The goal of drug product development is commercial success.  If this statement wasn’t true, how would patients access the life-saving or life-enhancing drugs we are developing?  Yet, all too many companies focus just on FDA approval, which in our view should be just a very important milestone. When embarking on the drug development journey, it is essential that companies have a clear idea of how the proposed product will be an improvement or preferred alternative to drugs currently used or in the development pipeline. A Target Product Profile (TPP) can be a helpful aid to identifying the desired product attributes and communicating them to the development team, stakeholders, and regulatory authorities.  We’ve asked one of our research associates, Jillian Orans, to provide the following background on TPP.

The utility of a Target Product Profile (TPP)

During the lengthy drug development process, getting from point A to point Z as quickly and efficiently as possible can save years of time and millions of dollars.  A Target Product Profile, or TPP, is a document that is intended to facilitate the complicated path that ends at the approval and optimal labeling of a safe and efficacious marketed drug product.  Including a TPP in your information package can streamline receipt of constructive feedback from the FDA by presenting all of the relevant medical and scientific information for your drug program in the context of your drug development goals.

What is a TPP?

A Target Product Profile is a format for a summary of a drug development program that is described in terms of drug labeling concepts and goals. The TPP should be developed with the commercial goals of the product in the forefront. These goals should be balanced against the pharmacology of the drug and the practicalities of the clinical development program. The resulting document should contain a optimized realistic view of the goals of drug development. This document normally contains a synopsis of what will end up on the drug label, listed for each of three categories: the ideal product description (Optimal or “best-case”), a minimally acceptable product description (Minimal or “worst-case”), and a description that falls in between these best- and worst-case scenarios that will likely resemble the actual commercial product label after approval (Target or “likely-case”).  The Optimal version should be the goal: what the sponsor hopes to claim on the final label, which will be used to guide the design, conduct, and analyses of clinical trials to provide maximum efficiency to the overall development program.  An annotations or comments section can be added to provide information on proposed, planned or completed studies that will support the target, including protocol numbers and relevant dates.  A TTP is a dynamic document which can be updated as the drug development program progresses and knowledge of the drug increases.

The FDA and TPPs

The FDA has issued a draft guidance in 2007 focusing on the use of Target Product Profiles as a strategic development tool. While the TPP is entirely voluntary, the FDA encourages sponsors to submit TPPs as a part of their Briefing Documents during all steps of the drug development process. The TPP is intended to improve interactions between the sponsor and the FDA throughout this process by focusing discussions and increasing understanding of specific sponsor goals.

From the perspective of the FDA, TPPs are helpful to the sponsor for several reasons.

• Use of the TPP can minimize the risk of late stage drug development failure.
• TPPs increase the probability that optimal safety and efficacy data are available in a timely manner.
• TPPs can save FDA meeting times; for example, by allowing the sponsor to bypass discussion of the history of the drug development programs.
•  Interactions with the FDA can be streamlined by focusing meetings on changes to the TPP, rather than revisiting established entries.

Ultimately, the TPP should allow for improved label content as well as a decrease in the total amount of time spent on the entire drug development process.

What else can a TPP do for you?

When used properly, the Target Product Profile can be an invaluable strategic planning tool.  TPPs can assess potential pitfalls and create mitigation plans at all stages of the clinical development process.  They can aid in planning through distribution to clinical and nonclinical research organizations in order to solicit advice and modify existing study plans to be more time- and cost-efficient.  These documents also promote a team-based approach to drug development, by raising awareness of the marketing goals and the clinical programs among team members and promoting collaboration within the project.

The TPP can also be used to estimate the market potential and establish the net present value of a given product. By taking into consideration the optimal (best-case) scenario, the target (likely-case) scenario, and the minimal (worst-case scenario), a sponsor can provide develop the competitive strategies required to make a successful product; keeping in mind that a successful product is not only an approved product, but also one that is optimally profitable.

Putting together a TPP

The TPP, in broad strokes, contains the overall intent of a drug development program, organized by key sections used in drug labeling.  The FDA views the TPP as dynamic, meaning that the document should change throughout the process, including regular updates to reflect changes in clinical development programs, outcomes of clinical trials, input from the FDA, or knowledge of the drug itself.  The TPP should be shorter than a drug label and will likely be brief early on in the development process.

Some possible sections of the TPP include the following:

• Therapeutic Area
• Indications and Usage
• Population
• Dosage Form and Strengths
• Dosage and Administration
• Clinical Pharmacology
• Proposed Clinical Program
• Nonclinical toxicology Program
• Contraindications
• Drug Interactions
• Adverse Reactions
• Storage and Handling
• Warnings and Precautions
• Overdose
• Potential for abuse
• Patient Counseling Information
• References

In addition to the optimal, minimal and target label scenarios outlined above, an annotations or comments section should also be added to highlight completed or proposed studies, protocol or serial numbers, and relevant dates.

Other sections that may not be included in the FDA document but contain marketing information that the sponsor would like to consider during the drug development process include:

• Primary markets
• Pharmacoeconomics
• Cost of Goods
• Target Price
• Current Gold Standard
• Possible Product Extensions

 Conclusions

A detailed Target Product Profile, when created early in the development program and updated as new information becomes available throughout the drug development process, can be extremely helpful in mapping out the strategic marketing and scientific pathway. The TPP can not only facilitate interactions with the FDA, but also help in the strategic planning of your clinical and nonclinical programs and provide a valuable tool in the assessment of the market value of the product. It defines the goals of the drug development early in the process, focusing team efforts and streamlining program implementation. All of these advantages contribute to the ultimate goal of driving greater efficiencies and shorter timelines to the approval of an optimally marketable and profitable product.