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Aligning for 505(b)(2) Success: Oncology Combination Product First-in-class Treatment Success

Throughout the life-cycle of a drug development program, sponsors are confronted with a range of key questions. They must decide on the correct…

  • Regulatory pathway,
  • FDA center,
  • Key Opinion Leaders (KOLs),
  • Target Product Profile (TPP) inclusions,
  • Data to rely on,
  • Studies to conduct,
  • Indication and patient population,
  • Commercial strategy, and much more.

When sponsors consider these as individual questions as they arise instead of taking a holistic view of a program from the beginning, they run the risk of missing out on time and cost savings during development and on revenue potential after launch. Beginning with the end in mind means having the right regulatory and commercial plan in place early on based on sound science and medical, regulatory, and commercial assessments.

The Need

Take, for instance, an emerging biopharmaceutical company dedicated to developing novel therapies to address specialty cancers and urologic diseases. The sponsor approached Premier Consulting early in an oncology combination product program, seeking to accelerate its development of the much-needed treatment for a rare and difficult-to-treat urologic cancer, and maintained the relationship through NDA approval.

When first approaching Premier Consulting, the sponsor noted that there were suboptimal medical solutions for the urologic cancer, mainly surgery, and a clear unmet need for an effective non-surgical product delivered to the site of action. The sponsor’s question for Premier Consulting was simple: “What do we need to do to accelerate this program?” Premier Consulting’s answer was to begin with the end in mind.

The Solutions

Using a patient-centered drug development approach, Premier Consulting provided the critical strategic regulatory and commercial insights and supported the product development; regulatory strategy (including key regulatory FDA meetings); nonclinical work; chemistry, manufacturing and controls (CMC) work; and electronic submissions. Premier Consulting also contributed its expertise in the 505(b)(2) pathway as well as orphan drug, fast-track, breakthrough, and priority review designations to advance and accelerate the product to approval.

Premier Consulting’s first task was to review the sponsor’s data and map out, from a holistic viewpoint, key gap areas inhibiting the immediate goal of accelerating the product development. Those key areas were in regulatory and commercial.


An important key to eventual success was to establish the “lead center” that would have primary jurisdiction for the drug-device combination product. While the sponsor initially planned to approach the Center for Radiological Health (CDRH), it soon became apparent that the combination product should be submitted to the Center for Drug Evaluation and Research (CDER). Likewise, the sponsor had planned to file a 510(k) application, but Premier Consulting determined that the 505(b)(2) regulatory pathway was the best fit. These key insights saved the sponsor time and cost by focusing team efforts on collecting the right data and streamlining the program implementation for the right FDA review team.

The sponsor also needed to engage in an early interaction with the FDA via the pre-Investigational New Drug meeting. Through this interaction, the sponsor was able to confirm regulatory alignment on the proposed development plan, the indication, and the patient population. Premier Consulting was also able to present real-world evidence collected under a Compassionate Use program to petition successfully for accelerated approval, and the product was granted orphan drug, fast-track, and priority review designations.


As discussed in a previous blog post, the TPP can be used as an invaluable strategic planning tool. Premier Consulting provided support by planning the key sections of the TPP to align sponsor goals, patient needs, and product labeling.

Strategic commercial insights to the program brought the sponsor’s previously accepted market size into question, as prior estimates of market size and cost were based on data from surgeries rather than in-office procedures conducted by urologists. While the sponsor’s original KOLs were surgeons, urologist KOLs were sought after Premier Consulting’s assessment.

The Result

Beginning with the end in mind drove greater time and savings efficiencies and shorter timelines to approval, and it delivered an optimally marketable and value-driven product to the patient. Premier Consulting’s early regulatory and commercial assessments ultimately led to a first-in-class NDA product approval for the sponsor in 2020.

Wherever you are in your product development cycle, Premier Consulting can help you get your “next inch” right. Contact us today to find out how we can help you accelerate your drug development program toward approval and through commercialization.


Ruth Stevens, PhD, MBA
Strategic Advisor



Ken Phelps
Strategic Advisor

Stacey Ayres, PhD
VP, Regulatory Strategy