What do your target product profiles (TPP) say about you? Are you the pragmatist? The dreamer? Or maybe the pessimist? Fortunately, we cannot gauge your personality type from the TPP, or at least we should not be able to. We should, however, be able to understand enough about your product to gauge the potential value it will bring. ¹

A TPP – a document outlining the goals and potential value of an individual product in development – has many roles. It can be used internally as something for teams to aspire to, and externally to demonstrate to stakeholders the future value of the product, backed up by a growing evidence base.

Drug developers may be wary of developing TPPs, seeing them as documents based simply on assumptions and preferring to wait for the evidence from Phase III data. However, the value a TPP can bring to a company is as wide as it is deep. It provides a basis for early testing of endpoints, allows researchers to review clinical trial design, helps health economics teams to conceptualise models, supports market access teams in strategic preparation, allows commercial teams to perform meaningful financial forecasts, and increases collaboration across the different groups.

Using the TPP wisely

The concept of the TPP has been around for many years and, like anything that has become habitual, its optimal use and value can sometimes be lost. Therefore, it’s always worth looking at it afresh.

The TPP, at its most useful, is a multifaceted tool containing the elements that will demonstrate value to the product’s stakeholders, both internally and externally. The TPP should:

• make the differentiating elements of a product clear so that value can be understood.
• drive product development so that outcomes substantiate this value.
• make explicit the value the product will bring, for forecasting, net present value (NPV) calculations, and pricing and market access strategy, for example.
• unify communications both internally and externally to strengthen the value story.
• drive the commercial strategy in terms of positioning, and leverage points for patients, physicians, and payers.

To be truly useful throughout the drug development process, the TPP needs cross-functional input. To avoid the risk of the TPP becoming overloaded with every endpoint considered to ensure that all functions have their say, the elements of the TPP need to be prioritised. It should highlight only the key aspects that will differentiate and support the value story. Any components that fall outside of these priority areas can be regarded as ‘nice to have’ but may not win market share.

The TPP should be developed with three levels in mind:

1. Basic profile: the minimum level of evidence required for the product to make it to market.
2. Realistic profile: what the product is most likely to achieve, to forecast sales, and to position the product in the market.
3. Aspirational profile: the best-case scenario.

Building a full product picture needs thought about all three levels, along with a set of messages for each level for external testing. As well as confirming the value story, and planning the route to market and criteria for go/no-go decisions, the outcomes from the testing process can be used to develop mitigation strategies should the assumptions in each profile be proven incorrect.

Creating the TPP

To be a useful part of the product development process, the TPP needs to be more than just a hurried profile pulled together using Phase III results for a quick piece of market or payer research. Pulling the TPP together should ideally begin in early clinical development, at the end of proof-of-concept trials or even earlier, and before engaging in Phase II.

Creating the TPP at this early point provides an in-depth understanding of the critical clinical outcomes and their minimum value for success. This will allow drug developers to:

• direct the development of the Phase II/III clinical plan.
• carry out early assessment of reimbursement and pricing (important for new or crowded indications).
• develop new tools for clinical trials if required, such as patient-reported outcomes (PROs).
• criteria for go/no-go decision-making on the back of Phase II results.

As each new set of clinical trial results become available, the TPP needs to be adapted to reflect the endpoints and outcomes from each trial. The TPP also needs to follow important changes in the market, and needs full understanding of the current treatment pathway.

The TPP should be complete by the time the Phase III trials are in preparation. It should be used as the basis for the design of the pivotal studies, aiming to back up all of its key elements. Once trials are underway, making any further changes to either the clinical development plan or the TPP, unless absolutely necessary, will be costly and will impact the pricing, uptake, access, and forecasting work already carried out. It could also delay drug approval and launch.

Developing the TPP: Learning and improving

There are several common pitfalls for companies developing TPPs. Firstly, companies need to make sure that the TPP clearly explains the value of the product, written in a language appropriate to the audience, with each key point supported with data, or including how that data will be collected. For example, simply stating the expected clinical endpoints is not enough. The TPP needs to explain how these endpoints will be proven for the specified population, such as placebo-controlled or comparator-controlled studies; treatment-naïve or treatment-experienced patients; or adults, adolescents, children or infants. These elements are important for regulatory and payer perception.

Secondly, target product profiles need to include clear values for outcomes where possible, adjusted for each of the basic, realistic and aspirational profiles. At the earlier stages of development, these outcome values may still need to be developed, for example, ascertaining which outcomes are needed for first- or second-line treatment, or how the figures will differ across patient groups. However, at later stages of development, if the values are too vague, or the ranges provided are too wide, it can create uncertainty.

Thirdly, there is the communication of the target product profiles, both internally and externally. It’s important to understand, and then to make clear, which profile is being discussed – the basic, realistic or aspirational profile – and why that particular profile is relevant.

Finally, and perhaps most importantly, it’s vital to be certain that the profile, at whichever level, is objective and based on evidence and reasoned argument. When things are going well, everyone involved in the product’s development will want to believe that the hard work they are putting in is worthwhile, and will create the best product possible. On the flip side, if the development process has been challenging, it can feel easier to have a negative perspective. Either viewpoint can impair the objective view that is needed to create and evaluate a TPP.

Overall, target product profiles should do what it describes. It should provide a tangible focus or target for the company to understand what it is working toward, and create a road map with a clear end in sight.

To discuss TPPs or other market access strategies, call us on: +44 (0) 203 750 9833 or email us at: discover@validinsight.com.

References

1. NIH (2017) Target product profile. Available at: https://neuroscienceblueprint.nih.gov/resources/target-product-profile.htm [Last accessed 27 September 2017].