Is it Time to Sharpen Your Target Product Profile (TPP)? We’ve heard it countless times: “Fail early and live to fight another day.” “Preserve capital, human resources, and energy for a project with a higher probability of success.” But how do you know if you are failing, if you have not described and quantified success? In the right hands, the Target Product Profile (TPP) is a powerful tool to define, refine, and increase the odds of commercial and clinical success.
Before the widespread adoption of the TPP in the 1980s and early 1990s, development plans were long, detailed technical documents, and would, for example, often dive deeply into pharmacology. They read more like textbooks and were not easily understood and digested by management. Development plans included a draft label, itself a long and complex matter-of-fact document that is often confused with the more concise, and sometimes aspirational, TPP.
In other words, development plans failed to bridge gaps between scientists, management, and others as they looked down the road. What was missing was a clear articulation of the end game. In the 1980s, the alarming spectacle of expensive late-stage failures – and the inevitable consequence of raising the cost of drug development across the board – engendered the desire for a more efficient way to develop drugs. Pondering recent wreckage, management wanted a road map, not a lengthy and technical owner’s manual, to guide the drug-development journey and avoid driving full-throttle off a cliff. Thus, the Target Product Profile was born.
So, what is the TPP? A quick search will yield straightforward answers – the TPP is a table that outlines the characteristics of the drug in key categories such as: therapeutic area, indication, patient population, efficacy, safety, dosage regimen, route of administration, formulation, and active pharmaceutical ingredients (APIs). Complete the template and check the box; you have a TPP!
But the TPP is not simply a document; it is the work product of a critical process that is in turn a reflection of the organization’s culture. The TPP can be thought of as a written contract among the members of the development team, a diverse collection of individuals hailing from discovery science, commercialization, legal, clinical and regulatory affairs, manufacturing, chemistry, and management. In drafting and updating the TPP, the entire development team takes ownership and holds themselves and each other accountable for defining and regularly measuring success. When questions arise and relationships between members grow strained, the Target Product Profile provides a framework to ask and resolve difficult questions.
When drafting and updating the TPP, the process is as important as the document. The process provides an opportunity to identify, describe, and resolve misalignments that can doom the product. For example, the organization’s pharmacologists may be excited by a molecule’s novel mechanism of action. It is up to marketing to point out that simply being new is not sufficient. What are the advantages compared to competing treatments? Novel is not good enough. Depending on when they are uncovered (lead selection vs. late-stage clinical), faulty assumptions, such as “new is better,” are either soon-forgotten or precipitate an existential crisis for the organization. Without clear, frequent, organized, and transparent communication between members of the development team with all eyes on the same road map, your organization runs the risk of a head-on collision with a burning dump truck on a one-way street.
Do you have a molecule that you believe can be developed into a drug? Or do you have a discovery platform that you wish to aim at specific unmet clinical need? Then it is time to draft your TPP. For example, if your organization is a mature drug company, you may have a valuable commercial operation in a particular therapeutic area. Your scientists have identified an approved product in the same therapeutic area with room for improvement, such as potency, dosing, or side effects. A good Target Product Profile provides medicinal chemists with guardrails to guide their work and develop a product with a market advantage based on its intrinsic characteristics.
In a pre-funded biopharma startup, the first draft of a TPP may be aspirational. Creativity and resourcefulness will be required to draft and maintain a useful TPP and produce a quality tool. You have reason to believe in your molecule, but you do not have a staff of scientists, clinicians, chemists, nor the resources to research ideas with patient groups or engage payors. One of the many challenges of a successful startup, however, is to bootstrap the access, goodwill, industry expertise, and research that is within your reach to scrutinize your product from every angle beginning on day 0. Imagination and educated guesses may be the only option at the start, until you can improve your organization’s resources and gain access to the necessary expertise. If nothing else, drafting the initial TPP may newly reveal gaping holes, blind spots, and unacceptable uncertainties. You may have arrived at a necessary pivot point sooner than expected.
The development teams that write the first draft of a Target Product Profile in a startup vs. a mature drug company may bear little resemblance to one another. In the case of the former, the organization may consist of little more than a founder who is “all of the above CXO: CEO, CSO, COO….” In contrast, the latter may be fully staffed with participants that reflect, represent, or research the perspectives of all stakeholders: patients, health care providers, regulators, payors, and, of course, the Sponsor. Whatever the team photo looks like on day one – and maybe it is a selfie - it is important to upgrade as resources expand with new team members who are charged with ratcheting up the rigor of the TPP. Every element of the TPP is subject to scrutiny.
In one example, the development team exclusively engaged internal stakeholders within the Sponsor organization such as staff chemists, regulatory specialists, clinicians, and toxicologists. Management had signed off on a product that decreased post-surgical healing time following a complex fracture from six months to three months. Fortunately, the organization engaged payors soon thereafter and avoided a futile phase 3 program: the reality was that decreasing healing time in this patient population would not translate into reduced costs, and the treatment would in fact increase costs for the payor. The drug would never make it to the formulary for reimbursement.
A TPP that is fully informed by accountable individuals decreases the risk of expensive and painful lessons. A Target Product Profile is a living document that is only as good as the team that owns and maintains it. It is the development team’s responsibility to make good use of the TPP to update their prior knowledge with new information.
A toxic organizational culture will undermine the development of the TPP and erode its utility. In order for the Target Product Profile process to succeed – which can mean abandoning a program sooner rather than later – the stakeholders within an organization must speak up and be fearless, especially when expressing uncertainty or concern. After all, drug development is probabilistic, not deterministic. Our task is to do our best to address and narrow uncertainty and enable well-informed decisions. When faced with questions such as: “Is twice daily dosing going to be accepted by patients and health care providers?” Sometimes the correct answer is “I don’t know, we should do some market research.” Does your development team feel brave and empowered, or afraid of saying the wrong thing and looking foolish? If it is the latter, then a TPP, no matter how good it is, is not a panacea for an organizational culture that is not prepared to embrace the process.
In a world driven by milestones, a TPP is a living reflection of your organization’s value, culture, and ability to execute. For example, a high-quality, regularly updated TPP is a powerful communication document when approaching and reporting to investors. Are the drug development assumptions that you made three years ago still valid? Are there new scientific data that impacts your drug development plans? Has the competitive landscape changed? Has the patient population changed? These are monumental questions, and the TPP is an indispensable tool to grapple with them. A quality TPP builds confidence, while a poor TPP created by checking the boxes amplifies uncertainty or worse, is written and then forgotten or ignored.
Are you a small organization that lacks the critical expertise to draft a high-quality TPP? Would you like the help of experts who have written, updated and managed dozens of TPPs over the years? We can help. Contact us today to learn how we can help with all of your regulatory needs, starting with the development of a Target Product Profile.
August 17, 2016
During product development some sponsors struggle to determine how their product will be regulated by the FDA. Will it be considered a drug, medical device, biologic, or combination product? Based on...
December 1, 2015
On September 25, 2015, FDA published a notice of proposed rulemaking (NPRM) regarding the regulation of products made or derived from tobacco. In the Federal Register announcement, “FDA requested...
November 27, 2018
On Tuesday, November 20th, FDA announced the establishment of a public docket to solicit comments and feedback on a proposed framework regarding the regulation of prescription drug-use-related...