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.

Addendum on estimands and sensitivity analysis to the guideline on statistical principles in clinical trials


The European Medicines Agency has released for public consultation following guidance document:

The E9(R1) addendum aims to clarify and extend ICH E9 by elaborating on the choice of estimand and sensitivity analysis in clinical trials, providing a framework to align its planning, design, conduct, analysis and interpretation. Having clarity in the trial objectives and accounting explicitly for intercurrent events when describing the treatment effect of interest at the planning stage of a clinical trial are crucial for a clear description of the effects of a medicine.

The proposed framework aims to facilitate dialogue for the disciplines involved at the planning level of the trial and also between sponsors and regulators regarding the treatment effects of interest that a clinical trial should address. The statistical analysis, aligned to the estimand, will be associated with assumptions and data limitations, the impact of which can be investigated through sensitivity analysis, whose definition and role are clarified in this addendum.

Deadline for comments will be 28 February 2018.

Please for more information please click on the link below:

European regulatory system for medicinesr brochure

EMA document

Advances in Stem Cell Therapy for Lung Diseases

University of North Carolina Health Care researchers have made strides toward a stem cell treatment for lung diseases such as pulmonary fibrosis, COPD, and cystic fibrosis.

In fact, they are discussing the start of clinical trials with regulatory authorities.

The team discussed its work in two recent studies. One proved that it is possible to isolate lung stem cells with a relatively non-invasive procedure. The other showed that stem cells reduce fibrosis in rats with pulmonary fibrosis.

“This is the first time anyone has generated potentially therapeutic lung stem cells from minimally invasive biopsy specimens,” Dr. Jason Lobo, director of the university’s lung transplant and interstitial lung disease program, said in a press release. He was co-senior author of both studies.

The research team had previously homed in on stem and support cells they could isolate from a lung tissue sample and grow in a lab. The tissue formed sphere-like structures in a lab dish, prompting the scientists to call them lung spheroid cells.

In 2015, the team showed that these cells had potent regenerative properties in animals with lung diseases. In fact, the stem cells they cultivated outperformed another type called mesenchymal stem cells.

For further information, please check the links below:

The EMA: Science and innovation for better medicines


The European Medicines Agency (EMA) ensures that medicines which are prescribed and used across the European Union (EU) are Safe, Effective and of Good Quality.All of them are carefully evaluated by the bests scientific experts through the European regulatory network assuring each new medicine it is recommended for authorisation if the benefits for the patients outweigh the risks of possible side effects.

The document created, entitled Enabling science that works for patients (at link below), try to explain  how The EMA promotes science and innovation in order to find better medicines, collaborates closely with patients to understand their point of view and to make sure that new medicines address their needs,   providing also a scientific advice and guidance to encourage the development of new and innovative medicines, especially in areas with limited treatment options such as rare diseases and illnesses in children.

EMA involves patients, consumers and their representative organisations in all decisions made during the lifecycle of a medicine in the:

  • Development of policies
  • Regulatory guidance
  • The evaluation and safety monitoring of medicines

To see the deatiled leaflet created by The EMA, pleach clink on the link below:

Enabling science that works for patients

A unique source of information on safety and effectiveness of authorised drugs is registered as EU trade mark

The EU PAS Register was launched in November 2010 as unique source of information on the safety and effectiveness of authorised medicines. This platform is openly and accessible which includes information on observational post-authorisation research in medicines already marketed in EuropePSNResearch

and some infor that includes is:

  • Study protocols
  • Study results
  • Related publications
Data from 31st f July 2017 – EU PAS Register Platform

All the information at the EU PAS Register helps to reduce publication bias through increased transparency of medicines research, improves the quality of post-authorisation studies by facilitating peer-review of protocols and results, promotes collaboration among stakeholders, and ensures compliance with EU pharmacovigilance legislation requirements.

 The platform is recommended in the following cases: scientific publications, guidelines and textbooks. Although initially the main aim of The EU PAS Register was to collect studies conducted in the European Union, researchers from outside the EU are also registering studies to increase transparency of their research.

The EU PAS Register was developed through the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP), which is coordinated by EMA to support research in pharmacoepidemiology and pharmacovigilance.

If you want to consult more info abour EU PAS Register Platform, please click on the link below:

EU PAS Register

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: