Speeding life-changing medicines to patients by minimizing risk in the preclinical stage

Drug development is an inherently risky business, a truth well understood by all stakeholders, including investors. New concepts and novel therapies don’t always succeed. However – fortunately – that’s never impeded innovation, especially in this exciting world of scientific advances. And while investors may have been more careful over the last couple of years, the near outlook is considerably more positive. Still, investors and partners expect to see a clear development path to preclinical and clinical proof of concept and a sound strategy for commercial success.

The question, then, is about how biotech companies can mitigate risk before going too far in the wrong direction. Or, to put a finer point on it, what are the critical actions to take in the preclinical phase that can ultimately drive the success of a product? To explore further, I sat down with experts from across Parexel, including my colleagues Balazs Felcsuti, partner at Health Advances, and Kristina Reeder, director and global head of Patient Insights. The following is a high-level overview of the first podcast in our five-part series called De-risking Drug Development.
 

Determine the value 

The short answer to “how to mitigate risk” is to consider the value proposition from the viewpoint of all key stakeholders at the very outset. Those stakeholders encompass not just investors but regulators, payers, and, of course, the patients themselves. Regardless of the short-term objective, which might be to transfer the science to another entity after reaching a certain milestone, biotechs must think ahead to commercialization. 
The best practice is close coordination between development, regulatory, and commercial strategies to determine the right indication and target population. From the earliest stages, the focus must be on the patients who will ultimately benefit from the treatment. Many companies neglect commercial adoption while pursuing the regulatory path for approval. 

Focus on the patient 

The best management teams understand how to capture the patient voice from the start, working with the end in mind. The perspectives involved in decision-making should comprise the patient, the clinician, caregivers, and ultimately the payer, such that the product's value is determined for each stakeholder. What is the patient’s lived experience, and how will the treatment impact that experience?  What about the burden on the patient and the caregiver? What are their preferences? What do they value – not just in terms of health, but in other aspects of their lives? 

In this regard, engaging in meaningful, mutually productive partnerships with patient and caregiver advocacy groups can inform the entire drug development process. Understanding their point of view is essential for developing the right instruments for assessments and collecting endpoints that measure the perceived value. Furthermore, regulators will want to ascertain that data points are predicated on patient input and that the product meets a specific unmet need. 

On that note, the competitive landscape is another key consideration. What products are currently on the market, and how will this new treatment improve the standard of care? 

Be agile 

That said, focus is the operative principle. Often, we see in the early stages that the abundance of possibilities of a mechanism or a drug can be so compelling that biotech leaders lose sight of their initial strategy. Trying to be all things to all people can cause them to miss the best opportunities and the most likely paths to success. 

By the same token, the ability to stay nimble and pivot when necessary is equally important. The best way to strike that balance is to plot a strategy that articulates several different paths – Plan A, Plan B, Plan C – with the ability to shift quickly. Knowing when to fail, and fail fast, will avoid throwing good money after bad. In fact, investors are often looking for an experienced management team capable of recognizing and acknowledging when the time has come to change course. 

Prepare a thoughtful communications strategy 

Finally, early-stage planning should encompass medical affairs. At Parexel, we have learned through our work, particularly in novel cell and gene therapy products, that a medical affairs partner can be fundamental to success. Medical affairs experts help guide patient-centricity, evidence generation, digital strategy, medical communications, and more. They can work with R&D teams, even before first-in-human trials, to ensure a clear understanding of the need to shape a strong evidence-based narrative for the product as well as the disease area. 

A communications strategy must be in place to ensure that the narrative is tailored in a way that will resonate with each stakeholder group – and to clearly identify each of those constituencies. They include patient and caregiver advocacy groups, potential future investigators on clinical trials, thought leaders who might become advocates as the product advances through development, and even regulators, in some cases. A key objective is to disseminate foundational information to establish the value story through peer-reviewed publications and other channels. Whether investor-focused assets, press releases, congress presentations, or patient-focused literature, the messages must be compliant, consistent, credible, and supported by scientific evidence. 

In short, through informed decision-making, we can minimize risk and maximize the probability of commercial success at critical points in the preclinical phase. Please listen to our series of five podcasts, De-Risking Drug Development, for more insights about how to take this approach throughout the development lifecycle.

About Parexel Consulting 

Parexel Consulting helps you facilitate business-critical decision-making at every stage to maximize the probability of commercial success. Our expertise encompasses medical affairs, growth strategies, strategic compliance, regulatory strategy and operations, market access, and medical communications. Our approach considers and addresses the needs of all stakeholders, including patients, physicians, regulators, payers, and other healthcare providers. We do this by designing, operating, and implementing strategies informed by expert knowledge, experience, and clinical trial expertise. We collaborate with you to help speed life-changing medicines to patients who need them. 

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