What is a Genome Wide Association Study (GWAS)?
A genome wide association study attempts to better understand how specific genetic mutations or alleles correlate with incidence or severity of a disease or other patient phenotype. They typically involve large datasets of patient-level data including both genetic and phenotypic (clinical and demographic characteristics) variables. The UK Biobank is a particularly famous example that continues to be used in support of drug development including identification of new targets for the treatment of specific disease.
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What is minimal clinically important difference (MCID)?
The minimal clinically important difference (MCID) is a threshold which needs to be met for a given intervention compared to standard of care in order to consider it of clinical relevance. The bar for MCID may be higher than that for statistical significance (although this is not necessary) and if so, a statistically significant result may still be considered not meaningful from a clinical perspective because it is not moving the needle enough for patients.
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What is probability of technical and regulatory success (PTRS)?
Probability of technical and regulatory success (PTRS) is the expected chance of success in a clinical trial or clinical program and successful regulatory approval to follow. It consists of two separate probabilities which are multiplied: the probability of technical success (PTS) which is the probability of success of the trial portion alone and probability of regulatory success (PRS) which is the probability of regulatory success alone.
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What are biomarkers?
Biomarkers are biological molecules found in the body that help discern some aspect about the pathophysiology of a disease. Biomarkers can be useful in helping understand which patients might do best on certain therapies (especially targeted therapies). Sometimes, development programs focus specifically on patients who meet a certain biomarker requirement (such as PDL1 expression above a certain thresold in solid tumors or existence of a specific genetic mutation)- examples such as this might be considered “precision medicine”.
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What is contribution of components (CoC)?
Contribution of components (CoC) refers to the impact that specific therapies that are part of a broader set of combination treatment are having for the overall outcome. For example, in a combination treatment of drug A and drug B, one may want to know what drug A means for outcomes when added to drug B compared to using drug B alone. The same question might come up for drug B as an add-on compared to drug A alone.
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What is a Target Product Profile (TPP)?
A target product profile refers to what the expected properties of a drug will be once development (ie studies) are complete. It usually consists of many elements including information on expected dosing, route of administration, potential efficacy and safety profile, etc. As new data emerge once studies readout the TPP is generally updated to reflect the latest knowledge.
The TPP is an important strategic tool used within pharma companies to plan for and assess investigational agents including the market potential, potential marketing strategies, etc.
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What is a Clinical Study Report (CSR)?
A Clinical Study Report (CSR) is a key deliverable that is required in many regulatory submissions and containing information about the methods and results of a clinical trial or clinical study. The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) provides an E3 guidance which orders the structure of the information presented within the document.
This information includes specifics on the proposed course of treatment, the medical information derived from the patients, patient demographics, and other information that gives insight into the trial methods and results.
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What is a Common Technical Document (CTD)?
The Common Technical Document (CTD) provides a shared format that is accepted by various regulatory bodies including the European Medicines Agency (EMA) in Europe, the Food and Drug Administration (FDA) in the United States, and the Ministry of Health, Labour and Welfare in Japan. It is mandatory to use this format for any new drug applications in Europe and Japan, and is recommended for new drug applications (NDAs) in the United States.
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What is Biostatistics?
Biostatistics is a specialization in statistics that focuses on application of statistical methods to studies within the realm of biology. In the context of pharma, biostatistics is a function which owns the statistical work associated with clinical trail design and analyses.
Specifically, biostatisticians in pharma can be part of clinical development teams and advise on things like proper sample sizes to ensure power, the types of statistical analyses which might be appropriate for the endpoints of interest, potential issues which may make statistical interpretation difficult and how to avoid them, etc.
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What is a Post Hoc Analysis?
A post hoc analysis is any analysis of a dataset that was not pre-specified prior to data collection. In pharma, it is most often used in the context of clinical trials and refers to analyses which were not specified in the original statistical analysis plan. Post hoc analyses may look at subgroups of interest or different endpoints than those in the primary analyses.
Post hoc analyses are often conducted following primary result communication and publication of clinical trials, especially pivotal studies.
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