert and
non-expert decision makers to understand the survival times In this original research article, Gyawali, et al evaluated whether
more easily. Kaplan-Meier plots remain an important tool in the response rates and durations of response of targeted
research and analysis and the development of a more visually cancer drugs observed in nonrandomized controlled trials (non-
meaningful presentation of the result is a great step forward. RCTs) are consistent when these drugs are tested in randomized
controlled trials (RCTs). The authors compared the response
rates and median durations of response in non-RCTs versus
RCTs using the ratio of response rates and the ratio of durations
of response (defined as the response rates [or durations
of response] in non-RCTs divided by the response rates [or
10 | March/April 2020 Value & Outcomes SpotlightISPOR CENTRAL
durations of response] in RCTs). The ratio of response rates or Note: The preceding texts are simplified summaries of the published
durations of response was pooled across the trial pairs using articles. They do not contain an opinion on an in-depth analysis
random-effects meta-analysis. Both non-RCTs and RCTs were the results obtained by the authors. The selection of these works
available for 19 drug-indication pairs selected. The response was made based on overall relevance to the HEOR community,
rates and durations of response in non-RCTs were greater than not a product of a literature review or of a methodological quality
those in RCTs in 63% and 87% of cases, respectively. The pooled selection.
ratio of response rates was 1.06 (95% CI, 0.95–1.20), and the
pooled ratio of durations of response was 1.17 (95% CI, 1.03–
1.33). Response rates and durations of response derived from
non-RCTs were also poor surrogates for overall survival derived
from RCTs.
Relevance
As more and more drugs, especially new targeted cancer drugs,
are slated to receive regulatory approval globally, based on
durable responses in non-RCTs, this is important research to
consider. A critical eye should be cast over the use of durable
responses data derived from non-RCTs, because the responses
could be overestimates and poor predictors of survival benefit.
The authors conclude that caution must be exercised when
approving or prescribing targeted drugs based on data on
durable responses derived from non-RCTs. •
Value & Outcomes Spotlight March/April 2020 | 11ISPOR—The Professional Society for Health Economics and Outcomes Research
ISPOR 2020 Conferences
Virtual ISPOR 2020
May 18-20
HEOR: Advancing Evidence to Action
May 14: New Plenary Session “HEOR in the Era of COVID-19”
May 18-20: Virtual Conference
ISPOR-FDA Summit 2020
Using Patient-Preference Information in Medical Device
Regulatory Decisions: Benefit-Risk and Beyond
Summit Postponed
Visit the website for updates
12-15 September
Seoul, South Korea
Next Generation Healthcare in Asia Pacific:
Where Technology Meets Patients to Improve Care
Abstract Notifications: 10 June
Early Registration Deadline: 28 July
14-18 November | Milan, Italy
Improving Health: Establishing
Incentives and Sharing Value
Early Registration Deadline: 6 October
Abstract Submissions Open Through 8 July
Looking ahead to 2021 Conferences:
ISPOR 2021 ISPOR Latin ISPOR Europe 2021
May 15-19 America 2021 6-10 November
Montreal, Canada Mexico City, Mexico Paris, France www.ispor.org
12 | March/April 2020 Value & Outcomes SpotlightThe Society’s Annual Conference
Is Now a Virtual Event
SAVE THE DATE
Virtual ISPOR 2020
MAY 18-20
HEOR: Advancing Evidence to Action
The 3-day virtual conference will include issue panels,
workshops, podium presentations and symposia, as well as
3 featured plenary sessions:
• Health Policy—Designing for the Future
• HEOR and Clinical Decision Making—Advancing Meaningful Progress
• On the Road to Enhanced Cost-Effectiveness Analysis—New Directions, New Milestones
A virtual plenary session, HEOR in the Era of COVID-19, scheduled for May 14
NEW
10:00AM-12:00PM EDT, will be offered free to members and non-members.
The Virtual Short Course Program will be available beginning in June.
Visit the ISPOR website for dates and times.
Detailed program information and registration will be announced soon.
Visit www.ispor.org/ISPOR2020 for updates.
Value & Outcomes Spotlight March/April 2020 | 13ISPOR CENTRAL
FROM THE JOURNALS
Section Editors:
Soraya Azmi, MBBS, MPH, Beigene, California, USA; Agnes Benedict, MSc, MA, Evidera, Budapest, Hungary
Value in Health. 2020; based health study and indicated they or polyclinic were preferred by 85% of
would be available for future studies. respondents; few preferred vaccinations
23(2):200–208. Participants were randomized into 4 in their own home or other options.
Optimal Design of Population-Level groups. People in Group 1 received Importantly, 76% perceived the vaccine
a survey regarding their thoughts as safe, but few people considered
Financial Incentives of Influenza
about the flu vaccine (which was to be themselves being at risk of infection
Vaccination for the Elderly completed in 2 months), and a SGD without the vaccine (35%).
Mu Yue, Yi Wang, PhD, Chng Kiat Low, $10 (US $6.90) shopping voucher (this
Joanne Su-yin Yoong, Alex R. Cook served as the control group). In the 3 Circumstances related to the flu vaccine
intervention groups, people were asked are unique in Singapore in many
Influenza (or the flu) is caused by 3 types to fill in the survey and also to go for ways: there is less seasonality due to
of influenza virus. It is a rapidly evolving the vaccine (at their own cost, at SGD its equatorial location, the funding of
virus, causing 3 to 5 million severe cases $32 [US$22.08]), in return for a small healthcare is based on medical savings
per year and approximately 260,000 compensation of SGD $10, $20, or $30 account, and it is a developed yet
to 560,000 deaths per year.1 The case (US $6.90, $13.80, and $20.69) in the small country. However, the topic of
fatality is highest among high-risk form of shopping vouchers. The outcome the paper is very important, especially
patients (ie, children, the elderly, and measured was “participation within 2 considering the current COVID-19 virus
people with other comorbidities). It is a months,” corresponding to returning the pandemic. Flu is a potentially deadly
seasonal disease mostly occurring in the survey in Group 1 and returning both the disease among high-risk groups and that
winter but can happen any time along survey and vaccination certificate dated can put additional strain on the health
the equator. Flu vaccines are developed within the study period. Letters returned system given the presence of COVID-19.
twice a year to match the predicted mix from unknown addresses and those Although a vaccine is available for the
of types of viruses for the coming season. vaccinated within the previous 6 months flu, the awareness of the severity of the
Vaccination is recommended annually were excluded from the denominator for disease and the uptake of the vaccine
for the high-risk groups, but several calculating the participation. are very low. Direct financial incentives
countries recommend it to everyone. may have an important role in targeting
There are some countries where it is Overall response was 9.3%, with highest the high-risk groups. However, more
offered free of charge (United Kingdom), in Group 1 (16.9%) and extremely low ideas will be needed to substantially
or at a relatively low cost.1 However, values in the 3 other groups (4.5%, 7.5, increase the number of patients showing
uptake of the flu vaccination is variable. and 9.2%, respectively). Nevertheless,
the increase in the total incentive from
up for their annual flu shots. •
In the United Kingdom, where a universal SGD $10 to $20 (US $6.90, $13.80) in
References
vaccine program for school children shopping voucher value was statistically
1. World Health Organization. Influenza
was introduced in 2013, a retrospective significant; further increase was not. (Seasonal). Ask the expert: Influenza Q&A. 6
observational study of 500 primary However, in terms of trends, both November 2018. Archived from the original
care practices (~700,000 children) males and females were more likely to on 30 November 2019. https://www.who.
showed an increase in vaccination participate if SGD $30 (US $20.69) was int/news-room/fact-sheets/detail/influenza-
uptake from 2012–2013 to 2013–2014 offered versus SGD $10 (US $6.90), (seasonal). Accessed March 20, 2020.
in targeted children aged 2 to 3 years, while some other demographic factors
2. Rajaram S, Steffey A, Blak B, Hickman
both in children with a high-risk medical mattered: Chinese elderly were more
M, Christensen H, Caspard H. Uptake
conditions (from 40.7% to 61.1%) and sensitive to incentives, as well as the
of childhood influenza vaccine from
those without (from 1.0% to 43.0%).2 nonworking elderly, and those over 75
2012–2013 to 2014–2015 in the UK and
According to the United Kingdom’s official years responded much more strongly the implications for high-risk children: a
statistics on seasonal flu vaccines, the to the incentives. The authors looked retrospective observational cohort study.
uptake among individuals over 65 years for the “optimal” financial incentive BMJ Open. 2016;6:e010625. doi:10.1136/
of age was 71.3% in 2018, and among but considered the vaccine uptake as bmjopen-2015-010625.
those at high risk, 46.9%.3 In Singapore an external variable. Therefore, this is
(Yue et al),4 the current uptake of the flu not a true optimization along multiple 3. GOV.UK. Official Statistics. Seasonal flu
vaccine uptake in GP patients: monthly
vaccine is around 15% among school parameters. Their key finding is that
data, 2019 to 2020. https://www.gov.uk/
children and 17% among the elderly, considering transmission dynamics, an
government/statistics/seasonal-flu-vaccine-
despite recommendations for vaccination incentive between SGD $10 and $20 (US
uptake-in-gp-patients-monthly-data-2019-
from the local ministry of health. We $6.90 and $13.90) minimizes the cost to-2020. Accessed March 20, 2020.
review the article by Yue et al studying per completed vaccination from a health
the impact of a financial incentives among system perspective. 4. Yue M, Wang Y, Low CK, Yoong J S-Y,
the elderly in Singapore. Cook AR. Optimal design of population-level
In terms of the survey results, of those financial incentives of influenza vaccination for
The authors invited 4000 individuals, who responded, vaccinations that took the elderly. Value Health. 2020;23(2):200-208.
who all participated in a population- place in a general practitioner’s office
14 | March/April 2020 Value & Outcomes SpotlightISPOR CENTRAL
FROM THE REGION
ISPOR’s Health Technology Assessment and Patient Representative Roundtables:
Strengthening Patient-Centered Decision Making in Asia Pacific and Globally
Robert Selby, MBA, Director, Global Networks (Asia Pacific and Latin America), ISPOR, Lawrenceville, NJ, USA
A gainst the backdrop of rapid
institutional reforms and
development for national health
Association of China, China Organization
for Rare Diseases, Philippine Alliance
of Patient Organizations, Vietnam
impact analysis, and health economics
(budget impact analysis and health
economic analysis were optional
technology assessment (HTA) in Hemophilia Group, China Rare Disease previously). Additionally, the government
China, ISPOR hosted HTA and Patient Organizations Development Network is also engaging in a program of high-
Representatives Roundtable discussions (Mainland China), and the Psoriasis volume centralized purchasing of
in Beijing, China on 25 October 2019. Association Taiwan. generics through their “4+7 Plan,” which
ISPOR’s HTA and Patient Representative
Roundtables are platforms to advance
scientific methods, facilitate information
sharing about the development of HTA,
A major thread of discussion centered around ways payers are
and strengthen the role HTA plays in bringing access of high-cost innovative therapies to patients while
optimizing healthcare decisions. These
roundtables are ideal opportunities
still maintaining acceptable budgets.
for ISPOR to bridge the gap between
technology assessors, private and public The key topics of the roundtables has led to lower prices for a wide variety
payers, regulators, and patients, and centered on managing high-cost of medicines.3 While such approaches
the discussions focus on innovative therapies and patient participation in have yielded rapid and dynamic results,
ways to improve health globally and HTA and healthcare decision making. how these changes will affect the
make healthcare decision making Participants presented specific cases of healthcare system in terms of systematic
more patient-centric. ISPOR HTA and managed entry schemes, negotiation and transparent processes toward value
Patient Representatives Roundtables mechanisms and approaches for pricing and efficacy assessment, prioritization
are convened regularly in Asia Pacific, and reimbursement, HTA harmonization and access, and health technology
Europe, Latin America, Middle East and across the globe, HTA in universal health innovation remains to be seen.
Africa, and North America.1,2 coverage implementation, and patient
involvement in healthcare decisions. Risk-sharing agreements or other
The ISPOR Asia-Pacific HTA and Patient managed-access programs have
Representatives Roundtables enjoyed Improving Patient Access to been in practice in South Korea and
broad representation from key experts Innovative Technologies Australia for several years and have
and decision-making bodies from the A major thread of discussion centered provided incentives and pathways
region, including the Pharmaceutical around ways payers are bringing access for the adoption of promising new
Benefits Advisory Committee (Australia); of high-cost innovative therapies to technologies for vulnerable patients
Health Technology Assessment in India patients while still maintaining acceptable where limited data may exist. While
(India); HTA Committee (Indonesia); budgets. Jurisdictions are taking highly there have been some examples of
Health Insurance Review & Assessment varied approaches to this issue, ranging success with these programs, significant
Service and National Evidence-based from direct centralized negotiation in challenges remain, particularly regarding
Healthcare Collaborating Agency China to outcomes-based arrangements capacity and bandwidth of payers
(South Korea); Center for Drug in Australia and South Korea. Chinese in collecting data and assessing the
Evaluation (Taiwan); National Institute payers have leveraged their purchasing relevant evidence. And since many of
of Public Health and National Institute power and large market to extract steep these arrangements are only active for
of Social Security and Population price cuts for many orphan drugs and 4 to 5 years per contract, the questions
Research (Japan); Health Technology have also sped up review and approval surrounding long-term efficacy and value
Assessment Section, Ministry of Health, processes significantly. The Chinese are harder to answer. Many studies
Malaysia; Philippine Health Insurance government is also conducting more that utilize narrow time horizons or
Corporation (Philippines); Agency for frequent comprehensive reviews and surrogate endpoints for the candidate
Care Effectiveness (Singapore); Health updates of the national reimbursement interventions are said not to adequately
Intervention and Technology Assessment drug list, with the latest update occurring capture the full costs and value that
Program (Thailand); and HTA department in 2019. Currently, all new therapies are expected to be realized throughout
(Mongolia). Patient organizations that under consideration are required to the technology’s life cycle. Additionally,
were represented included the Heart to undergo review in the areas of clinical the arrangements themselves can
Heart Foundation (Thailand), Lymphoma efficacy, pricing benchmarks, budget bring substantial risk and uncertainty.
Value & Outcomes Spotlight March/April 2020 | 15ISPOR CENTRAL
Thus, some payers still feel hesitant to inclusion of consumer representatives. assessment of treatment effects,
pursue these types of arrangements While these examples are encouraging, and key methodological issues in
(except in very special cases). It was there are still questions among pragmatic randomized controlled trials.
expressed by many participants that to patients as to whether this is enough, Regionally, however, there are questions
make such arrangements more feasible as these representatives do not have surrounding managing uncertainty,
in the region, additional work needs voting power in some jurisdictions including what structure and resources
to be done by innovators to generate and may have a limited capacity in are needed to clarify the impact and
acceptable evidence for payers to providing input. Patient groups also relevance of data. Specifically, how do
mitigate uncertainty and risk wherever question whether such a small patient we collectively define an intervention’s
possible. For example, there should be delegation on these committees could level of impact or magnitude of benefit?
enough of an initial correlation within be truly representative of the broader Is it just high unmet need being met?
the clinical trials and a sufficiently community, even if they may be expertly What is a significant clinical benefit—is
robust accompanying body of outcomes qualified. And for groups that still lack it defined in terms of breadth or depth?
data to support effective decision formal participation mechanisms in their How do patients value judgments
differ from society as a whole? And the
question of changing priorities and
According to one prominent patient advocate in the region, realities in the light of evolving evidence
data are an important tool for patient organizations to present and perspectives necessitates clarity of
approaches surrounding disinvestment
their case to decision makers, and that without data, a patient and de-listing of technologies.
is just another person with an opinion. What Can Patients Contribute?
Based on the notable efforts many
making.4 Payers should also have a respective jurisdictions or feel that such patient organizations are making in the
better understanding of the potential processes are lacking, advocacy remains region, it was clear that patient data are
market impact of reimbursement and their primary recourse, which has its own one of the most powerful witnesses they
renegotiation decisions, which could limitations. While progress is occurring, can provide. According to one prominent
affect the availability of certain products much more needs to be done to ensure patient advocate in the region, data
in their countries. that these processes are achieving the are an important tool for patient
ultimate objective of making decisions organizations to present their case
Patient Involvement in HTA in Asia patient-centric. To that end, key to decision makers, and that without
Pacific: Where Are We? questions have emerged, namely: data, a patient is just another person
As patients and patient advocates (1) What is the proper role of patients in with an opinion. Patient representative
are becoming empowered to take HTA and healthcare decision making? organizations have taken incredible
ownership of their healthcare, they (2) Where should patients get involved in efforts to generate patient-centric data
are increasingly laying pressure on the process? and (3) What can patients for decision makers, as well as publishing
HTA bodies and policy makers and meaningfully contribute to the process? reports and presenting to policymakers
emphasizing the importance of their to emphasize the special considerations
involvement in informing policy and Managing Uncertainty: Local Data that HTA needs to make for rare
HTA decisions. At the same time, there Constraints and Future Investment diseases. Patient-generated data can
is rising consensus in the region among Many jurisdictions in Asia Pacific provide insights into patient preferences
policymakers that healthcare decision struggle with a paucity of local and priorities for policymakers, and
making and delivery should be patient- population data, which means that patient inputs can help researchers to
centric and equitable. Many jurisdictions many important reimbursement better capture the burden of disease and
in the region have already formally decisions must be taken based on cost of illness. Jurisdictions in the Asia
incorporated patient involvement in potentially limited relevant evidence. Pacific region have incorporated various
their HTA processes. In Taiwan, 2 patient Challenges remain in making data and mechanisms for capturing patient data
representatives are invited to participate evidence available and adaptable for and perspectives. For example, Taiwan
in the Pharmaceutical Benefit and local considerations. China is taking has fielded a patient questionnaire
Reimbursement Standard joint meeting large strides toward incorporating with an online submission form and
as nonvoting members. In Australia, and utilizing big data in healthcare guidelines to generate patient feedback;
the Pharmaceutical Benefits Advisory decision making at all levels, with the Australia also utilized a similar feedback
Committee has 2 expert consumer establishment of a China Real World process. Nonetheless, quality of feedback
(patient) representatives, and schedules Data and Studies Alliance (ChinaREAL) and patient data remains a challenge, as
consumer hearings to facilitate dialogue. and investment in data infrastructures.5 there is no formal system for assessing
Australia also established the HTA The ChinaREAL collaboration validity or considering conflicts of interest
Consumer Consultative Committee has resulted in the production of (lobbying influences) in Australia.
in 2017 that provides strategic advice technical guidance documents
and support to the principal Health including databases and registries for A key challenge for the future will be
Technology Assessment Committees research purposes, epidemiological making patient inputs and data more
and the Department of Health with the and statistical considerations in the meaningful for payers and impactful in
20 | March/April 2020 Value & Outcomes Spotlight
16ISPOR CENTRAL
health policy. The first part of this relates needs to be done to strengthen health
to the ability of patient organizations infrastructures and to bridge evidence
Additional information
to effectively leverage their voice gaps globally through health economics
and position as a credible and vital and outcomes research. Finally, it will be The next ISPOR Asia Pacific HTA and Patient
stakeholder in the process. To lend essential for HTA stakeholders to more Representative Roundtables will take
more weight to their voices, “expert actively facilitate translation of their place during the ISPOR Asia Pacific 2020
patients” are needed—both globally and recommendations into policy. A model Conference, to be held on 12-15 September
regionally—to strengthen the foundation for this could be Malaysia, which involves 2020 in Seoul, South Korea. For more
for organizational/institutional government payers in assessment information on these and other initiatives,
participation and incorporation of priority setting through criteria and please visit: www.ispor.org/member-groups/
perspectives, and education will remain discusses evidence with decision makers councils-roundtables.
a critical part of this. Patients should on the local context.
also be better advocates (not just for
their specific diseases but for their This report is adapted from presentations
stakeholder group as a whole), as they and discussions that occurred during ISPOR
will be more effective in a unified way. HTA and Patient Representative roundtables
A “turfing” mentality still exists among
some patient societies as they vie for
- Asia Pacific on 25 October 2019. •
influence and limited resources. References
1. ISPOR Health Technology Assessment
The other part of this relates to the Roundtables. International Society for
quality of patient data. For policymakers Pharmacoeconomics and Outcomes
the question becomes: What kind of Research. https://www.ispor.org/
data are really helpful for decision member-groups/councils-roundtables/
making? With respect to qualitative data, health-technology-assessment-council/
decision makers count specific and rich health-technology-assessment-roundtables.
patient testimonials (ones that share Accessed January 2, 2020.
patients’ personal disease experience 2. ISPOR Patient Representatives Roundtables.
and effects on the quality of their lives ) International Society for Pharmacoeconomics
as most useful to them. From a research and Outcomes Research. https://www.ispor.
standpoint, patient perspectives have org/member-groups/councils-roundtables/
patient-council/patient-representatives-
the potential to ensure that clinical trial
roundtables. Accessed January 2, 2020.
and observational study designs have
assumptions, objectives and endpoints 3. Yue X.. “4+7” Drug procurement reform in
that are better aligned with the real China. China National Health Development
world to optimize outcomes.6 Research Center, Beijing, China, 2019. https://
www.cgdev.org/sites/default/files/CGD-
procurement-background-china-case.pdf.
Conclusions Accessed March 12, 2020.
For HTA to be successful, it should be
timely, relevant and practically usable for 4. Adamski J, Godman B, Ofierska-Sujkowska
G, et al. Risk sharing arrangements for
decision makers, and follow an inclusive
pharmaceuticals: potential considerations
and transparent process that proactively
and recommendations for European payers.
emphasizes local horizon scanning BMC Health Serv Res. 2010;10(153). https://doi.
and priority setting. Patients are a key org/10.1186/1472-6963-10-153.
stakeholder group for healthcare and
5. Sun X. Real world evidence in China:
should be actively involved in HTA, but
current practices, challenges, strategies and
where and how they are involved in the developments, presented at ISPOR Asia Pacific
process needs to be clarified further. 2018 Conference, Tokyo, Japan. https://www.
Moreover, there is an important role for ispor.org/docs/default-source/conference-
patients to play in clinical trial design ap-2018/china-2nd-plenary-for-handouts.
and in the design and interpretation of pdf?sfvrsn=5fbc7719_0. Accessed March 12,
observational studies. 2020.
6. Johnson LB, Smalley JB. Engaging the
Development and utilization of local patient: patient-centered research. In: Hall
data will be an essential priority for K, Vogel A, Croyle R, eds. Strategies for Team
Asia Pacific countries in the immediate Science Success. [[City, Country: Springer
term to mitigate global data reliance. [[full company name]]; 2019. https://doi.
Patient-reported outcomes data are org/10.1007/978-3-030-20992-6_10.
also set to play a more prominent role
in future evidence considerations,
including in China. Further works
Value & Outcomes Spotlight March/April 2020 | 15
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