On the 17th, the webinar jointly hosted by Medirama and GCCL (GC Cell) drew great success, with more than 550 people pre-registered. At the event, Hanlim Moon, CEO of Medirama, delivered a lecture titled “FDA’s Oncology Expedited Programs in Action: A Case-Based Approach to Strategic Drug Development.”

The Bio introduces the details of his lecture through two contributed articles.

Understanding the FDA’s Programs for Oncology Drug Development

Cancer drug development is one of the most complex and urgent areas of medicine. Bringing a new drug to patients requires going through multiple clinical phases and regulatory procedures, with a high risk of failure throughout the process. In fact, over the past decade, the ultimate approval success rate for oncology drugs has remained lower than in other therapeutic areas. Many candidates enter clinical trials, but most never reach patients, leaving families frustrated and developers facing massive losses.

Against this backdrop, the FDA’s expedited review programs were established to accelerate access to new therapies. These four pathways—Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval—each play a different role in speeding up drug development and providing patients with earlier access to innovative treatments.

Fast Track enables closer communication with the FDA from the early stages of development, reducing uncertainty.

Breakthrough Therapy provides intensive, cross-disciplinary support for drugs showing remarkable promise in early trials.

Priority Review shortens the review timeline for submitted applications, hastening commercialization.

Accelerated Approval allows early approval based on surrogate endpoints, aiming to deliver transformative therapies to patients as quickly as possible.

These mechanisms are not simply about “faster approvals.” The FDA’s Oncology Center of Excellence (OCE) emphasizes broader considerations such as trial design, dose optimization, and ensuring racial diversity.

For example:

Project Confirm monitors cases where accelerated approvals lack confirmatory evidence, preserving the program’s credibility.

Project Optimus focuses on tailoring dose optimization to targeted therapies, rather than relying on traditional chemotherapy standards.

Project Equity mandates multinational trials and racial diversity to ensure the U.S. patient population is properly represented.

Ultimately, the essence of expedited programs is not just speed but a balance between scientific rigor and patient access. Strategic use of these pathways requires holistic planning, from early development through confirmatory trials and patient diversity. For developers, this is both a challenge and an opportunity; for patients, it represents hope for better treatment.

Lessons from Case Studies

1. Tagrisso (osimertinib): A textbook success story of expedited approval

For patients with non-small cell lung cancer (NSCLC) carrying the EGFR T790M mutation, resistance to existing therapies left virtually no treatment options. To overcome this challenge, AstraZeneca developed osimertinib (brand name Tagrisso), a third-generation EGFR inhibitor.

Clinical development progressed at extraordinary speed. Within just eight months of first patient dosing in 2013, it gained Fast Track designation. A year later, it secured Breakthrough Therapy status, and within 2 years and 8 months, both Accelerated Approval and Priority Review were granted. What normally takes over a decade was achieved in record time.

Tagrisso thus became a rare example of a drug that benefitted from all four FDA expedited programs. In the phase 3 FLAURA trial, it also demonstrated significant overall survival benefits as a first-line therapy, ultimately establishing itself as the new global standard of care for lung cancer. This case underscores the effectiveness of using expedited programs early while subsequently building robust confirmatory evidence.

2. Lumakras (sotorasib): Rapid entry but an incomplete outcome

KRAS G12C mutations were long considered “undruggable.” Amgen broke through this barrier with sotorasib (brand name Lumakras), earning accelerated approval from the FDA in 2021. For patients, it represented the first new treatment option in decades.

However, problems soon followed. In the confirmatory CodeBreaK 200 trial, survival benefits were unclear, and serious concerns about data quality emerged. The FDA noted not only failure to meet endpoints but also signs of systemic bias undermining the credibility of the results.

Specific issues included:

Asymmetric dropouts between treatment arms,

Subjective investigator-based imaging assessments favoring sotorasib,

Early crossover distorting independent imaging review (BICR) outcomes.

Even dose optimization data was heavily criticized by oncologists, despite the company’s claims.

This case highlights the double-edged nature of accelerated approval: while early access is possible, failure to provide robust confirmatory evidence risks withdrawal of approval and market exit. Lumakras still has an opportunity as a first-line therapy in an ongoing phase 3 trial, but its future remains uncertain.

3. SN BioScience’s SNB-101: A Korean biotech’s global challenge

Korean biotech firm SN BioScience gained international attention when its investigational drug SNB-101 received Fast Track designation from the FDA. Despite being in the early stages, the FDA recognized its innovative mechanism, preclinical/early clinical results, and potential to address unmet medical needs.

Fast Track is more than just accelerated review—it provides frequent meetings with FDA reviewers, early alignment on development directions, and even rolling reviews. In essence, a small Korean biotech gained the type of collaborative channels usually reserved for multinational pharma giants investing billions of dollars.

Source: The Bio https://www.thebionews.net