From Rescue to FDA Approval: Reanalysis and Resubmission of an Ophthalmology Trial

Introduction

This case study highlights an ophthalmology clinical trial for a product to treat wet age-related macular degeneration (AMD). “Wet” AMD is a rare type of macular degeneration, accounting for just 10% of overall cases. However, it’s an advanced form of the condition, causing rapid onset and severe vision loss. Currently, there are very limited treatment options available, making this trial extremely important.

The results of this study were to be submitted to the FDA to allow for approval of this product. Veramed was tasked with rescuing the study after failings with the original CRO resulted in a lengthy FDA letter that identified issues with the statistical methodology and analysis, preventing the FDA from giving their approval.

The Issue

This client had selected a different CRO to perform the analysis and submission of the data to the FDA for approval of so that the product could go to market. The FDA found substantial uncertainty in the primary effectiveness outcome and the data that was submitted, resulting in the FDA not approving the submission. The FDA requested a complete re-analysis of the data using sound statistical techniques.

This proved to be the tipping point for the sponsor’s relationship with the existing CRO, which was already strained. Recognizing the need to bring in experts in statistical analysis who understand the FDA process and the use of correct statistical techniques, the client reached out to Veramed for support.

Veramed had only 60 days to prepare a new statistical plan detailing a complete re-design of the primary endpoint analysis and to re-analyse the data in accordance with the new statistical plan.

The Solution

With such a tight timeline of 60 days to work to, it was imperative that Veramed could come in and hit the ground running. With extensive rescue study and statistical experience, the Veramed team was able to quickly understand the issues raised by the FDA and prepare a new statistical analysis plan leading to re-working of the statistical techniques consistent with the FDA’s requirements and expectations. This required particularly high degree of technical expertise as the previous CRO had used an outdated analysis methodology that the FDA no longer accepted.

Veramed added extra resources to the project which allowed us to meet the tight timelines and be able to provide a quality submission using appropriate statistical techniques.

The FDA reviewed the new submission and analysis and responded with minor comments and updates. Any FDA updates and requests were performed by Veramed within 48 hours of the request, resulting in a streamlined process for review.

The Outcome

As a result of Veramed’s statistical re-analysis of the data and new submission, this novel treatment for wet AMD received FDA approval. We were able to get the analysis back on track, quickly and smoothly, accelerating the time to market for an advanced treatment that could have a life-changing impact for affected patients.

Veramed is now this trial sponsor’s provider of choice for statistical analysis, and they are looking to collaborate with Veramed on future studies.

Discover how Veramed can also provide you with flexible and collaborative statistical support, from study design through to submission and beyond.