scholarly journals Biomarkers and Surrogate Endpoints in Drug Development: A European Regulatory View

2017 ◽  
Vol 58 (6) ◽  
pp. BIO27 ◽  
Author(s):  
Kerstin Wickström ◽  
Jane Moseley
Keyword(s):  
Drug Development ◽  
Surrogate Endpoints ◽  
European Regulatory

scholarly journals Bayesian meta-analytical methods to incorporate multiple surrogate endpoints in drug development process

2015 ◽  
Vol 35 (7) ◽  
pp. 1063-1089 ◽  
Author(s):  
Sylwia Bujkiewicz ◽  
John R. Thompson ◽  
Richard D. Riley ◽  
Keith R. Abrams
Keyword(s):  
Drug Development ◽  
Analytical Methods ◽  
Development Process ◽  
Surrogate Endpoints ◽  
Drug Development Process

scholarly journals A European regulatory perspective on cystic fibrosis: current treatments, trends in drug development and translational challenges for CFTR modulators

2018 ◽  
Vol 27 (148) ◽  
pp. 170124 ◽  
Author(s):  
Stefano Ponzano ◽  
Giulia Nigrelli ◽  
Laura Fregonese ◽  
Irmgard Eichler ◽  
Fabio Bertozzi ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Drug Development ◽  
Forced Expiratory Volume ◽  
The European Union ◽  
End Points ◽  
Sweat Chloride ◽  
European Regulatory ◽  
Clinical Models ◽  
Cf Transmembrane Conductance Regulator ◽  
Cftr Modulators

In this article we analyse the current authorised treatments and trends in early drug development for cystic fibrosis (CF) in the European Union for the time period 2000–2016. The analysis indicates a significant improvement in the innovation and development of new potential medicines for CF, shifting from products that act on the symptoms of the disease towards new therapies targeting the cause of CF. However, within these new innovative medicines, results for CF transmembrane conductance regulator (CFTR) modulators indicate that one major challenge for turning a CF concept product into an actual medicine for the benefit of patients resides in the fact that, although pre-clinical models have shown good predictability for certain mutations, a good correlation to clinical end-points or biomarkers (e.g. forced expiratory volume in 1 s and sweat chloride) for all mutations has not yet been achieved. In this respect, the use of alternative end-points and innovative nonclinical models could be helpful for the understanding of those translational discrepancies. Collaborative endeavours to promote further research and development in these areas as well as early dialogue with the regulatory bodies available at the European competent authorities are recommended.

scholarly journals Biomarkers and Surrogate Endpoints: How and When might They Impact Drug Development?

2002 ◽  
Vol 18 (2) ◽  
pp. 83-90 ◽  
Author(s):  
Chetan D. Lathia
Keyword(s):  
Decision Making ◽  
Drug Development ◽  
Development Process ◽  
Surrogate Endpoints ◽  
Pharmacokinetic Data ◽  
Drug Development Process ◽  
High Likelihood ◽  
Molecular Pharmacology ◽  
Toxicity Biomarkers ◽  
The Cost

As the pharmaceutical industry starts developing novel molecules developed based on molecular biology principles and a better understanding of the human genome, it becomes increasingly important to develop early indicators of activity and/or toxicity. Biomarkers are measurements based on molecular pharmacology and/or pathophysiology of the disease being evaluated that may assist with decision-making in various phases of drug development. The utility of biomarkers in the development of drugs is described in this review. Additionally, the utility of pharmacokinetic data in drug development is described. Development of biomarkers may help reduce the cost of drug development by allowing key decisions earlier in the drug development process. Additionally, biomarkers may be used to select patients who have a high likelihood of benefit or they could be used by clinicians to evaluate the potential for efficacy after start of treatment.

Development of Orphan Drugs under European Regulatory Incentives and Patent Protection

2017 ◽  
Vol 24 (3) ◽  
pp. 239-263 ◽  
Author(s):  
Mari Minn
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Patent Protection ◽  
Market Access ◽  
Orphan Drugs ◽  
Patient Access ◽  
Generic Market ◽  
European Regulatory ◽  
Orphan Drug Development ◽  
Regulatory Incentives

AbstractThis article analyses how the regulatory incentives provided by Regulation 141/2000 affect orphan drug development and generic market entry. It seeks to find out how the regulatory incentives work in combination with patent protection, and whether in combination they foster orphan drug innovation and overall patient access, or rather hinder the latter. The article argues that even though the regulatory incentives are targeted to fostering innovation and early generic market access, when combined with patent protection, the generic entry is likely blocked or delayed.

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