MDTServices

NON-CLINICAL DATA 

MDT services provide trainings on general nonclinical programs needed to support clinical trials and the marketing authorization of new molecules or existing molecules in new therapeutic indications.

Training sessions cover non-clinical data requirements for innovative small molecules, large molecules (biopharmaceuticals) as well as advanced therapies (cell and gene therapies).

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MDT services also provide help on nonclinical studies to support different designs of first-in-man trials, such as single dose, multiple dose, estimation of starting doses as well as subsequent dosing schemes (link for http://www.ff.ul.pt/cf/recursos-humanos/docentes-e-investigadores/maria-beatriz-da-silva-lima/)

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SAFETY

Training sessions on safety assessment to support marketing authorization and benefit risk assessment during the product life cycle include:

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- Safety signal detection and evaluation during product life cycle

- Risk Management activities

- Risk Management Plans (RMPs)

- Risk Minimization Plans and strategies

- Benefit Risk assessment during product life cycle

CLINICAL DATA

Support on clinical data generation to support marketing authorization and relative effectiveness assessment cover in particular:

• Clinical development and assessment of all types of medicinal products including advanced therapies, targeted therapies and immunotherapies

• Specificities of co-developments (drug and diagnostic)

• Clinical development of other health technologies (diagnostics in vitro, medical devices)

• Methodology and management of clinical trials

• All aspects from methodological principles to presentation and interpretation of results
  • Choice of relevant trial designs (fit for purpose),

  • Choice of relevant patient populations

  • Choice of endpoints relevant both for clinicians and for patients

  • Choice of adequate comparator(s) and comparisons

• Non-inferiority clinical trials

  • From methodology to reading a publication

• Observational studies

  • Description of different studies design, biases, presentation and interpretation of results

• Relative efficacy and relative effectiveness, how to fill the gap

• Evidence generation: how to prepare scientific advice/early dialogues files at the national and European level

• Early access to market in Europe: pathways, possibilities

• Real world data: state of the art, difficulites

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MORE

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• Efficacy Effectiveness Gap

  • workshops on data requirements with anonymised examples of authorized drugs assessed for reimbursement in different European countries

• Patient-Reported Outcomes

  • From validation process to practice implementation in studies

  • What are the benefits of including PRO in a development program for a marketing authorization or for HTA evaluation?

  • How to choose the good questionnaire to use in a clinical development program?

  • How presenting and interpreting the results of PRO data for different stakeholder perspectives (marketing authorization, HTA, physicians)?

• Ethics and regulatory requirements of clinical research (France and Europe)

  • Landscape of regulatory regulation

  • Principles for drafting an (reasonable) information sheet

  • How to increase a successful submission to Ethics Committee?

  • Risk-based approach of clinical trials

• Critical reading of publications

  • Critical reading of an article, focus on the positive and negative points of the publication (methodology, population included, calculation of the sample size, endpoint model, follow-up of patients, statistical analysis, presentation and clinical relevance of results, extrapolation of results)

• Data Safety Monitoring Board

  • When, why, how : principles for a feasible implementation and management