How Project Optimus has impacted oncology-focussed biotech companies

After a slow adoption for many biotechs, Project Optimus is beginning to achieve its aims. Speaking on the BioCentury: This week podcast, ICON’s Dr Liz O’Brien, Senior Director, Drug Development Solutions assessed how biotechs have adapted to the FDA’s requirements on oncology dose-optimisation. She looked at how it fits in with regulatory requirements in other parts of the world and offers advice to biotechs to ensure they are set for success.

What is Project Optimus?

Launched in 2021, Project Optimus aims to ensure more accurate and appropriate dosing of oncology treatments. In the past there was a tendency towards leading with the highest tolerable dose and refining subsequent doses. Project Optimus makes oncology treatments more patient-centric, with developers tasked with finding the most appropriate dose before the drug enters the market.

Outside of oncology, dose-optimisation starting in drug development is the norm. The burden is on developers to define dosing in the early development of their treatment. Oncology developers, because of the huge unmet need for their treatments, were able to bring their products to market potentially sacrificing dose refinement for speed of access to innovation. For small oncology-focussed biotechs, this sea-change presents some initial challenges. There is limited precedent for this approach and, particularly for smaller companies, they may lack the necessary experience and bandwidth. Companies with prior commercialization experience and a global multiple therapy area footprint may innately have greater ability to adapt and embrace Optimus dose-optimisation requirements However, up to now biotech companies have not been doing the kind of post approval dose refinement clinical research that FDA Oncology Centre of Excellence Optimus aims to address, in early oncology drug development.

How are global regulatory agencies aligning with Optimus?

While Project Optimus is FDA Oncology Center of Excellence (OCE)-led, oncology drug development clinical trials by their nature are global and multi-regional.

“The beauty of oncology is that the patient pathway, the care pathway, treatment guidelines and clinical practice guidelines are very uniform globally,” O’Brien says.

Although agencies in other parts of the world don’t have the exact same framework as FDA OCE they are broadly aligned and receptive to dose-optimisation principles. For example, the European Medicines Agency (EMA) published whitepapers on the subject. At the same time, the EU Health Technology Assessment Regulation (JCA – EU Joint Clinical Assessment ) has been implemented which is also focused on demonstrating comparative clinical effectiveness. Key stakeholders such as the European Organisation for the Research and Treatment of Cancer (EORTC) endorsed the drive towards dose-optimisation, even before Project Optimus was formally published.

Next steps for biotechs

Biotechs with limited or no global commercialization experience or constrained inhouse expertise or resource do not have to go it alone. ICON Biotech helps to round out smaller biotech companies’ teams to provide strategic support. Biotechs may have insufficient bandwidth to cope with the requirements of post-marketing studies, medical affairs and investigator-initiated studies. Another challenge for biotechs is refining trial design for phase 3 studies. Beginning a phase 3 study without consulting relevant bodies is “navigating in the dark,” according to O’Brien. ICON Biotech provides strategic advice to ensure that companies maximise their consultations with regulatory agencies, resulting in better informed phase 3 study designs incorporating dose optimization from early development steps.

Conclusion

For smaller biotech companies, FDA’s Project Optimus represents more than a regulatory hurdle – it is a clinically meaningful initiative designed to ensure that innovative oncology drugs are dosed to maximise benefit while minimizing harm. By shifting focus from the highest tolerated dose to the most effective and patient-friendly dose, Optimus helps promising therapies to reach their full potential in the clinic and reduces the risk of drugs being withdrawn due to preventable toxicities. For biotechs, early adoption of these principles not only supports patients’ outcomes but also strengthens the long-term viability of their pipeline.

These challenges can be overcome with careful planning, consultations with regulatory agencies and external support from an experienced CRO.

Listen to the podcast episode

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What is Project Optimus?

How are global regulatory agencies aligning with Optimus?

“The beauty of oncology is that the patient pathway, the care pathway, treatment guidelines and clinical practice guidelines are very uniform globally,” O’Brien says.

Next steps for biotechs

Conclusion

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