EMA – institution of changes

Year 2015 is exceptional in the history of the European Medicines Agency (EMA). This year marks the 20th anniversary of the creation of the Agency as an institution responsible for medicines authorisation for the entire European Union. At first the Agency gathered representatives from the scientific world of medicine and pharmacology from each EU Member State working in specialised committees of experts on medicines authorisation in Europe and creating legislation that enables for general rules of medicines marketing at European level. But through these twenty years of existence, the activity of the Agency evolved, as did its structure, responsibilities and function.

Due to systematic changes in the role of patients from the passive users of health systems and medicines to active participants and partners in treatment and decision-making processes of health care, the Agency has been changing its policy involving more and more stakeholders helping in all fields the Agency is responsible for. Today’s shape of EMA is a result of this multi-stakeholder, sustainable cooperation and common efforts of all specialists involved, including patients and patients’ representatives. At present 7 independent Committees function in EMA:

Each of the Committees has its own regulations and goals but they share some common features: representativeness (members are delegated from each EU Member State offices responsible for medicines authorisation at national level), transparency (clear and publicly available materials from every single meeting) and what is the most important for us, patients – our presence in each of the Committees.

The main patients’ structure functioning in EMA is the ‘’Patients’ and Consumers’ Working Party’’ (PCWP) established 10 years ago within CHMP. The role of PCWP is to advise, initiate discussions and propose solutions to all matters that are important with regards to medicines authorisation and their safety but also to develop educational materials that are fully recognised and clear for each patient who takes medicines. PCWP members take part also in discussions about improving the functioning of the existing legislation (optimisation of the processes e.g. of medicines authorisation, and increase in use of IT tools) whilst progressing the development of the new legislation.

During PCWP meetings ECPC’s representatives speak out on current issues, lying in the interests of cancer patients and their treatment. Currently ECPC is involved in a multi-stakeholder discussion on a new European clinical trials database and portal, Pharmacovigilance legislation and procedures, projects that are currently under development e.g. WEBRADR project directed to mobile technology use in adverse drug reaction reporting. From this year, ECPC is a member of a sub-group working on involvement of young patients (children, teenagers and young adults) in EMA’s activity. Co-chair of the sub-group is Rafal Swierzewski, ECPC Board Member.

Cancer patients and benefit-risk evaluation in EMA.

The most important task within the medicine authorisation process is benefit-risk evaluation. The evaluation indicating if the benefits from the use of a new medicine exceed the risk (e.g. severe adverse drug reactions, including death) was made so far based on scientists’ expertise reported after clinical trials. But the last decade brought a significant change of that approach. Patients’ perspective and opinion became of the same importance as scientific data. The reason which is a pillar of this new approach is simple: patients who take medicines are unique experts not only in their own disease, but also in the area of treatment. It’s also worth to notice that the last decade brought a tremendous development of new treatments and medicines; progress made in technology, biotechnology and genetics highly influenced the level on perception of the patient in the entire system. More and more advanced and personalized medicines forced the main stakeholders i.e. regulators and pharma industry to reach for the opinions of patients. Those opinions are of great importance in benefit-risk evaluation of new medicines especially in cancer treatment.

Cancer patients’ role in benefit-risk evaluation is mainly to provide advice/protocol assistance, participate in Scientific Advisory Group meetings, express opinions during oral explanations preceding decision-making as well by written consultations, and take part in Pharmacovigilance Committee activities. In the last case, the crucial role of cancer patients is associated with adverse drug reaction reporting, especially with regards to newly approved medicines. Indeed, reporting any side effects helps patients by knowledge enhancement of new medicine application and use.  However, many patients do not know that they have the right to report any adverse drug reaction by themselves – not only to a doctor, nurse or pharmacists, but directly to the manufacturer or national responsible authority e.g. via internet websites.

Another activity of cancer patients in the benefit-risk evaluation process is their participation in Scientific Advisory/ad hoc expert groups meetings. In this case, meetings are convened at the request of the CHMP or PRAC to deliver answers to specific questions connected to a specific medicine application.

Several times per year, EMA asks ECPC’s office to recommend a patient with a specific cancer for which a new medicine is undergoing authorisation or a patient who follows a treatment with a medicine that is under after-marketing authorisation procedures. Due to a large list of ECPC members, we are able to cope with this task and propose patients that are able to meet with EMA’s specialists and experts and help them in the process of benefit-risk evaluation. What is crucial here is that it’s not expected that the cancer patient delegated to EMA is an expert in pharmacology or medicine. The most important aspect is the patient’s own experience with the disease and treatment. Sometimes, it’s also not important if the patient knows the English language or not. In such cases, EMA asks the help of interpreters/translators. Moreover, the costs related to the patients’ visit at the EMA office in London are entirely covered by the Agency, including travel and accommodation if needed.

Cancer patients and clinical trials regulation. EU clinical trials database and portal development.

The new EU Clinical Trials Regulation (Regulation 536/2014) forced all EMA’s stakeholders to put as much efforts as possible in creating a new database and portal dedicated to clinical trials. Due to new transparency rules in the Regulation, it is of high importance for cancer patients to monitor its development. The current electronic system that is available for each patent via the EMA or EC websites is limited, difficult and sometimes consists of not actual data.

As of 2014, ECPC is a member of the EU Clinical Trials Database and Portal Stakeholders Group, which is an expert body created specifically for the purposes directly connected to new version of the CT database and portal. The major assumption and line of work of this Group is that the portal would fulfil demands of every stakeholder, including cancer patients. Expectations from each side are huge and the regulator (EMA) is obliged to listen and - after discussion - implement the most suitable solution that can be applied and be useful for everyone involved: from professionals, through sponsors and scientists – to patients.

Although many CT search engines are available (including the one on ECPC’s website), the majority of them are constructed as patient-friendly websites, easy to navigate and delivering the most relevant information on specific clinical trials: when, where, inclusion criteria and contact data.

Information that must be available at the European CT portal should be much broader. Besides the basic data mentioned earlier, additional information is required according to the new Regulation. Due to transparency rules, such data as clinical trial application forms, clinical trial protocols, assessment reports, inspection reports, Union Control reports, serious breaches and corrective measures, urgent safety measures and reports, clinical study reports and many other documents should be accessible through the portal. Unfortunately, fulfilling transparency obligations translates directly into a higher degree of complexity. Our role, as patient representatives is to ensure that the portal will be as clear and readable as possible, despite this expected complexity. It’s also in our cancer patient interest to monitor the EU CT database development, just to ensure to other cancer patients that data presented there are and will remain accurate.

ECPC’s involvement in EMA’s activity – more than ever.

From 2013 the direct collaboration between the European Medicines Agency and the European Cancer Patient Coalition has begun to develop in many different directions. At present, our organisation is recognised as one of the leading patient representing stakeholders of the Agency. Our contribution to EMA is not only limited to the activities mentioned above. We participate actively in discussions focusing on: addressing existing public health priorities and new priority areas, including special populations and rare cancers; tackling supply issues and potential shortages/lack of medicines availability; facilitating access to new and innovative medicines (adaptive pathways of medicines authorisation) and a closer collaboration between EMA and HTA/pricing and reimbursement bodies. Presenting cancer patients opinions at the main European regulator level of medicines – the EMA – we are able to have a direct voice that is heard and hence an influence on the most important issues in medicines policy in Europe.

For more insights regarding EMA's activities, we invite you to watch EMA introductory video for patient representatives. You can also download 2015 Annual Report of EMA interactions with patients, consumers, healthcare professionals and their organisations.

 

Rafal Swierzewski, PhD
Former ECPC Board Member

 

                                          

 

 

 

*updated in August 2016