What happens before a medicine assessment starts?

A few months before the assessment starts, EMA provides guidance to medicine developers to ensure that their applications for marketing authorisation comply with legal and regulatory requirements to avoid unnecessary delays.

Who bears the cost of medicine evaluation?

European legislation requires that pharmaceutical companies contribute to the costs of regulation of medicines. As the companies will earn revenues from the sales of medicines, it is fair that they should bear most of the financial costs of regulating them. This means that EU taxpayers do not have to support all the costs of ensuring the safety and effectiveness of medicines.

Companies pay an administrative fee upfront before EMA assessment starts. The administrative fee applicable for each procedure is defined by EU legislation.

What information needs to be submitted in a marketing authorisation application

The data submitted by medicine developers in their application for marketing authorisation must comply with EU legislation. They must include a range of information, including on the way the medicine is manufactured, its effects in laboratory studies, benefits and side effects observed in patients, and how risks will be managed, as well as the proposed information to be provided to patients and doctors.

What is the key principle underpinning a medicine’s assessment?

The balance between the benefits and risks of a medicine is the key principle guiding a medicine’s assessment. A medicine can only be authorised if its benefits outweigh the risks.

All medicines have benefits as well as risks. When assessing the evidence gathered on a medicine, EMA determines whether the benefits of the medicine outweigh its risks in the group of patients for whom the medicine is intended.

As for all marketing authorisations, it must still be demonstrated that the benefits of the medicine outweigh the risks.

In addition, since not everything is known about a medicine’s safety at the time of its initial authorisation, the way risks will be minimised, managed and monitored once the medicine is more widely used is also an integral part of the assessment and is agreed at the time of authorisation.

While the authorisation of a medicine is based on an overall positive balance between the benefits and risks at population level, each patient is different and before a medicine is used, doctors and their patient should judge whether this is the right treatment option for them based on the information available on the medicine and on the patient’s specific situation.

Who is involved in the assessment of marketing authorisation applications?

A committee of experts (the CHMP) evaluates the applications. Each of its members is supported by a team of assessors.

How does the CHMP work?

Peer review and collegial decisions are at the heart of the CHMP assessments.

For each application for a new medicine, two committee members − known as rapporteur and co-rapporteur − from different countries are appointed to lead the assessment (for generics only one rapporteur is appointed). They are appointed according to objective criteria to make best use of the available expertise in the EU.

The role of the rapporteur and co-rapporteur is to conduct the scientific evaluation of the medicine independently from each other. They each form an assessment team with assessors from their national agency and sometimes from other national agencies.

Can the CHMP request more information during the evaluation?

During the evaluation, the CHMP raises questions on the evidence provided in the application and asks the applicant to provide clarifications or additional analyses to address these questions. Responses have to be provided within an agreed timeframe.

The CHMP can raise objections or concerns which can relate to any aspect of the medicine. If unresolved, major objections preclude marketing authorisation.

Major objections can relate for example to the way the medicine was studied, the way it is manufactured, or to the effects seen in patients such as the magnitude of the benefits or the seriousness of the side effects.

What additional expertise can the CHMP rely on?

Experts with specialised scientific knowledge or clinical experience are often consulted during the evaluation to enrich the scientific discussion.

How are patients and healthcare professionals involved?

Patients and healthcare professionals understand the issues ‘from the inside’. They are therefore consulted as experts, and provide views on whether the medicine can address their needs.

What are the measures to safeguard experts’ independence?

Independence is safeguarded by a high level of transparency and the application of restrictions if certain interests are considered to potentially impact impartiality.

EMA policies on handling competing interests have been put in place to restrict the involvement of members, experts and staff with possible competing interests in the Agency’s work while maintaining EMA’s ability to access the best available expertise.

How does the CHMP make its final recommendation?

The final CHMP recommendation is reached by a formal vote. Ideally, the CHMP will come to a consensus and unanimously recommend either the approval or refusal of the marketing authorisation; such a consensus is reached in 90% of cases. However, when a final recommendation by consensus cannot be reached, the committee’s final recommendation will represent the majority view.

What information is publicly available during the evaluation of a new medicine and once a decision has been made?

EMA provides a high level of transparency about its medicine assessment by publishing meeting agendas and minutes, reports describing how the medicine was assessed and the clinical study results submitted by medicine developers in their applications.