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 a systematic literature review (SLR)?
A systematic literature review (SLR) is a type of study in which a search of the published literature is conducted using a pre-specified and structured approach. They are a good way to get an understanding of what evidence is currently available on a specific question or topic and in turn can be used to inform understanding of key data gaps. They can also be the first step in collating studies that can then be used in meta-analyses.
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What is a Non-Interventional Study?
A non-interventional study (NIS) is a broad term that includes any type of research in which patients are treated as they otherwise would in the real world. This can include retrospective studies utilizing sources such as patient charts, electronic health record data, administrative claims data, or patient registries as well as prospective registries that do not have an interventional component. Typically, the term NIS (or NIR for non-interventional research) is used to describe studies of medical or clinical nature and not market research.
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What is a treatment sequencing model?
A treatment sequencing model attempts to incorporate data from different clinical or real world studies in order to understand how different treatment sequencing strategies may impact patient outcomes in specific populations. This is particularly important to understand in therapeutic settings in which there are multiple options and no clear consensus on the right order of medications to use. Treatment sequencing models can help inform decisionmaking in these settings by incorporating data from clinical trials or real world studies while attempting to reduce biases associated with comparisons when no direct comparison (randomized controlled trial or other head-to-head study) exists.
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What is a Statistical Analysis Plan (SAP)?
A statistical analysis plan is a detailed document outlining the statistical analyses (both descriptive and inferential) that are planned for a given study. It is usually created prior to start of a study and for pivotal studies must be in line with regulatory requirements in terms of acceptability of endpoint analysis and with regards to prespecification.
The study protocol might get into elements of the statistical analysis plan, but the SAP goes into much more detail.
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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 an Advisory Board?
An advisory board refers to a meeting of experts that is convened by a pharma company to gain insights around a particular topic. They are usually held by a specific brand or franchise and can include a set of key opinion leaders (KOLs), payers, or both. Advisory boards can last anywhere from a couple hours to a full 8 hours and can be conducted in person or remotely.
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What is a Budget Impact Model (BIM)?
A budget impact model is a standard deliverable for HEOR teams and allows for estimation of the costs associated with adding a drug to formulary for a given payer or health system. It is often used in discussions with payers by medical outcomes liaisons (HEOR field team) and potentially by market access account executives as well in the US. Outside of the US, it may be used by some affiliates with various payers depending on the country situation and laws.
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