REGULATORY
CASE STUDY


Benefit-Risk Profile, Data Presentation Strategy, & Briefing Document for Successful FDA Ad Comm.

STRATEGIC OVERVIEW

Vault Bioventures was engaged by a small (pre-commercial) biotech company to provide key regulatory support for a new combination drug/device product that had a long, complicated, and challenging development history. The company had received two Complete Response Letters from the FDA in answer to an earlier NDA filing for the product that necessitated new clinical trial data generation. In addition, the product faced potential questions regarding its long-term safety profile. As part of their current resubmission strategy, the company asked Vault Bioventures to put together a perspective on the product benefit-risk profile that would serve as a core element of the resubmission package.

After Reviewing the product characteristics and full clinical data, Vault was able to author a balanced summary that highlighted a favorable benefit-risk-profile for patients based on understanding the therapeutic environment and medical needs for this disease category. Having reviewed the full clinical trial data, both old (legacy) trial results, as well as newly-generated clinical data, Vault Bioventures proposed a comprehensive strategy to best highlight efficacy and safety across the many trials conducted over the years.

The company had been advised that the product would go before an FDA Advisory Committee, but given limited company resources, no work had been done to define the strategy for this important public pre-approval meeting until the NDA documents had been submitted. Leaving very little time for meeting preparation, Vault Bioventures responded to the company’s urgent request for assistance by authoring a comprehensive Briefing Document in the span of 2 months, which served as a roadmap for meeting presentation flow, the definitive summary of the product, its complex development, and its benefit-risk profile to Steering Committee members and others (e.g., analysts, potential partner pharmas). The Vault Bioventures team supported our client in all AdCom meeting preparation activities, including mock advisory committee meetings and during the meeting itself.


OUTCOME

  • Our client had a very successful FDA Advisory Committee meeting which resulted in an overwhelming recommendation by the committee members to approve the product.
  • Following the FDA advisory committee meeting, our client received FDA approval and successfully completed a licensing deal.

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