The NSMF Initiative: New Safe Medicines Faster
EFB Section on Medicines Development

Newsletter 14 May, 2004

Medicines Development

Dear Members of EFB Section on Medicines Development

This newsletter holds the following information;

EUREKA Cluster definition phase approved, E!3348 NewMedFaster

I am happy to inform you that the application for the definition Phase has been approved by EUREKA. You may find the details of the application here.

FP6 upcoming 3rd Call focus on NSMF

The text below is a draft circulating, I do not expect major changes. The official announcement is expected in late spring or early summer 2004. You should use this early information presented here to prepare yourself and find partners to exploit this opportunity. Please contact me, especially if you are interested in participating in A)

A) New approaches for accelerated development of new, safe and more effective medicines - INTEGRATED PROJECT.  The focus of this topic is to propose a new comprehensive approach to drug development utilising recent and emerging scientific accomplishments. Bottlenecks/barriers in the current drug development process must be identified and solutions elaborated to overcome them.  The project should contain two components:

1. To deliver a comprehensive strategy with a detailed roadmap to reduce the drug development time, encompassing the whole path from discovery of a new molecule to the validation and approval stages, ensuring high levels of drug safety and efficacy as well as fast availability of innovative medicines to the patients.

2. To conduct, in support of the strategy, exploratory and demonstrative research activities within one or two of the major chronic progressive disorders, where novel concepts for accelerated drug development can be tested and evaluated. The research must address key areas, which are linked to the bottlenecks in drug development and include regulatory aspects. 

The consortium should involve all relevant stakeholders (academia, clinicians, patient organisations, large industry, SMEs, regulatory and ethics specialists).

B) Pharmacogenomics for individualised medicines - INTEGRATED PROJECT. The research objective is to increase the specificity and to improve the risk/benefit ratio of new drugs, by using pharmacogenomic methods. Research should focus on the interaction of drugs with their receptors, transporters and metabolising enzymes. The deliverables are the development of in vitro and in vivo models to predict inter-individual variations in drug absorption, distribution, metabolism, elimination and clinical effects. This necessitates a close integration of clinical, epidemiological, regulatory, ethical, economic and industrial aspects, as well as those related to public perception. This expertise must equally be reflected in the consortium composition, including partners representing all relevant disciplines, and, in particular, ethical, regulatory and patients' issues.  (For orphan medicines see STREP in area "Combating cardiovascular diseases, diabetes and rare diseases" page 15.)

C) Standardised assays for determining immunogenicity of biopharmaceuticals -STREP. The aim of this topic is to improve and standardise the detection and prediction of immune reactions. Deliverables are the development and standardisation of assays (e.g. antibody assays) and reference preparations to allow for inter-laboratory comparisons, so as to predict and thereby avoid such complications arising from the use of biopharmaceuticals.  This implies a close co-operation of all relevant disciplines, including ethical and regulatory specialists, SMEs and the pharmaceutical industry.

D) 'Microdosing' studies to gain enhanced absorption-, distribution, metabolism, excretion (ADME) - parameters for biopharmaceuticals (especially orientated towards involvement of SMEs) - STREP.  The focus of this topic is the development of technologies enabling the conduct and evaluation of 'microdosing' studies in humans as a means to obtain basic pharmacological data on biopharmaceuticals. This includes the set-up of clinical trials for administration of sub-pharmacological doses to humans and its comparison to existing data on pharmacological doses.

E) Exploring the potential of stem cells and/or primary cells, for the understanding of monogenic rare diseases and the development of new drugs for their treatment - INTEGRATED PROJECT. Mutated stem cells and/or primary cells can be a powerful analytical tool to better understand the molecular and cellular mechanisms of monogenic rare diseases, especially using genomics and proteomics knowledge. This project aims to develop accurate cellular models for studying the diseases, to explore the capacity of stem cells differentiation for understanding the development of these diseases, and to explore the potential of these cells as screening tools for lead compound identification and/or drug targeting validation, using high-throughput and high-content technologies.

F) Targeted delivery of protein-based drugs (especially orientated towards involvement of SMEs)  - STREP. This topic focuses on research addressing the barriers involved in delivering protein-based drugs to their molecular receptor. The deliverable is a better understanding of the inter- and intracellular transport of protein-based drugs leading to specialised drug-delivery systems.

G) Cell systems as a means to enhance toxicity testing - INTEGRATED PROJECT. Cell-based model systems will be developed for testing the toxicity of biologicals, pharmaceuticals, chemical compounds, directly related to human health. Research is expected to make use of the opportunities offered by functional genomics to deliver faster tools with proven potential for multiple applications in relevant industrial sectors and to make a substantial contribution to the "Three Rs" (replacement, reduction and refinement of animal experiments, cfr. introduction). The involvement of SMEs in the various steps of the proposed work is expected. The development stages could include the use of array technology, the demonstration and validation ones will make use of the already existing (toxicological) data, thus avoiding unnecessary experiments.

H) Optimization and pre-validation of in-vitro models for the study of drug absorption, modification and detoxification in the liver and in the intestinal epithelium (especially orientated towards involvement of SMEs) - STREP.  Research in this STREP will contribute to the various phases of a fully integrated drug development programme. As this is an area that the pharmaceutical companies and other drug discovery and development entities are paying close attention to, specific emphasis has been placed on SME participation, also considering the fact that in vitro screening techniques represent a significant market opportunity, (hugely-expensive animal studies do take place in the process of drug development) growth is predicted in this market segment. Studying the absorption by the intestine, the distribution to the organism, and the metabolism by the liver will provide supportive information to augment the interpretation of toxicology findings, reinforcing drug efficacy.

I) Workshop: how to integrate and make optimal use of animal data, non-animal data and predictions from computer-based modelling in assessing the risk of chemical compounds? - SSA

J) Mini-pigs as models for toxicity testing of new drugs and chemicals: impact assessment - SSA

Kind regards, Jorgen Dirach

Dr. Jørgen Dirach, MD, MBA, MFPM
Section Chairman