March 29, 2022
Reece Armstrong, Editor
Hettie Stroebel, Founder and CEO of Launch Excellence looks at how target product profiles can be used by life sciences companies as a strategy for more effective product development.
The global effort to develop and distribute a SARS-CoV-2 vaccine in months is a triumph for the pharmaceutical industry. It is also unprecedented. Never in the history of drug development has a proposed vaccine gone from concept to regulatory approval in a matter of months.
A key tool in that effort was a Target Product Profile (TPP) for Covid vaccines. Shortly after the discovery of the virus, the World Health Organisation (WHO), in consultation with various stakeholders such as the manufacturing and scientific communities, developed a TPP for Covid vaccines. It laid out the roadmap for developing the vaccine and the acceptable criteria to be met to protect patients from the devastating effects.
As the name suggests, “Target Product Profile,” also known as the TPP, refers to the targeted or intended profile a pharmaceutical/biotechnology product or technology. The profile provides the product’s desired characteristics or features defining the value proposition, and key differentiators aimed at providing a competitive advantage to the product. The TPP is a dynamic multidisciplinary strategic development process tool embodying the notion of beginning with the goal in mind.
The TPP should be used throughout the drug development process from pre-IND phase to post marketing phase.
Target product profiles should not only be developed for drugs and vaccines, but also for diagnostic tests, medical devices, and other innovations.
The target product profile establishes a shared vision and strategy intra-company
Alignment of all stakeholders and decision markers around one common vision for the desired product based on unmet needs of the market is pivotal to bring a new product to market in the shortest and most cost-effective way. An integrated, cross-functional developed and approved TPP facilitates strategic product go/no-go decision making along the development pathway at the different stage gates, minimises risk, and improves contingency- and scenario planning. If a company loses sight of the original desired target, there is greater risk that decisions about the product are made on the spur of the moment.
The target product profile as business tool for determining asset value and capital commitment
A target product profile helps to prioritise the indication(s) and associated patient population -providing a long-term view of an asset’s lifecycle. Developing one or multiple indications have different impacts on time, cost, risk and ultimately return on investment for a drug. TPP answers questions such as should we proceed with the fastest route to PoC while keeping risk reasonably low and maintaining a high value.
The target product profile represents a critical path tool with regulatory agencies
A TPP can be prepared by a sponsor as a part of proprietary IND file and then shared with the FDA to facilitate communication regarding a particular drug development programme. Submission of TPP is therefore voluntary.
A TPP enables a sponsor to pursue the desired outcome (i.e., approval and optimal labelling of a safe and effective drug) in the most efficient manner with respect to FDA interaction because all such interaction is focused on the explicitly stated goals of the development programme.
An efficient dialogue between a sponsor and the FDA during the drug development process can minimise the risk of late-stage drug development failures, increase the probability that optimal safety and efficacy data are available in a timely manner, improve labelling content, and possibly decrease the total time involved with drug development.
FDA demonstrated that experience with TPP-focused meetings with sponsors at the FDA can be useful and beneficial.
Breder, and co-authors, demonstrated in an analysis comparing first-cycle actions by the FDA, that applications with a TPP had a significantly lower refuse to file decision than those without a TPP1. In addition to this, the authors showed that the time between NDA/BLA submission and approval was one month less in the TPP application group than non-TPP. While the value of a 30-day difference may not be readily apparent, to a patient with a serious disease this difference can be critical. From a business perspective, the average monthly sales revenue at the end of patent will constitute a significant difference.
The target product profile as an early drug discovery and development compass
Drug discovery and development is a fascinating, challenging, and multidisciplinary process where ideas for therapeutic intervention are devised, evaluated, and translated into medicines that will ultimately benefit patients. There are multiple points where decisions must be made which almost always impact time, cost, risk and ultimately return on investment.
The target product profile affects all research activities from lead optimisation to final product. Some salient TPP properties such as toxicological risks, predicted human dosing, and pharmaceutical properties can only be effectively, and practically, assessed for the first time in a project timeline during early drug development. TPP definition and compliance have therefore far-reaching effects across the drug discovery–drug development value chain: they dictate which compounds are made in the first place, which compounds will be selected for clinical development, and ultimately which compounds will be successful at the end of the development cycle.
The target product profile describes the critical quality attributes needed for manufacturing
Target Product Quality Profile (TPQP) is a term that is a natural extension of the TPP for product quality. It is the quality characteristics that the drug product should possess to reproducibly deliver the therapeutic benefit promised in the label. The TPQP guides formulation scientists to establish formulation strategies and keep the formulation effort focused and efficient. The desirable TPQP, ensures a product development scientist give serious consideration to the biopharmaceutical properties of the drug substance. These biopharmaceutical properties include physical, chemical, and biological properties. These properties directly impact the product quality and performance.
The target product profile is the foundation for commercial value
The business/commercial team TPPs are usually abbreviated versions of the regulatory TPP but also contain additional information necessary for the assessment of a product’s commercial potential and the net present value (NPV) of the product.
The target product profile as an early economic evaluation for reimbursement
Economic evaluation compares the costs and benefits/outcomes of different healthcare strategies to support technology adoption and reimbursement decisions. Whilst economic evaluation is usually conducted at the later stages of the evaluation pathway, early economic evaluation is increasingly performed at the proof-of-concept or pre-regulatory-approval stage as a means of directing stop/go decisions and informing the optimal trajectory of future research. The first step in economic modelling is to map the care pathway in which the new technology will sit. This typically involves reviewing clinical guidelines, literature and consulting clinicians, payers, patients, and caregiver on their individual experiences of the care pathway. This is crucial in the context of TPP development, because selecting a different test role and/or purpose can result in significantly different development requirements.
In conclusion, a target product profile, and its other extensions, is an investment that more life science companies need to incorporate in their plans to gain investment, focus and shorten development, gain earlier approval, and is ultimately a compelling value proposition for patients, providers, and payers.