Introduction/Background

An international company needed a partner with regulatory and strategic development expertise to realize the full potential of its product: a catheter tailored to meet the specific needs of a drug designed to cure an organ cancer. The client’s catheter device worked by mixing an off-the-shelf drug powder with a combination of polymers to yield a solution that gelled at body temperature.

With FDA approval, this potentially curative treatment could substantially increase the quality of life for patients and reduce overall treatment costs compared to the current standard of care, which involved surgery and subsequent maintenance therapies.

Challenge

At its initial meeting with the Premier Consulting team, the company outlined its current strategy: Focus on device approval for the catheter, targeting surgeons who treat patients with this type of organ cancer.

Solution

Premier Consulting found that the client had not considered two key factors:

• If a device and drug are needed together under a specific set of instructions, they can be packaged and labeled together as a combination product. Until recently, rules surrounding combination products were at odds in the United States and European Union, resulting in confusion and misinformation on the topic.
• Given the potentially curative power of the company’s therapy, the treatment decision was much more likely to be made by the patient’s specialist, not the surgeon.

With considerable experience in combination products and commercial market identification, Premier Consulting worked with the client to:

• Correctly recognize the product category and convince the FDA to agree to a combination product. Furthermore, given the drastic improvement in efficacy, Premier Consulting made the case to the FDA for orphan drug status, with fast-track review.
• Clearly identify the specialty medical market: specialists, not surgeons.

Takeaway

The FDA granted orphan drug status with fast-track review. Moreover, the new strategy changed the way the client approached its product development and marketing efforts. With better-supported return on investment, the client went public, raising money to accelerate the development of this and related products. It also rebranded with a new product name to reflect the company’s focus on therapeutic impact, not technology.

Outcome

FDA granted orphan drug status with fast-track review, and the client identified its true target market.