From Setback to Success: How a Biotech transformed their Rare Disease Clinical Study by leveraging Biostatistics and Programming expertise

Introduction

Rare disease studies can present unique challenges compared to other types of clinical studies. Limited patient pools and enrollment considerations are important, and the study typically has far fewer patients. These challenges can affect study design, data collection, data review throughout the trial, and how data is analyzed and presented both during and at the end of the study. In addition to the standard end-of-study reporting activities, rare disease studies may require a more flexible approach to reporting that includes regular status reports, parallel integrated analyses to accumulate evidence from ongoing and legacy studies, and data monitoring activities at an individual patient level.

Veramed was contracted by a Biotech to transition , biostatistics and programming deliverables of this rare disease study. The client wanted to contract a vendor that could offer better communication,prioritization, and a team that could really take ownership – traits they felt were lacking in the relationship with their incumbent CRO.

The Issue

The client had a critical need for a vendor well-versed in the orphan drug designation process, which is unique to rare diseases. Although this process had been handled by their previous supplier, the client required the submission strategy to be revisited and reassessed within a tight timeframe.

Communication between the client and the previous vendor was not optimal, and the client required a more structured and responsive framework with which to collaborate on data processing and analysis.

Additionally, the client specifically sought a team capable of generating customized reports promptly and demonstrating adaptability in response to evolving demands.

Finally, the DSMB activities of the trial were slow to take off, and the client was looking for leadership in the coordination of meetings and an informed specification of panel outputs.

The Solution

Veramed deployed a team with a proven track record in rare disease studies, and with the experience to handle and analyze data for rare diseases. Rare disease studies require different methodologies, such as statistical methods for small samples, including non-parametric methods and bespoke graphics, in which the Veramed team had prior experience. The programming team was used to conducting deep QC processes for individual patient data, ensuring that the review process could pick up inconsistencies early.

The work was launched with a structured transition plan that identified all required data and information sources, so that all members of the team were immediately proactive in identifying gaps, evaluating status, and finding solutions. The transition was supported at all levels within both Veramed and the client organization to ensure rapid uptake. A new framework of meetings and communication norms was proposed and implemented.

As a more agile organization, Veramed’s team had the ability to rapidly adapt to client requirements and respond efficiently to questions that were raised. Veramed was also able to produce orphan drug reports within a short time frame, and to quickly develop ongoing tailored status reports that contained a high level of detail. Veramed statistical consultants supported a re-evaluation of sample size for one of the studies.

As the client’s data monitoring needs expanded to separate safety and efficacy panels, Veramed coordinated the organization of both panels and quickly developed dummy outputs to support individual patient adjudications, with the programming macros to efficiently regenerate outputs for regular evaluations.

The Outcome

The client was extremely happy to be working with a vendor who had rare disease experience but whose core values also focused on forming a collaborative, interactive relationship, where Veramed became part of their team rather than a contracted separate entity.

The study is ongoing and has moved beyond the transition stage to put in place several status report mechanisms, support to 2 independent advisory committees, a pre-final orphan drug designation report, and 2 revised and agreed Statistical Analysis Plans.

In addition, the work has been extended with a new contract for a global safety data integration, which both supports the client’s PV function and lays the groundwork for an Integrated Summary of Safety.

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