Translational Sciences

Translational Sciences

Defining the path towards critical value inflection points and its timely execution while balancing risks and resources is a key strategic focus for small to medium enterprises. Integrating project specific development strategies and the respective milestone deliverables in the overall corporate objectives provides a strategic roadmap to valuable exit points and allows acceleration of high value projects.

At NDA we’ve brought together unmatched scientific and regulatory expertise that provides a unique strategic partnership. We can help you map out the development opportunities, assess the risks, provide risk mitigation and management tools and we can help you determine a clear path forward to the next value inflection point.
By applying a stage-gate approach, we can help you focus on defining input data to inform decision-making at critical stages.

The experts at NDA can provide a “sounding board” for business ideas, a “strategic partner” to help you get from idea to value and an “execution partner” to help you execute projects efficiently within the resource constraints.

The benefits of this approach are:

  • Tailored drug development advice to meet the regulatory requirements and expectations for both the EU and the US
  • Expert critical input and assessment of alternative routes for product development
  • Focus resources, accelerate development, provide choices for exit scenarios
  • Increased efficiency due to the identification of common (core) requirements, resulting in a core FIH clinical dossier for the EU and US

The stage-gate approach enables a clear establishment of acceptable product profile and identification of strategies to mitigate any challenges/gaps. In addition, this enables an early assessment of benefit/risk which is further developed through life cycle.

The Stage Gate Approach:

Opportunity Analysis

Helping you determine the commercial viability of proposed target and indication by:

  • Understanding target biology and relevance in disease or condition
  • Selecting technology platform
  • Determining the translational strategy to demonstrate meaningful treatment benefit

Lead candidate selection

Helping you determine the optimal molecular format:

  • To minimise immunogenic potential
  • To characterize mode of action
  • To determine if biomarker identification can be applied
  • To ensure appropriate pharmacology and toxicology profile

Develop-ability

Helping you ensure that adequate quality and productivity achieved:

  • To develop a manufacturing plan
  • To minimise product-quality related risk factors
  • To ensure compliance with global regulatory requirements

Translational Strategy

Helping you determine dose- and concentration-response relationships for pharmacology and adverse effects:

  • To ensure that appropriate in vitro assays and tools are being applied
  • To ensure selection of relevant animal models
  • To ensure use of in vitro studies to address limitations of animal models
  • To ensure that the PK-PD-ADA-Safety endpoints measured
  • To ensure that the translational strategy informs PK-PD and delivers IND-enabling data

IND-enabling Studies

Helping you ensure that all pertinent risks evaluated in relevant models:

  • To justify species
  • To justify size of study population, assessments and parameters
  • To justify relevance of findings in safety studies and translation in clinical investigations

First Time-in-Humans

Helping you ensure that there is a positive benefit-to-risk for administration to humans:

  • To justify study population
  • To justify clinical starting dose and dose escalation
  • To justify treatment algorithm, integration of translational elements, study analysis
  • To ensure safety assessment and risk management (including immunogenicity)

Clinical Proof of Concept

Helping you ensure that the evidence of therapeutic effect is demonstrated:

  • To understand the target patient population
  • To address safety risks and management
  • To justify of dose and schedule
  • To determine the heterogeneity of patient population
  • To measure the treatment outcome