An overview of the EU’s orphan designation programme

The Agency is responsible for reviewing applications from sponsors for orphan designation. To qualify for orphan designation, a medicine must meet a number of criteria:

  • It must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

The EU’s orphan designation programme was launched in the year 2000 to encourage companies to research and develop medicines for rare diseases.

To date, over 1,900 medicines have been designated as orphan medicines, giving access to specific incentives that make it more attractive for companies to develop them. By the end of 2017 over 140 of these medicines were on the market, providing treatment options for patients who previously often had none.

The new factsheet published by European Medicines Agency today explains what a rare disease is, how the EU programme works and what incentives are made available to developers.

FDA clears stereotactic radiotherapy system for use in treating breast cancer

Radiation therapy is an important treatment option for cancer patients.

Approximately 60 percent of all cancer patients will be treated with some form of radiation therapy. During radiation therapy, tumor cells are killed when their DNA is damaged by the radiation being absorbed into them. While radiation therapy has the potential to kill tumor cells, it can also damage healthy tissue around the tumor.

The GammaPod system is intended for use in the non-invasive stereotactic delivery of a radiation dose to a portion (partial volume) of the breast in conjunction with breast conserving treatment. During the procedure, radiation is delivered to specific areas of the breast. The GammaPod has not been shown to be as effective as whole breast radiation therapy (WBRT) and is not intended to replace WBRT.

The GammaPod system is a dedicated stereotactic radiation therapy technology designed to treat breast cancer. GammaPod uses thousands of focused beams of radiation from 36 rotating radioactive Cobalt-60 sources in combination with a two-layer, vacuum-assisted cup that immobilizes the breast to achieve a more accurate delivery of radiation. The GammaPod design to immobilize the breast during treatment provides the benefit of minimizing the radiation dose to the surrounding healthy tissues in the breast, heart and lungs.

The FDA reviewed scientific evidence including a clinical study of 17 patients that tested the feasibility of accurately delivering the prescribed dose to the breast tumor while minimizing radiation to the healthy tissue. The clinical evidence supports delivering the prescribed dose to the breast tumor with minimal radiation-induced side effects such as skin redness or erythema.


Merry Christmas from PSN Research Team!!!

The EMA: Brexit update, Annual work programme and Budget for 2018


How is the current situation with The EMA?

This is the first meeting of the Board since the General Affairs Council (Art.50) of 20 November and the decision of the EU 27 Member States to relocate the Agency to Amsterdam. EMA now has just over 15 months to prepare for the move and take up its new seat in Amsterdam by 30 March 2019 at the latest.

EMA’s collaboration with the Netherlands commenced promptly and agreement has been reached on the joint governance structure with plans to progress activities within five work streams relating to the temporary and permanent premises, staff relocation, financial and legal aspects, and external communication.

The EU27 Member States and EMA have developed a methodology for the redistribution of the work currently carried out by the UK’s Medicines & Healthcare products Regulatory Agency (MHRA) and Veterinary Medicines Directorate (VMD). The joint redistribution plan reflects the strengthened capacity of the European medicines regulatory network. The risk-based methodology takes into account the diverse expertise in the network and the workload associated with the medicines. EMA will communicate details of the methodology and next steps in early 2018.

What will be the 2018 work programme and budget for the EMA?

The EMA is preparing the UK’s withdrawal from the EU an what will be the impact on the activities of the Agency. This became clear in the discussion on the Agency’s work programme and the budget for 2018-2019.

The Board adopted a budget of 337 million euros for 2018, a 2% increase over the previous year, driven primarily by an increase in fee-generating activities. The budget for 2018 includes provisions for Brexit-related costs such as IT-related relocation expenses and costs of the physical relocation of the Agency staff and assets. As the relocation process evolves, the budget will need to be monitored carefully and any additional costs that cannot be absorbed should be discussed with the European Commission.

How is going the development of the EU clinical trial portal and database for the EU Clinical Trial Regulation?

The development of the EU Portal and Database is making important progress. A partially-completed version of the system has been subjected to user acceptance testing by representatives of the European Commission, Member States, academia, pharmaceutical industry and contract research organisation (CRO) associations throughout November, while technical testing and further development continue in parallel.

This is the most ambitious IT development project so far required by the EU pharmaceutical legislation. The Agency is working hard in collaboration with Member States, stakeholders and the developers to deliver a good, functional system to support the needs of EU clinical research, and to have the Clinical Trial Regulation enter in operation as soon as practicable.

The EMA relocated to Amsterdam (Netherlands)

On 29 March 2017, the United Kingdom (UK) notified the European Council of its intention to withdraw from the European Union (EU), a process known as ‘Brexit’. EMA is making preparations to ensure that the Agency can continue to deliver on its mission and protect public and animal health after the UK leaves the EU on 30 March 2019, the date currently set by the timeframe provided in Article 50 of the Treaty on European Union.

The EU 27 Member States took this decision on 20 November in the margins of the General Affairs Council (Article 50) of the European Council

The Agency will begin working immediately with the Dutch government to prepare for the move and take up its operations in Amsterdam on 30 March 2019 at the latest.

EMA and the Netherlands will establish a joint governance structure to steer and oversee the relocation project.

In early December 2017, the Agency will publish a monitoring chart on its website that will allow stakeholders to track progress.

Amsterdam was one of 19 offers to host EMA submitted by the Member States at the end of July 2017. For more information, see Background to relocation decision.

New guidelines on good manufacturing practices for advanced therapies

The European Commission has published a set of guidelines on good manufacturing practice (GMP) specific to advanced therapy medicinal products (ATMPs).

ATMPs are medicines for human use that are based on genes or cells. These therapies offer ground-breaking new opportunities for the treatment of diseases and injuries. They are particularly important for severe, untreatable or chronic diseases for which conventional approaches have proven to be inadequate.

The new guidelines adapt the European Union GMP requirements to the specific characteristics of ATMPs and address the novel and complex manufacturing scenarios utilised for these products. The guidelines foster a risk based approach to manufacture and testing of such products.

The guidelines ensure that these novel medicinal productsare consistently produced and controlled according to high quality standards, for the benefit and the safety of patients. This initiative is part of the joint action plan launched by the Directorate General for Health and Food Safety (DG SANTE) and the European Medicines Agency (EMA) in October 2017 to foster the development of ATMPs.

The European Commission drafted these guidelines with extensive input from the Agency’s Committee for Advanced Therapies (CAT) and GMP/Good distribution parctice (GDP) inspectors working group (GMDP IWG), together with national competent authorities and other external stakeholders. The adaptations introduced in the GMP framework for ATMPs will continue ensuring a high level of quality for ATMPs and a high level of patient protection.

Go-live planning of the new EudraVigilance system

The EudraVigilance System is going to be scheduled for downtime from 8th  to 21st of  November 2017.

The Agency will be releasing the new EudraVigilance system on 22th of November. This follows the EMA Management Board announcement in May that the EudraVigilance (Human) system has achieved full functionality. A number of planned tasks and activities are necessary to prepare for the release of the new EudraVigilance system, requiring a downtime period of 10 business days from 8 to 21 November. The scheduled downtime will affect a number of key EudraVigilance functionalities and several other IT systems and business processes.

For more information on the impacted IT systems, including EudraVigilance, business processes and the alternative arrangements, please click here or on the link below:

EudraVigilance System downtime

PSN Research at 25th UEG Week Barcelona 2017

Taking place for the 25th time, UEG Week is the largest and most prestigious GI meeting in Europe and is now a global congress. UEG Week attracts around 14,000 participants each year, from around the world.

UEG, or United European Gastroenterology Week is an opportunity to present new research and thinking across a wide range of digestive disease areas, cutting-edge post-graduate teaching sessions, some of the best GI abstracts and posters and simultaneous live streams to a global audience and endoscopic, ultrasound and surgical hands-on training.

The focus for the 25th UEG Week 2017 in Barcelona will be to advance science and link people in the global GI community. UEG’s mission is to continuously improve standards of care in gastroenterology, and promote ever greater understanding of digestive and liver disease – among the public and medical experts alike.


For further information please clink the link below:


Sermes CRO a PSN Research Company is a Tester of the New EMA´s EU Portal (UATs)

The EMA (European Medicines Agency), retakes the development of the new portal for the online presentation of clinical trials at European level (UATs European portal).
This October 2017 will take place the next step, “test the new portal by a group of experts” carefully selected by EMA itself. In total, the EMA has entrusted 16 European representatives with the task of testing “onsite” the new Portal (UATs) through on-site E2E scenarios, in a face-to-face manner in their offices in London.

EUCROF (European CRO Federation), has determined that Sermes CRO a PSN Research Company, represented by their Head of Unit of start-up (UPM), Lidya Dominguez, will be person chosen to go to the offices of the EMA and to carry out the “on-site” UATs test to analyze possible errors and To bring new ideas of improvement to implant in the Portal.

For more information, click on the following links:

Presentation of Eu Portal and Database project

The EMA presents the EU regulatory system

The European Medicines Agency (EMA)  hosted an awareness session to present the European Union’s (EU) medicines regulatory system and EMA’s role in it to international regulators and non-governmental organisations.

All the speakers were part of the Agency and will present on the European medicines regulatory network and the various activities carried out by EMA as part of it. The issue will be to show a clear understanding of how medicines are regulated in different parts of the word is of prime importance in an increasingly globalised world where regulators rely on close cooperation.

Topics include:

  • European Union (EU) marketing authorisation procedures
  • Support to innovation
  • Benefit/risk assessment of new medicines
  • Pharmacovigilance activities
  • Stakeholder engagement

The European medicines regulatory system is based on a network of around 50 regulatory authorities from the 31 EEA countries (28 EU Member States plus Iceland, Liechtenstein and Norway), the European Commission and EMA.

This network is what makes the EU regulatory system unique. The network is supported by a pool of thousands of experts drawn from across Europe, allowing it to source the best possible scientific expertise for the regulation of medicines in the EU and to provide scientific advice of the highest quality.

EMA and the Member States cooperate and share expertise in the assessment of new medicines and of new safety information. They also rely on each other for exchange of information in the regulation of medicine, for example regarding the reporting of side effects of medicines, the oversight of clinical trials and the conduct of inspections of medicines’ manufacturers. This works because EU legislation requires that each Member State operates to the same rules and requirements regarding the authorisation and monitoring of medicines.

For more information, please see the programme.