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Presentation - The Economics of the Market for Medicines, Jorge Mestre-Ferrandiz 17 March 2016
1. Economics of the market for
medicines
Dr Jorge Mestre-Ferrandiz
City University
17th March 2016
2. 2
Agenda
1. The supply side – R&D
2. Demand for medicines
3. NICE – the cost-effectiveness ‘4th hurdle’
4. Regulating medicine prices
3. 3
Structure
Time
£
Supply Issues
• R&D process
• Cost of an NME
• Public/private
collaborations
• R&D incentives
• Capital market
Nature of competition
• Follow-on compounds
(dynamic)
Competition in the
off-patent segment
Demand / Regulation
• Role of HTA
• Uptake drivers
• Prescribing Incentives
• Demand vs. Supply
controls
t0
t1
t2 t3
Launch
Patent
expiry
4. 4
Five global challenges
•Challenge 1: Increasing importance of specialised and
stratified medicines
•Challenge 2: Rising drug development costs
•Challenge 3: Closer benefit-risk monitoring by regulators
over a medicines’ life cycle
•Challenge 4: Increase in demand for real world evidence of
relative effectiveness by HTA, payers and regulators
•Challenge 5: Disconnect between regulators and payers/HTA
bodies evidence needs
Key variables: Price * Volume * Time (and VALUE!)
5. 5
Characteristics of Medicines Markets
•Supply is R&D intensive, which implies:
• Intellectual property rights (patents)
• Long lead times
• High risk
• Dynamic competition is as important as static
• Generic competition after patent expiry
•Demand is regulated – governments and social insurers
are major buyers of medicines
•Prices are regulated
6. 6
Supply Side – Main Characteristics (1)
•Patents are an incentive for dynamic efficiency –
by promising temporary monopoly if successful
•Patents last 20 years; first 9-11 of which are
spent getting the medicine to market, i.e.
research & development (R&D)
•Commercial success in R&D-based companies
has depended on finding ‘blockbusters’
7. 7
Supply Side – Main Characteristics (2)
•Average R&D cost of a new medicine up to
launch c£1.2bn
•Includes costs of failures
•Out of pocket costs ≈ 50%
•Opportunity cost of capital ≈ 50%
•Only ≈ 30% of launched medicines earn
revenues that exceed their lifetime costs
8. 8
REGULATION
TIME (YEARS)
PHASES OF
DRUG
DEVELOPMENT
Phase III
Development research
Final patent
application
Marketing
application
Post-mktg
research*
Phase
IV
2002-7
Discovery research
Investigational new
drug application (US)
1999
Phase I Phase IISynthesis
Biological testing &
pharmacologic
screening
2008
Marketing
approval/
product launch
2010
Regulatory
review
Basic
research
Short-term animal testing
Long-term animal testing
Toxicology and pharmacokinetic studies
Chemical development
Pharmaceutical development
Discovery & Development of a New Medicine
*Phase IV is not part of the R&D process, although data collected during that time may affect how the drug is used.
Source: Mestre-Ferrandiz, J., Sussex, J. and Towse, A. (2012) The R&D Cost of a New Medicine. London: Office of Health Economics
10. 10
Understanding the R&D process: basic
concepts
Most new medicines are developed simultaneously
The innovation race stimulates competition
Being the first in class does not imply being the best
in class
The market (clinical practice) determines the
‘winners’
There exists spillovers in the R&D process
Alliances have an important role to play
11. 11
Cash Flow for a Successful Medicine
Launch
Patent
expiry
£ p.a. +
_
0
12. 12
Supply Side – Main Characteristics (3)
•R&D costs are sunk (global) joint costs
•R&D costs ≈ 17% of pharmaceutical sales p.a.
But ≈ 31% of costs on net present value basis
•=> (even long-run) marginal cost << average cost
•=> Price discrimination (based on Ramsey rule?) if non-
linear pricing is impractical
• Parallel trade
13. 13
% of ‘World’ Pharmaceutical Industry R&D
Spend
Source: ABPI R&D Sourcebook (2015). http://www.abpi.org.uk/our-
work/library/industry/Pages/131115.aspx
14. 14
Agenda
1.The supply side – R&D
2.Demand for medicines
3.NICE – the cost-effectiveness
‘4th hurdle’
4.Regulating medicine prices
15. 15
Types of Prescription Medicines
•In 2011 generics accounted for c64% of the total number of
prescriptions dispensed by pharmacies in England, compared
with fewer than one in six as recently as 1982
•Proportion of prescriptions written generically (c90% in 2011
vs. 35% in 1985)
•Source: OHE Compendium (2013)
[OTCs = over the counter medicines]
Original brand Branded Unbranded OTCs
On-patent Off-patent generics generics
NHS
Private
16. 16
Expenditure on medicines per head of population is low
in the UK relative to OECD comparators
Expenditure on medicines per person £ for selected OECD countries
17. 17
UK spending on medicines as a percentage of GDP is already
amongst the lowest in developed countries
(1) IMS Health World Review Analyst 2012. OECD Health Database. All data accessed March 2012
0.9
1.2
1.2
1.2
1.3
1.3
1.4
1.5
1.5
1.8
2.1
1.0
1.0
1.4
1.4
0.9
1.4
1.4
1.4
1.7
2.0
2.1
UK
Sweden
Denmark
Austria
Ireland
Germany
Italy
Spain
Belgium
France
US
2008
2011
Spending on medicines as a percentage of GDP in various countries in
2011(1)
18. 18
The UK has among the highest penetration of and use
generics across European countries, c. 65%
Generics market share in Europe by volume
%
51%
34% 35% 35% 36% 37%
47% 48% 51% 53% 55% 55% 57% 58% 60% 61% 64% 65% 66%
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
GR
34%
BE
35%
AT
36%
CH
39%
IT
40%
IE
42%
NO
43%
ES
45%
PT
45%
FR
47%
FI
49%
TK
52%
SE
53%
NE
63%
DK
64%
CA
65%
DE
65%
UK
66%
MEAN
49%
% GENERIC PRODUCTS
% NON GENERIC PRODUCTS
Source: IMS 2012 (2011 data)
21. 21
Demand Side Characteristics
Chooses Pays Consumes
Normal
market
Consumer Consumer Consumer
Prescription
medicines
market
Prescriber Government
/ insurer
Patient
22. 22
Measures Affecting Prescriber Price
Sensitivity
•Primary Care Trust budgets
•Practice budgets and prescribing incentive
schemes
•Provision of information (PRODIGY, PACT,
NICE guidance, pharmaceutical advisers,
etc.)
23. 23
Agenda
1. The supply side – R&D
2. Demand for medicines
3. NICE – the cost-effectiveness ‘4th hurdle’
4. Regulating medicine prices
24. 24
National Institute for Health and
Care Excellence
•NICE established in 1999
• Faster access to medicines
• Address the ‘postcode lottery’
• Provide evidence-based guidance to the health system on
new technologies
•Independent special health authority
•Expanded remit to cover public health in 2005
•Covers England and Wales
25. 25
NICE
•NICE’s guidance falls into three main areas
Clinical
guidelines
Health technology
evaluation
Public health guidance
• Recommendations on the
use of new and existing
technologies within NHS
• Medicines
• Medical devices
• Diagnostic techniques
• Surgical procedures
• Therapeutic technologies
other than medicinal
products
• Systems of Care
• Interventional procedures
• Screening tools
• Recommendations on
the appropriate NHS
treatment and care of
people with specific
diseases and conditions
• Recommendations for
NHS and local
government
professionals on
promoting a healthy
lifestyle and reducing
the risk of developing a
disease
26. 26
Technology Appraisal Criteria - April 2013
•The Institute and Appraisal Committee take into
account:
• the broad clinical priorities of the Secretary of
State for Health and the Welsh Assembly
Government
• the degree of clinical need of the patients with
the condition under consideration
• the broad balance of benefits and costs
• any guidance from the Secretary of State for
Health and the Welsh Assembly Government on
the resources likely to be available and on such
other matters as they think fit
• the effective use of available resources
27. 27
NICE’s Guide to Methods of Technology
Appraisal, April 2013
•Below a most plausible ICER of £20,000 per QALY gained, the decision to
recommend the use of a technology is normally based on the cost-effectiveness
estimate and the acceptability of a technology as an effective use of NHS
resources
•Above a most plausible ICER of £20,000 per QALY gained, judgements about the
acceptability of the technology as an effective use of NHS resources will
specifically take account of the following factors
• The degree of certainty around the ICER
• Whether there are strong reasons to indicate that the assessment of the
change in health-related quality of life has been inadequately captured,
and may therefore misrepresent the health utility gained
• The innovative nature of the technology, specifically if the innovation adds
demonstrable and distinctive benefits of a substantial nature which may
not have been adequately captured in the reference case QALY measure
• The technology meets the criteria for special consideration as a 'life-
extending treatment at the end of life'
• Aspects that relate to non-health objectives of the NHS
28. 28
NICE’s Guide to Methods of
Technology Appraisal, April 2013
•As the ICER of an intervention increases in the
range of £20,000 to £30,000 per QALY gained, the
Committee's judgement about the acceptability of
the technology as an effective use of NHS resources
will make explicit reference to the relevant factors
listed before
•Above a most plausible ICER of £30,000 per QALY
gained, the Committee will need to identify an
increasingly stronger case for supporting the
technology as an effective use of NHS resources,
with regard to the factors listed
31. 31
Not all QALYs are equal to NICE
even now: “special weighting”*
•“Life extending treatment at the end of life” = less
than 2 years’ life expectancy, extends life by ≥ 3
months for patient population ≤ 7,000 in England
•Severity of underlying condition
•Stakeholder persuasion
•Significant innovation not captured by QALYs
•Disadvantaged populations
•Children
* Rawlins M et al (2009) “Pharmacoeconomics: NICE’s approach to decision-making” British Journal of
Clinical Pharmacology
32. 32
There was a big (?) change in prospect…
Implementation of Value Based Pricing (VBP)
replacing the PPRS in 2014 (when 2009 PPRS
expired) – but now called Value Based Assessment
July 2010 December2010 January 2014
33. 33
NICE’s Current Approach
£20,000 per QALY
£30,000 per QALY
£50,000 per QALY
Source: NICE Consultation Paper – Value Based Assessment of Health Technologies
35. 35
NICE’s Response
•“Following a consultation, the Institute has
decided to undertake further work before
making changes to the way it appraises new
medicines and other technologies for use by
the NHS. It argues that any changes to NICE’s
methods need to be made as part of a wider review
of the innovation, evaluation and adoption of new
treatments (including those for cancers) involving
patients, people working in or with the NHS, the
life sciences industries and health researchers” (18
September 2014)
•https://www.nice.org.uk/news/press-and-
media/nice-calls-for-a-new-approach-to-managing-
the-entry-of-drugs-into-the-nhs
36. 36
Economic Evaluation Elsewhere
•Focused on pharmaceuticals
•Fourth hurdle i.e. reimbursement decisions:
• Public reimbursement: Australia, Baltic countries,
Belgium, Canada (British Columbia, Ontario), Czech
Republic, Denmark, Finland, France, Hungary,
Netherlands, New Zealand, Norway, Portugal,
Russia, Slovenia, Sweden
• US managed care formularies
•Pricing negotiations
• Australia, France, Italy, New Zealand
•Advice to health service
• England and Wales (NICE), Scotland (SMC)
•Risk sharing arrangements
• Australia, New Zealand, UK (few cases)
37. 37
HTAs – Some Issues
What products to
evaluate?
When to evaluate? How to evaluate?
For what purpose?
All vs. limited
• Clinical effectiveness &/or
cost effectiveness
• Additional modelling
• Independence of agency
• Information sources: RCT
vs. other
• Mandatory vs. advisory…
P&R vs. prescribing guidelines/use
Pre-launch (i.e. pre-requisite
to launch) vs. post-launch
38. 38
Agenda
1. The supply side – R&D
2. Demand for medicines
3. NICE – the cost-effectiveness ‘4th hurdle’
4. Regulating medicine prices
39. 39
Why Regulate? - Market Failure
•Public goods and the free-rider problem (e.g.
research)
•Externalities
• E.g. your vaccination reduces my risk of catching
an infection
• E.g. the caring externality: I’m happy if you’re
cared for
•Incomplete or asymmetric information
• Moral hazard (= ‘hidden action’)
• Selection problem (= ‘hidden information’)
• Principal/agent problems
•Government procurement
40. 40
Monopoly Power
•Economies of scale and/or scope
•Natural (local) monopoly
•Input constraints
•Patents: dynamic efficiency vs static monopoly
41. 41
Options: Types of Regulation
•‘No regulation’ = 1998 Competition Act only
•Profit, i.e. rate of return, control:
• Unbanded
• Banded
•Price control:
• Baskets of products, as with ‘RPI-X’ control of
utilities’ prices
• Individual products, e.g. via reference prices, or
‘cost-plus’, or related to therapeutic benefit
42. 42
1998 Competition Act
•Came into force March 2000
•Based on EU Treaty - Articles 81 & 82
•Prohibitions:
• Chapter 1 – Agreements preventing, restricting
or distorting competition
• Chapter 2 – Abuse of a dominant market
position
•Fines up to 10% of turnover; 3rd parties may
sue for damages
44. 44
RPI-X Regulation of a Basket of ‘n’ Products
w1p1
1 + w2p1
2 + w3p1
3 + …….. + wnp1
n
--------------------------------------------------- -1 x 100 ≤ ΔRPI - X
w1p0
1 + w2p0
2 + w3p0
3 + …….. + wnp0
n
Where:
wi = weight for product ‘i’ (e.g. quantity sold in period 0)
pt
i = price of product ‘i’ in period t = 0,1
ΔRPI = % change in retail price index between period 0 and period 1
X = efficiency factor
{ {
45. 45
Regulation Criteria
•Static efficiency:
• Productive efficiency: making the right choice
between different ways of achieving the same
outcome
• Allocative efficiency: doing the things that people
want and ensuring that the right people get them
•Dynamic efficiency
•Benefit to UK plc – economic rent
•Regulatory (administrative) burden
•Equity/other social policy objectives
46. 46
(How) Should Pharmaceuticals be Regulated in
the UK?
• What, if anything, to regulate?
• On- and/or off-patent?
• Branded and/or unbranded?
• Prescribed and/or over-the-counter?
• Sales to NHS only, or all UK sales?
• If so, how?
• Rate of return control, unbanded
• Rate of return control, banded
• Price control – basket, RPI-X
• Price control – individual products, reference prices
• From 3 perspectives:
• General public: patients and taxpayers
• Government
• Industry
47. 47
Key Questions
1. How price-sensitive are the people making the consumption
choices?
2. How much competition is there between one medicine and
another, or between medicines and alternative treatments?
3. Do producers have incentives to keep costs down?
4. Will production and consumption choices become increasingly
distorted over time?
5. Do producers have incentives to invest in the UK, especially in
R&D?
6. Would the regulatory system be costly for the regulator to
administer and the companies to comply with?
48. 48
2 Forms of Price Regulation in UK
•Pharmaceutical Price Regulation Scheme
(PPRS) regulates manufacturers’ profits
earned on sales to the National Health Service
of branded medicines (on- and off-patent)
•Schemes M ands W control the reimbursed
price of generic medicines paid to dispensing
pharmacists and doctors
49. 49
The PPRS (2014)
•Have been variants of PPRS since 1960s
•Department of Health acts as regulator for whole UK
•Objectives of 2014 PPRS:
• Provide stability and predictability to Government and industry
• Support the NHS by ensuring that the branded medicines bill stays within
affordable limits
• Improve access to innovative medicines commensurate to outcomes they
offer patients by ensuring that medicines approved by NICE are available
widely in the NHS
• Reduce bureaucracy and duplication
• Support the Government’s growth and innovation agenda for life sciences
•Voluntary – but statutory alternative scheme for firms
that opt out
50. 50
The PPRS (2014)
•Covers branded pharmaceuticals sold to the
NHS
•Negotiated every 5 years or so between the
ABPI and the Department of Health
•Current scheme commenced 1/1/14
•Scheme applies to all companies supplying
BRANDED medicines to the NHS ≈ 80% by
value of pharma sales to NHS
•Indirectly controls price by regulating
profits earned by these firms
•Freedom of pricing at launch
51. 51
PPRS (2014)
For the first time, it sets limits to the growth of sales
of branded medicines to the NHS
52. 52
PPRS (2014)
Innovation and access to medicines
•NICE will not negotiate, publicly set or
publicly indicate prices
•The basic cost-effectiveness threshold will
be retained at a level consistent with the
current range and not changed for the
duration of the agreement
53. 53
The PPRS 2009 – still applies
•Flexible Pricing Schemes: where a company can increase
or decrease its original list price in light of new evidence or a
different indication being developed
•Patient Access Schemes: which will facilitate earlier patient
access for medicines that are not in the first instance found to
be cost and clinically effective by NICE within a framework
that preserves the independence of NICE
54. 54
Patient Access Schemes (1)
•Patient Access Schemes are schemes proposed by a
pharmaceutical company and agreed between the Department
(with input from NICE) and a pharmaceutical company in order to
improve the cost-effectiveness of a drug and enable patients to
receive access to cost-effective innovative medicines
•Note: only relates to England and Wales, as different HTA
arrangements are in place in Scotland and Northern Ireland
55. 55
Patient Access Schemes (2)
(Some) Key principles:
•Arrangements must respect the role of NICE
•Schemes are to be discussed first and agreed in principle
by the Department and the company
•Schemes should be clinically robust, clinically plausible,
appropriate and monitorable
•Any scheme should be operationally manageable for the
NHS without unduly complex monitoring, disproportionate
additional costs and bureaucracy
•Schemes should be consistent with existing financial flows
in the NHS and with local commissioning
•The more systematic use of such Schemes will need to be
reviewed in light of experience. The timing of such a review
will be jointly agreed but will be initiated not later than two
years after the commencement of this Agreement
56. 56
Patient Access Schemes
2014 PPRS:
•Simple discount schemes; and
•Complex schemes
•Simple discount schemes must meet the simple discount criteria which ensure
that a PAS imposes no significant ongoing additional burden on the NHS, as set
out in the PASLU process guide and the relevant PAS proposal template. The
other option for a scheme member would be to change the list price of the
product.
•Complex schemes include all other types of PAS. This could potentially
incorporate a wide range of models. To date, components of PAS have included:
• Rebates;
• Stock supplied at zero cost;
• Dose capping;
• Outcome-based schemes.
57. 57
UK PAS’ by type/therapeutic area
(July 2015)
Originally schemes were more
complex and varied in nature; now,
there is a trend towards simple
discounts due to confidentiality
agreements
Source: OHE analysis
Two thirds of schemes are
for cancer and one
quarter for
immuomodulating
biologics (mainly TNF’s)
58. 58
Cancer Drugs Fund (1)
• For oncology medicines specifically, a “Cancer Drugs
Fund” (CDF) was introduced in England in 2010
• Originally announced to run until 2014, it has now been
given extra money and extended until 2016
• The CDF provides a means of improving patient access
to cancer drugs, and is used to fund drug treatments,
including radiopharmaceuticals, for patients who have
been unable to access a drug recommended by their
oncologist
• This includes drugs that are either not routinely
available on the NHS or have not been approved or
appraised by NICE. It also provides fast track access to
cancer drugs that are awaiting NICE guidance as well as
access to drugs for less common cancers
59. 59
Distribution of NICE decisions for cancer drugs: before and
after introduction of Cancer Drugs Fund (2006 – July 2015)
Cancer Drugs Fund associated with increase in NICE rejections and
fall in restricted recommendations for cancer medicines
Source: OHE analysis
65. 65
Generics: M and W Schemes (2005)
•The reimbursed price (the Drug Tariff price) is the
volume-weighted average price charged by
manufacturers
•Manufacturers and wholesalers are required to
submit quarterly data to the Department of Health
on, among other things, net sales values and net
acquisition costs, on a product by product basis i.e.
including discounts
•Greater reliance on competition to control prices,
but the generics market is more closely monitored
than ever before
67. 67
But International Price Comparisons
are Sensitive to ….
•Manufacturers’ prices or final selling price to the payer?
•Brands or generics or molecules?
•Sample size and selection (value versus volume, degree of market
coverage)
•Bilateral versus multilateral
•Match single pack, match product form or price per unit (tablet,
DDD, IMS SUs, Kg)?
•Volume weights: unweighted, own country (Paasche) or foreign
weights (Laspeyres)?
•Choice of exchange rate
•What exactly is the question you are trying to answer?
68. 68
Useful Reading
•Cockburn. I. and Henderson, R., “Racing to Invest? The Dynamics of Competition in Ethical Drug Discovery”, Journal of Economics and Management
Strategy, Volume 3, No. 3, Fall 1994, pp. 481-519, 1994
•Danzon, P. and Chao. L., “Prices, Competition and Regulation in Pharmaceuticals: A Cross National Perspective”, Office of Health Economics, London,
2000a.
•Danzon, P. and Chao. L., “Cross-National Price Differences for Pharmaceuticals: How Large and Why?” Journal of Health Economics, 2000b.
•Danzon, P. and Chao. L., “Does Regulation Drive out Competition in Markets for Pharmaceuticals?” Journal of Law and Economics, 2000c.
•Danzon, P. and Kim, J., “The Life Cycle of Pharmaceuticals: A Cross-National Perspective” Office of Health Economics, London, 2002
•Di Masi, J., Hansen, R. and Grabowski, H., “The Price of Innovation: New Estimates of Drug Development Costs”, Journal of Health Economics 22, pp.
151-185, 2003
•Garau and Sussex, 2007, “Estimating pharmaceutical companies’ value to the UK economy. Case study of the BPG”, Office of Health Economics, London
•Grabowski, H., Vernon, J. and DiMasi, J., “Returns on Research and Development for 1990s New Drug Introductions”, Pharmacoeconomics, Supplement
3, 2002.
•Henderson, R. and Cockburn, I., “Scale, Scope and Spillovers: The Determinants of Research Productivity in Drug Discovery”, RAND Journal of
Economics, Vol. 27, No. 1, pp. 32-59, Spring 1996.
•Kettler, H., “Updating the Cost of a New Chemical Entity”, Office of Health Economics, London, 1999.
•Mason. A., Towse, A., Drummond, M. and Cooke, J., “Influencing Prescribing in a Primary Care Led NHS”, Office of Health Economics, London, 2002
•Mestre-Ferrandiz, 2006, “The Faces of Regulation. Profit and price regulation of the UK pharmaceutical industry after the 1998 Competition Act”, Office of
Health Economics, London
•Office of Health Economics, 2006, The Many Faces of Innovation
•Pharmaceutical Industry Competitiveness Task Force (PICTF), 2005, available at http://www.advisorybodies.doh.gov.uk/pictf/publications.htm
•PPRS: Documents available at: http://www.dh.gov.uk/en/Healthcare/Medicinespharmacyandindustry/Pharmaceuticalpriceregulationscheme/DH_494
•Sussex, J. and Marchant, N., (eds.), Risk and Return in the Pharmaceutical Industry, Office of Health Economics, 1999.
•Towse, A., Pritchard, C. and Devlin, N., (eds.), 2002, Cost-Effectiveness Thresholds: Economic and Social Issues, Office of Health Economics and King’s
Fund
•Towse, A. and Danzon, P., 2003, “Differential Pricing for Pharmaceuticals: Reconciling Access, R&D and Patents”, Working Paper 03-7, AEI-Brookings
Joint Centre for Regulatory Studies
•Wertheimer, A. Levy, R. and O’Connor, T. 200, “Too many drugs? The clinical and economic value of incremental innovations” in Investing in Health: The
social and economic benefits of health care innovations, Volume 14, pp. 77-118, Elsevier Science Ltd.