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Clinical Research

Study Design & Methodology

Align the study with overall medical, access to market, regulatory goals

Designing and executing a clinical study that encompasses medical, market access and regulatory considerations is crucial for generating reliable evidences and ensuring successful outcomes. The goal is develop a meaningful protocol for patients and optimize it to tackle challenges from regulators and at sites. 

It is crucial to tailor the study design and methodologies to address specific factors effectively such as microbiome, medical device specificities. Collaborating with multidisciplinary enhances the rigor and validity of the study.

Our integrated solutions

We collaborate closely with Key Opinion Leaders and healthcare professionals and tailor the study design to capture relevant clinical endpoints, patient-reported outcomes and other essential measures specific to ensure the study design is aligned with medical needs. 

We engage our market access experts early in the study design to gain insights into pricing, and reimbursement requirements. We also consider patient engagement to demonstrate the value proposition to payers and patients. 

Closely collaborating with regulatory authorities from the outset to address their specific requirements to develop a robust study protocol that adheres to ethical principles, data protection regulations and local and international regulatory standards.

How to choose an appropriate study design?

What does “patient-centric protocol” mean?

How to choose the meaningful endpoint?

What we offer

  • Align the study with overall medical, access to market & regulatory goals
  • Author study synopsis and patient-centric protocol
  • Leverage patient organizations
  • Combined in-house expertise with medical and statistical inputs
  • Statistical review of the protocol according to ICH E9 or other specific guidelines
  • Optimize study design to streamline the development plan
  • Randomization strategy
  • Accurately define the level of blind
  • Sample size calculation
  • Test multiplicity handling and missing data imputation  
  • Interim analyses strategy
  • Statistical analysis plan, study population definition
  • Ensure data eligibility for registration, price & reimbursement
  • ​Fields of expertise in rare diseases, medical devices, microbiome-based product, etc.


How to choose the appropriate study design?

Design of clinical study is a key step allowing to optimize and de-risk the overall clinical development program up to access to market as well as its acceptability by several stakeholders, notably, regulators, payers and patients. 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.

Patient-centered protocol places the needs, preferences, priorities and experiences of patients at the heart of the research process. 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.

A relevant endpoint is essential to measure the efficacy/effectiveness of a medical intervention in an objective manner, influencing the validity of the results obtained and the applicability of the conclusions in medical practice. 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.

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