Ophthalmology Market Access by Dr Steven Bradshaw

©2015 Market Access Solutions Ltd. All rights reserved.
Market Access Solutions, Ltd Market Access Strategies for the Global Healthcare Environment
Therapeutic landscape in posterior segment disease: opportunities for
patients, clinicians and for industry
Presented at the 15th EURETINA Congress NICE; 17th September 2015: #FP-7265
Steven Bradshaw BSc (Hons), MB BChir, MRCOphth
European Director, Market Access Solutions

Disclosure
Disclosure
2
 Market Access Solutions (Market Access Solutions Ltd and Market Access Solutions LLC) receive fees from
industry for consulting on new and existing medicines
 We did not receive any sponsorship to attend or present at EURETINA
 We will not be endorsing any particular product

1. Bringing a new eye therapy to market
2. White space assessment in retinal disease
• Objectives and methods
• Findings
• Considerations for clinical development in dry AMD
4. Conclusions and recommendations
3
Overview
Report Content

Once upon a time … efficacy and safety used to be enough
Background
4
... provided that pharmaceutical products were safe
and effective, doctors could prescribe them, patients
could get their prescriptions filled, and payers would
reimburse the costs

Now a number of challenges exist … payers need to balance healthcare demands
with budgetary constraints
Background
5
To optimize market access, pharma
companies also need to demonstrate the
value of the product during national and
regional pricing and reimbursement
negotiations … the requirements of payers
are different to those of regulators
Phase 1 Phase 2 Phase 3
Clinical Development
MA P&R Launch
Commercialisation
Quality
Safety
Efficacy
Payer value = Market access
To achieve market
authorisation/regulatory
approval, pharma companies
need to demonstrate that a
product is safe, effective and
meets quality standards
4th hurdle
The difference between market access and marketing is that market access
requires evidence that payers value ...

What payers need to know … and what they tend not to consider
Background
6
 Is the product needed? (unmet clinical need)
 Does it work? (clinical efficacy)
 How well does it work? (clinical effectiveness)
 Can we control its use? (patient population)
 Is it worth it? (cost effectiveness)
 Can we afford it? (budget impact)
 Tolerability
 Mechanism of action
 Pharmacokinetics
 Societal costs
 Route of administration
 Convenience for patients
 Indirect costs
Usually not factored in payer decision makingUsual factors in payer decision making

• Findings
7
Overview
Report Content

Assessing opportunities for new retinal therapies was the ultimate aim of this research
Objectives and Methods
Overarching
goal Understand access opportunities for new entrants in posterior segment disorders
Key focus
areas of the
assessment
Understand the preceding and current/future clinical and market perspectives
 Identify the remaining unmet needs in each indication
 Outline the value drivers for access for future entrants
 Review HTA recommendations for each publically available evaluation
 Key insights for clinical trial design and launch in multiple indications and how
future agents can expect to achieve access and price
Indications Wet AMD, dry AMD, DME, non-infectious posterior uveitis and vitreomacular traction
8

©2015 Market Access Solutions Ltd. All rights reserved. 9
Secondary research and qualitative interviews with 15 payers, decision makers and key
opinion leaders across three major markets in Europe, plus Canada and Japan
Objectives and Methods
JapanFrance
Germany
United Kingdom
Market in scope
Canada

• Findings
10
Overview
Report Content

Characteristic Wet AMD Dry AMD DME
Non-infectious
posterior uveitis
Vitreomacular
traction
Prevalence
170 per 10,000 and
increasing
380+ per 10,000
and increasing
Average of 7% of
diabetics affected
38 per 100,000 22.5 per 100 000
Disease burden
A leading cause of
blindness
Minor progressive
symptoms
Major cause of
visual impairment
Debilitating in
acute phase
Symptoms are mild
and develop slowly
Economic burden /
Therapy costs
High cost anti-VEGF
drugs
No reimbursed
treatment
High cost anti-VEGF
drugs
Low cost drugs
available
One off surgical
costs
Unmet need
Burdensome
disease but good
treatments
No current
therapies
Burdensome
disease but good
treatments
Treatments
available
Treatments
available
Competitive environment
(pipeline)
Fovista and
biosimilars
Lampalizumab and
other products in
pipeline
Several licensed
products launched
Few treatments in
pipeline
Few treatments in
pipeline
Payer management
PAS required for
cost-effectiveness
No cost control but
no available budget
PAS required for
recommendation
Off-label products
available
PAS maybe
required for
recommendation
HTA challenges
Current therapies
required PAS
Unknown
environment
Population
restrictions likely
Low incentive to
review
Payers more
aware since Jetrea
Overall opportunity of
entering therapy area
MEDIUM:
Competitive and
highly managed with
effective SoC
HIGH: First to market
product could have
significant impact
MEDIUM: High
population but
treatments available
LOW: Small
population with off-
label treatments
used
LOW: Small
population and
increased Payer
awareness
Opportunity Assessment
11
Opportunity in posterior segment disorders based on multicriteria assessment of
perceived clinical burden, unmet need, market dynamics and payer environment
PAS = patient access scheme; SoC = standard of care

12
The market landscape is expected to transform should one or more pipeline
developments prove successful
 Lampalizumab is expected to be the
first drug to enter the dry AMD
market
• 20.4% reduction in geographic
atrophy progression from month
6 through month 18 in its phase II
study
• phase III is ongoing and includes
patients that are positive for the
complement factor I biomarker;
the primary endpoint is reduction
in the rate of geographic atrophy
progression
 Cell therapies are still in early phase
of development, showing promising
results and indicate a high price tag
Therapies being developed for dry AMD - Clinicatrials.gov Dec ’14
The key challenge for any product to reach patients, is to demonstrate clinical and
economic outcomes that resonate with payers ...

• Findings
13
Overview
Report Content

1 2 3 4 5
Payers (n=4) KOLs (n=10)
Clinical Development Considerations
14
Perceptions vary between payers and KOLs, with the former suggesting change in
BCVA and the latter change in GA as the optimal primary efficacy measure in dry AMD
Change in Best Corrected Visual Acuity (BCVA)
Functional
Change in central visual fields
Near acuity/ Reading speed
Contrast sensitivity
Visual Function Questionnaire (VFQ-25)
AMSLER testing
Change in drusen size/ area
Anatomical
Hyper/ hypo-pigmentation
Central retinal thickness
Rate of change in the area of geographical atrophy (GA)


← Value →
Notes on interpretation: Interviewees were asked to score the value of each endpoint on a scale of 1 (low) to 5 (high). Scores were averaged
for payers (n=4) and KOLs (n=10).
Low High
Perceptions of dry AMD endpoints
1 2 3 4 5
Payers (n=4) KOLs(n=10)
=Payer-preferred =KOL-preferred

1 2 3 4 5
Payers (n=4) KOLs (n=10)
15
Other anatomical endpoints with no clear relevance to patient outcomes are scored
lower in value, especially by payers
 Anatomical endpoints are scored low in value because:
• Payers are unfamiliar with terms and clinical meaningfulness of these changes
and how vision is affected as a direct consequence
• KOLs are not sure about the objectivity of these measurements and question
their correlation to dry AMD progression
“These are just surrogate endpoints and so far I have not seen convincing reasons for why they are
important. If there were publications to suggest otherwise then my opinion would be different. The first
thing I do when evaluating a new therapy is to put down the manufacturer’s submission and look at
PubMed to see the validity of the claims for myself.”– Payer, Germany
“Can usually use OCT for AMD and DME but it is not clear what is the best measure to use for dry AMD and
which one best correlates with disease progression. Nothing good at the moment.”– KOL, Germany
← Value →Low High
Perceptions of dry AMD endpoints
1 2 3 4 5
Payers (n=4) KOLs(n=10)
Notes on interpretation: Interviewees were asked to score the value of each endpoint on a scale of 1 (low) to 5 (high). Scores were averaged
for payers (n=4) and KOLs (n=10).
Change in drusen size/area
AnatomicalHyper/hypo-pigmentation
Central retinal thickness

• Findings
16
Overview
Report Content

Conclusions and Recommendations
17
Majority of threats and payer uncertainty to funding patient access can be mitigated
through clinical community support of effective therapies and payer education
Uncertainty around acceptability of
predominantly anatomic endpoints
Publish, even if it is obvious to
ophthalmologists that GA size matters,
because payers need evidence to support their
decisions
Size of patient population may necessitate
restriction to subgroups to control budget
impact
Help get therapies to those groups with higher
need through research and publications
Once treatments come to market, later
entrants will require H2H trials
Champion effective and safe treatments to
best inform industry so that future trials use
the most appropriate comparator and patients
will be able to access the best medicines
Slower progression rates and reduced
burden for earlier stages means that
payers may be initially apprehensive about
up front payments
Work to develop real world data from
registries or small local studies to help to
support access to therapies that are safe and
effective, particularly for high cost medicines

Market Access Strategies for the Global Healthcare Environment
For further information
Steve Bradshaw, Director Market Access Solutions
1000 Great West Road, London, TW8 9DW
Tel: +44.203.675.8995
E: sbradshaw@mktxs.com
.mktxs.com

19
Dry AMD represents a considerable market opportunity especially for agents that
can successfully target and prevent the progression of geographic atrophy
Opportunities Threats
 Large and increasing patient population with no
satisfactory therapies
 Payers’ willingness to pay is thought to be high
due to high unmet need and potential to avoid
progression to expensive-to-treat wet AMD
 Initial clinical outcome requirements may be more
relaxed due to current lack of comparative
treatments; for the first entrant a placebo-
controlled trial will be satisfactory
 Market opportunity is estimated at US$5 billion
globally
 Uncertainty around acceptability of predominantly
anatomic endpoints
 Size of patient population may necessitate
restriction to subgroups to control budget impact
 Once new treatments penetrate the market, later
entrants will require H2H trials
 Slower progression rates and reduced burden for
earlier stages means that payers may be initially
apprehensive about up front payments –
especially for high-cost regenerative medicines
 ×

20
From a combined payer-KOL perspective, a placebo-controlled trial of at least 2 years
in patients with moderate visual loss represents the best design (for a first entrant)
Feature Requirement Rationale
Pivotal design Placebo control (first entry)
 Because no SoC available, placebo trial would be acceptable
 Vitamin supplements can be used in both arms, as usually recommended
Duration 2 years minimum
 As a slowly progressing disease 2 years is judged the minimum acceptable
 Most interviewees also ask for an additional 1-3 years extension trial
Population
Stage: Intermediate
Size: 250+ patients per arm
Exclusions: Late-stage, other
retinal diseases
 Intermediate stage patients with moderate visual loss (e.g., 20/80 vision)
recommended as motivated to complete study and likely to see better
differentiation between study arms than early or late stage
Primary
endpoints
Payers: BCVA stab/improvement
KOLs: GA progression
 BCVA most important for licensing and for payers
 GA progression (as measured) is a KOL favourite, but correlation with VA
would be needed to improve payer acceptance
Outcomes
3 lines difference in ETDRS
20% relative change in GA
 Payers familiar with 3 lines gain on ETDRS from previous experience with
wet AMD trials; % change in GA in line with lampalizumab’s outcomes
Safety
Ocular AEs: e.g., infections
Other AEs: e.g., immunological
 Any therapy requiring an invasive procedure in patients with early disease
and low-moderate disease burden must have few side effects
“There’s no treatment at the moment so sham study is fine, but you
compare against nutritional supplementation … and, because it's a
slowly progressing disease I’d want data for 5 years.”– KOL, France
“The best ones are those with some element of visual loss and then you'd see a
gradual decline in the control group. These patients are also more highly
motivated to see out the trial because they have the early signs.” – KOL, UK
Requirements for a dry AMD clinical trial based on interviewee responses

Characteristic Wet AMD Dry AMD DME
Non-infectious
posterior uveitis
Vitreomacular
traction
Prevalence
Clinical burden
Economic burden
Unmet need
Competitive environment
Payer management
HTA challenges
Overall opportunity of
entering therapy area
MEDIUM:
Competitive and
highly managed with
effective SoC
HIGH: First to market
product could have
significant impact
MEDIUM: High
population but
treatments available
LOW: Small
population with off-
label treatments
used
LOW: Small
population and
increased Payer
awareness
21
Opportunity in posterior segment disorders based on multicriteria assessment of
perceived clinical burden, unmet need, market dynamics and payer environment
Large Medium SmallKEY:

To facilitate market access, manufacturers can offer support to physicians and
patients in the form of education, administrative assistance and research funding
Markets Focus Manufacturer provided services
 Educational programs for patients are well-received by French ophthalmologists and patient
groups provide a forum for support
 Educational and administrative support programs in place
 German payers are skeptical of manufacturer involvement at local level, preferring straight
discounting at the national or regional level
 Novartis have entered partnerships with the NHS to provide static and mobile eye clinics to
improve service to local areas
• Currently 3 schemes are running in England and Wales
 Novartis and Bayer provide local and personal assistance to patients who may have trouble
paying for Lucentis and Eylea through Special Access Programs
• If patients do not have full coverage (e.g. they are under 65 years old) then copayment rates of 10-
20% can be prohibitive on expensive VEGF therapies
• Manufacturers will investigate with the health insurers for personal plans so patients can access
treatment
• This ensures patients can receive treatment at initial consultation without the need for complicated
insurance assessment
 Japanese ophthalmologists are open to approach from manufacturers who may offer prescriber/
patient incentives
= Patient-focused= Physician-focused
22

23
Inherent differences between regenerative medicines and traditional
pharmaceuticals present multiple challenges for access and reimbursement
Pricing, reimbursement and access issues
Gene
therapies
Traditional
pharmaceuticals
Implications
Involves multiple procedural steps that may
be separately reimbursable
Often Rarely
• Similar to reimbursement for a
device/procedure
• Failure to achieve reimbursement of any
component may jeopardize reimbursement of
the entire procedure
HTA will focus on the cost-effectiveness of
the entire procedure
Yes
Not often
applicable
• Requires HEOR data collection regarding entire
procedure
May involve multiple billing codes/tariffs
and/or payment centers for reimbursement
of the full procedure
Yes No
• Lack of appropriate codes/tariffs or payment
may limit or preclude access and uptake
May involve requirements for longer-term
data collection to demonstrate value (incl.
post-market follow-up data)
Yes Sometimes
• This is a top HTA criticism of most regenerative
therapies in global markets to date
Strong potential to be more costly than
standard of care alternatives
Often Sometimes
• Higher cost means that therapies must
demonstrate significant outcome
improvements vs. standard of care
May enable a disease cure or prolonged
therapeutic effect
Yes Rarely
• Can alter the balance of benefit-cost tradeoffs
in value assessment

Summary
24
“They need to provide data beyond the regulatory approval standards – it does no good to compare a new and expensive therapy
to a placebo if a comparator already exists.” – National Payer, Germany
Ophthalmologists are
central to identification of
existing unmet needs,
supporting clinical
development and
monitoring through
research and in shaping
payer beliefs, particularly
where there are
misconceptions
For high cost regenerative
medicines (gene therapies
anticipated to cost
>€1 million per dose), ‘payer
centric’ clinical development
and consideration of
innovative funding models
(pay for performance,
annuity-based, risk pools …
even crowd funding) are
needed to ensure patient
access
Payers have established
their own therapeutic
guidelines, manage access
more tightly and scrutinize
the price of new therapies,
meaning pharma companies
need to provide the
evidence sought by payers,
specifically information such
as comparative
effectiveness, cost
effectiveness and real world
data
Evidence needs Clinical support Funding models

Ophthalmology Market Access by Dr Steven Bradshaw

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