A key decision in the planning of a clinical trial is that of the sample size. As we’ve discussed previously, the number of participants in a trial is key to whether it will provide useful results – specifically whether the trial will have enough statistical power. Simultaneously, the cost and complexity of a trial can…
To perform any experiment, it is vital to ensure that enough data is collected. Too little data can not only mean imprecise results, but also makes distinguishing between reality and statistical fluke difficult. This concept of statistical power – and, inherent within, sample size – is a key component of trial design: consideration must be…
One key challenge when developing vaccines for human use is that it is often infeasible or unethical to assess the efficacy of the candidate vaccine directly in humans. As we’ve discussed previously, diseases which are severe, rare, or both are difficult to accommodate in human testing. This poses a quandary: these diseases are often the…
When we discuss the performance of a vaccine, properties such as its immunogenicity, adverse event frequency, and any correlates of protection are usually among the first to be mentioned. A property which is considered less often is when a patient can expect to be protected after they have received the vaccine. This is known as…
Vaccines are one of the few medical interventions which are regularly undertaken by healthy individuals. As a result, the bar for ensuring the safety of a vaccine is extremely high. It would be difficult to justify administering a preventative vaccine for a disease an individual might contract if that vaccine regularly causes serious side effects…
Vaccine development often only hits the headlines when there’s a breakthrough clinical trial or final approval. Behind every successful vaccine, however, is a lesser-known stage of research that quietly lays the groundwork: preclinical studies. Don’t be fooled: these early investigations are essential for answering crucial questions which underpin the future of the vaccine. Is the…
The earliest stages of the development of any vaccine involve laying out the roadmap for its design and testing. A vast network of interlinked paths from a new idea to a needle in a patient’s arm stretch ahead into the future, each with unique regulatory and ethical implications. Should the vaccine target a certain strain…
If you’ve ever taken a rapid test for COVID-19, then you’ve used an in vitro diagnostic device (IVD). At the start of the pandemic, the only reliable method for determining whether a patient was currently infected with COVID was a slow PCR test which required processing in a lab to return a result. By contrast,…
When scientific a study is evaluated, the sample size is often among the first issues brought up. Whether it’s the number of participants in a study, the number of times an experiment is run, or even the number of people asked to complete an electoral poll, it is well-known that – in general – a…
The idiom “to pull oneself up by their bootstraps” originated in the late 19th century. At first, it implied impossibility, but has since shifted to mean advances achieved through grit, determination, and hard work, often in business or in one’s career. Thankfully, the statistical method – bootstrapping – named for the idiom rarely requires such…
Statistics is divided between two camps: the frequentists and the Bayesians. The latter of these has begun to attract more popular attention in recent years with the rise of AI and other similar technologies. This has extended to the life sciences, in which there are several fields where Bayesian approaches are taking centre stage. So,…
In previous blogs, we’ve examined the statistics behind Non-Compartmental PK/PD analysis. The existence of the Non-Compartmental Analysis (NCA) implies the existence of compartmental analysis, and, indeed, it is among the most commonly used methodologies for PK/PD statistics. Rather than extract results from data alone as in an NCA, a compartmental analysis involves fitting a model…
In our blog introducing bioequivalence, we described how studies establishing bioequivalence can be a way to avoid expensive clinical trials without compromising on the safety or efficacy of a drug product. In particular, this applies for generic versions of already approved drugs, or for different dosage routes for those drugs. In these cases, bioequivalence studies…
Validation of statistical software is required for use in regulatory work. Many auditors expect to see the traditional IQ, OQ, PQ approach applied to the base system (SAS, R, etc). This approach, however, was never mandated by the FDA – or anyone else – and was never really suited to software validation because it ignores…
One of the greatest challenges in the development of new drugs and therapeutics is that it’s just plain expensive. The average spend on a drug which passes clinical trial is on the order of billions of dollars – and don’t forget that more than 90% of drug candidates don’t make it to market – meaning…
Crucial in understanding the safety and efficacy of a drug are how it percolates through the body, or pharmacokinetics (PK), and how the body responds to its presence, or pharmacodynamics (PD). As with any aspect of drug development, testing PK and PD requires collecting data, and, as with all data, interpreting that data requires statistics….
Unless you’ve been hiding under a rock for the past few months (and, honestly, who could blame you), you will have interacted with artificial intelligence (AI) in some form. Whether it’s using ChatGPT to write an email to that one client you wish would go to your competitor or bemoaning the vagaries of the TikTok…
There are essentially two types of allocation we can make when forming clinical trial groups. The first is a random allocation, which, as you would expect, is unpredictable: the study group to which the subject is assigned is unknown before the process takes place. The second is formally known as deterministic, but we’re going to…
Population Pharmacokinetics (PopPK) is the gold standard method of understanding drug safety and efficacy, particularly as it provides a unique insight into the variation of drug behaviour from person to person. While not a novel methodology—Sheiner, Rosenberg and Marathe first demonstrated the analysis in 1977—techniques and approaches in PopPK have undergone continual improvement in the…
This blog introduces the concept of multiplicity, why it is important and offers some techniques for dealing with the issue. When planning a clinical study, lots of thought is put into what to measure – i.e., which data needs to be collected, how it should be analysed. This leads to the definition of so-called endpoints….