The importance of the health care payer view in the development of a new compund is exemplarily shown and was presented at a conference in London in March 2012

1. Case Study:
Launching and Selling
a Pharmaceutical Compound
with the Payer in Mind
Dr. Stefan Walzer
General Manager
AiM Assessment in Medicine GmbH
Tel.: +49 7621 705 105 20
stefan.walzer@assessment-in-medicine.de

2. The ultimate goal for a new compound
• The ultimate goal is Commercial Success
• Commercial success is defined by revenue
generation
Revenue = Sales * price
• Payers could influence the success of a new
compound on each side of the equation
• Sales: Caps, volume agreements, paybacks, etc.
• Price: Discount, Cost share agreement, etc.
2

3. Payer perspective critical for
commercial success
• Payers dictate global pharmaceutical pricing and
market access today.
• While there are critical costs and resource trade-
offs to consider, companies should focus on the
payer perspective throughout the drug
development and commercialization processes.
• Those that build the payer perspective into phase
III drug trials and ensure the availability of
supportive evidence for all key claims will create
a strong payer value case.
3
Source: Schonveld, Think like a payer, 2011

4. What happens if payers decide to not
reimburse a new drug?
• Implications for the pharmaceutical
company:
• No revenue generation
• No Return on Investment
• Potential lay-offs
• Implications for the patients
• No new (more efficacious) drug available
• Potential negative impact on Quality of Life
• No new future innovations which might be based on a
new compound
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5. General market access flow combined
with key 7 payer questions
1 Why do patients need this?
Determine Funding Path or Benefit
Coverage
What am I currently paying for treating this
2 disease?
Evaluate Product
Clinical Value Economic Value How is your drug better than the
3 alternatives in my specific setting?
How much is your drug and why is it worth
4 the cost?
Determine Reimbursement Rate
Can I afford it and what part of my budget
5
will I use to fund it?
Negotiate Price & Contract
6 What are the consequences of not funding?
What patients should get it and how can
Manage Utilization 7 use be limited to appropriate patients?
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6. When could a company influence a
positive payer decision?
• Between phase I and II program
• Early pricing & reimbursement evaluation
• Investment decisions using early modelling
• Before start of phase III program
• Payer consultation (e.g. NICE UK, GBA Germany, …)
• Joint regulatory and payer consultations
• Design of the study including the payer requirements:
– Endpoints
– Comparator
– Sample size & study power
– Subgroups / biomarker
– Minimum effect size
– Potential bias
– …
6

7. When could a company influence a
positive payer decision?
• After the availability of the phase III data
• Availability of data (final vs interim data)
• Payer consultation (e.g. NICE UK, GBA Germany, …)
• Payer story aligned with regulatory submissions
• Final pricing decision
• Health Economic data including cost-effectiveness
• Strategic Publication Planning
– Primary efficacy article
– Secondary articles
– Health Economics
7

8. Case study: Sunitinib in Renal Cell
Carcinoma
• Renal Cell Carcinoma from a payer‘s
perspective:
• Relatively low incidence / prevalence
• Few ‚old-school‘ treatments
• High clinical trial failure rate
• Low budget impact
• Oncology: Key endpoints are Overall Survival (OS) and
Progression Free Survival (PFS)
8

9. Case study: Sunitinib in Renal Cell
Carcinoma
• Early development phase:
• Dosing studies
• Potential price anchors:
– ‚Old-school‘ RCC treatments
– Innovative oncology treatments in other indications
» Erlotinib
» Bevacizumab
» Cetuximab
» Trastuzumab
» Imatinib
» Rituximab
»…
9

10. Case study: Sunitinib in Renal Cell
Carcinoma
• Preparation of the market: Awareness of
disease burden
• Epidemiological review
• Economic review
10

11. Case study: Sunitinib in Renal Cell
Carcinoma
– Start with the „worst“
patients: 2L RCC
– No comparator in trials
– Phase II trials as basis
– As efficacy results were so
astonishing Pfizer decided to
request a marketing
authorization and
reimbursement
11

12. Case study: Sunitinib in Renal Cell
Carcinoma
• However, the feedback by payers was different
(examples)
• France: ASMR III, conditional market access (before
Mediator scandal!)
• Germany: Full market access (no AMNOG at that time!)
• Italy: Long negotiations, cost-share agreement
• UK: No NICE recommendation for 2L
12

13. Case study: Sunitinib in Renal Cell
Carcinoma
• Cost-Effectiveness in 2L: Various publications
13

14. Case study: Sunitinib in Renal Cell
Carcinoma
• Break-through data in 1L
• Confirming the strong signals in 2L in front of clinical
and payer audience
• Beneficial QoL outcomes
14

15. Case study: Sunitinib in Renal Cell
Carcinoma
• Indirect Comparison and Cost-
Effectiveness in 1L
• NICE confirming cost-
effectiveness
• ASMR II in France
• Market access and fast uptake in
most countries around the world
15

16. Conclusions
• Payers dictate global pharmaceutical pricing and
market access today.
• In order to have a successful market access story the
payer perspective would need to be included as early
as possible, the latest for the information of the
phase III design
• In the case study Pfizer planned especially for a
market access in 1st line and developed an optimized
strategy for it
16

17. Thank you
Dr. Stefan Walzer
General Manager
AiM Assessment in Medicine GmbH
Tel.: +49 7621 705 105 20
stefan.walzer@assessment-in-medicine.de