Investigation of managed entry agreements to address uncertainty around orphan drug pricing and reimbursement decisions, poster session, Dolon LTD, ECRD 2016

1. Objectives
• National payers have increasingly looked to use managed entry
agreements (MEAs) to expedite patient access to orphan medicines,
while managing uncertainty around their clinical evidence and
budget impact.
• We aim to identify, describe and classify different MEAs that have
been used to enable patient access to orphan medicines.
• We propose a taxonomy to categorise different types of MEA,
including an assessment of when it makes sense to use each
type of scheme, and in which countries different schemes are
most commonly used (Figure 1). We also propose a framework
that categorises the level of financial risk being shared between
the pharmaceutical company and healthcare payer across different
MEA types (Figure 2).
Methods
• A literature search was conducted to identify non-confidential
MEAs involving orphan medicines.
• Interviews with pharmaceutical industry and health system
stakeholders were carried out to complement information identified
though the literature search.
• A bespoke taxonomy was created and a database was established
to record identified schemes.
• It should be noted that the output of this research is not expected
to be comprehensive, given the confidentiality of MEAs in a number
of countries.
Results
• Seventy examples of managed entry agreements involving EMA
orphan designated drugs were identified, which were broadly
classified into two categories: (1) outcomes-linked schemes; and
(2) financial-based schemes (Figure 1).
• Outcomes-linked schemes require the tracking of pre-determined
patient outcomes in order to adjust the pricing and reimbursement
status of the medicine e.g., via re-assessment of inclusion on a
‘positive list’, re-assessment of price, or adjusting price via rebate
mechanisms. Examples of identified outcomes-linked schemes
were further categorised into three subtypes: (1.1) coverage with
evidence development, whereby reimbursement status and/or
pricing is dependent on the outcome of evidence developed
post-marketing (e.g., Vimzim for MPS IVa in England); (1.2)
conditional treatment continuation, whereby continued
reimbursement is conditional on meeting short-term treatment
goals (e.g., Solaris for aHUS in Australia); (1.3) outcomes guarantee,
which requires a rebate, refund or price adjustment if a performance
target is not met (e.g., Adcetris for HL and ALCL in Italy).
52
Investigationofmanagedentryagreements
toaddressuncertaintyaroundorphandrug
pricingandreimbursementdecisions
Sear R­1
, Hutchings A2
.
1. Dolon Ltd, London, UK. richard.sear@dolon.com 2. Dolon Ltd, London, UK. adam.hutchings@dolon.com
Conclusion
• MEAs have been implemented with varying degrees of success
across different markets as a tool to manage patient access to
orphan products, whilst managing the risk of uncertainty of clinical
outcomes and budget impact. However, with the proliferation of
real world healthcare data, combined with the increasing need
to drive efficiencies in health system budgets while making
innovative new treatments available to patients, there is both an
increasing capability and desire to experiment with MEAs.
• Rare disease medicines provide a good basis for MEAs, considering
patient populations are smaller than non-rare populations (and it
is potentially easier to track treatment outcomes). In addition,
there is typically a high degree of uncertainty around patient
population (prevalence) numbers and trial evidence (which is often
based on smaller trial populations and occasionally surrogate
endpoints) compared with trials in non-rare indications.
• MEAs can be used to manage some of the uncertainty around
patient population numbers, and the transfer of clinical trial evidence
to real world patient populations (whilst helping health systems
and pharma companies to learn more through the collection of
post-launch data).
• Different scheme constructs are suited to manage different
uncertainties – for example, where there is uncertainty about the
transfer of trial outcomes to a real world population, an outcomes-
guarantee, or coverage with evidence development scheme would
help address this. Alternatively, if there is uncertainty around
prevalence or incidence claims across a specified population (or
expectation of off-label use), a population spend cap, or price
volume agreement would be better suited.
• Consideration should be given to the capability requirements and
costs associated with implementing such schemes in different
countries. Outcomes-linked schemes will typically require the use
of registries and require incentives to ensure high-quality data
entry. The administrative burden of data collection and rebate
processes can be high on a health system, and should be
considered in designing appropriate schemes.
WHAT IS THE COMPANY SELLING
Figure 2. Level of risk shared in different MEAs
FULL SUITE OF
SERVICES WITH
POPULATION RISK:
UNIT BEING SOLDDRUG/REGIMEN
OUTCOMES-LINKED
SCHEMES:
FINANCIAL-BASED
SCHEMES:
TOTAL CARE
OF PATIENT
PATIENT
OUTCOME:
UNIT:
SELL INTEGRATED HEALTH
SERVICES TO PAYORS AND
PROVIDERS ON PERFORMANCE
BASE (NOT EXPLORED HERE)
E.G., RESPONSE TO
TREATMENT, MONTHS
SURVIVAL
FULL RISK1
NO RISK
SHARED
SHARED RISK
(E.G.,
OUTCOMES-
LINKED
REBATE)
ONE-SIDED
RISK SHARE
(E.G., COST
CEILING)
E.G., PILL, PACKAGE
SEEK COLLABORATIVE
APPROACHES TO SHARE RISK
ASSOCIATED WITH A
MEDICINE PERFORMING IN
THE REAL WORLD BETWEEN
THE COMPANY AND PAYER
SELL INNOVATIVE MEDICINES
UNDER FINANCE-BASED
MODELS
What is the
level of
financial
risk
shared?
1
Pharma company take on risk
of full care of patient (beyond
just medicine treatment)
• Financial-based schemes involve contracting around unit price
and/or volume, rather than patient outcomes brackets (Figure 2).
Identified examples include; space (2.1) patient spend caps,
patient-level utilisation caps, In which any additional volume is
provided free of charge, or retrospectively reimbursed via rebates
(e.g., Firdapse for LEMS in UK); (2.2) Manufacturer-funded initiation,
in which the manufacturer provides the product free of charge
(or at a discount) for treatment initiation (e.g., first cycle); (2.3)
Population spent caps, in which a maximum spend is agreed for
a specified population, regardless of volume (e.g., Naglazyme for
NPS VI in France); (2.4) Price volume agreements, in which different
price points are applied, depending on volume used (e.g., Firazyr
for symptomatic treatment of acute attacks of hereditary angioedema
in adults in Belgium).
Figure 1. Taxonomy of Managed Entry Agreements
Coverage and/or price level
adjusted based on evidence
developed post-marketing
• When there is uncertainty
about transfer of trial
outcomes to RW population
Coverage conditioned on
short-term treatment goals
• When there is uncertainty about transfer
of trial outcomes to RW population
Rebate, refund or price
adjustment in case performance
target not met
• When a product is brought to market
using surrogate endpoints
• When there is uncertainty about trial
outcomes transferring to RW population
Patient-level utilisation caps, with
additional treatment either free of
charge or not payer funded
• When there is significant variation
in dose/duration of treatment
between individual patients
Manufacturer supplies
product free of charge for
treatment initiation
• When there is uncertainty about
response rate, or adverse events that
present early after treatment initiation
Maximum spend amount
agreed, irrespective of product
volume supplied
• When there is uncertainty about the
prevalence/incidence claims across
specified population
Different price points apply,
depending on volume
thresholds achieved
• When there is uncertainty about the
prevalence/incidence claims across
specified population
DESCRIPTION WHEN USED: COUNTRIES IDENTIFIED: CONSIDERATIONS
MANAGED ENTRY
AGREEMENTS
1. OUTCOMES-
LINKED SCHEMES
2. FINANCIAL-
BASED SCHEMES
1.1 COVERAGE
WITH EVIDENCE
DEVELOPMENT
CONDITIONAL
COVERAGE
OUTCOMES
GUARANTEE
PATIENT
LEVEL
1.2 CONDITIONAL
TREATMENT
CONTINUATION
1.3 OUTCOMES
GUARANTEE
2.1 PATIENT
COST CAP
2.2.
MANUFACTURER
FUNDED INITIATION
2.3 REVENUE CAP
2.4 PRICE-VOLUME
ADVANTAGES
• INCENTIVISE INNOVATION TO
IMPROVE PATIENT OUTCOMES
• HAVE POTENTIAL UPSIDE AND
DOWNSIDE FOR BOTH CONTRACTING
PARTIES (SHARED RISK)
DISADVANTAGES:
• HIGH ADMINISTRATIVE BURDEN
ADVANTAGES
• INCENTIVISE INNOVATION TO
IMPROVE PATIENT OUTCOMES
• TYPICALLY EASIER TO IMPLEMENT
(NO TRACKING OF PATIENT
OUTCOMES)
• APPEAL TO COST-SENSITIVE PAYER
(RISK IS WHOLLY TRANSFERRED TO
COMPANY)
DISADVANTAGES:
• PURELY COST-BASED, PROVIDE NO
INCENTIVE TO IMPROVE PATIENT
OUTCOMES
FINANCIAL-BASED
SCHEMES:
OUTCOMES-LINKED
SCHEMES:
POPULATION
LEVEL
8th European Conference on Rare Diseases Orphan Products 2016 in Edinburgh, Scotland