Financing and Implementing Innovation in Healthcare Systems: A Component of the Precision Medicine Readiness Principles - WHITE PAPER JUNE 2021
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Financing and Implementing Innovation in Healthcare Systems: A Component of the Precision Medicine Readiness Principles WHITE PAPER JUNE 2021
Cover: Majcot, Getty Images – Inside: Getty Images, Unsplash
Contents
3 Foreword
4 Executive summary
6 Introduction
8 Innovation ecosystem overview
9 A Defining the innovation ecosystem
9 B Innovation ecosystem components
11 1 Innovation governance
12 1.1 Precision medicine implementation roadmap from national strategic plans
14 1.2 Open science principles
15 1.3 Precision medicine IP policy and benefit sharing
16 1.4 Adapted health technology assessment processes for precision medicine
technologies
18 2 Innovation financing
19 2.1 Public funding and fiscal space usage
20 2.2 Private-sector financial backing
22 2.3 Blended finance and the diagonal approach
23 2.4 Reimbursement conditions and coverage models
24 2.5 Experimentation with value-based care instruments
26 3 Community and partnerships
27 3.1 Entrepreneurship, research and innovation hubs
28 3.2 University-industry collaboration
29 3.3 Learning healthcare system
31 4 Workforce and infrastructure
32 4.1 Foundational health systems strengthening
32 4.2 Economic development
33 4.3 Absorptive capacity
35 Gaps and future research areas
37 Conclusion
38 Appendix
41 References
43 Secondary sources
45 Contributors
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Financing and Implementing Innovation in Healthcare Systems 2
June 2021 Financing and Implementing Innovation
in Healthcare Systems:
A Component of the Precision Medicine
Readiness Principles
Foreword
This white paper highlights the need to bridge
existing gaps in investment approaches.
Genya Dana Elissa Prichep
Head of Healthcare Lead, Shaping the Future
Transformation, of Health and Healthcare,
World Economic Forum World Economic Forum
The ability to design and implement targeted and circumstances of the geographic location, patient
personalized approaches to screening, preventing, population, economic situation and political realities.
diagnosing and treating diseases continues to
improve. There is great promise in precision The World Economic Forum wishes to aid policy-
medicine, with improved outcomes and more makers, funders, investors, clinicians and others who
efficient use of scarce resources. are preparing their healthcare systems for a future
state that includes precision medicine approaches.
As precision medicine approaches continue to make We are pleased to present this instalment in the
their way into more care systems, we are still woefully Precision Medicine Readiness Principles, which
lopsided in terms of who has access to the necessary identifies capabilities stakeholders can use to
research, technologies, treatments and skill sets. benchmark progress in nurturing a system that is
Trust in healthcare systems’ ability to deliver care in ready for precision medicine approaches.
equitable and agile ways continues to be tested and
found wanting during the COVID-19 pandemic. This white paper on the financing and
implementation of precision medicine approaches
Many healthcare systems, clinicians and policy- explores the necessary components to build
makers who want to harness the power of and sustain advanced healthcare innovation
precision medicine approaches find themselves ecosystems. While the focus of this paper is on
faced with the daunting task of determining the mechanisms that can be used today, as well
where to begin. This is especially true in places as case studies describing how different countries
that are under-resourced or making a transition have implemented such initiatives, the work also
from prioritizing basic primary care and infectious highlights the need to bridge existing gaps in
disease treatment to treating diseases such as investment approaches. The white paper offers
cancer and rare diseases. Investments must be both a path to innovation in healthcare ecosystems
made across many areas, including workforce, and a call for continued investment in delivering
infrastructure, technology and strategic planning, in the highest standards of health and healthcare to
ways that are complementary and responsive to the people around the globe.
Financing and Implementing Innovation in Healthcare Systems 3
Executive summary
In 2016, the National Academy of Medicine precision medicine innovation ecosystem. Case
published a discussion paper, Realizing the Full studies from developed and emerging economy
Potential of Precision Medicine in Health and countries are used to illustrate the implementation
Healthcare, that would respond to the growing of such capabilities by countries at any (early,
interest in supporting precision medicine through intermediate, advanced) stage of precision medicine
US health policy by proposing a framework that readiness. Developed under the Leapfrogging with
leaders in policy, business and healthcare could Precision Medicine project, this paper also calls
reference for the development and integration of attention to tools and case studies that can help
precision medicine. Informed by this work and address inequities in the application of innovation
developed through desk research, interviews drivers and the accessibility of precision medicine
and a multistakeholder workshop at the 2019 approaches globally.
World Health Assembly, the World Economic
Forum’s Platform for Shaping the Future of Health Go-to-market strategies for precision medicine
and Healthcare developed a framework for the innovations remain challenging given the incentive
Precision Medicine Readiness Principles: a gaps for technology producers, funders and
global roadmap that identifies precision medicine service provider adopters and payors alike.
capabilities from which policy-makers and others Forming the necessary enablers and conditions
looking to advance precision medicine in their for the innovation ecosystem requires novel policy
countries can find benchmarks for readiness. instruments, financing approaches, collaboration
models and economic development as explored in
Under the broader faculty of the Precision this white paper.
Medicine Readiness Principles, the innovation
ecosystem topic aims to address the processes Figure 1 below summarizes the core discussion
that enable the creation and expansion of a areas explored in this work – the exemplary
precision medicine marketplace. Specifically, capabilities and implementation milestones across
this paper discusses the necessary functions four main categories: innovation governance;
of business producers, resource funders and innovation financing; community and partnerships;
regulatory governors to encourage, establish and and workforce and infrastructure. These
build a continuously growing economic marketplace benchmarks are further positioned along the
for precision medicine technologies. To this end, the readiness axis to inform the criteria for progress
paper presents: (1) a framework for the capabilities and capability maturity. “Exploring” health systems
of the precision medicine innovation ecosystem; are early in their adoption of precision medicine
(2) benchmarks to assess the stage of readiness approaches, potentially conducting some research
across these capabilities; and (3) outstanding gaps studies or piloting programmes, but will generally
in the investment approaches needed to drive not have precision medicine approaches accessible
precision medicine implementation. to members of the population. “Activating” health
systems have some clinical use of precision
Policy-makers and others looking to advance medicine approaches with limited accessibility,
precision medicine in their countries are invited to while “implementing” systems have several
use this guidance document to gauge the maturity precision medicine approaches accessible to a
of the various capabilities that build and sustain a large number of people.
Financing and Implementing Innovation in Healthcare Systems 4
Insights from this work include the importance of: value-based care approaches – e.g. evidence-
based modelling studies, performance-based
Establishing national innovation policy contracts – may align stakeholder incentives and
instruments: The innovation policy mix consists of invite consensus on precision medicine’s value.
the initiatives – e.g. intellectual property legislation,
cluster initiatives, market review processes, federal Expanding collaboration and operating models:
budgets – that build the early national public Various multistakeholder partnership models have
confidence and market demand for precision emerged across the value chain to advance precision
medicine approaches that private entities cannot medicine research and development (R&D) and
build alone. commercialization. Further collaborations between
both public and private parties are encouraged to
Developing novel financing approaches that explore novel operating models such as the learning
capture value: Novel financing models for precision healthcare system and open science for genomics.
medicine technologies that capture value and
promote equity and access are encouraged to spur Nurturing incremental innovation and
private investment activity. sustainable adoption: Incremental innovation in
precision medicine remains the most prevalent form
Public funding and philanthropy dollars may be of development. Early initiatives such as systems for
insufficient or unavailable in the longer term for collecting family health history or national disease
low- and middle-income countries (LMICs) seeking surveillance registries may be initial steps towards
to develop their precision medicine capabilities. precision medicine adoption.
Cross-cutting diagonal funding approaches that
support health-system strengthening are particularly While this work represents only one part of the
encouraged to close this financing gap. Precision Medicine Readiness Principles,
it is the hope of the authors that the readiness
Actualizing value-based care: Coverage and principles are used by policy and healthcare leaders
reimbursement gaps persist globally, in part as central points of departure from which to build
because of insufficient data on precision medicine robust precision medicine capabilities into their
value-add and payment uncertainty. Emerging health systems.
FIGURE 1 Precision Medicine Readiness Principles for the innovation ecosystem topic
100
Exploring Activating Data
Implementing
90
AReadinessA
80 National strategic plan Diverse public
Data initiative types
Data
Innovation governance
GO FAIR* principles
Public legislation
70 and regulatory Mexico
considerations in the provision of Benefit sharing of genomics IP Clear IP ownership decisions
precision medicine
Y-Axis (Y-axis subhead)
Data
Data
60 HTA** adaptations
Brazil Data
50 Public and philanthropy-driven funding Diverse private financiers
Innovation financing
Blended finance models Alternative payment models
China
Funding mix to both set up and
40reimburse precision
regularly
Evidence-based modelling studies
medicine programmes
Data
Data Value-based reimbursement
30
Data
Data initiatives
Innovation cluster Data
Community and partnerships Data
20
Academic research
Data consortia Data
TTOs***/industry collaborations
Methods by which multistakeholder Data Data
collaboration emerges in an South-south collaborations Data
10 ecosystem Public-private partnership mix
innovation
Learning healthcare system
Data
0 Data
1 2 3 4 5 6 7
SDG 3**** foundational effectors
Workforce and infrastructure X-Axis
Family health history records National patient registries Technology-intensive infrastructure
Health system, human capital and
economic foundations core to Workforce development Precision health upskilling
precision medicine
Technology absorption Production-driven innovation
*Findability, Accessibility, Interoperability and Reuse of digital assets, **Health technology assessment, ***Technology transfer organization, ****United Nations Sustainable Development Goals
Financing and Implementing Innovation in Healthcare Systems 5Introduction
Leaders in policy, business and healthcare, in the form of “capabilities”, defined as the working
while receptive to the potential of precision set of key elements employed to drive development
medicine applications, do not have the benefit of precision medicine approaches within healthcare
of a consistent and standardized framework ecosystems. These capabilities are neither an
to both evaluate their health system’s current exhaustive list nor mandatory criteria, but represent
readiness for the practical implementation and a distillation of critical drivers in developing a
integration of precision medicine and elucidate precision medicine environment within a country.
the capabilities needed to drive future growth.
Stakeholders in emerging economies, in particular, This white paper focuses on one pillar of the
indicated that they would value a guidance Precision Medicine Readiness Principles: the
document that includes case studies and access innovation ecosystem. Hence, the capabilities
to partner communities as they think about how described in this paper should be considered in the
to strategically grow the precision medicine context provided by the foundational elements and
capabilities of their healthcare ecosystems. other categories captured in the Precision Medicine
Readiness Principles.
In alignment with this need, the World Economic
Forum’s Platform for Shaping the Future of Health
and Healthcare is developing the Precision Medicine
Readiness Principles: a thought leadership
project, inspired by a National Academy of Medicine
discussion paper, that includes developing a living
document from which those aiming to advance
precision medicine in their countries can find
benchmarks for readiness. Precision medicine offers
a more personalized and targeted approach to
preventing and addressing disease through screening,
diagnosing and treating patients by considering
their genetic and biological make-up, surrounding
environmental factors and lifestyle behaviours.
By identifying best practices and strategic
opportunities for precision medicine adoption
in a healthcare setting, the Precision Medicine
Readiness Principles serve as a gauge that
policy-makers and others looking to advance
precision medicine in their countries can use to:
(1) find maturity assessment benchmarks for
readiness; and (2) identify potential steps forward to
implementation. These benchmarks are illustrated
Financing and Implementing Innovation in Healthcare Systems 6FIGURE 2 Precision Medicine Readiness Principles framework. This framework comprises five
core topics that establish criteria that countries or stakeholders can use to evaluate their
progress towards precision medicine
PM
CAPABILITIES
FRAMEWORK Actionable Evidence Innovation
Engagement health data generation Care integration ecosystem
Implementing
“several precision medicine Focus: public Focus: actionable Focus: evidentiary Focus: bringing precision Focus: innovation-
approaches accessible to a engagement and and ethical data frameworks for the medicine into routine oriented
large percentage of the population” inclusion, provider collection evaluation of precision clinical care stakeholder
education and medicine approaches communities
relationship building Capabilities may Capabilities may for marketplace
include: Capabilities may include: growth
Activating Capabilities may biobanking, omics, include: clinical care providers,
“in some clinical use with include: clinical data and approval frameworks, care guidelines, Capabilities may
limited accessibility” patient awareness, real-world data outcome technologies such as include:
provider training, collection, data policy measurements, diagnostics and governance,
diversity and inclusion, and governance, value assessments, treatments, accessibility financing,
Exploring community standards and ties to reimbursement partnerships,
engagement interoperability workforce and
“able to pilot”
and trust frameworks infrastructure
Foundational elements (enablers)
Include: general infrastructure, basic healthcare systems, regulatory oversight, coverage mechanisms, information technology systems, social determinants
Financing and Implementing Innovation in Healthcare Systems 7Below: Ipopba, Getty Images
Innovation ecosystem
overview
What are the capabilities that build and
sustain a precision medicine marketplace?
Financing and Implementing Innovation in Healthcare Systems 8A Defining the innovation ecosystem
The innovation ecosystem topic aims to address to the various forms of novel developments in
the processes that pave the way for the creation precision medicine and includes new products,
and expansion of a precision medicine marketplace firms, technology, business models, behavioural
and its enabling conditions. Specifically, the changes and policies. Such forms of precision
white paper discusses the necessary functions medicine “innovation” manifest differently in a global
of business producers, resource funders and context; innovation may range from the preferential
regulatory governors to encourage, establish introduction of incremental, marginal improvements
and build a continuously growing economic in process to significant technological adoption
marketplace for precision medicine technologies. or novel product development. Hence, while this
The guiding research question for this topic can be white paper provides capabilities that may serve
framed as “What are the capabilities that build and as a preparatory model for the implementation
sustain a precision medicine marketplace?” of a precision medicine innovation ecosystem,
it excludes specific, prescriptive guidelines as
Under the broader umbrella of precision medicine programmes almost always require modifications
implementation, the innovation ecosystem refers and adaptations to fit local contexts.
B Innovation ecosystem components
FIGURE 3 Innovation ecosystem framework. This topic area in the Precision Medicine Readiness
Principles covers four foundational areas: innovation governance, innovation financing,
community and partnerships, and workforce and infrastructure
Innovation Innovation
governance financing
Open science principles Public funding
IP ownership and benefit sharing Private funding
Market approval process (HTA) Blended capital
National strategic plan Reimbursement and coverage
Value-based care instruments
INNOVATION
ECOSYSTEM
Community and Workforce and
partnerships infrastructure
Research and innovation hubs Health systems strengthening
University R&D and tech transfer Economic development
Learning healthcare system Absorptive capacity
The innovation ecosystem white paper was financing; (3) community and partnerships; (4)
developed through a multistakeholder workshop on workforce and infrastructure. For the purposes of
the sidelines of the 2019 World Health Assembly, this paper, these areas and their corresponding
desk research, the compilation of a Precision capabilities are defined below.
Medicine Readiness Principles: Innovation Loop
Resource Guide and individual consultations with Innovation governance refers to public legislation
approximately 40 professionals across international and regulatory considerations in the provision
health systems representing a range of industries, of precision medicine. These include mandates
organizations and governments. This research set forth by ministries of health, market-review
pointed to the four foundational areas outlined in processes and policy-driven initiatives enabled by
Figure 3: (1) innovation governance; (2) innovation the country’s governing bodies.
Financing and Implementing Innovation in Healthcare Systems 9Innovation financing refers to the funding mix Workforce and infrastructure describes the
to both set up and regularly reimburse precision broader health system, human capital and economic
medicine programmes. This may range from considerations necessary to support a precision
stand-alone public or private financiers, public- medicine marketplace. Such foundational elements
private funding partnerships and specific financing are necessary to power the discovery and economic
mechanisms for precision medicine. sustainability of precision medicine innovations.
Community and partnerships refers to the The following sections will explore these four areas
methods by which multistakeholder collaboration and their attendant capabilities. The discussion of
emerges in an innovation ecosystem. In the each area will reflect how capabilities mature as
precision medicine context, this area explores health ecosystems adopt and incorporate more
the changing role of various stakeholders and the precision medicine approaches overall. Each area is
conditions needed to enable innovation. supported by illustrative ongoing case studies.
Financing and Implementing Innovation in Healthcare Systems 10Below: 101cats, Getty Images
1 Innovation governance
The public legislation and regulatory
considerations in the provision of
precision medicine.
Financing and Implementing Innovation in Healthcare Systems 111.1 Precision medicine implementation roadmap
from national strategic plans
Summary: Some level of government-driven implementation builds the national public confidence and
market demand for precision medicine approaches that private entities cannot build alone. The national
strategic plan is created based on country-specific population health burdens and a shift to preventative care.
A precision medicine national strategy consists first adopted at the private health provider level.
of both: (1) the aspirational metrics of the ministry Even in this latter scenario, performance evaluation
of health (or equivalent government decision- of such private activity may provide compelling
making agencies) to incorporate precision medicine cases for national precision medicine programmes.
approaches into care; and (2) the steps and These national initiatives have taken a variety of
partnerships outlined to achieve such targets within a approaches: rare diseases, oncology, population
certain time frame. Top-down policy entrepreneurship health surveillance and infrastructure development
from key government leaders inspires the early activity are among the many focus areas of the 36-plus
of the precision medicine innovation ecosystem formal national or international strategies for precision
through large-scale national initiatives, barring specific medicine developed as of 2019. A snapshot is
cases in which precision medicine approaches are captured in Figure 4 below.
FIGURE 4 Ongoing government-funded national genomic-medicine initiatives
100
Data
90
80
Data Data
70 Mexico
Y-Axis (Y-axis subhead)
Data
Data
60
Brazil Data
50
China
40
Data
Data
30
Data
Data Data
Source: Stark et. al (2019) Data
20
Data Data
Data Data
Data
10
Data
0 Data
1 2 3 4 5 6 7
United Kingdom France United States of America Qatar
X-Axis
Genomics England 2012 – 100,000 Genomic Medicine Plan 2016-2025 – National Human Genome Research Qatar Genome 2015 – Infrastructure,
genomes: rare disease, cancer £350M Rare disease, cancer, diabetes Institute 2007 – Infrastructure and population cohort
($485M) €670M ($799M) clinical cohorts $427M
Scottish genomes: £6M ($8M) All of Us 2016-2025 – Population
Welsh Genomics for Precision cohort $500M (first two years) Japan
Medicine: £6.8M ($9M)
Northern Ireland Genomic Medicine
Switzerland Japan Genomic Medicine Program
Centre: £3.3M ($4.6M) Swiss personalized health network 2015 – Infrastructure, clinical and
2017-2020 – Infrastructure CHF68M Brazil population-based cohorts, drug
($69M) Brazil 2015 – Brazil Initiative on discovery JPY10.2B ($90.05M)
Precision Medicine
Netherlands Infrastructure, disease and population
RADICON-NL 2016-2025 – cohorts China
Rare disease
Estonia
Health Research Infrastructure Estonian Genome Project 2000 – Precision Medicine Initiative –
Infrastructure and population-based 1,000,000 genomes CNY60B
Saudi Arabia
cohort 2017: €5M for 100,000 ($9.2B)
individuals Saudi Human Genome Program
Denmark 2013 – Infrastructure, clinical cohorts
and population-based cohorts
Genome Denmark 2012 – DK86M Australia
($13.5M) SAR300M ($80M)
Finland Australian Genomics 2016-2021 –
FarGen 2011-2017: DK10M
($1.6M) National Genome Strategy Infrastructure, rare disease and cancer
Infrastructure, population-based 2015-2020 – Infrastructure €50M Turkey AUD$125M ($95M)
cohort, pathogen project ($59M) Genomics Health Futures Mission
Turkish Genome Project 2017-2023 – 2018-2028 – AUD$500M ($372M)
Infrastructure, clinical and population-
based cohortsInitial use cases The readiness of a national strategic plan will as to the value of such initiatives. In countries
may be prioritized rest heavily on the government’s prioritization of where precision medicine is further embedded in
based on: (1) precision medicine in its health system. In countries clinical care – more commonly occurring in high-
where precision medicine implementation is income countries – there is greater opportunity
disease burden;
low – typically in LMICs – the ministry of health to implement public-driven initiatives given the
(2) applicability to
(or equivalent) will likely have existing strategic increased resourcing and established foundation.
population health programmes for other disease areas such as As markets develop and more research and funding
surveillance; and (3) infectious or neglected tropical diseases, given are able to drive discovery, the national strategy
a shift to preventative other national health priorities. If precision medicine may include additional initiative types or enter
care, particularly for is included in the national strategy, however, other disease areas. A table of illustrative initiative
non-communicable initial use cases may be prioritized based on: (1) types are presented in Table 1, referenced from the
diseases, and disease burden; (2) applicability to population Global Alliance for Genomics and Health (GA4GH).
provision of health surveillance; and (3) a shift to preventative Greater private-sector participation is also observed
information to the care, particularly for non-communicable diseases, alongside maturing markets, which is further
government. and provision of information to the government discussed in the innovation financing section.
TA B L E 1 Selected precision medicine innovation types drawn from the 221 initiatives categorized
by the Global Alliance for Genomics and Health (GA4GH) Catalogue
Initiative type Description
Biological material repositories that collect and store
Biobank/repository
biospecimens for medical research
Operational or infrastructure collaborations with
Consortium/collaborative network
multiple partner organizations
Storage of organized health and genomic-relevant
Database
information, often for catalogue and research use
Collaborative partnerships with industry
stakeholders such as health providers, payers,
Industry
pharmaceutical companies or diagnostic/
sequencing companies
Standardized vocabularies and terminology of
Ontology or nomenclature tool
biological or clinical information
Knowledge-generating and sharing initiatives, often
Research network/project
through academic-driven partnerships
Standards Common, shared-information frameworks
Other frameworks, platforms, architecture or
Tool
programmes relevant to precision medicine
Case study – Nigeria’s Cancer Control Plan by guidelines for cancer management from the
the Ministry of Health formalizing support of National Comprehensive Cancer Network (NCCN)
precision medicine implementation: In 2015, Clinical Practice Guidelines in Oncology, which is
the Nigerian Ministry of Health announced its new now taught in Nigeria’s medical training curriculums.
Cancer Control Plan (CCP), which lends formalized The CCP outlines measures to support the Nigerian
support for country-wide implementation of government’s screening programme, with the goal
approaches such as DNA-based cancer testing. of screening half the eligible Nigerian population
Supported by the Clinton Health Access Initiative, by 2022. Over the long term, the Ministry of Health
the American Cancer Society and the US National aims to incorporate routine screening of eligible
Cancer Institute, the CCP established updated cancers into existing clinical programmes.
Financing and Implementing Innovation in Healthcare Systems 13Case study – US NIH All of Us initiative: In that include data and research centres (DRCs),
2018, the US National Institutes of Health (NIH) biobanks, patient participant centres, healthcare
launched the All of Us initiative (formerly known as provider organizations and community engagement
the Precision Medicine Initiative Cohort Program) partnerships. Specific steps exist through the data
with the objective of collecting genomic and collection process (consent forms, survey modules,
health data on a longitudinal basis from 1 million partner protocols) and particular attention is paid
volunteers in the United States. The initiative acts to strategic enrolment of demographics under-
as a consortium with 100-plus programme partners represented in biomedical research.
1.2 Open science principles
Summary: Policy applications of open science can inspire data-driven research and development (R&D) of
precision medicine innovations. The guiding principles are described by the international GO FAIR initiative,
and should be considered alongside relevant biodata standards.
In contrast to the more proprietary intellectual Open science for precision medicine research may
property ownership models based on trade secrecy manifest in various initiatives – e.g. international
and competition, open science – as defined by multistakeholder genome research consortia,
the Organisation for Economic Co-operation and centrally accessible databases for research and
Development (OECD)’s Making Open Science clinical use or collaborative efforts that avoid
a Reality paper – is a movement enabled by duplicated work. In moving towards implementation,
information and communication technologies various open science guiding principles described
towards transparent data sharing, rapid knowledge by the international GO FAIR initiative enable
dissemination and broad research accessibility. researchers to find, access, interoperate and reuse
Emerging policy applications of open science each other’s research objects. These are described
have particular relevance in the field of precision in Figure 5 below. Readiness benchmarks for
medicine as they are: (1) changing how genomics- innovation can be found in: (1) the extent to
related research is conducted and disseminated; which countries follow the FAIR principles; (2) the
and (2) reducing the otherwise high costs and agreed-upon standards for exchanging genomic,
uncertainty risk of precision medicine if actors were phenotypic and clinical data attributes; (3) the
to invest resources in R&D as stand-alone initiatives. recognition of participant consent and privacy; and
Importantly, the access to higher volumes of data (4) the extent to which open science principles are
as a result of open science principles can improve implemented alongside proper intellectual property
both the granularity of information for stratified care (IP) ownership regulations.
delivery and the applicability of clinical research
discoveries to increased patient populations.
FIGURE 5 The FAIR Guiding Principles
To be Findable: To be Interoperable:
F1. (meta)data are assigned a globally unique and persistent identifier I1. (meta)data use a formal, accessible, shared and broadly applicable language for
knowledge representation.
F2. data are described with rich metadata (defined by R1 below)
I2. (meta)data use vocabularies that follow FAIR principles
F3. metadata clearly and explicitly include the identifier of the data it describes
I3. (meta)data include qualified references to other (meta)data
F4. (meta)data are registered or indexed in a searchable resource
To be Accessible: To be Reusable:
A1. (meta)data are retrievable by their identifier using a standardized R1. meta(data) are richly described with a plurality of accurate and relevant attributes
communications protocol
R1.1. (meta)data are released with a clear and accessible data usage licence
A1.1 the protocol is open, free and universally implementable
R1.2. (meta)data are associated with detailed provenance
A1.2 the protocol allows for an authentication and authorization procedure,
where necessary R1.3. (meta)data meet domain-relevant community standards
A2. metadata are accessible, even when the data are no longer available
Source: Wilkinson et. al (2016)
Financing and Implementing Innovation in Healthcare Systems 14Early collaboration with large-scale research groups environment, policy-makers may note inefficiencies,
and involvement in international human genomics with current access restrictions to scientific and
initiatives may accelerate the formation of a country’s research data (e.g. isolated research groups/health
precision medicine innovation ecosystem. To start this systems, data-sharing restrictions across country
process, policy-makers have a greater opportunity borders), which limits the potential for holistic
to design early opt-in incentives for institutionalizing precision medicine R&D advancement for all involved
open science and set a precedent for national, participants. The Canadian government’s Roadmap
centralized biorepositories with interoperability and for Open Science provides an excellent model
collective contribution requirements. Such early to move beyond such restrictions. The Canadian
attention to open-science principles can prevent roadmap outlines the steps they took to make federal
the emergence of the potentially ad hoc or isolated science open to all, while respecting privacy, security,
genomic research programmes more common in ethical considerations and appropriate intellectual
existing precision medicine programmes. In this latter property protection.
Case study – Structural Genomics Consortium precompetitive principles adopted by the SGC
(SGC): A charitable open science public-private translate into other open science conditions
partnership, the SGC was started in 2003 with imposed on its researchers and collaborators via
the core mandate of determining 3D structures a trust agreement that prohibits the patenting of
of human proteins and parasite proteins that are research outputs using SGC data and stipulates
of biomedical importance (e.g. potential drug fast dissemination and prepublication sharing
targets). It is recognized as one of the earliest and of its structural and chemical biology outputs.
largest pioneers of open science and releases The current collaborators with SGC include
structural genomics data into the public domain pharmaceutical companies, governments,
through the Protein Data Bank annually. The academic institutions and NGOs.
Case study – Estonian Genome Center of the as the Biobanking and Biomolecular Resources
University of Tartu (EGCUT) Biobank: Estonia’s Research Infrastructure (BBMRI), the European
biobank initiative, the EGCUT Biobank, has been Research Infrastructure Consortium (BBMRI-ERIC)
funded by the Estonian government since 2000 and the Public Population Project in Genomics
and includes a collection of health and genetics (P3G). The EGCUT maintains a data-sharing policy
data from 5% of Estonia’s adult population. The in which collaborators accessing EGCUT resources
EGCUT actively collaborates with many universities, must send any scientific results obtained from the
research institutions and consortia given its research project using EGCUT data or samples back
membership in various international networks such to EGCUT for addition to the Estonian Biobank.
1.3 Precision medicine IP policy and benefit sharing
Summary: Clear intellectual property (IP) policy for precision medicine technologies acts as a market
incentive for precision medicine innovators. These policies may not be so effective in all locations,
particularly those with little precision medicine infrastructure, and require other push-and-pull measures.
Benefit-sharing models aim to close the gap regarding equitable distribution of value from international
precision medicine projects.
Policy-makers The core question to be addressed by policy- awareness of the risks and rewards associated
must assess the makers for precision medicine IP can be with an innovation, such as when conducting
strength of this summarized as: “Do current national patent regimes freedom-to-operate searches. Likewise, it may
promote R&D and innovation in precision medicine offer similar risk-and-reward insight to public-sector
‘pull’ incentive
while also benefitting the collective interest of public organizations considering opening their databases
on private-sector
health?” While IP protections are critical incentives to commercial parties.
engagement relative for precision medicine innovators, policy-makers
to the benefits of must assess the strength of this “pull” incentive on However, the incentive effects of IP policies may
open innovation private-sector engagement relative to the benefits be limited or non-existent for precision medicine
principles that aim to of open innovation principles that aim to collectively innovations addressing health problems mainly
collectively advance advance precision medicine in the long term. affecting LMICs due to the small and uncertain
precision medicine in market demand for such innovations. The World
the long term. A recommended benchmark for readiness is Health Organization states that there is a need
the clear articulation of IP policies for precision for other governance responsibilities, financial
medicine innovations. Clearly defined rules provide mechanisms and multistakeholder partnership
researchers and technology producers with an models to fill the IP incentive gap.
Financing and Implementing Innovation in Healthcare Systems 15IP incentive gaps are also observed among LMIC- used by LMICs; or (2) arrange country-specific
based researchers participating in international socially responsible licensing via negotiations with
open genome projects; while data is contributed by respective government agencies if their citizens
and accessible to all involved countries, wealthier provided data. To be sure, this does not address
nations are more likely to patent innovations mechanisms to overcome the lack of representation
from such projects. The MalariaGEN case study of LMIC populations in large genomic datasets.
illustrates an early use of benefit-sharing models More work on equitable collaboration models is
to close this gap in its international open science encouraged, particularly for genomics IP and locally
initiative. Under this model, the IP owner can: (1) relevant data. This topic is further discussed in the
provide differential access or pricing for innovations “workforce and infrastructure” section.
Case study – Malaria Genomic Epidemiology types (including genetic data) across collaborators.
Network’s (MalariaGEN) data release policy: Under its online data release policy, the website
MalariaGEN is an international open science project states that “the owner of the IP agrees to license it
to identify the specific genetic underpinnings of on a reasonable basis for use in the developing world
malaria funded by the Grand Challenges in Global and on a preferential basis to the countries whose
Health Initiative through the Bill & Melinda Gates citizens are the subject of the database”. This acts
Foundation and the Wellcome Trust. Thirty-nine as a benefit-sharing mechanism for LMICs that can
countries currently participate in the MalariaGEN more equitably gain from participating in MGEN’s
research consortium, sharing and integrating data open data project in the event of royalty flow from IP.
1.4 Adapted health technology assessment
processes for precision medicine technologies
Summary: The integration of precision medicine technologies into standard market-review processes
remains a gap in both high- and low-income countries. If traditional health technology assessment
methods are unable to properly evaluate precision medicine, novel refinements must be adopted by
pharmacoeconomic groups globally.
The inclusion of Novel precision medicine technologies may not ways through the approval process and may
precision medicine fit traditional evaluation and market approval include, but is not limited to, faster regulatory review
in clinical practice processes for biotechnologies. While conventional timelines, clear pricing standards and relevant
HTA aims to evaluate the social, economic, medical feedback. However, in creating robust
has been recognized
organizational and ethical issues of a health frameworks to formally evaluate precision medicine
to ‘impact each intervention, its systematic evaluation varies from technologies, HTA bodies may struggle to keep
stage of the country to country and often does not cover pace or may attempt some level of ad hoc review
Health Technology novel scenarios posed by precision medicine processes on such technologies. Nonetheless,
Assessment technologies. The inclusion of precision medicine these traditional review processes may not fully
(HTA) process, in clinical practice has been recognized to “impact account for accurate end points that capture the
from scoping and each stage of the Health Technology Assessment value-add of a precision medicine innovation.
modelling through to (HTA) process, from scoping and modelling through
decision-making and to decision-making and review”. For instance, To date, only a few HTA bodies internationally
review’. pharmacogenomic tests (PGx) that assess the accommodate specific considerations or traditional
adverse event (AE) risks of certain therapeutics evaluation methods for precision medicine – e.g.
may not fit into traditional assessment models the Diagnostic Assessment Programme at the
given their indirect effect on patient outcomes if the National Institute for Health and Care Excellence
resulting information gain from the PGx diagnostic (NICE) in the UK, the Health Interventions and
changes the course of care. This variability in the Technology Assessment Program (HITAP) in
patient pathway may be more difficult to capture in Thailand and the HTA Access Point in Australia.
standard HTA analyses. Australia’s entry point for companion diagnostics
(CDx) is illustrated as a case study below. The
Refinements to various components of HTA are conduct of enabling studies to collect the necessary
encouraged, so as to adapt to the influx of precision data for HTA processes is further discussed in the
medicine algorithms, digital health applications and “reimbursement conditions and coverage models”
“omics”-based tests. This may manifest in various section under innovation financing.
Financing and Implementing Innovation in Healthcare Systems 16Case study – Australia’s HTA Access Point for to be assessed by more than one expert advisory
Co-dependent Diagnostic Technologies: The committee. The latter is particularly relevant for
Australian government’s Department of Health designated “co-dependent technologies” such
established a “single entry point”, the HTA Access as targeted therapies that require companion
Point (HTAAP), that assists specific applicants for diagnostic technology, in which HTAAP will work
HTA and reimbursement if they are uncertain about with the applicant to form a tailored assessment
the funding programme for which their technology methodology for the eligible technology in the hope
may be eligible, or where their technology may need that assessment timelines may be reduced.
Financing and Implementing Innovation in Healthcare Systems 17Below: THANIT, Getty Images
2 Innovation financing
The funding mix to both set up and regularly
reimburse precision medicine programmes.
Financing and Implementing Innovation in Healthcare Systems 182.1 Public funding and fiscal space usage
Summary: Government spending on precision medicine programmes demonstrates national support and
encourages private-sector funding for the innovation ecosystem. LMIC settings with constrained fiscal
space may seek to prioritize precision medicine initiatives when relevant to other national health priorities.
Expansion of targeted budgetary allocations over time remains necessary for sustainable public funding of
precision medicine.
Public funding is often vital in spurring precision financing involvement from private innovators as the
medicine programmes and such investment precision medicine market matures. This funding
will depend on the fiscal space – defined as the shift from top-down public spending to greater
budgetary allocation for a targeted investment private investment activity is commonly observed
area – available from the ministry of finance or as the innovation ecosystem supports further
respective treasury agency. While constrained translation of R&D.
by limited resources, especially relative to private
funding, public spend can either fully establish or While LMICs may not have the available fiscal
partially underwrite early innovation programmes via space for precision medicine due to the focus
an initial mix of grant-based and/or loan financing. on other national health priorities (e.g. infectious
Additionally, public grants are often used to finance disease or neglected tropical diseases), public
research initiatives in both academic institutions funding is crucial in the early stages of the
and relevant private projects. These government innovation ecosystem. In such scenarios where
investments often take the form of milestone-based precision medicine is a national investment
tranche payments or upfront disbursements but will interest, precision medicine programmes may
depend on the project arrangement. be incorporated into related health initiatives of
national focus with already ongoing fiscal spending
While national precision medicine programmes may (e.g. infectious disease surveillance or genomic
ultimately be financed by a variety of public- and studies of rare disease prevalent in the country). For
private-funding sources, the readiness criteria for emerging and more developed economies able to
public funding are defined by: (1) the fiscal space consider greater public investment, the fiscal space
allocation to precision medicine from existing remains particularly important to de-risk a reliance
budgets or new funding channels; and (2) the on private aid. The World Health Organization
diversity of investment partners supporting the (WHO) notes that creating fiscal space expansions,
project portfolio. The former is based on the ability often driven by macroeconomic growth, budget
of a government’s budgetary leadership to audit reprioritization and efficiency improvements,
the return on investment (ROI) of potential precision enables the long-term financial sustainability of
medicine efforts, measure the national priority level precision medicine approaches. The government
and understand the value from precision medicine of Thailand case study below demonstrates
approaches. As the innovation ecosystem matures, the effective use of public funding for precision
a greater evidence base of early precision medicine medicine approaches as an upper-middle-income
initiatives can provide traction for further public country, first within Stevens-Johnson syndrome
funding. Similarly self-perpetuating, the latter and toxic epidermal necrolysis (TEN) in 2004,
benchmark highlights the diversity within types of now into larger-scale five-year genome catalogue
precision medicine innovation and the growing co- initiatives (2019).
Case study – public financing of the Genomics syndrome (SJS) and toxic epidermal necrolysis
Thailand Initiative and pharmacogenomics (TEN) screening, with investments from the Thai
(PGx) studies: In early 2019, the Thai government Ministry of Science and Technology. The project
approved a $150 million five-year initiative to used genetic testing to identify epilepsy patients
catalogue the genomes of 50,000 citizens. This at risk of developing these severe responses to a
project, named the Genomics Thailand Initiative, widely prescribed generic medication, and ultimately
aims to expand the understanding of South-East reduced such cases by 80% in 2018. Continuing
Asia’s under-represented genomic composition its precision medicine efforts, the most recent
and advance Thailand’s genomics capabilities Genomics Thailand Initiative falls under the broader
in personalized diagnostics, drug selection and remit of the Thailand 4.0 government campaign
treatment in various disease areas. Thailand pursued to close the middle-income country gap towards
precision medicine research as early as 2004 an innovation-driven economy, starting with a
through the Thai PGx Project for Stevens-Johnson competitive genomic medicine industry.
Financing and Implementing Innovation in Healthcare Systems 192.2 Private-sector financial backing
Summary: Private funding provides the funding scale and expertise to support precision medicine
initiatives. Engagement by different types of financiers (innovation producers, investors, aid donors and
service providers) may shift as the innovation ecosystem grows. Private financing of precision medicine is
scarce in LMICs, even from development assistance for health (DAH) groups.
In tandem with the public fiscal space, private specific funds and development assistance for
funding provides the funding scale and expertise health (DAH) groups (e.g. foundations, family
to support the continuous development of the offices or high-net-worth individuals [HNWIs]). As
precision medicine innovation ecosystem. These the marketplace develops, producers – ranging
private-sector financiers range in funding size and from biopharmaceutical to diagnostic/sequencing
involvement – examples may include development companies (and investors), including private equity
aid agencies, global health philanthropies, and venture capital (PEVC) groups – actively deploy
biopharmaceutical companies, technology financial and in-kind advisory/expertise resources
manufacturers and impact investing groups among for precision medicine projects. Such private-sector
various other NGO and corporate investors. The stakeholders enter as the innovation ecosystem
expansion of the precision medicine marketplace grows to provide lower-risk operating environments
is often supported by increased public-private co- and tenable investment exit opportunities. At
financing partnerships and individual private-sector advanced stages of readiness, service providers
activity (further discussed as a readiness benchmark such as private provider systems are able to self-
under the “public funding” section above). finance precision medicine approaches within their
clinical practices.
Readiness benchmarks for private-sector financing
activity can be viewed as the extent to which Private financing of precision medicine technologies
private stakeholders are able to participate in and in the LMIC context remains scarce. Attention
fund projects in the innovation ecosystem. Policy- from DAH groups and global innovative financing
makers are encouraged to conduct a stakeholder mechanisms (IFIs), including The Global Fund, Gavi,
analysis of relevant private actors in order to the Vaccine Alliance and Unitaid, have historically
map potential collaboration opportunities and focused on preventing and treating infectious
current funding gaps in the precision medicine diseases such as HIV/AIDs, tuberculosis and
marketplace. Private funders for precision medicine malaria. As such, non-communicable diseases
can be broadly categorized into: (1) innovation (NCDs) including cancers and cardiovascular
producers; (2) investors; (3) aid donors; and disease – areas in which precision medicine
(4) service providers. Importantly, the mix of approaches are currently most applicable – remain
stakeholders shifts depending on the maturity of underfunded through traditional global health DAH
the innovation ecosystem. For instance, countries and IFI channels. This remains a notable gap that
in the early stages of private financing activity is further highlighted in the DAH financing disease
primarily attract aid donors in the form of disease- area breakdown in Figure 6.
FIGURE 6 Development assistance for health (DAH) by financing channels
Development assistance for health by channel of assistance, 1990-2018
40 Regional development banks UNICEF, UNFPA, UNAIDS, Unitaid, PAHO
World Bank Other bilateral development agencies
US foundations China bilateral
35 International NGOs Australia bilateral
US NGOs Canada bilateral
Gates Foundation France bilateral
30
CEPI Germany bilateral
Global Fund UK bilateral
Gavi USA bilateral
25
Billions of 2018 $
WHO
20
15
10
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1990
1991
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1993
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1996
1997
1998
1999
2000
2001
2002
2002
2003
2004
2005
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2011
2012
2013
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2015
2016
2017
YearCase study – series A financing of 54Gene by has predominantly taken a private-financing approach
Adjuvant Capital: Founded in 2019, 54Gene is an to grow its capabilities and positioning in the wider
African genomics research, services and development precision medicine marketplace, with a total of $21.5
company established with the aim of including million raised venture capital funding stimulated by
under-represented African genomic data in health initial participation in accelerators (Y Combinator
research and drug development. In April 2020, the and Google Developers Launchpad). While servicing
US- and Nigeria-based genomics company 54Gene more than 100,000 participants in its biobank to
announced a $15 million series A financing round expand African representation in population genetics
led by Adjuvant Capital, a global health-focused as of 2021, 54Gene has also been able to build up
investment fund for life science technologies backed infrastructural gaps on the African continent by both
by the Bill & Melinda Gates Foundation, Novartis and upskilling the workforce and strengthening genomics
the International Finance Corporation (IFC). 54Gene R&D capabilities.
Case study – private equity financing of largest oncology treatments alongside an array of imaging
precision medicine provider in Morocco by and genomic diagnostic services. In addition
Alta Semper: Frontier markets private equity firm to financial capital, Alta Semper aims to help
Alta Semper has invested in Morocco’s largest ODM’s expansion strategy by providing operating
oncology, radiology and diagnostics services expertise across north and sub-Saharan Africa,
platform, Oncologie Diagnostic du Maroc (ODM). alongside access to its existing healthcare holdings
The provider owns and operates five facilities for in Morocco.
Case study – UBS $500 million Oncology Impact Foundation will manage any such generated capital
Fund: Swiss multinational bank UBS Group raised from initial early-stage oncology investments and
~$500 million from HNWIs for investment by MPM, specifically direct this flow to: (1) academic grants
a healthcare venture capital firm with oncology to promising oncology-related research; and (2)
expertise. The fund serves as an impact investment health access initiatives in developing countries.
initiative that invests in early-stage cancer This combination of HNWI impact investors
treatments and, in turn, manages the royalties (scale) and VC expertise (domain knowledge and
and performance fees from successful drug experience) presents a potential replicable financing
sales of such investments for impact-based key model for private-driven impact investing in
performance indicators (KPIs). The UBS Optimus precision medicine technologies.
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