Oncology Drug Development: Webinar Learnings on the Use of Expedited Pathways and Oncology Center of Excellence Programs

When it comes to cancer treatment, there is an inherent urgency to “leave no stone unturned.” Whether as caregivers, patients, or drug development professionals, we all share a commitment to doing everything possible to bring life-saving therapies to those who need them most. In oncology drug development, this means leveraging every available regulatory tool to accelerate timelines without compromising the benefit-risk equation.

During a recent installment of the MMS Expert Insights webinar series, Dr. Uma Sharma, CEO of MMS, and Ben Kaspar, Senior Director of Regulatory Affairs and Strategic Consulting, shared actionable advice on navigating the FDA’s expedited pathways and programs offered through the Oncology Center of Excellence.

Mapping the Path to Accelerate Oncology Drug Development

To make it easier to understand during the webinar, Kaspar likened the process to navigation, requiring a map (your clinical development plan), a compass (data and its analysis), and identifiable landmarks (regulatory milestones). By aligning these elements, Sponsors can move strategically and efficiently toward approval.

Additionally, the key FDA programs and critical decision points that can make or break an expedited timeline were highlighted, as follows.

Fast Track Designation

Fast Track offers benefits like rolling review and increased communication with the FDA, but Sponsors must demonstrate the potential to address an unmet need. In oncology drug development, this often involves presenting early clinical or nonclinical data for patients with limited treatment options. Sponsors should plan carefully, ensuring that their development plan answers how they will confirm efficacy in subsequent trials.

Breakthrough Therapy Designation

With Fast Track benefits and the added involvement of FDA senior managers, Breakthrough Therapy is designed for therapies that show substantial improvement over available treatments. During the webinar, Kaspar emphasized the importance of preliminary clinical evidence, often at Phase 2, and a clear plan for confirming these early results in later stages.

Accelerated Approval

For treatments relying on surrogate or intermediate endpoints, Accelerated Approval allows earlier market entry, contingent on confirmatory post-marketing studies. Sponsors must justify the surrogate endpoint’s ability to predict clinical benefit. This is a challenge that requires rigorous planning and alignment with the latest 2024 guidance.

Priority Review

Priority Review shortens NDA/BLA timelines by four months, offering a faster path to market for treatments addressing significant unmet needs. While the FDA ultimately decides, Kaspar highlighted that a well-prepared submission, including a clear benefit-risk profile, increases the likelihood of success.

Real-Time Oncology Review (RTOR)

RTOR enables early FDA engagement, with reviews starting as soon as topline results become available. This program can cut months off the approval process but requires exceptional preparedness. The webinar underscored the importance of managing parallel Information Requests (IRs) and pre-submission activities, which can strain unprepared teams.

Project Orbis

For Sponsors aiming to achieve simultaneous oncology drug development approvals across multiple countries, Project Orbis streamlines regulatory reviews through collaboration between international health authorities. However, the complexity of coordinating multinational submissions requires careful planning, robust data management, and aligned timelines.

Four Key Takeaways for Sponsors

Success in oncology drug development requires a fine balance of understanding regulatory pathways and early alignment with FDA expectations. By focusing on proactive planning, integrating data-driven processes, and maintaining a sharp benefit-risk focus, Sponsors can better navigate expedited oncology programs.

Here are four considerations to ensure that Sponsors make the most of these opportunities, as follows:

1. Plan Early, Align Often: Engage the FDA proactively to ensure that you are leveraging expedited pathways at the right milestones. Early alignment helps identify critical regulatory touchpoints and mitigates risks that can delay progress, setting a clear path for accelerated oncology drug development.
2. Integrate Data, Process, and Strategy: A cohesive approach to regulatory planning that includes combining data organization, streamlined processes, and strategic oversight prevents delays and ensures consistency across submissions. This integration enables Sponsors to anticipate challenges and deliver stronger, more effective applications.
3. Build for Compliance: Preparing for REMS, post-marketing studies, and regulatory inspections ahead of time can mitigate risks at approval. Early planning not only ensures smoother reviews but also demonstrates a Sponsor’s commitment to long-term safety and efficacy.
4. Prioritize Benefit-Risk Assessment: Demonstrating clear, evidence-backed improvements over existing therapies remains central to success. A focused benefit-risk strategy throughout development provides the foundation for a compelling regulatory submission and positions therapies for expedited approval.

On top of having a stronger advantage with a well-integrated regulatory approach, Kaspar that proprietary tools like Datacise®, which enable rapid, cross-study data visualization and validation, provide critical support to accelerate decision-making throughout development.

Watch the video on Datacise here.

A Roadmap for Accelerating Oncology Approvals

As Sponsors face increasing pressure to deliver oncology therapies faster, understanding and optimizing these pathways is imperative. By mapping out key programs, aligning benefit-risk strategies, and leveraging cross-functional expertise, Sponsors can accelerate approvals with internal resources or through the help of tenured oncology experts at MMS without sacrificing quality.

From Fast Track and Breakthrough Therapy to Real-Time Oncology Review (RTOR) and Project Orbis, these pathways hold immense potential for those who know how and when to use them to maximize their impact.

For those who missed the live session, access the full “No Stone Unturned” webinar and watch it on-demand – Click here