The EMA Agreed on principles for involving young people under 18 in their activities

New principles give a voice to patients, consumers and carers under age 18 can make an important contribution to EMA’s committee discussions on medicines by sharing their experience and perspectives of living with a disease or condition.

These principles set out best practice for the interaction between parties on:

  • Discussion in small groups
  • Provision of specific support
  • Use of appropriate language
  • Obtaining parental consent
  • Protection of personal data
  • The privacy of the young patients.


The key forum in which young people could participate in the Agency’s activities would be its Paediatric Committee (PDCO), but experience has demonstrated that the Committee for Medicinal Products for Human Use (CHMP), the Pharmacovigilance Risk Assessment Committee (PRAC) and the Scientific Advice Working Party (SAWP) can also benefit from this input when these groups review medicines for children.

For more information about this notice, please check the links below:

Involving young people in EMA activities



FDA Panel Recommends Approval for the First Gene Therapy in Leukemia Treatment

A FDA panel opened a new era in medicine unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer, transforming them into what scientists call “a living drug” that powerfully bolsters the immune system to shut down the disease.

The treatment will be The First Gene Therapy ever to reach the market throw FDA approval. 

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.

In 2012, as a 6-year-old patient was treated in a study at the Children’s Hospital of Philadelphia. Severe side effects — raging fever, crashing blood pressure, lung congestion — nearly killed her. But she emerged cancer free, and has remained so. 

Her father said to FDA comitte“I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”

The main evidence  presented by the sponsor to the FDA came from a study of 63 patients who received the treatment from April 2015 to August 2016:

  • 52 of them, (82.5%) went into remission — a high rate for such a severe disease.
  • 11 of them died

As NY times sahred on their interview: “It’s a new world, an exciting therapy,” said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment. The next step,  will be to determine “what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight. This is the beginning of something big.”

FDA takes steps in Drug Competition to Improve Patient Access

The U.S. Food and Drug Administration (FDA), is taking two new important steps to increase competition in the market for prescription drugs, and facilitate entry of lower-cost alternatives.

The agency published a list of off-patent, off-exclusivity branded drugs without approved generics, and also implemented, for the first time, a new policy to expedite the review of generic drug applications where competition is limited.

To encourage generic drug development, the FDA posted a list of branded drugs that have no listed patents or exclusivities and for which the agency has yet to approve a generic drug application (known as an Abbreviated New Drug Application or ANDA). The agency also intends to expedite the review of any generic drug application for a product on this list to ensure that they come to market as expeditiously as possible.

The FDA is also announcing a change to its policy on how the agency prioritizes its review of generic drug applications. The FDA will expedite the review of generic drug applications until there are three approved generics for a given drug product. The agency is revising the policy based on data that indicate that consumers see significant price reductions when there are multiple FDA-approved generics available.

These actions follow closely the FDA’s announcement of a public meeting to be held on July 18, 2017, to solicit input on places where the FDA’s rules – including the standards and procedures related to generic drug approvals – are being used in ways that may create obstacles to generic access, instead of ensuring the vigorous competition Congress intended.

These are the first of a series of steps the agency intends to take to help tackle this important issue. The agency will unveil additional aspects of this plan in the near future and will continue to communicate with the public as additional elements of this plan are implemented. These actions reflect the administration’s broader work to improve access to prescription drugs.


New Platform for parallel consultation will provide advice to medicine developers and facilitate access to medicines for patients


The European Medicines Agency (EMA) and the European Network for Health Technology Assessment (EUnetHTA) are stepping up their efforts to provide developers of medicines with simultaneous, coordinated advice on their development plans and facilitate alignment of data requirements.

This new initiative replaces the existing tool for parallel scientific advice by EMA and HTA bodies which required medicine developers to contact Member States’ HTA bodies individually. It also builds on previous initiatives and pilots on HTA-regulatory collaboration led by EMA, EUnetHTA and the European CommissionExternal link icon (see notes).

Medicines developers will need to notify simultaneously EMA and EUnetHTA of their intention to request parallel advice. EUnetHTA’s Early Dialogue Secretariat, recently created to facilitate such consultations, will then coordinate the involvement of the HTA bodies that will take part in the parallel advice, taking into account the preferences of the requester.

EUnetHTA created the Early Dialogues Working Party (EDWP), composed of HTA bodies with demonstrated experience in early dialogues/scientific advice, to ensure high-quality advice and consistency over time.

The main benefits of this new platform will be:

  • increased mutual understanding and problem solving ability through a more structured interaction between EMA and HTA bodies;
  • improved coordination with, and greater participation of HTA bodies, as a result of the creation of an Early Dialogue Working Party and an Early Dialogue Secretariat at EUnetHTA;
  • streamlined logistics for the requesters.

These advantages are expected to lead to more robust outcomes resulting from the parallel consultation on evidence-generation plans for pharmaceuticals.


For more information, just click at the following link:
EMA – interaction to align data requirements


PSN Research attended the 18th EULAR Congress of Reumathology celebrated in Madrid last 14th to17th of June




Rafael Zurita (Genaral Director) and Alain Baleydier (France office Director) from PSN Research, attended the 18th EULAR Congress 2017 14-17 of June  in Madrid. 

This is one of the most relevant event during the year for PSN Research as Reumathology is one of the most important Areas of Expertise for PSN RESEARCH 

The Congress, offered a wide range of topics including:

  • Clinical Innovations

  • Clinical Translational Research

  • Basic Science


In addition, PSN Research attended at EULAR 2017 meetings organised by People with Arthritis and Rheumatism in Europe (PARE), by Health Professionals in Rheumatology (HPR), and by the Healthcare Industry. 


EULAR congress have been received tremendous interest

in terms of participation and quality of contributions





It received more than 4.850 abstract submissions, which is by far the highest ever at a EULAR congress. A total of 347 were accepted as oral presentations this year, and the congress features 180 sessions and poster tours with 355 speakers. Out of the 2,336 poster displays spread over 3 days, 461 posters will be explained in 45 themed poster tours.

These numbers reflect the availability of increased information on the impact, burden, and cost of these diseases for society and a significantly improved ability to diagnose and treat them.

For more information about the EULAR Congress please click the links below:



Pulmonary Arterial Hypertension (PAH): Workshop on 12th June co-organised by The EMA for Children with PAH

Pulmonary Arterial Hypertension is a disorder of the blood vessels leading to the lungs, in which the blood pressure in the pulmonary artery becomes high. PAH is a serious, debilitating disease that affects both children and adults but the condition is rare in children and the signs and symptoms often differ from those seen in adults.

The European Medicines Agency (EMA), the United States Food and Drug Administration (FDA) and Health Canada are co-organising a workshop to discuss the requirements for the development of medicines for pulmonary arterial hypertension (PAH) that address the high unmet medical needs of children.

The workshop will bring together leading experts and stakeholders in PAH across the globe:


The objectives of the event are to:

  1. Analyse the problems related to the conduct of clinical trials in children with PAH,
  2. Refine endpoints and study design to address the challenges identified,
  3. Set priorities for future research in specific medicine development aspects,
  4. Provide medicine developers with more guidance specific to global product development, taking into account current limitations 

For further information about this Workshop. please click at the links below:




The EMA publish a New Action Plan to support SMEs in pharmaceutical innovation



The European Medicines Agency (EMA) has published today an action plan for small and medium-sized enterprises (SMEs), which aims to foster innovation and support SMEs in the development of novel human and veterinary medicines.

SMEs are the backbone of Europe’s economy, representing:

  • The 99% of all businesses in the European Union (EU) 
  • SMEs are providing 2/3 of total private sector employment
  • SMEs are a motor of innovation and play a major role in the development of new medicines.

To support SMEs throughout all stages of medicine development, EMA’s SME Office provides active regulatory, financial and administrative support to registered SMEs. More than 1,700 companies are currently registered with the Agency operating in the human and veterinary fields.


The plan covers four key areas and lists a series of new and enhanced actions:

1. Awareness of EMA’s SME initiative

2. Training and education

3. Support the development of innovative medicines

4. Engagement with SMEs, EU partners and stakeholders

EMA has also published today its SME office 2016 annual report providing an overview of SME-related activities in 2016 and highlights incentives and platforms that SMEs can leverage to advance innovative developments and regulatory strategies.

Please if you want to consult the original documents just click on the links below:

Framework for collaboration with academia

Europe’s next leaders: the Start-up and Scale-up Initiative

SME office 2016 annual report

10th Anniversary of the SME initiative

Approval for New EudraVigilance system for collection and monitoring of suspected Adverse Reactions

The European Medicines Agency (EMA) will launch a new and improved version of EudraVigilance, the European information system of suspected adverse reactions to medicines that are authorised or being studied in clinical trials in the European Economic Area (EEA). The new version of EudraVigilance will go live on 22 November 2017 (announcement of the EMA Management Board) with enhanced functionalities for reporting and analysing suspected adverse reactions.

The enhancements for reporting and analysing suspected adverse reactions of the new EudraVigilance system will support better safety monitoring of medicines and a more efficient reporting process for stakeholders.

Expected benefits include:

  1. Simplified reporting of individual case safety reports (ICSRs) and the re-routing of ICSRs to Member States as marketing authorisation holders will no longer have to provide these reports to national competent authorities, but directly to EudraVigilance, which will ultimately reduce duplication of efforts. An ICSR provides information on an individual case of a suspected adverse reaction to a medicine;
  2. Better detection of new or changing safety issues, enabling rapid action to protect public health;
  3. Increased transparency based on broader access to reports of suspected adverse reactions by healthcare professionals and general public via the portal, the public interface of the EudraVigilance database;
  4. Enhanced search and more efficient data analysis capabilities;
  5. Increased system capacity and performance to support large volumes of users and reports (including non-serious adverse reactions originating from the EEA);
  6. More efficient collaboration with the World Health Organization (WHO) as EMA will make the reports of individual cases of suspected adverse reactions within the EEA available to the WHO Uppsala Monitoring Centre (UMC) directly from EudraVigilance; Member States will no longer need to carry out this task.


The Agency will support national competent authorities, marketing authorisation holders and sponsors of clinical trials in the EEA through targeted e-learning and face-to-face trainings, webinars and information days. Users can trial the new functions of the EudraVigilance system and the internationally agreed format for ICSRs in a test environment as of 26 June 2017. Further information is available on the EudraVigilance training and support webpage.

If you want more info, just click on the links below:


EudraVigilance training and support webpage

Clinical Trial Regulation

Announcement of the EMA Management Board

The European Medicines Agency (EMA) has published its 2016 Annual Report

The European Medicines Agency (EMA) has published its 2016 Annual Report. The report focuses on the Agency’s key achievements in the areas of medicine evaluation, support to research and development of new and innovative treatments and the safety monitoring of medicines in real life.

In 2016, the Agency recommended a marketing authorisation for:

  • 81 medicines for human use, including 27 new active substances
  • 11 medicines from the veterinary side were recommended for approval, including six new active substances

As a result of the safety monitoring of all medicines marketed in the European Union (EU), the product information for over 300 medicines for human use was updated on the basis of new safety data.

This document also contains three interviews with stakeholders and EMA representatives on topics of major interest :

Vaccine hesitancy – a threat to public health;

Creating an agile organisation for the 21st century;

How to reinforce surveillance of antimicrobial consumption.

The last part of the report provides a large amount of data and figures that illustrate the work of EMA and its impact.

Please click on the following links for more info:


The EMA and the European Commission have published a New Guide on Biosimilar medicines

The guide was launched last  5th  of May of 2017 at the European Commission’s third stakeholder event on biosimilar medicines, a discussion forum that provides a platform for stakeholders interested in biosimilars, including healthcare professionals, patients, payers, regulators and industry. The EU has pioneered the regulation of biosimilar medicines by establishing a solid framework for their approval and by shaping biosimilar development globally.

The main objective of this document is to increase, even more, the understanding of Biosimilar medicines by Healthcare proffesionals and also by all the stake holders involve as it was previously requested by Organisations from across the EU representing: doctors, nurses, pharmacists and patients have also shared useful views, to ensure that the guide adequately addresses questions relevant to healthcare professionals.

Dr Juan Garcia Burgos, Head of EMA’s Public Engagement Department, was in charge of the public presentation of the guide at launch, referring to the guide as: “this comprehensive reference material is a joint effort to support information and continuous education of healthcare professionals in the EU, and facilitate dialogue with patients.”


If you are interesting to check more information or download the Guide, please click on the links below: