Five questions Biotech executives should be asking of a statistician

Five questions Biotech executives should be asking of a statistician

Five questions Biotech executives should be asking of a statistician 558 344 Exploristics

By Kimberley Hacquoil, Exploristics CDSO

Biotechnology companies have recently been plagued with waves of layoffs, longer roads to IPO and increasing difficulty in finding investors to support the new and innovative drug discovery and development plans. Biotech companies who can secure funding do so with cautious optimism, as the funding will often only provide enough to get them to the next milestone. The twists and turns of drug development mean that future funding rounds are still lurking around the corner for developers. With many challenges, much uncertainty and numerous landmarks remaining along a route, what questions should Biotech executives be asking to set them apart from the competitors? And how do statisticians play a vital role in answering these.

1. What different clinical development strategies are viable?

Before statisticians even start to look at sample sizes, Biotechs should be asking far more strategic questions about a development program. One of which is taking a more holistic view and asking the question “are we aiming for the right destination?” Statisticians can support decision-makers by providing quantitative information regarding indication sequencing, defining the target product profile and competitor analysis. They can also provide advice related to transversing the complexities of the regulatory landscape especially in novel or unprecedented areas. Often Biotechs only get “one shot” and so it’s imperative that this is used wisely. Engaging with statisticians early ensures developers are asking the right questions of potential medicines from the start, improving their chances of success.

2. What are the main risks in the clinical development of our asset and how can we mitigate these?

Drug development is hard and there is no “easy lane” to cruise in. Being proactive and accepting the potential pitfalls that could jeopardise a clinical development program or expose a company to misfortune is an important step in the journey. Identification of key risks is not enough in this VUCA world, and to successfully mitigate risks its critical to have data and insights regarding the likelihood and impact of such events. Statisticians can evaluate different data sources, use expert opinion, and explore different scenarios to provide information to rank and decide between alternative options and mitigation strategies. For example, recruitment delays or difficulties often curse clinical trials, but prospective statistical modelling of different situations and subsequent consequences can allow for mitigation strategies to be put into place, thus increasing the likelihood of smooth recruitment. Therefore, Biotechs can avoid tunnel vision and be able to adapt accordingly with changing circumstances or environment.

3. Are we leveraging historical and real-world data in our clinical development plan?

We live in a data rich world which can sometimes be overwhelming to steer through effectively. Statisticians and data scientists have tools which explore, curate, and utilise vast amounts of historical and real-world data. Combining this with simulation, Statisticians can estimate clinical trial outcomes prior to starting any trials for real. This vital step in the planning process empowers Biotechs to explore “what-if” scenarios and factor in learnings to the development plan. It facilitates efficient decision-making by leveraging insights from prior knowledge and having a test run in a virtual environment before spending any actual resource or time on clinical trials.

4. How can we effectively de-risk our clinical programs to guarantee effective use of resources?

Biotechs are troubled more than most with constrained resources and difficult decisions regarding prioritisation. This presents a unique opportunity to think innovatively about the sequencing of studies and decision criteria within and between clinical trials. Statisticians are skilled at exploring different approaches including adaptive designs to learn from data in ongoing trials and adapt accordingly to optimise the allocation of patients, speed up decision timelines and increase the probability of success. Adaptive designs allow for Biotechs to spend a (relatively) small amount of money to gain knowledge and predict the overall likelihood of success should a trial continue. These predictions allow decision-makers to make far more informed choices on how to spend remaining money. De-risking clinical programs in this way is very appealing to investors as it shows prudent planning and resource utilisation as well as good stewardship of capital. During your journey, Statisticians can help you get investor ready by defining the best places to stop and assess, and then plan the right path forward.

5. How can we gain the most robust evidence to ensure future collaborations and partnerships?

As mentioned already, the funding pot is not limitless and Biotechs will need to show how the value of their asset has grown with each new milestone. The evidence and data collected for each decision point will need to be resilient to scrutiny like with any drug development process, but it will also need to withstand shifting surroundings depending on the external ecosystem. For example, if a Biotech wants to partner with a Pharma company for full development, then they will need to satisfy certain hurdles which may vary across companies. Statisticians can help Biotechs to design clinical trials and gather external data which fulfil multiple perspectives and objectives which can set the Biotech up for successful future partnerships. Alternatively, if further investment is the aim, then Statisticians are best placed to quantify any outstanding risks and the benefits of a development program, putting a Biotech on the right path for positive funding outcomes.

The course to success may not be straightforward, it may not be easy, and it may not be as expected when first set out. However, a statistician can help executives and leaders in Biotechs every step of the way to make the hard decisions. Used more strategically, statisticians can be valuable  guides and critical co-pilots on a Biotech’s development journey, ensuring it doesn’t  go off-track or end up with a sub-optimal itinerary. By asking statisticians these five key questions before setting off along the path towards the next milestone, Biotechs and their investors can have greater confidence that the right route has been selected to reach it.

 

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