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

• 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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The ultimate goal for a new compound

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

• 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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What happens if payers decide to not reimburse a new drug?

General market access flow combined with key 7 payer questions

Why do patients need this?

What am I currently paying for treating this disease?

What are the consequences of not funding?

What patients should get it and how can use be limited to appropriate patients?

How is your drug better than the alternatives in my specific setting?

How much is your drug and why is it worth the cost?

Can I afford it and what part of my budget will I use to fund it?

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Evaluate Product

Determine Funding Path or Benefit Coverage

Negotiate Price & Contract

Manage Utilization

Determine Reimbursement Rate

Economic Value Clinical Value

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• 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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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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When could a company influence a positive payer decision?

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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• 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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Case study: Sunitinib in Renal Cell Carcinoma

• Preparation of the market: Awareness of disease burden

• Epidemiological review • Economic review

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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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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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Case study: Sunitinib in Renal Cell Carcinoma

• Cost-Effectiveness in 2L: Various publications

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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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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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Case study: Sunitinib in Renal Cell Carcinoma

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• 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

Conclusions

Thank you

Dr. Stefan Walzer General Manager

AiM Assessment in Medicine GmbH Tel.: +49 7621 705 105 20

stefan.walzer@assessment-in-medicine.de