Lecture 2: Management of a clinical development project

A target product profile (TPP)

A summary statement of the key claims that a company wants to be able to talk about their product when on the market. —> provides the vision

A target product claim (TPC)

A detailed description of:

• Claims and the customers to which they are targeted (customers = patients).

• How those claims will be delivered and which studies will provide the supporting information, together with GO/NO decisions

What kind of other business processes do the TPP and TPC drive?

• Discovery & Licensing

• Clinical development & health economics programmes (via TPC)

• Regulatory requirements, pricing, forecasting, portfolio review

What is the TPP/TPC based on?

• Clinical experience

• - Physicians (internal/external)

• - Advisory board

• - Patient interviews – patient insight important

• Market research

• Public information

• Right balance between meeting market needs and technical feasibility

What are key questions for the TPP?

• What is the medical need?

• - Patient perspective

• - Physician perspective

• Competitor insight

• - What is on the market today?

• - What will be on the market tomorrow?

• What do the payors want?

How can you expand the vision in the TPP into a more tangible form?

• Clear and detailed links of claims to clinical studies and customers

• Description of these studies e.g. numbers, comparators, measures/end-points

• Detailed description of claims, customers and how they will be delivered

What does a TPC drive?

Decision making - GO/NO GO

What else is the TPC specific on?

Specific on claim types e.g. regulatory, promotional, data display

• What statement may be allowed in e.g. the label

Name 4 claim types.

1. Label claim

2. Promotable claim

3. Data display

4. Scientific exchange

Label claim

The data to support the claim must be included in the label

Promotable claim

The data to support the claim does not need to be included in the label but must be supported by adequate and well-controlled trials

Data display

When a specific promotable claim cannot be made but the data can be displayed in promotional or scientific materials to convey message

Scientific exchange

Data cannot be displayed in promotional materials, but may be communicated to health care professionals through e.g. scientific publications

Which to organisations have harmonised data requirements and a review process

US and EMA

What does China specifically require?

Safety+PK study in Chinese patients.

Who work together on target labeling

1. Commercial

• Product information claims

2. Regulatory

• Guidelines, authority advice, precedents, ‘risk assessment’

3. Clinical

• Treatment tradition, culture, opinion leaders, study results

Name 6 clinical development programme objectives.

• High quality data

• Faster Development

• Minimized costs

• Reduced late stage attrition

• Fast and uncomplicated Regulatory review

• Desired label

What is the purpose of the CDP?

To define all the clinical activities that will deliver the Target Product Claims and fulfil the requirements for global development and launch. CDP includes both strategic and operational clinical considerations.

What are 8 key clinical and regulatory strategy considerations?

• Are there regulatory guidelines to follow?

• Are there precedent study designs we need to adhere to?

• Mechanisms of action implications?

• Drug profile implications?

• Surrogate endpoints available?

• Primary endpoint recommendations?

• Competitors on the market?

• Competitors in development?

What are 6 contents of the CDP?

1. Situation Analysis and Clinical Strategy

2. Overview of Clinical Programme

• Flow chart of clinical studies

• Table of clinical studies

• TPP/TPC and regulatory requirements checks

3. Activities and Timelines

• Key clinical development milestones and deliverables

4. Resource requirements for Clinical Programme

5. Critical success factors and issues

6. Risk management and contingency plans

Phase I decision tree example

Regulatory strategy

Integrated part of the overall disease area strategy and CDP

Of which parts does the regulatory strategy consist?

1. Situation analysis (including regulatory environment)

2. Strategic considerations

3. Issue identification / opportunities:

• Regulatory guideline development

• Learning from agency interactions with similar products

• Use/participate in agency initiatives

4. Consultation and advice strategy

• Pre-Phase II meeting

• FDA end of Phase II-meeting

5. Filing strategy

• Strategic placement of studies