Mathematical Models Target Disease with Drugs Chosen by Your DNA

MP900337348HERSHEY, Pa. — Medicines that are personally tailored to your DNA are becoming a reality, thanks to the work of U.S. and Chinese scientists who developed statistical models to predict which drug is best for a specific individual with a specific disease.

“Traditional medicine doesn’t consider mechanistic drug response,” saidRongling Wu, director of the Center for Statistical Genetics and professor of public health sciences within the division of biostatistics and bioinformatics at the Penn State College of Medicine. “We want to look at how an individual person responds to an individual drug by deriving and using sophisticated mathematical models, such as differential equations.”

The current method of prescribing medication now sometimes includes a pharmacogenomic approach, but researchers recognize the limitation of this approach in predicting a response to a particular drug and dosage combination.

Pharmacogenomics uses a person’s genes to explain the difference between how one person responds to a drug compared to another. The team’s equations take this field one step further by also including information about how the body processes a drug and how the drug acts in the body.

Wu focused on the big picture by studying drug response and drug reaction. In particular, the team looked at pharmacokinetics, which influences the concentration of a drug reaching its target, and pharmacodynamics, which determines the drug response. Metabolic, environmental and developmental factors also play a role in medication response.

The researchers created a statistical analysis framework of differential equations that they expect will help doctors and pharmacists, by simulating such variables as protein-protein and protein-DNA interactions a drug has in a patient. The framework characterizes a drug’s absorption, distribution and elimination properties, yielding information on pharmacological targets, physiological pathways and, ultimately, disease systems in patients, resulting in predictions of treatment effectiveness.

“The results from this framework will facilitate the quantitative prediction of the responses of individual subjects as well as the design of optimal drug treatments,” the researchers noted in a recent special issue of Advanced Drug Delivery Reviews.

This framework will expand to shed light on the variability of drug response based on information the medical community continues to gather about how an individual responds to a particular drug and dose combination. The information collected is then combined with information about the patient’s genes, proteins and metabolism to help determine what drug and dose might be best for that person.

“If we know how genes control drug response, we can create a statistical model that shows us what will happen before using the drug,” said Wu, who is also a member of Penn State’s Huck Institutes of the Life Sciences. This means a future with more effective medications, faster healing and fewer side effects. “That is our final goal,” Wu said.

Working with Wu were Yaqun Wang and Ningtao Wang, both graduate students in the Center for Statistical Genetics at Penn State; and JianxinWang, professor, information science, and Zhong Wang, computer scientist, both at the Center for Computational Biology at Beijing Forestry University.

The National Center for Advancing Translational Sciences of the National Institutes of Health supported this research.

Source: Penn State – original article was written by Victoria M. Indivero.

EMA publishes guidance on preparing and reviewing summaries of product characteristics

logo_ema21/01/2013 – The European Medicines Agency has published guidance for pharmaceutical companies on how to prepare and review summaries of product characteristics (SmPCs) for human medicines. The guidance consists of a set of presentations detailing the information that should be included in each of the sections of the SmPC, together with background information on SmPCs both as a presentation and a video. Two videos explaining how to complete the SmPC sections on the therapeutic indication and pharmacodynamic properties of a medicine and on undesirable effects are also available.

The guidance, prepared by the Agency’s SmPC Advisory Group, outlines the principles in the European Commission’s guideline on SmPC. It is intended to enable companies to make sure that the information in SmPCs is of high quality when they submit them to the Agency as part of applications for new marketing authorisations or updates to existing marketing authorisations.

The background presentation and video also aim to raise awareness of the information provided in SmPCs among healthcare professionals.

SmPCs are a key part of the marketing authorisation of all medicines authorised in the European Union and the basis of information for healthcare professionals on how to use a medicine safely and effectively. They are kept updated throughout the lifecycle of a medicine as new efficacy or safety data emerge.

SmPCs are also the basis for the preparation of package leaflets, so are important documents in enabling information on medicines to reach patients.

Source: EMA News

Medical/Pharmaceutical Translations 2012-2013 Trends

Weather Vane with Dollar SignBack in January 2012, I made the following forecasts for 2012 compared with 2011.

  • A higher volume of work
  • An increase in rate levels for qualified translators
  • The social networks growing in significance
  • The specialised ‘tools of the trade’ are required as ever, but the definition of exchange formats and workflows needs to be driven ahead
  • Machine translation has yet to fulfil its promises
  • Translation associations should be looking at extending their range of educational and CPD facilities
  • Representing the interests of the translation profession must be reinforced

The original article is here (only available in German)

Now that the year 2012 has come to an end (and the world has survived – contrary to expectations in some quarters), it is worth considering to what extent these predictions have changed and whether indeed new and interesting trends have developed.

Volume of Work/Rate Levels

Here, we would benefit from data that are more topical and reliable. The first two statements for the medical/pharmaceutical sector are still applicable in my opinion; albeit based upon data from a small group of LSPs with which I maintain close contact in that respect. Nevertheless, I increasingly note suggestions in various blogs and forums that could lead one to conclude that the market should be substantially more dynamic than it is from my vantage point. I would like to see more information about the scope of orders and rates, since information like this could help us to identify seasonal and absolute trends. Using such data, it would be possible to react and the data would lessen the partly hysterical cries about sinking rates which – in my opinion – are certainly to the detriment of our profession.

Social Networks/Internet Culture

The social and professional network tools (Twitter, Facebook, LinkedIn, Xing and Google+) are becoming ever more important and the previous translation platforms (Proz.com, Translatorscafe etc.) are suffering from increasingly less importance. This can be seen variously in the increasing number of translation groups e.g. on Facebook, LinkedIn, Xing, where more and more business is transacted and also in the range of CPD facilities being made available via these groups.  Professional associations such as the German BDÜ took their time to set foot onto the social networks but in the meantime, they have understood the significance and are presenting themselves professionally on these platforms.

Unfortunately this development does not just have positive aspects. As a freelancer, it is impossible to follow all groups within which interesting projects are posted and also as an LSP, it is becoming ever more difficult to find specialists for specific projects on the various platforms.

For this reason it will be necessary to develop aggregators that bundle the various offers. On Twitter, we have made a first step towards combining job offers from various sources by means of our @Translate_Jobs account. We also offer similar services to embrace news from the translation profession with @Translate_News, interesting blogs and events in the profession with @Translate_Blogs and @TranslateEvents.

These solutions are, however, limited by the facilities that Twitter offers, which is one of the reasons why we launched our Alexandria platform to cover the area of CPD opportunities.

Specialised Tools/Interoperability/Crowd and Cloud Services

In the field of interoperability, good things are happening as the two top dogs MemoQ and Trados benefit from ever more functions to improve interoperability between the individual programs. Here it only seems natural that recent weeks have seen massive criticism of the hermetically-sealed protected design of the across program. I am somewhat more cautious in this respect, since I thoroughly recognize the necessity for closed workflows and would prefer an appropriately optional functionality from other vendors. At the same time, I would naturally appreciate it should across deign to open up.

What I cannot, however, understand is how one can work as a translator with the cloud services that are springing up like mushrooms. This is a TM solution that can only bring disadvantages to the translator with a lack of their own TM, no traceability of tasks performed etc. etc.

Machine Translation

I would appreciate having a functional system, but unfortunately have yet to find one. There is nothing more to be said, other than the fact that I will keep my eyes open. What I find interesting are two aspects:

a) We translators are told more and more that there is a an enormous and ever-growing market for bad ( i.e. machine) translations. Well, that is fine for those who are happy to read dross, of which there is an appalling abundance on the Internet. The main problem as I see it is that the time will come when readers actually believe these to be bona fide translations.

b) At the same time, I hear that trained MT systems within limited domains and certain language pairs can produce results that are supposed to be better than those produced by human translators. But the decisive point is that so far, nobody has been capable of showing me such a system or its results. Last year, several MT vendors explained to me just how remarkable their systems were, but when push came to shove, I saw nothing convincing other than impressive statistics that were of no consequence whatsoever.

Now that I have set up Trados Studio with TMs including several millions of words and autosuggest dictionaries of up to 1 GB in size, I can reach a level of productivity where I can indeed ask myself to what extent I need MT for our language pairs and specialized areas.

Education and Continued Training

Here, there is something afoot. Germany’s BDÜ and DVÜD, as well as other providers, have significantly increased the range of their online CPD facilities. In fact at first glance, it might seem to be superfluous that we are entering the market with our own offering (http://alexandria-library.com). However, with the Alexandria Project, we do indeed have several objectives in mind. With it, we would like to create a central platform (by means of collaboration with as many vendors as possible e.g. Diléal and Localize.pl), upon which we can offer continued training and resources for new entrants to the profession and specialists within the various languages. In addition to that, we would like to offer specialists a platform that enables them to present themselves in order to improve their reputation in the profession and with future clients. Thirdly, we want to start using this platform as soon as possible to draw the attention of potential customers to the necessity of qualitatively acceptable translation, whilst attempting to educate them about how they can identify suitable language service providers, or rather what they themselves can contribute in order to achieve optimal results. In that department, we still ‘have the builders in’ but we shall soon be expanding what we have on offer. Feedback and suggestions will be very welcome indeed because Alexandria is – after all – intended to provide an interesting service to as many translators and customers as possible.

The Interests of the Translation Profession

So far, I was disappointed to observe that translation associations carry out too little to promote the profession externally in a way that generates interest. Translators and translation associations seem to be too occupied with themselves (i.e. with translation per se) and enter much too little into contact with possible customers, whose lack of information about translation, quality, processes and rates tends to lead them down into the depths frequented by the so-called ‘bottom feeders’. It would be laudable to see several national associations deciding upon closer cooperation with each other and being outwardly active in terms of customer education and representing the profession. A common European job portal of translation associations could help in this respect. Here, customers looking for translation service providers would at least have the reassurance that the translators fulfil certain minimal criteria of professionalism. This would draw attention away from the Internet platforms such as Proz and TC, where all the cut price vendors who often provide bad quality lurk, since customers seeking quality would finally have a qualitatively more valuable service at their disposal.

Conclusions

I am not sure to what extent much changed in the profession during 2012, but I see a careful trend for translators taking on more responsibility for their own fate and success and emancipating themselves from the clutches of major organisations and company groups. In 2013, this positive development can lead to a wider movement coming together that brings us forward as a profession. I will be delighted if we can make our contribution to that with Alexandria and Trikonf 2013.

Proactive publication of clinical trial data – discussing the way forward

09/08/2012 – The European Medicines Agency has announced that it will proactively publish clinical trial data and enable access to full data sets by interested parties. A number of practical and policy issues need to be addressed before complex data sets can be made available.

The Agency is organising a workshop on 22 November 2012 to listen to the views, interests, and concerns from a broad range of institutions, groups and individuals. Results from the workshop will help the Agency define the modalities of proactive access to clinical trial data, in a way that best serves patients and public health in an open and transparent forum.

Registration is open until 31 October 2012. Expressions of interest should be sent to ctdataworkshop@ema.europa.eu by 31 October 2012. Places are limited to 150 and will be offered on a first-come-first-served basis.

Source: EMA News and press releases – http://www.ema.europa.eu

International IT Regulations and Compliance. Quality Standards in the Pharmaceutical and Regulated Industries

Research and Markets has announced the addition of John Wiley and Sons Ltd’s new report International IT Regulations and Compliance. Quality Standards in the Pharmaceutical and Regulated Industries to their offering.

After taking part in an EU Leonardo da Vinci project to create a complete curriculum for a Master’s degree in IT Validation, Siri H. Segalstad decided to write this book as she realised there was a need for a comprehensive book that rings together current thinking on the implementation of standards and regulations in relation to IT for a wide variety of industries.

This text allows the readers to acquire not only knowledge but also understanding of quality thinking and to apply this knowledge. It will allow them to use the Quality Management System (QMS) as a tool for further development in their organization and to assess QMS from other companies during a supplier audit.

This book will enable the user to understand the process of validation, how to divide validation into manageable pieces, and what is included in the validation for different types of systems.

Topics covered include:

- Quality standards
– Regulatory requirements for IT systems
– Quality Management Systems-QMS
– Organization for an IT system
– Legal implications of an IT system
– Advanced quality management systems
– Validation process and validation techniques
– Validation of IT systems
– Risk assessment and risk management
– Laboratory Information Management Systems (LIMS) and Building Management Systems (BMS)

This comprehensive text will be useful to those working with quality assurance and validation of IT systems and in regulated industries, regardless of which standards they are using.
For more information visit Research and Markets

Source: John Wiley and Sons Ltd
Business Wire 
Source: Pharmiweb.com

New pharmacovigilance legislation comes into operation

Better protection of public health through strengthened EU system for medicines safety

02/07/2012 – The European Medicines Agency (EMA) welcomes the start of new European Union (EU) legislation on pharmacovigilance. This new piece of legislation aims to promote and protect public health by strengthening the existing Europe-wide system for monitoring the safety and benefit-risk balance of medicines.

“The progress made in healthcare would not have been possible without medicines and the research and development community behind them. But we need smart regulation for the system to be able to continue to deliver safe and effective medicines,” said the Agency’s Executive Director, Guido Rasi.

“The new pharmacovigilance legislation will help us to make the system more robust for public health and more transparent. It gives regulators a range of new or improved tools to ensure that patients are not exposed to unnecessary risks when taking medicines. It also increases the efficiency of medicines regulation for the benefit of all stakeholders.”

The new legislation was proposed by the European Commission in 2008 and adopted by the European Parliament and the Member States in December 2010. Highlights of the new legislation include:

  • establishment of a new scientific committee, the Pharmacovigilance Risk Assessment Committee (PRAC);
  • clarification of the roles and responsibilities of all actors involved in the monitoring of the safety and efficacy of medicines in Europe and strengthened coordination, leading to more robust and rapid EU decision-making;
  • engagement of patients and healthcare professionals in the regulatory process, including direct consumer reporting of suspected adverse drug events;
  • improved collection of key information on medicines, e.g. through risk-proportionate, mandatory post-authorisation safety and efficacy studies;
  • more transparency and better communication, including publication of agendas and minutes of the PRAC and the possibility to hold public hearings.

The Agency is ready for the first meeting of the PRAC on 19 and 20 July 2012. All Member States have nominated their members. The European Commission has appointed six independent scientific experts who will also serve as members. The nomination of PRAC members representing patients and healthcare professionals will follow a new public call for expressions of interest.

Information about the roles and the functioning of the PRAC has been published today and can be found on the Agency’s website, together with more information about the new pharmacovigilance legislation.

Notes
1. This press release, together with all related documents, is available on the Agency’s website.
2. More information on the new pharmacovigilance legislation is available here.
3. The status report ‘Countdown to July 2012: the Establishment and Functioning of the PRAC’ is available on the Agency’s website.
4. The new pharmacovigilance legislation is comprised of Directive 2010/84/EU and Regulation (EU) No 1235/2010.
5. More information on the work of the European Medicines Agency can be found on its website

EMA boosts EU transparency with online publication of suspected side effect reports

 

 

 

Member States and the Agency release data on medicines in compliance with EudraVigilance access policy

31/05/2012 – The European Medicines Agency has today begun publishing suspected side effect reports for medicines authorised in the European Economic Area (EEA) on a new public website: http://www.adrreports.eu. The reports come directly from the European Union (EU) medicines safety database EudraVigilance, and are one of the many types of data used by regulators to monitor the benefits and risks of a medicine once authorised. The launch of the new website is part of the Agency’s continuing efforts to ensure EU regulatory processes are transparent and open and is a key step in the implementation of the EudraVigilance access policy.

The information published today relates to approximately 650 medicines and active substances authorised through the centralised procedure, which is managed by the Agency. Information on the website is presented in the form of a single report per medicine or active substance. Each report pulls together the total number of individual suspected side effect reports submitted to EudraVigilance by Member States and marketing-authorisation holders. These aggregated data can be viewed by age group, sex, type of suspected side effect and by outcome. Within a year the Agency aims to additionally publish suspected side effect reports for common drug substances used in nationally authorised medicines.

A side effect (also known as an adverse drug reaction) includes side effects arising from use of a medicine within the terms of the marketing authorisation as well as from use outside the terms of the marketing authorisation, including overdose, misuse, abuse and medication errors, and those associated with occupational exposure.

All information on the website relates to suspected side effects. Suspected side effects may not be related to or caused by the medicine, and as a result, the published information cannot be used to determine the likelihood of experiencing a side effect or as an indication that a medicine is harmful. All users of the website are asked to read and accept a disclaimer explaining how to understand the information before they view a web report.

Medicines are an important part of modern healthcare, providing effective treatments for many diseases and conditions. For a medicine to be authorised for use in the EU the benefits of the medicine must always outweigh the risks.

Today’s launch also highlights the importance of side effect reporting and pharmacovigilance in safeguarding public health within the EU. Side-effect reporting is a key element in ensuring the detection of new or changing safety issues, and the Agency continues to further strengthen its work with partners and stakeholders across Europe to ensure a robust system for safety signal detection.

In June, the Agency will launch the website in the remaining 22 official EU languages.

Press release issued May 31st, 2012 on the EMA website – http://www.ema.europa.eu