The European Medicines Agency (EMA) was founded in 1995, and since then has worked across the EU and globally to protect public and animal health by assessing medicines and by providing independent, science based information on medicines. In 2000, patient representatives joined for the first time as members of one of EMA’s scientific committees. Today, their representatives take part in most of EMA’s scientific committees as full members, adding their unique perspective and experiences to discussions. They play an increasingly important role in the assessment of the risks and benefits of medicines.

The EMA is one of the central topics you can learn about at EURORDIS summer school. Claudia Finis, a psychologist from Germany, attended EURORDIS summer school on behalf of OIFE in 2019. Read her experience and report here:

“Indeed EURORDIS summer school was a school. We had to do a lot of work. Prior to the actual week of face-to-face training we had to read and work on real and invented articles as well as to do online training. The latter consists of several online training modules and is available online for free for anyone to use. So feel free to try it.

We learned a lot about pharmaceutical trials, medical research and how to assess it. Furthermore, we learned how the responsible people come to their decision whether they authorize a drug or not. One way for patient engagement is to work together with the EMA.

But what is the EMA?
The European Medicines Agency (EMA) is an agency of the European Union (EU) responsible for the scientific evaluation, supervision and safety monitoring of medicines in the EU. The mission of the EMA is to foster scientific excellence in the evaluation and supervision of medicines, for the benefit of public and animal health in the EU.

And why is that important for us?
Because the EMA is responsible for the orphan designation and marketing authorisation. It is responsible for the scientific evaluation of marketing authorisation applications for all orphan medicines in the European Economic Area, as they fall under the mandatory scope of the centralised procedure.

What are orphan drugs?
Orphan drugs are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare. A disease or disorder is defined as rare in Europe when it affects less than 1 in 2,000 citizens. These drugs are called “orphan” because under normal market conditions the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients. Pharmaceutical companies can apply for an orphan drug authorization and when granted they will get several benefits. But it’s a long way from the idea to the available medicine. And the work of the EMA goes even further. After authorization the drug is still monitored for several reasons e.g. side effects or long term reactions.

Reporting adverse effects is important
By the way, every patient is allowed or even encouraged to report about adverse effects to the EMA. This means drugs in off-label use, too. This is important and might lead to consequences such as the withdrawal of a drug if needed.

Where and when should patients contribute?
In order to prove that a new drug is safe and efficacious a lot of research has to be done, valid and meaningful data has to be collected. We as patients can contribute along the way. In fact, researchers can be surprisingly ignorant of what really matters to us. For example, a drug that causes measurable changes anywhere in the body but does not improve the issues that are important to us can be completely uninteresting to us and nobody would take it. That would be a waste of time and money for the pharmaceutical company while disappointing for us. So everyone will benefit from good collaboration. There are many opportunities to participate along the way. This graphic gives a brief impression.

Every orange speech bubble means a chance to get involved. In addition patients can give valuable input at a community advisory board (CAB), which is a group of patients who offer their expertise to several public or private sponsors of clinical research.

Research on children is getting more and more important.
In the past, research was mostly done on adult men. That is why there are far less drugs for children. Many drugs are nevertheless used for children, some as off-label use. With varying degrees of success. Nowadays the EMA tries to test new medicines as far as possible and necessary also for children. This is important because children often react differently. They have a different metabolism, they are still growing and they have different needs. But of course they are not able to participate in a committee. So the Paediatric Committee is probably the only one where not the patients themselves participate but their parents.

Which questions arise when one participates in a committee?
There may be ethical questions and decisions to be made and sometimes the trial has to be modified in order to get valid data. In other cases the research conditions may be so stressful for the participants that a high cancellation rate is to be expected. It looks like sometimes researchers think that it is no problem to attend the trial. But for the family this may mean to drive four times a week many kilometers after school (and work), wait at the hospital for one hour, get treated or examined for ten minutes, drive back all the way but they have to take care to arrive at the hospital before 3 p.m. with a child with special needs. The researchers often don’t take into account that people with a rare disease live far away from the performing university/ hospital and due to their disability they are not able to bare this stress. In addition, the parents may get problems with their employers. No wonder that such trials have a high dropout rate. Here again we can prevent a waste of time and money with our input. What are we willing to do and endure? What is possible, what is not? These researchers have sometimes never seen or talked to a patient with the disease or disability they are researching. They don’t know what it means to live with this disease or disability.

Besides the dry and important work, we also could chat, get to know nice people and do some networking, which of course is also important. And there are these encounters and moments that just delight the heart. I highly recommend the summer school for patient advocates especially for those who are willing to work on a European level.”

Written by Claudia Finis