PRMA Insights: Pricing and Reimbursement

Transcription

PRMA Insights:
Pricing and Reimbursement Success
in Rheumatoid Ar thritis
PRMA Insights provide in-depth understanding of current and
future market access realities, developed by industry-experienced
experts with comprehensive cross-functional knowledge. Critical
insight into the clinical and payor landscape is supported by
actionable strategic insights, providing a cornerstone on which to
build a successful market access strategy.
www.prmainsights.com
Pricing and
reimbursement
success
Achieving market success is becoming ever more challenging
as payors seek to rationalize restricted budgets on increasingly
numerous – and frequently expensive – products. Success
requires a thorough understanding of the current P&R landscape,
and how it is changing, and careful, strategic planning – from as
early as Phase 2.
PRMA Insights are a key resource that provides the basis for your
planning. We have analyzed the current treatment landscape
in detail, and have identified the key issues that need to be
addressed in developing the market access strategy.
info@prmainsights.com
Tel (UK): +44 (0)1252 624429
Tel (US): +1 (415) 655 6798
1
Introduction
The RA market is crowded, with eight biologics already available. However, up to a
quarter of patients do not respond adequately to these drugs and thus unmet need
remains. Whilst most of the biologics are reimbursed in the scope countries, use is heavily
restricted by payors seeking to limit the budget impact of these expensive products.
A robust market access strategy is key to
commercial success
will be tempered by patent expiries of the major biologics
and the subsequent launch of biosimilars. Nevertheless,
the market is expected to grow to US$27 bn by 2015.
Manufacturers face considerable challenges in terms of
proving differentiation and defining a place for their products
in the treatment paradigm. However, the biologics provide
a strong argument in favor of earlier intervention in order to
limit progression to joint damage and disability. This offers a
potential an opportunity for manufacturers who can rise to the
challenge of generating robust data to support this approach.
Rheumatoid arthritis (RA) is a chronic, progressive, and
ultimately destructive autoimmune disorder that, in a clinically
established form, affects 0.5–1% of the population worldwide.
Although the biologics are disease-modifying compounds that
slow the progression of joint damage, use is largely restricted
to later lines of therapy, when conventional DMARDS such
as methotrexate have failed. However, about a quarter of
patients have disease that is not adequately controlled
despite the wide range of treatment options available. Thus,
unmet needs remain, even though the market is crowded.
New products with novel mechanisms of action may meet
some of this need. However, demonstrating differentiation
is challenging, and head-to-head trials will be critical.
The global RA market experienced strong growth during
2005–2010, due largely to the launch of biologics such as
Enbrel, Remicade, Humira, and Rituxan, and fuelled further
by the more recent launches of Simponi, RoActemra,
Orencia, and Cimzia. In 2008, the global RA market was worth
US$11.6 bn and accounted for 1.7% of global pharmaceutical
sales. Moderate growth over the next 10 years will be driven
by the expected launch of the eight products currently in
Phase 3 development, offering new mechanisms of action
and in some cases oral administration. However, growth
The biologics offer a realistic possibility of slowing disease
progression in RA, and intervention before signs of joint
damage become manifest has the potential to reduce
joint destruction and subsequent disability and also the
comorbidities associated with RA, such as cardiovascular
disease. However, for earlier treatment to become a reality for
patients, a paradigm shift towards diagnosis of RA at a much
earlier stage is needed. Some authorities are beginning to
advocate earlier diagnosis and therefore intervention; however,
the means by which this can become a reality are far from clear.
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Manufacturers launching into this crowded market face
considerable challenges in terms of demonstrating
differentiation and value for money to increasingly demanding
payors faced with budgetary restrictions. However, a shift
towards earlier diagnosis and therefore treatment of RA
could also represent an opportunity in terms of market access
– for both established and new products – provided that
manufacturers can rise to the challenge in terms of clinical
development. Manufacturers need a clear understanding
of how to design clinical trials and economic models in order
to demonstrate the benefits of intervention at an earlier stage
of the disease. Clearly, building the HTA strategy to support
such an argument will need careful consideration.
Because of their high budget impact, the biologics are high on
payors’ priority lists for limiting expenditure, and the barriers
to market access will continue to increase. Access to biologics
is already restricted in most countries through budgetary caps
and funding restrictions at the national and/or local levels,
restrictive national guidelines, and inter-regional differences
in either the availability of authorized prescribing centers or
the procedures involved in prescription of these products.
Payors will expect manufacturers to proactively offer
financially based risk-sharing models, a strategy used by UCB
to ensure a positive NICE recommendation when introducing
Cimzia into a crowded UK market. Interestingly, this RSA was
subsequently adopted by some Regions in Spain.
Changes in the RA market are being played out against a
backdrop of P&R reform across Europe, no more so than
in Germany with the new AMNOG legislation. Value-based
pricing seems inevitable in the UK when the current PPRS
expires in 2014, and a royal decree in Spain in August 2011
has set out the requirement for cost-effectiveness analysis
in the P&R submission. Manufacturers need to keep
abreast of changes to P&R requirements in the scope
countries and ensure that clinical development plans are
flexible to accommodate changing requirements.
A compelling evidence-based payor value proposition is
required to overcoming market access hurdles, ideally
including head-to-head clinical data. Generation of an
evidence package that is consistent with a manufacturer’s
pricing and commercial ambitions requires early planning
(from Phase 1) and broad cross-functional cooperation.
Failure to adequately plan and invest in the market
access strategy could lead to commercially disastrous
consequences such as HTA and P&R rejections, lower
achievable price, smaller-than-planned reimbursed
population, and extensive market access delays.
PRMA Insights: The Roadmap to Pricing and
Reimbursement Success in Rheumatoid Arthritis
This publication focuses on the P&R issues around the
following scope treatments:
•
•
•
•
•
•
Humira® (adalimumab, Abbott)
Enbrel® (etanercept, Pfizer/Amgen)
Remicade® (infliximab, Merck)
Simponi® (golimumab, Centocor/Merck)
Cimzia® (certolizumab pegol, UCB Pharma)
RoActemra® (tocilizumab, Roche; marketed as Actemra
in the US and Japan)
• Kineret® (anakinra, Biovitrum AB)
• Orencia® (abatacept, Bristol-Myers Squibb)
• MabThera® (rituximab, Roche; marketed as Rituxan in
the US).
A backdrop of global P&R reform
In planning a successful market access strategy, manufacturers need to understand and
accommodate the reforms that are influencing regulatory requirements, HTA, and P&R
across many of the major markets. This PRMA Insights strategic resource considers
such issues, and highlights those that are likely to affect strategy development.
• How will the stringent benefit assessment in Germany
introduced with the AMNOG legislation influence
market access and prices – here and further afield?
• How will value-based pricing be implemented in the UK
and what will be the impact in terms of demonstrating
cost-effectiveness in areas of high unmet need such as RA?
• What impact will greater centralized use of costeffectiveness analysis and collaborative HTA by the
Regions have in Spain?
• How will the market access landscape in Japan
change as an HTA framework is established and costeffectiveness analysis becomes more important?
• How will measures such as comparative trials for
marketing authorization included in the recent reform in
France play out?
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6 steps for pricing and reimbursement success
1
Understanding the current treatment landscape
4
• Reviews of international, national, and local
clinical guidelines
• Epidemiology of the disease and relevant
subpopulations
• Changing approaches and attitudes to treatment
• Analysis of cost burden and presentation of
relevant key data
• Regulatory indications for the scope products
• Measurement of utilities
• Pivotal trial data for the scope products and
overview of key elements of clinical trial design,
such as subpopulations, comparators, and
endpoints
• Critique of cost-effectiveness analysis and
budget impact models
• Payors’ expectations relating to cost arguments,
and how these are changing
• Future treatment landscape – products in Phase 3
and the likely challenges these products will face
2
3
Disease burden
Understanding the HTA and P&R environment
5
Understanding the role of PROs
• PROs as a potential differentiating factor
• Overview of the current P&R process in the
scope countries (US, EU major 5, and Japan)
• What needs to be measured
• Focus on areas that are changing and how this
could affect strategy development
• Expectations of the regulatory and HTA agencies
Comprehensive review and analysis of relevant
HTAs and P&R decisions
• Critical analysis of reviews and decisions made by:
> NICE
> SMC
> AWMSG > CEPS
> TC
> IQWiG > G-BA
> DGFPS
> CIPM
> GENESIS > AIFA
> MHLW
> regional and hospital evaluations in Spain and
Italy
• The value propositions and arguments that
manufacturers submitted
• What worked – and what didn’t
• Payors’ criticisms and expectations
• Suitable instruments
• Strategies for inclusion of PRO claims on the
product label
6
Designing the clinical development
program to support market access
• Expectations of regulatory bodies and payors
• Choice of comparators in clinical trials to meet
payors’ expectations
• Appropriate endpoints
• Importance of patient subpopulations and how
these should be defined
• Measurement of PROs – relevant tools and
instruments
• Ways to achieve differentiation
PRMA Strategic Insight
Area to watch
Developed by our in-house experts, PRMA Strategic Insights
provide critical advice to manufacturers in planning their
market access strategy. They are listed together in each Chapter
Summary and in the Executive Summary, as well as being
located at relevant points in the text.
Issues that could affect
strategy development and
that need to be monitored
are flagged as areas to
watch.
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Table of contents
Each chapter starts with a Chapter Summary, PRMA Strategic Insights, and Areas to watch.
Executive summary
1
Clinical overview
1.1 Disease overview
1.1.1 The pre-articular phase
1.2 Pathogenesis
1.2.1 T and B cells
1.2.2 Cytokines
1.2.3 Morphologic manifestations
1.2.4 Inflammatory mediators as new therapeutic
targets
1.3 Etiology
1.3.1 Genetics
1.3.2 Risk factors
1.4 Clinical presentation and diagnosis
1.5 Classification criteria
1.5.1 ACR criteria
1.5.2 ACR/EULAR criteria
1.6 Measurement of severity
1.6.1 Joint counts
1.6.2 Radiography
1.6.3 Disease Activity Score
1.6.4 SDAI and CDAI
1.6.5 ACR classification of functional status
1.6.6 HAQ-DI
1.7 Disease progression
1.8 Prognostic factors
1.8.1 Biomarkers in RA
1.9 Extra-articular manifestations of RA
2
Epidemiology of rheumatoid arthritis
2.1 Incidence
2.2Prevalence
2.2.1 Prevalence by country
2.3 Epidemiology of early RA
2.4 Impact of RA on morbidity and mortality
2.4.1 Extra-articular manifestations
2.4.2 Other comorbidities
2.4.3 Burden of comorbidities
2.4.4 Mortality
3
Patient-reported outcomes
3.1 Patient-reported outcomes
3.2 Definitions
3.2.1 PROs
3.2.2HRQoL
3.3 Importance of PROs
3.3.1 Why measure PROs in RA?
3.4 PRO instruments in RA
3.4.1 Generic instruments
3.4.2 Rheumatology-specific PRO instruments
3.4.3 PRO instruments used to measure symptoms of RA
3.5 Impact of RA on HRQoL and other PROs
3.5.1 France
3.5.2 Germany
3.5.3 Italy
3.5.4 Japan
3.5.5 Spain
3.5.6 UK
3.5.7 US
3.6 Guidance on use of PROs
3.6.1 FDA guidance
3.6.2 EMA guidance on the measurement of HRQoL
3.7 EMA and FDA guidance on PROs in RA
3.7.1 Achieving a PRO claim
3.7.2 Label claims
Appendix A: PRO instruments used in RA
A.1
Rheumatology-specific instruments
A.1.1 AIMS/AIMS2
A.1.2 MACTAR
A.1.3 QoL-RA Scale
A.1.4 RAID
A.2 Symptom-specific instruments
A.2.1 Pain
A.2.2 Productivity
A.2.3 Fatigue
A.2.4 Sleep
A.2.5 Depression
4
Treatment of rheumatoid arthritis
4.1
Overview
4.2
Non-drug interventions
4.3
Pharmacologic interventions
4.3.1 NSAIDs
4.3.2 Glucocorticoids
4.3.3 Non-biological DMARDs
4.3.4 Biological DMARDs
4.3.5 Paradigm shift in treatment towards early
intervention
4.3.6 Impact of treatment delay
4.4
Clinical guidelines
4.4.1 ACR clinical guidelines
4.4.2 EULAR clinical guidelines
4.4.3 BSR/BHPR clinical guidelines
4.5
Measurement of response to RA therapies in clinical trials
4.5.1 ACR response criteria
4.5.2 EULAR response criteria (DAS28)
4.5.3 BSR definitions of response
4.5.4 Radiographic outcomes
4.5.5 Remission
4.6
Label claims for RA products
4.6.1 FDA guidance
4.6.2 EMA guidance
4.6.3 Overview of label claims
4.7
Biologics approved for the treatment of RA
4.7.1 Enbrel
4.7.2 Humira
4.7.3 Remicade
4.7.4 Simponi
4.7.5 MabThera/Rituxan
4.7.6 Orencia
4.7.7 RoActemra/Actemra
4.7.8 Cimzia
4.7.9 Kineret
4.8
Key characteristics of RA trials
4.9
Safety of the biological DMARDs
4.9.1 Withdrawals
4.9.2 Infections
4.9.3 Tuberculosis
4.9.4 Malignancies and lymphoma
4.10 Products in development
4.10.1 Tofacitinib
4.10.2 Ofatumumab
4.10.3 Sarilumab
4.10.4 Fostamatinib
4.10.5 Iguratimod
4.10.6 Secukinumab
4.10.7 LY2127399
4.10.8 Masitinib
5
Economic impact of rheumatoid arthritis
5.1
National annual costs of RA
5.1.1 Direct and indirect costs
5
5.2
5.3
6
6.1
6.2
6.3
6.4
6.5
6.6
6.7
6.8
6.9
7
7.1
7.2
7.3
7.4
7.5
7.6
7.7
7.8
5.1.2 France
5.1.3 Germany
5.1.4 Japan
5.1.5 Spain
5.1.6 UK
5.1.7 US
5.1.8 Cost of RA by functional status
Utilities
5.2.1 Utilities in rheumatoid arthritis
Pharmacoeconomic evaluations
5.3.1 Assumptions used in the models
5.3.2 Cost-effectiveness studies for biologics
Pricing and reimbursement in the US
Key stakeholders
Healthcare reform
6.2.1 The Affordable Care Act
6.2.2 Effect of healthcare reform on RA
6.2.3 Biosimilars
Pricing of pharmaceuticals in the US
6.3.1 Pricing of infused and injectable drugs
6.3.2 Pricing of oral drugs
6.3.3 Prices and OOP burden depend on type of insurance
6.3.4 Government regulation of drug prices
6.3.5 Manufacturer contracting in RA
Obtaining formulary listing
Formulary management
6.5.1 Tiering
6.5.2 Injectable products
6.5.3 Other utilization management techniques
6.5.4 Management of the pharmacy benefit
6.5.5 Coverage of drug costs
Payor mix for RA
6.6.1 Importance of RA biologics for employers
Formulary status of therapies for RA
6.7.1 Commercial and Medicare Part D formularies
6.7.2 Medicaid state formularies
6.7.3 Coverage policies for biologics differ between
MCOs
Current dynamics in RA
6.8.1 Impact of novel oral agents entering the RA
market
6.8.2 Patient advocacy
6.8.3 Infusion nurses
6.8.4 Patient assistance programs
6.8.5 Use of health technology assessment
6.8.6 Comparative effectiveness research
6.8.7 AHRQ guidance on RA
6.8.8 MCOs are requesting Phase 4 naturalistic studies
6.8.9 Risk evaluation and mitigation strategies
Annual costs of scope products in the US
Pricing and reimbursement in the UK
Key stakeholders
The pricing and reimbursement process
Free pricing in the UK within the context of the PPRS
7.3.1 Value-based pricing likely from 2014
7.3.2 Data requirements for market access
7.3.3 Drugs and Therapeutics Committees
NICE
7.4.1 Selection of technologies to be evaluated
7.4.2 Single versus multiple technology appraisals
7.4.3 Single technology appraisals
7.4.4 NICE can make one of four decisions
7.4.5 Patient access schemes
7.4.6 Scientific advice scheme
NICE clinical guidelines
NICE clinical guidelines in RA
7.6.1 NICE commissioning guides in RA
7.6.2 Quality and Outcomes Framework
BSR Biologics Register
NICE assessments of biologics in RA
7.8.1 Humira, Enbrel, and Remicade are recommended
for patients with RA
7.9
7.10
7.11
7.12
7.13
7.14
8
8.1
8.2
8.3
8.4
8.5
8.6
8.7
8.8
8.9
8.10
9
9.1
9.2
7.8.2 Biologics recommended after inadequate
response to a TNFI
7.8.3 Cimzia is approved for the treatment of RA
7.8.4 RoActemra is recommended for the treatment
of RA
7.8.5 Simponi is recommended for the treatment
of RA in patients who have inadequately
responded to DMARDs
7.8.6 Orencia is not recommended for the treatment
of RA following inadequate response to DMARDs
including MTX
7.8.7 Kineret is not recommended for the treatment
of RA
Future NICE appraisals in RA
The Scottish Medicines Consortium
7.10.1 Horizon scanning
7.10.2 The SMC has a disproportionate influence
7.10.3 Patient access schemes
SMC assessments in RA
7.11.1 MabThera
7.11.2 Orencia
7.11.3 RoActemra
7.11.4 Cimzia
7.11.5 Kineret
All Wales Medicines Strategy Group
Summary of HTA decisions in the UK
Annual costs of scope products in the UK
Pricing and reimbursement in France
Key stakeholders
P&R process, key timings, and data requirements
TC clinical evaluation
8.3.1 The evaluation of actual medical benefit – the
SMR
8.3.2 Public health interest
8.3.3 Incremental benefit – ASMR – is the key
determinant of subsequent pricing
8.3.4 ASMR versus SMR
8.3.5 Target population
8.3.6 Médicament d’exception restriction
Hospital versus community funding
8.4.1 T2A exemption
8.4.2 Retrocession pharmaceuticals
Pricing
8.5.1 Pricing for community products – CEPS
negotiations
8.5.2 Pricing for retrocession and T2A-exempt
products – price notification process
8.5.3 Pricing for hospital-only products
8.5.4 Pricing for non-reimbursable drugs
A range of P&R controls apply beyond product launch
Proposed regulatory reform
National treatment framework
8.8.1 Guidelines within the ALD scheme
8.8.2 Other clinical guidelines
8.8.3 Health networks
8.8.4 Patient registries
Overview of TC assessments of scope products
8.9.1 Remicade
8.9.2 Kineret
8.9.3 Enbrel
8.9.4 Humira
8.9.5 MabThera
8.9.6 RoActemra
8.9.7 Orencia
8.9.8 Cimzia
8.9.9 Simponi
8.9.10 Overview of TC comments on scope products
Annual costs of scope products in France
Pricing and reimbursement in Germany
Key stakeholders
The German healthcare system
9.2.1 Statutory health insurance
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9.3
9.4
9.5
9.6
9.7
9.8
9.9
9.10
10
10.1
10.2
10.3
10.4
10.5
10.6
10.7
11
11.1
11.2
The new market access landscape in Germany
Benefit assessment by the G-BA
9.4.1 The benefit dossier
9.4.2 The assessment process and timelines
9.4.3 G-BA scientific advice
9.4.4 Determination of the appropriate comparator
9.4.5 Cost data
9.4.6 Definitions of additional benefit
9.4.7 Indication/line extensions
9.4.8 Assessments of drugs already marketed
The changing role of IQWiG
9.5.1 Cost–benefit assessments
9.5.2 Benefit assessment
Pricing and reimbursement
9.6.1 Expenditure controls
9.6.2 Free pricing at launch
9.6.3 Price negotiation with the GKV-Spitzenverband
9.6.4 Indication/line extensions
9.6.5 Arbitration
9.6.6 Individual contracting with SHI funds
9.6.7 Reference pricing
9.6.8 Reimbursement and cost coverage in hospitals
9.6.9 Long-term impact of AMNOG legislation on
prices
RA in Germany
9.7.1 Treatment guidelines
9.7.2 G-BA therapy recommendations
9.7.3 AKdÄ guideline
9.7.4 DGRh guidelines
9.7.5 IQWiG assessment of biological DMARDs
RABBIT registry
Pricing landscape in RA
Annual costs of scope products in Germany
Pricing and reimbursement in Spain
Key stakeholders
10.1.1 The central Government
10.1.2 The Regions
10.1.3 The hospitals
10.2.1 The pricing and reimbursement processes are
combined
10.2.2 “Fast-track” process for innovative products
10.2.3 Reimbursement
10.2.4 Pricing negotiations
10.2.5 The use of cost-effectiveness
10.2.6 Cost-containment measures
Treatment guidelines for RA
10.3.1 SER GUIPCAR clinical practice guidelines
10.3.2 Health plans
National P&R status of key treatments
10.4.1 Monitoring use of biologics
Regional and local reviews of RA treatments
10.5.1 Cimzia
10.5.2 Enbrel
10.5.3 Humira
10.5.4 Kineret
10.5.5 Orencia
10.5.6 RoActemra
10.5.7 Simponi
Key issues highlighted by local and regional reviews of
RA products
Annual costs of scope products in Spain
Pricing and reimbursement in Italy
Key stakeholders
11.1.1 The central Government
11.1.2 The Regions
11.1.3 Local health units and hospitals
11.2.1 The P&R processes are combined
11.2.2 Therapeutic innovation can lead to a premium
price
11.2.3 Determination of therapeutic innovation
11.3 Regional HTA
11.3.1 Regional reviews
11.3.2 Budgetary responsibilities
11.3.3 Payback measures
11.3.4 Risk-sharing agreements
11.3.5 Pharmacovigilance
11.3.6 Discounts are applied at hospitals
11.3.7 Generics and reference pricing
11.4 Overview of RA in Italy
11.5 Management of RA
11.5.1 National initiatives
11.5.2 Regional initiatives
11.5.3 Clinical guidelines
11.5.4 Regional guidelines
11.5.5 Adherence to clinical guidelines
11.5.6 Use of the scope products in Italy
11.6 P&R status of the scope products
11.7 Economic evaluation of scope products
11.7.1 Economic studies
11.7.2 HTA/economic assessments by the Regions
11.7.3 Overview of HTAs
11.7.4 Regulation of biologics
11.8 Annual costs of scope products in Italy
12
Pricing and reimbursement in Japan
12.1 Key organizations and stakeholders
12.2 P&R process, key timings, and data requirements
12.2.1 Data requirements for reimbursement
12.3 Pricing process
12.4 Pricing methods for patented products
12.4.1 The comparator pricing method
12.4.2 The cost calculation method
12.4.3 Inter-strength adjustment
12.4.4 Foreign price adjustment
12.4.5 Analysis of methods used to calculate prices
12.5 Pricing of generics
12.6 Key current P&R issues
12.6.1 Biennial price revisions
12.6.2 Generic substitution
12.7 Future trends that will affect market access
12.7.1 Improved regulatory approval timelines
12.7.2 Price revision reforms
12.7.3 The use of pharmacoeconomic data in price
determination
12.8 Overview of the RA market in Japan
12.8.1 Epidemiology
12.8.2 Cost
12.9 Management of RA in Japan
12.9.1 Availability of products
12.9.2 Treatment guidelines
12.10 P&R status of biologics for RA
12.10.1 Remicade
12.10.2 Enbrel
12.10.3 Humira
12.10.4 Actemra
12.10.5 Orencia
12.10.6 Simponi
12.10.7 Cimzia
12.11 Products in development
12.11.1 Tofacitinib
12.11.2 Iguratimod
12.12 P&R issues identified from recent approvals
12.12.1 The time lag between Japan and the US/EU
12.12.2 Indication sequencing
12.13 Annual costs of scope products in Japan
13
International price comparison
13.1 Introduction and methodology
13.2 Dosage regimens
13.3 Unit prices
13.4 International treatment cost comparison
13.4.1 Annual cost of treatment
13.4.2 Annual cost of treatment rebased to US prices
References
7
Author profiles
PRMA Insights are developed by consultants with wide-ranging experience of all
aspects of market access, pricing, and reimbursement and are validated by national
and international opinion leaders.
Sotiria Papanicolaou
David Sykes (strategic advisor)
Sotiria has more than 10 years’ experience in the
pharmaceutical industry in corporate leadership, EMA
and global strategic market access teams, and a European
health outcomes research group, where she developed
health economics and outcomes strategies to support local
P&R negotiations for new products.
David is the founding partner of PRMA Consulting. He has
more than 15 years’ experience in P&R, market access,
and health outcomes and has held senior leadership roles.
David has developed European and global P&R and market
access programs to quantify, capture, and communicate
product value, including several products launched across
multiple therapy areas.
Jacqui Buchanan
Jacqui has 10 years’ experience in clinical and health economic
assessment. She has worked in strategic marketing and
health policy, health economics and pricing. She also has
wide-ranging experience in technology assessment and costeffectiveness consulting across the US, UK, and Australia.
Dr Helen Barham
Helen is managing editor at PRMA Consulting and has led
content development of all the PRMA Insights series. She
has more than 15 years’ experience in medical publishing,
with expertise in a wide range of therapeutic areas and
broad-ranging knowledge of market access and P&R.
Professor Deborah Saltman AM
(medical advisor)
Deborah has worked in the pharmaceutical industry for
more than 15 years, and has 20 years’ experience in
leadership positions in academic medicine, delivering
education programs for health workers in Europe, Asia,
and Australia. She also has extensive experience in health
research, postgraduate medical education, and medical
publishing. Deborah is currently Honorary Professor
in the Faculty of Medicine at the University of Sydney.
Deborah was made a member of the Order of Australia
in 2004, and was awarded the Rose Hunt Medal by the
RCGP (UK) in 2006.
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Monika Behrens
Dr Divyagiri Seshagiri
Monika has more than 15 years’ experience in the
pharmaceutical industry and statutory health insurance
and has been responsible for market access strategy for
GlaxoSmithKline in Germany, the UK, and Europe for a
broad range of disease areas. Monika has an in-depth
knowledge of the German healthcare system, particularly
the AMNOG legislation. She holds a diploma in national
economics and an MSc in health economics from York
University.
Divyagiri is a qualified physician and has over 6 years’
experience in the healthcare industry, including clinical
practice and clinical research and all stages of the healthcare
system, from prescribing through to drug research and
public health setup in India and various European countries.
Vanessa Mirsky
Vanessa has more than 10 years’ experience in
healthcare and life sciences commercialization strategy,
with a strong background in managed markets,
including reimbursement dynamics among public and
private payors in the US. Vanessa has worked with
multinational pharmaceutical companies and small
boutique firms in the US, and has significant experience
in oncology and biologics.
Cécile Matthews
Cécile has 13 years’ experience in pharmaceutical
consultancy, covering a broad range of strategic issues
and therapeutic areas. She has specialist knowledge of
European pricing and reimbursement issues that affect
the pharmaceutical industry, particularly in France.
Dr Alicia Gil
Alicia has more than 14 years’ experience in the
pharmaceutical and biotechnology industry, having held global,
regional, and local positions where she was involved in the
development and execution of regulatory affairs and market
access strategies through all phases of drug development and
commercialization, in a range of therapeutic areas. Alicia has
an in-depth understanding of the Spanish healthcare system
and market access challenges that it presents.
Dr Alberto Redaelli
Alberto has more than 25 years’ experience in the
pharmaceutical industry. He has been an international
manager in P&R and outcomes research, with broad
experience in business planning, market research, and
new product development at corporate level. Alberto
has published numerous articles and reviews relating to
the health economics of oncology in international peerreviewed journals, and has an in-depth understanding of
the Italian healthcare system and market access challenges.
Sample pages
9
1 Clinical overview
3 Patient-reported outcomes
PRMA Insights: Pricing and Reimbursement Success in Rheumatoid Arthritis
approval for a new therapy on the basis of a PRO claim. It is imperative that concepts match the
claims sought, and that the PRO instrument is developed and validated for the target population.
difficult to assess. The BATTER-UP consortium is undertaking a prospective study of a biomarker
panel that might predict response to TNFIs.
Extra-articular manifestations of RA occur in 40–50% of patients and can affect the CV system,
skin, eyes, blood, mucosa, and pulmonary system, and contribute to morbidity and mortality.
Incidence increases with disease duration.
•
PRMA Strategic Insights: Clinical overview
3.3.1
1i
Defining RA is currently a moving target. Trials to date have used the 1987 ACR criteria
for case recognition, which are based on features of established RA. However, the 2010
ACR/EULAR criteria provide a standardized approach to the identification of patients
with early RA and thus the framework for trials to investigate earlier intervention. This
will have an impact on trial design and the comparability of trial outcomes.
1ii
The majority of data on disease progression currently available are based on patient
cohorts before the widespread use of biological DMARDs. However, the pattern of
disease progression has changed, as TNFIs effectively suppress the pathophysiological
mechanisms associated with cartilage degradation and bone erosion, slowing
radiographic progression. As the treatment paradigm shifts towards earlier intervention,
the need to predict disease progression at the pre-articular phase becomes important,
particularly if intervention at this stage can avoid initiation of the disease process. RF
and ACPA can be used as diagnostic tools for early RA as well as prognostic factors.
•
•
PRMA Strategic Insight 3i
Why measure PROs in RA?
In the absence of clinical
superiority data, PRO benefits
may be key to differentiation of a
new product. A well-designed and
robust PRO strategy is therefore
essential, especially as most of the
existing products have PRO claims
in the labels. In addition to the
standard physical function (HAQ)
and generic PRO instruments,
instruments that evaluate pain,
fatigue, productivity, and quality of
sleep should be considered. The PRO
strategy should be developed and
externally validated with payors and
key opinion leaders from Phase 1
onwards, in order to identify the
instruments that should be included
in Phase 2/3 programs.
Given that there is no cure for RA, the aim of treatment
is to slow disease progression and lessen the impact
of the disease on patients’ lives. Measurement of
PROs in people with RA is important because it:
•
•
•
•
•
•
encourages patient-centered care
can define the severity of the condition and
inform decision-making
determines the effectiveness of treatments and
improvement of RA in routine practice
can be used to assess quality of care
is recommended by the EMA and FDA (see
Section 3.6)
is used as a decision-making tool for policy
makers who need to allocate limited resources.
PRO claims are important for helping to secure
market access.
Areas to watch
•
The multidimensionality of PROs is exemplified in the variety of outcomes that have been
included in FDA labeling claims: of PRO labels for drugs approved in 2007 and 2008, 75% of claims
were granted for signs and symptoms, 13% for activity limitations, and 13% for HRQoL (Doward
et al., 2010).
Interest in biomarkers as predictors of disease progression and treatment response is
increasing. Various potential biomarkers have been proposed but further work is needed
to establish any role in clinical practice (Section 1.8.1).
The BATTER-UP consortium is due to publish a validation of eight biomarkers as
predictors of the response to TNFIs in April 2012 (Section 1.8.1.3).
Algorithms to predict response to treatment on the basis of clinical indices have been
proposed and a mathematical model for predicting response to TNFI therapy after
2 weeks of treatment has been developed. The likely value of such algorithms remains
to be seen (Section 1.8.1.4).
3.4
PRO instruments in RA
Many generic and disease-specific instruments are used to assess PROs in RA. These are discussed
in the following sections. Details of individual instruments are given in Appendix A (page 89).
3.4.1
Generic instruments
Generic instruments enable comparisons across groups and interventions. All pivotal clinical trials
of new therapeutics in RA now include at least one generic instrument, usually the HAQ-DI (see
Section 1.6.6) to assess function and/or the SF-36 to assess HRQoL (Tugwell et al., 2007). These
instruments have proven validity and sensitivity for assessment of changes in health status or
HRQoL in clinical trials of DMARDs and biologics. These and other generic PRO instruments that are
widely used in trials of RA therapeutics are summarized in Table 3.1.
3.4.2
Rheumatology-specific PRO instruments
Rheumatology-specific instruments are designed to address problems specific to the RA population.
The Arthritis Impact Measurement Scale (AIMS) was one of the first PRO measures developed to
evaluate HRQoL in RA (Meenan et al., 1980). AIMS2 is a more comprehensive and sensitive version
of this questionnaire (Meenan et al., 1992). Use of the AIMS2 instrument is recommended by both
the FDA and EMA (see Section 3.7), although it has not yet been widely used in clinical trials of
investigational drugs for RA.
The RA Impact of Disease (RAID) is new patient-derived composite response score recently developed
by EULAR for use in clinical trials as a measure of the impact of RA on HRQoL (Gossec et al., 2011).
EULAR’s aim in developing RAID was to bring additional information into the assessment of RA that
accounts for discordance between the patient’s and the physician’s perspective. Current standard
assessments of RA include some dimensions or domains assessed by PROs (patient assessment of
This report is a licensed product and is not to be reproduced
or photocopied without the appropriate permissions.
38
68
4 Treatment of rheumatoid arthritis
5 Economic impact of rheumatoid arthritis
Table 4.38: Summary of the RAPID-1 trial of Cimzia
References
PRMA Strategic Insights
Keystone et al. (2008) Certolizumab pegol plus MTX is significantly more effective than placebo
plus MTX in active rheumatoid arthritis: findings of a fifty-two-week, Phase 3, multicenter,
randomized, double-blind, placebo-controlled, parallel-group study. Arthritis Rheum 58: 3319–29
Strand et al. (2009) Rapid and sustained improvements in health-related quality of life, fatigue, and
other patient-reported outcomes in rheumatoid arthritis patients treated with certolizumab pegol
plus MTX over 1 year: results from the RAPID 1 randomized controlled trial. Arthritis Res Ther 11: R170
Study
design
52 week Phase 3 multicenter randomized double-blind placebo-controlled parallel-group trial
Sample size
982
Treatment
and dosing
Cimzia 400/200: Cimzia 400 mg at weeks 0, 2, and 4, then Cimzia 200 mg every 2 weeks + MTX
weekly
5i
Drug costs and hospitalizations account for the greatest proportion of direct costs;
however, many published studies have not considered high-cost biologics. To build
the value proposition, new observational and costing studies are needed to assess the
clinical and economic impact of these agents. Relevant data need to be collected to allow
stratification of results according to disease severity, functional status, and comorbidities.
5ii
Existing registries may present an opportunity to characterize treatment patterns and
resource utilization profiles to inform costing and cost-effectiveness studies.
Figure 5.2:
Cimzia 400/400: Cimzia 400 mg at weeks 0, 2, and 4, then Cimzia 400 mg every 2 weeks + MTX
weekly
25,000
Placebo + MTX
Patient
population
Active rheumatoid arthritis despite MTX
Endpoints
Primary
22,458
16,441
15,000
• ACR 20 response rate at week 24
• Mean change from baseline in mTSS at week 52
15,417
16,502
13,463
Indirect
Informal care
Non-medical
Medical
(excluding drugs)
Drugs
10,000
Major secondary
• Mean change from baseline in mTSS at week 24
• Mean change in HAQ-DI score at week 52
• ACR 20, 50, and 70 response rates at week 24
Results
21,908
21,069
20,000
5,000
0
ACR 20: Cimzia 400/200, 58.8%; Cimzia 400/400, 60.8%; placebo, 13.6%
Mean change in mTSS at week 52: Cimzia 400/200, 0.4; Cimzia 400/400, 0.2; placebo, 2.8
Mean improvement in HAQ-DI: Cimzia 400/200, 0.6; Cimzia 400/400, 0.63; MTX, 0.18
US
Europe
France Germany
Italy
Spain
UK
Source: Lundkvist et al., 2008.
P < 0.001 Cimzia groups vs placebo for all results
HRQoL
Several studies have assessed the total per-patient costs associated with RA, summarized in
Table 5.1. Some studies have included both direct and indirect costs. Few studies have evaluated
the cost of RA in recent years and some studies date back to the early 1990s. (Only full key papers
published in English are considered in this discussion.)
SF-36 PCS: Cimzia 400/200, 7.8; Cimzia 400/400, 8.6%; placebo, 1.7
SF-36 MCS: Cimzia 400/200, 6.4; Cimzia 400/400, 6.4; placebo, 2.1
Fatigue Assessment Scale: Cimzia 400/200, –2.6; Cimzia 400/400, –2.5; placebo, –0.8
All values are mean change from baseline at week 52; P < 0.001 Cimzia groups vs placebo for all
results
Safety and
tolerability
Treatment-emergent AEs: Cimzia 400/200, 96.6; Cimzia 400/400, 94.5; placebo, 125.9
The most frequent non-infectious AEs were headache, hypertension, and back pain
Serious AEs: Cimzia 400/200, 14.8; Cimzia 400/400, 15.2; placebo, 12.0
AEs leading to study withdrawal: Cimzia 400/200, 5.6; Cimzia 400/400, 7.0; placebo, 3.3
Malignancy: Cimzia 400/200, 2.3; Cimzia 400/400, 1.3; placebo, 1.1
All rates are incidence per 100 patient-years
Conclusions
Compared with placebo plus MTX, Cimzia 200 or 400 mg plus MTX reduced signs and symptoms of
rheumatoid arthritis in a rapid and sustained manner, inhibited structural damage, and improved
physical function
Abbreviations: ACR 20/50/70, 20/50/70% improvement in number of tender or swollen joints and three of five other American
College of Rheumatology criteria; AE, adverse event; HAQ-DI, Health Assessment Questionnaire disability index; HRQoL, healthrelated quality of life; MCS, mental component summary; mTSS, modified total Sharp score; MTX, methotrexate; PCS, physical
component summary; SF-36, Short-form (36) Health Survey
5.1.2
France
Kobelt and colleagues reported costs associated with RA from a sample of 1,487 patients who
completed an anonymous mail survey, summarized in Table 5.2 (page 174). From a societal
perspective, productivity losses were the major cost (24%), followed by hospitalization (21%),
medication (20%), and informal care (16%). From the perspective of public payors, hospitalization
was the largest cost (34%), followed by drugs (26%) and indemnities (20%) (Kobelt et al., 2008).
The mean cost per patient in this latter perspective was estimated at €11,658 and represented
only 54% of total societal costs, which amounted to €21,690. Direct medical and non-medical costs
represented a high proportion of total costs (77% in the societal perspective and 80% in the public
payor perspective), reflecting the high age of the sample, and consequently low indirect costs. Use
of biological treatments was higher than the expected use – 27% of patients in the sample (Enbrel,
48.1%; Remicade, 26.5%; Humira, 25.7%; Kineret or MabThera, 3%).
© PRMA Consulting 2012
152
172
prma insights
®
Sample pages
5 Economic impact of rheumatoid arthritis
7 Pricing and reimbursement in the UK
measured by the HAQ-DI (P ≤ 0.001) and both approaches (HUI-3 and the EQ-5D utilities) showed
decreasing utility values as RA severity increased (Table 5.8).
Table 7.9: Cost-effectiveness results for Cimzia, based on the manufacturer’s model
Treatment Comparator
Table 5.8: HUI-3 and EQ-5D utility values according to disease severity assessed by the HAQ-DI
HUI-3
EQ-5D
HAQ-DI ≤0.5
1.1 ≤ HAQ-DI
< 1.6
1.6 ≤ HAQ-DI
< 2.1
HAQ-DI ≥2.1
0.91 ± 0.08
0.80 ± 0.15
0.75 ± 0.14
0.58 ± 0.20
0.45 ± 0.20
(n = 62)
(n = 56)
(n = 31)
(n = 34)
(n = 23)
0.87 ± 0.14
0.74 ± 0.19
0.62 ± 0.21
0.42 ± 0.30
0.17 ± 0.42
(n = 68)
(n = 66)
(n = 39)
(n = 35)
(n = 27)
In acknowledgement of the heterogeneous nature of RA, work is now starting to address different
HAQ-DI trajectories for different levels of sustained disease activity. HAQ-DI is mainly influenced
by disease activity in the early stages of disease but by joint damage in established disease. Thus
DMARDs and biologics can have a greater influence on the HAQ-DI during early disease stages but
less effect in established disease. According to recent feedback from advisors in our network, this
is particularly important when the HAQ is mapped to utility scores and costs per QALY in health
economic evaluations.
Enbrel +
MTX
−0.005/0.065
£2,675/2,993
Cimzia less effective and
more costly/£46,192
Cimzia +
MTX
Humira +
MTX
0.102/0.242
£3,563/3,124
£34,930/12,937
Cimzia +
MTX
Remicade +
MTX
0.211/0.458
−£4,468/−6,441
Cimzia
Humira
0.127/0.215
£5,347/1,223
£42,197/5,687
Cimzia
Enbrel
−0.047/−0.130
−£297/−517
£6,341/Cimzia less
effective and less costly
Cimzia +
MTX
Enbrel +
MTX
−0.005/0.065
−£900/−582
Cimzia less effective and
less costly/dominated
Cimzia +
MTX
Humira +
MTX
0.102/0.242
−£12/−451
Dominated/dominated
Cimzia +
MTX
Remicade +
MTX
0.211/0.458
−£8,043/−10,016
Dominated/dominated
Cimzia
Humira
0.127/0.215
£1,772/−2,352
£13,982/dominated
Enbrel
Cimzia
−0.047/−0.130
−£3,872/−4,092
£82,695/31,582
The revised economic model including the PAS for combination therapy showed that Cimzia dominated Enbrel, Humira, and Remicade. Cimzia monotherapy also dominated Humira monotherapy.
The ERG outlined the following concerns regarding the methodology used for the indirect
comparison.
Different instruments and
methodologies have been used to
derive utilities in RA. For example,
a 1 point reduction in the HAQ-DI
or DAS28 has been associated with
higher QALY gains if the EQ-5D rather
than the SF-6D is used. Thorough
research on this area is needed
before defining the PRO strategy and
health outcomes research.
•
•
•
•
•
The ERG questioned the appropriateness of using an indirect treatment comparison in which
only multiple two-way comparisons between biological DMARDs was possible, rather than
using a mixed-treatment comparison.
The method used for selecting the studies to be included in the indirect treatment comparison
was unclear. It was possible that relevant information from several excluded studies, including
one of Cimzia plus MTX versus placebo plus MTX (C87014 trial), could have been used.
The inclusion of data from the included studies was not consistent.
There was insufficient consideration and exploration of underlying heterogeneity among the
studies included in the indirect comparison.
Of the 10 trials used in the indirect comparison for combination therapy, participants in the two
Cimzia trials at 6 months had a low previous exposure to DMARDs compared with participants
in trials of comparator treatments. Also, the mean MTX dosage at the time of entry to the
10 trials was lowest in the Cimzia trials.
276
10 Pricing and reimbursement in Spain
12 Pricing and reimbursement in Japan
•
•
•
10.7
Reviewers and decision-makers have criticized the lack of real-world evidence and costeffectiveness studies. In the absence of such studies, economic evaluations are performed on
the basis of daily treatment, cost of complete treatment, and incremental cost versus
alternatives. Evaluations may also consider an estimation of the number of patients likely to
receive a treatment per year, annual costs, and annual efficacy units.
The reviewers criticized the general lack of
PRMA Strategic Insight 10xiv
data relevant to clinical practice in Spain (e.g.,
comparison with the treatments used locally,
Regional reviewers have criticized
rather than the international perspective) – this
the lack of data relevant to clinical
has important consequences since, in most
practice in Spain, which has resulted
in restrictions over their approved
cases, products are approved/introduced with
indication and reimbursement
restrictions over their approved indication and
conditions. Manufacturers need to
reimbursement conditions.
understand local clinical practice
Finally, while increased efforts are being made
in Spain, and ensure that relevant
to standardize approaches and methodology,
comparators (those actually used
discrepancies in conclusions reached after
in clinical practice) are included in
evaluating the same data set still remain, which
studies.
can be confusing for manufacturers.
Annual costs of scope products in Spain
Figure 10.3 shows the annual cost of treatments of RA with the scope products in Spain. The
methodology used to calculate these prices is described in Section 13.1.
Figure 12.1: Overview of the pricing and reimbursement process in Japan
New drug approval
19,881
17,216
20,345
18,675
15,000
13,500
14,492
HIB MED
In principle within
60–90 days
Applicant
DPO (1st)
1. Presence of similar drugs
2. Suitability of similar drugs
3. Necessity of applying premium
4. Validity of cost
Reimbursement discussed with
Japan Medical Association and
Japan Pharmaceutical Association
Applicant submits
appeal statement
DPO (2nd)
Draft pricing report
Added to NHI price list
Chuikyo: final decision on price/approval
Abbreviations: DPO, Drug Pricing Organization; EAD, Economic Affairs Division; HIB, Health Insurance Bureau; HPB,
Health Policy Bureau; MED, Medical Economics Division; NHI, national health insurance
Source: adapted from JPMA, 2012
applications to the NHI drug list has decreased significantly over time. For example, in the late 1990s
almost 50% of applications included some form of economic evaluation, compared with fewer than
10% in 2006–2008. An industry survey published in 2008 reported that manufacturers chose not
to submit health economic data because such studies are not considered by the authorities as
they are not part of the formal reimbursement process, and there are insufficient data for robust
analyses (Koike et al., 2007; Toshiya and Sannomiya, 2008).
18,712
15,861
12.3
Pricing process
The pricing process in Japan is based on a series of rules and there is limited scope for manufacturers
to influence or negotiate price. This section outlines the steps for achieving a price on the NHI drug
list. This process is illustrated in Figure 12.2.
10,000
5,000
0
Discussion
HIB’s draft price
25,000
20,000
HPB EAD
Applicant’s draft price
Figure 10.3: Annual costs of scope products for rheumatoid arthritis in Spain
21,124
Dominated/dominated
Cimzia monotherapy compared with Humira monotherapy gave a QALY gain of 0.215 and
incremental costs of £1,223, resulting in an ICER for Cimzia of £5,687 per QALY gained. Cimzia
monotherapy was less effective and less costly than Enbrel monotherapy.
PRMA Strategic Insight 5v
186
Cimzia +
MTX
The mapping technique proposed by Hurst and
colleagues (from a Scottish cohort of RA patients)
has been used in economic evaluations of TNFIs and
was used in the Birmingham RA Model (BRAM) which evaluated the cost-effectiveness of Enbrel,
Humira, and Remicade for the treatment of RA (Chen et al., 2006b). Bansback and colleagues
estimated EQ-5D and SF-6D scores from HAQ-DI items using linear regression models. The HAQ-DI
Cost per QALY
BC/revised
In all cases, dominated means Cimzia was more effective and less costly
Abbreviations: BC, base case; MTX, methotrexate; PAS, patient access scheme; QALY, quality-adjusted life-year
Source: NICE, 2010c
A recently published league table of utility scores in various diseases showed RA to be associated with
the worst HRQoL (Figure 5.8) (Currie et al., 2005; Lundkvist et al., 2008; Orme et al., 2007).
Clinical trials in RA have not typically collected
generic preference-based utility measures, so
methods of estimating utility values from diseasespecific instruments have been developed. Formulas
to map the HAQ-DI to the EQ-5D and SF-6D have
been developed in order to estimate the average
utility of a cohort (Bansback et al., 2007; Hurst et
al., 1997). The use of mapping techniques, while not
based on the primary collection of data, is a practical
solution when no utility measure has been collected.
Incremental cost
BC/revised
Including PAS
Adams and colleagues used regression analysis to calculate utility scores from the HAQ-DI and the
DAS28 in an RA sample (n = 345) at baseline and after 12 months of biological therapy (Adams
et al., 2010). A 1 point reduction in the HAQ-DI was associated with a gain in QALYs per annum of
0.24 using the EQ-5D but only 0.08 using the SF-6D. In addition, a 1 point reduction in the DAS28
score was associated with a gain in QALYs per annum of 0.084 using the EQ-5D and 0.029 using the
SF-6D. These differences highlight that comparison of cost-effectiveness is problematic when utility
scores have been derived using different methods.
Area to
watch
Incremental utility
BC/revised
Not including PAS
0.5 ≤ HAQ-DI
< 1.1
Values are mean ± SD (valid n)
Abbreviations: EQ-5D, EuroQol five-dimension questionnaire; HAQ-DI, Health Assessment Questionnaire disability
index; HUI-3, Health Utilities Index; SD, standard deviation
Source: Carreno et al., 2011
Cost of treatment per patient per year (US$)
10
Cimzia
Enbrel
Humira
Kineret MabThera Orencia Remicade RoActemra Simponi
Costs are for the first year of treatment, based on the regimens defined in Table 13.1, calculated ex-factory prices,
and the following exchange rates (27 July 2011; www.oanda.com): US$1 = £0.61, ¥78, €0.69
Source: PRMA Consulting, 2011
Once a manufacturer achieves regulatory approval, it submits a pricing proposal to the EAD in
the Health Policy Bureau. The price proposal needs to justify the pricing method, the choice of
comparator, and any price premium proposed (these methods are discussed in further detail in
Section 12.4). The EAD submits a recommendation to the Medical Economics Division (MED) in
the Health Insurance Bureau; the MED then sends its own proposal to the manufacturer. If the
manufacturer is satisfied with the proposal, the MED will consult with the DPO on the basis of this
proposal. If the manufacturer is not satisfied with the proposal, it is allowed to submit its original
proposal to the DPO. In this case, both the manufacturer and the MED present their separate
proposals at a meeting with the DPO and both are allowed to respond to questions from the DPO.
The manufacturer can be accompanied by an expert at this meeting.
The DPO’s price proposal that goes to Chuikyo is based on its assessment of the following:
•
388
the existence of similar drugs (the validity of either the comparator pricing method or the cost
calculation method)
438
11
Ways to use PRMA Insights
PRMA Insights provide a key resource for development of your P&R and market access strategy.
This diagram illustrates just a few of the many ways that this can support your planning.
Development of an HEOR
strategy and evidencegeneration plan
All chapters
Development of a PRO
strategy
Chapter 3
Development of a
preference-based utility
strategy
Chapter 5
Chapter 7 (UK)
Critical understanding of
existing HE models
Chapter 5
Country-specific chapters
(6–12; particularly 7 [UK])
Competitor AE profiles
Chapter 4
and country-specific
chapters
Inform clinical development
program (endpoints,
subgroups)
Inform HTA strategy
Inform cost-effectiveness
model
All chapters
Develop and inform value
propositions
Chapters 3–5
Country-specific chapters
Internal education
All chapters
Cross-functional
collaboration
All chapters
Understand:
• Competitor data packages
• Adverse-effect profiles
• Indirect comparisons
• Existing utility estimates
• Registries in the scope countries (data captured and how these
can be used)
• Supporting development of economic models
• Evidence gaps and future evidence generation
Understand:
• Role of PROs in product differentiation
• Impact of PROs on HTA and P&R submissions
• Existing PRO data for marketed products and label claims
• Evolution of a PRO strategy
Understand:
• Existing utility estimates in the literature and how these have been
applied to cost-effectiveness models of competitor products
• Mapping to EQ-5D to generate utilities
• How such estimates map onto different symptoms that may be
able to drive utility differences
Understand:
Critically assess existing models, assumptions, and inputs
(cost, utility, and clinical)
Understand:
• Impact of AEs in economic models
• HTA feedback on relevant AEs to include in models
Understand:
• Competitors’ HTA and clinical development strategies (existing
products and products in development)
• Benefits according to different subgroups and definitions used in trials
• Feedback from payors and HTA agencies on submitted evidence
and needs for future research
• Understand the value propositions of competitors and their
acceptance by payors
• Provide the basis for the global value dossier of a new product
Use as educational materials to enable colleagues to rapidly become
familiar with a new therapy area and the market challenges
Ensure common understanding across the organization in order to develop
a single coherent strategy that meets various stakeholders’ needs in:
• Market access challenges and opportunities
• HEOR strategy development
• Clinical development strategy
• Regulatory expectations in terms of indication, clinical trial design,
and PROs
• Accurate forecasting of the market opportunity
• A consistent value proposition that supports both market access
and marketing
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PRMA Insights: Pricing and Reimbursement

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