Right study design is crucial for generating reliable evidence, optimal recruitment and retention levels and ensuring successful outcome in a cost-effective manner.

The conception of the clinical trial should start with the definition of the research question and study objectives. Once this crucial step is achieved, a clinical scientist, a physician and statistician work together to define the most appropriate study design based on numerous parameters such as disease, type of product, targeted population, SoC. As an example, a design applicable for a dose finding trial with cytotoxic products with predictable safety/efficacy correlation will not be applicable for targeted therapies, where more innovative designs (mTPI, BOIN, CRM, adaptive trials) will need to be considered.

The phase of the development brings also additional points of control that influences the choice of the right design. These points of control can be the availability of the non clinical data & selection of the starting dose in case of a first in human trial, accumulated clinical data, considerations related to the choice of the comparator and targeted positioning of the product for late stages of the development.

Integrating the patient input and perspectives (patient engagement) in the clinical research process aims to ensure that clinical trials are tailored to meet patient expectations, improve their participation, and produce results much more relevant to clinical practice.

In a patient-centered clinical protocol, particular attention is paid to understanding the real impacts of the disease on the daily lives of patients, understand patient ‘s relevant outcomes and optimizing the constraints imposed by participation in the study.

By promoting such a collaborative approach, patient participation and retention can be increased significantly; and data generated from such trials is more representative of the reality experienced by patients, thus strengthening the external validity of the trials.

It is essential to clearly define the primary and secondary objectives of the clinical study.

The product development stage is a starting point that will constitute a basis for further considerations based on disease or patient severity/targeted population to be enrolled. The main objective of a phase 1/First in Human study would be above all to determine the safety profile and the pharmacokinetics/pharmacodynamics of the product while for the late phases the trials mainly seek to generate conclusive data on the efficacy/effectiveness & the significant benefit to guide decision-making in clinical practice and in policy.

Regulatory agencies such as the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) provide detailed guidelines aiming to assist sponsors in this process. Health technology assessment (HTA) bodies also play a crucial role in defining relevant criteria, ensuring that results are of clinical and economic interest.