Start with the end in mind. That is one of the most important considerations for companies seeking to conduct clinical trials in the global market.
A common mistake made by smaller biotech companies — and even older companies that have not done a lot of R&D in recent years — is to think only of the interim goal. For example, the goal of a small biotech might be to get phase 1 results and license out the product, but if they don’t consider the broader objective, which is to get a product on the market, it’s possible the study design won’t be robust enough to help achieve all endpoints.
These problems can be avoided by developing a solid Target Product Profile (TPP) that clearly lists what the product is, what it does, what you want the label to say and what you need to do to support those claims. The TPP defines the desired and minimally acceptable limits for critical features of the final product, including:
- Indication and patient population/s
- Dose and administration
- Forms, strengths and presentation
- Safety profile
- Drug interactions and co-administration
- Warnings and precautions
- Adverse Reactions, Drug Abuse and Dependence, Overdose
- Shelf-life and storage
Include all stakeholders
Multi-functional collaboration is crucial to a robust TPP since consideration must be given to potential conflicts of interest. For example, commercial may have certain goals for the label but clinicians might disagree or manufacturing might note that a proposed formulation requires further studies.
Ensuring all stakeholders are consulted when developing the TPP will help to determine protocol outlines, primary endpoints for the study, and where potential data gaps lie. For example, are there robust enough study statistics to support the endpoints? Is a comparator study needed? What are competitors doing?
If all stakeholders are not brought together to develop agreed-upon goals, and compromises, companies can lose a lot of time to differing or misunderstood priorities between departments. The objective should be to keep the whole team focused on the overarching goal, which is to get the product to patients in need, and that brings the important issue of patient-centricity to the fore. When that is the main objective in developing the TPP, it makes it easier to get agreement from different functions. For example, finance understands why R&D is asking for extra funding for the non-clinical budget, and clinical understands why marketing wants to conduct more research into the patient market, or why more needs to be done to bring in the patient voice.
The TPP ties all these criteria together so everyone knows the target and where the goal posts are. Those goal posts can, and will, be moved as development progress and new data emerges, but those changes need to be agreed to and there needs to be ownership of the document signed off by people in multiple departments.
For small companies working with many outsourcing partners, the TPP is even more important since it is the benchmark for communicating with everyone involved. It helps you and your partners know what you are aiming for and, importantly, it helps you to avoid ‘own goals’, such as missing important pre-clinical work or not looking at the right endpoint or dose ranges. Having a clear target laid out in the TPP helps structure strategies that take you in the right direction, helps project managers follow a well-delineated path, keeps company budgets from blowing out, and ensures your application won’t be rejected simply because your strategy wasn’t thought through.