The case study discusses the importance of considering the payer perspective in the development and commercialization of pharmaceutical compounds, emphasizing how payers influence pricing and market access. Companies should integrate payer insights early in the drug development process to create a strong value case, as illustrated by the case of sunitinib in renal cell carcinoma, which faced various payer reactions and negotiations. The conclusion highlights that a successful market access strategy requires incorporating payer considerations from the beginning, particularly in Phase III design.

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.
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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.
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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
– …
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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
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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)
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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
»…
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10. Case study: Sunitinib in Renal Cell
Carcinoma
• Preparation of the market: Awareness of
disease burden
• Epidemiological review
• Economic review
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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
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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
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13. Case study: Sunitinib in Renal Cell
Carcinoma
• Cost-Effectiveness in 2L: Various publications
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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
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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
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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
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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