Clinical drug development is often described as consisting of four sequential phases (Phase I-IV). The evolution of drug development studies can be classified according to when the study occurs during Clinical Development.
Adaptive Trial Design According with the FDA, an adaptive design clinical study is defined as a study that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data (usually interim data) from subjects in the study.
Concerns the regulatory and legal requirements of biomedical product development to the scientific research needed to ensure the safety and efficacy of such products.
Regulatory intelligence Relates to processing targeted information and data from various sources, analysing the data in its relevant context and generating a meaningful output to the regulatory strategy. The process is driven by business needs and linked to decisions and actions.
Regulatory strategy Is the plan outlined for the successful approval of the development candidate. It is heavily based on a through intelligence work (for example, knowledge of relevant guidance documents, regulatory framework) and is highly interactive with other disciplines (for example, a clinical development plan based on scientific advice from a Health Authority which was set out according with the regulatory rules).
Is the science and activities relating to the detection, assessment, understanding and prevention of adverse events and any other drug-related problem (according to the definition of the WHO).
Risk Management Plans Is the plan that defines the process by which risks factors are systematically identified, assessed and dealt with. Sometimes these plans are integrated conditions for Marketing Approval (MA), examples are the Post-Authorization Safety Studies (PASS) and Post-Authorization Efficacy Studies (PAES).
Is defined as a set of activities in a process that companies (pharma and biotech) need to go through to ensure their products are made available in as many countries as possible and most importantly that these products are reimbursed and made available to patients who need them.
Pharmacoeconomy Is the science that considers cost-benefit, cost-effectiveness, cost-minimization, cost-of-illness and cost-utility analyses to compare pharmaceutical products and treatment strategies relating to health care systems and society.
HEOR/HTA Evidence-based information that is able to provide meaningful insight on product value and its potential in real-world clinical practice is provided through Health Economics and Outcomes Research (HEOR). Moreover, the process for reimbursement is now characterized by a Health Technology Assessment (HTA) approach whereby payer organizations evaluate new technologies on the bases of their clinical value, cost-effectiveness and other health economic factors is a process which is increasingly becoming more significant in market access.
Real World Evidence
Data that is collected outside the controlled limitations of conventional randomised clinical trials to evaluate what is happening in normal clinical practice. Real world data as well as analytics can be a valuable support to clinical trial design, patient selection schema, and inform the collection of specific patient reported outcomes and economic data to establish ‘value’ throughout clinical development phases. This information can be collected in a number of ways.
Prevalence/Incidence Studies In epidemiology, prevalence is the proportion of the population with a given disease or condition over a specific period of time. The incidence of a disease is the rate at which new cases occur in a population during a specified period. These type of studies are examples of the type of data that can contribute
Disease Registry As the name implies, these studies involve registering and subsequently analysing all patients treated at a particular centre for a particular condition on a continuous basis. The use of registries – both large and small – is increasing as a way to collect real world data.
When establishing an R&D program, it is important to identify critical R&D milestones in the path of achieving the company’s goals, so building a solid strategic basis for that program is key.
Technology Transfer By definition this is the process of converting scientific and technical advances into marketable goods or services.
Translational Medicine It is a bidirectional process, covering so-called bench-to-bedside factors, which aim to increase the efficiency by which new therapeutic strategies developed through basic research are tested clinically, and bedside-to-bench factors, which provide feedback about the applications of new treatments and how they can be improved.