As the astronaut, Neil Armstrong once said, ‘Research is creating new knowledge’.
Within the pharmaceutical industry, Phase 1 trials represent both a cornerstone of our knowledge about a new compound and a significant milestone in the drug development lifecycle.
For smaller biopharma companies that may be relying on a handful of promising drug candidates, the move into Phase 1 often also represents an important pivot point in their organisation’s evolution.
At this stage, we assess the safety, side effects, best dose, and formulation method for a given compound and gather valuable data to inform the next development phase. Common study types encompass single ascending dose, multiple ascending dose, food-effect, and potential drug-drug interaction. Sponsors may also conduct other assessments to investigate the effect of sex, age, bioavailability and bioequivalence on the drug’s safety, side effects and dosing.
Phase 1 studies tend to be relatively short and usually conducted at a single site or dedicated Phase 1 unit with a small number of healthy subjects. As well as being first-in-human trials, additional clinical pharmacology Phase 1 trials may form an integral part of phase 3 planning as sponsors seek to gather further insights on different elements of the compound. Phase 1 trials, therefore, offer stimulating work, variety, and knowledge development for Statisticians. One recent programme Veramed has supported incorporates eight phase 1 trials studying aspects from bioequivalence in different ethnicities and age demographics, and pharmacodynamic interactions.
This scale of programme is not unusual, and sponsors and CROs may be regularly managing multiple Phase 1 studies at one time. While they are certainly lower complexity in terms of trial design, number of subjects, and nature of data collected, they nevertheless pose several challenges that we can help address as Statisticians and Programmers.
Protocol amendments
Early phase trials tend to be quite volatile, and protocol amendments are common. When protocol amendments occur, all the other associated documents need to be updated, including the case report form and the statistical analysis plan. As Statisticians working on early phase trials, we aim to be as flexible as possible and build that adaptability into our working days. Often these documents will require a short turnaround time to review, and it is vital to provide quick feedback. With other studies depending on the outcome, our fast responses ensure seamless progress.
Quick turnaround
The turnaround time between database lock and reporting for Phase 1 trials is often as little as ten business days. Early phase Statisticians need to work proficiently to achieve both speed and high-quality outputs. This focus on quality is essential and should extend to ensuring impeccable formatting and presentation of deliverables as well as the 100% integrity of the analyses. Just as we have a quick turnaround to create the outputs, the sponsor Statistician will also need to review swiftly, and our deliverables should reflect that.
Managing multiple projects
With several Phase 1 studies often running concurrently, strong project management and organisation across the statistical and programming team is critical. There are multiple opportunities to gain efficiencies across studies and apply consistent approaches and decisions that can improve standardisation as well as save time and oversight. For one of our clients, our team reported eight Phase 1 studies within a year by applying flexibility, smart resourcing and efficient processes – it was demanding but extremely rewarding.
Budget efficiency
Phase 1 trials tend to have lean budgets. As Statisticians, we can look for opportunities to be creative and streamlined in proposing which tables, listings and figures need reporting and what data points need to be collected to get the most from the trial budget. More isn’t always better – we must look at what is necessary and what is going to add value to the research question we are asking?
Veramed has extensive experience meeting the particular challenges of Phase 1 trials and supporting clients on a range of Phase 1 study designs across various indications. We developed a team of Statisticians and Programmers with particular know-how in these demanding, fast-paced trials. We work collaboratively as a statistical partner to sponsors at this critical point in development, ensuring the right answers to early research questions and giving the new compound of interest the best opportunity to progress to the next milestone.
To learn more about how Veramed can help your business and clinical trial needs, simply get in touch with us below.
