Science and technology in drug development are changing at a fast pace and bring with them a lot of promises. To cope with this evolution, the regulatory environment will need to adapt if it wants to keep with its mission to best enable the delivery of novel products to patients.

Representing small and medium-sized pharmaceuticals and health technologies companies, EUCOPE welcomed the opportunity to take part in the recent consultation of the European Medicines Agency (EMA) on its Regulatory Science Strategy 2025. The strategy sets out working proposals on the key areas with which the agency intends to engage. It ensures the EMA has the tools to continue supporting the European medicines regulatory network and fulfil its ongoing mission in light of upcoming scientific challenges.

In its response to the EMA, EUCOPE identified three priorities that we believe will have the most significant benefit for European citizens.

Diversify and Integrate the Provision of Regulatory advice along the Development Continuum

It is essential to advise on how science impacts the evaluation of medicines and benefit-risk assessment in a collaborative and timely manner. EUCOPE strongly supports the Agency’s intent to invest the necessary resources to strengthen and streamline the current scientific advisory platforms so that multiple development options can be comprehended effectively.

To avoid creating an additional complimentary advice procedure, a more flexible and integrated R&D product support mechanism is needed, providing agile rolling advice that effectively addresses the key challenges and development milestones.

The new system should be leaner, more flexible and faster while maintaining the inestimable and high-level communication, interaction, and alignment between the relevant stakeholders. The EMA has always been spearheading the systematic involvement of patients and stakeholders such as HTA bodies, payers, or healthcare professionals. We believe this is essential to achieve greater access to innovative therapies.

Reinforce Patients’ Relevance in Evidence Generation

Thanks to the recent developments in technologies and a stronger patient movement, patients are today very aware of their condition and treatment options. It is particularly true for rare diseases. Patients, parents and/or caregivers know more about their disease, symptoms and daily living than any other stakeholder.

In addition, patient’s data preferences, reported outcomes (PRO) and other types of patient input to drug development and evaluation are being increasingly used in several regulatory environments (as illustrated by the EUPATI scheme). We need a stronger collaboration on how this evolution can advance regulatory science and ultimately contribute to better patient-centred drug development, access and care.

Patients, parents and/or caregivers know more about their disease, symptoms and daily living than any other stakeholder.

The use of PROs in clinical trials enable to make the patients’ input heard more effectively and broadly during the development and evaluation process. Resulting in helping sponsors, regulators and other stakeholders like HTA bodies and payers to place efficacy and safety data in a patient-centred context. A coordinated approach to PROs across therapeutic areas and a proactive update by the EMA of specific clinical guidelines on these would be welcomed.

Integrated Pathway including Medical Technologies and Drug-Device Combination Products (DDCP)

Over the past years, the EMA has received an increase of scientific advice requests and marketing authorization applications for drug-device combination (DDC) products. The DDC market is indeed growing and will continue to do so with advances in medical technology paving the way towards safer and more efficient patient care.

Developments in science and technology for medical devices may advance more rapidly than for medicinal products alone.

Therefore, an integrated evaluation pathway is essential to ensure timely access to innovative treatments for often unmet medical needs. Concerns around the timely implementation of the Medical Device Regulation 2017/745 have clearly shown the relevance of effective collaboration between National Competent Authorities (NCA) and Notified Bodies and the necessity of adequate capacities being allocated.

With the new definition of In Vitro Diagnostics (IVDs) under the In Vitro Diagnostics Devices Regulation 2017/746, the move to a risk-based classification and the introduction of new rules for companion diagnostic devices, the IVD Regulation has changed the framework entirely.

The Competent Authorities (EMA and NCA), the Notified Bodies and the developers are faced with challenges, which can only be handled in collaboration. Companion diagnostics are generally codeveloped with an innovative medicinal product and regarded as novel and complex technologies in the area of precision medicines. This field of innovative research and their regulatory implications should be a priority for the EMA regarding the mission to foster scientific excellence in the evaluation and supervision of medicines, for the benefit of public health in the EU.

Finally, an integrated pathway could be particularly important in the context of digital health and EUCOPE encourages the EMA to ensure strong collaboration with the medical device community and Notified Bodies to ensure aspects such as qualification of new digital methodologies for drug development are carried out with the best available expertise and in a holistic manner.

The EMA has always been a role model in providing a platform for a collaborative regulatory environment, supporting the European researchers, small and medium-sized companies and the technology sector in developing world-leading medicinal products and health technologies, for the benefit of all patients.