Latest orphan designations and/or marketing authorisations

Medicinal products intended for rare diseases can receive an “orphan drug” label based on a defined number of criteria as summarised below:

• The product is intended for an indication with a prevalence not exceeding 5 in 10 000 persons in the EU.
• The disease is life-threatening, seriously debilitating or a serious and chronic condition.
• No satisfactory method of diagnosis, prevention or treatment of the condition is already authorised in the EU. If any method exists, then the medicinal product has to demonstrate that it provides a significant benefit compared to the product for which orphan designation is being requested.

Committee for Orphan Medicinal Products

A positive opinion on orphan designation is given by the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) based in London with the final decision made by the European Commission.

The COMP was created as part of the EU Regulation on Orphan Medicinal Products to review EU applications for the label "orphan drug”.

Orphan designation at any stage of development

Orphan designation is possible at any stage of drug development as long as proper scientific justification of the medical plausibility of the product in the indication requested is demonstrated.

The research may be pre-clinical (not yet tested on human subjects) or may have reached the human clinical trial phase.

Orphan designation does not indicate an endorsement for the use of the drug for the designated condition. Efficacy, safety and quality criteria first need to be satisfied for the granting of a marketing authorisation.

In addition to the position of Vice-Chair, EURORDIS has held two of the three seats assigned to patient organisation representatives on the COMP since its creation in 2000.

Detailed information on European orphan drug designation applications is available on theEMA website

A full list of designated and authorised orphan drugs in Europe available at: ec.europa.eu

6 June 2012

Treatment of meningioma

N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide

Treatment of schwannoma

N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide

Treatment of Wiskott-Aldrich syndrome

Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene

Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity

Letermovir

Treatment of pancreatic cancer

Polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine

Treatment of ovarian cancer

1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl) urea

Treatment of glioma

Adenovirus-associated vector containing human Fas-c gene

Treatment of adrenoleukodystrophy

Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA

25 May 2012

Treatment of soft tissue sarcoma

Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10

Source

EURORDIS

Online access to suspected side-effect reports

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: 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.

Notes

• The European database of suspected adverse drug reaction reports:http://www.adrreports.eu
• Patients, consumers and healthcare professionals report suspected side effects to either the national medicines regulatory authority or thepharmaceutical company that holds the marketing authorisation for the medicine. These reports are then transmitted electronically toEudraVigilance. The Agency, on behalf of European Member States, is responsible for the development, maintenance and coordination of EudraVigilance.
• Pharmaceutical companies that hold the marketing authorisation for a medicine in the European Economic Area (EEA) are also legally required to submit to EudraVigilance all reports of suspected unexpected adverse reactions that are serious and that occurred in a third country (non-EEA) where they hold a marketing authorisation. The web reports present information on suspected side effects that have occurred in both the EEA and outside the EEA.  
• A web report does not include reports from studies (e.g. clinical trials and non-interventional studies) or other types of reports, i.e. they only include spontaneous reports.
• Web reports can only be viewed with Adobe Reader 10.x and Adobe FlashPlayer 10.2.
• Frequently asked questions and a short guide on how to interpret web reports can be found online.
• The Management Board of the European Medicines Agency approved the EudraVigilance access policy in December 2010. The access policy describes how stakeholders, such as national medicines regulatory authorities in EEA countries, healthcare professionals, patients and consumers, as well as marketing authorisation holders and research organisations, can access information on suspected side effects submitted electronically to EudraVigilance.
• More information on the 2010 pharmacovigilance legislation can be found on the Agency’s website.

Source

http://www.adrreports.eu/
EMA

EAHP Academy Seminar "What do we need to know about building or reconstructing a hospital pharmacy?"

 
Target group of the seminar
The target group comprises hospital pharmacy managers, mainly head of pharmacies or deputies from the new EU- Countries and new members of EAHP.

Teaching goals
To ensure patient safety hospital pharmacists have to ensure:

• the correct medical supply including purchasing, stock- keeping and delivery of drugs
• the compliance with pharmaceutical and drug-related legal regulations as well as with all other laws and regulations related to pharmacies
• the compliance with the general rules of hygiene and of the science of pharmacy
• the quality assured production and compounding of medicines
• pharmaceutical counselling like drug information and clinical pharmacy services.

EU- legislation on production of medicines in hospital pharmacies like GMP guidelines and influences the technical and interior equipment of the production areas. Recent developments of logistic of drugs were characterised by the improvement of the supply chain using automation and track and trace.
A huge number of hospital pharmacies are more then 15 years old and do not fulfil the state of technology and science. Hospitals therefore are forced to reconstruct or rebuild the hospital pharmacy.
The seminar should give support in planning or replanning the hospital pharmacy.

Learning objectives

Seminar I: Planning of a state of the art hospital pharmacy production

The seminar gives theoretical information about regulatory requirements for hospital pharmacy production. This includes process description, quality risk management, quality management, feasibility study and user requirements to design qualification. In the following workshops the participants will work on the above mentioned topics on practical examples.
At the end of the seminar the participants will:

• have the skills to identify the relevant requirements for the hospital pharmacy they are working for.

Seminar II:Planning the logistic of a hospital pharmacy

This seminar gives theoretical information about logistics of hospital pharmacies and examples of the recent developments in the supply chain of drugs.
At the end of the seminar the participants will:

• get an overview about actual developments in the supply of medicines
• know about advantages and disadvantages of automation systems for picking packages
• know about advantages and disadvantages of unit dose systems
• know about track and trace during transport and delivery including the use of RFID
• know about the latest developments in barcoding
• be able to take in consideration the different developments either material or administrative and adapt to the hospital and hospital pharmacy needs.

 Speakers' biographies

View the bios of presenting speakers for the Academy seminar 2012.

2012 Academy seminar programme

View the detailed programme schedule.

The city of Thessaloniki

Some useful information on the most interesting places to visit in Thessaloniki.

10/01/2012 

 

Registration and hotel reservation

Welcome participants to the 2012 EAHP Academy Seminar!

Venue and public transportation

View the Academy Seminar's venue and how to reach the hotel by public transportation

Source 

EAHP

EU: PONTE platform prototype to be released soon

A prototype of the 'Efficient Patient Recruitment for Innovative Clinical Trials of Existing Drugs to other Indications' platform (PONTE platform) will be released this month (April 2012). The wider research community will thus have the opportunity to test run and review the tools that the platform will offer.


PONTE is a European project intended to establish a platform to help new uses for existing drugs. It will offer vital assistance throughout each stage of the process - from the moment an idea is generated through to the selection of suitable volunteers for the trial.

Once work on refining and perfecting the system is complete, PONTE will act as a one-stop search, query and decision support tool. By linking to existing medical knowledge databases, it will give near-instant access to information about any disease, drug or drug properties. This will aid researchers as they develop their hypotheses for new applications for existing medicines. If, for example, there is the possibility that a particular drug for high blood pressure may be useful in treating arthritis, PONTE will help to uncover it. The platform will then utilise data stored by individual hospitals to establish whether sufficient patients meet the criteria for a trial to be able to proceed. PONTE can select pre-consenting volunteers immediately - ensuring the right people for the right trial at the right time. To complete the jigsaw, the system will provide a step-by-step guide for setting up clinical protocol, in turn ensuring all aspects have been completed well in advance of the start of the trial.

PONTE will simplify the whole clinical trials process so that it will be easier to come up with the right concept and select the right patients. As well as saving money, this will give the public quicker access to drugs which may be able to help them. The platform is expected to be launched in a year's time.

Further information: 

• Source - PONTE Project
• PONTE Project website  

Source

ePractice

European Commission establishes new eHealth Stakeholder Group

European Commission establishes new eHealth Stakeholder Group

Following the recent call for expression of interest (closed in January 2012), the European Commission has selected the members to participate in its new eHealth Stakeholder Group. Members of the group, appointed for a period of three years, are expert representatives of European umbrella organisations active in the eHealth sector.

The group is expected to contribute to the development of legislation and policy related to eHealth. Policy areas of specific interest include relevant actions of theDigital Agenda for Europe on eHealth and the forthcoming eHealth Action Plan.

Complete list of the 29 selected member organisations, representatives and substitutes (pdf)

Source

http://ec.europa.eu/information_society/activities/health/policy/stakeholders_group/index_en.htm

New 24-hour drug robot at Worthing Hospital, UK

WORTHING Hospital has employed a new worker who never sleeps.

The newest addition to the pharmacy department at Worthing Hospital is a drug-dispensing robot, which works 24 hours a day to give staff instant access to 1,700 different medicines around the clock.

While the department has had a pharmacy robot for the last four years, the development means the majority of drugs can now be dispensed to ward staff at all times, compared to just 200 of the most common drugs. The new machine will also increase the reliability and accuracy of dispensing the drugs.

Saffron Mawby, head of medicines management at Western Sussex Hospitals NHS Trust, said:

“This latest development, giving patients those benefits around the clock, is a significant improvement to the service we can provide. Having key medicines available when they are needed is an important part of our patients’ clinical care and this new technology will help our in-patients get the medicines they need, faster than ever before, no matter what the time.”

The £20,000 system was bought by the Friends of Worthing Hospital, and will mean the trust will save on the expense of on-call pharmacy staff having to travel to hospital out of hours.

Lucy Shade, senior pharmacist at Worthing Hospital, said:

“The machine brings real benefits in terms of speed, safety, and releasing pharmacy staff to spend more time on the wards during the day, seeing patients. This new improvement to our out-of-hours service will significantly improve the service to our in-patients at night and at weekends.”

Source

http://www.shorehamherald.co.uk/