Start-ups and emerging biotech companies often start out with lofty goals about what their products can do. That’s very noble but it’s also an easy way to run out of money halfway through the development process.
While it’s good to have long-term goals, success depends on developing a well-defined target product profile (TPP). Start with a clear understanding of what your product can do and what your label would, ideally, say at the end of the process, considering the key attributes and taking advise from all functions to align the goals. By starting with the end in mind, your programme will be much more focused and effective. With a clearly documented TPP, you can more efficiently design your product development and prepare for the regulatory interactions that will be needed.
For example, too often oncology start-ups hope their therapies will cure all — or at least many — different kinds of cancer. They begin with preliminary trials and talk to the regulators about multiple cancer indications, then start work on them all at once. While perhaps that product does have huge potential, at the end of the day you need to get it to market and make money in order to conduct the extensive research and gather the data needed to demonstrate the safety and efficacy across all of those indications. That means you need to focus on one subset of cancers or take an even more specific and focused route through the orphan drug designation to get to market. You may even be able to cover multiple indications using adaptive advanced trial designs. Once you’ve proven your mechanisms of action, have carried out preliminary efficacy and safety studies in the clinic, and are well on the way to building your first marketing authorisation dossier and bringing in revenue, that’s the time to scale up investment in the rest of the pipeline.
It all comes down to starting with the end in mind and maintaining a single, clear and powerful focus.