Access to Medicine Index 2018 - Access to Medicine Index 2018 Methodology Report 2017
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Access to Medicine Foundation
Access to Medicine
Index 2018
METHODOLOGY
Access toREPORT
Medicine Index 2018
Methodology Report 2017
ACCESS TO M E D I CI N E FOU N DATI O N
September 2017
1
Methodology for the 2018 Access to Medicine Index
ACKNOWLE DG E M E NTS
The Access to Medicine Foundation would like to thank the following
people and organisations for their contributions to this report.
FU N D E RS
Bill & Melinda Gates Foundation
The UK government
The Netherlands Ministry of Foreign Affairs
E XPE RT R E VI E W CO M M IT TE E
Hans Hogerzeil (Chair)
Sanne Frost-Helt
Fumie Griego
Suzanne Hill
Frasia Karua
Dennis Ross-Degnan
Dilip Shah
Yo Takatsuki
Joshua Wamboga
Prashant Yadav
TECH N I C AL SU BCO M M IT TE ES R ES E ARCH TE AM E D ITO R IAL TE AM
Esteban Burrone Danny Edwards Anna Massey
Nick Chapman Beth Boyer Deirdre Cogan
Jennifer Dent Clarke Cole
Michele Forzley Luca Genovese
Warren Kaplan Catherine Gray
Jillian Kohler Nestor Papanikolaou
Niranjan Konduri Tara Prasad
Stine Trolle
AD D ITI O NAL CO NTR I B UTO RS
We would like to thank the many other experts who contributed their
views to the development of this methodology (see page 56).
ACCESS TO M E D I CI N E FO U N DATI O N
The Access to Medicine Foundation is an independent non-profit org-
anisation based in the Netherlands. It aims to advance access to medi-
cine in low- and middle-income countries by stimulating and guiding the
pharmaceutical industry to play a greater role in improving access.
Naritaweg 227-A
1043 CB, Amsterdam
The Netherlands
For questions about this report, please contact Danny Edwards,
Research Programme Manager
dedwards@accesstomedicinefoundation.org
+31 (0) 20 215 35 35
www.accesstomedicineindex.org
2Access to Medicine Foundation
A good practice
framework
There is no simple blueprint for making medicine accessible During ten years of research, we have identified real progress
to all who need them. Often, the poorest people must tackle from the pharmaceutical industry and best practices in many
complex and changeable barriers before they can access the areas linked to access: in R&D for neglected diseases, in new
health products they need. Nevertheless, huge strides are business models that serve low-income populations, and in
being made on major global health challenges – eradicat- a variety of maturing access initiatives that are making real
ing guinea worm, bringing out new medicines for tuberculo- change. Yet in other areas, the pace of change remains slow,
sis and hepatitis C, vaccinating a generation of girls against most notably in pricing.
cervical cancer. There are indeed tools and solutions availa-
ble that can take us a long way forward in improving access to In 2018, we will publish a new update in our Index research.
medicine. We will be working in the meantime to show how this meth-
odology report can be used to prioritise which actions com-
Our focus at the Access to Medicine Foundation is on the panies should take. Pharmaceutical companies need willing
role of the pharmaceutical industry. My team and I pres- and able partners to work with them to improve access and
ent here the current framework for pharmaceutical industry to continue the slow-burning move away from the traditional
good practice regarding access to medicine in low- and mid- pharma business model. We invite global health teams work-
dle-income countries, in the form of the metrics for the 2018 ing with and within companies, as well as investors, donors
Access to Medicine Index. They have been identified through and governments, academics and NGOs to use this method-
our proven consensus-building model. We conducted a series ology when working to develop healthy markets and healthy
of targeted stakeholder consultations to test and explore populations.
society’s current expectations of pharmaceutical companies
in 2017.
Our discussions resulted in a tightly focused methodology
that efficiently identifies where companies have the greatest
potential to make change. In priority areas, the Index analysis
will also deepen. For example, in 2018, the Index R&D analy-
sis will match company pipelines against the urgent R&D pri-
orities set by WHO and others. The timely inclusion of cancer
in the scope of the Index reflects the view that a transac- Jayasree K. Iyer
tional relationship is no longer enough. Companies must Executive Director
also engage in improving the continuum of care for cancer Access to Medicine Foundation
patients, and align with the growing prioritisation of cancer
care in low and middle income countries.
3Methodology for the 2018 Access to Medicine Index
Table of contents
Acknowledgements 2 TECH N ICAL ARE AS
Message 3 A General Access to Medicine Management 36
B Market Influence & Compliance 37
EXECUTIVE SU M MARY 6 C Research & Development 38
D Pricing Manufacturing & Distribution 39
I NTRO DUCTI O N E Patents & Licensing 40
Improving access to medicine in 2017 10 F Capacity Building 41
G Product Donations 42
REVI EWI NG TH E M ETHO DO LOGY
How the Index captures changes in the I N D ICATO RS PE R TECH N ICAL AREA
access-to-medicine landscape 12 A General Access to Medicine Management 43
B Market Influence & Compliance 44
KEY D ECISI O NS AN D D ISCUSSIONS 14 C Research & Development 46
▶ C A N CER I N SCO PE D Pricing Manufacturing & Distribution 49
How can the Access to Medicine Index bring cancer E Patents & Licensing 51
into its scope? 15 F Capacity Building 52
▶ PR I O R I T Y R& D G Product Donations 54
What are pharmaceutical companies doing to answer
calls for urgently needed R&D? 17 APPE N D ICES
▶ ACCESS PL A N N I N G I. Contributors to this report 56
Is it time for access planning to become standard II. Priority countries for pricing and registration –
practice during development? 18 2018 update 58
▶ A SSESSI N G I M PAC T III. Cancers in scope for the 2018 Access to
How should pharmaceutical companies assess the Medicine Index 61
impact of access initiatives? 19 IV. Good practice standards framework for the
▶ D O N AT I O NS Capacity Building analysis 63
Can donation programmes provide sustainable V. Priority diseases and pathogens for R&D analysis 64
access to medicine? 20 VI. ICD-10 coverage & (cancers only)
WHO EML relevance 66
WHAT WE M E ASU RE VII. References 78
Company Scope 22 VIII. Definitions and acronyms 79
Disease Scope 24
Geographic Scope 28
Product Type Scope 31
HOW TH E I N D EX M E ASU RES
The Analytical Framework: revealing the actions
that matter most for access 34
Analytical framework 35
4Access to Medicine Foundation
LIST O F FI GU RES LIST O F TAB LES
Figure 1. Analytical Framework for the 2018 Access Table 1. Analysis scopes for the 2018 Access
to Medicine Index 8, 35 to Medicine Index 7
Figure 2. 2017 Methodology Review for the 2018 Table 2. Companies included in the 2018 Access to
Access to Medicine Index 12 Medicine Index - 20 companies 22
Figure 3. Cancers in scope for R&D: poorer countries Table 3. List of diseases, conditions and pathogens
shoulder large burdens 15 included in the 2018 Access to Medicine Index 27
Figure 4. Market cap & revenue of companies listed Table 4. List of countries included in the 2018 Access
in the 2018 Access to Medicine Index 23 to Medicine Index – 106 countries 30
Figure 5. Breaking down the 2018 disease scope 24 Table 5. Priority countries 58
Figure 5a. Diseases and pathogens only in scope Table 6. Exceptions to the priority country table 60
for R&D 24 Table 7. Cancer types in scope and basis for inclusion
Figure 5b. Diseases and pathogens on independent for the R&D Technical Area 61
R&D priority lists 24 Table 8. Cancer types in scope and basis for inclusion
Figure 6. Low- and middle-income countries shoulder for product deployment analyses 62
the bulk of disease burdens 25 Table 9. Inclusion and scoring criteria for capacity
Figure 7. Defining the disease scope – building initiatives 63
screening protocol 26 Table 10. Priority diseases and pathogens analysed in
Figure 8. Countries included in the 2018 Access to the Research & Development Technical Area 64
Medicine Index - 106 Countries 28 Table 11. Priority pathogens 65
5Methodology for the 2018 Access to Medicine Index
Executive Summary
Globally, two billion people cannot access the medicine they lytical scopes and the development of new measurements
need. Huge advances in global health are being made, and yet where needed. Throughout this process, the team debated
new challenges continue to emerge. Among the many stake- a range of issues with governments, multilateral organisa-
holders working to improve access, pharmaceutical companies tions, research institutions, non-governmental organisations
have a critical role to play. In 2017, the Access to Medicine Foun- (NGOs), investors, patient organisations, policy centers and
dation has built consensus on how pharmaceutical companies pharmaceutical companies.
can address current global health priorities. This report descri-
bes the consensus-building process and how the latest cycle Discussions covered specific questions relating to pharma-
has shaped the methodology for the 2018 Access to Medicine ceutical company policy and practice, as well as broader per-
Index. The refined methodology has a tighter focus on where spectives on the role for the industry regarding access. With
companies have the largest potential for impacting access. the assistance of its formal committees of independent
experts, the Index team balanced the viewpoints provided to
The Access to Medicine Index analyses 20 of the largest identify workable ways forward. Strategic guidance was pro-
research-based pharmaceutical companies with products for vided by the Foundation’s Expert Review Committee (ERC),
high-burden diseases in low- and middle-income countries. an independent body of experts from, among others, WHO,
The Index ranks these companies according to their efforts governments, NGOs, patient organisations, the industry, aca-
to improve access to medicine in these countries. It identifies demia and investors.
best practices, highlights where progress is being made, and
uncovers where critical action is still required. In this way, the Analysis scopes in 2018
Index provides both a guide and an incentive for pharmaceuti- The 2018 Index will measure the same 20 companies as in
cal companies working to do more for people who lack access 2016, as they remain the largest R&D-based pharmaceuti-
to medicine. cal companies with the most relevant expertise and portfo-
lios. Considering their size, resources, pipelines, portfolios and
In 2016, the Access to Medicine Index reported that pharma- global reach, these companies have a critical role to play in
ceutical companies are getting more sophisticated in how improving access to medicine. The majority have consistently
they get essential products to poor people. However, good qualified for inclusion since 2008. Their efforts to improve
practice was found to be limited to a narrow range of prod- access to medicine will be assessed across 106 low- and mid-
ucts and countries, and many opportunities to expand good dle-income countries and in relation to 77 high-burden dis-
practice are yet to be acted upon. eases, conditions and pathogens.
The Index methodology is updated every two years to take 69 indicators
account of new developments and emerging challenges in The Index research team applied stricter standards than
access to medicine. Each methodology review is informed by in 2015 for deciding when to retain, strengthen, merge or
a wide-ranging multi-stakeholder dialogue coordinated by the remove a metric. As a result, the methodology has a tighter
Access to Medicine Foundation. For more than ten years, the focus on where action by pharmaceutical companies has
Foundation has built stakeholder consensus on what we can the greatest potential for improving access to medicine. It
expect from pharmaceutical companies. provides a robust framework for efficiently tracking com-
pany performance. The 2017 methodology comprises 69
Fine-grained review and consensus building indicators: four are mergers of pre-existing ones and 15 have
The Index research team began the 2017 review with a fine- been removed. Five new indicators were developed in
grained evaluation of the 2016 indicators and data sets, response to changes in global health priorities, including one
checking the robustness and continuing relevance of each that specifically recognises R&D targeting priority R&D gaps
measure in turn. The outcomes led to adjustments to ana- or needs, as identified by stakeholders such as WHO.
6Access to Medicine Foundation
KE Y CHAN G ES
• Targeted analysis of priority R&D. WHO and others have • Closer analysis of behaviours that facilitate access to
called for R&D to be urgently prioritised for specific diseases medicine. The Access to Medicine Index measures four
in order to address urgent public health issues. The 2018 aspects of pharmaceutical company behaviour – transpar-
Index will analyse how companies are responding through an ency, commitment, performance and innovation (referred to
assessment of R&D for priority diseases. More than half of as Strategic Pillars). Their relative importance varies depend-
the disease scope (45 out of 77) have an identified priority ing on the action in question, whether it is negotiating volun-
R&D gap or need, including for new diagnostic products, vac- tary licenses, marketing activities or capacity building initia-
cines or medicines. tives, for example. For the first time, this variation has been
captured in the Index’s analytical framework.
• Cancer is now in scope. Cancer incidence continues to rise
in low- and middle-income countries. These countries shoul- • New metrics for capturing the quality and impact of
der a considerable proportion of the global cancer burden, access initiatives. In 2018, the Index will take a deeper look at
and are increasingly prioritising cancer care in national health- the quality of companies’ capacity building initiatives, by com-
care plans. In 2018, the Index will assess companies’ actions paring them against a framework of good practice standards
to improve access to cancer control for the first time. Cancers developed by the Index research team. The Index will also
that place a high burden on public health will be analysed in expand its analysis of where and how companies monitor and
R&D, while cancer medicines on the latest WHO Model List of measure the impact of their access-to-medicine activities.
Essential Medicines (2017) qualify for analyses of pricing, pat-
enting and donations practices.
Table 1. Analysis scopes for the 2018 Access to Medicine Index
CO M PAN Y SCO PE G EO G R APH I C SCO PE
20 companies 106 low and middle-income countries
• Selected based on a combination of market • 31 low-income countries
capitalisation and relevance of portfolio for • 52 lower-middle-income countries
access to medicine. • 23 upper-middle-income countries
D I S E A S E SCO PE PRO D U C T T Y PE SCO PE
77 diseases, conditions and pathogens 8 types
• 21 communicable diseases Medicines, microbicides, preventive vaccines,
• 14 non-communicable diseases therapeutic vaccines, vector control products,
• 20 neglected tropical diseases platform technologies, diagnostics, contraceptive
• 10 maternal & neonatal health conditions methods and devices.
• 12 priority pathogens
7Methodology for the 2018 Access to Medicine Index
Figure 1. Analytical Framework for the 2018 Access to Medicine Index
The 2018 Access to Medicine Index analyses company behaviour using a The new approach to weighting the Strategic Pillars has been developed
framework of 69 indicators organised in seven Technical Areas. The frame- by the Foundation research team and tested both with an external expert in
work’s four pillars correspond to four aspects of behaviour. For the first time ranking analytics and the Index’s Expert Review Committee. Final weights
in 2018, the weight of each pillar now varies between the Technical Areas, of each Technical Area within the four pillars will be set during data analy-
giving a more sensitive reflection of where these behaviours matter most. sis, once new indicators have been confirmed as robust and can be fully inte-
grated into the 2018 Framework. Target weights are indicated in the figure
In 2018, the target overall weights of the Strategic Pillars remain closely below.
aligned with the weights agreed in 2015 by the Expert Review Committee.
In 2015, these weights were: 15% for Commitments, 25% for Transparency,
50% for Performance and 10% for Innovation.
Analytical Framework for the 2018 Access to Medicine Index
4 STRATEGIC PILLARS
Commitments Transparency Performance Innovation
13.3% (avg.) 23.2% (avg.) 54.1% (avg.) 9.4% (avg.)
10% General Access to Medicine Management
10% Market Influence & Compliance
7 TECHNICAL AREAS
20% Research & Development
25% Pricing, Manufacturing & Distribution
15% Patents & Licensing
15% Capacity Building
5% Product Donations
Strategic Pillar weights: the target distrubution of pillar weights across Technical Areas in 2018
Commitments Transparency Performance Innovation
13.3% (avg.) 23.2% (avg.) 54.1% (avg.) 9.4% (avg.)
8Access to Medicine Foundation
The 2018 Access to
Medicine Index
Methodology 2017
9Methodology for the 2018 Access to Medicine Index
I NTRO DUCTI O N
Improving access to medicine in 2017
All people share the right to the highest attainable stand- from globalisation,9 which could present a deeper crisis for
ard of health, as noted in the WHO Constitution. Yet access global cooperation in areas such as health.
to medicine continues to be out of reach for an estimated
two billion people worldwide. Huge advances are being Progress is being made
made toward internationally agreed global health targets. Nevertheless, during the same period, progress toward global
Nevertheless, new and complex health challenges continue to health targets has continued, demonstrating that effective
emerge, demanding sustained commitment and deeper coop- approaches are being developed and applied. For example,
eration from many different sides, as well as wider adoption child mortality dropped by almost 50% between 1990 and
of proven solutions. Providing access depends on a complex 2013. There has been a 48% decline in AIDS-related deaths
range of factors and stakeholders. since the peak of the HIV/AIDS epidemic in 2005,10 and more
than half of all people living with HIV are accessing antiret-
Development aid for health has slipped since the first decade roviral therapy.11 In 2015, 71% of countries had an NCD plan
of this century as government budgets have tightened. Aid addressing cancer, up from 50% in 2010.12 In 2017, the WHO
grew only 0.1% between 2015 and 2016, compared to growth World Health Assembly, endorsed a set of measures to
rates of up to 11.4% annually between 2000 and 2010.1 This improve cancer control.13
slow-down is particularly concerning for low-income coun-
tries that rely heavily on aid to maintain the health of their Breakthroughs in R&D continue to bring new promise. Direct-
populations.1 Yet, in many cases this gap is not being filled acting antivirals mean country-by-country elimination of hep-
by recipient governments. In many countries in sub-Saha- atitis C is a real possibility. Immunotherapy has become a
ran Africa and in low-income countries, government health clinically validated treatment for many cancers,14 with mor-
expenditure as a percentage of GDP has also been in decline tality from cancer dropping by 23% since 1991 in the United
in recent years.2 States.15 Recent advances in gene-editing technology hold
further promise for cancer control.16
While budget growth has slowed, crises and new trends have
posed further challenges to global health. For example, the Cooperation to limit antimicrobial resistance (AMR) is also
Ebola outbreak in 2014 led to over 11,000 deaths in West strengthening, with multiple initiatives starting up in recent
Africa.3 This was followed by the Zika outbreak in early 2015, years, such as the Combating Antibiotic Resistant Bacteria
which quickly spiked to almost 3,500 suspected and con- Biopharmaceutical Accelerator (CARB-X) and the Global
firmed cases in Central America in early 2016.4 Antimicrobial Antibiotic Research and Development Partnership (GARDP) in
resistance is growing and already causes more than 700,000 the field of R&D. Numerous pharmaceutical companies have
deaths each year worldwide.5 Rapid urbanisation, worsen- signalled their readiness to play a part in addressing AMR
ing diets, increasingly sedentary lifestyles and aging popu- by signing the Davos Declaration on Antibiotic Resistance
lations are contributing to a rise in non-communicable dis- and the Industry Roadmap for Progress on Combating
eases (NCDs).6 Climate change is expected to cause a quarter Antimicrobial Resistance.17,18
of a million additional deaths per annum from malnutrition,
malaria, diarrhoea and heat stress between 2030 and 2050.7 Critical role for pharmaceutical companies
In 2017, the need for all stakeholders to take action on access
Geopolitical and societal factors are also influencing the to medicine remains clear, with each having their own appro-
shape of the global health landscape. The World Economic priate role and responsibilities. Pharmaceutical companies
Forum has identified economic disparity and global govern- control unique products that can greatly alleviate disease bur-
ance failures, the decline of trust in institutions, and persisting dens; they also have the expertise to meet the need for new
gender inequalities as contributors to a fractured health land- and adapted innovative products; the power to address the
scape.8 Some see these global risks as factors in a move away affordability of those products; and the ability to strengthen
10Access to Medicine Foundation
supply chains and support healthcare infrastructures. When
pharmaceutical companies take positive action, it can have a
profound effect on people’s lives. Over the past ten years, the Access to Medicine Index has identified increas-
ing engagement by pharmaceutical companies in access to medicine.
In 2016, the Access to Medicine Index reported that pharma-
ceutical companies are getting more sophisticated in how
they get products to poor people, and are addressing global For example, the disease scope has been adjusted in line with
health priorities for example in R&D. However, good practice changing views on which diseases should be prioritised for
was found to be limited to a narrow range of products and improving access to medicine. In 2008, the Index focused
countries, and many opportunities to expand good practice mainly on Neglected Tropical Diseases (NTDs) as defined by
are yet to be acted upon. WHO, expanding to include high-burden diseases including
NCDs in 2010. The latest refinement in this direction is the
The work of the Access to Medicine Foundation inclusion of cancer in the 2018 Access to Medicine Index.
The Access to Medicine Index analyses 20 of the top
research-based pharmaceutical companies with products for The geographic scope has also been refined, to ensure it
high-burden diseases in low- and middle-income countries. covers countries where greater access to medicine is needed
The Index ranks these companies according to their efforts to most. Many countries have moved into higher World Bank
improve access to medicine. It identifies best practices, high- classifications over the lifespan of the Index: 72% of the
lights where progress is being made, and uncovers where crit- world’s poor now live in middle-income countries.19 To adapt
ical action is still required. In this way, the Index provides both to these demographic changes, the 2014 Index adopted
an incentive and a guide for pharmaceutical companies to do measures of human development and inequality in its country
more for people who still lack access to medicine. inclusion framework, to bring some higher income countries
with low levels of equality into the Index scope.
Over the past decade, the Access to Medicine Foundation has
developed a robust process for building consensus among a The 2018 Access to Medicine Index
wide range of stakeholders on what society expects of phar- In 2017 the Foundation has completed the 6th review of its
maceutical companies regarding access to medicine in low- methodology for the Access to Medicine Index. The 2018
and middle-income countries. These expectations are then Access to Medicine Index will measure the same 20 compa-
translated into metrics that form the basis of the methodol- nies as in 2016. Considering their size, resources, pipelines,
ogy for the Access to Medicine Index. portfolios and global reach, these companies have a criti-
cal role to play in improving access to medicine. The refined
The Index methodology is updated every two years in methodology comprises 69 indicators, covers 106 countries
line with developments in access to medicine following a and 77 diseases, conditions and pathogens. The Foundation
wide-ranging multi-stakeholder dialogue coordinated by the will now begin the process of data collection, verification,
Access to Medicine Foundation. The dialogue draws together scoring and analysis, before publishing the next Access to
the views of NGOs, governments, the industry and multi-lat- Medicine Index in late 2018. The Foundation will also use this
eral organisations, in order to build consensus on how and latest methodology to provide guidance to pharmaceuti-
where pharmaceutical companies can and should be improv- cal companies on where the priorities now lie, and how they
ing access to medicine. match with the many solutions and practices identified in pre-
vious iterations of the Index.
How the Index has responded to global challenges
As a result, the Index methodology has evolved continually
since the first Access to Medicine Index was publishd in 2008.
11Methodology for the 2018 Access to Medicine Index
REVI EWI NG TH E M ETHODOLOGY
How the Index captures changes in the
access-to-medicine landscape
Each Access to Medicine Index is the result of a two-year pro- CO M M IT TE E CO N SU LTATI O N S
cess known as the ‘Index cycle’, which begins with a targeted Throughout each Methodology Review, formal committees
review of the Index methodology. The aim is to confirm the support the Index team. Summaries of discussions and deci-
global priorities regarding access to medicine and define how sions are provided in the next section. Recommendations
society expects pharmaceutical companies to contribute. for specific areas of the Index are provided by Technical
The emphasis is on defining ambitious, but achievable actions Subcommittees of specialists in different aspects of access
for companies to take. For this latest review, the Foundation to medicine. Strategic guidance is provided by the Expert
drew on more than a decade of experience in building con- Review Committee (ERC), an independent body of experts,
sensus on where pharmaceutical companies can take action, including from WHO, governments, patient organisations, the
before translating it into robust metrics. The result is the industry, non-governmental organisations (NGOs), academia
methodology for the 2018 Access to Medicine Index. and investors. The ERC met twice to review proposals for the
scope, structure and analytical approach of the 2018 Index
The process for the methodology review has been developed and to ratify the final methodology.
over six Index cycles. It includes a series of internal checks on
indicators, data sets, measures of behaviour and on analyti- Expert Review Committee in 2018
cal approaches. This is followed by an external review, during Hans Hogerzeil (Chair) University of Groningen
which the consensus view is sought between a range of Sanne Frost-Helt Denmark Ministry of Foreign Affairs*
stakeholders on specific access topics and the role for phar- Fumie Griego International Federation of Pharma-
maceutical companies, as well as on the analytical scopes and ceutical Industry Associations
the appropriate weights for the areas measured by the Index. Suzanne Hill World Health Organization
Frasia Karua Amref Health Africa
The primary principles of the Methodology Review are: (1) Dennis Ross-Degnan Harvard Medical School
that all metrics are robust and data can efficiently and fea- Dilip Shah Indian Pharmaceutical
sibly be collected; (2) that the Index is responsive to chang- Association
ing access needs; and (3) that all metrics are relevant to the Yo Takatsuki BMO Global Asset Management
appropriate role for pharmaceutical companies in improving Joshua Wamboga International Alliance of Patients’
access to medicine. Organizations
Prashant Yadav Harvard Medical School
Figure 2. 2017 Methodology Review for the 2018 Access to Medicine Index
ERC Meeting I ERC Meeting II
June 2017 July 2017 Methodology
Index 2016 for 2018 Index
Sept 15 2016 Sept 2017
Company Scopes Resolving Final adjust-
Indicator and
feedback on and metrics specific ments following
data checks
2016 Index discussions questions ratification
INTERNAL EXTERNAL REVIEWS & CONSENSUS BUILDING FINALISATION
REVIEWS
*At time of ERC meetings
12Access to Medicine Foundation
I NTE R NAL R E VI E W O F I N D I C ATO RS AN D DATA CO N S E N SUS B U I LD I N G AN D D IALO G U E
The Foundation reviewed each of the indicators of the 2016 The Foundation has built stakeholder consensus on what we
Access to Medicine Index for robustness, response quality and can expect from pharmaceutical companies for more than
the potential for companies to improve access to medicine a decade. While disagreement persists in key areas, such as
through a series of quantitative and qualitative analyses: pricing and the management of intellectual property, overall
• Distribution analyses. Assessing the distribution of scores the depth of consensus on the appropriate role for pharma-
per indicator to check the spread of company behaviour in ceutical companies has grown. In 2017, the Foundation’s pro-
the 2016 Index. This indicates whether expectations of com- cess of consensus building has once again underpinned meth-
panies are fair (large clusters of low scores may indicate odological changes for the 2018 Access to Medicine Index.
expectations may be too high) and the extent of room for The Foundation strives to ensure that the consultation pro-
improvement. Outcomes inform refinements to indicators cess is wide-ranging, independent, transparent and includes
and scoring guidelines. the engagement of key global health experts.
• Response rate analyses: Assessing company response rates
to each data point requested in the 2016 Index. This con- Review and engagement process
firms whether questions are clear and whether companies The stakeholder dialogue was targeted toward priority areas
can feasibly gather data per question; it can also indicate the and topics identified by the Foundation’s research team for
relevance companies assign per question and/or their will- discussion with experts. Topics were prioritised through:
ingness to disclose information. internal analyses of data and indicators, independent reviews
• Correlation analyses: Indicator-level assessments of score of the Index research during the 2015-16 period of analysis,
correlations, which help diagnose less relevant indicators, and a review of developments in access-to-medicine theory
and can reveal or confirm positive or negative relationships and practice. The Foundation team also engaged with the
between related areas of company behaviour. companies measured by the 2016 Index and its associated
• Qualitative indicator review: A battery of qualitative assess- data-collection processes.
ments of each indicator, including clarity of expectations
and role for companies, continuing relevance to access to The Foundation’s research team then reached out to a broad
medicine, potential for longitudinal comparisons and the range of experts through a targeted stakeholder engage-
‘change-making’ potential of each indicator. ment exercise. Experts were identified from relevant organi-
sations, through a review of the literature, and recommenda-
These tests were used to identify where scoring guidelines tions from other stakeholders. The research team engaged
could be tightened, detect and eliminate the risk of redun- with experts and stakeholders from a wide range of back-
dant measures, and pinpoint opportunities for enhancing data grounds to ensure alternative viewpoints and technical exper-
quality. In 2017, the Foundation applied stricter standards for tise were incorporated. This included discussions with repre-
deciding when to merge or remove a metric. These standards sentatives of multilateral organisations, research institutions,
were linked to: the relevance of the measured behaviour to NGOs, investors, and companies (see Appendix).
access to medicine; clarity regarding the industry’s role; and
the degree of consensus among stakeholders regarding how The Foundation used the views gathered to inform its pro-
companies should behave. During the indicator review, topics posals for modifications to the methodology. These propos-
were identified for discussion during the next phase of con- als were discussed in detail with the Index’s Technical Sub-
sensus building and stakeholder dialogue. Committees and ERC. The recommendations and strategic
guidance provided by the ERC in particular helped to identify
ways forward where disagreement or uncertainty existed in
areas of measurement.
13Methodology for the 2018 Access to Medicine Index
REVI EWI NG TH E M ETHODOLOGY
Key decisions and discussions
Discussions held during the method- In this section:
ology review were wide-ranging and
rich. In many cases, there was align- ▶ C A N CER I N SCO PE
ment on the behaviours that the 2018 How can the Access to Medicine Index bring cancer into its scope?
Access to Medicine Index should meas-
ure and how. In others, it was difficult to ▶ PR I O R I T Y R& D
find consensus. In these cases, the Index What are pharmaceutical companies doing to answer calls for
team, with its Technical Subcommittees urgently needed R&D?
and Expert Review Committee, iden-
tified workable ways forward, balanc- ▶ ACCESS PL A N N I N G
ing the evidence and viewpoints gath- Is it time for access planning to become standard practice during
ered. This section highlights discussions development?
where the appropriate decision was
contested, or where discussions led to ▶ A SSESSI N G I M PAC T
new areas of measurement. How should pharmaceutical companies assess the impact of access
initiatives?
▶ D O N AT I O NS
Can donation programmes provide sustainable access to medicine?
14Access to Medicine Foundation
▶ C A N CER I N SCO PE
HOW CAN TH E ACCESS TO M E D ICI N E I N D EX B RI NG CANCE R I NTO ITS SCO PE?
Cancer is one of the world’s leading causes of death, and now comparing evidence gathered during the 2015 review, with
accounts for 1 in 6 deaths worldwide.20 Clearly, cancer is a pri- new developments and viewpoints on cancer prioritisation
ority global health issue. However, providing good cancer care and the opportunities for pharma companies.
is an almost uniquely complex challenge, requiring prevention,
screening, diagnosis, referral, treatment and palliative care, Cancer incidence continues to rise in low- and middle-income
among other steps. In poorer countries, the necessary infra- countries, with such countries shouldering a large propor-
structure and resources for delivering this care are typically tion of the burden (see figure 3).22 Three further medicines
weak or limited. Although the majority of countries have a have been added to the WHO EML (in 2017).23 In the same
national cancer control plan (NCCP) in place, in poorer coun- period, R&D activity for cancer treatment has expanded rap-
tries, the necessary infrastructure and resources for deliver- idly, and the global oncology market is now expected to grow
ing cancer care are typically weak or limited. China, India and by almost a third to USD 150 billion by 2020.24
Brazil, for example, have relatively strong
health systems that are better equipped for the
management of cancer, while countries such Figure 3. Cancers in scope for R&D: poorer countries shoulder
as Kenya and South Africa do not yet meet large burdens
basic infrastructure requirements for cancer The 2018 Index will analyse company pipelines for 17 cancer types. These have been
treatment.21 selected based on their incidence either globally or in countries in scope. For most of
these cancer types, more than 50% of the incidence is in the 106 low- and middle-income
Cancer has not previously been included in the countries in the scope of the Index.
scope of the Access to Medicine Index. When
Lung
its inclusion was last discussed, in 2015, stake-
Breast
holders expressed contrasting views: for exam-
Stomach
ple, that the need for greater action to improve
Liver
cancer control had triggered WHO to add 16
Colorectal
cancer medicines to its Model List of Essential
Cervical
Medicines (WHO EML); that there is a need to
Oesophageal
stimulate companies to address the affordabil-
Prostate
ity of cancer medicines in countries with con-
Leukaemia
strained finances; that companies can only play
Lip, oral cavity
a limited role in improving cancer support sys-
Non-Hodgkin lymphoma
tems; that the Index should instead prioritise
Brain, nervous system
diseases with a more critical need for access
Bladder
to treatment, including typical childhood kill-
Gallbladder
ers with known and effective treatments on the
Other pharynx
market. Nasopharynx
Kaposi sarcoma
During the 2017 Methodology Review, the
0 0.5 1 1.5 2
research team once again examined whether
● Incidence in countries in scope Incidence (mn)
cancer should be brought into the Index scope, ● Global incidence rate
15Methodology for the 2018 Access to Medicine Index
At the 2017 WHO World Health Assembly, delegates agreed pharmaceutical companies can build capacity at all levels of
a resolution on cancer prevention and control, urging greater health services across the cancer continuum of care in low-
efforts to “promote the availability and affordability of qual- and middle-income countries. Furthermore, the companies
ity, safe and effective medicines (for cancer), in particular, share an opportunity to increase access to affordable medi-
but not limited to, those on the WHO Model List of Essential cine. Together, these companies produce 34 of the 46 unique
Medicines (WHO EML).25 cancer medicines on the WHO EML (2015).
In 2017, the Access to Medicine Foundation carried out a
first study of how pharmaceutical companies are address-
ing cancer control. It found that 16 companies were engaged
in 129 diverse initiatives in low- and middle-income coun-
tries.26 The range and volume of initiatives indicates that
D ECI S I O N : C AN CE R I S I N SCO PE FO R TH E 2018 I N D E X
On reviewing new developments with stakeholders, and with type in low-resource settings. The cancers in scope for R&D
strategic guidance from the Expert Review Committee, the are selected based on global incidence, on incidence in coun-
Foundation decided to include cancer for the first time in the tries in the scope of the Index, and where the burden was dis-
scope of the Access to Medicine Index in 2018. In its analy- proportionately high in low- and middle-income countries
sis, the Index will acknowledge where possible the context
of national cancer care systems. The 2018 Index will examine When it comes to registered products, an external prioriti-
27 cancer types: 17 in the R&D Technical Area, and 19 in the sation does exist. Cancers in scope for product deployment
Technical Areas relating to pricing, patenting and donations are selected based on whether there are relevant registered
(see Appendix). Nine cancers are in both sets. In Capacity products on the WHO EML (2017), highlighted in the recent
Building, the Index will include all initiatives related to cancer. cancer resolution as those needing particular focus when
considering availability and affordability. This focuses the
Bringing cancers into scope for R&D highlighted a signifi- analysis on a subset of cancer products identified by WHO as
cant omission in the global health landscape: an absence of essential for the treatment of cancer.
prioritisation regarding cancer-care research needs in low-
and middle-income countries. Therefore a proxy was needed; The decision to include cancer in the 2018 Index scope is
incidence was highlighted as the most robust indication of described in more detail in Cancer Control 2017 (by the
whether further R&D was needed to treat a particular cancer International Network for Cancer Treatment and Research).
16Access to Medicine Foundation
▶ PR I O R I T Y R& D
WHAT ARE PHARMACEUTICAL CO M PAN I ES DO I NG TO ANSWE R CALLS FO R U RG E NTLY N E E D E D R&D?
There are many diseases without adequate or effective treat- Stakeholders were clear that companies could be expected to
ments available, or where the products are not sufficiently tai- act and incentivised to do more in this low-incentive space.
lored to meet the needs of people living in low- and middle-
income countries. Pharmaceutical companies have much to Following these discussions, the Foundation identified and
add in this space: addressing such ‘product gaps’ is a core reviewed published, independently defined lists of prior-
expertise of the industry. However, there is a mismatch in ity product gaps and research needs. Such prioritisations can
incentives. Commercial incentives remain a primary driver for stimulate R&D by providing guidance and directing resources
pharmaceutical R&D. The product gaps and research needs to where they are most needed. The Index offers an addi-
that matter more to people living in low- and middle-income tional incentive in the form of recognition for R&D that tar-
countries and less to people in wealthier countries typically gets these priorities. The Index team found R&D prior-
offer little or no commercial incentive to engage in pharma- ity lists defined by global health stakeholders, such as WHO,
ceutical R&D. for a range of Communicable Diseases, Neglected Tropical
Diseases and Maternal & Neonatal Health Conditions.
Despite this, companies can and do engage in R&D with However, no priority list has yet been developed to iden-
less commercial promise, for example through collaborative tify R&D needs within the field of Non-Communicable
models such as Product Development Partnerships (PDPs), Diseases (NCDs). On reaching out to stakeholders, it was rec-
which can facilitate risk- and expertise-sharing. As a first anal- ognised that very limited work has been done in this field.
ysis in this space, the 2016 Access to Medicine Index looked Stakeholders identified the absence of an external prioritisa-
at whether pharmaceutical companies were addressing ‘high- tion list for NCDs as a significant concern that needed to be
need, low-incentive’ product gaps. It found that 31 out of 84 addressed by the global health community.
of the gaps analysed were being addressed by one or more
companies, largely through partnerships, and through a com- The prioritisation lists identified by the Index team define spe-
bined total of 151 projects. This analysis compared companies’ cific product gaps that are disproportionately needed by pop-
pipelines with priority product gaps identified by Policy Cures ulations in low- and middle-income countries, as well as gaps
Research (G-FINDER) for diseases already included in the linked to potential global health threats, such as emerging
Index disease scope.27 infectious diseases and pathogens that have developed anti-
biotic resistance. To address the lack of a prioritisation list for
During the 2017 methodology review, the Foundation sought NCDs, stakeholders endorsed the Index team’s proposition to
to expand this analysis to draw in a more comprehensive include R&D projects for NCDs that demonstrably address a
range of diseases where a pressing research need or need specific to populations in low- and middle-income
product gap had been identified. In discussions with stake- countries.
holders, emerging infectious diseases such as Ebola and Zika
were cited as diseases that were not within the Index scope,
but where R&D was of critical value – and where companies
have shown clear evidence of engagement.
D ECI S I O N : E XPAN D D I S E A S E SCO PE TO C APTU R E I N D USTRY R ES PO N S ES TO R& D PR I O R ITI ES CU R R E NTLY I D E NTI FI E D
The disease scope has been expanded to include all diseases, The five lists are:
conditions and pathogens with an identified product gap on
the five independently compiled lists of product gaps and • G-FINDER neglected diseases, products and technologies
R&D needs that are deemed priorities for public health. The (2017);27
aim is to provide a complete analysis of how the companies • G-FINDER reproductive health areas, products and technol-
in scope are addressing such R&D priorities. This analysis will ogies (2014);28
aid global health stakeholders in understanding where R&D • WHO R&D Blueprint (2017);29
is taking place, and recognise and encourage companies to • WHO Initiative for Vaccine Research gaps (2017)30
address all priority gaps on these lists. • WHO pathogen priority list for R&D of new antiobiotics
(2017)31
17Methodology for the 2018 Access to Medicine Index
▶ PL A N N I N G FO R ACCESS
IS IT TI M E FO R ACCESS PL AN N I NG TO B ECO M E STAN DARD PR ACTI CE DU RI NG PRO DUCT
D EVE LO PM E NT ?
Pharmaceutical companies start working on their market Views among stakeholders have since shifted; the con-
access strategies while products are still in development. sensus now is that companies should apply the lessons
Their aim is to secure strong market positions for new prod- they have learned from access planning in PDPs and bring
ucts, and they generally target markets with high potential them in-house. Indeed, companies in many cases already
profitability. It is less common for companies to also plan for do so. Looking only at late-stage R&D projects, the 2016
access for populations in less profitable markets during devel- Index showed that 41% of projects conducted by compa-
opment. These access plans aim to make successful innova- nies in-house had associated access provisions. Importantly,
tions rapidly available for patients in low- and middle-income expanding this expectation would capture companies’ plans
countries, and at affordable prices and support their rapid for more projects targeting NCDs. These projects typically
uptake. happen in-house, rather than in collaboration. Given the
increasing burden of NCDs in low-and middle income coun-
During the methodology review, the Foundation’s research tries, the need for companies to also make new NCD products
team asked stakeholders to consider whether companies can rapidly accessible is growing.
be expected to step up their access-planning and integrate it
more deeply into their businesses. Is it time for access plan- On the question of timing (i.e., when access planning should
ning to become standard practice during development? take place), stakeholders tended to agree that broad com-
mitments – e.g., to ensure the affordability of the product on
In the 2016 Index, access provisions were expected only when approval – can be made very early in development. However,
they were part of collaborative research projects, usually with they were also clear that, in most cases, it is not possible to
PDPs. During previous methodology reviews, the stakeholder develop detailed access provisions tailored to local contexts
view was that access planning was more likely in such part- until at least phase II clinical development.
nerships than in projects conducted by companies in-house.
The 2014 Index showed 39% of projects carried out in collab-
oration had plans for access in place, rising to 51% in the 2016
Index.
D ECI S I O N : B ROAD E R M E A SU R E M E NT O F ACCESS PROVI S I O N S
Stakeholders agreed that it was now time to broaden the This means it will now look more comprehensively at access
focus of the Index’s measurement of access provisions: that provisions for R&D projects targeting NCDs. Regarding
companies can now be expected to plan for access for all pro- timing, the 2018 Index will expect advance planning for access
spective products that are needed in low- and middle-income for projects from phase II. This provides a clearer expectation
countries. Following this shift, the Index adjusted its measure and point of focus for early consideration of access.
to recognise all access provisions, whether for R&D carried
out in partnership or in-house.
18Access to Medicine Foundation
▶ A SSESSI N G I M PAC T
HOW CAN TH E I N D EX M E ASU RE TH E I M PACT O F ACCESS I N ITIATIVES?
The pressure to show that initiatives to improve access to Discussions held during the Foundation’s 2017 Methodology
medicine actually work is growing, particularly as pharmaceu- Review confirmed that such moves are viewed as a step in
tical companies are expanding their engagement in access ini- the right direction. Stakeholders see value in pharmaceuti-
tiatives in low- and middle-income countries. Governments, cal companies working with third parties and each other to
NGOs, patient groups and communities increasingly expect develop and fine tune their approaches to impact measuring,
to see a measureable impact. The companies themselves as well as in sharing information about their results and suc-
also seek a greater understanding of what works and what cesses. However, there is still no agreement among stake-
doesn’t, to demonstrate and build on success and avoid holders on how to best define impact, or on the most appro-
repeating past failures. priate models for assessing the impact of pharmaceutical
companies’ access initiatives. Stakeholders have also high-
The increasing focus on impact measuring started with the lighted risks that stem from confusion between outcome and
global development community, driven by several economic impact measurements.
and political factors. For example, many funding agencies
have reduced or retargeted their development budgets, while Stakeholders argued for transparency regarding impact meas-
major donors have pushed hard for greater demonstration of urement, specifically in terms of companies sharing informa-
‘value for money’. At the same time, there is a growing public tion about their approaches and whether they work, as well
perception that five decades of development assistance – in as the results of their evaluations, so that a wider community
time, money and other resources – have not led to the hoped- of actors can learn from them.
for effects. This perception has put pressure on donors, and
consequently other actors in international development such In 2016, the Index evaluated whether companies or their
as NGOs, academia and the private sector, to do a better job partners carry out impact assessments for donation pro-
of demonstrating clear, tangible results that can be under- grammes. When the measure was developed in 2015, these
stood by both their peers and the general public. programmes were identified through stakeholder consulta-
tion as the most likely focus of impact measurement. A study
Several pharmaceutical companies have already started to from Boston University has since confirmed this position;33 it
announce, plan and carry out impact assessments of their found that 31 out of 47 published evaluations related to dona-
access initiatives. For example, University College London tion programmes. However, many stakeholders and pharma-
recently carried out a study of Novo Nordisk’s Base of the ceutical companies now expect more; impact assessments are
Pyramid projects. This initiative aims to facilitate access to now viewed as possible and potentially instructive in a variety
diabetes care for people in work, but on low incomes, in cer- of access initiatives, from inclusive business models, to health
tain low- and middle-income countries.32 Boston University systems strengthening activities.
has started a programme that aims to measure the impact of
initiatives associated with Access Accelerated: an industry ini-
tiative to prevent NCDs and improve access to care in low-
and lower-middle income countries.
TH E D ECI S I O N : I N D E X TO LO O K FO R B ROAD E R E FFO RTS TO E VALUATE I M PAC T
For the 2018 Index, companies’ efforts to evaluate impact will nise those companies taking steps and making plans to meas-
also be measured in the Technical Areas of General Access ure impact, share information about the variety of ways they
to Medicine Management and Capacity Building, as well as engage in this work, including with third parties, and credit
in Product Donations. More specifically, the Index will recog- those companies that take steps to publish the results.
19Methodology for the 2018 Access to Medicine Index
▶ D O N AT I O NS
CAN DO NATI O N PROG R AM M ES PROVI D E SUSTAI NAB LE ACCESS TO M E D I CI N E?
The donation of pharmaceutical products can help to ensure access to pharmaceuticals is better guaranteed through
that the poorest populations – people with no ability to pay models such as equitable pricing or licensing than through
– are able to access the medicines they need. Donations con- donations. Such approaches emphasise affordability for
tinue to demonstrate particular value during humanitarian payers and encourage low- and middle-income country gov-
emergencies, when healthcare infrastructure is damaged and ernments to invest in their health systems. At the same time,
populations are especially vulnerable. Donations have become equitable pricing and licensing can provide companies with a
a core component of global efforts to eliminate, eradicate return on their investments as an incentive to remaining in a
and control neglected tropical diseases, which predominantly given market longer-term.
affect the poorest populations across the world.
Stakeholders agree that donation programmes remain an
Recently, however, some have raised concerns regarding the appropriate approach for improving access to medicine in
long-term sustainability of product donations. For example, certain contexts, particularly for reaching the poorest and
Médecins Sans Frontières recently rejected an offer of pneu- most vulnerable populations. There is also a critical difference
mococcal vaccine donations calling instead for the vaccine between programmes that aim for disease eradication and
to be sold at a discounted price.34 The organisation’s ration- those where eradication cannot be seen as a goal (i.e., pro-
ale is that donation programmes are vulnerable to changing grammes targeteing chronic diseases). Where donations are
priorities within companies, while market-based approaches deemed appropriate, the consensus view is that programmes
are more likely to last.34 Other commentators have noted that must include assessments of how access can be sustainable
donations can disrupt market incentives for generic compe- in the long-term. This means companies working with govern-
tition,35 and emphasised the importance of taking long-term ments to establish plans to ensure recipient populations can
sustainability into account when donating products, especially continue to access treatments for as long as they are needed,
for those targeting chronic diseases.36 even after donation programmes end. Once again, sustainable
approaches are especially pressing where patients suffer from
During the 2017 Methodology Review, the Foundation found a chronic diseases.
growing consensus among stakeholders that sustainable
TH E D ECI S I O N : G R E ATE R E M PHA S I S O N SUSTAI NAB I LIT Y PL AN N I N G ; R E D U CE D W E I G HT
FO R PRO D U C T D O NATI O N S OV E R ALL
Following these discussions, the research team carried out a implemented in a sustainable manner, with a view to the long-
close examination of the Product Donations Technical Area term needs of the populations they serve. Finally, the Index
with the sustainability of access in mind. This led to a reduc- will differentiate between programmes targeting communi-
tion in the overall weight of the Technical Area, from 10% to cable diseases and those targeting NCDs, recognising that
5% of companies’ final Index score. NCD programmes cannot aim for disease eradication and/or
The Index will also apply a more stringent standard regard- elimination.
ing the quality of donation programmes; companies are now
expected to ensure donations programmes are designed and
20Access to Medicine Foundation
What the Index
measures
The Access to Medicine Index assesses company policies and
behaviour regarding specific diseases and product types and
in a specific geographic scope. The following pages set out
the rationale for these analytical scopes and how they have
been defined.
In this section:
CO M PAN Y SCO PE
20 companies
• Selected based on a combination of market capitalisation
and relevance of portfolio for access to medicine.
D I S E A S E SCO PE
77 diseases, conditions and pathogens
• 21 Communicable Diseases
• 14 Non-Communicable Diseases
• 20 Neglected Tropical Diseases
• 10 Maternal & Neonatal Health Conditions
• 12 Priority Pathogens
G EO G R APH I C SCO PE
106 Low and middle-income countries
• 31 Low-Income Countries (LICs)
• 52 Lower-Middle-Income countries (LMICs)
• 23 Upper-Middle-Income Countries (UMICs)
PRO D U C T T Y PE SCO PE
Medicines, microbicides, preventive vaccines, therapeutic vac-
cines, vector control products, platform technologies, diag-
nostics, contraceptive methods and devices.
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