Executive Summary
In assembling a team for a new trial studying a rare form of cancer, acute myeloid leukemia (AML), a prestigious cancer research center was looking for a CRO partner with unique qualities. Large or boutique, the partner had to first have highly-skilled staff with extensive experience in oncology studies. The partner selected would also have to provide hands-on involvement from its leaders throughout the complex trial, and an uncommon level of responsiveness. As the search unfolded, PROMETRIKA stood out amongst its competitors as the right choice for a partner for this sponsor.
A Transparent Approach to Finding a Partner
In embarking on the selection of a CRO partner for their AML program, this sponsor articulated a clear and transparent approach: Rather than focusing on the size of the CRO, they were more interested in finding a flexible and responsive organization that could best address the institutional challenges and anticipate their needs.
To that end, they asked a number of general questions, such as:
- Who will give us undivided, high-touch attention?
- Who has the expertise and experience to provide feedback on the study design to avoid later issues?
- Are they willing and able to deep-dive into the study design complexities?
- Are they willing and able to assign highly-qualified resources to provide the level of expertise needed during the study from start-up through writing of the Clinical Study Report?
In short… Who is the partner who will help us conquer cancer?
The team also had more specific questions, including:
- Who will execute the Phase 1 trial efficiently and effectively?
- Who will bring it to the next inflection point (positive or negative)?
- Who will be able to present a suitable dataset for an external organization or partner to evaluate whether the compound is a suitable asset for future development?
In the end, the sponsor found the partner that could meet its specific needs.
Sponsor chooses PROMETRIKA as Partner
The sponsor selected PROMETRIKA because of its full support services, its industry experience and academic background of its staff, and its deep experience and expertise in oncology trials. Other attributes that factored into the decision included:
- A clear focus on a shared goal: to make a difference for patients with cancer
- A willingness for seamless integration and partnership with the sponsor team
- A seasoned team throughout the full execution of the study who knows how to minimize the risks of the trial, including out of scope budgets and delayed timelines
- A collaborative cross-functional approach, to ensure the timely delivery of high quality data.
With an experienced and cohesive team in place, PROMETRIKA is currently working collaboratively with the sponsor team to navigate through the inevitable challenges that occur during the conduct of most studies, let alone complex oncology studies. Utilizing industry-leading database technology of its partner, Medidata, PROMETRIKA is responsible for implementing customized solutions for the trial’s many complexities and leveraging its long history of working together to deliver quality data.
There’s Power in a Strong Partnership
What this sponsor and PROMETRIKA have affirmed is the impactful synergy that is produced when two organizations come together to undertake one mission. Powered by a shared appreciation of patients’ perspective and a focus on the end goal, this partnership is destined to deliver outstanding results.
The first steps in this trial were to identify the anticipated challenges and develop proactive solutions to minimize their impact. These fall into five key areas:
- Study start-up
- Study execution and conduct
- Management
- Safety monitoring
- Budget
Study start-up
In launching this trial, the sponsor team faced a long list of challenges, not the least of which was the large group of stakeholders involved. Staff hours are limited, as researchers have time commitments for treating non-study patients. Drug accessibility was another issue, as the drug is not manufactured in the United States and importation created a high potential for delays.
The PROMETRIKA team was able to help them address their many challenges by first building a strong rapport with all stakeholders through face-to-face and online meetings and then developing an accurate and achievable timeline with a back-up mitigation plan to avoid delays. A seasoned clinical supply expert worked with the team to expedite the importing process and distribution of the study drug so that deadlines could be met.
Study execution and conduct
The complex nature of the protocol design with dose escalation also presents challenges. They include the frequency and the imperative for timeliness of Safety Review Committee meetings and the reliance on the local laboratory to provide on-time data. There are also recruitment challenges for relapsing or refractory patients with AML.
To manage these issues, PROMETRIKA conducted an in-depth protocol review, and identified critical influences that would affect the timeline. To become familiar with the patient community and ensure that enrollment will track toward success, the team conducted a thorough feasibility study. The team also reached out to members of the sponsor’s oncology practice to facilitate recruitment success.
Management
One of the management challenges of the study is that it is conducted by two organizations geographically distanced from each other. To address that challenge, regional monitors who had previously worked with the sponsor were recruited to bridge the distance gap. As the study progresses, investment of PROMETRIKA’s time and that of the sponsor is being continually assessed with an eye toward the scalability of the solutions for future collaborations. The PROMETRIKA team has also leveraged state of the art EDC and other technologies to provide integral real-time reports of the data to assist the stakeholders in monitoring operational performance and detecting data trends.
Throughout the study, involvement of PROMETRIKA’s upper management team ensures effective operations through a detailed communication plan, and efficient regular remote meetings with occasional face-to-face sessions as necessary.
Safety monitoring
The challenges of safety monitoring in this First-in-Man trial are met by frequent Safety Review Committee Meetings, ongoing reconciliation of clinical and safety data, and a vigilant approach to detecting and reporting unexpected events.
PROMETRIKA’s solutions incorporate consideration of both clinical research and clinical practice concerns and priorities to ensure that the diverse reviewers’ objectives are met using tools such as adaptable reports. Prospective plans for pharmacovigilance, clinical operations, data management, and statistical analysis ensure a harmonized and integrated approach to safety management, reporting, and analysis. The team has a keen focus on data triggers for early signal detection (dose limiting toxicities, discontinuations, missed doses and visits) and on applying early intervention to ensure best trial outcomes.
Budget
The funding mechanism for this trial requires a continual financial focus and avoidance of out of scope costs.
PROMETRIKA’s approach to handing these requirements involves advising on the collection of necessary data and avoidance of “nice to have” data collection, using data management tools to provide early feedback and minimize data query generation, and instituting monthly internal budget reviews to allow rapid response to operational challenges that may result in budgetary scope-creep.
Conclusion
The successful partnership between the sponsor’s program team and PROMETRIKA, all started with transparency and a clear understanding of the desired approach. They knew that finding a flexible and responsive organization was paramount to addressing the needs they anticipated and they kept that their focus as they searched for the right CRO for this program.