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Putting Patients at the Heart of Future Innovation

Апрель 2015

The European Medicines Agency celebrates its 20th anniversary in 2015

The European Medicines Agency (EMA) is celebrating its 20th anniversary in 2015. To mark the occasion, it held a conference to discuss how best to deliver its role of putting patients at the heart of innovative medicines development.

The conference was the opportunity for Yann Le Cam, EURORDIS Chief Executive Officer, to speak on the importance of reinforcing the ‘health’ pillar of the EMA’s ‘Science, Medicines, Health’ mission through increased and better engagement of patients in the medicines development and assessment journey.

He commented, «An approved medicine that does not address a real patient need is only an invention, not an innovation that is meaningful to patients». Better definition of patient engagement throughout the entire life cycle of medicines development is required to increase the availability of innovative medicines that respond to patients’ needs. This can be achieved by:

  • Ensuring that patients have a role in steering industry towards those areas where they need new therapeutic options that can provide a cure or stabilise a disease, in particular areas of unmet medical needs.
  • Making clinical development patient-relevant; patients should be able to engage in dialogue with companies on all aspects related to the design of a clinical trial to allow for patient-relevant clinical trial outcomes.
  • Guaranteeing that patients have a role in directly reporting additional data that stems from their day-to-day experience of living with their condition. This allows for patient-reported clinical trial outcomes.
  • Including patients in risk-benefit assessment; when assessing the risks and benefits of a new medicine, only patients, as experts of their disease, can legitimately determine how much uncertainty, risk or harm they are willing to accept in exchange for the proposed benefit of a medicine.

In 2014, patient representatives and consumers took part in EMA activities 633 times. Tools that allow patients to provide their input to the medicines development journey in a timely manner are in constant development. These include EMA mechanisms (parallel scientific advice with health-technology-assessment (HTA) bodies, scientific advisory groups, the adaptive pathways pilot project and the benefit-risk assessment pilot project) and on a HTA level, the SEED (Shaping European Early Dialogues for health technologies) Project.

However, the challenge is to make patient input credible, so that it is accepted by all stakeholders and has a real impact. To do this, regulators, HTA bodies and patients need to continue to work together to put in place the best and most appropriate methodologies to incorporate patient input into the regulatory process, especially so that this input is independent from the influence of medicines companies.

We must also keep in mind that scientific innovation in medicines development must not be disconnected from patients’ access to medicines. The continuous engagement of patients throughout the medicines development pathway should also involve all stakeholders who have a role in determining patient access to medicines, such as regulatory agencies and HTA bodies. Mr Le Cam added, «Today, assessments and decisions made by various stakeholders are often siloed and made in isolation. A series of reasonable but disconnected assessments doesn’t make for a good final decision. A seamless pathway is needed to ensure that new, innovative medicines reach the patients that they have been developed for».

A video of the EMA conference is available online; Mr Le Cam’s participation can be viewed here. The EMA’s 20th anniversary book is available here (EURORDIS contribution on page 30).

Eva Bearryman, Junior Communications Manager, EURORDIS